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Pilot Trial of Pregnenolone in Autism

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ClinicalTrials.gov Identifier: NCT02627508
Recruitment Status : Recruiting
First Posted : December 11, 2015
Last Update Posted : May 9, 2019
Sponsor:
Collaborator:
Simons Foundation
Information provided by (Responsible Party):
Antonio Hardan, Stanford University

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.

Condition or disease Intervention/treatment Phase
Autism Spectrum Disorder Autism Drug: Pregnenolone Drug: Placebo Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 40 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Randomized Controlled Pilot Trial of Pregnenolone in Autism
Actual Study Start Date : January 29, 2016
Estimated Primary Completion Date : December 2019
Estimated Study Completion Date : December 2019

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Pregnenolone (up to 500 mg per day)

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
orally administered Pregnenolone capsules

Placebo Comparator: Placebo
Placebo
Drug: Placebo
orally administered placebo capsules




Primary Outcome Measures :
  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 Outcome Measures :
  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 ]


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Ages Eligible for Study:   14 Years to 25 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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 Autism Diagnostic Observation Schedule (ADOS);
  • 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02627508


Contacts
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Contact: Lakshmi Vrittamani (650) 736-1235 vrittama@stanford.edu
Contact: Jaelyn Edwards (650) 736-1235 jedwrds@stanford.edu

Locations
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United States, California
Stanford University School of Medicine Recruiting
Stanford, California, United States, 94305
Contact: Robin Libove    650-736-1235    rlibove@stanford.edu   
Principal Investigator: Antonio Y. Hardan, MD         
Sub-Investigator: Lawrence K. Fung, MD, PhD         
Sponsors and Collaborators
Stanford University
Simons Foundation
Investigators
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Principal Investigator: Antonio Y. Hardan, MD Stanford University

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Responsible Party: Antonio Hardan, Professor of Psychiatry and Behavioral Sciences, Stanford University
ClinicalTrials.gov Identifier: NCT02627508     History of Changes
Other Study ID Numbers: IRB-34762
First Posted: December 11, 2015    Key Record Dates
Last Update Posted: May 9, 2019
Last Verified: May 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes

Keywords provided by Antonio Hardan, Stanford University:
Autism Spectrum Disorder
Autism
Pregnenolone

Additional relevant MeSH terms:
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Autistic Disorder
Autism Spectrum Disorder
Child Development Disorders, Pervasive
Neurodevelopmental Disorders
Mental Disorders