Pilot Trial of Pregnenolone in Autism

Study Overview
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Brief Summary
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This is a research study to examine the tolerability and effectiveness of pregnenolone in individuals with autism. Pregnenolone is a naturally occurring steroid hormone in the brain that has been implicated in treating various psychiatric conditions. The investigators hope to learn the effects and safety of using pregnenolone in reducing irritability and sensitivity to sensory differences and improving social communication in individuals with autism. The investigators hope by studying the effects of pregnenolone in more detail, the investigators can design better ways to treat individuals with autism.

Official Title
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Randomized Controlled Pilot Trial of Pregnenolone in Autism

Conditions
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Autism, Autism Spectrum Disorder

Intervention / Treatment
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* Drug: Pregnenolone
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: outpatients between 14 and 25 years of age with a Tanner stage of IV or V; male and female subjects who were physically healthy; diagnosis of ASD based on DSM-5, expert clinical opinion and confirmed with Autism Diagnostic Interview - Revised (ADI-R) and either Autism Diagnostic Observation Schedule (ADOS) or Childhood Autism Rating Scale (CARS-2); Aberrant Behavior Checklist -Irritability (ABC-I)≥ 18 and Clinical Global Impression (CGI)-Severity subscale ≥ 4; stable concomitant medications for at least 2 weeks; no planned changes in psychosocial interventions during the trial. Exclusion Criteria: Diagnostic and Statistical Manual (DSM-5) diagnosis of schizophrenia, schizoaffective disorder, alcohol use disorder; prior adequate trial of pregnenolone; active medical problems: unstable seizures (>2 in past month), significant physical illness; pregnant or sexually active female subjects who do not adhere to use an appropriate form of external prophylactics; participants taking steroid medications.

Ages Eligible for Study
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Minimum Age: 14 Years
Maximum Age: 25 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
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Pregnenolone (up to 500 mg per day)<br>Twice daily intake of orally administered Pregnenolone will occur on a schedule as described below. Weeks 1 and 2: 30mg twice daily (total 60mg per day) Weeks 3 and 4: 60mg twice daily (total: 120mg per day) Weeks 5 and 6: 90mg twice daily (total: 180mg per day) Weeks 7 and 8: 150mg twice daily (total: 300mg per day) Weeks 9 and 10: 210mg twice daily (total: 420mg per day) Weeks 11 to 14: 250mg twice daily (total: 500mg per day) | Drug: Pregnenolone<br>* orally administered Pregnenolone capsules<br>|
| Placebo Comparator: Placebo<br>Placebo | Drug: Placebo<br>* orally administered placebo capsules<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change from baseline in parent rated Aberrant Behavior Checklist Irritability (ABC-I) score during treatment |  | 2-week, 4-week, 6-week, 8-week, 10-week, 14-week | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change from baseline in parent rated Aberrant Behavior Checklist (ABC) scores during treatment |  | 2-week, 4-week, 6-week, 8-week, 10-week, 14-week | 
| Change from baseline in parent rated Sensory Profile Questionnaire (SPQ) scores during treatment |  | 2-week, 4-week, 6-week, 8-week, 10-week, 14-week | 
| Change from baseline in Vineland Adaptive Behavior Scales Second Edition (VABS-II) |  | 2-week, 6-week, 10-week, 14-week | 
| Change from baseline in Clinical Global Impression (CGI) scores during treatment |  | 2-week, 4-week, 6-week, 8-week, 10-week, 14-week | 
| Change from baseline in levels of pregnenolone and its related neurosteroids in peripheral blood |  | 14-week | 
| Change from baseline on the Autism Diagnostic Observation Schedule (ADOS) |  | 14-week | 
| Change from baseline in laboratory based eye-gaze measures |  | 14-week | 
| Change from baseline in laboratory based electroencephalography (EEG) measures |  | 14-week | 
| Change from baseline in laboratory clinical lab values including complete metabolic panel, complete blood count with differential, cholesterol panel and routine urinalysis. |  | 14-week | 
| Change from baseline in parent rated Social Responsiveness Scale (SRS) scores during treatment |  | 2-week, 6-week, 10-week, 14-week | 
| Change from baseline on the Dosage Record Treatment Emergent Symptom Scale (DOTES) scores during treatment |  | 2-week, 4-week, 6-week, 8-week, 10-week, 14-week | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Autism Spectrum Disorder, Autism, Pregnenolone