What side effects are associated with this type of intervention?

Common treatments for Autism Spectrum Disorder (ASD) such as omega-3 fatty acids and intranasal oxytocin have been studied for their potential benefits. Omega-3 fatty acids are generally well-tolerated but can cause gastrointestinal distress, such as nausea and diarrhea. Intranasal oxytocin has shown some potential but is not recommended for continuous use due to limited efficacy and potential side effects like temporary gynecomastia. Pregnenolone, a naturally occurring steroid hormone, is being studied for its effects on irritability and social communication in ASD, but detailed side effect profiles are not yet well-established.

What prior FDA approvals exist?

There are no specific FDA-approved treatments for the core symptoms of Autism Spectrum Disorder (ASD) that are similar to pregnenolone, a naturally occurring steroid hormone. Current research, including pilot trials, is exploring the potential of pregnenolone for improving social communication and reducing irritability in individuals with ASD. However, these studies are still in the investigational stages, and no definitive FDA approvals have been granted for such treatments.

What other types of research exist?

Promising novel alternatives to pregnenolone for treating Autism Spectrum Disorder in 2024 include: <ol> <li>Oxytocin: Known for its role in social bonding, oxytocin has shown potential in improving social interaction and reducing repetitive behaviors in ASD patients.</li> <li>Balovaptan: A vasopressin V1a receptor antagonist that has demonstrated improvements in socialization and communication in clinical trials.</li> <li>Arbaclofen: A GABA-B receptor agonist that may help reduce social withdrawal and improve social behaviors.</li> <li>Cannabidiol (CBD): Emerging evidence suggests that CBD may help reduce anxiety and improve behavior in ASD patients.</li> <li>Suramin: An antipurinergic drug that has shown promise in early trials for improving language and social behavior in children with ASD.</li> </ol>

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Corticosteroid

Pregnenolone for Autism

Phase 2

Recruiting

Led By Antonio Y. Hardan, MD

Research Sponsored by Stanford University

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

outpatients between 14 and 25 years of age with a Tanner stage of IV or V;

outpatients between 14 and 25 years of age with a Tanner stage of IV or V

Must not have

pregnant or sexually active female subjects who do not adhere to use an appropriate form of external prophylactics

participants taking steroid medications

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

Summary

This trial is testing pregnenolone, a natural hormone in the brain, to see if it can help people with autism. Pregnenolone is part of a neurosteroid pathway that has been studied for its potential therapeutic value in stress and drug abuse-related disorders. The goal is to find out if it can reduce irritability and sensitivity to sensory differences, and improve social communication. Researchers hope this will lead to better treatments for autism.

Who is the study for?

This trial is for healthy males and females aged 14-25 with Autism Spectrum Disorder (ASD), confirmed by specific diagnostic tools. Participants must have a certain level of irritability and severity of ASD symptoms, be in the late stages of puberty, and on stable medications without changes planned during the study. Those with unstable seizures, significant illness, other major psychiatric disorders, or women who are pregnant or not using contraception cannot join.

What is being tested?

The study tests pregnenolone's safety and effectiveness compared to a placebo in reducing irritability, improving sensitivity to sensory differences, and enhancing social communication in individuals with autism. Pregnenolone is a hormone thought to help various psychiatric conditions.

What are the potential side effects?

Potential side effects may include hormonal imbalances since pregnenolone is a steroid hormone. Specific side effects aren't listed but could resemble those associated with other steroids such as mood swings, weight gain, or increased susceptibility to infections.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am between 14 and 25 years old and have reached a late stage of puberty.

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I am between 14 and 25 years old and have reached a late stage of puberty.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am not pregnant or I use birth control if sexually active.

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I am currently taking steroid medications.

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I do not have frequent seizures or severe illness.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Side effects data

From 2011 Phase 4 trial • 80 Patients • NCT01409096

Sinusitis

Corneal ulceration

Skin abscess followed by allergic reaction to antibiotic used to treat it

Skin rash

100%

80%

60%

40%

20%

Study treatment Arm

Placebo

Pregnenolone

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: Pregnenolone (up to 500 mg per day)Experimental Treatment1 Intervention

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)

Group II: PlaceboPlacebo Group1 Intervention

Placebo

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Pregnenolone

2011

Completed Phase 4

~770

0 / 5Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for Autism Spectrum Disorder (ASD) often target neurochemical pathways to alleviate symptoms. Pregnenolone, a naturally occurring steroid hormone, is being studied for its potential to reduce irritability and sensory sensitivity while improving social communication by modulating neurosteroid activity in the brain. Melatonin, another treatment, helps regulate sleep patterns by influencing the circadian rhythm, which is crucial for improving sleep disturbances common in ASD. Omega-3 fatty acids, although not strongly recommended, are thought to support brain function and reduce inflammation. These treatments matter for ASD patients as they address core symptoms and improve quality of life by targeting underlying neurobiological mechanisms.

Replication Pilot Trial of Therapeutic Horseback Riding and Cortisol Collection With Children on the Autism Spectrum.Advances in the research of melatonin in autism spectrum disorders: literature review and new perspectives.