Autism Spectrum Disorder
Conditions
Keywords
Autism Spectrum Disorder, IGF-1
Brief summary
The proposed project will pilot the use of IGF-1 as a novel treatment for core symptoms of autism. We will use a double-blind, placebo-controlled crossover trial design in five children with autism to evaluate the impact of IGF-1 treatment on autism-specific impairments in socialization, language, and repetitive behaviors. We expect to provide evidence for the safety and feasibility of IGF-1 in ameliorating social withdrawal in children with Autistic Disorder. Further, we expect to demonstrate that IGF-1 is associated with improvement on secondary outcomes of social impairment, language delay, and repetitive behavior, as well as on functional outcomes of global severity.
Detailed description
IGF-1 is an FDA approved, commercially available compound that crosses the blood-brain barrier and has beneficial effects on synaptic development by promoting neuronal cell survival, synaptic maturation, and synaptic plasticity. IGF-1 is effective in reversing mouse and neuronal models of Rett syndrome and Phelan McDermid syndrome, both single gene causes of ASD and may therefore be effective in treating autism spectrum disorders (ASD) more broadly.
Interventions
DRUGIGF-1
IGF-1 is an FDA-approved (IND exemption #113450), commercially available compound that crosses the blood-brain barrier and has beneficial effects on synaptic development by promoting neuronal cell survival, synaptic maturation, and synaptic plasticity.
DRUGPlacebo/saline
Placebo
Sponsors
Autism Science Foundation
Icahn School of Medicine at Mount Sinai
Study design
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
5 Years to 12 Years
Healthy volunteers
No
Inclusion criteria
* Meet DSM-5 criteria for Autism Spectrum Disorder confirmed by the Autism Diagnostic Interview-Revised (ADI-R) and the Autism Diagnostic Observation Schedule- Generic (ADOS-G) * Children between the ages of 5-12 years of age * Language delay (lack of fluent phrase speech) reflected by use of ADOS Module 1 or 2 * Must be on stable medication regimens for at least three months prior to enrollment, assuming the concomitant medication is safe for use with IGF-1
Exclusion criteria
* Closed epiphyses * Active or suspected neoplasia * Intracranial hypertension * Hepatic insufficiency * Renal insufficiency * Cardiomegaly/valvulopathy * History of allergy to IGF-1 * Patients with comorbid conditions deemed too medically compromised to tolerate the risk of experimental treatment with IGF-1
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Aberrant Behavior Checklist - Social Withdrawal Subscale | Phase 1 - Week 4 and 12, Phase 2 - Week 4 and 8 | Aberrant Behavior Checklist - Social Withdrawal Subscale has16 items, Each item is scored as 0 (never a problem), 1 (slight problem), 2 (moderately serious problem), or 3 (severe problem). Total Subscale scores range from 0 to 48. Higher score indicates poorer health outcomes. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Repetitive Behavior | Phase 1 - Week 4 and 12, Phase 2 - Week 4 and 8 | Repetitive Behavior: Repetitive Behavior Scale (RBS) - Includes 43 items scored 0 (behavior does not occur), 1 (behavior occurs and is a mild problem), 2 (behavior occurs and is a moderate problem), 3 (behavior occurs and is a severe problem). Full scale from 0-129, with higher scores indicating worse outcomes. |
| Caregiver Strain | Phase 1 - Week 4 and 12, Phase 2 - Week 4 and 8 | Caregiver Strain Index - 21 question tool, with response 1 (not al all) - 5 (very much). Total score on scale from 21-105. Higher score indicates poorer health outcomes. |
| Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Week 4 of Phase 1 | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) - Each item is marked as 0 (does not show the behavior), 1 (rarely shows the behavior), 2 (sometimes/partially shows the behavior), 3 (usually/typically shows the behavior), or ? (uncertain/don't understand). The Sensory, Ritual, Social Pragmatic, Semantic Pragmatic, Arouse, Specific Fears, and Aggressiveness subscales range from 0 to 100, where higher score indicates worse severity than the average child with autism. The Social Approach, Expressive, and Learning Memory subscales range from 0 to 100, where higher score indicates better skills relative to the average child with autism. Scores reported in subscales. Scores were not reported as full scale. |
| The Clinical Global Impression - Severity Scale (CGI-S) | Phase 1 - Week 4 and 12, Phase 2 - Week 4 and 8 | The CGI-S is a 7-point scale that requires the clinician to rate the severity of the patient's illness at the time of assessment, relative to the clinician's past experience with patients who have the same diagnosis. Considering total clinical experience, a patient is assessed on severity of mental illness at the time of rating. Full scale is 1 (normal, not at all ill) to 7 (extremely ill). |
| Vineland Adaptive Behavior Composite Score | Baseline and Phase 1 Week 12 | The Vineland Adaptive Behavior Scales, Second Edition (Survey Interview Form) yields an overall standard score (Adaptive Behavior Composite, ABC) and age standard scores in four domains. ABC scores have a mean of 100 and a standard deviation of 15 (range = 20 to 160). Higher scores suggest a higher level of adaptive functioning. |
| Children's Yale-Brown Obsessive Compulsive Disorder Scale for Pervasive Developmental Disorders | Phase 1 - Week 4 and 12, Phase 2 - Week 4 and 8 | Clinician-rated interview designed to evaluate repetitive behavior in children with pervasive developmental disorders (PDDs). It is a modification of the Children's Yale-Brown Obsessive Compulsive Scale (CY-BOCS), developed to assess typically-developing children with obsessive compulsive behavior. Because of language limitations in children with PDDs the CY-BOCS-PDD only includes the five compulsion items: Time Spent, Interference, Distress, Resistance of repetitive behavior, and Control of repetitive behavior. Each item is rated from 0 (none) through 4 (extreme), and scores can range from 0 to 20, with higher scores reflecting more severe symptoms. Usually a score 8 is considered clinically significant. |
Countries
United States
Participant flow
Recruitment details
Decision made to terminate due to difficulty in procurement of adequate supply of treatment drug. Decision made on April 2023 to terminate study. However last participant visit was Sept 2017.
Participants by arm
| Arm | Count |
|---|---|
| Children With Autism Spectrum Disorders Randomized, placebo-controlled, crossover format with 12 weeks in each treatment arm (IGF-1 and placebo), separated by a four-week wash-out phase. | 3 |
| Total | 3 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| First Treatment Phase 12 Weeks | Withdrawal by Subject | 1 | 1 |
| Second Treatment Phase 12 Weeks | Withdrawal by Subject | 0 | 1 |
Baseline characteristics
| Characteristic | Children With Autism Spectrum Disorders |
|---|---|
| Age, Categorical <=18 years | 3 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 0 Participants |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 3 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 1 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 2 Participants |
| Sex: Female, Male Female | 0 Participants |
| Sex: Female, Male Male | 3 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 2 | 0 / 2 |
| other Total, other adverse events | 2 / 2 | 2 / 2 |
| serious Total, serious adverse events | 0 / 2 | 0 / 2 |
Outcome results
Primary
Aberrant Behavior Checklist - Social Withdrawal Subscale
Aberrant Behavior Checklist - Social Withdrawal Subscale has16 items, Each item is scored as 0 (never a problem), 1 (slight problem), 2 (moderately serious problem), or 3 (severe problem). Total Subscale scores range from 0 to 48. Higher score indicates poorer health outcomes.
