Autism Spectrum DisorderPhase 2NCT01970345
A Pilot Treatment Study of Insulin-Like Growth Factor-1 (IGF-1) in Autism Spectrum Disorder
A Phase 2 study testing IGF-1 in people with Autism Spectrum Disorder, run by Icahn School of Medicine at Mount Sinai at 1 site in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 5 to 12 years
- Conditions the study is for
- Autism Spectrum Disorder
- What is being tested?
- IGF-1
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 3 participants
- Where
- 1 siteUnited States
- Study dates
- May 2014to 13 Sep 2017
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 to 12 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Icahn School of Medicine at Mount Sinai
New York, New York, 10029
About this study
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.
Read the 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.
- Conditions
- Autism Spectrum Disorder
- Being tested
- IGF-1, Placebo/saline
- Keywords
- Autism Spectrum Disorder, IGF-1
- Medical subject headings
- Autism Spectrum Disorder
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IGF-1Drug
Also called Increlex, mecasermin
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.
Placebo/salineDrug
Also called Saline
Placebo
| Study group | Role | What they receive |
|---|---|---|
| IGF-1 | Experimental | 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. Receives: Drug: IGF-1 |
| Placebo | Placebo comparator | Placebo Receives: Drug: Placebo/saline |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Phase 1 - Week 4 and 12, Phase 2 - Week 4 and 8
Secondary outcomes (9)
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.
Measured: Week 4 of Phase 1
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.
Measured: Week 12 of Phase 1
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.
Measured: Week 4 of Phase 2
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.
Measured: Week 8 of Phase 2
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.
Measured: Phase 1 - Week 4 and 12, Phase 2 - Week 4 and 8
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.
Measured: Phase 1 - Week 4 and 12, Phase 2 - Week 4 and 8
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.
Measured: Phase 1 - Week 4 and 12, Phase 2 - Week 4 and 8
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).
Measured: Phase 1 - Week 4 and 12, Phase 2 - Week 4 and 8
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.
Measured: Baseline and Phase 1 Week 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Oct 2013 (estimated)
- Study startMay 2014
- Primary completion13 Sep 2017
- Study completion13 Sep 2017
Why it stopped: Decision made to terminate due to difficulty in procurement of adequate supply of treatment drug. Decision made on April 2023 to terminate study.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Autism Spectrum DisorderStudies of the same condition, open and under way.
- More trials run by Icahn School of Medicine at Mount SinaiThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- Icahn School of Medicine at Mount Sinai
- Sponsor type
- University, hospital or other
- Collaborators
- Autism Science Foundation
- Responsible party
- Principal investigator, Alexander Kolevzon, Icahn School of Medicine at Mount Sinai
- Investigators
- Alexander Kolevzon, MD, Principal investigator, Icahn School of Medicine at Mount Sinai
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01970345
- Sponsor's protocol ID
- GCO 13-1206
- Status last verified
- Jan 2024
- First submitted
- 22 Oct 2013
- Last updated
- 8 Feb 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Long 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.
PubMed 40351246Iffland 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.
PubMed 37811711
Study at a glance
From the registry record, last updated by the sponsor on 8 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Treatment Study of Insulin-Like Growth Factor-1 (IGF-1) in Autism Spectrum Disorder
- ClinicalTrials.gov ID
- NCT01970345
- Acronym / sponsor ID
- GCO 13-1206
- Conditions
- Autism Spectrum Disorder
- Keywords
- Autism Spectrum Disorder, IGF-1
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Decision made to terminate due to difficulty in procurement of adequate supply of treatment drug. Decision made on April 2023 to terminate study.
- Enrolment
- 3 participants (actual)
- Lead sponsor
- Icahn School of Medicine at Mount SinaiUniversity, hospital or other
- Collaborators
- Autism Science Foundation
- Study start
- May 2014
- Primary completion
- 13 Sep 2017 (actual)
- Study completion
- 13 Sep 2017 (actual)
- First posted
- 28 Oct 2013 (estimated)
- Results
- Posted 8 Feb 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| IGF-1 | Experimental | Drug: IGF-1Randomized, 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 | Placebo comparator | Drug: Placebo/salinePlacebo |
Who may qualify
- Ages
- 5 to 12 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (8 listed)
- Closed epiphyses
- Active or suspected neoplasia
- Intracranial hypertension
- Hepatic insufficiency
- Renal insufficiency
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| IGF-1Also: Increlex, mecaserminIGF-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. | Drug | IGF-1 |
| Placebo/salineAlso: SalinePlacebo | Drug | Placebo |
Primary outcome
Aberrant Behavior Checklist - Social Withdrawal Subscale
Time frame: 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.
9 secondary outcomes
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Time frame: Week 4 of Phase 1
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Time frame: Week 12 of Phase 1
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Time frame: Week 4 of Phase 2
Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
Time frame: Week 8 of Phase 2
Repetitive Behavior
Time frame: Phase 1 - Week 4 and 12, Phase 2 - Week 4 and 8
Caregiver Strain
Time frame: Phase 1 - Week 4 and 12, Phase 2 - Week 4 and 8
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
The Clinical Global Impression - Severity Scale (CGI-S)
Time frame: Phase 1 - Week 4 and 12, Phase 2 - Week 4 and 8
Vineland Adaptive Behavior Composite Score
Time frame: Baseline and Phase 1 Week 12
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Feb 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 40351246Long 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.
- Derived from this study: PubMed 37811711Iffland 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Alexander Kolevzon, MD principal investigator
Icahn School of Medicine at Mount Sinai
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing IGF-1 in people with Autism Spectrum Disorder, run by Icahn School of Medicine at Mount Sinai at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 8 Feb 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 8 Feb 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Aberrant Behavior Checklist - Social Withdrawal Subscale (time frame: Phase 1 - Week 4 and 12, Phase 2 - Week 4 and 8). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01970345, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Feb 2024.