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A Study of Pregnenolone in the Treatment of Individuals With Autism

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01881737
First Posted: June 20, 2013
Last Update Posted: March 29, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Antonio Hardan, Stanford University
  Purpose
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.

Condition Intervention Phase
Autistic Disorder Drug: Pregnenolone Phase 2

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label Pilot Study of Pregnenolone in the Treatment of Individuals With Autism

Resource links provided by NLM:


Further study details as provided by Antonio Hardan, Stanford University:

Primary Outcome Measures:
  • Number of Participants With Adverse Events According to Dosage Record and Treatment Emergent Symptom (DOTES) as Assessed at All Follow-up Visits (2, 4, 6, 8, 10, 12, and 16 Weeks) [ Time Frame: 2, 4, 6, 8, 10, 12, and 16 weeks ]

Secondary Outcome Measures:
  • Social Responsiveness Scale (SRS) Total Score [ Time Frame: 12 weeks ]
    SRS total score (total range 0-195); higher scores mean more abnormal social behaviors.

  • Sensory Profile Questionnaire Total Score [ Time Frame: 12 ]
    scores on a scale (range: 38-190); lower scores mean more abnormal sensory problems.

  • Vineland Adaptive Behavior Scale [ Time Frame: 12 weeks ]
    Adaptive Behavior Composite Score (score range 20-160); higher scores mean more typical adaptive behaviors.

  • Repetitive Behavior Scale [ Time Frame: 12 weeks ]
  • Pregnenolone Level in Peripheral Blood as Measured at Baseline and After 12 Weeks [ Time Frame: 12 weeks ]

Enrollment: 15
Study Start Date: July 2011
Study Completion Date: September 2013
Primary Completion Date: September 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Pregnenolone
Pregnenolone up to 500 mg per day
Drug: 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.


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.

  Eligibility

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   18 Years to 45 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01881737


Locations
United States, California
Stanford University School of Medicine
Stanford, California, United States, 94305
Sponsors and Collaborators
Stanford University
Investigators
Principal Investigator: Antonio Hardan, MD Stanford University
  More Information

Publications:
Responsible Party: Antonio Hardan, Professor, Stanford University
ClinicalTrials.gov Identifier: NCT01881737     History of Changes
Other Study ID Numbers: SU-08092011-8246
First Submitted: November 28, 2011
First Posted: June 20, 2013
Results First Submitted: August 12, 2016
Results First Posted: March 29, 2017
Last Update Posted: March 29, 2017
Last Verified: February 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes

Keywords provided by Antonio Hardan, Stanford University:
autism

Additional relevant MeSH terms:
Autistic Disorder
Child Development Disorders, Pervasive
Neurodevelopmental Disorders
Mental Disorders