Autistic DisorderPhase 2NCT01881737

A Study of Pregnenolone in the Treatment of Individuals With Autism

A Phase 2 study testing Pregnenolone in people with Autistic Disorder, run by Stanford University at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

Who is it for?: Ages 18 to 45 years
Conditions the study is for: Autistic Disorder
What is being tested?: Pregnenolone
Type of treatment: Pill or oral medicine
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 15 participants
Where: 1 siteUnited States
Study dates: Jul 2011to Sep 2013
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

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 18 to 45 years
All sexes
No healthy volunteers
Adult

Who may be able to join

Outpatients 18-45 years of age;
Males and females who are physically healthy;
Diagnosis of autism based on Diagnostic and Statistical Manual (DSM-IV-TR) criteria, the Autism Diagnostic Interview-Revised, and expert clinical evaluation;
Total Aberrant Behavior Checklist (ABC) greater then 21;
Care provider who can reliably bring subject to clinic visits, can provide trustworthy ratings, and interacts with subject on a regular basis;
Ability of subject to swallow the compound;
Stable concomitant medications for at least 2 weeks; and
No planned changes in psychosocial interventions during the open-label pregnenolone trial.

Who cannot join

Diagnostic and Statistical Manual (DSM-IV-TR) diagnosis of schizophrenia, schizoaffective disorder, or psychotic disorder, not otherwise specified;
Prior adequate trial of pregnenolone;
Active medical problems: unstable seizures, significant physical illness (e.g., serious liver or renal pathology);
Pregnancy or sexually active females (as determined by a urinary pregnancy test in the beginning of the study); and
Subjects taking oil or fat based nutritional supplements will be excluded from the study unless they have been off these compounds for at least 4 weeks

Trial locations and contacts

1 site in 1 country.

United States1 site

Stanford University School of Medicine
Stanford, California, 94305

Other trials in United States

About this study

Official title: An Open-Label Pilot Study of Pregnenolone in the Treatment of Individuals With Autism

This study will assess the tolerability and effectiveness of pregnenolone in the treatment of behavioral deficits in adults with autism. Pregnenolone is a naturally occurring hormone found in the body which has been shown to help with the function of nerve cells. It is also shown to modulate the activity of certain brain receptors implicated in autism. We hope to examine the tolerability of pregnenolone in adults with autism.

Read the detailed description

Pregnenolone has been used safely in research studies involving individuals with schizophrenia. In the proposed trial, we hope to examine the tolerability of pregnenolone in adults with autism. We hope to see improvement in behavioral outcomes as measured by standardized behavioral measures. Further, we will measure concentrations of pregnenolone and related neuroactive compounds in the blood. The use of pregnenolone has been studied in a number of mental disorders but not autism. Thus, we hope the study will identify new avenues of research for the treatment of autism.

Conditions: Autistic Disorder
Being tested: Pregnenolone
Keywords: autism
Medical subject headings: Autistic Disorder

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

PregnenoloneDrug
With Baseline serving as approximately day 1, twice daily intake of orally administered pregnenolone will occur on a schedule consisting of an up-titration followed by a down-titration as described below.
Week 1 and 2: 100 mg
Week 3 and 4: 200 mg
Week 5 and 6: 300 mg
Week 7 and 8: 400 mg
Week 9 -12: 500 mg
At the end of Week 12, pregnenolone was decreased by 50 mg twice a day every 3 days until it was discontinued.
If the participant is unable to tolerate a specific dose then he/she will be maintained at the highest tolerated dose until down titration occurs.

Study groups in NCT01881737
Study group	Role	What they receive
Pregnenolone	Experimental	Pregnenolone up to 500 mg per day Receives: Drug: Pregnenolone

What is being measured

The primary outcome is the main question the study is designed to answer.

