Back to resultsA Study of Pregnenolone in the Treatment of Individuals With Autism
Primary Purpose
Autistic Disorder
Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Pregnenolone
Sponsored by
About this trial
This is an interventional treatment trial for Autistic Disorder focused on measuring autism
Eligibility Criteria
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
Sites / Locations
- Stanford University School of Medicine
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Pregnenolone
Arm Description
Pregnenolone up to 500 mg per day
Outcomes
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)
Secondary Outcome Measures
Social Responsiveness Scale (SRS) Total Score
SRS total score (total range 0-195); higher scores mean more abnormal social behaviors.
Sensory Profile Questionnaire Total Score
scores on a scale (range: 38-190); lower scores mean more abnormal sensory problems.
Vineland Adaptive Behavior Scale
Adaptive Behavior Composite Score (score range 20-160); higher scores mean more typical adaptive behaviors.
Repetitive Behavior Scale
Pregnenolone Level in Peripheral Blood as Measured at Baseline and After 12 Weeks
Full Information
NCT ID
NCT01881737
First Posted
November 28, 2011
Last Updated
February 8, 2017
Sponsor
Stanford University
1. Study Identification
Unique Protocol Identification Number
NCT01881737
Brief Title
A Study of Pregnenolone in the Treatment of Individuals With Autism
Official Title
An Open-Label Pilot Study of Pregnenolone in the Treatment of Individuals With Autism
Study Type
Interventional
2. Study Status
Record Verification Date
February 2017
Overall Recruitment Status
Completed
Study Start Date
July 2011 (undefined)
Primary Completion Date
September 2013 (Actual)
Study Completion Date
September 2013 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Stanford University
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Autistic Disorder
Keywords
autism
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
15 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Pregnenolone
Arm Type
Experimental
Arm Description
Pregnenolone up to 500 mg per day
Intervention Type
Drug
Intervention Name(s)
Pregnenolone
Intervention Description
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 Outcome Measure Information:
Title
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 Measure Information:
Title
Social Responsiveness Scale (SRS) Total Score
Description
SRS total score (total range 0-195); higher scores mean more abnormal social behaviors.
Time Frame
12 weeks
Title
Sensory Profile Questionnaire Total Score
Description
scores on a scale (range: 38-190); lower scores mean more abnormal sensory problems.
Time Frame
12
Title
Vineland Adaptive Behavior Scale
Description
Adaptive Behavior Composite Score (score range 20-160); higher scores mean more typical adaptive behaviors.
Time Frame
12 weeks
Title
Repetitive Behavior Scale
Time Frame
12 weeks
Title
Pregnenolone Level in Peripheral Blood as Measured at Baseline and After 12 Weeks
Time Frame
12 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
45 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Antonio Hardan, MD
Organizational Affiliation
Stanford University
Official's Role
Principal Investigator
Facility Information:
Facility Name
Stanford University School of Medicine
City
Stanford
State/Province
California
ZIP/Postal Code
94305
Country
United States
12. IPD Sharing Statement
Plan to Share IPD
Yes
Citations:
PubMed Identifier
24849255
Citation
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.
Results Reference
result
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A Study of Pregnenolone in the Treatment of Individuals With Autism
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