Retinopathy of Prematurity (ROP)
Conditions
Brief summary
The main purpose of this study is to evaluate the long-term efficacy and safety outcomes following short-term exposure to rhIGF-1/rhIGFBP-3 versus standard neonatal care in Study ROPP-2008-01 (NCT01096784).
Detailed description
Long-term Safety and Efficacy Outcome Study Comparing Children Previously Enrolled in Study ROPP-2008-01 for the Prevention of Retinopathy of Prematurity (ROP)
Interventions
Participants who received rhIGF-1/rhIGFBP-3 in study ROPP-2008-01 (NCT01096784) will be enrolled to this study. No investigational product will be administered in this study.
Sponsors
Shire
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
40 Weeks to 108 Weeks
Healthy volunteers
No
Inclusion criteria
* Participant was randomized in Study ROPP-2008-01 Section D (NCT01096784). * Participants parent or legally authorized representative(s) must provide written informed consent prior to performing any study-related activities. Study-related activities are any procedures that would not have been performed during normal management of the participant.
Exclusion criteria
* Any other condition or therapy that, in the Investigator's opinion, may pose a risk to the Participant or interfere with the participants ability to be compliant with this protocol or interfere with the interpretation of results. * The participant or participants parent or legally authorized representative(s) is unable to comply with the protocol as determined by the Investigator.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 12 Months CA | At 12 Months CA | Standard age-appropriate methods were used to assess visual acuity (how well a participant sees at different distances) with teller acuity cards, LEA symbols test and Charts. It was categorized as following: normal (measurable acuity \>= 20/40 or \>= 15 cycles/degree); below normal (20/200 \<= measurable acuity \< 20/40 or 3 cycles/degree \<= measurable acuity \< 15 cycles/degree); poor (measurable acuity \<= 20/200 or \<= 3 cycles/degree). Corrected Age (weeks) was defined as Chronological Age (weeks) - 40 + Gestational Age (weeks) in this study. |
| Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 20 Months CA | At 20 Months CA | Standard age-appropriate methods were used to assess visual acuity (how well a participant sees at different distances) with teller acuity cards, LEA symbols test and Charts. It was categorized as following: normal (measurable acuity \>= 20/40 or \>= 15 cycles/degree); below normal (20/200 \<= measurable acuity \< 20/40 or 3 cycles/degree \<= measurable acuity \< 15 cycles/degree); poor (measurable acuity \<= 20/200 or \<= 3 cycles/degree). Corrected Age (weeks) was defined as Chronological Age (weeks) - 40 + Gestational Age (weeks) in this study. |
| Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 24 Months CA | At 24 Months CA | Standard age-appropriate methods were used to assess visual acuity (how well a participant sees at different distances) with teller acuity cards, LEA symbols test and Charts. It was categorized as following: normal (measurable acuity \>= 20/40 or \>= 15 cycles/degree); below normal (20/200 \<= measurable acuity \< 20/40 or 3 cycles/degree \<= measurable acuity \< 15 cycles/degree); poor (measurable acuity \<= 20/200 or \<= 3 cycles/degree). Corrected Age (weeks) was defined as Chronological Age (weeks) - 40 + Gestational Age (weeks) in this study. |
| Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 4.75 Years CA | At 4.75 Years CA | Standard age-appropriate methods were used to assess visual acuity (how well a participant sees at different distances) with teller acuity cards, LEA symbols test and Charts. It was categorized as following: normal (measurable acuity \>= 20/40 or \>= 15 cycles/degree); below normal (20/200 \<= measurable acuity \< 20/40 or 3 cycles/degree \<= measurable acuity \< 15 cycles/degree); poor (measurable acuity \<= 20/200 or \<= 3 cycles/degree). Corrected Age (weeks) was defined as Chronological Age (weeks) - 40 + Gestational Age (weeks) in this study. |
| Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 5 Years CA | At 5 Years CA | Standard age-appropriate methods were used to assess visual acuity (how well a participant sees at different distances) with teller acuity cards, LEA symbols test and Charts. It was categorized as following: normal (measurable acuity \>= 20/40 or \>= 15 cycles/degree); below normal (20/200 \<= measurable acuity \< 20/40 or 3 cycles/degree \<= measurable acuity \< 15 cycles/degree); poor (measurable acuity \<= 20/200 or \<= 3 cycles/degree). Corrected Age (weeks) was defined as Chronological Age (weeks) - 40 + Gestational Age (weeks) in this study. |
| Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 6 Months Corrected Age (CA) | At 6 Months CA | Standard age-appropriate methods were used to assess visual acuity (how well a participant sees at different distances) with teller acuity cards, LEA symbols test and Charts. It was categorized as following: normal (measurable acuity \>= 20/40 or \>= 15 cycles/degree); below normal (20/200 \<= measurable acuity \< 20/40 or 3 cycles/degree \<= measurable acuity \< 15 cycles/degree); poor (measurable acuity \<= 20/200 or \<= 3 cycles/degree). Corrected Age (weeks) was defined as Chronological Age (weeks) - 40 + Gestational Age (weeks) in this study. |
| Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 12 Months CA | At 12 Months CA | Ocular alignment was assessed in primary gaze by comparing the position of the corneal light reflection in left eye and right eye (corneal light reflection assessment). Presence or absence of strabismus was recorded in primary gaze and in as many of the 9 positions of gaze as feasible with the cover test assessment of refixation movement. Extraocular muscle over action or deficiency was recorded. Ocular motility referred to eye movements governed by the 6 extraocular muscles in each eye. It was assessed by examiner observation of the participants ability to abduct, adduct, supra, and inferoduct each eye. Ocular alignment and motility included the presence or absence of strabismus (classified as Esotropia \[inward turn of the eye\], Exotropia \[outward turn of the eye\], Hypertropia \[upward turn of the eye\], Hypotropia \[downward turn of the eye\]) was recorded. Number of participants with combined data for ocular alignment and oculomotor examination (motility) at 12 months CA were reported. |
| Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 24 Months CA | At 24 Months CA | Ocular alignment was assessed in primary gaze by comparing the position of the corneal light reflection in left eye and right eye (corneal light reflection assessment). Presence or absence of strabismus was recorded in primary gaze and in as many of the 9 positions of gaze as feasible with the cover test assessment of refixation movement. Extraocular muscle over action or deficiency was recorded. Ocular motility referred to eye movements governed by the 6 extraocular muscles in each eye. It was assessed by examiner observation of the participants ability to abduct, adduct, supra, and inferoduct each eye. Ocular alignment and motility included the presence or absence of strabismus (classified as Esotropia, Exotropia, Hypertropia, Hypotropia) was recorded. Number of participants with combined data for ocular alignment and oculomotor examination (motility) assessed by corneal light reflex and by the cover test at 24 months CA were reported. |
| Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 5-Years CA | At 5 Years CA | Ocular alignment was assessed in primary gaze by comparing the position of the corneal light reflection in left eye and right eye (corneal light reflection assessment). Presence or absence of strabismus was recorded in primary gaze and in as many of the 9 positions of gaze as feasible with the cover test assessment of refixation movement. Extraocular muscle over action or deficiency was recorded. Ocular motility referred to eye movements governed by the 6 extraocular muscles in each eye. It was assessed by examiner observation of the participants ability to abduct, adduct, supra, and inferoduct each eye. Ocular alignment and motility included the presence or absence of strabismus (classified as Esotropia, Exotropia, Hypertropia, Hypotropia) was recorded. Number of participants with combined data for ocular alignment and oculomotor examination (motility) assessed by corneal light reflex and by the cover test at 5-Years CA were reported. |
| Number of Participants With Nystagmus at 12 Months CA | At 12 Months CA | Nystagmus was observed during the ocular alignment assessments. Presence and absence of nystagmus was reported at 12 Months CA. |
| Number of Participants With Nystagmus at 24 Months CA | At 24 Months CA | Nystagmus was observed during the ocular alignment assessments. Presence and absence of nystagmus was reported at 24 Months CA. |
| Number of Participants With Nystagmus at 5 Years CA | At 5 Years CA | Nystagmus was observed during the ocular alignment assessments. Presence and absence of nystagmus was reported at 5 Years CA. |
| Refraction With Cycloplegia as Assessed by Retinoscopy at 6 Months CA | At 6 Months CA | Refraction was a measure of the lens power required for a focused image on the retina. Refraction with cycloplegia was measured and recorded in diopters for each eye individually (left eye and right eye). Refraction with cycloplegia performed as part of the corrective lens determination procedure included measurements of sphere, cylinder, axis, and prism for each individual eye (left eye and right eye). For sphere, the negative values are summarized as 'nearsighted', and the positive values (including sphere of 0.00) are summarized as 'farsighted'. |
| Refraction With Cycloplegia as Assessed by Retinoscopy at 12 Month CA | At 12 Months CA | Refraction was a measure of the lens power required for a focused image on the retina. Refraction with cycloplegia was measured and recorded in diopters for each eye individually (left eye and right eye). Refraction with cycloplegia performed as part of the corrective lens determination procedure included measurements of sphere, cylinder, axis, and prism for each individual eye (left eye and right eye). For sphere, the negative values are summarized as 'nearsighted', and the positive values (including sphere of 0.00) are summarized as 'farsighted'. |
| Refraction With Cycloplegia as Assessed by Retinoscopy at 20 Month CA | At 20 Months CA | Refraction was a measure of the lens power required for a focused image on the retina. Refraction with cycloplegia was measured and recorded in diopters for each eye individually (left eye and right eye). Refraction with cycloplegia performed as part of the corrective lens determination procedure included measurements of sphere, cylinder, axis, and prism for each individual eye (left eye and right eye). For sphere, the negative values are summarized as 'nearsighted', and the positive values (including sphere of 0.00) are summarized as 'farsighted'. |
| Refraction With Cycloplegia as Assessed by Retinoscopy at 4.75 Years CA | At 4.75 Years CA | Refraction was a measure of the lens power required for a focused image on the retina. Refraction with cycloplegia was measured and recorded in diopters for each eye individually (left eye and right eye). Refraction with cycloplegia performed as part of the corrective lens determination procedure included measurements of sphere, cylinder, axis, and prism for each individual eye (left eye and right eye). For sphere, the negative values are summarized as 'nearsighted', and the positive values (including sphere of 0.00) are summarized as 'farsighted'. |
| Number of Participants With Stereoacuity as Assessed With the Lang Stereotest At 5 Years CA | At 5 Years CA | Stereoacuity, a measure of depth perception, was assessed using the Lang Stereotest. Number of participants with presence and absence of stereopsis (the ability to perceive depth and 3-dimensional structure) was reported. |
| Number of Participants With Adverse Events (AEs) | From start of study up to end of study (up to 6.5 years) | An AE is any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Child Behaviour as Assessed by Child Behaviour Checklist (CBCL) Based on T-Score Clinical Categories | At 24 Months CA and 5 Years CA | The CBCL (1 ½ to 5) was parent-reported outcome measure used to assess behavioral, emotional, and social functioning of toddlers and preschool children aged 18-60 months. It is composed of 99 items rated on Likert scale and includes 7 syndrome scales under 2 domains (Internalizing and Externalizing Problems): Internalizing includes syndromes of Emotionally Reactive, Anxious/Depressed, Somatic Complaints, and Withdrawn. Externalizing includes syndromes of Aggressive Behavior and Attention Problems. The 2 domains of all 7 scales were combined to form total score range of 23 to 100. For each question, raw scores of all subscales are converted to standardized T-scores. Higher T score indicates more behavior problems. For Internalization, Externalization and Total Behavior Problem, T-scores of less than 60 are considered non-clinical, 60-63 are borderline, and 64 or more are considered clinical. |
| Child Behavior as Assessed by Attention-Deficit/Hyperactivity Disorder Rating Scale (ADHD-RS) Based on Total Score | At 5 Years CA | The ADHD-RS measures the behaviors of children with Attention-Deficit/Hyperactivity Disorder (ADHD). It consisted of 18 items designed to reflect current symptomatology of ADHD based on DSM-IV criteria. Each item was scored on a 4-point scale ranging from 0 (reflecting no symptoms) to 3 (reflecting severe symptoms) with total scores ranging from 0 to 54. Higher scores represent greater severity of ADHD symptoms. |
| Child Behavior as Assessed by Social Communication Questionnaire (SCQ) | At 5 Years CA | The SCQ is a brief instrument evaluate communication skills and social functioning in children used for screening for autism or autism spectrum disorders (ASD). The SCQ has 40 dichotomous (yes, no) items, and each item scored 1 point for abnormal behavior and 0 point for absence of abnormal behavior/normal behavior. It yields total score ranging from 0 to 39, higher scores represent more social communication impairment. The first item, Is she/he now able to talk using short phrases or sentences?, is not scored, but determines if six items relating to abnormal language are assigned. Only verbal children (children with yes response to first question) are assigned the six items relating to abnormal language and can score a total of 0 to 39 points; non-verbal children (children with no response to first question) are not assigned six items in relation to abnormal language and score a total of 0 to 33 points where higher scores represent more social communication impairment. |
| Number of Participants With Pulmonary Morbidity | At 6 Months CA, 12 Months CA and 24 Months CA | Pulmonary morbidity was assessed with questions related to family history and smoking status as well as diagnosis of select pulmonary symptoms, conditions and related hospitalizations. Anyone living in the same home with participant smoke, Participants had asthma, wheezing, bronchopulmonary dysplasia (BPD) exacerbation or flare-up, Participants had bronchiolitis, bronchitis, or pneumonia diagnosed, Participants had to use oxygen at home, Participants had to visit emergency room or urgent care for respiratory problem, and Participants had to stay in a hospital overnight for respiratory problem were reported. |
| Number of Participants With Abnormal Physical Examination | From start of study drug administration up to end of study (up to 6.5 years) | Physical examination includes general appearance; head and neck, eyes, ears, nose and throat; chest and lungs; endocrine, cardiovascular system, abdomen, genitourinary, skin and musculoskeletal system. |
| Change From Baseline in Height Z-score | Baseline, 6 Months CA, 12 Month CA, 24 Months CA and 5 Years CA | Height was measured using Z-score. Z-score was calculated based on the participants chronological age and utilizing World Health Organization child growth standards by adjusting age and sex- matched means and standard deviations (norm). Z-score is a standard score that gives an idea of how far from the mean a data point is. If a Z-score is 0, it indicates that the data point's score is identical to the mean score. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean. Baseline was defined as the first assessment in the antecedent study (ROPP-2008-01 \[NCT01096784\]). Change from baseline in Height Z-score at 6-month, 12-month, 24-month and 5-year CA were reported. |
| Change From Baseline in Head Circumference Z-score | Baseline, 6 Months CA, 12 Months CA and 24 Months CA | Head circumference measured for all participants using a lasso type, non-stretchable measuring tape such as the Lasso-o tape. Z-score calculated based on participants chronological age and utilizing World Health Organization child growth standards by adjusting age, sex matched means and standard deviations (norm).Z-score is standard score that gives idea of how far from mean a data point is. If a Z-score is 0, it indicates that data point's score is identical to mean score. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean. Baseline was defined as the first assessment in the antecedent study (ROPP-2008-01 \[NCT01096784\]). Change from baseline in Head Circumference Z-score at 6, 12 and 24-months reported. |
| Number of Participants With Survival Status | From start of study drug administration up to end of study (up to 6.5 years) | Survival status was assessed by number participants who died and were censored during the study. |
| Change From Baseline (3 Months CA) in Pediatric Quality of Life Inventory (PedsQL™) Scale: Infant Scale | Baseline (3 Months CA) up to 6 Months CA, 12 Months CA and 24 Months CA | The PedsQL was a generic health related quality of life instrument designed specifically for a pediatric population. PedsQL infant scale encompasses 5 dimensions of functioning (Physical Functioning, Physical Symptoms, Emotional Functioning, Social Functioning, and Cognitive Functioning). The infant for ages 1-12 months (36 Items); for ages 13-24 months (45 Items) rated on a 5-point Likert scale (0 to 4). Overall total scores were calculated as average of all dimensional sub-score items of Infant scale, ranging from 0 to 100 where 0=100 (Never), 1=75 (almost never), 2=50 (sometimes), 3=25 (often), and 4=0 (almost always). Higher scores indicate improved quality of life. A negative value indicates decreased quality of life. The 3 months CA was considered as baseline for this outcome measure. |
| Change From Baseline (24 Months CA) in Pediatric Quality of Life Inventory (PedsQL™) Scale: Generic Core Scale (GCS) Total Score | Baseline (24 Months CA), up to 3 Years CA, 4 Years CA and 5 Years CA | The PedsQL was a generic health related quality of life instrument designed specifically for a pediatric population. The GCS encompasses 4 dimensions of functioning (physical, emotional, social, school). The GCS that apply to toddler for 2-4 years of age (21 Items) rated on a 5-point Likert scale (0 to 4); and Young Child for 5-7 years of age (23 Items) rated on a 3-point Likert scale (0, 2, 4). Overall total scores were calculated as average of all dimensional sub-score items of GCS (Toddler and young children's) ranging from 0 to 100 where 0=100 (Never), 1=75 (almost never), 2=50 (sometimes), 3=25 (often), and 4=0 (almost always). Higher scores indicate improved quality of life. A negative value indicates decreased quality of life. The 24 month CA was considered as baseline for this outcome measure. |
| Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 24 Months CA, 3 Years CA, 4 Years CA and 5 Years CA | HSCS-PS was a validated instrument used via parent proxy within age 2.5 - 5 years. The instrument is composed of 12 domains (Vision, Hearing, Speech, Mobility, Dexterity, Self-care, Emotion, Learn/Remember, Think/Problem Solve, Pain, General Health, Behavior) with up to 6 levels (0-5). The 12 dimensions grouped into 4 categories: neurosensory (vision and hearing), motor (mobility, dexterity, and self-care), learning/remembering (speech, learn/remember, think/problem solve), and quality of life (emotion, pain, general health, behavior). For each category, data were recorded into following levels: no problem (scoring 0 on any attribute); a mild problem (scoring 1 on a scale of 0 to 3, or 1 to 2 on a scale of 0 to 5 for any attribute);moderate/severe problem (scoring \> 1 on a scale of 0 to 3, or \>2 on a scale of 0 to 5 for any attribute). Higher numbers indicating better child's health status. |
