A Study of Pregnenolone in the Treatment of Individuals With Autism

An Open-Label Pilot 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.

Study Overview

• Status: Completed
• Conditions: Autistic Disorder
• Intervention / Treatment: Drug: Pregnenolone

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.

• Study Type: Interventional
• Enrollment (Actual): 15
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • California
    • Stanford, California, United States, 94305
      • Stanford University School of Medicine

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 14 years to 41 years (Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
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)	2, 4, 6, 8, 10, 12, and 16 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Social Responsiveness Scale (SRS) Total Score	SRS total score (total range 0-195); higher scores mean more abnormal social behaviors.	12 weeks
Sensory Profile Questionnaire Total Score	scores on a scale (range: 38-190); lower scores mean more abnormal sensory problems.	12
Vineland Adaptive Behavior Scale	Adaptive Behavior Composite Score (score range 20-160); higher scores mean more typical adaptive behaviors.	12 weeks
Repetitive Behavior Scale		12 weeks
Pregnenolone Level in Peripheral Blood as Measured at Baseline and After 12 Weeks		12 weeks

Collaborators and Investigators

• Sponsor: Stanford University

Publications and helpful links

General Publications

• 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.

Study record dates

Study Major Dates

• Study Start: July 1, 2011
• Primary Completion (Actual): September 1, 2013
• Study Completion (Actual): September 1, 2013

Study Registration Dates

• First Submitted: November 28, 2011
• First Submitted That Met QC Criteria: June 17, 2013
• First Posted (Estimate): June 20, 2013

Study Record Updates

• Last Update Posted (Actual): March 29, 2017
• Last Update Submitted That Met QC Criteria: February 8, 2017
• Last Verified: February 1, 2017

More Information

Terms related to this study

• Keywords: autism
• Additional Relevant MeSH Terms: Mental Disorders; Neurodevelopmental Disorders; Child Development Disorders, Pervasive; Autism Spectrum Disorder; Autistic Disorder
• Other Study ID Numbers: SU-08092011-8246

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Yes