Clinical trial NCT01970345: A Pilot Treatment Study of Insulin-Like Growth Factor-1 (IGF-1) in Autism Spectrum Disorder
Official title: A Pilot Treatment Study of Insulin-Like Growth Factor-1 (IGF-1) in Autism Spectrum Disorder
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Autism Spectrum Disorder
Keywords: Autism Spectrum Disorder
IGF-1
Lead sponsor: Icahn School of Medicine at Mount Sinai
Sponsor class: OTHER
Collaborators: name: Autism Science Foundation
First posted: 2013-10-28
Enrollment: 3
Sex: ALL
Minimum age (years): 5
Maximum age (years): 12
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: Aberrant Behavior Checklist - Social Withdrawal Subscale
timeFrame: Phase 1 - Week 4 and 12, Phase 2 - Week 4 and 8
Secondary outcomes:
measure: Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
timeFrame: Week 4 of Phase 1
measure: Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
timeFrame: Week 12 of Phase 1
measure: Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
timeFrame: Week 4 of Phase 2
measure: Social Pervasive Developmental Disorder-Behavior Inventory (PDDBI)
timeFrame: Week 8 of Phase 2
measure: Repetitive Behavior
timeFrame: Phase 1 - Week 4 and 12, Phase 2 - Week 4 and 8
measure: Caregiver Strain
timeFrame: Phase 1 - Week 4 and 12, Phase 2 - Week 4 and 8
measure: Children's Yale-Brown Obsessive Compulsive Disorder Scale for Pervasive Developmental Disorders
timeFrame: Phase 1 - Week 4 and 12, Phase 2 - Week 4 and 8
measure: The Clinical Global Impression - Severity Scale (CGI-S)
timeFrame: Phase 1 - Week 4 and 12, Phase 2 - Week 4 and 8
measure: Vineland Adaptive Behavior Composite Score
timeFrame: Baseline and Phase 1 Week 12
Other outcomes:
Locations:
city: New York
country: United States
facility: Icahn School of Medicine at Mount Sinai
state: New York
Study officials:
affiliation: Icahn School of Medicine at Mount Sinai
name: Alexander Kolevzon, MD
role: PRINCIPAL_INVESTIGATOR
References: