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

• Status: Recruiting
• Trial end date: 2022-05-01
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Icahn School of Medicine at Mount Sinai
• Collaborator: Autism Science Foundation
• Treatments: Mecasermin; Mitogens

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