Time frame: Phase 1 - Week 4 and 12, Phase 2 - Week 4 and 8
Population: Results for participants who were still on study.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| IGF-1 | Aberrant Behavior Checklist - Social Withdrawal Subscale | Week 4, phase 1 | 20 score on a scale | — |
| IGF-1 | Aberrant Behavior Checklist - Social Withdrawal Subscale | Week 4, phase 2 | 3 score on a scale | — |
| IGF-1 | Aberrant Behavior Checklist - Social Withdrawal Subscale | Week 8, Phase 2 | 2 score on a scale | — |
| Placebo | Aberrant Behavior Checklist - Social Withdrawal Subscale | Week 4, phase 1 | 20 score on a scale | Standard Deviation 18.4 |
| Placebo | Aberrant Behavior Checklist - Social Withdrawal Subscale | Week 12, Phase 1 | 4 score on a scale | — |
Secondary
Caregiver Strain
Caregiver Strain Index - 21 question tool, with response 1 (not al all) - 5 (very much). Total score on scale from 21-105. Higher score indicates poorer health outcomes.
Time frame: Phase 1 - Week 4 and 12, Phase 2 - Week 4 and 8
Population: Data provided for participants who were on study
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| IGF-1 | Caregiver Strain | Week 8, Phase 2 | 54 score on a scale | — |
| IGF-1 | Caregiver Strain | Week 4, Phase 1 | 71 score on a scale | — |
| IGF-1 | Caregiver Strain | Week 4, Phase 2 | 54 score on a scale | — |
| Placebo | Caregiver Strain | Week 4, Phase 1 | 87 score on a scale | Standard Deviation 9.9 |
| Placebo | Caregiver Strain | Week 12, Phase 1 | 73 score on a scale | — |
Secondary
Children's Yale-Brown Obsessive Compulsive Disorder Scale for Pervasive Developmental Disorders
Clinician-rated interview designed to evaluate repetitive behavior in children with pervasive developmental disorders (PDDs). It is a modification of the Children's Yale-Brown Obsessive Compulsive Scale (CY-BOCS), developed to assess typically-developing children with obsessive compulsive behavior. Because of language limitations in children with PDDs the CY-BOCS-PDD only includes the five compulsion items: Time Spent, Interference, Distress, Resistance of repetitive behavior, and Control of repetitive behavior. Each item is rated from 0 (none) through 4 (extreme), and scores can range from 0 to 20, with higher scores reflecting more severe symptoms. Usually a score 8 is considered clinically significant.
Time frame: Phase 1 - Week 4 and 12, Phase 2 - Week 4 and 8
Population: data not collected
Secondary
Repetitive Behavior
Repetitive Behavior: Repetitive Behavior Scale (RBS) - Includes 43 items scored 0 (behavior does not occur), 1 (behavior occurs and is a mild problem), 2 (behavior occurs and is a moderate problem), 3 (behavior occurs and is a severe problem). Full scale from 0-129, with higher scores indicating worse outcomes.
Time frame: Phase 1 - Week 4 and 12, Phase 2 - Week 4 and 8
Population: Data provided for participants who were on study
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| IGF-1 | Repetitive Behavior | Week 4 of Phase 1 | 31 score on a scale | — |
| IGF-1 | Repetitive Behavior | Week 4 of Phase 2 | 11 score on a scale | — |
| IGF-1 | Repetitive Behavior | Week 8 of Phase 2 | 8 score on a scale | — |
| Placebo | Repetitive Behavior | Week 4 of Phase 1 | 44 score on a scale | Standard Deviation 41 |
| Placebo | Repetitive Behavior | Week 12 of Phase 1 | 11 score on a scale | — |
Secondary
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) - Each item is marked as 0 (does not show the behavior), 1 (rarely shows the behavior), 2 (sometimes/partially shows the behavior), 3 (usually/typically shows the behavior), or ? (uncertain/don't understand). The Sensory, Ritual, Social Pragmatic, Semantic Pragmatic, Arouse, Specific Fears, and Aggressiveness subscales range from 0 to 100, where higher score indicates worse severity than the average child with autism. The Social Approach, Expressive, and Learning Memory subscales range from 0 to 100, where higher score indicates better skills relative to the average child with autism. Scores reported in subscales. Scores were not reported as full scale.
