Clinical trial NCT02627508: Pilot Trial of Pregnenolone in Autism
Official title: Randomized Controlled Pilot Trial of Pregnenolone in Autism
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Autism Spectrum Disorder
Autism
Keywords: Autism Spectrum Disorder
Autism
Pregnenolone
Lead sponsor: Stanford University
Sponsor class: OTHER
Collaborators: name: Simons Foundation
First posted: 2015-12-11
Enrollment: 40
Sex: ALL
Minimum age (years): 14
Maximum age (years): 25
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


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 Autism Spectrum Disorder (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.

Primary outcomes:
measure: Change from baseline in parent rated Aberrant Behavior Checklist Irritability (ABC-I) score during treatment
timeFrame: 2-week, 4-week, 6-week, 8-week, 10-week, 14-week
Secondary outcomes:
measure: Change from baseline in parent rated Aberrant Behavior Checklist (ABC) scores during treatment
timeFrame: 2-week, 4-week, 6-week, 8-week, 10-week, 14-week
measure: Change from baseline in parent rated Sensory Profile Questionnaire (SPQ) scores during treatment
timeFrame: 2-week, 4-week, 6-week, 8-week, 10-week, 14-week
measure: Change from baseline in Vineland Adaptive Behavior Scales Second Edition (VABS-II)
timeFrame: 2-week, 6-week, 10-week, 14-week
measure: Change from baseline in Clinical Global Impression (CGI) scores during treatment
timeFrame: 2-week, 4-week, 6-week, 8-week, 10-week, 14-week
measure: Change from baseline in levels of pregnenolone and its related neurosteroids in peripheral blood
timeFrame: 14-week
measure: Change from baseline on the Autism Diagnostic Observation Schedule (ADOS)
timeFrame: 14-week
measure: Change from baseline in laboratory based eye-gaze measures
timeFrame: 14-week
measure: Change from baseline in laboratory based electroencephalography (EEG) measures
timeFrame: 14-week
measure: Change from baseline in laboratory clinical lab values including complete metabolic panel, complete blood count with differential, cholesterol panel and routine urinalysis.
timeFrame: 14-week
measure: Change from baseline in parent rated Social Responsiveness Scale (SRS) scores during treatment
timeFrame: 2-week, 6-week, 10-week, 14-week
measure: Change from baseline on the Dosage Record Treatment Emergent Symptom Scale (DOTES) scores during treatment
timeFrame: 2-week, 4-week, 6-week, 8-week, 10-week, 14-week
Other outcomes:
Locations:
city: Stanford
country: United States
facility: Stanford University School of Medicine
state: California
Study officials:
affiliation: Stanford University
name: Antonio Y. Hardan, MD
role: PRINCIPAL_INVESTIGATOR
References: