JZP541 for Autism

Overseen By Clinical Trial Disclosure & Transparency

Age: 18 - 65

Sex: Any

Trial Phase: Phase 2

Sponsor: Jazz Pharmaceuticals

Must not be taking: Cannabinoids, Bupropion

Disqualifiers: Psychosis, Major depression, Seizures, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: 12 weeks

Trial Summary

What is the purpose of this trial?

This trial is testing a medication called JZP541 to see if it can help reduce irritability in adults with autism. The study will check both how well the medication works and if it is safe. Irritability is a big concern for adults with autism, and current treatments might not always help.

Will I have to stop taking my current medications?

The trial requires that all medications affecting ASD symptoms, behavior, anxiety, or sleep must be stable for 4 weeks (or 8 weeks for long-acting forms) before starting the study. Participants must keep their medication regimen stable throughout the study.

What data supports the effectiveness of the drug JZP541 for autism?

The study on mGluR5-antagonists, like MPEP, shows that targeting glutamate receptors can reduce repetitive behaviors in a mouse model of autism, suggesting potential benefits for similar drugs. ^{1 2 3 4 5}

How is the drug JZP541 unique for treating autism?

JZP541 is unique because it may target the core symptoms of autism by modulating glutamatergic signaling through mGluR5 receptors, which is different from other treatments like aripiprazole that focus on managing irritability or sensory abnormalities. ^{1 2 6 7 8}

Eligibility Criteria

Adults aged 18-45 with Autism Spectrum Disorder (ASD) experiencing irritability, who have a stable treatment regimen and can self-report side effects. They need an informed study partner and must not be pregnant or planning pregnancy. Excluded are those with recent seizures, psychosis, significant heart disease, suicidal behavior within the last month, severe intellectual disability, or substance abuse.

Inclusion Criteria

You have frequent episodes of anger, aggression, or hurting yourself or others that affect your ability to function or impact those around you.

Male or female aged 18 to 45 years inclusive at the time of signing the informed consent

Has a suitable study partner who keeps in regular contact with the participant, has sufficient knowledge of the participant's behavior to complete the study assessments, able to communicate with site personnel, willing to comply with protocol requirements, and has adequate literacy to complete the protocol-specified questionnaires

Exclusion Criteria

You have had a seizure in the last 2 years.

You have been diagnosed with a serious mental health condition like schizophrenia or bipolar disorder with psychotic features.

You have a history of mental disorders that affect your thoughts and perception, or you have close family members who have been diagnosed with similar conditions.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive JZP541 or placebo for 12 weeks to assess efficacy and safety in treating irritability associated with ASD

12 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment discontinuation

up to approximately 2 years

Treatment Details

Interventions

• JZP541
• Placebo

Trial Overview The trial is testing JZP541's effectiveness in reducing irritability in adults with ASD compared to a placebo. Participants will receive either the actual drug or a placebo without knowing which one they're getting to measure true effects of JZP541.

Participant Groups

Group I: JZP541 Experimental Treatment 1 Intervention

Participants who will be randomized to receive JZP541.

Group II: Placebo Placebo Group 1 Intervention

Participants who will be randomized to receive placebo.

Who Is Running the Clinical Trial?

Jazz Pharmaceuticals

Lead Sponsor

Trials

252

Recruited

35,100+

Bruce C. Cozadd

Jazz Pharmaceuticals

Chief Executive Officer since 2009

BA in Economics from Yale University, MBA from Stanford University

Dr. Austin

Jazz Pharmaceuticals

Chief Medical Officer since 2023

MD from the Royal College of Surgeons in Ireland

GW Research

Collaborator

Trials

1

Findings from Research

In a study of 35 children with autism, aripiprazole treatment for 8 weeks significantly reduced illness severity and improved various behavioral symptoms, as measured by the Clinical Global Impression and Autism Treatment Evaluation Checklist.

The treatment was found to be safe, with no severe adverse effects reported, indicating that aripiprazole can be a viable option for managing autism-related behavioral disorders in children.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Clinical observation of aripiprazole in the treatment of autism]. Xie, Q., Tang, J., Xu, Y., et al.[2015]

Risperidone and aripiprazole are the most supported pharmacological treatments for irritability in autism, with multiple randomized controlled trials (RCTs) showing significant improvements in symptoms.

While several alternative treatments like clonidine and methylphenidate also showed efficacy, they are less established than the FDA-approved antipsychotics, highlighting the need for behavioral therapy alongside medication for optimal management.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Review of the pharmacotherapy of irritability of autism. Elbe, D., Lalani, Z.[2021]

In a study of 13 children and adolescents with autism, aripiprazole was found to improve sensory deficits and disruptive behaviors over an average treatment duration of 24.4 weeks.

The medication showed specific benefits in areas such as auditory processing and visual input modulation, indicating its potential effectiveness in addressing sensory abnormalities associated with autism.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A retrospective review of the effectiveness of aripiprazole in the treatment of sensory abnormalities in autism. Fung, LK., Chahal, L., Libove, RA., et al.[2015]

In an 8-week study involving 92 children and adolescents with autism spectrum disorder, aripiprazole significantly reduced irritability compared to placebo, as measured by parent/caregiver and clinician ratings.

Aripiprazole was found to be effective and well-tolerated, with no serious adverse events reported, indicating a favorable safety profile for treating irritability in this population.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Aripiprazole in the Treatment of Irritability in Children and Adolescents with Autism Spectrum Disorder in Japan: A Randomized, Double-blind, Placebo-controlled Study. Ichikawa, H., Mikami, K., Okada, T., et al.[2022]

References

1. United States pubmed.ncbi.nlm.nih.gov

mGluR5-antagonist mediated reversal of elevated stereotyped, repetitive behaviors in the VPA model of autism. [2022]

2. China pubmed.ncbi.nlm.nih.gov

[Clinical observation of aripiprazole in the treatment of autism]. [2015]

3. Canada pubmed.ncbi.nlm.nih.gov

Review of the pharmacotherapy of irritability of autism. [2021]

4. United States pubmed.ncbi.nlm.nih.gov

An open-label naturalistic pilot study of acamprosate in youth with autistic disorder. [2021]

5. Switzerland pubmed.ncbi.nlm.nih.gov

Mediators of Placebo Response to Cannabinoid Treatment in Children with Autism Spectrum Disorder. [2023]

6. United States pubmed.ncbi.nlm.nih.gov

A retrospective review of the effectiveness of aripiprazole in the treatment of sensory abnormalities in autism. [2015]

7. United States pubmed.ncbi.nlm.nih.gov

Aripiprazole in the Treatment of Irritability in Children and Adolescents with Autism Spectrum Disorder in Japan: A Randomized, Double-blind, Placebo-controlled Study. [2022]

8. United States pubmed.ncbi.nlm.nih.gov

A Placebo-Controlled Crossover Trial of Gastrin-Releasing Peptide in Childhood Autism. [2018]