Clinical trial NCT01881737: A Study of Pregnenolone in the Treatment of Individuals With Autism
Official title: An Open-Label Pilot Study of Pregnenolone in the Treatment of Individuals With Autism
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Autistic Disorder
Keywords: autism
Lead sponsor: Stanford University
Sponsor class: OTHER
First posted: 2013-06-20
Enrollment: 15
Sex: ALL
Minimum age (years): 18
Maximum age (years): 45
Standard ages: ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Detailed 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.

Eligibility criteria: Inclusion Criteria:

1. Outpatients 18-45 years of age;
2. Males and females who are physically healthy;
3. Diagnosis of autism based on Diagnostic and Statistical Manual (DSM-IV-TR) criteria, the Autism Diagnostic Interview-Revised, and expert clinical evaluation;
4. Total Aberrant Behavior Checklist (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. Diagnostic and Statistical Manual (DSM-IV-TR) diagnosis of schizophrenia, schizoaffective disorder, or psychotic disorder, not otherwise specified;
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

Primary outcomes:
measure: 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)
timeFrame: 2, 4, 6, 8, 10, 12, and 16 weeks
Secondary outcomes:
measure: Social Responsiveness Scale (SRS) Total Score
timeFrame: 12 weeks
measure: Sensory Profile Questionnaire Total Score
timeFrame: 12
measure: Vineland Adaptive Behavior Scale
timeFrame: 12 weeks
measure: Repetitive Behavior Scale
timeFrame: 12 weeks
measure: Pregnenolone Level in Peripheral Blood as Measured at Baseline and After 12 Weeks
timeFrame: 12 weeks
Other outcomes:
Locations:
city: Stanford
country: United States
facility: Stanford University School of Medicine
state: California
Study officials:
affiliation: Stanford University
name: Antonio Hardan, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.