Number of Participants With Adverse Events According to Dosage Record and Treatment Emergent Symptom (DOTES) as Assessed at All Follow-up Visits (2, 4, 6, 8, 10, 12, and 16 Weeks)
Measured: 2, 4, 6, 8, 10, 12, and 16 weeks

Secondary outcomes (5)

Social Responsiveness Scale (SRS) Total Score
SRS total score (total range 0-195); higher scores mean more abnormal social behaviors.
Measured: 12 weeks
Sensory Profile Questionnaire Total Score
scores on a scale (range: 38-190); lower scores mean more abnormal sensory problems.
Measured: 12
Vineland Adaptive Behavior Scale
Adaptive Behavior Composite Score (score range 20-160); higher scores mean more typical adaptive behaviors.
Measured: 12 weeks
Repetitive Behavior Scale
Measured: 12 weeks
Pregnenolone Level in Peripheral Blood as Measured at Baseline and After 12 Weeks
Measured: 12 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Jul 2011Sep 2013

First posted20 Jun 2013 (estimated)
Study startJul 2011
Primary completionSep 2013
Study completionSep 2013

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Autistic 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: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 15 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01881737
Sponsor's protocol ID: SU-08092011-8246
Status last verified: Feb 2017
First submitted: 28 Nov 2011
Last updated: 29 Mar 2017
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
Individual data sharing: Yes

Publications

Fung LK, Libove RA, Phillips J, Haddad F, Hardan AY. Brief report: an open-label study of the neurosteroid pregnenolone in adults with autism spectrum disorder. J Autism Dev Disord. 2014 Nov;44(11):2971-7. doi: 10.1007/s10803-014-2144-4.
PubMed 24849255

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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All Autistic Disorder trials

Study at a glance

From the registry record, last updated by the sponsor on 29 Mar 2017. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: An Open-Label Pilot Study of Pregnenolone in the Treatment of Individuals With Autism
ClinicalTrials.gov ID: NCT01881737
Acronym / sponsor ID: SU-08092011-8246
Conditions: Autistic Disorder
Keywords: autism
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 15 participants (actual)
Lead sponsor: Stanford UniversityUniversity, hospital or other
Study start: Jul 2011
Primary completion: Sep 2013 (actual)
Study completion: Sep 2013 (actual)
First posted: 20 Jun 2013 (estimated)
Results: Posted 29 Mar 2017

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Pregnenolone	Experimental	Drug: PregnenolonePregnenolone up to 500 mg per day

Who may qualify

Ages: 18 to 45 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult

Key inclusion criteria (8 listed)

Outpatients 18-45 years of age;
Males and females who are physically healthy;
Diagnosis of autism based on Diagnostic and Statistical Manual (DSM-IV-TR) criteria, the Autism Diagnostic Interview-Revised, and expert clinical evaluation;
Total Aberrant Behavior Checklist (ABC) greater then 21;
Care provider who can reliably bring subject to clinic visits, can provide trustworthy ratings, and interacts with subject on a regular basis;

Key exclusion criteria (5 listed)

Diagnostic and Statistical Manual (DSM-IV-TR) diagnosis of schizophrenia, schizoaffective disorder, or psychotic disorder, not otherwise specified;
Prior adequate trial of pregnenolone;
Active medical problems: unstable seizures, significant physical illness (e.g., serious liver or renal pathology);
Pregnancy or sexually active females (as determined by a urinary pregnancy test in the beginning of the study); and
Subjects taking oil or fat based nutritional supplements will be excluded from the study unless they have been off these compounds for at least 4 weeks

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
PregnenoloneWith Baseline serving as approximately day 1, twice daily intake of orally administered pregnenolone will occur on a schedule consisting of an up-titration followed by a down-titration as described below. Week 1 and 2: 100 mg Week 3 and 4: 200 mg Week 5 and 6: 300 mg Week 7 and 8: 400 mg Week 9 -12: 500 mg At the end of Week 12, pregnenolone was decreased by 50 mg twice a day every 3 days until it was discontinued. If the participant is unable to tolerate a specific dose then he/she will be maintained at the highest tolerated dose until down titration occurs.	Drug	Pregnenolone

