A Study of Pregnenolone in the Treatment of Individuals With Autism

NCT ID: NCT01881737

Last Updated: 2017-03-29

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 15 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2011-07-31
• Study Completion Date: 2013-09-30

Brief Summary

This study will assess the tolerability and effectiveness of pregnenolone in the treatment of behavioral deficits in adults with autism. Pregnenolone is a naturally occurring hormone found in the body which has been shown to help with the function of nerve cells. It is also shown to modulate the activity of certain brain receptors implicated in autism. We hope to examine the tolerability of pregnenolone in adults with autism.

Detailed Description

This study will assess the tolerability and effectiveness of pregnenolone in the treatment of behavioral deficits in adults with autism. Pregnenolone is a naturally occurring hormone found in the body which has been shown to help with the function of nerve cells. It is also shown to modulate the activity of certain brain receptors implicated in autism.

Pregnenolone has been used safely in research studies involving individuals with schizophrenia. In the proposed trial, we hope to examine the tolerability of pregnenolone in adults with autism. We hope to see improvement in behavioral outcomes as measured by standardized behavioral measures. Further, we will measure concentrations of pregnenolone and related neuroactive compounds in the blood. The use of pregnenolone has been studied in a number of mental disorders but not autism. Thus, we hope the study will identify new avenues of research for the treatment of autism.

Conditions

Autistic Disorder

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Pregnenolone

Pregnenolone up to 500 mg per day

Group Type: EXPERIMENTAL

Pregnenolone

Intervention Type: DRUG

With Baseline serving as approximately day 1, twice daily intake of orally administered pregnenolone will occur on a schedule consisting of an up-titration followed by a down-titration as described below.

Week 1 and 2: 100 mg

Week 3 and 4: 200 mg

Week 5 and 6: 300 mg

Week 7 and 8: 400 mg

Week 9 -12: 500 mg

At the end of Week 12, pregnenolone was decreased by 50 mg twice a day every 3 days until it was discontinued.

If the participant is unable to tolerate a specific dose then he/she will be maintained at the highest tolerated dose until down titration occurs.

Interventions

Pregnenolone

With Baseline serving as approximately day 1, twice daily intake of orally administered pregnenolone will occur on a schedule consisting of an up-titration followed by a down-titration as described below.

Week 1 and 2: 100 mg

Week 3 and 4: 200 mg

Week 5 and 6: 300 mg

Week 7 and 8: 400 mg

Week 9 -12: 500 mg

At the end of Week 12, pregnenolone was decreased by 50 mg twice a day every 3 days until it was discontinued.

If the participant is unable to tolerate a specific dose then he/she will be maintained at the highest tolerated dose until down titration occurs.

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

1. Outpatients 18-45 years of age;

2. Males and females who are physically healthy;

3. Diagnosis of autism based on Diagnostic and Statistical Manual (DSM-IV-TR) criteria, the Autism Diagnostic Interview-Revised, and expert clinical evaluation;

4. Total Aberrant Behavior Checklist (ABC) greater then 21;

5. Care provider who can reliably bring subject to clinic visits, can provide trustworthy ratings, and interacts with subject on a regular basis;

6. Ability of subject to swallow the compound;

7. Stable concomitant medications for at least 2 weeks; and

8. No planned changes in psychosocial interventions during the open-label pregnenolone trial.

Exclusion Criteria

1. Diagnostic and Statistical Manual (DSM-IV-TR) diagnosis of schizophrenia, schizoaffective disorder, or psychotic disorder, not otherwise specified;

2. Prior adequate trial of pregnenolone;

3. Active medical problems: unstable seizures, significant physical illness (e.g., serious liver or renal pathology);

4. Pregnancy or sexually active females (as determined by a urinary pregnancy test in the beginning of the study); and

5. Subjects taking oil or fat based nutritional supplements will be excluded from the study unless they have been off these compounds for at least 4 weeks

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 45 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Stanford University

OTHER

Sponsor Role: lead

Responsible Party

Antonio Hardan

Professor

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Antonio Hardan, MD

Role: PRINCIPAL_INVESTIGATOR

Stanford University

Locations

Stanford University School of Medicine

Stanford, California, United States

Countries

United States

References

Fung LK, Libove RA, Phillips J, Haddad F, Hardan AY. Brief report: an open-label study of the neurosteroid pregnenolone in adults with autism spectrum disorder. J Autism Dev Disord. 2014 Nov;44(11):2971-7. doi: 10.1007/s10803-014-2144-4.

Reference Type: RESULT

PMID: 24849255 (View on PubMed)

Other Identifiers

SU-08092011-8246

Identifier Type: -

Identifier Source: org_study_id