A Study of Pregnenolone in the Treatment of Individuals With Autism

Autistic Disorder Clinical Trial

Official title:

An Open-Label Pilot Study of Pregnenolone in the Treatment of Individuals With Autism

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01881737
• Other study ID #: SU-08092011-8246
• Status: Completed
• Phase: Phase 2
• First received: November 28, 2011
• Last updated: December 2, 2015
• Start date: July 2011
• Est. completion date: September 2013

Study information

• Verified date: December 2015
• Source: Stanford University
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

Description:

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.

Pregnenolone has been used safely in research studies involving individuals with schizophrenia. In the proposed trial, we hope to examine the tolerability of pregnenolone in adults with autism. We hope to see improvement in behavioral outcomes as measured by standardized behavioral measures. Further, we will measure concentrations of pregnenolone and related neuroactive compounds in the blood. The use of pregnenolone has been studied in a number of mental disorders but not autism. Thus, we hope the study will identify new avenues of research for the treatment of autism.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 15
• Est. completion date: September 2013
• Est. primary completion date: September 2013
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years to 45 Years

Eligibility

Inclusion Criteria:

1. Outpatients 18-45 years of age;

2. Males and females who are physically healthy;

3. Diagnosis of autism based on DSM-IV-TR criteria, the Autism Diagnostic Interview-Revised, and expert clinical evaluation;

4. Total ABC greater then 21;

5. Care provider who can reliably bring subject to clinic visits, can provide trustworthy ratings, and interacts with subject on a regular basis;

6. Ability of subject to swallow the compound;

7. Stable concomitant medications for at least 2 weeks; and

8. No planned changes in psychosocial interventions during the open-label pregnenolone trial.

Exclusion Criteria:

1. DSM-IV-TR diagnosis of schizophrenia, schizoaffective disorder, or psychotic disorder NOS;

2. Prior adequate trial of pregnenolone;

3. Active medical problems: unstable seizures, significant physical illness (e.g., serious liver or renal pathology);

4. Pregnancy or sexually active females (as determined by a urinary pregnancy test in the beginning of the study); and

5. 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

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Autistic Disorder

Intervention

Drug:
• Pregnenolone
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. Days 1-14: 100 mg Week 1 and 2: 200 mg Week 3 and 4: 350 mg Week 5 and 6: 400 mg Week 7 -12: 500 mg 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.

Locations

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

Sponsors (1)

Lead Sponsor	Collaborator
Stanford University

Country where clinical trial is conducted

United States, 

References & 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. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change from Baseline in Dosage Record and Treatment Emergent Symptom (DOTES) at 2, 4, 6, 8, 10, 12, and 16 weeks		Baseline, 2, 4, 6, 8, 10, 12, and 16 weeks	Yes
Secondary	Social Responsiveness Scale		12 and 16 weeks	No
Secondary	Sensory Profile Questionnaire		12 and 16 weeks	No
Secondary	Vineland Adaptive Behavior Scale		12 and 16 weeks	No
Secondary	Repetitive Behavior Scale		12 and 16 weeks	No
Secondary	Levels of pregnenolone and its related neurosteroids in peripheral blood		12 weeks	No