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

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

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

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 Summary: 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.
Sponsor: Icahn School of Medicine at Mount Sinai

Current Primary Outcome: Aberrant Behavior Checklist - Social Withdrawal Subscale [ Time Frame: at 12 weeks ]

Aberrant Behavior Checklist - Social Withdrawal Subscale


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Social [ Time Frame: at 12 weeks ]
    Social: Pervasive Developmental Disorder-Behavior Inventory (PDD-BI; Cohen 2003).
  • Language [ Time Frame: at 12 weeks ]
    Language: Vineland Adaptive Behavior Communication Domain (Sparrow, 1984).
  • Repetitive Behavior [ Time Frame: at 12 weeks ]
    Repetitive Behavior: Repetitive Behavior Scale (RBS; Bodfish et al., 1999); Children's Yale-Brown Obsessive Compulsive Disorder Scale for Pervasive Developmental Disorders (CY-BOCS-PDD; Scahill et al., 2006
  • Overall Functional Outcomes [ Time Frame: at 12 weeks ]
    Overall Functional Outcomes: CGI-Improvement and Severity Scales (Guy, 1976); Vineland Adaptive Behavior Composite
  • Caregiver Strain [ Time Frame: at 12 weeks ]
    Caregiver Strain Index (CSI; Brannan et al., 1997).


Original Secondary Outcome: Same as current

Information By: Icahn School of Medicine at Mount Sinai

Dates:
Date Received: October 22, 2013
Date Started: May 2014
Date Completion: January 2020
Last Updated: January 10, 2017
Last Verified: January 2017