What is the mechanism of action of this treatment?

Common treatments for Autism, particularly those similar to JZP541, often work by modulating neurotransmitter activity or other pathways involved in mood and behavior regulation. For instance, medications may target serotonin or dopamine receptors to help stabilize mood and reduce irritability. Oxytocin, a hormone linked to social bonding, is also being explored for its potential to improve social interactions in Autism patients. These mechanisms are crucial as they directly address core symptoms of Autism, such as irritability and social deficits, thereby improving the quality of life for patients.

What side effects are associated with this type of intervention?

Common treatments for Autism Spectrum Disorder (ASD) that modulate neurotransmitter activity or other pathways involved in mood and behavior regulation include antipsychotics, antidepressants, and mood stabilizers. Antipsychotics like risperidone and aripiprazole can cause weight gain, sedation, and metabolic changes. Antidepressants such as SSRIs (e.g., fluoxetine) may lead to gastrointestinal issues, sleep disturbances, and increased anxiety. Mood stabilizers like valproate can result in weight gain, tremors, and gastrointestinal problems. The severity and occurrence of these side effects can vary among individuals.

What prior FDA approvals exist?

The FDA has approved several medications for the treatment of irritability associated with Autism Spectrum Disorder (ASD). Notably, risperidone and aripiprazole are antipsychotic drugs that have been approved for this purpose. These medications work by modulating neurotransmitter activity, particularly dopamine and serotonin receptors, to help manage symptoms such as aggression, self-injury, and severe tantrums. These treatments are somewhat similar to the investigational drug JZP541, which is being studied for its efficacy and safety in treating irritability in adults with ASD, likely through modulation of neurotransmitter pathways.

What other types of research exist?

Promising novel alternatives to JZP541 for treating irritability in autism patients include: 1. Ascorbic acid (AA) for its potential modulation of GABA receptors. 2. FG 7142, an inverse agonist at benzodiazepine receptors. 3. JNJ-55511118, a non-competitive NMDA receptor antagonist. 4. Benzodiazepines like lorazepam. 5. Hypnotics like zolpidem for their impact on sleep and mood.

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JZP541 for Autism

Phase 2

Waitlist Available

Research Sponsored by Jazz Pharmaceuticals

Eligibility Criteria Checklist

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

Must have

Be between 18 and 65 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up baseline up until end of study or discontinuation (whichever occurs first), up to approximately 2 years

Summary

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.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

While specific side effects for JZP541 aren't listed here, common ones may include digestive issues, changes in mood or behavior, allergic reactions if sensitive to ingredients; participants should report any discomforts they experience.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ baseline up until end of study or discontinuation (whichever occurs first), up to approximately 2 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~baseline up until end of study or discontinuation (whichever occurs first), up to approximately 2 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline up until end of study or discontinuation (whichever occurs first), up to approximately 2 years for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Mean Change From Baseline to Week 12 in the Aberrant Behavior Checklist-Irritability (ABC-I) Subscale Score

Secondary study objectives

Mean Change From Baseline to Week 12 in Aberrant Behavior Checklist (ABC) Modified Social Withdrawal Subscale Score

Mean Change From Baseline to Week 12 in Aberrant Behavior Checklist (ABC) Stereotypic Behavior Subscale Score

Mean Change From Baseline to Week 12 in Repetitive Behavior Scale - Revised (RBS-R) Subscale Scores

+2 more

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: JZP541Experimental Treatment1 Intervention

Participants who will be randomized to receive JZP541.

Group II: PlaceboPlacebo Group1 Intervention

Participants who will be randomized to receive placebo.

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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, particularly those similar to JZP541, often work by modulating neurotransmitter activity or other pathways involved in mood and behavior regulation. For instance, medications may target serotonin or dopamine receptors to help stabilize mood and reduce irritability. Oxytocin, a hormone linked to social bonding, is also being explored for its potential to improve social interactions in Autism patients. These mechanisms are crucial as they directly address core symptoms of Autism, such as irritability and social deficits, thereby improving the quality of life for patients.

Study protocol of the ASD-Net, the German research consortium for the study of Autism Spectrum Disorder across the lifespan: from a better etiological understanding, through valid diagnosis, to more effective health care.New directions in the treatment of autism spectrum disorders from animal model research.The oxytocin system in drug discovery for autism: animal models and novel therapeutic strategies.