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Interagency Autism Coordinating Committee (IACC)
Autism Research Database
Office of Autism Research Coordination (OARC)
 
Project Element Element Description

Project Title

Project Title

Human Clinical Trial of IGF-1 in Children with Idiopathic ASD

Principal Investigator

Principal Investigator

Kolevzon, Alexander

Description

Description

A double-blind, randomized, placebo controlled, phase 2 human clinical trial of Insulin-Like Growth Factor-1 (IGF-1) in children with a genetic cause of autism (Phelan McDermid Syndrome) is currently underway at the Seaver Autism Center for Research and Treatment at Mount Sinai.  That study builds on previous work in a mouse model system of Phelan McDermid Syndrome which showed that IGF-1 treatment reversed the effects of disrupted glutamate signaling associated with impaired learning and memory. This Treatment Grant will expand the current study to add a cohort of children with autism but without Phelan McDermid Syndrome. IGF-1 is a commercially available compound that is known to promote synaptic maturation and plasticity. It has already been shown to reverse behavioral and physiological deficits associated with Rett Syndrome in mouse models and preliminary results in children with Rett syndrome are promising. Results from this trial are expected to provide evidence that IGF-1 is safe, well tolerated, and efficacious in targeting core symptoms of autism in children without Phelan McDermid Syndrome because of the potential that the glutamate signaling pathway is relevant to diverse forms of autism.

Funder

Funder

Autism Science Foundation

Fiscal Year Funding

Fiscal Year Funding

0

Current Award Period

Current Award Period

2013-2015

Strategic Plan Question

Strategic Plan Question

Question 4: Which Treatments And Interventions Will Help?

Strategic Plan Objective

Strategic Plan Objective

Yellow dot: Objective has some degree of funding, but less than the recommended amount. 4LC. Conduct at least one study to evaluate the safety and effectiveness of medications commonly used in the treatment of co-occurring conditions or specific behavioral issues in people with ASD by 2015. IACC Recommended Budget: $10,000,000 over 5 years.

Project Link

Project Link

Human Clinical Trial of IGF-1 in Children with Idiopathic ASD (External web link)

Institution

Institution

ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI

State/Country

State/Country

New York

Project Number

Project Number

Federal or Private?

Federal or Private?

Private

Received ARRA Funding?

Received ARRA Funding?

No

History/Related Projects

History/Related Projects

Human Clinical Trial of IGF-1 in Children with Idiopathic ASD | 0 | 2014 |
Human Clinical Trial of IGF-1 in Children with Idiopathic ASD | 25000 | 2013 | n/a

 
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