Autism Spectrum DisorderPhase 2NCT02627508
Pilot Trial of Pregnenolone in Autism
A Phase 2 study testing Pregnenolone in people with Autism Spectrum Disorder and Autism, run by Stanford University at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 14 to 25 years
- Conditions the study is for
- Autism Spectrum Disorder, Autism
- What is being tested?
- Pregnenolone
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 40 participants
- Where
- 1 siteUnited States
- Study dates
- 29 Jan 2016to 17 Mar 2026
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 14 to 25 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
Stanford University School of Medicine
Stanford, California, 94305
About this study
Official title: Randomized Controlled 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.
- Conditions
- Autism Spectrum Disorder, Autism
- Being tested
- Pregnenolone, Placebo
- Keywords
- Autism Spectrum Disorder, Autism, Pregnenolone
- Medical subject headings
- Autism Spectrum Disorder, Autistic Disorder
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PregnenoloneDrug
orally administered Pregnenolone capsules
PlaceboDrug
orally administered placebo capsules
| Study group | Role | What they receive |
|---|---|---|
| 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) Receives: Drug: Pregnenolone |
| Placebo | Placebo comparator | Placebo Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline in parent rated Aberrant Behavior Checklist Irritability (ABC-I) score during treatment
Measured: 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
Measured: 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
Measured: 2-week, 4-week, 6-week, 8-week, 10-week, 14-week
Change from baseline in Vineland Adaptive Behavior Scales Second Edition (VABS-II)
Measured: 2-week, 6-week, 10-week, 14-week
Change from baseline in Clinical Global Impression (CGI) scores during treatment
Measured: 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
Measured: 14-week
Change from baseline on the Autism Diagnostic Observation Schedule (ADOS)
Measured: 14-week
Change from baseline in laboratory based eye-gaze measures
Measured: 14-week
Change from baseline in laboratory based electroencephalography (EEG) measures
Measured: 14-week
Change from baseline in laboratory clinical lab values including complete metabolic panel, complete blood count with differential, cholesterol panel and routine urinalysis.
Measured: 14-week
Change from baseline in parent rated Social Responsiveness Scale (SRS) scores during treatment
Measured: 2-week, 6-week, 10-week, 14-week
Change from baseline on the Dosage Record Treatment Emergent Symptom Scale (DOTES) scores during treatment
Measured: 2-week, 4-week, 6-week, 8-week, 10-week, 14-week
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Dec 2015 (estimated)
- Study start29 Jan 2016
- Primary completion13 Mar 2026
- Study completion17 Mar 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Autism Spectrum DisorderStudies of the same condition, open and under way.
- More trials run by Stanford UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 40 participants (actual)
Sponsor and investigators
- Lead sponsor
- Stanford University
- Sponsor type
- University, hospital or other
- Collaborators
- Simons Foundation
- Responsible party
- Principal investigator, Antonio Hardan, Stanford University
- Investigators
- Antonio Y. Hardan, MD, Principal investigator, Stanford University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02627508
- Sponsor's protocol ID
- IRB-34762
- Status last verified
- Mar 2026
- First submitted
- 2 Dec 2015
- Last updated
- 30 Mar 2026
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 30 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Randomized Controlled Pilot Trial of Pregnenolone in Autism
- ClinicalTrials.gov ID
- NCT02627508
- Acronym / sponsor ID
- IRB-34762
- Conditions
- Autism Spectrum Disorder, Autism
- Keywords
- Autism Spectrum Disorder, Autism, Pregnenolone
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 40 participants (actual)
- Lead sponsor
- Stanford UniversityUniversity, hospital or other
- Collaborators
- Simons Foundation
- Study start
- 29 Jan 2016 (actual)
- Primary completion
- 13 Mar 2026 (actual)
- Study completion
- 17 Mar 2026 (actual)
- First posted
- 11 Dec 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Pregnenolone (up to 500 mg per day) | Experimental | Drug: PregnenoloneTwice 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) |
| Placebo | Placebo comparator | Drug: PlaceboPlacebo |
Who may qualify
- Ages
- 14 to 25 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- 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;
Key exclusion criteria (5 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Pregnenoloneorally administered Pregnenolone capsules | Drug | Pregnenolone (up to 500 mg per day) |
| Placeboorally administered placebo capsules | Drug | Placebo |
Primary outcome
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
11 secondary outcomes
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
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
Change from baseline in Vineland Adaptive Behavior Scales Second Edition (VABS-II)
Time frame: 2-week, 6-week, 10-week, 14-week
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
Change from baseline in levels of pregnenolone and its related neurosteroids in peripheral blood
Time frame: 14-week
Change from baseline on the Autism Diagnostic Observation Schedule (ADOS)
Time frame: 14-week
Change from baseline in laboratory based eye-gaze measures
Time frame: 14-week
Change from baseline in laboratory based electroencephalography (EEG) measures
Time frame: 14-week
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
Change from baseline in parent rated Social Responsiveness Scale (SRS) scores during treatment
Time frame: 2-week, 6-week, 10-week, 14-week
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Mar 2026.
- Individual participant dataSharing planned: Yes
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Antonio Y. Hardan, MD principal investigator
Stanford University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Pregnenolone in people with Autism Spectrum Disorder and Autism, run by Stanford University at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 30 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Mar 2026.