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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

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

NCT ID

NCT02627508

First Posted

December 2, 2015

Last Updated

July 13, 2023

Sponsor

Stanford University

Collaborators

Simons Foundation

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

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

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

autismdd@stanford.edu

First Name & Middle Initial & Last Name or Official Title & Degree

Jared Gong

Phone

(650) 736-1235

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

autismdd@stanford.edu

First Name & Middle Initial & Last Name & Degree

Jared Gong

Phone

(650) 736-1235

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

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Pilot Trial of Pregnenolone in Autism

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