| Health Status Measured by the Health Utilities Index (HUI) Mark 2 and 3 | At 5 Years CA | The HUI is a family of generic health profiles and preference-based systems used for measuring health status, reporting HRQoL, and producing utility scores. The HUI2 score includes 6 attributes: Sensation, Mobility, Cognition, Self-Care, Emotion, and Pain with up to 5 levels of severity (higher numbers indicate worse level of severity). The HUI3 score includes 8 attributes: Vision, Hearing, Speech, Cognition, Ambulation, Dexterity, Emotion, and Pain with up to 6 levels to indicate the severity (with higher numbers indicating worse levels). HUI2/3 single-attribute scores of morbidities are defined on a scale such the worst level has a score of 0.00 and the best level has a score of 1.00. HUI mark 2 and 3 Overall utility score was reported. |
| Health Care Resource Use (HCRU) | At 3 Months CA, 6 Months CA, 12 Months CA, 24 Months CA, 3 Years CA, 4 Years CA and 5 Years CA | The total number of private office/hospital outpatient visits of health care resource at 3 Months CA, 6 Months CA, 12 Months CA, 24 Months CA, 3 Years CA, 4 Years CA and 5 Years CA were reported. |
| Health Care Resource Utilization (HCRU): Number of Visits to Emergency Department | At 3 Months CA, 6 Months CA, 12 Months CA , 24 Months CA, 3 Years CA, 4 Years CA and 5 Years CA | Number of visits to emergency department at 3 Months CA, 6 Months CA, 12 Months CA, 24 Months CA, 3 Years CA, 4 Years CA and 5 Years CA were reported. |
| Health Care Resource Utilization (HCRU): Duration of Hospitalization | At 3 Months CA, 6 Months CA, 12 Months CA , 24 Months CA, 3 Years CA, 4 Years CA and 5 Years CA | The participant's duration of hospitalizations at 3 Months CA, 6 Months CA, 12 Months CA, 24 Months CA, 3 Years CA, 4 Years CA and 5 Years CA were reported. |
| Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 3 Months CA, 6 Months CA, 12 Months CA, 24 Months CA, 3 Years CA, 4 Years CA and 5 Years CA | Number of participants who required prescription eyeglasses and educational support at 3 Months CA, 6 Months CA, 12 Months CA, 24 Months CA, 3 Years CA, 4 Years CA and 5 Years CA were reported. |
| Change From Baseline (12 Months CA) in Cognitive Development as Assessed by Bayley Scales of Infant and Toddler Development (BSID-III) Composite Scores at 24 Months CA | Baseline (12 Months CA), 24 Months CA | BSID-III was to assess cognitive, motor, and language skills, and applicable to children aged 1-42 months. There are 5 subscales, cognitive subscale (Ranges: 55-145) stands alone while 2 language subscales (expressive and receptive) combine to make a total language score (Ranges: 47-153) and 2 motor subtests (fine and gross motor) form combined motor scale (Ranges: 46-154). Higher scores represent greater cognitive, language and motor abilities. Positive value indicates improvement and negative value indicate worsening in cognitive development. The 12 Months CA considered as baseline for this outcome measure. Composite scores derived from various sums of subtest scaled scores and scaled to a metric with a mean of 100 and a standard deviation of 15 and range from 40 to 160. Higher values denote stronger skills and abilities in the domain, indicating better outcomes. |
| Cognitive Development as Assessed by Wechsler Preschool and Primary Scale of Intelligence (WPPSI-IV) Full Scale at 5 Years CA | At 5 Years CA | The WPPSI-IV is a measure of general cognitive development in children that has components of both verbal and non-verbal tasks. It is applicable to preschoolers and young children aged 2 years +6 months to 7 years +7 months and is a direct assessment of a child's cognitive skills. The test framework of the WPPSI-IV is organized into five Primary Index sub scales: Verbal Comprehension, Visual Spatial, Fluid Reasoning, Working Memory, and Processing Speed. The Full Scale includes all of the sub scales at the Primary Index scale level, as well as any additional supplemental subtests that may be used to derive the Full Scale IQ. Composite scores are derived from the sum of sub scaled scores with a mean of 100 and a standard deviation of 15. Composite score for Full Scale IQ and subscales (Verbal Comprehension, Visual Spatial, Fluid Reasoning, Working Memory, and Processing Speed) ranges from 40 (extremely low) to 160 (very superior). Data for Full scale IQ was reported here. |
| Change From Baseline in Body Weight Z-score | Baseline, 6 Months CA, 12 Months CA, 24 Months CA and 5 Years CA | Body weight collected using calibrated scales (type of scale was dependent upon participant's age). Measure recorded to the nearest 0.1 kilogram (kg). Z-score based on participants chronological age and utilizing World Health Organization child growth standards by adjusting age and sex- matched means and standard deviations (norm). Z-score is standard score that gives idea of how far from the mean a data point is. If Z-score is 0, it indicates that the data point's score is identical to the mean score. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean. Baseline is first assessment in the antecedent study (ROPP-2008-01 \[NCT01096784\]). Change from baseline in body weight Z-score at 6-month, 12-month, 24-month and 5-year CA were reported. |
| Number of Participants Diagnosed With Neurological Examination for Cerebral Palsy (CP) by Neurological Examination at 24 Months CA | At 24 Months CA | A comprehensive neurological examination for the diagnosis of CP was conducted. The Amiel-Tison neurological examination framework was utilized for this assessment and it was conducted by trained medical professionals. Number of Participants diagnosed with CP by neurological examination at 24 Months CA were reported. |
| Number of Participants With Normal and Abnormal Hearing Screening Status | At 6 Months CA and 5 Years CA | Number of participants with any hearing status (normal and abnormal) were reported. |
| Change From Baseline (6 Months CA) in Child Behavior as Assessed by Vineland Adaptive Behaviour Scales (VABS-II) | Baseline (6 Months CA), 12 Months CA, 24 Months CA, and 5 Year CA | The VABS-II was used to measure the personal and social skills of participants serially over time. This test measures the following 4 key domains: Communication, Daily Living Skills, Socialization, and Motor skills. The domains have the same range and directionality as the adaptive behavior composite score (20-160). Higher scores indicate higher level of cognitive ability. A positive change value indicates improvement and negative value indicates worsening in adaptive functioning. The 6 months CA was considered as baseline for this outcome measure. Change from baseline (6 Months CA) in child behavior as assessed by VABS-II adaptive behavior composite was reported here. |
Countries
Italy, Netherlands, Poland, Sweden, United Kingdom, United States
Participant flow
Recruitment details
This study was conducted at 16 sites from 26 March 2015 (first participant first visit) to 28 September 2021 (last participant last visit).
Pre-assignment details
A total of 76 participants who previously treated with rhIGF 1/rhIGFBP-3 or Standard Neonatal Care in ROPP-2008-01 (NCT01096784) study were enrolled in this long-term safety and efficacy study.
Participants by arm
| Arm | Count |
|---|---|
| Antecedent Standard of Care Participants who were treated with standard neonatal care in study ROPP-2008-01 (NCT01096784). | 36 |
| Antecedent rhIGF-1/rhIGFBP-3 Participants who were treated with rhIGF-1/rhIGFBP-3 in study ROPP-2008-01 (NCT01096784). | 40 |
| Total | 76 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Death | 1 | 0 |
| Overall Study | Lost to Follow-up | 4 | 4 |
| Overall Study | Other (Covid 19 Pandemic) | 0 | 1 |
| Overall Study | Other (Site Terminated by Sponsor) | 1 | 0 |
| Overall Study | Other (unspecified) | 1 | 0 |
| Overall Study | Withdrawal by Subject | 9 | 10 |
Baseline characteristics
| Characteristic | Antecedent rhIGF-1/rhIGFBP-3 | Total | Antecedent Standard of Care |
|---|---|---|---|
| Age, Continuous | 6.73 Weeks STANDARD_DEVIATION 6.224 | 6.93 Weeks STANDARD_DEVIATION 5.911 | 7.15 Weeks STANDARD_DEVIATION 5.622 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 13 Participants | 19 Participants | 6 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 27 Participants | 55 Participants | 28 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 2 Participants | 2 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 4 Participants | 4 Participants |
| Race (NIH/OMB) Black or African American | 1 Participants | 6 Participants | 5 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 5 Participants | 6 Participants | 1 Participants |
| Race (NIH/OMB) White | 34 Participants | 60 Participants | 26 Participants |
| Sex: Female, Male Female | 14 Participants | 27 Participants | 13 Participants |
| Sex: Female, Male Male | 26 Participants | 49 Participants | 23 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 1 / 36 | 0 / 40 |
| other Total, other adverse events | 5 / 36 | 2 / 40 |
| serious Total, serious adverse events | 3 / 36 | 2 / 40 |
Outcome results
Primary
Number of Participants With Adverse Events (AEs)
An AE is any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment.
Time frame: From start of study up to end of study (up to 6.5 years)
Population: Safety Set included all participants in the Enrolled Set who have safety follow-up data in this long-term outcome study.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Antecedent Standard of Care | Number of Participants With Adverse Events (AEs) | 11 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Adverse Events (AEs) | 9 Participants |
Primary
Number of Participants With Nystagmus at 12 Months CA
Nystagmus was observed during the ocular alignment assessments. Presence and absence of nystagmus was reported at 12 Months CA.
Time frame: At 12 Months CA
Population: Enrolled set included of all participants for whom written informed consent was obtained for this long-term outcome study. Here, Overall Number of participants analyzed signifies those participants who were evaluable for this outcome measure.
| Arm | Measure | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Antecedent Standard of Care | Number of Participants With Nystagmus at 12 Months CA | Participants with presence of nystagmus | 1 Participants |
| Antecedent Standard of Care | Number of Participants With Nystagmus at 12 Months CA | Participants with absence of nystagmus | 24 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Nystagmus at 12 Months CA | Participants with presence of nystagmus | 2 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Nystagmus at 12 Months CA | Participants with absence of nystagmus | 34 Participants |
Primary
Number of Participants With Nystagmus at 24 Months CA
Nystagmus was observed during the ocular alignment assessments. Presence and absence of nystagmus was reported at 24 Months CA.
Time frame: At 24 Months CA
Population: Enrolled set included of all participants for whom written informed consent was obtained for this long-term outcome study. Here, Overall Number of participants analyzed signifies those participants who were evaluable for this outcome measure.
| Arm | Measure | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Antecedent Standard of Care | Number of Participants With Nystagmus at 24 Months CA | Participants with presence of nystagmus | 0 Participants |
| Antecedent Standard of Care | Number of Participants With Nystagmus at 24 Months CA | Participants with absence of nystagmus | 23 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Nystagmus at 24 Months CA | Participants with presence of nystagmus | 2 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Nystagmus at 24 Months CA | Participants with absence of nystagmus | 32 Participants |
Primary
Number of Participants With Nystagmus at 5 Years CA
Nystagmus was observed during the ocular alignment assessments. Presence and absence of nystagmus was reported at 5 Years CA.
Time frame: At 5 Years CA
Population: Enrolled set included of all participants for whom written informed consent was obtained for this long-term outcome study. Here, Overall Number of participants analyzed signifies those participants who were evaluable for this outcome measure.
| Arm | Measure | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Antecedent Standard of Care | Number of Participants With Nystagmus at 5 Years CA | Participants with presence of nystagmus | 0 Participants |
| Antecedent Standard of Care | Number of Participants With Nystagmus at 5 Years CA | Participants with absence of nystagmus | 14 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Nystagmus at 5 Years CA | Participants with presence of nystagmus | 2 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Nystagmus at 5 Years CA | Participants with absence of nystagmus | 15 Participants |
Primary
Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 12 Months CA
Ocular alignment was assessed in primary gaze by comparing the position of the corneal light reflection in left eye and right eye (corneal light reflection assessment). Presence or absence of strabismus was recorded in primary gaze and in as many of the 9 positions of gaze as feasible with the cover test assessment of refixation movement. Extraocular muscle over action or deficiency was recorded. Ocular motility referred to eye movements governed by the 6 extraocular muscles in each eye. It was assessed by examiner observation of the participants ability to abduct, adduct, supra, and inferoduct each eye. Ocular alignment and motility included the presence or absence of strabismus (classified as Esotropia \[inward turn of the eye\], Exotropia \[outward turn of the eye\], Hypertropia \[upward turn of the eye\], Hypotropia \[downward turn of the eye\]) was recorded. Number of participants with combined data for ocular alignment and oculomotor examination (motility) at 12 months CA were reported.
Time frame: At 12 Months CA
Population: Enrolled set included all participants for whom written informed consent was obtained for this long-term outcome study. Here, Overall Number of participants analyzed signifies those participants who were evaluable for this outcome measure.
| Arm | Measure | Group | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|---|
| Antecedent Standard of Care | Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 12 Months CA | Participants with Esotropia | Presence | 1 Participants |
| Antecedent Standard of Care | Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 12 Months CA | Participants with Esotropia | Absence | 24 Participants |
| Antecedent Standard of Care | Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 12 Months CA | Participants with Exotropia | Presence | 0 Participants |
| Antecedent Standard of Care | Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 12 Months CA | Participants with Exotropia | Absence | 25 Participants |
| Antecedent Standard of Care | Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 12 Months CA | Participants with Hypertropia | Presence | 0 Participants |
| Antecedent Standard of Care | Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 12 Months CA | Participants with Hypertropia | Absence | 25 Participants |
| Antecedent Standard of Care | Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 12 Months CA | Participants with Hypotropia | Presence | 0 Participants |
| Antecedent Standard of Care | Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 12 Months CA | Participants with Hypotropia | Absence | 25 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 12 Months CA | Participants with Hypotropia | Absence | 36 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 12 Months CA | Participants with Esotropia | Presence | 4 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 12 Months CA | Participants with Hypertropia | Presence | 0 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 12 Months CA | Participants with Esotropia | Absence | 32 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 12 Months CA | Participants with Hypotropia | Presence | 0 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 12 Months CA | Participants with Exotropia | Presence | 0 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 12 Months CA | Participants with Hypertropia | Absence | 36 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 12 Months CA | Participants with Exotropia | Absence | 36 Participants |
Primary
Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 24 Months CA
Ocular alignment was assessed in primary gaze by comparing the position of the corneal light reflection in left eye and right eye (corneal light reflection assessment). Presence or absence of strabismus was recorded in primary gaze and in as many of the 9 positions of gaze as feasible with the cover test assessment of refixation movement. Extraocular muscle over action or deficiency was recorded. Ocular motility referred to eye movements governed by the 6 extraocular muscles in each eye. It was assessed by examiner observation of the participants ability to abduct, adduct, supra, and inferoduct each eye. Ocular alignment and motility included the presence or absence of strabismus (classified as Esotropia, Exotropia, Hypertropia, Hypotropia) was recorded. Number of participants with combined data for ocular alignment and oculomotor examination (motility) assessed by corneal light reflex and by the cover test at 24 months CA were reported.
Time frame: At 24 Months CA
Population: Enrolled set included all participants for whom written informed consent was obtained for this long-term outcome study. Here, Overall Number of participants analyzed signifies those participants who were evaluable for this outcome measure.
| Arm | Measure | Group | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|---|
| Antecedent Standard of Care | Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 24 Months CA | Participants with Esotropia | Presence | 1 Participants |
| Antecedent Standard of Care | Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 24 Months CA | Participants with Esotropia | Absence | 22 Participants |
| Antecedent Standard of Care | Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 24 Months CA | Participants with Exotropia | Presence | 0 Participants |
| Antecedent Standard of Care | Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 24 Months CA | Participants with Exotropia | Absence | 23 Participants |
| Antecedent Standard of Care | Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 24 Months CA | Participants with Hypertropia | Presence | 0 Participants |
| Antecedent Standard of Care | Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 24 Months CA | Participants with Hypertropia | Absence | 23 Participants |
| Antecedent Standard of Care | Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 24 Months CA | Participants with Hypotropia | Presence | 0 Participants |
| Antecedent Standard of Care | Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 24 Months CA | Participants with Hypotropia | Absence | 23 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 24 Months CA | Participants with Hypotropia | Absence | 34 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 24 Months CA | Participants with Esotropia | Presence | 5 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 24 Months CA | Participants with Hypertropia | Presence | 0 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 24 Months CA | Participants with Esotropia | Absence | 29 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 24 Months CA | Participants with Hypotropia | Presence | 0 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 24 Months CA | Participants with Exotropia | Presence | 1 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 24 Months CA | Participants with Hypertropia | Absence | 34 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 24 Months CA | Participants with Exotropia | Absence | 33 Participants |
Primary
Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 5-Years CA
Ocular alignment was assessed in primary gaze by comparing the position of the corneal light reflection in left eye and right eye (corneal light reflection assessment). Presence or absence of strabismus was recorded in primary gaze and in as many of the 9 positions of gaze as feasible with the cover test assessment of refixation movement. Extraocular muscle over action or deficiency was recorded. Ocular motility referred to eye movements governed by the 6 extraocular muscles in each eye. It was assessed by examiner observation of the participants ability to abduct, adduct, supra, and inferoduct each eye. Ocular alignment and motility included the presence or absence of strabismus (classified as Esotropia, Exotropia, Hypertropia, Hypotropia) was recorded. Number of participants with combined data for ocular alignment and oculomotor examination (motility) assessed by corneal light reflex and by the cover test at 5-Years CA were reported.
