Skip to main content
OpenTrials
Completed

NCT Number: NCT01881737

A Study of Pregnenolone in the Treatment of Individuals With Autism

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.

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Autistic Disorder Autism Spectrum Disorder Child Development Disorders, Pervasive Mental Disorders Neurodevelopmental Disorders

Age range

18 year–45 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Stanford University School of Medicine

Stanford, California, 94305, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Outpatients 18-45 years of age;
  • Males and females who are physically healthy;
  • Diagnosis of autism based on Diagnostic and Statistical Manual (DSM-IV-TR) criteria, the Autism Diagnostic Interview-Revised, and expert clinical evaluation;
  • Total Aberrant Behavior Checklist (ABC) greater then 21;
  • Care provider who can reliably bring subject to clinic visits, can provide trustworthy ratings, and interacts with subject on a regular basis;
  • Ability of subject to swallow the compound;
  • Stable concomitant medications for at least 2 weeks; and
  • No planned changes in psychosocial interventions during the open-label pregnenolone trial.

Exclusion criteria

  • Diagnostic and Statistical Manual (DSM-IV-TR) diagnosis of schizophrenia, schizoaffective disorder, or psychotic disorder, not otherwise specified;
  • Prior adequate trial of pregnenolone;
  • Active medical problems: unstable seizures, significant physical illness (e.g., serious liver or renal pathology);
  • Pregnancy or sexually active females (as determined by a urinary pregnancy test in the beginning of the study); and
  • 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

Treatment and study plan

Pregnenolone

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.

Primary outcomes

  1. 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 outcomes

  1. Social Responsiveness Scale (SRS) Total Score

    Time frame: 12 weeks

    SRS total score (total range 0-195); higher scores mean more abnormal social behaviors.

  2. Sensory Profile Questionnaire Total Score

    Time frame: 12

    scores on a scale (range: 38-190); lower scores mean more abnormal sensory problems.

  3. Vineland Adaptive Behavior Scale

    Time frame: 12 weeks

    Adaptive Behavior Composite Score (score range 20-160); higher scores mean more typical adaptive behaviors.

  4. Repetitive Behavior Scale

    Time frame: 12 weeks

  5. Pregnenolone Level in Peripheral Blood as Measured at Baseline and After 12 Weeks

    Time frame: 12 weeks

Sponsors and collaborators

Lead sponsor

Stanford University

Other

Registry information

Official study title

An Open-Label Pilot Study of Pregnenolone in the Treatment of Individuals With Autism

Important dates

Study start
2011
Primary completion
2013
Study completion
2013
First posted
Jun 20, 2013
Registry last updated
Mar 29, 2017

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.