Primary outcome

Number of Participants With Adverse Events According to Dosage Record and Treatment Emergent Symptom (DOTES) as Assessed at All Follow-up Visits (2, 4, 6, 8, 10, 12, and 16 Weeks)
Time frame: 2, 4, 6, 8, 10, 12, and 16 weeks

5 secondary outcomes

Social Responsiveness Scale (SRS) Total Score
Time frame: 12 weeks
Sensory Profile Questionnaire Total Score
Time frame: 12
Vineland Adaptive Behavior Scale
Time frame: 12 weeks
Repetitive Behavior Scale
Time frame: 12 weeks
Pregnenolone Level in Peripheral Blood as Measured at Baseline and After 12 Weeks
Time frame: 12 weeks

Results and safety

Results posted to the registry on 29 Mar 2017, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Pregnenolone	15	10

Primary outcome: Number of Participants With Adverse Events According to Dosage Record and Treatment Emergent Symptom (DOTES) as Assessed at All Follow-up Visits (2, 4, 6, 8, 10, 12, and 16 Weeks)

Time frame: 2, 4, 6, 8, 10, 12, and 16 weeks. Number. Unit: participants

Measure	Pregnenolone n = 12
Tiredness	1
Diarrhea	2
Depressive Affect	2
Increased Excitement/Agitation	3
Sleep Problems	1
Drowsiness	1
Anorexia/Decreased Appetite	2
Increased Motor Activity	1

Population analysed: During the 12-week treatment period, two participants dropped out of the study. The follow-up observations for the two participants who dropped out were included in the analyses.

5 secondary outcomes are reported in the full results.

Adverse events

Time frame: Time Frame: Baseline, 2, 4, 6, 8, 10, 12, and 16 weeks. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Pregnenolone	0 of 12 (0%)	6 of 12 (50%)	Not reported

Limitations and caveats (from the sponsor): This is an open-label trial with a very small sample size. No measures of plasma or salivary concentrations of metabolites were completed in this study.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 29 Mar 2017.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Results publication: PubMed 24849255Fung LK, Libove RA, Phillips J, Haddad F, Hardan AY. Brief report: an open-label study of the neurosteroid pregnenolone in adults with autism spectrum disorder. J Autism Dev Disord. 2014 Nov;44(11):2971-7. doi: 10.1007/s10803-014-2144-4.
Individual participant dataSharing planned: Yes

Related trials for Autistic Disorder

Recruiting
Cumulative and Booster Effects of Multisession Prefrontal tDCS in ASD Adolescents
Transcranial Direct Current Stimulation, Autistic Spectrum Disorder
RecruitingPhase 3
A Study to Evaluate the Long-term Safety and Tolerability of Xanomeline and Trospium Chloride for the Treatment of Psychiatric Disorders in Children and Adolescents
Schizophrenia, Autism-Related Irritability
Recruiting
Neuroimaging Predictors of Improvement to Pivotal Response Treatment (PRT) in Young Children With Autism
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RecruitingPhase 2
A Study of Esomeprazole in Children With Autism
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Recruiting
Transcranial Magnetic Stimulation for Restricted and Repetitive Behavior in ASD
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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 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 Autistic Disorder, 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 8 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 29 Mar 2017. 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. 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?

Yes. Results are posted in the registry (first posted 29 Mar 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Number of Participants With Adverse Events According to Dosage Record and Treatment Emergent Symptom (DOTES) as Assessed at All Follow-up Visits (2, 4, 6, 8, 10, 12, and 16 Weeks) (time frame: 2, 4, 6, 8, 10, 12, and 16 weeks). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01881737, which also lists 1 linked publication.

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 29 Mar 2017; AzTrials last refreshed its copy on 8 Nov 2026.