Time frame: At 5 Years CA
Population: Enrolled set included all participants for whom written informed consent was obtained for this long-term outcome study. Here, Overall Number of participants analyzed signifies those participants who were evaluable for this outcome measure.
| Arm | Measure | Group | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|---|
| Antecedent Standard of Care | Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 5-Years CA | Participants with Esotropia | Presence | 1 Participants |
| Antecedent Standard of Care | Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 5-Years CA | Participants with Esotropia | Absence | 13 Participants |
| Antecedent Standard of Care | Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 5-Years CA | Participants with Exotropia | Presence | 0 Participants |
| Antecedent Standard of Care | Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 5-Years CA | Participants with Exotropia | Absence | 14 Participants |
| Antecedent Standard of Care | Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 5-Years CA | Participants with Hypertropia | Presence | 0 Participants |
| Antecedent Standard of Care | Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 5-Years CA | Participants with Hypertropia | Absence | 14 Participants |
| Antecedent Standard of Care | Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 5-Years CA | Participants with Hypotropia | Presence | 0 Participants |
| Antecedent Standard of Care | Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 5-Years CA | Participants with Hypotropia | Absence | 14 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 5-Years CA | Participants with Hypotropia | Absence | 17 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 5-Years CA | Participants with Esotropia | Presence | 1 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 5-Years CA | Participants with Hypertropia | Presence | 0 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 5-Years CA | Participants with Esotropia | Absence | 16 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 5-Years CA | Participants with Hypotropia | Presence | 0 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 5-Years CA | Participants with Exotropia | Presence | 0 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 5-Years CA | Participants with Hypertropia | Absence | 17 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Oculomotor Examination (Motility) and Ocular Alignment (Assessed by Corneal Light Reflex and Cover Test) at 5-Years CA | Participants with Exotropia | Absence | 17 Participants |
Primary
Number of Participants With Stereoacuity as Assessed With the Lang Stereotest At 5 Years CA
Stereoacuity, a measure of depth perception, was assessed using the Lang Stereotest. Number of participants with presence and absence of stereopsis (the ability to perceive depth and 3-dimensional structure) was reported.
Time frame: At 5 Years CA
Population: Enrolled set included all participants for whom written informed consent was obtained for this long-term outcome study. Here, Overall Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure.
| Arm | Measure | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Antecedent Standard of Care | Number of Participants With Stereoacuity as Assessed With the Lang Stereotest At 5 Years CA | Participants with presence of stereopsis | 13 Participants |
| Antecedent Standard of Care | Number of Participants With Stereoacuity as Assessed With the Lang Stereotest At 5 Years CA | Participants with absence of stereopsis | 1 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Stereoacuity as Assessed With the Lang Stereotest At 5 Years CA | Participants with absence of stereopsis | 1 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Stereoacuity as Assessed With the Lang Stereotest At 5 Years CA | Participants with presence of stereopsis | 16 Participants |
Primary
Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 12 Months CA
Standard age-appropriate methods were used to assess visual acuity (how well a participant sees at different distances) with teller acuity cards, LEA symbols test and Charts. It was categorized as following: normal (measurable acuity \>= 20/40 or \>= 15 cycles/degree); below normal (20/200 \<= measurable acuity \< 20/40 or 3 cycles/degree \<= measurable acuity \< 15 cycles/degree); poor (measurable acuity \<= 20/200 or \<= 3 cycles/degree). Corrected Age (weeks) was defined as Chronological Age (weeks) - 40 + Gestational Age (weeks) in this study.
Time frame: At 12 Months CA
Population: Enrolled set included all participants for whom written informed consent was obtained for this long-term outcome study. Here, Overall Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure and Number analyzed signifies those participants who were evaluable at the specified categories.
| Arm | Measure | Group | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|---|
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 12 Months CA | Participants With Right Eye | Below Normal | 21 Participants |
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 12 Months CA | Participants With Left Eye | Poor | 1 Participants |
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 12 Months CA | Participants With Left Eye | Normal | 0 Participants |
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 12 Months CA | Participants With Both Eyes | Normal | 0 Participants |
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 12 Months CA | Participants With Right Eye | Poor | 0 Participants |
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 12 Months CA | Participants With Both Eyes | Below Normal | 23 Participants |
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 12 Months CA | Participants With Left Eye | Below Normal | 20 Participants |
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 12 Months CA | Participants With Both Eyes | Poor | 1 Participants |
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 12 Months CA | Participants With Right Eye | Normal | 0 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 12 Months CA | Participants With Both Eyes | Poor | 2 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 12 Months CA | Participants With Right Eye | Normal | 1 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 12 Months CA | Participants With Right Eye | Below Normal | 26 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 12 Months CA | Participants With Right Eye | Poor | 5 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 12 Months CA | Participants With Left Eye | Normal | 1 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 12 Months CA | Participants With Left Eye | Below Normal | 25 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 12 Months CA | Participants With Left Eye | Poor | 6 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 12 Months CA | Participants With Both Eyes | Normal | 1 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 12 Months CA | Participants With Both Eyes | Below Normal | 33 Participants |
Primary
Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 20 Months CA
Standard age-appropriate methods were used to assess visual acuity (how well a participant sees at different distances) with teller acuity cards, LEA symbols test and Charts. It was categorized as following: normal (measurable acuity \>= 20/40 or \>= 15 cycles/degree); below normal (20/200 \<= measurable acuity \< 20/40 or 3 cycles/degree \<= measurable acuity \< 15 cycles/degree); poor (measurable acuity \<= 20/200 or \<= 3 cycles/degree). Corrected Age (weeks) was defined as Chronological Age (weeks) - 40 + Gestational Age (weeks) in this study.
Time frame: At 20 Months CA
Population: Enrolled set included all participants for whom written informed consent was obtained for this long-term outcome study. Here, Overall Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure and Number analyzed signifies those participants who were evaluable at the specified categories.
| Arm | Measure | Group | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|---|
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 20 Months CA | Participants With Right Eye | Below Normal | 17 Participants |
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 20 Months CA | Participants With Left Eye | Poor | 2 Participants |
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 20 Months CA | Participants With Left Eye | Normal | 0 Participants |
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 20 Months CA | Participants With Both Eyes | Normal | 0 Participants |
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 20 Months CA | Participants With Right Eye | Poor | 2 Participants |
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 20 Months CA | Participants With Both Eyes | Below Normal | 20 Participants |
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 20 Months CA | Participants With Left Eye | Below Normal | 18 Participants |
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 20 Months CA | Participants With Both Eyes | Poor | 3 Participants |
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 20 Months CA | Participants With Right Eye | Normal | 0 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 20 Months CA | Participants With Both Eyes | Poor | 2 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 20 Months CA | Participants With Right Eye | Normal | 2 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 20 Months CA | Participants With Right Eye | Below Normal | 23 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 20 Months CA | Participants With Right Eye | Poor | 4 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 20 Months CA | Participants With Left Eye | Normal | 1 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 20 Months CA | Participants With Left Eye | Below Normal | 24 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 20 Months CA | Participants With Left Eye | Poor | 4 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 20 Months CA | Participants With Both Eyes | Normal | 1 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 20 Months CA | Participants With Both Eyes | Below Normal | 30 Participants |
Primary
Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 24 Months CA
Standard age-appropriate methods were used to assess visual acuity (how well a participant sees at different distances) with teller acuity cards, LEA symbols test and Charts. It was categorized as following: normal (measurable acuity \>= 20/40 or \>= 15 cycles/degree); below normal (20/200 \<= measurable acuity \< 20/40 or 3 cycles/degree \<= measurable acuity \< 15 cycles/degree); poor (measurable acuity \<= 20/200 or \<= 3 cycles/degree). Corrected Age (weeks) was defined as Chronological Age (weeks) - 40 + Gestational Age (weeks) in this study.
Time frame: At 24 Months CA
Population: Enrolled set included all participants for whom written informed consent was obtained for this long-term outcome study. Here, Overall Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure and Number analyzed signifies those participants who were evaluable at the specified categories.
| Arm | Measure | Group | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|---|
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 24 Months CA | Participants With Right Eye | Below Normal | 17 Participants |
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 24 Months CA | Participants With Left Eye | Poor | 3 Participants |
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 24 Months CA | Participants With Left Eye | Normal | 0 Participants |
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 24 Months CA | Participants With Both Eyes | Normal | 1 Participants |
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 24 Months CA | Participants With Right Eye | Poor | 3 Participants |
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 24 Months CA | Participants With Both Eyes | Below Normal | 19 Participants |
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 24 Months CA | Participants With Left Eye | Below Normal | 17 Participants |
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 24 Months CA | Participants With Both Eyes | Poor | 3 Participants |
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 24 Months CA | Participants With Right Eye | Normal | 0 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 24 Months CA | Participants With Both Eyes | Poor | 4 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 24 Months CA | Participants With Right Eye | Normal | 2 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 24 Months CA | Participants With Right Eye | Below Normal | 25 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 24 Months CA | Participants With Right Eye | Poor | 4 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 24 Months CA | Participants With Left Eye | Normal | 2 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 24 Months CA | Participants With Left Eye | Below Normal | 24 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 24 Months CA | Participants With Left Eye | Poor | 2 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 24 Months CA | Participants With Both Eyes | Normal | 5 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 24 Months CA | Participants With Both Eyes | Below Normal | 24 Participants |
Primary
Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 4.75 Years CA
Standard age-appropriate methods were used to assess visual acuity (how well a participant sees at different distances) with teller acuity cards, LEA symbols test and Charts. It was categorized as following: normal (measurable acuity \>= 20/40 or \>= 15 cycles/degree); below normal (20/200 \<= measurable acuity \< 20/40 or 3 cycles/degree \<= measurable acuity \< 15 cycles/degree); poor (measurable acuity \<= 20/200 or \<= 3 cycles/degree). Corrected Age (weeks) was defined as Chronological Age (weeks) - 40 + Gestational Age (weeks) in this study.
Time frame: At 4.75 Years CA
Population: Enrolled set included all participants for whom written informed consent was obtained for this long-term outcome study. Here, Overall Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure.
| Arm | Measure | Group | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|---|
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 4.75 Years CA | Participants With Right Eye | Below Normal | 4 Participants |
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 4.75 Years CA | Participants With Left Eye | Poor | 0 Participants |
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 4.75 Years CA | Participants With Left Eye | Normal | 11 Participants |
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 4.75 Years CA | Participants With Both Eyes | Normal | 10 Participants |
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 4.75 Years CA | Participants With Right Eye | Poor | 0 Participants |
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 4.75 Years CA | Participants With Both Eyes | Below Normal | 5 Participants |
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 4.75 Years CA | Participants With Left Eye | Below Normal | 4 Participants |
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 4.75 Years CA | Participants With Both Eyes | Poor | 0 Participants |
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 4.75 Years CA | Participants With Right Eye | Normal | 11 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 4.75 Years CA | Participants With Both Eyes | Poor | 0 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 4.75 Years CA | Participants With Right Eye | Normal | 15 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 4.75 Years CA | Participants With Right Eye | Below Normal | 3 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 4.75 Years CA | Participants With Right Eye | Poor | 0 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 4.75 Years CA | Participants With Left Eye | Normal | 14 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 4.75 Years CA | Participants With Left Eye | Below Normal | 4 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 4.75 Years CA | Participants With Left Eye | Poor | 0 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 4.75 Years CA | Participants With Both Eyes | Normal | 17 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 4.75 Years CA | Participants With Both Eyes | Below Normal | 1 Participants |
Primary
Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 5 Years CA
Standard age-appropriate methods were used to assess visual acuity (how well a participant sees at different distances) with teller acuity cards, LEA symbols test and Charts. It was categorized as following: normal (measurable acuity \>= 20/40 or \>= 15 cycles/degree); below normal (20/200 \<= measurable acuity \< 20/40 or 3 cycles/degree \<= measurable acuity \< 15 cycles/degree); poor (measurable acuity \<= 20/200 or \<= 3 cycles/degree). Corrected Age (weeks) was defined as Chronological Age (weeks) - 40 + Gestational Age (weeks) in this study.
Time frame: At 5 Years CA
Population: Enrolled set included all participants for whom written informed consent was obtained for this long-term outcome study. Here, Overall Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure.
| Arm | Measure | Group | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|---|
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 5 Years CA | Participants With Right Eye | Below Normal | 5 Participants |
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 5 Years CA | Participants With Left Eye | Poor | 0 Participants |
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 5 Years CA | Participants With Left Eye | Normal | 8 Participants |
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 5 Years CA | Participants With Both Eyes | Normal | 9 Participants |
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 5 Years CA | Participants With Right Eye | Poor | 0 Participants |
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 5 Years CA | Participants With Both Eyes | Below Normal | 5 Participants |
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 5 Years CA | Participants With Left Eye | Below Normal | 6 Participants |
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 5 Years CA | Participants With Both Eyes | Poor | 0 Participants |
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 5 Years CA | Participants With Right Eye | Normal | 9 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 5 Years CA | Participants With Both Eyes | Poor | 0 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 5 Years CA | Participants With Right Eye | Normal | 11 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 5 Years CA | Participants With Right Eye | Below Normal | 6 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 5 Years CA | Participants With Right Eye | Poor | 0 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 5 Years CA | Participants With Left Eye | Normal | 11 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 5 Years CA | Participants With Left Eye | Below Normal | 6 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 5 Years CA | Participants With Left Eye | Poor | 0 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 5 Years CA | Participants With Both Eyes | Normal | 11 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 5 Years CA | Participants With Both Eyes | Below Normal | 6 Participants |
Primary
Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 6 Months Corrected Age (CA)
Standard age-appropriate methods were used to assess visual acuity (how well a participant sees at different distances) with teller acuity cards, LEA symbols test and Charts. It was categorized as following: normal (measurable acuity \>= 20/40 or \>= 15 cycles/degree); below normal (20/200 \<= measurable acuity \< 20/40 or 3 cycles/degree \<= measurable acuity \< 15 cycles/degree); poor (measurable acuity \<= 20/200 or \<= 3 cycles/degree). Corrected Age (weeks) was defined as Chronological Age (weeks) - 40 + Gestational Age (weeks) in this study.
Time frame: At 6 Months CA
Population: Enrolled set included all participants for whom written informed consent was obtained for this long-term outcome study. Here, Overall Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure and Number analyzed signifies those participants who were evaluable at the specified categories.
| Arm | Measure | Group | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|---|
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 6 Months Corrected Age (CA) | Participants With Right Eye | Below Normal | 12 Participants |
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 6 Months Corrected Age (CA) | Participants With Left Eye | Poor | 7 Participants |
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 6 Months Corrected Age (CA) | Participants With Left Eye | Normal | 0 Participants |
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 6 Months Corrected Age (CA) | Participants With Both Eyes | Normal | 0 Participants |
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 6 Months Corrected Age (CA) | Participants With Right Eye | Poor | 12 Participants |
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 6 Months Corrected Age (CA) | Participants With Both Eyes | Below Normal | 18 Participants |
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 6 Months Corrected Age (CA) | Participants With Left Eye | Below Normal | 14 Participants |
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 6 Months Corrected Age (CA) | Participants With Both Eyes | Poor | 9 Participants |
| Antecedent Standard of Care | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 6 Months Corrected Age (CA) | Participants With Right Eye | Normal | 0 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 6 Months Corrected Age (CA) | Participants With Both Eyes | Poor | 9 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 6 Months Corrected Age (CA) | Participants With Right Eye | Normal | 0 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 6 Months Corrected Age (CA) | Participants With Right Eye | Below Normal | 19 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 6 Months Corrected Age (CA) | Participants With Right Eye | Poor | 13 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 6 Months Corrected Age (CA) | Participants With Left Eye | Normal | 0 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 6 Months Corrected Age (CA) | Participants With Left Eye | Below Normal | 18 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 6 Months Corrected Age (CA) | Participants With Left Eye | Poor | 14 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 6 Months Corrected Age (CA) | Participants With Both Eyes | Normal | 0 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Visual Acuity as Assessed by an Age-appropriate Method at 6 Months Corrected Age (CA) | Participants With Both Eyes | Below Normal | 25 Participants |
Primary
Refraction With Cycloplegia as Assessed by Retinoscopy at 12 Month CA
Refraction was a measure of the lens power required for a focused image on the retina. Refraction with cycloplegia was measured and recorded in diopters for each eye individually (left eye and right eye). Refraction with cycloplegia performed as part of the corrective lens determination procedure included measurements of sphere, cylinder, axis, and prism for each individual eye (left eye and right eye). For sphere, the negative values are summarized as 'nearsighted', and the positive values (including sphere of 0.00) are summarized as 'farsighted'.