Time frame: Week 8 of Phase 2
Population: Data provided for participants who completed week 8. Data not provided by participant for one subscale.
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| IGF-1 | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Sensory | 5 score on a scale |
| IGF-1 | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Ritual | 1 score on a scale |
| IGF-1 | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Semantic Pragmatic | 9 score on a scale |
| IGF-1 | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Arouse | 10 score on a scale |
| IGF-1 | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Specific Fears | 7 score on a scale |
| IGF-1 | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Aggressiveness | 6 score on a scale |
| IGF-1 | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Social Approach | 60 score on a scale |
| IGF-1 | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Expressive | 43 score on a scale |
| IGF-1 | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Learning Memory | 14 score on a scale |
Secondary
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) - Each item is marked as 0 (does not show the behavior), 1 (rarely shows the behavior), 2 (sometimes/partially shows the behavior), 3 (usually/typically shows the behavior), or ? (uncertain/don't understand). The Sensory, Ritual, Social Pragmatic, Semantic Pragmatic, Arouse, Specific Fears, and Aggressiveness subscales range from 0 to 100, where higher score indicates worse severity than the average child with autism. The Social Approach, Expressive, and Learning Memory subscales range from 0 to 100, where higher score indicates better skills relative to the average child with autism. Scores reported in subscales. Scores were not reported as full scale.
Time frame: Week 4 of Phase 2
Population: Data provided for participants who completed week 4
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| IGF-1 | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Sensory | 11 score on a scale |
| IGF-1 | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Ritual | 2 score on a scale |
| IGF-1 | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Social Pragmatic | 13 score on a scale |
| IGF-1 | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Semantic Pragmatic | 5 score on a scale |
| IGF-1 | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Arouse | 16 score on a scale |
| IGF-1 | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Specific Fears | 10 score on a scale |
| IGF-1 | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Aggressiveness | 10 score on a scale |
| IGF-1 | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Social Approach | 52 score on a scale |
| IGF-1 | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Expressive | 36 score on a scale |
| IGF-1 | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Learning Memory | 14 score on a scale |
Secondary
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) - Each item is marked as 0 (does not show the behavior), 1 (rarely shows the behavior), 2 (sometimes/partially shows the behavior), 3 (usually/typically shows the behavior), or ? (uncertain/don't understand). The Sensory, Ritual, Social Pragmatic, Semantic Pragmatic, Arouse, Specific Fears, and Aggressiveness subscales range from 0 to 100, where higher score indicates worse severity than the average child with autism. The Social Approach, Expressive, and Learning Memory subscales range from 0 to 100, where higher score indicates better skills relative to the average child with autism. Scores reported in subscales. Scores were not reported as full scale.
Time frame: Week 12 of Phase 1
Population: Data provided for participants who completed week 12
| Arm | Measure | Group | Value (MEAN) |
|---|---|---|---|
| Placebo | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Sensory | 9 score on a scale |
| Placebo | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Ritual | 4 score on a scale |
| Placebo | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Social Pragmatic | 16 score on a scale |
| Placebo | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Semantic Pragmatic | 8 score on a scale |
| Placebo | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Arouse | 12 score on a scale |
| Placebo | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Specific Fears | 22 score on a scale |
| Placebo | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Aggressiveness | 18 score on a scale |
| Placebo | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Social Approach | 59 score on a scale |
| Placebo | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Expressive | 27 score on a scale |
| Placebo | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Learning Memory | 15 score on a scale |
Secondary
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) - Each item is marked as 0 (does not show the behavior), 1 (rarely shows the behavior), 2 (sometimes/partially shows the behavior), 3 (usually/typically shows the behavior), or ? (uncertain/don't understand). The Sensory, Ritual, Social Pragmatic, Semantic Pragmatic, Arouse, Specific Fears, and Aggressiveness subscales range from 0 to 100, where higher score indicates worse severity than the average child with autism. The Social Approach, Expressive, and Learning Memory subscales range from 0 to 100, where higher score indicates better skills relative to the average child with autism. Scores reported in subscales. Scores were not reported as full scale.
