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Growth Factor

IGF-1 for Autism

Phase 2
Waitlist Available
Led By Alexander Kolevzon, MD
Research Sponsored by Icahn School of Medicine at Mount Sinai
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Children between the ages of 5-12 years of age
Be younger than 18 years old
Must not have
Closed epiphyses
Active or suspected neoplasia
Timeline
Screening 3 weeks
Treatment Varies
Follow Up phase 1 - week 4 and 12, phase 2 - week 4 and 8
Awards & highlights

Summary

This trial will test if a new treatment, IGF-1, is safe and effective in children with autism. The children will be given either the treatment or a placebo and then switched after a period of time. The study will measure if the treatment improves socialization, language, and repetitive behaviors.

Who is the study for?
This trial is for children aged 5-12 with Autism Spectrum Disorder, confirmed by specific diagnostic tools. They must have a language delay and be on stable medication regimens that are safe with IGF-1 for three months prior to joining. Children can't join if they have certain medical conditions like closed epiphyses, tumors, high brain pressure, liver or kidney issues, heart enlargement/valve problems, or allergies to IGF-1.Check my eligibility
What is being tested?
The study tests the effects of Insulin-Like Growth Factor-1 (IGF-1) versus a placebo in improving socialization, language skills, and repetitive behaviors in autistic children. It's a small-scale double-blind trial where neither the participants nor researchers know who receives IGF-1 or placebo during the study period.See study design
What are the potential side effects?
Potential side effects of IGF-1 treatment could include reactions at the injection site since it's similar to insulin but may also affect growth due to its role in development. The exact side effects aren't listed here but would typically be monitored closely given this is a pilot study.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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My child is between 5 and 12 years old.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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My growth plates have fully matured.
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I have or might have a new tumor.
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I have high pressure inside my skull.
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My liver is not working properly.
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My kidneys do not work well.
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I have an enlarged heart or heart valve problems.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~phase 1 - week 4 and 12, phase 2 - week 4 and 8
This trial's timeline: 3 weeks for screening, Varies for treatment, and phase 1 - week 4 and 12, phase 2 - week 4 and 8 for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Aberrant Behavior Checklist - Social Withdrawal Subscale
Secondary outcome measures
Caregiver Strain
Children's Yale-Brown Obsessive Compulsive Disorder Scale for Pervasive Developmental Disorders
Repetitive Behavior
+3 more

Side effects data

From 2013 Phase 1 & 2 trial • 44 Patients • NCT01207908
41%
Headache
23%
Nausea and vomiting
18%
Leg pain
14%
fever
14%
Back pain
5%
Finger injury
5%
rash and skin welt
5%
Foot pain
5%
seizure
100%
80%
60%
40%
20%
0%
Study treatment Arm
Standard Steroid Treatment Alone
IGF-1

Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: IGF-1Experimental Treatment1 Intervention
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.
Group II: PlaceboPlacebo Group1 Intervention
Placebo
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
IGF-1
2010
Completed Phase 2
~70

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.
Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
Common treatments for Autism Spectrum Disorder (ASD) often target neuronal growth and synaptic plasticity, which are crucial for improving core symptoms such as social impairments, language delays, and repetitive behaviors. IGF-1, for instance, promotes neuronal growth and synaptic plasticity, potentially ameliorating social withdrawal and enhancing overall functional outcomes. Oxytocin, another treatment, is believed to improve social behaviors by modulating social bonding and emotional responses. Melatonin, while primarily used for sleep disturbances, can indirectly benefit ASD patients by improving sleep patterns, which in turn can enhance daytime behaviors and cognitive functions. These treatments matter for ASD patients as they address underlying neurological deficits, offering potential improvements in quality of life and daily functioning.
Neural Mechanisms of Qigong Sensory Training Massage for Children With Autism Spectrum Disorder: A Feasibility Study.Dietary Supplement for Core Symptoms of Autism Spectrum Disorder: Where Are We Now and Where Should We Go?Clinical and neural effects of six-week administration of oxytocin on core symptoms of autism.

Find a Location

Who is running the clinical trial?

Icahn School of Medicine at Mount SinaiLead Sponsor
871 Previous Clinical Trials
527,269 Total Patients Enrolled
7 Trials studying Autism Spectrum Disorder
232 Patients Enrolled for Autism Spectrum Disorder
Autism Science FoundationOTHER
4 Previous Clinical Trials
190 Total Patients Enrolled
3 Trials studying Autism Spectrum Disorder
180 Patients Enrolled for Autism Spectrum Disorder
Alexander Kolevzon, MD4.34 ReviewsPrincipal Investigator - Icahn School of Medicine at Mount Sinai
Icahn School of Medicine at Mount Sinai
13 Previous Clinical Trials
504 Total Patients Enrolled
7 Trials studying Autism Spectrum Disorder
373 Patients Enrolled for Autism Spectrum Disorder
5Patient Review
Dr. Alexander Kolevzon is a caring and attentive doctor who went above and beyond for my son. We are grateful to have him as our physician.

Media Library

IGF-1 (Growth Factor) Clinical Trial Eligibility Overview. Trial Name: NCT01970345 — Phase 2
Autism Spectrum Disorder Research Study Groups: IGF-1, Placebo
Autism Spectrum Disorder Clinical Trial 2023: IGF-1 Highlights & Side Effects. Trial Name: NCT01970345 — Phase 2
IGF-1 (Growth Factor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT01970345 — Phase 2
~0 spots leftby Jul 2025