Primary outcomeMean· Phase 1 - Week 4 and 12, Phase 2 - Week 4 and 8
Aberrant Behavior Checklist - Social Withdrawal Subscale
| Group | Value (score on a scale) | Spread |
|---|---|---|
| IGF-1 | 20 | NA |
| Placebo | 20 | 18.4 |
Trial · Interventional
TerminatedPhase 2Results postedRegistry dataA Pilot Treatment Study of Insulin-Like Growth Factor-1 (IGF-1) in Autism Spectrum Disorder
NCTNCT01970345 (opens ClinicalTrials.gov in a new tab)Other idGCO 13-1206
This trial was stopped early.
Reason given at the registry: Decision made to terminate due to difficulty in procurement of adequate supply of treatment drug. Decision made on April 2023 to terminate study.
Results were posted for this trial.
Overview
- Status
- Terminated
- Phase
- Phase 2
- Enrollment
- 3 participants (actual)
- Sponsor
- Icahn School of Medicine at Mount Sinai · Other
- Design
- Randomized · Quadruple masking
- Countries
1 country
United States
- Start
- May 2014
- Primary completion
- 13 Sept 2017
- Completion
- 13 Sept 2017
- First posted
- 28 Oct 2013 (estimated)
- Last update posted
- 8 Feb 2024
- Conditions
- Autism Spectrum Disorder
Timeline
1 trial · 1 stopped early · 1 with posted results
- Completed
- Ongoing
- Not yet recruiting
- Stopped
- Estimated dates (est.)
- Results posted
20142015201620172018
- Ph 2Terminated
A Pilot Treatment Study of Insulin-Like Growth Factor-1 (IGF-1) in Autism Spectrum Disorder
NCT01970345May 2014 to 13 Sept 2017
Registry summary
Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.
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.
Interventions
- IGF-1Drug
- Placebo/salineDrug
Arms2
Experimental
IGF-1
Randomized, placebo-controlled, crossover format with 12 weeks in each treatment arm (IGF-1 and placebo), separated by a four-week wash-out phase. Dose titration will be initiated at 0.04 mg/kg twice daily by subcutaneous injection, and increased, as tolerated, every week by 0.04 mg/kg per dose to a maximum of 0.12 mg/kg twice daily. Doses may be decreased according to tolerability by 0.04 mg/kg per dose. Medication will be administered twice daily with meals, and preprandial glucose monitoring will be performed by parents at treatment initiation, prior to each injection, and until a well tolerated dose is established.
Placebo comparator
Placebo
Placebo
Primary outcomes1
Outcomes the sponsor listed as primary.
Aberrant Behavior Checklist - Social Withdrawal Subscale
Time frame · Phase 1 - Week 4 and 12, Phase 2 - Week 4 and 8
Registry description
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 outcomes(9)
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Time frame · Week 4 of Phase 1
Registry description
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.
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Time frame · Week 12 of Phase 1
Registry description
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.
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Time frame · Week 4 of Phase 2
Registry description
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.
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Time frame · Week 8 of Phase 2
Registry description
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.
Repetitive Behavior
Time frame · Phase 1 - Week 4 and 12, Phase 2 - Week 4 and 8
Registry description
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
Time frame · Phase 1 - Week 4 and 12, Phase 2 - Week 4 and 8
Registry description
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.
Children's Yale-Brown Obsessive Compulsive Disorder Scale for Pervasive Developmental Disorders
Time frame · Phase 1 - Week 4 and 12, Phase 2 - Week 4 and 8
Registry description
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.
The Clinical Global Impression - Severity Scale (CGI-S)
Time frame · Phase 1 - Week 4 and 12, Phase 2 - Week 4 and 8
Registry description
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
Time frame · Baseline and Phase 1 Week 12
Registry description
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.
Posted results
Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.
Primary outcome results
Secondary and other outcome results(7)
Secondary outcomeMean· Week 4 of Phase 1
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
| Group | Value (score on a scale) | Spread |
|---|---|---|
| IGF-1, Week 4, Phase 1 | 21 | NA |
| Placebo, Week 4, Phase 1 | 16 | 14.1 |
Secondary outcomeMean· Week 12 of Phase 1
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
| Group | Value (score on a scale) | Spread |
|---|---|---|
| IGF-1 | not posted | – |
| Placebo | 9 | NA |
Secondary outcomeMean· Week 4 of Phase 2
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
| Group | Value (score on a scale) | Spread |
|---|---|---|
| IGF-1 | 11 | NA |
| Placebo | not posted | – |
Secondary outcomeMean· Week 8 of Phase 2
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
| Group | Value (score on a scale) | Spread |
|---|---|---|
| IGF-1 | 5 | NA |
| Placebo | not posted | – |
Secondary outcomeMean· Phase 1 - Week 4 and 12, Phase 2 - Week 4 and 8
Repetitive Behavior
| Group | Value (score on a scale) | Spread |
|---|---|---|
| IGF-1 | 31 | NA |
| Placebo | 44 | 41.0 |
Secondary outcomeMean· Phase 1 - Week 4 and 12, Phase 2 - Week 4 and 8
Caregiver Strain
| Group | Value (score on a scale) | Spread |
|---|---|---|
| IGF-1 | 71 | NA |
| Placebo | 87 | 9.9 |
Secondary outcome· Phase 1 - Week 4 and 12, Phase 2 - Week 4 and 8
Children's Yale-Brown Obsessive Compulsive Disorder Scale for Pervasive Developmental Disorders
| Group | Value |
|---|---|
| IGF-1 | not posted |
| Placebo | not posted |
Adverse events, as posted
| Group | Deaths | Serious events | Other events |
|---|---|---|---|
| IGF-1Dose titration will be initiated at 0.04 mg/kg twice daily by subcutaneous injection, and increased, as tolerated, every week by 0.04 mg/kg per dose to a maximum of 0.12 mg/kg twice daily. Doses may be decreased according to tolerability by 0.04 mg/kg per dose. Medication will be administered twice daily with meals, and pre-prandial glucose monitoring will be performed by parents at treatment initiation, prior to each injection, and until a well-tolerated dose is established. | 0 / 2 | 0 / 2 | 2 / 2 |
| PlaceboPlacebo/saline: Placebo | 0 / 2 | 0 / 2 | 2 / 2 |
Each cell reads participants affected / participants at risk, as the registry posted them.
Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works
Studies0
Studies that cite this trial.
No study cites this trial.
Other registry references
References listed at the registry that are not in PepBank. They link to PubMed.
- DerivedLong J, Liao X, Han K, Niu M, Chen J, Wang X, Liu J, Zhang Y, Zhang H. Association of Thyroid Hormone and Insulin-Like Growth Factor-1 Levels With Autism Spectrum Disorders: A Systematic Review and Meta-Analysis. Autism Res. 2025 Jul;18(7):1497-1512. doi: 10.1002/aur.70052. Epub 2025 May 12. PMID 40351246
- DerivedIffland M, Livingstone N, Jorgensen M, Hazell P, Gillies D. Pharmacological intervention for irritability, aggression, and self-injury in autism spectrum disorder (ASD). Cochrane Database Syst Rev. 2023 Oct 9;10(10):CD011769. doi: 10.1002/14651858.CD011769.pub2. PMID 37811711
Eligibility
Who could join, as posted.
- Sex
- All
- Age
- from 5 Years up to 12 Years
- Healthy volunteers
- Not accepted
Inclusion and exclusion criteria, as posted
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
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