Time frame: At 12 Months CA
Population: Enrolled set included all participants for whom written informed consent was obtained for this long-term outcome study. Here, Overall Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure and Number analyzed signifies those participants who were evaluable at the specified categories.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Antecedent Standard of Care | Refraction With Cycloplegia as Assessed by Retinoscopy at 12 Month CA | Right Eye: Sphere (Nearsighted) | -1.564 Diopter | Standard Deviation 1.8752 |
| Antecedent Standard of Care | Refraction With Cycloplegia as Assessed by Retinoscopy at 12 Month CA | Right Eye: Sphere (Farsighted) | 2.042 Diopter | Standard Deviation 1.1796 |
| Antecedent Standard of Care | Refraction With Cycloplegia as Assessed by Retinoscopy at 12 Month CA | Right Eye: Cylinder | 0.783 Diopter | Standard Deviation 0.7163 |
| Antecedent Standard of Care | Refraction With Cycloplegia as Assessed by Retinoscopy at 12 Month CA | Right Eye: Axis | 86.0 Diopter | Standard Deviation 62.82 |
| Antecedent Standard of Care | Refraction With Cycloplegia as Assessed by Retinoscopy at 12 Month CA | Right Eye: Prism | 0.00 Diopter | Standard Deviation 0 |
| Antecedent Standard of Care | Refraction With Cycloplegia as Assessed by Retinoscopy at 12 Month CA | Left Eye: Sphere (Nearsighted) | -2.875 Diopter | Standard Deviation 1.9203 |
| Antecedent Standard of Care | Refraction With Cycloplegia as Assessed by Retinoscopy at 12 Month CA | Left Eye: Sphere (Farsighted) | 1.857 Diopter | Standard Deviation 1.3171 |
| Antecedent Standard of Care | Refraction With Cycloplegia as Assessed by Retinoscopy at 12 Month CA | Left Eye: Cylinder | 0.663 Diopter | Standard Deviation 0.6334 |
| Antecedent Standard of Care | Refraction With Cycloplegia as Assessed by Retinoscopy at 12 Month CA | Left Eye: Axis | 107.3 Diopter | Standard Deviation 60.19 |
| Antecedent Standard of Care | Refraction With Cycloplegia as Assessed by Retinoscopy at 12 Month CA | Left Eye: Prism | 0.00 Diopter | Standard Deviation 0 |
| Antecedent rhIGF-1/rhIGFBP-3 | Refraction With Cycloplegia as Assessed by Retinoscopy at 12 Month CA | Left Eye: Cylinder | 0.851 Diopter | Standard Deviation 0.6795 |
| Antecedent rhIGF-1/rhIGFBP-3 | Refraction With Cycloplegia as Assessed by Retinoscopy at 12 Month CA | Right Eye: Sphere (Nearsighted) | -2.438 Diopter | Standard Deviation 1.4773 |
| Antecedent rhIGF-1/rhIGFBP-3 | Refraction With Cycloplegia as Assessed by Retinoscopy at 12 Month CA | Left Eye: Sphere (Nearsighted) | -2.500 Diopter | Standard Deviation 1.7048 |
| Antecedent rhIGF-1/rhIGFBP-3 | Refraction With Cycloplegia as Assessed by Retinoscopy at 12 Month CA | Right Eye: Sphere (Farsighted) | 1.491 Diopter | Standard Deviation 1.0908 |
| Antecedent rhIGF-1/rhIGFBP-3 | Refraction With Cycloplegia as Assessed by Retinoscopy at 12 Month CA | Left Eye: Prism | 0.00 Diopter | Standard Deviation 0 |
| Antecedent rhIGF-1/rhIGFBP-3 | Refraction With Cycloplegia as Assessed by Retinoscopy at 12 Month CA | Right Eye: Cylinder | 0.926 Diopter | Standard Deviation 0.6423 |
| Antecedent rhIGF-1/rhIGFBP-3 | Refraction With Cycloplegia as Assessed by Retinoscopy at 12 Month CA | Left Eye: Sphere (Farsighted) | 1.606 Diopter | Standard Deviation 1.1962 |
| Antecedent rhIGF-1/rhIGFBP-3 | Refraction With Cycloplegia as Assessed by Retinoscopy at 12 Month CA | Right Eye: Axis | 105.7 Diopter | Standard Deviation 50.53 |
| Antecedent rhIGF-1/rhIGFBP-3 | Refraction With Cycloplegia as Assessed by Retinoscopy at 12 Month CA | Left Eye: Axis | 112.6 Diopter | Standard Deviation 55.11 |
| Antecedent rhIGF-1/rhIGFBP-3 | Refraction With Cycloplegia as Assessed by Retinoscopy at 12 Month CA | Right Eye: Prism | 0.00 Diopter | Standard Deviation 0 |
Primary
Refraction With Cycloplegia as Assessed by Retinoscopy at 20 Month CA
Refraction was a measure of the lens power required for a focused image on the retina. Refraction with cycloplegia was measured and recorded in diopters for each eye individually (left eye and right eye). Refraction with cycloplegia performed as part of the corrective lens determination procedure included measurements of sphere, cylinder, axis, and prism for each individual eye (left eye and right eye). For sphere, the negative values are summarized as 'nearsighted', and the positive values (including sphere of 0.00) are summarized as 'farsighted'.
Time frame: At 20 Months CA
Population: Enrolled set included all participants for whom written informed consent was obtained for this long-term outcome study. Here, Overall Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure and Number analyzed signifies those participants who were evaluable at the specified categories.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Antecedent Standard of Care | Refraction With Cycloplegia as Assessed by Retinoscopy at 20 Month CA | Right Eye: Cylinder | 0.750 Diopter | Standard Deviation 0.6755 |
| Antecedent Standard of Care | Refraction With Cycloplegia as Assessed by Retinoscopy at 20 Month CA | Left Eye: Sphere (Nearsighted) | -3.500 Diopter | Standard Deviation 1.8028 |
| Antecedent Standard of Care | Refraction With Cycloplegia as Assessed by Retinoscopy at 20 Month CA | Left Eye: Sphere (Farsighted) | 1.800 Diopter | Standard Deviation 1.2237 |
| Antecedent Standard of Care | Refraction With Cycloplegia as Assessed by Retinoscopy at 20 Month CA | Right Eye: Sphere (Farsighted) | 1.903 Diopter | Standard Deviation 0.9931 |
| Antecedent Standard of Care | Refraction With Cycloplegia as Assessed by Retinoscopy at 20 Month CA | Left Eye: Cylinder | 0.900 Diopter | Standard Deviation 0.8288 |
| Antecedent Standard of Care | Refraction With Cycloplegia as Assessed by Retinoscopy at 20 Month CA | Right Eye: Axis | 105.0 Diopter | Standard Deviation 62.33 |
| Antecedent Standard of Care | Refraction With Cycloplegia as Assessed by Retinoscopy at 20 Month CA | Left Eye: Axis | 117.8 Diopter | Standard Deviation 53.82 |
| Antecedent Standard of Care | Refraction With Cycloplegia as Assessed by Retinoscopy at 20 Month CA | Right Eye: Sphere (Nearsighted) | -2.350 Diopter | Standard Deviation 2.4083 |
| Antecedent Standard of Care | Refraction With Cycloplegia as Assessed by Retinoscopy at 20 Month CA | Left Eye: Prism | 0.00 Diopter | Standard Deviation 0 |
| Antecedent Standard of Care | Refraction With Cycloplegia as Assessed by Retinoscopy at 20 Month CA | Right Eye: Prism | 0.00 Diopter | Standard Deviation 0 |
| Antecedent rhIGF-1/rhIGFBP-3 | Refraction With Cycloplegia as Assessed by Retinoscopy at 20 Month CA | Left Eye: Prism | 0.00 Diopter | Standard Deviation 0 |
| Antecedent rhIGF-1/rhIGFBP-3 | Refraction With Cycloplegia as Assessed by Retinoscopy at 20 Month CA | Right Eye: Sphere (Nearsighted) | -1.700 Diopter | Standard Deviation 1.6808 |
| Antecedent rhIGF-1/rhIGFBP-3 | Refraction With Cycloplegia as Assessed by Retinoscopy at 20 Month CA | Right Eye: Sphere (Farsighted) | 1.278 Diopter | Standard Deviation 0.8996 |
| Antecedent rhIGF-1/rhIGFBP-3 | Refraction With Cycloplegia as Assessed by Retinoscopy at 20 Month CA | Right Eye: Cylinder | 0.778 Diopter | Standard Deviation 0.6841 |
| Antecedent rhIGF-1/rhIGFBP-3 | Refraction With Cycloplegia as Assessed by Retinoscopy at 20 Month CA | Right Eye: Axis | 122.8 Diopter | Standard Deviation 58.69 |
| Antecedent rhIGF-1/rhIGFBP-3 | Refraction With Cycloplegia as Assessed by Retinoscopy at 20 Month CA | Right Eye: Prism | 0.00 Diopter | Standard Deviation 0 |
| Antecedent rhIGF-1/rhIGFBP-3 | Refraction With Cycloplegia as Assessed by Retinoscopy at 20 Month CA | Left Eye: Sphere (Farsighted) | 1.389 Diopter | Standard Deviation 1.108 |
| Antecedent rhIGF-1/rhIGFBP-3 | Refraction With Cycloplegia as Assessed by Retinoscopy at 20 Month CA | Left Eye: Cylinder | 0.681 Diopter | Standard Deviation 0.5665 |
| Antecedent rhIGF-1/rhIGFBP-3 | Refraction With Cycloplegia as Assessed by Retinoscopy at 20 Month CA | Left Eye: Axis | 110.1 Diopter | Standard Deviation 53.56 |
| Antecedent rhIGF-1/rhIGFBP-3 | Refraction With Cycloplegia as Assessed by Retinoscopy at 20 Month CA | Left Eye: Sphere (Nearsighted) | -2.600 Diopter | Standard Deviation 1.9733 |
Primary
Refraction With Cycloplegia as Assessed by Retinoscopy at 4.75 Years CA
Refraction was a measure of the lens power required for a focused image on the retina. Refraction with cycloplegia was measured and recorded in diopters for each eye individually (left eye and right eye). Refraction with cycloplegia performed as part of the corrective lens determination procedure included measurements of sphere, cylinder, axis, and prism for each individual eye (left eye and right eye). For sphere, the negative values are summarized as 'nearsighted', and the positive values (including sphere of 0.00) are summarized as 'farsighted'.
Time frame: At 4.75 Years CA
Population: Enrolled set included all participants for whom written informed consent was obtained for this long-term outcome study. Here, Overall Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure and Number analyzed signifies those participants who were evaluable at the specified categories.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Antecedent Standard of Care | Refraction With Cycloplegia as Assessed by Retinoscopy at 4.75 Years CA | Right Eye: Sphere (Nearsighted) | -8.500 Diopter | — |
| Antecedent Standard of Care | Refraction With Cycloplegia as Assessed by Retinoscopy at 4.75 Years CA | Right Eye: Sphere (Farsighted) | 1.691 Diopter | Standard Deviation 1.1094 |
| Antecedent Standard of Care | Refraction With Cycloplegia as Assessed by Retinoscopy at 4.75 Years CA | Right Eye: Cylinder | 0.593 Diopter | Standard Deviation 0.6505 |
| Antecedent Standard of Care | Refraction With Cycloplegia as Assessed by Retinoscopy at 4.75 Years CA | Right Eye: Axis | 99.1 Diopter | Standard Deviation 69.95 |
| Antecedent Standard of Care | Refraction With Cycloplegia as Assessed by Retinoscopy at 4.75 Years CA | Right Eye: Prism | 0.02 Diopter | Standard Deviation 0.075 |
| Antecedent Standard of Care | Refraction With Cycloplegia as Assessed by Retinoscopy at 4.75 Years CA | Left Eye: Sphere (Nearsighted) | -6.500 Diopter | — |
| Antecedent Standard of Care | Refraction With Cycloplegia as Assessed by Retinoscopy at 4.75 Years CA | Left Eye: Sphere (Farsighted) | 1.853 Diopter | Standard Deviation 1.2376 |
| Antecedent Standard of Care | Refraction With Cycloplegia as Assessed by Retinoscopy at 4.75 Years CA | Left Eye: Cylinder | 0.717 Diopter | Standard Deviation 0.7126 |
| Antecedent Standard of Care | Refraction With Cycloplegia as Assessed by Retinoscopy at 4.75 Years CA | Left Eye: Axis | 88.6 Diopter | Standard Deviation 65.64 |
| Antecedent Standard of Care | Refraction With Cycloplegia as Assessed by Retinoscopy at 4.75 Years CA | Left Eye: Prism | 0.02 Diopter | Standard Deviation 0.056 |
| Antecedent rhIGF-1/rhIGFBP-3 | Refraction With Cycloplegia as Assessed by Retinoscopy at 4.75 Years CA | Left Eye: Cylinder | 0.574 Diopter | Standard Deviation 0.5574 |
| Antecedent rhIGF-1/rhIGFBP-3 | Refraction With Cycloplegia as Assessed by Retinoscopy at 4.75 Years CA | Right Eye: Sphere (Nearsighted) | -0.500 Diopter | — |
| Antecedent rhIGF-1/rhIGFBP-3 | Refraction With Cycloplegia as Assessed by Retinoscopy at 4.75 Years CA | Left Eye: Sphere (Nearsighted) | -0.750 Diopter | — |
| Antecedent rhIGF-1/rhIGFBP-3 | Refraction With Cycloplegia as Assessed by Retinoscopy at 4.75 Years CA | Right Eye: Sphere (Farsighted) | 1.383 Diopter | Standard Deviation 1.5465 |
| Antecedent rhIGF-1/rhIGFBP-3 | Refraction With Cycloplegia as Assessed by Retinoscopy at 4.75 Years CA | Left Eye: Prism | 0.00 Diopter | Standard Deviation 0 |
| Antecedent rhIGF-1/rhIGFBP-3 | Refraction With Cycloplegia as Assessed by Retinoscopy at 4.75 Years CA | Right Eye: Cylinder | 0.603 Diopter | Standard Deviation 0.6254 |
| Antecedent rhIGF-1/rhIGFBP-3 | Refraction With Cycloplegia as Assessed by Retinoscopy at 4.75 Years CA | Left Eye: Sphere (Farsighted) | 1.517 Diopter | Standard Deviation 1.9966 |
| Antecedent rhIGF-1/rhIGFBP-3 | Refraction With Cycloplegia as Assessed by Retinoscopy at 4.75 Years CA | Right Eye: Axis | 119.0 Diopter | Standard Deviation 56.05 |
| Antecedent rhIGF-1/rhIGFBP-3 | Refraction With Cycloplegia as Assessed by Retinoscopy at 4.75 Years CA | Left Eye: Axis | 114.2 Diopter | Standard Deviation 55.17 |
| Antecedent rhIGF-1/rhIGFBP-3 | Refraction With Cycloplegia as Assessed by Retinoscopy at 4.75 Years CA | Right Eye: Prism | 0.00 Diopter | Standard Deviation 0 |
Primary
Refraction With Cycloplegia as Assessed by Retinoscopy at 6 Months CA
Refraction was a measure of the lens power required for a focused image on the retina. Refraction with cycloplegia was measured and recorded in diopters for each eye individually (left eye and right eye). Refraction with cycloplegia performed as part of the corrective lens determination procedure included measurements of sphere, cylinder, axis, and prism for each individual eye (left eye and right eye). For sphere, the negative values are summarized as 'nearsighted', and the positive values (including sphere of 0.00) are summarized as 'farsighted'.
Time frame: At 6 Months CA
Population: Enrolled set included all participants for whom written informed consent was obtained for this long-term outcome study. Here, Overall Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure and Number analyzed signifies those participants who were evaluable at the specified categories.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Antecedent Standard of Care | Refraction With Cycloplegia as Assessed by Retinoscopy at 6 Months CA | Right Eye: Sphere (Nearsighted) | -1.833 Diopter | Standard Deviation 1.0916 |
| Antecedent Standard of Care | Refraction With Cycloplegia as Assessed by Retinoscopy at 6 Months CA | Right Eye: Sphere (Farsighted) | 1.976 Diopter | Standard Deviation 1.2572 |
| Antecedent Standard of Care | Refraction With Cycloplegia as Assessed by Retinoscopy at 6 Months CA | Right Eye: Cylinder | 0.875 Diopter | Standard Deviation 0.8534 |
| Antecedent Standard of Care | Refraction With Cycloplegia as Assessed by Retinoscopy at 6 Months CA | Right Eye: Axis | 116.4 Diopter | Standard Deviation 58.03 |
| Antecedent Standard of Care | Refraction With Cycloplegia as Assessed by Retinoscopy at 6 Months CA | Left Eye: Sphere (nearsighted) | -1.750 Diopter | Standard Deviation 0.9747 |
| Antecedent Standard of Care | Refraction With Cycloplegia as Assessed by Retinoscopy at 6 Months CA | Left Eye: Sphere (farsighted) | 2.034 Diopter | Standard Deviation 1.2731 |
| Antecedent Standard of Care | Refraction With Cycloplegia as Assessed by Retinoscopy at 6 Months CA | Left Eye: Cylinder | 0.820 Diopter | Standard Deviation 0.6395 |
| Antecedent Standard of Care | Refraction With Cycloplegia as Assessed by Retinoscopy at 6 Months CA | Left Eye: Axis | 120.3 Diopter | Standard Deviation 52.27 |
| Antecedent rhIGF-1/rhIGFBP-3 | Refraction With Cycloplegia as Assessed by Retinoscopy at 6 Months CA | Left Eye: Axis | 108.8 Diopter | Standard Deviation 54.18 |
| Antecedent rhIGF-1/rhIGFBP-3 | Refraction With Cycloplegia as Assessed by Retinoscopy at 6 Months CA | Right Eye: Sphere (Nearsighted) | -1.417 Diopter | Standard Deviation 1.4216 |
| Antecedent rhIGF-1/rhIGFBP-3 | Refraction With Cycloplegia as Assessed by Retinoscopy at 6 Months CA | Left Eye: Sphere (nearsighted) | -1.250 Diopter | Standard Deviation 0.9014 |
| Antecedent rhIGF-1/rhIGFBP-3 | Refraction With Cycloplegia as Assessed by Retinoscopy at 6 Months CA | Right Eye: Sphere (Farsighted) | 1.838 Diopter | Standard Deviation 1.3256 |
| Antecedent rhIGF-1/rhIGFBP-3 | Refraction With Cycloplegia as Assessed by Retinoscopy at 6 Months CA | Left Eye: Cylinder | 0.962 Diopter | Standard Deviation 0.8341 |
| Antecedent rhIGF-1/rhIGFBP-3 | Refraction With Cycloplegia as Assessed by Retinoscopy at 6 Months CA | Right Eye: Cylinder | 0.932 Diopter | Standard Deviation 0.8801 |
| Antecedent rhIGF-1/rhIGFBP-3 | Refraction With Cycloplegia as Assessed by Retinoscopy at 6 Months CA | Left Eye: Sphere (farsighted) | 1.831 Diopter | Standard Deviation 1.3494 |
| Antecedent rhIGF-1/rhIGFBP-3 | Refraction With Cycloplegia as Assessed by Retinoscopy at 6 Months CA | Right Eye: Axis | 106.0 Diopter | Standard Deviation 52.87 |
Secondary
Change From Baseline (12 Months CA) in Cognitive Development as Assessed by Bayley Scales of Infant and Toddler Development (BSID-III) Composite Scores at 24 Months CA
BSID-III was to assess cognitive, motor, and language skills, and applicable to children aged 1-42 months. There are 5 subscales, cognitive subscale (Ranges: 55-145) stands alone while 2 language subscales (expressive and receptive) combine to make a total language score (Ranges: 47-153) and 2 motor subtests (fine and gross motor) form combined motor scale (Ranges: 46-154). Higher scores represent greater cognitive, language and motor abilities. Positive value indicates improvement and negative value indicate worsening in cognitive development. The 12 Months CA considered as baseline for this outcome measure. Composite scores derived from various sums of subtest scaled scores and scaled to a metric with a mean of 100 and a standard deviation of 15 and range from 40 to 160. Higher values denote stronger skills and abilities in the domain, indicating better outcomes.
