Examining the effects of pregnenolone in individuals with autism

Randomized Controlled Pilot Trial of Pregnenolone in Autism

Phase 2 Interventional Stanford University · NCT02627508

This study is testing whether pregnenolone can help young people with autism feel less irritable and more comfortable in social situations.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment40 (estimated)
Ages14 Years to 25 Years
SexAll
SponsorStanford University Academic / other
Locations1 site (Stanford, California)
Trial IDNCT02627508 on ClinicalTrials.gov

What this trial studies

This pilot trial investigates the tolerability and effectiveness of pregnenolone, a naturally occurring steroid hormone, in individuals diagnosed with Autism Spectrum Disorder (ASD). The study aims to assess how pregnenolone may reduce irritability and sensory sensitivity while improving social communication. Participants aged 14 to 25 will receive either pregnenolone or a placebo, with careful monitoring of their responses and safety throughout the trial. The findings could inform future treatment strategies for autism.

Who should consider this trial

Good fit: Ideal candidates are outpatients aged 14 to 25 with a diagnosis of Autism Spectrum Disorder and specific behavioral criteria.

Not a fit: Patients with schizophrenia, significant medical issues, or those who have previously tried pregnenolone may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could lead to improved treatment options for individuals with autism, enhancing their quality of life.

How similar studies have performed: While this approach is novel in the context of autism, similar studies exploring hormonal treatments for psychiatric conditions have shown promise.

Eligibility criteria

Show full inclusion / exclusion 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.

Where this trial is running

Stanford, California

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions Autism Spectrum DisorderAutismPregnenolone
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.