A Study of Pregnenolone in the Treatment of Individuals With Autism

Study Overview
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Brief Summary
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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
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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.

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

Conditions
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Autistic Disorder

Intervention / Treatment
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* Drug: Pregnenolone


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 45 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Pregnenolone<br>Pregnenolone up to 500 mg per day | Drug: Pregnenolone<br>* 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.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | 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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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autism