A Pilot Treatment Study of Insulin-Like Growth Factor-1 (IGF-1) in Autism Spectrum Disorder

NCT ID: NCT01970345

Last Updated: 2024-02-08

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE2
• Total Enrollment: 3 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2014-05-31
• Study Completion Date: 2017-09-13

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.

Conditions

Autism Spectrum Disorder

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: CROSSOVER
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

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.

Group Type: EXPERIMENTAL

IGF-1

Intervention Type: DRUG

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

Placebo

Group Type: PLACEBO_COMPARATOR

Placebo/saline

Intervention Type: DRUG

Placebo

Interventions

IGF-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.

Intervention Type: DRUG

Placebo/saline

Placebo

Intervention Type: DRUG

Other Intervention Names

Increlex; mecasermin; Saline

Eligibility Criteria

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

• Minimum Eligible Age: 5 Years
• Maximum Eligible Age: 12 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Autism Science Foundation

OTHER

Sponsor Role: collaborator

Icahn School of Medicine at Mount Sinai

OTHER

Sponsor Role: lead

Responsible Party

Alexander Kolevzon

Clinical Director, Seaver Autism Center for Research and Treatment

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Alexander Kolevzon, MD

Role: PRINCIPAL_INVESTIGATOR

Icahn School of Medicine at Mount Sinai

Locations

Icahn School of Medicine at Mount Sinai

New York, New York, United States

Countries

United States

References

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.

Reference Type: DERIVED

PMID: 40351246 (View on PubMed)

Iffland 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.

Reference Type: DERIVED

PMID: 37811711 (View on PubMed)

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

GCO 13-1206

Identifier Type: -

Identifier Source: org_study_id