Time frame: Baseline (12 Months CA), 24 Months CA
Population: Enrolled set included all participants for whom written informed consent was obtained for this long-term outcome study. Here, Overall Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure and Number analyzed signifies those participants who were evaluable at the specified time point.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Antecedent Standard of Care | Change From Baseline (12 Months CA) in Cognitive Development as Assessed by Bayley Scales of Infant and Toddler Development (BSID-III) Composite Scores at 24 Months CA | Cognitive Composite Scores: Change at 24 Months CA | 7.0 Score on a scale | Standard Deviation 18.45 |
| Antecedent Standard of Care | Change From Baseline (12 Months CA) in Cognitive Development as Assessed by Bayley Scales of Infant and Toddler Development (BSID-III) Composite Scores at 24 Months CA | Language Composite Scores: Change at 24 Months CA | 2.4 Score on a scale | Standard Deviation 18.73 |
| Antecedent Standard of Care | Change From Baseline (12 Months CA) in Cognitive Development as Assessed by Bayley Scales of Infant and Toddler Development (BSID-III) Composite Scores at 24 Months CA | Motor Composite Scores: Change at 24 Months CA | 12.2 Score on a scale | Standard Deviation 23.54 |
| Antecedent rhIGF-1/rhIGFBP-3 | Change From Baseline (12 Months CA) in Cognitive Development as Assessed by Bayley Scales of Infant and Toddler Development (BSID-III) Composite Scores at 24 Months CA | Motor Composite Scores: Change at 24 Months CA | 10.8 Score on a scale | Standard Deviation 18.25 |
| Antecedent rhIGF-1/rhIGFBP-3 | Change From Baseline (12 Months CA) in Cognitive Development as Assessed by Bayley Scales of Infant and Toddler Development (BSID-III) Composite Scores at 24 Months CA | Cognitive Composite Scores: Change at 24 Months CA | 6.6 Score on a scale | Standard Deviation 21.27 |
| Antecedent rhIGF-1/rhIGFBP-3 | Change From Baseline (12 Months CA) in Cognitive Development as Assessed by Bayley Scales of Infant and Toddler Development (BSID-III) Composite Scores at 24 Months CA | Language Composite Scores: Change at 24 Months CA | -0.9 Score on a scale | Standard Deviation 15.79 |
Secondary
Change From Baseline (24 Months CA) in Pediatric Quality of Life Inventory (PedsQL™) Scale: Generic Core Scale (GCS) Total Score
The PedsQL was a generic health related quality of life instrument designed specifically for a pediatric population. The GCS encompasses 4 dimensions of functioning (physical, emotional, social, school). The GCS that apply to toddler for 2-4 years of age (21 Items) rated on a 5-point Likert scale (0 to 4); and Young Child for 5-7 years of age (23 Items) rated on a 3-point Likert scale (0, 2, 4). Overall total scores were calculated as average of all dimensional sub-score items of GCS (Toddler and young children's) ranging from 0 to 100 where 0=100 (Never), 1=75 (almost never), 2=50 (sometimes), 3=25 (often), and 4=0 (almost always). Higher scores indicate improved quality of life. A negative value indicates decreased quality of life. The 24 month CA was considered as baseline for this outcome measure.
Time frame: Baseline (24 Months CA), up to 3 Years CA, 4 Years CA and 5 Years CA
Population: Enrolled set included all participants for whom written informed consent was obtained for this long-term outcome study. Here, Overall Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure and Number analyzed signifies those participants who were evaluable at specified time points.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Antecedent Standard of Care | Change From Baseline (24 Months CA) in Pediatric Quality of Life Inventory (PedsQL™) Scale: Generic Core Scale (GCS) Total Score | Change at 3 Years CA: Total Score | 0.62 Score on scale | Standard Deviation 12.22 |
| Antecedent Standard of Care | Change From Baseline (24 Months CA) in Pediatric Quality of Life Inventory (PedsQL™) Scale: Generic Core Scale (GCS) Total Score | Change at 4 Years CA: Total Score | 1.50 Score on scale | Standard Deviation 14.376 |
| Antecedent Standard of Care | Change From Baseline (24 Months CA) in Pediatric Quality of Life Inventory (PedsQL™) Scale: Generic Core Scale (GCS) Total Score | Change at 5 Years CA: Total Score | -4.96 Score on scale | Standard Deviation 15.561 |
| Antecedent rhIGF-1/rhIGFBP-3 | Change From Baseline (24 Months CA) in Pediatric Quality of Life Inventory (PedsQL™) Scale: Generic Core Scale (GCS) Total Score | Change at 3 Years CA: Total Score | -1.13 Score on scale | Standard Deviation 8.533 |
| Antecedent rhIGF-1/rhIGFBP-3 | Change From Baseline (24 Months CA) in Pediatric Quality of Life Inventory (PedsQL™) Scale: Generic Core Scale (GCS) Total Score | Change at 4 Years CA: Total Score | -3.46 Score on scale | Standard Deviation 7.917 |
| Antecedent rhIGF-1/rhIGFBP-3 | Change From Baseline (24 Months CA) in Pediatric Quality of Life Inventory (PedsQL™) Scale: Generic Core Scale (GCS) Total Score | Change at 5 Years CA: Total Score | -4.49 Score on scale | Standard Deviation 8.225 |
Secondary
Change From Baseline (3 Months CA) in Pediatric Quality of Life Inventory (PedsQL™) Scale: Infant Scale
The PedsQL was a generic health related quality of life instrument designed specifically for a pediatric population. PedsQL infant scale encompasses 5 dimensions of functioning (Physical Functioning, Physical Symptoms, Emotional Functioning, Social Functioning, and Cognitive Functioning). The infant for ages 1-12 months (36 Items); for ages 13-24 months (45 Items) rated on a 5-point Likert scale (0 to 4). Overall total scores were calculated as average of all dimensional sub-score items of Infant scale, ranging from 0 to 100 where 0=100 (Never), 1=75 (almost never), 2=50 (sometimes), 3=25 (often), and 4=0 (almost always). Higher scores indicate improved quality of life. A negative value indicates decreased quality of life. The 3 months CA was considered as baseline for this outcome measure.
Time frame: Baseline (3 Months CA) up to 6 Months CA, 12 Months CA and 24 Months CA
Population: Enrolled set included all participants for whom written informed consent was obtained for this long-term outcome study. Here, Overall Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure and Number analyzed signifies those participants who were evaluable at specified time points.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Antecedent Standard of Care | Change From Baseline (3 Months CA) in Pediatric Quality of Life Inventory (PedsQL™) Scale: Infant Scale | Change at 6 Months CA: Total Score (Infant scale) | -1.10 Score on scale | Standard Deviation 10.056 |
| Antecedent Standard of Care | Change From Baseline (3 Months CA) in Pediatric Quality of Life Inventory (PedsQL™) Scale: Infant Scale | Change at 12 Months CA: Total Score (Infant scale) | -0.16 Score on scale | Standard Deviation 12.875 |
| Antecedent Standard of Care | Change From Baseline (3 Months CA) in Pediatric Quality of Life Inventory (PedsQL™) Scale: Infant Scale | Change at 24 Months CA: Total Score (Infant scale) | 7.84 Score on scale | Standard Deviation 7.339 |
| Antecedent rhIGF-1/rhIGFBP-3 | Change From Baseline (3 Months CA) in Pediatric Quality of Life Inventory (PedsQL™) Scale: Infant Scale | Change at 6 Months CA: Total Score (Infant scale) | -0.60 Score on scale | Standard Deviation 11.088 |
| Antecedent rhIGF-1/rhIGFBP-3 | Change From Baseline (3 Months CA) in Pediatric Quality of Life Inventory (PedsQL™) Scale: Infant Scale | Change at 12 Months CA: Total Score (Infant scale) | -0.57 Score on scale | Standard Deviation 11.902 |
| Antecedent rhIGF-1/rhIGFBP-3 | Change From Baseline (3 Months CA) in Pediatric Quality of Life Inventory (PedsQL™) Scale: Infant Scale | Change at 24 Months CA: Total Score (Infant scale) | -4.82 Score on scale | Standard Deviation 14.205 |
Secondary
Change From Baseline (6 Months CA) in Child Behavior as Assessed by Vineland Adaptive Behaviour Scales (VABS-II)
The VABS-II was used to measure the personal and social skills of participants serially over time. This test measures the following 4 key domains: Communication, Daily Living Skills, Socialization, and Motor skills. The domains have the same range and directionality as the adaptive behavior composite score (20-160). Higher scores indicate higher level of cognitive ability. A positive change value indicates improvement and negative value indicates worsening in adaptive functioning. The 6 months CA was considered as baseline for this outcome measure. Change from baseline (6 Months CA) in child behavior as assessed by VABS-II adaptive behavior composite was reported here.
Time frame: Baseline (6 Months CA), 12 Months CA, 24 Months CA, and 5 Year CA
Population: Enrolled set included all participants for whom written informed consent was obtained for this long-term outcome study. Here, Overall Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure and Number analyzed signifies those participants who were evaluable at the specified time point.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Antecedent Standard of Care | Change From Baseline (6 Months CA) in Child Behavior as Assessed by Vineland Adaptive Behaviour Scales (VABS-II) | Change at 12 Months CA: Adaptive Behavior Composite | -0.3 Score on a scale | Standard Deviation 14.5 |
| Antecedent Standard of Care | Change From Baseline (6 Months CA) in Child Behavior as Assessed by Vineland Adaptive Behaviour Scales (VABS-II) | Change at 24 Months CA: Adaptive Behavior Composite | 7.8 Score on a scale | Standard Deviation 16.47 |
| Antecedent Standard of Care | Change From Baseline (6 Months CA) in Child Behavior as Assessed by Vineland Adaptive Behaviour Scales (VABS-II) | Change at 5 Years CA: Adaptive Behavior Composite | -3.2 Score on a scale | Standard Deviation 20.92 |
| Antecedent rhIGF-1/rhIGFBP-3 | Change From Baseline (6 Months CA) in Child Behavior as Assessed by Vineland Adaptive Behaviour Scales (VABS-II) | Change at 12 Months CA: Adaptive Behavior Composite | -1.5 Score on a scale | Standard Deviation 12.41 |
| Antecedent rhIGF-1/rhIGFBP-3 | Change From Baseline (6 Months CA) in Child Behavior as Assessed by Vineland Adaptive Behaviour Scales (VABS-II) | Change at 24 Months CA: Adaptive Behavior Composite | 0.8 Score on a scale | Standard Deviation 14.89 |
| Antecedent rhIGF-1/rhIGFBP-3 | Change From Baseline (6 Months CA) in Child Behavior as Assessed by Vineland Adaptive Behaviour Scales (VABS-II) | Change at 5 Years CA: Adaptive Behavior Composite | -3.9 Score on a scale | Standard Deviation 19.86 |
Secondary
Change From Baseline in Body Weight Z-score
Body weight collected using calibrated scales (type of scale was dependent upon participant's age). Measure recorded to the nearest 0.1 kilogram (kg). Z-score based on participants chronological age and utilizing World Health Organization child growth standards by adjusting age and sex- matched means and standard deviations (norm). Z-score is standard score that gives idea of how far from the mean a data point is. If Z-score is 0, it indicates that the data point's score is identical to the mean score. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean. Baseline is first assessment in the antecedent study (ROPP-2008-01 \[NCT01096784\]). Change from baseline in body weight Z-score at 6-month, 12-month, 24-month and 5-year CA were reported.
Time frame: Baseline, 6 Months CA, 12 Months CA, 24 Months CA and 5 Years CA
Population: Enrolled set included all participants for whom written informed consent was obtained for this long-term outcome study. Here, Overall Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure and Number analyzed signifies those participants who were evaluable at the specified time points.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Antecedent Standard of Care | Change From Baseline in Body Weight Z-score | Change at 5 Years CA | 5.80 Z-score | Standard Deviation 1.149 |
| Antecedent Standard of Care | Change From Baseline in Body Weight Z-score | Change at 12 Months CA | 5.08 Z-score | Standard Deviation 1.3 |
| Antecedent Standard of Care | Change From Baseline in Body Weight Z-score | Change at 6 Months CA | 4.40 Z-score | Standard Deviation 1.323 |
| Antecedent Standard of Care | Change From Baseline in Body Weight Z-score | Change at 24 Months CA | 5.17 Z-score | Standard Deviation 0.885 |
| Antecedent rhIGF-1/rhIGFBP-3 | Change From Baseline in Body Weight Z-score | Change at 6 Months CA | 4.30 Z-score | Standard Deviation 1.146 |
| Antecedent rhIGF-1/rhIGFBP-3 | Change From Baseline in Body Weight Z-score | Change at 5 Years CA | 5.60 Z-score | Standard Deviation 1.66 |
| Antecedent rhIGF-1/rhIGFBP-3 | Change From Baseline in Body Weight Z-score | Change at 24 Months CA | 4.99 Z-score | Standard Deviation 0.983 |
| Antecedent rhIGF-1/rhIGFBP-3 | Change From Baseline in Body Weight Z-score | Change at 12 Months CA | 4.88 Z-score | Standard Deviation 1.026 |
Secondary
Change From Baseline in Head Circumference Z-score
Head circumference measured for all participants using a lasso type, non-stretchable measuring tape such as the Lasso-o tape. Z-score calculated based on participants chronological age and utilizing World Health Organization child growth standards by adjusting age, sex matched means and standard deviations (norm).Z-score is standard score that gives idea of how far from mean a data point is. If a Z-score is 0, it indicates that data point's score is identical to mean score. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean. Baseline was defined as the first assessment in the antecedent study (ROPP-2008-01 \[NCT01096784\]). Change from baseline in Head Circumference Z-score at 6, 12 and 24-months reported.
Time frame: Baseline, 6 Months CA, 12 Months CA and 24 Months CA
Population: Enrolled set included all participants for whom written informed consent was obtained for this long-term outcome study. Here, Overall Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure and Number analyzed signifies those participants who were evaluable at the specified time points.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Antecedent Standard of Care | Change From Baseline in Head Circumference Z-score | Change at 6 Months CA | 7.61 Z-score | Standard Deviation 1.535 |
| Antecedent Standard of Care | Change From Baseline in Head Circumference Z-score | Change at 12 Months CA | 8.29 Z-score | Standard Deviation 1.472 |
| Antecedent Standard of Care | Change From Baseline in Head Circumference Z-score | Change at 24 Months CA | 8.44 Z-score | Standard Deviation 1.415 |
| Antecedent rhIGF-1/rhIGFBP-3 | Change From Baseline in Head Circumference Z-score | Change at 6 Months CA | 7.12 Z-score | Standard Deviation 1.576 |
| Antecedent rhIGF-1/rhIGFBP-3 | Change From Baseline in Head Circumference Z-score | Change at 12 Months CA | 7.83 Z-score | Standard Deviation 1.567 |
| Antecedent rhIGF-1/rhIGFBP-3 | Change From Baseline in Head Circumference Z-score | Change at 24 Months CA | 8.05 Z-score | Standard Deviation 1.692 |
Secondary
Change From Baseline in Height Z-score
Height was measured using Z-score. Z-score was calculated based on the participants chronological age and utilizing World Health Organization child growth standards by adjusting age and sex- matched means and standard deviations (norm). Z-score is a standard score that gives an idea of how far from the mean a data point is. If a Z-score is 0, it indicates that the data point's score is identical to the mean score. Lower numbers indicate values lower than the mean and higher numbers indicate values higher than the mean. Baseline was defined as the first assessment in the antecedent study (ROPP-2008-01 \[NCT01096784\]). Change from baseline in Height Z-score at 6-month, 12-month, 24-month and 5-year CA were reported.
