Pilot Trial of Pregnenolone in Autism

NCT ID: NCT02627508

Last Updated: 2026-01-13

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 40 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2016-01-29
• Study Completion Date: 2026-05-31

Brief Summary

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.

Conditions

Autism Spectrum Disorder; Autism

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Pregnenolone (up to 500 mg per day)

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)

Group Type: EXPERIMENTAL

Pregnenolone

Intervention Type: DRUG

orally administered Pregnenolone capsules

Placebo

Placebo

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

orally administered placebo capsules

Interventions

Pregnenolone

orally administered Pregnenolone capsules

Intervention Type: DRUG

Placebo

orally administered placebo capsules

Intervention Type: DRUG

Eligibility Criteria

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 Autism Spectrum Disorder (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.

• Minimum Eligible Age: 14 Years
• Maximum Eligible Age: 25 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Simons Foundation

OTHER

Sponsor Role: collaborator

Stanford University

OTHER

Sponsor Role: lead

Responsible Party

Antonio Hardan

Professor of Psychiatry and Behavioral Sciences

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Antonio Y. Hardan, MD

Role: PRINCIPAL_INVESTIGATOR

Stanford University

Locations

Stanford University School of Medicine

Stanford, California, United States

Countries

United States

Other Identifiers

IRB-34762

Identifier Type: -

Identifier Source: org_study_id