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NCT Number: NCT02627508

Pilot Trial of Pregnenolone in Autism

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.

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Key information

Conditions

Autism Spectrum Disorder Autism Autistic Disorder Child Development Disorders, Pervasive Mental Disorders Neurodevelopmental Disorders

Age range

14 year–25 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Stanford University School of Medicine

Stanford, California, 94305, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Pregnenolone

Drug

orally administered Pregnenolone capsules

Placebo

Drug

orally administered placebo capsules

Primary outcomes

  1. Change from baseline in parent rated Aberrant Behavior Checklist Irritability (ABC-I) score during treatment

    Time frame: 2-week, 4-week, 6-week, 8-week, 10-week, 14-week

Secondary outcomes

  1. Change from baseline in parent rated Aberrant Behavior Checklist (ABC) scores during treatment

    Time frame: 2-week, 4-week, 6-week, 8-week, 10-week, 14-week

  2. Change from baseline in parent rated Sensory Profile Questionnaire (SPQ) scores during treatment

    Time frame: 2-week, 4-week, 6-week, 8-week, 10-week, 14-week

  3. Change from baseline in Vineland Adaptive Behavior Scales Second Edition (VABS-II)

    Time frame: 2-week, 6-week, 10-week, 14-week

  4. Change from baseline in Clinical Global Impression (CGI) scores during treatment

    Time frame: 2-week, 4-week, 6-week, 8-week, 10-week, 14-week

  5. Change from baseline in levels of pregnenolone and its related neurosteroids in peripheral blood

    Time frame: 14-week

  6. Change from baseline on the Autism Diagnostic Observation Schedule (ADOS)

    Time frame: 14-week

  7. Change from baseline in laboratory based eye-gaze measures

    Time frame: 14-week

  8. Change from baseline in laboratory based electroencephalography (EEG) measures

    Time frame: 14-week

  9. Change from baseline in laboratory clinical lab values including complete metabolic panel, complete blood count with differential, cholesterol panel and routine urinalysis.

    Time frame: 14-week

  10. Change from baseline in parent rated Social Responsiveness Scale (SRS) scores during treatment

    Time frame: 2-week, 6-week, 10-week, 14-week

  11. Change from baseline on the Dosage Record Treatment Emergent Symptom Scale (DOTES) scores during treatment

    Time frame: 2-week, 4-week, 6-week, 8-week, 10-week, 14-week

Sponsors and collaborators

Lead sponsor

Stanford University

Other

Collaborators

  • Simons Foundation

Registry information

Official study title

Randomized Controlled Pilot Trial of Pregnenolone in Autism

Important dates

Study start
2016
Primary completion
2026
Study completion
2026
First posted
Dec 11, 2015
Registry last updated
Mar 30, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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