Time frame: Baseline, 6 Months CA, 12 Month CA, 24 Months CA and 5 Years CA
Population: Enrolled set included all participants for whom written informed consent was obtained for this long-term outcome study. Here, Overall Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure and Number analyzed signifies those participants who were evaluable at the specified time points.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Antecedent Standard of Care | Change From Baseline in Height Z-score | Change at 6 Months CA | 6.36 Z-Score | Standard Deviation 1.711 |
| Antecedent Standard of Care | Change From Baseline in Height Z-score | Change at 12 Months CA | 6.79 Z-Score | Standard Deviation 1.747 |
| Antecedent Standard of Care | Change From Baseline in Height Z-score | Change at 24 Months CA | 7.30 Z-Score | Standard Deviation 2.029 |
| Antecedent Standard of Care | Change From Baseline in Height Z-score | Change at 5 Years CA | 7.84 Z-Score | Standard Deviation 1.856 |
| Antecedent rhIGF-1/rhIGFBP-3 | Change From Baseline in Height Z-score | Change at 5 Years CA | 8.17 Z-Score | Standard Deviation 1.505 |
| Antecedent rhIGF-1/rhIGFBP-3 | Change From Baseline in Height Z-score | Change at 6 Months CA | 6.19 Z-Score | Standard Deviation 1.215 |
| Antecedent rhIGF-1/rhIGFBP-3 | Change From Baseline in Height Z-score | Change at 24 Months CA | 7.78 Z-Score | Standard Deviation 1.376 |
| Antecedent rhIGF-1/rhIGFBP-3 | Change From Baseline in Height Z-score | Change at 12 Months CA | 7.25 Z-Score | Standard Deviation 1.155 |
Secondary
Child Behavior as Assessed by Attention-Deficit/Hyperactivity Disorder Rating Scale (ADHD-RS) Based on Total Score
The ADHD-RS measures the behaviors of children with Attention-Deficit/Hyperactivity Disorder (ADHD). It consisted of 18 items designed to reflect current symptomatology of ADHD based on DSM-IV criteria. Each item was scored on a 4-point scale ranging from 0 (reflecting no symptoms) to 3 (reflecting severe symptoms) with total scores ranging from 0 to 54. Higher scores represent greater severity of ADHD symptoms.
Time frame: At 5 Years CA
Population: Enrolled set included all participants for whom written informed consent was obtained for this long-term outcome study. Here, Overall Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Antecedent Standard of Care | Child Behavior as Assessed by Attention-Deficit/Hyperactivity Disorder Rating Scale (ADHD-RS) Based on Total Score | 11.9 Score on scale | Standard Deviation 9.3 |
| Antecedent rhIGF-1/rhIGFBP-3 | Child Behavior as Assessed by Attention-Deficit/Hyperactivity Disorder Rating Scale (ADHD-RS) Based on Total Score | 8.3 Score on scale | Standard Deviation 10.1 |
Secondary
Child Behavior as Assessed by Social Communication Questionnaire (SCQ)
The SCQ is a brief instrument evaluate communication skills and social functioning in children used for screening for autism or autism spectrum disorders (ASD). The SCQ has 40 dichotomous (yes, no) items, and each item scored 1 point for abnormal behavior and 0 point for absence of abnormal behavior/normal behavior. It yields total score ranging from 0 to 39, higher scores represent more social communication impairment. The first item, Is she/he now able to talk using short phrases or sentences?, is not scored, but determines if six items relating to abnormal language are assigned. Only verbal children (children with yes response to first question) are assigned the six items relating to abnormal language and can score a total of 0 to 39 points; non-verbal children (children with no response to first question) are not assigned six items in relation to abnormal language and score a total of 0 to 33 points where higher scores represent more social communication impairment.
Time frame: At 5 Years CA
Population: Enrolled set included all participants for whom written informed consent was obtained for this long-term outcome study. Here, Overall Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Antecedent Standard of Care | Child Behavior as Assessed by Social Communication Questionnaire (SCQ) | 5.9 Score on scale | Standard Deviation 6.8 |
| Antecedent rhIGF-1/rhIGFBP-3 | Child Behavior as Assessed by Social Communication Questionnaire (SCQ) | 5.2 Score on scale | Standard Deviation 7.88 |
Secondary
Cognitive Development as Assessed by Wechsler Preschool and Primary Scale of Intelligence (WPPSI-IV) Full Scale at 5 Years CA
The WPPSI-IV is a measure of general cognitive development in children that has components of both verbal and non-verbal tasks. It is applicable to preschoolers and young children aged 2 years +6 months to 7 years +7 months and is a direct assessment of a child's cognitive skills. The test framework of the WPPSI-IV is organized into five Primary Index sub scales: Verbal Comprehension, Visual Spatial, Fluid Reasoning, Working Memory, and Processing Speed. The Full Scale includes all of the sub scales at the Primary Index scale level, as well as any additional supplemental subtests that may be used to derive the Full Scale IQ. Composite scores are derived from the sum of sub scaled scores with a mean of 100 and a standard deviation of 15. Composite score for Full Scale IQ and subscales (Verbal Comprehension, Visual Spatial, Fluid Reasoning, Working Memory, and Processing Speed) ranges from 40 (extremely low) to 160 (very superior). Data for Full scale IQ was reported here.
Time frame: At 5 Years CA
Population: Enrolled set included all participants for whom written informed consent was obtained for this long-term outcome study. Here, Overall Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Antecedent Standard of Care | Cognitive Development as Assessed by Wechsler Preschool and Primary Scale of Intelligence (WPPSI-IV) Full Scale at 5 Years CA | 97.3 Scores on a scale | Standard Deviation 17.28 |
| Antecedent rhIGF-1/rhIGFBP-3 | Cognitive Development as Assessed by Wechsler Preschool and Primary Scale of Intelligence (WPPSI-IV) Full Scale at 5 Years CA | 91.6 Scores on a scale | Standard Deviation 14.17 |
Secondary
Health Care Resource Use (HCRU)
The total number of private office/hospital outpatient visits of health care resource at 3 Months CA, 6 Months CA, 12 Months CA, 24 Months CA, 3 Years CA, 4 Years CA and 5 Years CA were reported.
Time frame: At 3 Months CA, 6 Months CA, 12 Months CA, 24 Months CA, 3 Years CA, 4 Years CA and 5 Years CA
Population: Enrolled set included all participants for whom written informed consent was obtained for this long-term outcome study. Here, Overall Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure and Number analyzed signifies those participants who were evaluable at the specified time point.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Antecedent Standard of Care | Health Care Resource Use (HCRU) | At 12 Months CA: Total Health Care Resource Use | 25.0 Visits | Standard Deviation 23.81 |
| Antecedent Standard of Care | Health Care Resource Use (HCRU) | At 3 Years CA: Total Health Care Resource Use | 22.8 Visits | Standard Deviation 41.38 |
| Antecedent Standard of Care | Health Care Resource Use (HCRU) | At 6 Months CA: Total Health Care Resource Use | 15.5 Visits | Standard Deviation 16.06 |
| Antecedent Standard of Care | Health Care Resource Use (HCRU) | At 4 Years CA: Total Health Care Resource Use | 21.8 Visits | Standard Deviation 39 |
| Antecedent Standard of Care | Health Care Resource Use (HCRU) | At 24 Months CA: Total Health Care Resource Use | 20.1 Visits | Standard Deviation 31.95 |
| Antecedent Standard of Care | Health Care Resource Use (HCRU) | At 5 Years CA: Total Health Care Resource Use | 15.7 Visits | Standard Deviation 39.8 |
| Antecedent Standard of Care | Health Care Resource Use (HCRU) | At 3 Months CA: Total Health Care Resource Use | 9.8 Visits | Standard Deviation 6.48 |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Care Resource Use (HCRU) | At 5 Years CA: Total Health Care Resource Use | 32.7 Visits | Standard Deviation 51.22 |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Care Resource Use (HCRU) | At 3 Months CA: Total Health Care Resource Use | 8.3 Visits | Standard Deviation 7.67 |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Care Resource Use (HCRU) | At 6 Months CA: Total Health Care Resource Use | 10.3 Visits | Standard Deviation 9.4 |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Care Resource Use (HCRU) | At 12 Months CA: Total Health Care Resource Use | 22.5 Visits | Standard Deviation 33.95 |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Care Resource Use (HCRU) | At 24 Months CA: Total Health Care Resource Use | 30.0 Visits | Standard Deviation 40.92 |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Care Resource Use (HCRU) | At 3 Years CA: Total Health Care Resource Use | 34.8 Visits | Standard Deviation 55.25 |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Care Resource Use (HCRU) | At 4 Years CA: Total Health Care Resource Use | 41.2 Visits | Standard Deviation 69.93 |
Secondary
Health Care Resource Utilization (HCRU): Duration of Hospitalization
The participant's duration of hospitalizations at 3 Months CA, 6 Months CA, 12 Months CA, 24 Months CA, 3 Years CA, 4 Years CA and 5 Years CA were reported.
Time frame: At 3 Months CA, 6 Months CA, 12 Months CA , 24 Months CA, 3 Years CA, 4 Years CA and 5 Years CA
Population: Enrolled set included all participants for whom written informed consent was obtained for this long-term outcome study. Here, Overall Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure and Number analyzed signifies those participants who were evaluable at the specified categories.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Antecedent Standard of Care | Health Care Resource Utilization (HCRU): Duration of Hospitalization | At 3 Months CA | 12.9 Days | Standard Deviation 31.27 |
| Antecedent Standard of Care | Health Care Resource Utilization (HCRU): Duration of Hospitalization | At 6 Months CA | 6.4 Days | Standard Deviation 4.56 |
| Antecedent Standard of Care | Health Care Resource Utilization (HCRU): Duration of Hospitalization | At 12 Months CA | 7.2 Days | Standard Deviation 7.76 |
| Antecedent Standard of Care | Health Care Resource Utilization (HCRU): Duration of Hospitalization | At 24 Months CA | 4.1 Days | Standard Deviation 5.01 |
| Antecedent Standard of Care | Health Care Resource Utilization (HCRU): Duration of Hospitalization | At 3 Years CA | 4.3 Days | Standard Deviation 3.3 |
| Antecedent Standard of Care | Health Care Resource Utilization (HCRU): Duration of Hospitalization | At 4 Years CA | 3.5 Days | Standard Deviation 3.54 |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Care Resource Utilization (HCRU): Duration of Hospitalization | At 3 Years CA | 4.8 Days | Standard Deviation 6.87 |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Care Resource Utilization (HCRU): Duration of Hospitalization | At 3 Months CA | 5.5 Days | Standard Deviation 4.55 |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Care Resource Utilization (HCRU): Duration of Hospitalization | At 24 Months CA | 2.5 Days | Standard Deviation 1.73 |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Care Resource Utilization (HCRU): Duration of Hospitalization | At 6 Months CA | 7.4 Days | Standard Deviation 9.58 |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Care Resource Utilization (HCRU): Duration of Hospitalization | At 4 Years CA | 4.0 Days | Standard Deviation 3.61 |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Care Resource Utilization (HCRU): Duration of Hospitalization | At 12 Months CA | 9.0 Days | Standard Deviation 11.87 |
Secondary
Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support
Number of participants who required prescription eyeglasses and educational support at 3 Months CA, 6 Months CA, 12 Months CA, 24 Months CA, 3 Years CA, 4 Years CA and 5 Years CA were reported.
Time frame: At 3 Months CA, 6 Months CA, 12 Months CA, 24 Months CA, 3 Years CA, 4 Years CA and 5 Years CA
Population: Enrolled set included all participants for whom written informed consent was obtained for this long-term outcome study. Here, Overall Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure and Number analyzed signifies those participants who were evaluable at the specified categories.
| Arm | Measure | Group | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|---|
| Antecedent Standard of Care | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 5 Years CA: Used Prescription Eyeglasses | No | 16 Participants |
| Antecedent Standard of Care | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 24 Months CA: Used Prescription Eyeglasses | Yes | 1 Participants |
| Antecedent Standard of Care | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 3 Months CA: Received Educational Support | Yes | 0 Participants |
| Antecedent Standard of Care | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 5 Years CA: Received Educational Support | Yes | 3 Participants |
| Antecedent Standard of Care | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 3 Months CA: Received Educational Support | No | 32 Participants |
| Antecedent Standard of Care | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 24 Months CA: Used Prescription Eyeglasses | No | 24 Participants |
| Antecedent Standard of Care | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 6 Months CA: Received Educational Support | Yes | 0 Participants |
| Antecedent Standard of Care | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 6 Months CA: Used Prescription Eyeglasses | No | 28 Participants |
| Antecedent Standard of Care | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 6 Months CA: Received Educational Support | No | 28 Participants |
| Antecedent Standard of Care | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 3 Years CA: Used Prescription Eyeglasses | Yes | 3 Participants |
| Antecedent Standard of Care | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 12 Months CA: Received Educational Support | Yes | 0 Participants |
| Antecedent Standard of Care | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 3 Months CA: Used Prescription Eyeglasses | No | 32 Participants |
| Antecedent Standard of Care | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 12 Months CA: Received Educational Support | No | 26 Participants |
| Antecedent Standard of Care | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 3 Years CA: Used Prescription Eyeglasses | No | 21 Participants |
| Antecedent Standard of Care | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 24 Months CA: Received Educational Support | Yes | 0 Participants |
| Antecedent Standard of Care | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 12 Months CA: Used Prescription Eyeglasses | Yes | 0 Participants |
| Antecedent Standard of Care | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 24 Months CA: Received Educational Support | No | 25 Participants |
| Antecedent Standard of Care | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 4 Years CA: Used Prescription Eyeglasses | Yes | 2 Participants |
| Antecedent Standard of Care | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 3 Years CA: Received Educational Support | Yes | 1 Participants |
| Antecedent Standard of Care | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 3 Months CA: Used Prescription Eyeglasses | Yes | 0 Participants |
| Antecedent Standard of Care | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 3 Years CA: Received Educational Support | No | 23 Participants |
| Antecedent Standard of Care | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 4 Years CA: Used Prescription Eyeglasses | No | 21 Participants |
| Antecedent Standard of Care | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 4 Years CA: Received Educational Support | Yes | 5 Participants |
| Antecedent Standard of Care | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 12 Months CA: Used Prescription Eyeglasses | No | 26 Participants |
| Antecedent Standard of Care | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 4 Years CA: Received Educational Support | No | 18 Participants |
| Antecedent Standard of Care | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 5 Years CA: Used Prescription Eyeglasses | Yes | 3 Participants |
| Antecedent Standard of Care | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 5 Years CA: Received Educational Support | No | 16 Participants |
| Antecedent Standard of Care | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 6 Months CA: Used Prescription Eyeglasses | Yes | 0 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 5 Years CA: Received Educational Support | No | 15 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 3 Months CA: Used Prescription Eyeglasses | Yes | 0 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 3 Months CA: Used Prescription Eyeglasses | No | 39 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 6 Months CA: Used Prescription Eyeglasses | Yes | 0 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 6 Months CA: Used Prescription Eyeglasses | No | 39 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 12 Months CA: Used Prescription Eyeglasses | Yes | 1 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 12 Months CA: Used Prescription Eyeglasses | No | 35 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 24 Months CA: Used Prescription Eyeglasses | Yes | 2 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 24 Months CA: Used Prescription Eyeglasses | No | 34 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 3 Years CA: Used Prescription Eyeglasses | Yes | 7 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 3 Years CA: Used Prescription Eyeglasses | No | 26 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 4 Years CA: Used Prescription Eyeglasses | Yes | 5 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 4 Years CA: Used Prescription Eyeglasses | No | 22 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 5 Years CA: Used Prescription Eyeglasses | Yes | 4 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 5 Years CA: Used Prescription Eyeglasses | No | 16 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 3 Months CA: Received Educational Support | Yes | 0 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 3 Months CA: Received Educational Support | No | 39 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 6 Months CA: Received Educational Support | Yes | 1 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 6 Months CA: Received Educational Support | No | 38 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 12 Months CA: Received Educational Support | Yes | 1 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 12 Months CA: Received Educational Support | No | 35 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 24 Months CA: Received Educational Support | Yes | 0 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 24 Months CA: Received Educational Support | No | 36 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 3 Years CA: Received Educational Support | Yes | 4 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 3 Years CA: Received Educational Support | No | 29 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 4 Years CA: Received Educational Support | Yes | 3 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 5 Years CA: Received Educational Support | Yes | 5 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Care Resource Utilization (HCRU): Number of Participants Who Required Prescription Eyeglasses and Educational Support | At 4 Years CA: Received Educational Support | No | 24 Participants |
Secondary
Health Care Resource Utilization (HCRU): Number of Visits to Emergency Department
Number of visits to emergency department at 3 Months CA, 6 Months CA, 12 Months CA, 24 Months CA, 3 Years CA, 4 Years CA and 5 Years CA were reported.
Time frame: At 3 Months CA, 6 Months CA, 12 Months CA , 24 Months CA, 3 Years CA, 4 Years CA and 5 Years CA
Population: Enrolled set included all participants for whom written informed consent was obtained for this long-term outcome study. Here, Overall Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure and Number analyzed signifies those participants who were evaluable at the specified categories.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Antecedent Standard of Care | Health Care Resource Utilization (HCRU): Number of Visits to Emergency Department | At 12 Months CA: Emergency Department Visits | 1.6 Visits | Standard Deviation 0.73 |
| Antecedent Standard of Care | Health Care Resource Utilization (HCRU): Number of Visits to Emergency Department | At 3 Years CA: Emergency Department Visits | 2.5 Visits | Standard Deviation 2.38 |
| Antecedent Standard of Care | Health Care Resource Utilization (HCRU): Number of Visits to Emergency Department | At 6 Months CA: Emergency Department Visits | 2.2 Visits | Standard Deviation 2.17 |
| Antecedent Standard of Care | Health Care Resource Utilization (HCRU): Number of Visits to Emergency Department | At 4 Years CA: Emergency Department Visits | 1.9 Visits | Standard Deviation 1.46 |
| Antecedent Standard of Care | Health Care Resource Utilization (HCRU): Number of Visits to Emergency Department | At 24 Months CA: Emergency Department Visits | 2.1 Visits | Standard Deviation 1.39 |
| Antecedent Standard of Care | Health Care Resource Utilization (HCRU): Number of Visits to Emergency Department | At 5 Years CA: Emergency Department Visits | 1.7 Visits | Standard Deviation 0.58 |
| Antecedent Standard of Care | Health Care Resource Utilization (HCRU): Number of Visits to Emergency Department | At 3 Months CA: Emergency Department Visits | 2.5 Visits | Standard Deviation 1.87 |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Care Resource Utilization (HCRU): Number of Visits to Emergency Department | At 5 Years CA: Emergency Department Visits | 1 Visits | — |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Care Resource Utilization (HCRU): Number of Visits to Emergency Department | At 3 Months CA: Emergency Department Visits | 1.6 Visits | Standard Deviation 1.13 |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Care Resource Utilization (HCRU): Number of Visits to Emergency Department | At 6 Months CA: Emergency Department Visits | 1.3 Visits | Standard Deviation 0.67 |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Care Resource Utilization (HCRU): Number of Visits to Emergency Department | At 12 Months CA: Emergency Department Visits | 2.2 Visits | Standard Deviation 1.14 |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Care Resource Utilization (HCRU): Number of Visits to Emergency Department | At 24 Months CA: Emergency Department Visits | 1.4 Visits | Standard Deviation 0.61 |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Care Resource Utilization (HCRU): Number of Visits to Emergency Department | At 3 Years CA: Emergency Department Visits | 1.6 Visits | Standard Deviation 0.84 |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Care Resource Utilization (HCRU): Number of Visits to Emergency Department | At 4 Years CA: Emergency Department Visits | 1.7 Visits | Standard Deviation 0.82 |
Secondary
Health Status Measured by the Health Utilities Index (HUI) Mark 2 and 3
The HUI is a family of generic health profiles and preference-based systems used for measuring health status, reporting HRQoL, and producing utility scores. The HUI2 score includes 6 attributes: Sensation, Mobility, Cognition, Self-Care, Emotion, and Pain with up to 5 levels of severity (higher numbers indicate worse level of severity). The HUI3 score includes 8 attributes: Vision, Hearing, Speech, Cognition, Ambulation, Dexterity, Emotion, and Pain with up to 6 levels to indicate the severity (with higher numbers indicating worse levels). HUI2/3 single-attribute scores of morbidities are defined on a scale such the worst level has a score of 0.00 and the best level has a score of 1.00. HUI mark 2 and 3 Overall utility score was reported.
