An Open-Label Pilot Study of Pregnenolone in the Treatment of Individuals With Autism
Trial Snapshot
- Phase
- Phase 2
- Status
- Completed
- Sponsor
- Stanford University
- Enrollment
- 15
- Locations
- 1
- Primary Endpoint
- 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)
Study Overview
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.
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 18 Years to 45 Years (Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
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
Arms & Interventions
Pregnenolone
Pregnenolone up to 500 mg per day
Intervention: Pregnenolone (Drug)
Outcomes
Primary Outcomes
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
- Pregnenolone Level in Peripheral Blood as Measured at Baseline and After 12 Weeks(12 weeks)
- Social Responsiveness Scale (SRS) Total Score(12 weeks)
- Sensory Profile Questionnaire Total Score(12)
- Vineland Adaptive Behavior Scale(12 weeks)
- Repetitive Behavior Scale(12 weeks)
Investigators
Antonio Hardan
Professor
Stanford University