Pilot Trial of Pregnenolone in Autism

NCT02627508 | Completed Phase 2 DMC

• 1 other identifier: IRB-34762
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 1

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

Keywords

Autism Spectrum Disorder; Autism; Pregnenolone

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (11)

• 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

Study Arms (2)

Pregnenolone (up to 500 mg per day)

EXPERIMENTAL

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

Placebo

PLACEBO COMPARATOR

Placebo

Drug: Placebo

Interventions

Pregnenolone DRUG

orally administered Pregnenolone capsules

Pregnenolone (up to 500 mg per day)

Placebo DRUG

orally administered placebo capsules

Placebo

Eligibility Criteria

• Age: 14 Years - 25 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Stanford University: lead
• Simons Foundation: collaborator

Study Sites (1)

Stanford University School of Medicine

Stanford, California, 94305, United States

Location

MeSH Terms

Conditions

Autism Spectrum Disorder; Autistic Disorder

Interventions

Pregnenolone

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Intervention Hierarchy (Ancestors)

Pregnenes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Hydroxycorticosteroids; Adrenal Cortex Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Progesterone Congeners; Gonadal Steroid Hormones; Gonadal Hormones

Study Officials

• Antonio Y. Hardan, MD

  Stanford University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor of Psychiatry and Behavioral Sciences

Study Record Dates

• First Submitted: December 2, 2015
• First Posted: December 11, 2015
• Study Start: January 29, 2016
• Primary Completion: March 13, 2026
• Study Completion: March 17, 2026
• Last Updated: March 30, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will share

Locations

US (1)