Time frame: Week 4 of Phase 1
Population: Data provided for participants who completed week 4
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| IGF-1 | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Sensory | 21 score on a scale | — |
| IGF-1 | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Ritual | 9 score on a scale | — |
| IGF-1 | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Social Pragmatic | 20 score on a scale | — |
| IGF-1 | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Semantic Pragmatic | 19 score on a scale | — |
| IGF-1 | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Arouse | 23 score on a scale | — |
| IGF-1 | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Specific Fears | 13 score on a scale | — |
| IGF-1 | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Aggressiveness | 7 score on a scale | — |
| IGF-1 | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Social Approach | 75 score on a scale | — |
| IGF-1 | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Expressive | 36 score on a scale | — |
| IGF-1 | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Learning Memory | 0 score on a scale | — |
| Placebo | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Social Approach | 31 score on a scale | Standard Deviation 15.6 |
| Placebo | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Sensory | 16 score on a scale | Standard Deviation 14.1 |
| Placebo | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Specific Fears | 25 score on a scale | Standard Deviation 1.4 |
| Placebo | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Ritual | 13 score on a scale | Standard Deviation 14.1 |
| Placebo | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Learning Memory | 8.5 score on a scale | Standard Deviation 2.1 |
| Placebo | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Social Pragmatic | 14 score on a scale | Standard Deviation 0 |
| Placebo | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Aggressiveness | 30 score on a scale | Standard Deviation 25.5 |
| Placebo | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Semantic Pragmatic | 0.5 score on a scale | Standard Deviation 0.7 |
| Placebo | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Expressive | 18.5 score on a scale | Standard Deviation 20.5 |
| Placebo | Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI) | Arouse | 20.5 score on a scale | Standard Deviation 6.4 |
Secondary
The Clinical Global Impression - Severity Scale (CGI-S)
The CGI-S is a 7-point scale that requires the clinician to rate the severity of the patient's illness at the time of assessment, relative to the clinician's past experience with patients who have the same diagnosis. Considering total clinical experience, a patient is assessed on severity of mental illness at the time of rating. Full scale is 1 (normal, not at all ill) to 7 (extremely ill).
Time frame: Phase 1 - Week 4 and 12, Phase 2 - Week 4 and 8
Population: Data provided for participants who were on study
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| IGF-1 | The Clinical Global Impression - Severity Scale (CGI-S) | Week 4, Phase 1 | 5 score on a scale | — |
| IGF-1 | The Clinical Global Impression - Severity Scale (CGI-S) | Week 8, Phase 2 | 4 score on a scale | — |
| Placebo | The Clinical Global Impression - Severity Scale (CGI-S) | Week 4, Phase 1 | 5 score on a scale | Standard Deviation 1.4 |
| Placebo | The Clinical Global Impression - Severity Scale (CGI-S) | Week 12, Phase 1 | 4 score on a scale | — |
| Placebo | The Clinical Global Impression - Severity Scale (CGI-S) | Week 4, Phase 2 | 4 score on a scale | — |
Secondary
Vineland Adaptive Behavior Composite Score
The Vineland Adaptive Behavior Scales, Second Edition (Survey Interview Form) yields an overall standard score (Adaptive Behavior Composite, ABC) and age standard scores in four domains. ABC scores have a mean of 100 and a standard deviation of 15 (range = 20 to 160). Higher scores suggest a higher level of adaptive functioning.
Time frame: Baseline and Phase 1 Week 12
Population: Data collected at baseline and Week 12 and only for participants on study.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| IGF-1 | Vineland Adaptive Behavior Composite Score | Baseline | 55 T-score | — |
| Placebo | Vineland Adaptive Behavior Composite Score | Baseline | 51 T-score | Standard Deviation 8.5 |
| Placebo | Vineland Adaptive Behavior Composite Score | Week 12, Phase 1 | 57 T-score | — |