Time frame: At 5 Years CA
Population: Enrolled set included all participants for whom written informed consent was obtained for this long-term outcome study. Here, Overall Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Antecedent Standard of Care | Health Status Measured by the Health Utilities Index (HUI) Mark 2 and 3 | HUI2 Overall Utility Score | 0.929 Score on scale | Standard Deviation 0.1246 |
| Antecedent Standard of Care | Health Status Measured by the Health Utilities Index (HUI) Mark 2 and 3 | HUI3 Overall Utility Score | 0.893 Score on scale | Standard Deviation 0.196 |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Status Measured by the Health Utilities Index (HUI) Mark 2 and 3 | HUI2 Overall Utility Score | 0.867 Score on scale | Standard Deviation 0.2115 |
| Antecedent rhIGF-1/rhIGFBP-3 | Health Status Measured by the Health Utilities Index (HUI) Mark 2 and 3 | HUI3 Overall Utility Score | 0.852 Score on scale | Standard Deviation 0.2665 |
Secondary
Number of Participants Diagnosed With Neurological Examination for Cerebral Palsy (CP) by Neurological Examination at 24 Months CA
A comprehensive neurological examination for the diagnosis of CP was conducted. The Amiel-Tison neurological examination framework was utilized for this assessment and it was conducted by trained medical professionals. Number of Participants diagnosed with CP by neurological examination at 24 Months CA were reported.
Time frame: At 24 Months CA
Population: Enrolled set included all participants for whom written informed consent was obtained for this long-term outcome study. Here, Overall Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Antecedent Standard of Care | Number of Participants Diagnosed With Neurological Examination for Cerebral Palsy (CP) by Neurological Examination at 24 Months CA | 1 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants Diagnosed With Neurological Examination for Cerebral Palsy (CP) by Neurological Examination at 24 Months CA | 5 Participants |
Secondary
Number of Participants With Abnormal Physical Examination
Physical examination includes general appearance; head and neck, eyes, ears, nose and throat; chest and lungs; endocrine, cardiovascular system, abdomen, genitourinary, skin and musculoskeletal system.
Time frame: From start of study drug administration up to end of study (up to 6.5 years)
Population: Safety Set included all participants in the Enrolled Set who have safety follow-up data in this long-term outcome study.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Antecedent Standard of Care | Number of Participants With Abnormal Physical Examination | 13 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Abnormal Physical Examination | 18 Participants |
Secondary
Number of Participants With Child Behaviour as Assessed by Child Behaviour Checklist (CBCL) Based on T-Score Clinical Categories
The CBCL (1 ½ to 5) was parent-reported outcome measure used to assess behavioral, emotional, and social functioning of toddlers and preschool children aged 18-60 months. It is composed of 99 items rated on Likert scale and includes 7 syndrome scales under 2 domains (Internalizing and Externalizing Problems): Internalizing includes syndromes of Emotionally Reactive, Anxious/Depressed, Somatic Complaints, and Withdrawn. Externalizing includes syndromes of Aggressive Behavior and Attention Problems. The 2 domains of all 7 scales were combined to form total score range of 23 to 100. For each question, raw scores of all subscales are converted to standardized T-scores. Higher T score indicates more behavior problems. For Internalization, Externalization and Total Behavior Problem, T-scores of less than 60 are considered non-clinical, 60-63 are borderline, and 64 or more are considered clinical.
Time frame: At 24 Months CA and 5 Years CA
Population: Enrolled set included all participants for whom written informed consent was obtained for this long-term outcome study. Here, Overall Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure and Number analyzed signifies those participants who were evaluable at the specified time point.
| Arm | Measure | Group | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|---|
| Antecedent Standard of Care | Number of Participants With Child Behaviour as Assessed by Child Behaviour Checklist (CBCL) Based on T-Score Clinical Categories | At 5 Years CA: Internalizing | Clinical | 3 Participants |
| Antecedent Standard of Care | Number of Participants With Child Behaviour as Assessed by Child Behaviour Checklist (CBCL) Based on T-Score Clinical Categories | At 5 Years CA: Externalizing | Borderline | 0 Participants |
| Antecedent Standard of Care | Number of Participants With Child Behaviour as Assessed by Child Behaviour Checklist (CBCL) Based on T-Score Clinical Categories | At 5 Years CA: Internalizing | Non-Clinical | 15 Participants |
| Antecedent Standard of Care | Number of Participants With Child Behaviour as Assessed by Child Behaviour Checklist (CBCL) Based on T-Score Clinical Categories | At 5 Years CA: Externalizing | Clinical | 3 Participants |
| Antecedent Standard of Care | Number of Participants With Child Behaviour as Assessed by Child Behaviour Checklist (CBCL) Based on T-Score Clinical Categories | At 24 Months CA: Externalizing | Non-Clinical | 21 Participants |
| Antecedent Standard of Care | Number of Participants With Child Behaviour as Assessed by Child Behaviour Checklist (CBCL) Based on T-Score Clinical Categories | At 24 Months CA: Total Behavior Problems | Non-Clinical | 22 Participants |
| Antecedent Standard of Care | Number of Participants With Child Behaviour as Assessed by Child Behaviour Checklist (CBCL) Based on T-Score Clinical Categories | At 24 Months CA: Internalizing | Borderline | 0 Participants |
| Antecedent Standard of Care | Number of Participants With Child Behaviour as Assessed by Child Behaviour Checklist (CBCL) Based on T-Score Clinical Categories | At 24 Months CA: Total Behavior Problems | Borderline | 1 Participants |
| Antecedent Standard of Care | Number of Participants With Child Behaviour as Assessed by Child Behaviour Checklist (CBCL) Based on T-Score Clinical Categories | At 24 Months CA: Externalizing | Borderline | 1 Participants |
| Antecedent Standard of Care | Number of Participants With Child Behaviour as Assessed by Child Behaviour Checklist (CBCL) Based on T-Score Clinical Categories | At 24 Months CA: Total Behavior Problems | Clinical | 0 Participants |
| Antecedent Standard of Care | Number of Participants With Child Behaviour as Assessed by Child Behaviour Checklist (CBCL) Based on T-Score Clinical Categories | At 5 Years CA: Internalizing | Borderline | 1 Participants |
| Antecedent Standard of Care | Number of Participants With Child Behaviour as Assessed by Child Behaviour Checklist (CBCL) Based on T-Score Clinical Categories | At 5 Years CA: Total Behavior Problems | Non-Clinical | 14 Participants |
| Antecedent Standard of Care | Number of Participants With Child Behaviour as Assessed by Child Behaviour Checklist (CBCL) Based on T-Score Clinical Categories | At 24 Months CA: Externalizing | Clinical | 1 Participants |
| Antecedent Standard of Care | Number of Participants With Child Behaviour as Assessed by Child Behaviour Checklist (CBCL) Based on T-Score Clinical Categories | At 5 Years CA: Total Behavior Problems | Borderline | 3 Participants |
| Antecedent Standard of Care | Number of Participants With Child Behaviour as Assessed by Child Behaviour Checklist (CBCL) Based on T-Score Clinical Categories | At 24 Months CA: Internalizing | Clinical | 0 Participants |
| Antecedent Standard of Care | Number of Participants With Child Behaviour as Assessed by Child Behaviour Checklist (CBCL) Based on T-Score Clinical Categories | At 5 Years CA: Total Behavior Problems | Clinical | 2 Participants |
| Antecedent Standard of Care | Number of Participants With Child Behaviour as Assessed by Child Behaviour Checklist (CBCL) Based on T-Score Clinical Categories | At 5 Years CA: Externalizing | Non-Clinical | 16 Participants |
| Antecedent Standard of Care | Number of Participants With Child Behaviour as Assessed by Child Behaviour Checklist (CBCL) Based on T-Score Clinical Categories | At 24 Months CA: Internalizing | Non-Clinical | 23 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Child Behaviour as Assessed by Child Behaviour Checklist (CBCL) Based on T-Score Clinical Categories | At 24 Months CA: Internalizing | Clinical | 2 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Child Behaviour as Assessed by Child Behaviour Checklist (CBCL) Based on T-Score Clinical Categories | At 24 Months CA: Internalizing | Non-Clinical | 32 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Child Behaviour as Assessed by Child Behaviour Checklist (CBCL) Based on T-Score Clinical Categories | At 24 Months CA: Internalizing | Borderline | 0 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Child Behaviour as Assessed by Child Behaviour Checklist (CBCL) Based on T-Score Clinical Categories | At 5 Years CA: Internalizing | Non-Clinical | 15 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Child Behaviour as Assessed by Child Behaviour Checklist (CBCL) Based on T-Score Clinical Categories | At 5 Years CA: Internalizing | Borderline | 2 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Child Behaviour as Assessed by Child Behaviour Checklist (CBCL) Based on T-Score Clinical Categories | At 5 Years CA: Internalizing | Clinical | 3 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Child Behaviour as Assessed by Child Behaviour Checklist (CBCL) Based on T-Score Clinical Categories | At 24 Months CA: Externalizing | Non-Clinical | 32 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Child Behaviour as Assessed by Child Behaviour Checklist (CBCL) Based on T-Score Clinical Categories | At 24 Months CA: Externalizing | Borderline | 0 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Child Behaviour as Assessed by Child Behaviour Checklist (CBCL) Based on T-Score Clinical Categories | At 24 Months CA: Externalizing | Clinical | 2 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Child Behaviour as Assessed by Child Behaviour Checklist (CBCL) Based on T-Score Clinical Categories | At 5 Years CA: Externalizing | Non-Clinical | 17 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Child Behaviour as Assessed by Child Behaviour Checklist (CBCL) Based on T-Score Clinical Categories | At 5 Years CA: Externalizing | Borderline | 2 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Child Behaviour as Assessed by Child Behaviour Checklist (CBCL) Based on T-Score Clinical Categories | At 5 Years CA: Externalizing | Clinical | 1 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Child Behaviour as Assessed by Child Behaviour Checklist (CBCL) Based on T-Score Clinical Categories | At 24 Months CA: Total Behavior Problems | Non-Clinical | 31 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Child Behaviour as Assessed by Child Behaviour Checklist (CBCL) Based on T-Score Clinical Categories | At 24 Months CA: Total Behavior Problems | Borderline | 3 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Child Behaviour as Assessed by Child Behaviour Checklist (CBCL) Based on T-Score Clinical Categories | At 24 Months CA: Total Behavior Problems | Clinical | 0 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Child Behaviour as Assessed by Child Behaviour Checklist (CBCL) Based on T-Score Clinical Categories | At 5 Years CA: Total Behavior Problems | Non-Clinical | 17 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Child Behaviour as Assessed by Child Behaviour Checklist (CBCL) Based on T-Score Clinical Categories | At 5 Years CA: Total Behavior Problems | Borderline | 2 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Child Behaviour as Assessed by Child Behaviour Checklist (CBCL) Based on T-Score Clinical Categories | At 5 Years CA: Total Behavior Problems | Clinical | 1 Participants |
Secondary
Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS)
HSCS-PS was a validated instrument used via parent proxy within age 2.5 - 5 years. The instrument is composed of 12 domains (Vision, Hearing, Speech, Mobility, Dexterity, Self-care, Emotion, Learn/Remember, Think/Problem Solve, Pain, General Health, Behavior) with up to 6 levels (0-5). The 12 dimensions grouped into 4 categories: neurosensory (vision and hearing), motor (mobility, dexterity, and self-care), learning/remembering (speech, learn/remember, think/problem solve), and quality of life (emotion, pain, general health, behavior). For each category, data were recorded into following levels: no problem (scoring 0 on any attribute); a mild problem (scoring 1 on a scale of 0 to 3, or 1 to 2 on a scale of 0 to 5 for any attribute);moderate/severe problem (scoring \> 1 on a scale of 0 to 3, or \>2 on a scale of 0 to 5 for any attribute). Higher numbers indicating better child's health status.
Time frame: At 24 Months CA, 3 Years CA, 4 Years CA and 5 Years CA
Population: Enrolled set included all participants for whom written informed consent was obtained for this long-term outcome study. Here, Overall Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure and Number analyzed signifies those participants who were evaluable at the specified time points.
