search
Back to results

Pilot Trial of Pregnenolone in Autism

Primary Purpose

Autism Spectrum Disorder, Autism

Status
Recruiting
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Pregnenolone
Placebo
Sponsored by
Stanford University
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Autism Spectrum Disorder focused on measuring Autism Spectrum Disorder, Autism, Pregnenolone

Eligibility Criteria

14 Years - 25 Years (Child, Adult)All SexesDoes not accept healthy volunteers

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 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.

Sites / Locations

  • Stanford University School of MedicineRecruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

Pregnenolone (up to 500 mg per day)

Placebo

Arm Description

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)

Placebo

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

Change from baseline in parent rated Aberrant Behavior Checklist (ABC) scores during treatment
Change from baseline in parent rated Sensory Profile Questionnaire (SPQ) scores during treatment
Change from baseline in Vineland Adaptive Behavior Scales Second Edition (VABS-II)
Change from baseline in Clinical Global Impression (CGI) scores during treatment
Change from baseline in levels of pregnenolone and its related neurosteroids in peripheral blood
Change from baseline on the Autism Diagnostic Observation Schedule (ADOS)
Change from baseline in laboratory based eye-gaze measures
Change from baseline in laboratory based electroencephalography (EEG) measures
Change from baseline in laboratory clinical lab values including complete metabolic panel, complete blood count with differential, cholesterol panel and routine urinalysis.
Change from baseline in parent rated Social Responsiveness Scale (SRS) scores during treatment
Change from baseline on the Dosage Record Treatment Emergent Symptom Scale (DOTES) scores during treatment

Full Information

First Posted
December 2, 2015
Last Updated
July 13, 2023
Sponsor
Stanford University
Collaborators
Simons Foundation
search

1. Study Identification

Unique Protocol Identification Number
NCT02627508
Brief Title
Pilot Trial of Pregnenolone in Autism
Official Title
Randomized Controlled Pilot Trial of Pregnenolone in Autism
Study Type
Interventional

2. Study Status

Record Verification Date
July 2023
Overall Recruitment Status
Recruiting
Study Start Date
January 29, 2016 (Actual)
Primary Completion Date
March 2025 (Anticipated)
Study Completion Date
March 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Stanford University
Collaborators
Simons Foundation

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Autism Spectrum Disorder, Autism
Keywords
Autism Spectrum Disorder, Autism, Pregnenolone

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
40 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Pregnenolone (up to 500 mg per day)
Arm Type
Experimental
Arm Description
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)
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Placebo
Intervention Type
Drug
Intervention Name(s)
Pregnenolone
Intervention Description
orally administered Pregnenolone capsules
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
orally administered placebo capsules
Primary Outcome Measure Information:
Title
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 Measure Information:
Title
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
Title
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
Title
Change from baseline in Vineland Adaptive Behavior Scales Second Edition (VABS-II)
Time Frame
2-week, 6-week, 10-week, 14-week
Title
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
Title
Change from baseline in levels of pregnenolone and its related neurosteroids in peripheral blood
Time Frame
14-week
Title
Change from baseline on the Autism Diagnostic Observation Schedule (ADOS)
Time Frame
14-week
Title
Change from baseline in laboratory based eye-gaze measures
Time Frame
14-week
Title
Change from baseline in laboratory based electroencephalography (EEG) measures
Time Frame
14-week
Title
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
Title
Change from baseline in parent rated Social Responsiveness Scale (SRS) scores during treatment
Time Frame
2-week, 6-week, 10-week, 14-week
Title
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

10. Eligibility

Sex
All
Minimum Age & Unit of Time
14 Years
Maximum Age & Unit of Time
25 Years
Accepts Healthy Volunteers
No
Eligibility 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 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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Joe McGrath
Phone
(650) 736-1235
Email
autismdd@stanford.edu
First Name & Middle Initial & Last Name or Official Title & Degree
Jared Gong
Phone
(650) 736-1235
Email
xjgong@stanford.edu
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Antonio Y. Hardan, MD
Organizational Affiliation
Stanford University
Official's Role
Principal Investigator
Facility Information:
Facility Name
Stanford University School of Medicine
City
Stanford
State/Province
California
ZIP/Postal Code
94305
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Joe McGrath
Phone
650-736-1235
Email
autismdd@stanford.edu
First Name & Middle Initial & Last Name & Degree
Jared Gong
Phone
(650) 736-1235
Email
xjgong@stanford.edu
First Name & Middle Initial & Last Name & Degree
Antonio Y. Hardan, MD
First Name & Middle Initial & Last Name & Degree
Lawrence K. Fung, MD, PhD

12. IPD Sharing Statement

Plan to Share IPD
Yes

Learn more about this trial

Pilot Trial of Pregnenolone in Autism

We'll reach out to this number within 24 hrs