A Study of Pregnenolone in the Treatment of Individuals With Autism

NCT01881737 | Completed Phase 2 Results DMC

• 1 other identifier: SU-08092011-8246
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Autistic Disorder

Keywords

autism

Outcome Measures

Primary Outcomes (1)

• 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)

  2, 4, 6, 8, 10, 12, and 16 weeks

Secondary Outcomes (5)

• Social Responsiveness Scale (SRS) Total Score

  12 weeks

• Sensory Profile Questionnaire Total Score

  12

• Vineland Adaptive Behavior Scale

  12 weeks

• Repetitive Behavior Scale

  12 weeks

• Pregnenolone Level in Peripheral Blood as Measured at Baseline and After 12 Weeks

  12 weeks

Study Arms (1)

Pregnenolone

EXPERIMENTAL

Pregnenolone up to 500 mg per day

Drug: Pregnenolone

Interventions

Pregnenolone DRUG

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.

Pregnenolone

Eligibility Criteria

• Age: 18 Years - 45 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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

Sponsors & Collaborators

• Stanford University: lead

Study Sites (1)

Stanford University School of Medicine

Stanford, California, 94305, United States

Location

Related Publications (1)

• 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.

  PMID: 24849255 RESULT

MeSH Terms

Conditions

Autistic Disorder

Interventions

Pregnenolone

Condition Hierarchy (Ancestors)

Autism Spectrum Disorder; Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Intervention Hierarchy (Ancestors)

Pregnenes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Hydroxycorticosteroids; Adrenal Cortex Hormones; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Progesterone Congeners; Gonadal Steroid Hormones; Gonadal Hormones

Limitations and Caveats

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.

Results Point of Contact

• Title: Antonio Hardan, MD
• Organization: Stanford University School of Medicine

hardanay@stanford.edu

(650) 736-1235

Study Officials

• Antonio Hardan, MD

  Stanford University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: November 28, 2011
• First Posted: June 20, 2013
• Study Start: July 1, 2011
• Primary Completion: September 1, 2013
• Study Completion: September 1, 2013
• Last Updated: March 29, 2017
• Results First Posted: March 29, 2017

Record last verified: 2017-02

Data Sharing

• IPD Sharing: Will share

Locations

US (1)