JZP541 for Autism

No longer recruiting at 1 trial location

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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores a new treatment called JZP541 to reduce irritability in adults with autism spectrum disorder (ASD). The researchers aim to assess the effectiveness and safety of this treatment compared to a placebo, a non-active substance used for comparison. Adults with ASD who experience temper outbursts, aggression, or self-harm that disrupt daily life might be suitable for this trial. Participants must maintain a stable routine with their current medications and have no severe intellectual disabilities or certain mental health conditions. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group of people.

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.

Is there any evidence suggesting that JZP541 is likely to be safe for humans?

Research shows that JZP541 is being tested to determine its effectiveness in reducing irritability in adults with autism spectrum disorder (ASD). Early results suggest that this treatment is generally well-tolerated. A previous study found JZP541 to be safe and noted some positive effects on behavior. While some side effects might occur, they are not expected to be serious or common. However, as JZP541 is still under study, more information is needed to fully understand its safety. Prospective participants should consult a healthcare professional to consider the possible risks and benefits of joining a trial. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

JZP541 is unique because it introduces a novel approach to treating autism. Unlike standard treatments, which often focus on behavioral therapies or medications targeting symptoms like irritability, JZP541 may work by addressing underlying neurological pathways specific to autism. Researchers are excited about JZP541's potential to offer a more targeted and effective treatment option, potentially improving the quality of life for individuals with autism in ways current therapies do not.

What evidence suggests that JZP541 might be an effective treatment for irritability in adults with autism?

Research has shown that JZP541, which participants in this trial may receive, may help reduce irritability in adults with autism spectrum disorder (ASD). In earlier studies, JZP541 improved behavior issues related to ASD, supported by changes in biological markers linked to these behaviors. Although data on adults is limited, similar studies in children with ASD using a related treatment, aripiprazole, showed significant reductions in irritability. These early findings suggest that JZP541 could potentially help manage irritability in adults with ASD. ^{1 2 3 4 6}

Are You a Good Fit for This Trial?

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.

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

Able to cooperate and take part in study assessments, including blood sampling

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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

Published Research Related to This Trial

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]

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]

Citations

1.

hra.nhs.uk

hra.nhs.uk/planning-and-improving-research/application-summaries/research-summaries/a-study-of-jzp541-in-adults-with-irritability-associated-with-autism-spectrum-disorder/

A Study of JZP541 in Adults with Irritability Associated ...

The primary objective of the study is to compare the efficacy of JZP541 relative to placebo in reducing irritability in adults with Autism Spectrum Disorder ( ...

2.

withpower.com

withpower.com/trial/phase-2-child-development-disorders-pervasive-4-2023-adf8e

JZP541 for Autism · Info for Participants

In an 8-week study involving 92 children and adolescents with autism spectrum disorder, aripiprazole significantly reduced irritability compared to placebo, as ...

3.

cdek.pharmacy.purdue.edu

cdek.pharmacy.purdue.edu/trial/NCT05733390/

Trial | NCT05733390

This randomized, double-blind, placebo-controlled, phase 2 study will evaluate the efficacy and safety of JZP541 in treating irritability in adults with ASD.

4.

synapse.patsnap.com

synapse.patsnap.com/drug/78fe188a1e344764920df411d01a28c4

JZP-541 - Drug Targets, Indications, Patents

... Efficacy and Safety of JZP541 in Adults With Irritability Associated With Autism Spectrum Disorder. 100 Clinical Results associated with JZP-541.

5.

sciencedirect.com

sciencedirect.com/science/article/pii/S002839082500156X

Behavioral and transcriptomic effects of a novel ...

Our findings demonstrate JZP541 was able to ameliorate ASD associated behavioral deficits, and this was supported by improvements in putative transcriptional ...

6.

staging-fdaaa.ebmdatalab.net

staging-fdaaa.ebmdatalab.net/trial/NCT05733390/

A trial that was reported late by Jazz Pharmaceuticals

Full data ; A Phase 2, Randomized, Double-blind, Placebo-Controlled Study to Investigate the Efficacy and Safety of JZP541 in Adults With Irritability Associated ...