| Arm | Measure | Group | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|---|
| Antecedent Standard of Care | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 24 Months CA: Neurosensory | Mild | 3 Participants |
| Antecedent Standard of Care | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 4 Years CA: Neurosensory | No Problem | 21 Participants |
| Antecedent Standard of Care | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 3 Years CA: Neurosensory | No Problem | 20 Participants |
| Antecedent Standard of Care | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 4 Years CA: Neurosensory | Mild | 2 Participants |
| Antecedent Standard of Care | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 24 Months CA: Learning/Remembering | No Problem | 10 Participants |
| Antecedent Standard of Care | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 4 Years CA: Neurosensory | Moderate/Severe | 0 Participants |
| Antecedent Standard of Care | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 3 Years CA: Neurosensory | Mild | 2 Participants |
| Antecedent Standard of Care | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 4 Years CA: Motor | No Problem | 16 Participants |
| Antecedent Standard of Care | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 4 Years CA: Motor | Mild | 5 Participants |
| Antecedent Standard of Care | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 24 Months CA: Motor | No Problem | 19 Participants |
| Antecedent Standard of Care | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 4 Years CA: Motor | Moderate/Severe | 2 Participants |
| Antecedent Standard of Care | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 3 Years CA: Neurosensory | Moderate/Severe | 2 Participants |
| Antecedent Standard of Care | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 4 Years CA: Learning/Remembering | No Problem | 16 Participants |
| Antecedent Standard of Care | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 24 Months CA: Learning/Remembering | Mild | 6 Participants |
| Antecedent Standard of Care | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 4 Years CA: Learning/Remembering | Mild | 4 Participants |
| Antecedent Standard of Care | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 3 Years CA: Motor | No Problem | 16 Participants |
| Antecedent Standard of Care | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 4 Years CA: Learning/Remembering | Moderate/Severe | 3 Participants |
| Antecedent Standard of Care | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 24 Months CA: Neurosensory | No Problem | 21 Participants |
| Antecedent Standard of Care | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 4 Years CA: Quality of Life | No Problem | 13 Participants |
| Antecedent Standard of Care | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 3 Years CA: Motor | Mild | 4 Participants |
| Antecedent Standard of Care | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 4 Years CA: Quality of Life | Mild | 8 Participants |
| Antecedent Standard of Care | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 24 Months CA: Learning/Remembering | Moderate/Severe | 8 Participants |
| Antecedent Standard of Care | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 4 Years CA: Quality of Life | Moderate/Severe | 2 Participants |
| Antecedent Standard of Care | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 3 Years CA: Motor | Moderate/Severe | 4 Participants |
| Antecedent Standard of Care | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 5 Years CA: Neurosensory | No Problem | 16 Participants |
| Antecedent Standard of Care | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 24 Months CA: Motor | Mild | 3 Participants |
| Antecedent Standard of Care | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 5 Years CA: Neurosensory | Mild | 3 Participants |
| Antecedent Standard of Care | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 3 Years CA: Learning/Remembering | No Problem | 13 Participants |
| Antecedent Standard of Care | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 5 Years CA: Neurosensory | Moderate/Severe | 0 Participants |
| Antecedent Standard of Care | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 24 Months CA: Quality of Life | No Problem | 11 Participants |
| Antecedent Standard of Care | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 5 Years CA: Motor | No Problem | 13 Participants |
| Antecedent Standard of Care | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 3 Years CA: Learning/Remembering | Mild | 6 Participants |
| Antecedent Standard of Care | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 5 Years CA: Motor | Mild | 5 Participants |
| Antecedent Standard of Care | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 24 Months CA: Neurosensory | Moderate/Severe | 0 Participants |
| Antecedent Standard of Care | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 5 Years CA: Motor | Moderate/Severe | 1 Participants |
| Antecedent Standard of Care | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 3 Years CA: Learning/Remembering | Moderate/Severe | 5 Participants |
| Antecedent Standard of Care | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 5 Years CA: Learning/Remembering | No Problem | 10 Participants |
| Antecedent Standard of Care | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 24 Months CA: Quality of Life | Mild | 11 Participants |
| Antecedent Standard of Care | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 5 Years CA: Learning/Remembering | Mild | 7 Participants |
| Antecedent Standard of Care | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 3 Years CA: Quality of Life | No Problem | 13 Participants |
| Antecedent Standard of Care | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 5 Years CA: Learning/Remembering | Moderate/Severe | 2 Participants |
| Antecedent Standard of Care | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 24 Months CA: Motor | Moderate/Severe | 2 Participants |
| Antecedent Standard of Care | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 5 Years CA: Quality of Life | No Problem | 11 Participants |
| Antecedent Standard of Care | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 3 Years CA: Quality of Life | Mild | 9 Participants |
| Antecedent Standard of Care | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 5 Years CA: Quality of Life | Mild | 8 Participants |
| Antecedent Standard of Care | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 24 Months CA: Quality of Life | Moderate/Severe | 2 Participants |
| Antecedent Standard of Care | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 5 Years CA: Quality of Life | Moderate/Severe | 0 Participants |
| Antecedent Standard of Care | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 3 Years CA: Quality of Life | Moderate/Severe | 2 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 5 Years CA: Quality of Life | Moderate/Severe | 1 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 24 Months CA: Neurosensory | No Problem | 30 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 24 Months CA: Neurosensory | Mild | 4 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 24 Months CA: Neurosensory | Moderate/Severe | 1 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 24 Months CA: Motor | No Problem | 21 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 24 Months CA: Motor | Mild | 9 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 24 Months CA: Motor | Moderate/Severe | 5 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 24 Months CA: Learning/Remembering | No Problem | 10 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 24 Months CA: Learning/Remembering | Mild | 13 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 24 Months CA: Learning/Remembering | Moderate/Severe | 12 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 24 Months CA: Quality of Life | No Problem | 21 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 24 Months CA: Quality of Life | Mild | 11 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 24 Months CA: Quality of Life | Moderate/Severe | 3 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 3 Years CA: Neurosensory | No Problem | 28 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 3 Years CA: Neurosensory | Mild | 3 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 3 Years CA: Neurosensory | Moderate/Severe | 2 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 3 Years CA: Motor | No Problem | 16 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 3 Years CA: Motor | Mild | 13 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 3 Years CA: Motor | Moderate/Severe | 4 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 3 Years CA: Learning/Remembering | No Problem | 17 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 3 Years CA: Learning/Remembering | Mild | 11 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 3 Years CA: Learning/Remembering | Moderate/Severe | 5 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 3 Years CA: Quality of Life | No Problem | 21 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 3 Years CA: Quality of Life | Mild | 8 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 3 Years CA: Quality of Life | Moderate/Severe | 4 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 4 Years CA: Neurosensory | No Problem | 23 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 4 Years CA: Neurosensory | Mild | 4 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 4 Years CA: Neurosensory | Moderate/Severe | 0 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 4 Years CA: Motor | Mild | 8 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 4 Years CA: Motor | Moderate/Severe | 3 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 4 Years CA: Learning/Remembering | No Problem | 12 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 4 Years CA: Learning/Remembering | Mild | 11 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 4 Years CA: Learning/Remembering | Moderate/Severe | 4 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 4 Years CA: Quality of Life | No Problem | 17 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 4 Years CA: Quality of Life | Mild | 9 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 4 Years CA: Quality of Life | Moderate/Severe | 1 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 5 Years CA: Neurosensory | No Problem | 17 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 5 Years CA: Neurosensory | Mild | 3 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 5 Years CA: Neurosensory | Moderate/Severe | 0 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 5 Years CA: Motor | No Problem | 14 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 5 Years CA: Motor | Mild | 5 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 5 Years CA: Motor | Moderate/Severe | 1 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 5 Years CA: Learning/Remembering | No Problem | 12 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 5 Years CA: Learning/Remembering | Mild | 7 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 5 Years CA: Learning/Remembering | Moderate/Severe | 1 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 5 Years CA: Quality of Life | No Problem | 15 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 5 Years CA: Quality of Life | Mild | 4 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Health Status Measured by the Health Status Classification System-Preschool (HSCS-PS) | At 4 Years CA: Motor | No Problem | 16 Participants |
Secondary
Number of Participants With Normal and Abnormal Hearing Screening Status
Number of participants with any hearing status (normal and abnormal) were reported.
Time frame: At 6 Months CA and 5 Years CA
Population: Enrolled set included all participants for whom written informed consent was obtained for this long-term outcome study. Here, Overall Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure and Number analyzed signifies those participants who were evaluable at the specified time point.
| Arm | Measure | Group | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|---|
| Antecedent Standard of Care | Number of Participants With Normal and Abnormal Hearing Screening Status | At 6 Months CA | Normal Hearing | 20 Participants |
| Antecedent Standard of Care | Number of Participants With Normal and Abnormal Hearing Screening Status | At 6 Months CA | Abnormal Hearing | 1 Participants |
| Antecedent Standard of Care | Number of Participants With Normal and Abnormal Hearing Screening Status | At 5 Years CA | Normal Hearing | 15 Participants |
| Antecedent Standard of Care | Number of Participants With Normal and Abnormal Hearing Screening Status | At 5 Years CA | Abnormal Hearing | 2 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Normal and Abnormal Hearing Screening Status | At 5 Years CA | Abnormal Hearing | 1 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Normal and Abnormal Hearing Screening Status | At 6 Months CA | Normal Hearing | 24 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Normal and Abnormal Hearing Screening Status | At 5 Years CA | Normal Hearing | 20 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Normal and Abnormal Hearing Screening Status | At 6 Months CA | Abnormal Hearing | 4 Participants |
Secondary
Number of Participants With Pulmonary Morbidity
Pulmonary morbidity was assessed with questions related to family history and smoking status as well as diagnosis of select pulmonary symptoms, conditions and related hospitalizations. Anyone living in the same home with participant smoke, Participants had asthma, wheezing, bronchopulmonary dysplasia (BPD) exacerbation or flare-up, Participants had bronchiolitis, bronchitis, or pneumonia diagnosed, Participants had to use oxygen at home, Participants had to visit emergency room or urgent care for respiratory problem, and Participants had to stay in a hospital overnight for respiratory problem were reported.
Time frame: At 6 Months CA, 12 Months CA and 24 Months CA
Population: Enrolled set included all participants for whom written informed consent was obtained for this long-term outcome study. Here, Overall Number of Participants Analyzed signifies those participants who were evaluable for this outcome measure and Number analyzed signifies those participants who were evaluable at the specified time point.
| Arm | Measure | Group | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|---|
| Antecedent Standard of Care | Number of Participants With Pulmonary Morbidity | At 24 Months CA: Had to visit emergency room or urgent care for respiratory problem | No | 20 Participants |
| Antecedent Standard of Care | Number of Participants With Pulmonary Morbidity | At 6 Months CA: Anyone living in the same home with participant smoke | Yes | 6 Participants |
| Antecedent Standard of Care | Number of Participants With Pulmonary Morbidity | At 6 Months CA: Anyone living in the same home with participant smoke | No | 22 Participants |
| Antecedent Standard of Care | Number of Participants With Pulmonary Morbidity | At 6 Months CA: Had asthma, wheezing, BPD exacerbation or flare-up | Yes | 8 Participants |
| Antecedent Standard of Care | Number of Participants With Pulmonary Morbidity | At 6 Months CA: Had asthma, wheezing, BPD exacerbation or flare-up | No | 20 Participants |
| Antecedent Standard of Care | Number of Participants With Pulmonary Morbidity | At 6 Months CA: Had bronchiolitis, bronchitis, or pneumonia diagnosed | Yes | 4 Participants |
| Antecedent Standard of Care | Number of Participants With Pulmonary Morbidity | At 6 Months CA: Had bronchiolitis, bronchitis, or pneumonia diagnosed | No | 24 Participants |
| Antecedent Standard of Care | Number of Participants With Pulmonary Morbidity | At 6 Months CA: Had to use oxygen at home | Yes | 8 Participants |
| Antecedent Standard of Care | Number of Participants With Pulmonary Morbidity | At 6 Months CA: Had to use oxygen at home | No | 20 Participants |
| Antecedent Standard of Care | Number of Participants With Pulmonary Morbidity | At 6 Months CA: Had to visit emergency room or urgent care for respiratory problem | Yes | 5 Participants |
| Antecedent Standard of Care | Number of Participants With Pulmonary Morbidity | At 6 Months CA: Had to visit emergency room or urgent care for respiratory problem | No | 23 Participants |
| Antecedent Standard of Care | Number of Participants With Pulmonary Morbidity | At 6 Months CA: Had to stay in a hospital overnight for respiratory problem | Yes | 5 Participants |
| Antecedent Standard of Care | Number of Participants With Pulmonary Morbidity | At 6 Months CA: Had to stay in a hospital overnight for respiratory problem | No | 23 Participants |
| Antecedent Standard of Care | Number of Participants With Pulmonary Morbidity | At 12 Months CA: Anyone living in the same home with participant smoke | Yes | 3 Participants |
| Antecedent Standard of Care | Number of Participants With Pulmonary Morbidity | At 12 Months CA: Anyone living in the same home with participant smoke | No | 23 Participants |
| Antecedent Standard of Care | Number of Participants With Pulmonary Morbidity | At 12 Months CA: Had asthma, wheezing, BPD exacerbation or flare-up | Yes | 4 Participants |
| Antecedent Standard of Care | Number of Participants With Pulmonary Morbidity | At 12 Months CA: Had asthma, wheezing, BPD exacerbation or flare-up | No | 22 Participants |
| Antecedent Standard of Care | Number of Participants With Pulmonary Morbidity | At 12 Months CA: Had bronchiolitis, bronchitis, or pneumonia diagnosed | Yes | 1 Participants |
| Antecedent Standard of Care | Number of Participants With Pulmonary Morbidity | At 12 Months CA: Had bronchiolitis, bronchitis, or pneumonia diagnosed | No | 25 Participants |
| Antecedent Standard of Care | Number of Participants With Pulmonary Morbidity | At 12 Months CA: Had to use oxygen at home | Yes | 2 Participants |
| Antecedent Standard of Care | Number of Participants With Pulmonary Morbidity | At 12 Months CA: Had to use oxygen at home | No | 24 Participants |
| Antecedent Standard of Care | Number of Participants With Pulmonary Morbidity | At 12 Months CA: Had to visit emergency room or urgent care for respiratory problem | Yes | 3 Participants |
| Antecedent Standard of Care | Number of Participants With Pulmonary Morbidity | At 24 Months CA: Had bronchiolitis, bronchitis, or pneumonia diagnosed | Yes | 2 Participants |
| Antecedent Standard of Care | Number of Participants With Pulmonary Morbidity | At 24 Months CA: Had bronchiolitis, bronchitis, or pneumonia diagnosed | No | 23 Participants |
| Antecedent Standard of Care | Number of Participants With Pulmonary Morbidity | At 24 Months CA: Had to use oxygen at home | Yes | 1 Participants |
| Antecedent Standard of Care | Number of Participants With Pulmonary Morbidity | At 24 Months CA: Had to use oxygen at home | No | 24 Participants |
| Antecedent Standard of Care | Number of Participants With Pulmonary Morbidity | At 24 Months CA: Had to visit emergency room or urgent care for respiratory problem | Yes | 5 Participants |
| Antecedent Standard of Care | Number of Participants With Pulmonary Morbidity | At 24 Months CA: Had to stay in a hospital overnight for respiratory problem | Yes | 2 Participants |
| Antecedent Standard of Care | Number of Participants With Pulmonary Morbidity | At 24 Months CA: Had to stay in a hospital overnight for respiratory problem | No | 23 Participants |
| Antecedent Standard of Care | Number of Participants With Pulmonary Morbidity | At 12 Months CA: Had to visit emergency room or urgent care for respiratory problem | No | 23 Participants |
| Antecedent Standard of Care | Number of Participants With Pulmonary Morbidity | At 12 Months CA: Had to stay in a hospital overnight for respiratory problem | Yes | 1 Participants |
| Antecedent Standard of Care | Number of Participants With Pulmonary Morbidity | At 12 Months CA: Had to stay in a hospital overnight for respiratory problem | No | 25 Participants |
| Antecedent Standard of Care | Number of Participants With Pulmonary Morbidity | At 24 Months CA: Anyone living in the same home with participant smoke | Yes | 3 Participants |
| Antecedent Standard of Care | Number of Participants With Pulmonary Morbidity | At 24 Months CA: Anyone living in the same home with participant smoke | No | 22 Participants |
| Antecedent Standard of Care | Number of Participants With Pulmonary Morbidity | At 24 Months CA: Had asthma, wheezing, BPD exacerbation or flare-up | Yes | 4 Participants |
| Antecedent Standard of Care | Number of Participants With Pulmonary Morbidity | At 24 Months CA: Had asthma, wheezing, BPD exacerbation or flare-up | No | 21 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Pulmonary Morbidity | At 12 Months CA: Had bronchiolitis, bronchitis, or pneumonia diagnosed | No | 30 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Pulmonary Morbidity | At 24 Months CA: Had asthma, wheezing, BPD exacerbation or flare-up | Yes | 8 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Pulmonary Morbidity | At 6 Months CA: Anyone living in the same home with participant smoke | Yes | 7 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Pulmonary Morbidity | At 12 Months CA: Had to use oxygen at home | Yes | 0 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Pulmonary Morbidity | At 6 Months CA: Anyone living in the same home with participant smoke | No | 32 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Pulmonary Morbidity | At 24 Months CA: Had to stay in a hospital overnight for respiratory problem | Yes | 0 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Pulmonary Morbidity | At 6 Months CA: Had asthma, wheezing, BPD exacerbation or flare-up | Yes | 4 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Pulmonary Morbidity | At 12 Months CA: Had to use oxygen at home | No | 35 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Pulmonary Morbidity | At 6 Months CA: Had asthma, wheezing, BPD exacerbation or flare-up | No | 35 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Pulmonary Morbidity | At 12 Months CA: Had to stay in a hospital overnight for respiratory problem | No | 31 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Pulmonary Morbidity | At 6 Months CA: Had bronchiolitis, bronchitis, or pneumonia diagnosed | Yes | 5 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Pulmonary Morbidity | At 12 Months CA: Had to visit emergency room or urgent care for respiratory problem | Yes | 7 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Pulmonary Morbidity | At 6 Months CA: Had bronchiolitis, bronchitis, or pneumonia diagnosed | No | 34 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Pulmonary Morbidity | At 24 Months CA: Had to stay in a hospital overnight for respiratory problem | No | 36 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Pulmonary Morbidity | At 6 Months CA: Had to use oxygen at home | Yes | 5 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Pulmonary Morbidity | At 24 Months CA: Had bronchiolitis, bronchitis, or pneumonia diagnosed | Yes | 1 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Pulmonary Morbidity | At 6 Months CA: Had to use oxygen at home | No | 34 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Pulmonary Morbidity | At 24 Months CA: Anyone living in the same home with participant smoke | No | 33 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Pulmonary Morbidity | At 6 Months CA: Had to visit emergency room or urgent care for respiratory problem | Yes | 7 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Pulmonary Morbidity | At 24 Months CA: Had bronchiolitis, bronchitis, or pneumonia diagnosed | No | 35 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Pulmonary Morbidity | At 6 Months CA: Had to visit emergency room or urgent care for respiratory problem | No | 32 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Pulmonary Morbidity | At 12 Months CA: Had to visit emergency room or urgent care for respiratory problem | No | 28 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Pulmonary Morbidity | At 6 Months CA: Had to stay in a hospital overnight for respiratory problem | Yes | 7 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Pulmonary Morbidity | At 24 Months CA: Had to use oxygen at home | Yes | 0 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Pulmonary Morbidity | At 6 Months CA: Had to stay in a hospital overnight for respiratory problem | No | 32 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Pulmonary Morbidity | At 24 Months CA: Anyone living in the same home with participant smoke | Yes | 3 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Pulmonary Morbidity | At 12 Months CA: Anyone living in the same home with participant smoke | Yes | 5 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Pulmonary Morbidity | At 24 Months CA: Had to use oxygen at home | No | 36 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Pulmonary Morbidity | At 12 Months CA: Anyone living in the same home with participant smoke | No | 30 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Pulmonary Morbidity | At 12 Months CA: Had to stay in a hospital overnight for respiratory problem | Yes | 4 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Pulmonary Morbidity | At 12 Months CA: Had asthma, wheezing, BPD exacerbation or flare-up | Yes | 7 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Pulmonary Morbidity | At 24 Months CA: Had to visit emergency room or urgent care for respiratory problem | Yes | 5 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Pulmonary Morbidity | At 12 Months CA: Had asthma, wheezing, BPD exacerbation or flare-up | No | 28 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Pulmonary Morbidity | At 24 Months CA: Had asthma, wheezing, BPD exacerbation or flare-up | No | 28 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Pulmonary Morbidity | At 12 Months CA: Had bronchiolitis, bronchitis, or pneumonia diagnosed | Yes | 5 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Pulmonary Morbidity | At 24 Months CA: Had to visit emergency room or urgent care for respiratory problem | No | 31 Participants |
Secondary
Number of Participants With Survival Status
Survival status was assessed by number participants who died and were censored during the study.
Time frame: From start of study drug administration up to end of study (up to 6.5 years)
Population: Safety set included all participants in the Enrolled Set who have safety follow-up data in this long-term outcome study.
| Arm | Measure | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Antecedent Standard of Care | Number of Participants With Survival Status | Participants who Died | 1 Participants |
| Antecedent Standard of Care | Number of Participants With Survival Status | Participants who were Censored | 35 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Survival Status | Participants who Died | 0 Participants |
| Antecedent rhIGF-1/rhIGFBP-3 | Number of Participants With Survival Status | Participants who were Censored | 40 Participants |