Pilot Trial of Pregnenolone in Autism

Autism Spectrum Disorder Clinical Trial

Official title:

Randomized Controlled Pilot Trial of Pregnenolone in Autism

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT02627508
• Other study ID #: IRB-34762
• Status: Recruiting
• Phase: Phase 2
• Start date: January 29, 2016
• Est. completion date: March 2025

Study information

• Verified date: June 2024
• Source: Stanford University
• Contact: Briana Hernandez
• Phone: (650) 736-1235
• Email: autismdd[@]stanford.edu
• Is FDA regulated: No
• Study type: Interventional

Clinical 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. 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 40
• Est. completion date: March 2025
• Est. primary completion date: March 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 14 Years to 25 Years

Eligibility

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.

Related Conditions & MeSH terms

• Autism
• Autism Spectrum Disorder
• Autistic Disorder

Intervention

Drug:
• Pregnenolone
orally administered Pregnenolone capsules
• Placebo
orally administered placebo capsules

Locations

Country	Name	City	State
United States	Stanford University School of Medicine	Stanford	California

Sponsors (2)

Lead Sponsor	Collaborator
Stanford University	Simons Foundation

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change from baseline in parent rated Aberrant Behavior Checklist Irritability (ABC-I) score during treatment		2-week, 4-week, 6-week, 8-week, 10-week, 14-week
Secondary	Change from baseline in parent rated Aberrant Behavior Checklist (ABC) scores during treatment		2-week, 4-week, 6-week, 8-week, 10-week, 14-week
Secondary	Change from baseline in parent rated Sensory Profile Questionnaire (SPQ) scores during treatment		2-week, 4-week, 6-week, 8-week, 10-week, 14-week
Secondary	Change from baseline in Vineland Adaptive Behavior Scales Second Edition (VABS-II)		2-week, 6-week, 10-week, 14-week
Secondary	Change from baseline in Clinical Global Impression (CGI) scores during treatment		2-week, 4-week, 6-week, 8-week, 10-week, 14-week
Secondary	Change from baseline in levels of pregnenolone and its related neurosteroids in peripheral blood		14-week
Secondary	Change from baseline on the Autism Diagnostic Observation Schedule (ADOS)		14-week
Secondary	Change from baseline in laboratory based eye-gaze measures		14-week
Secondary	Change from baseline in laboratory based electroencephalography (EEG) measures		14-week
Secondary	Change from baseline in laboratory clinical lab values including complete metabolic panel, complete blood count with differential, cholesterol panel and routine urinalysis.		14-week
Secondary	Change from baseline in parent rated Social Responsiveness Scale (SRS) scores during treatment		2-week, 6-week, 10-week, 14-week
Secondary	Change from baseline on the Dosage Record Treatment Emergent Symptom Scale (DOTES) scores during treatment		2-week, 4-week, 6-week, 8-week, 10-week, 14-week