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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

Randomized Controlled Pilot Trial of Pregnenolone in Autism

Principal Investigator

Principal Investigator

Hardan, Antonio

Description

Description

Medications for treating the core symptoms of autism spectrum disorder (ASD) continue to be an unmet need. The only medications approved by the U.S. Food and Drug Administration (FDA) for the treatment of individuals with ASD are effective in treating irritability and associated aggressive behaviors, but these medications can also cause severe long-term side effects such as diabetes and involuntary motor movements. Effective medications with more tolerable side effect profiles are highly desirable. Antonio Hardan and his colleagues at Stanford University are planning to examine the effectiveness of pregnenolone in the treatment of adolescents with ASD. Pregnenolone belongs to a new class of hormones known as neurosteroids, which have been shown to be effective in treating various psychiatric conditions, including bipolar depression and schizophrenia. Compared with current FDA-approved medications, preliminary data from Hardan’s group in a small open-label study of pregnenolone suggests that this compound represents a potentially effective and well-tolerated agent for treating irritability in individuals with ASD 1. In addition, preliminary evidence suggests that pregnenolone may be helpful in improving select core ASD symptoms, such as social deficits and sensory abnormalities. Hardan’s team aims to extend their work by performing a 12-week randomized, double-blind, placebo-controlled pilot trial to examine the effectiveness of orally administered pregnenolone in reducing irritability and associated behaviors in adolescents with ASD. As ASD is a heterogeneous disorder, the team also plans to examine the usefulness of biomeasures (including blood levels of neurosteroids, eye-tracking and brain wave recording) in predicting treatment response and assessing biological changes with pregnenolone treatment. The use of biomeasures may help identify whether a select subgroup of ASD individuals are responsive to pregnenolone. The results of this pilot trial will further clarify the potential utility of pregnenolone as a medical treatment for ASD. References: 1. Fung L.K. et al. J. Autism Dev. Disord. 44, 2971-2977 (2014) PubMed

Funder

Funder

Simons Foundation

Fiscal Year Funding

Fiscal Year Funding

0

Current Award Period

Current Award Period

2015-2017

Strategic Plan Question

Strategic Plan Question

Question 4: Which Treatments And Interventions Will Help?

Strategic Plan Objective

Strategic Plan Objective

Green dot: Objective has greater than or equal to the recommended funding. 4LA. Complete at least three randomized controlled trials on medications targeting core symptoms in people with ASD of all ages by 2014. IACC Recommended Budget: $22,200,000 over 5 years.

Project Link

Project Link

Randomized Controlled Pilot Trial of Pregnenolone in Autism (External web link)

Institution

Institution

Stanford University

State/Country

State/Country

California

Project Number

Project Number

345949

Federal or Private?

Federal or Private?

Private

Received ARRA Funding?

Received ARRA Funding?

No

History/Related Projects

History/Related Projects

N/A

 
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