What side effects are associated with this type of intervention?

Common treatments for Autism Spectrum Disorder (ASD) that promote neuronal growth and synaptic plasticity, such as IGF-1, omega-3 fatty acids, and cannabinoids, have varying side effects. Omega-3 fatty acids can cause gastrointestinal issues like nausea and diarrhea, while cannabinoids may lead to somnolence, decreased appetite, irritability, and restlessness. The efficacy of these treatments in improving core ASD symptoms remains inconsistent.

What prior FDA approvals exist?

Currently, there are no FDA-approved treatments specifically targeting the core symptoms of Autism Spectrum Disorder (ASD) that function similarly to IGF-1, which promotes neuronal growth and synaptic plasticity. Most FDA-approved medications for ASD, such as risperidone and aripiprazole, primarily address irritability and associated behavioral symptoms rather than core social and communication deficits. The pilot study of IGF-1 aims to explore its potential in improving socialization, language, and repetitive behaviors in children with autism, which represents a novel approach compared to existing treatments.

What other types of research exist?

Promising novel alternatives to IGF-1 for treating Autism Spectrum Disorder include: 1) Antisense oligonucleotide therapies, which aim to reactivate the expression of specific genes in the brain, 2) Gene therapy using viral vectors to correct genetic abnormalities, and 3) Trofinetide, a compound that has shown efficacy in clinical trials for Rett syndrome, which shares some overlapping features with ASD.

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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 IGF-1, a substance that helps brain cells grow and connect, in children with autism. The goal is to see if it can improve social skills, language, and reduce repetitive behaviors. IGF-1 is already approved for other uses and works by making brain cells stronger and better connected. IGF-1 has been studied for its potential to improve behavior in mouse models of autism and has shown promise in clinical studies for neurodevelopmental disorders, including autism spectrum disorder (ASD).

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.

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.

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 weeks3 visits

Treatment ~ Varies

Follow Up ~ phase 1 - week 4 and 12, phase 2 - week 4 and 8

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

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Aberrant Behavior Checklist - Social Withdrawal Subscale

Secondary study objectives

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

Finger injury

rash and skin welt

Foot pain

seizure

100%

80%

60%

40%

20%

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

0 / 7Eligibility criteria met

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.