Pilot Trial of Pregnenolone in Autism

Stanford University

Status and phase: Active, not recruiting; Phase 2

Conditions: Autism Spectrum Disorder; Autism

Treatments: Drug: Pregnenolone; Drug: Placebo

Study type: Interventional

Funder types: Other

Identifiers

NCT02627508

IRB-34762

Details and patient eligibility

About

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.

Enrollment: 40 patients

Sex: All

Ages: 14 to 25 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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40 participants in 2 patient groups, including a placebo group

Pregnenolone (up to 500 mg per day)

Experimental group

Description:

Twice daily intake of orally administered Pregnenolone will occur on a schedule as described below. Weeks 1 and 2: 30mg twice daily (total 60mg per day) Weeks 3 and 4: 60mg twice daily (total: 120mg per day) Weeks 5 and 6: 90mg twice daily (total: 180mg per day) Weeks 7 and 8: 150mg twice daily (total: 300mg per day) Weeks 9 and 10: 210mg twice daily (total: 420mg per day) Weeks 11 to 14: 250mg twice daily (total: 500mg per day)

Treatment:

Drug: Pregnenolone

Placebo

Placebo Comparator group

Description:

Placebo

Treatment:

Drug: Placebo