Retinopathy of Prematurity (ROP)
Conditions
Interventions
Product Name: mecasermin rinfabate
Product Code: rhIGF-1/rhIGFBP-3
Pharmaceutical Form: Solution for injection
Sponsors
Premacure AB, A Member of the Shire Group of Companies
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Each subject must meet the following criteria to be enrolled in this study. 1. Subject was randomized in Study ROPP-2008-01, Section D 2. Subject’s 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 subject. Are the trial subjects under 18? yes Number of subjects for this age range: 120 F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Subjects who meet any of the following criteria will be excluded from the study. 1. Any other condition or therapy that, in the Investigator’s opinion, may pose a risk to the subject or interfere with the subject’s ability to be compliant with this protocol or interfere with the interpretation of results 2. The subject or subject’s parent or legally authorized representative(s) is unable to comply with the protocol as determined by the Investigator
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objectives of this study are: - To evaluate the long-term efficacy outcomes following short-term exposure to rhIGF-1/rhIGFBP-3 versus standard neonatal care in Study ROPP-2008-01 (Section D) as assessed by ROP-associated visual outcomes - To evaluate the long-term safety outcomes following short-term exposure to rhIGF-1/rhIGFBP-3 versus standard neonatal care in Study ROPP-2008-01 (Section D);Secondary Objective: The secondary objectives of this study are to evaluate the effect following short-term exposure to rhIGF-1/rhIGFBP-3 versus standard neonatal care in Study ROPP-2008-01 (Section D) on: Growth parameters; Cognitive development; Physical development; Child behavior; Pulmonary morbidity; Survival; Health-related quality of life (HRQoL); Health utility; Health care resource use (HCRU) ;Primary end point(s): The primary efficacy endpoints of this study are: - Visual acuity as assessed by an age appropriate method - Ocular alignment and ocular motor examination in primary gaze and in as many of 9 positions of gaze as possible as assessed by corneal light reflex and by the cover test - Assessment of nystagmus by observation - Refraction as assessed by retinoscopy with cycloplegia - Stereoacuity as assessed with the Lang Stereotest;Timepoint(s) of evaluation of this end point: will vary depending upon age at enrollment, but subjects will not be followed beyond age 5.5 years corrected age (CA) | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): The secondary efficacy endpoints of this study are: - Growth parameters including body weight, body length (or height), and head circumference - Cognitive development as assessed by the following standardized, age-appropriate tools: o Bayley Scales of Infant and Toddler Development, Third Edition (BSID-III) o Wechsler Preschool and Primary Scale of Intelligence, Fourth Edition (WPPSI-IV) - Physical development as assessed by standardized, age appropriate tools including physical exam, neurological examination for assessment of cerebral palsy, and hearing assessment - Child behavior as assessed by the following: o Vineland Adaptive Behavior Scales, Second Edition (VABS-II) o Child Behavior Checklist (CBCL; 1 ½ to 5) o Attention Deficit/Hyperactivity Disorder Rating Scale-fourth edition (ADHD RS-IV) for the assessment of symptoms of attention deficit/hyperactivity disorder (ADHD) o Social Communication Questionnaire (SCQ) for screening of Autism Spectrum Disorder (ASD) - Pulmonary morbidity data (eg, hospitalizations, emergency room [ER] visits, pulmonary medications) - Cerebral magnetic resonance imaging (MRI): Participation in the MRI assessment is optional and has no impact on participating in the main matter, corpus callosum, frontal lobes, cerebellum and total volume will be analysed for the purposes of the study) - Survival as assessed by death during the study due to any cause - Blood pressure, heart rate and respiratory rate will be measured at the 5 year CA visit. The health economic outcome research endpoints of this study are: - Health-related quality of life (HRQoL) will be assessed by the Pediatric Quality of Life Inventory (PedsQL™) Scales appropriate for the child's age of development with the Total Scale Score and 5 domains within Physical Health (Physical Functioning and Physical Symptoms) and Psychosocial Health Scores (Emotional, So | — |
Countries
Canada, Italy, Netherlands, Poland, Sweden, United Kingdom, United States
Contacts
Public ContactEmily Jochim
Shire
Outcome results
None listed