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

ID#: NCT01970345

Age: 5 - 12 years

Gender: All

Healthy Subjects: No

Study Phase: Phase 2

Recruitment Status: Recruiting

Start Date: May 01, 2014

End Date: January 01, 2020

Contact Information:
Lexie Massa
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.

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