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

  • End date
    Jul 28, 2024
  • participants needed
  • sponsor
    Icahn School of Medicine at Mount Sinai
Updated on 4 October 2022
pervasive developmental disorder


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.


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.

Condition Autism Spectrum Disorder
Treatment IGF-1, Placebo/saline
Clinical Study IdentifierNCT01970345
SponsorIcahn School of Medicine at Mount Sinai
Last Modified on4 October 2022


Yes No Not Sure

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
History of allergy to IGF-1
Patients with comorbid conditions deemed too medically compromised to tolerate the risk of experimental treatment with IGF-1
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