A Phase 2, Randomized, Double-blind, Placebo-Controlled Study to Investigate the Efficacy and Safety of JZP541 in Adults With Irritability Associated With Autism Spectrum Disorder

Jazz Pharmaceuticals1 site in 1 country1 target enrollmentJune 30, 2023

ConditionsIrritability Associated With Autism Spectrum Disorder Autism Spectrum Disorder ASD

InterventionsJZP541 Placebo

DrugsJZP541

Overview

Phase: Phase 2
Intervention: JZP541
Conditions: Irritability Associated With Autism Spectrum Disorder
Sponsor: Jazz Pharmaceuticals
Enrollment: 1
Locations: 1
Primary Endpoint: Mean Change From Baseline to Week 12 in the Aberrant Behavior Checklist-Irritability (ABC-I) Subscale Score
Status: Withdrawn
Last Updated: last year

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

Behavior dysregulation is commonly associated with people with autism spectrum disorder (ASD). Irritability is a major safety concern in adults with ASD. This study will assess the efficacy and safety of JZP541 in the treatment of adults with irritability associated with ASD.

Detailed Description

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

Registry

clinicaltrials.gov

Start Date

June 30, 2023

End Date

November 9, 2023

Last Updated

last year

Study Type

Interventional

Study Design

Parallel

Sex

All

Investigators

Sponsor

Jazz Pharmaceuticals

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•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
•Participant has full scale intelligence quotient (IQ) or General Ability Index (GAI) \>45, measured in adulthood using the Wechsler Adult Intelligence Scale - Fourth Edition (WAIS-IV) or Wechsler Adult Intelligence Scale - Second Edition (WASI-II), and the ability to self-report on adverse events (AEs) as determined by the investigator
•Able to cooperate and take part in study assessments, including blood sampling
•Has a current diagnosis of autism spectrum disorder (ASD) as per DSM-5 criteria for ASD \[confirmed at screening\], which has been confirmed by the Autism Diagnostic Observational Schedule-2nd Edition (ADOS-2) or the Autism Diagnostic Interview-Revised (ADI-R) \[lifetime assessment is acceptable\]. If no lifetime assessment is available, the ADOS-2 or ADI-R should be performed at screening for diagnosis confirmation
•Has episodes of temper outbursts, aggression, self-injurious behavior or a combination of these issues that interfere with the participant's performance or affect others
•Participant's irritability per Clinical Global Impression of Severity (CGIS) is at least 3 (moderate) at screening and randomization
•All medications taken by the participant that may have an effect on ASD symptoms, behavior, anxiety, or sleep must have been stable for 4 weeks, or 8 weeks for long-acting formulations, prior to the Screening Visit and Visit 2
•Willing to maintain a stable regimen for all participant medications and interventions throughout the study
•Participant is compliant with their current medications

Exclusion Criteria

•Has a severe or profound intellectual disability (ie, IQ or GAI ≤ 45)
•Participant is unable to self-report AEs, as determined by the investigator
•Has a current diagnosis of psychosis spectrum disorders (bipolar disorder with psychotic features, schizo-affective disorder, delusional disorder, or schizophrenia)
•Has a current diagnosis of major depression (participants with depression in remission for at least 12 months may be included)
•Has an established history of psychotic spectrum disorders, early signs of psychosis at screening or Visit 2, or first degree relative with a lifetime diagnosis of psychosis spectrum disorders
•Has had a seizure within the past 2 years
•Has had changes in anticonvulsive therapy within the last 12 weeks
•Has experienced myocardial infarction or clinically significant cardiac dysfunction within the 12 months prior to Visit 1 or has a history of clinically significant arterial vascular disease, including cerebrovascular and cardiovascular disease
•Has supine systolic blood pressure \< 90 mmHg or \> 150 mmHg or supine diastolic blood pressure \< 50 mmHg or \> 105 mmHg or a postural drop in systolic blood pressure ≥ 20 mmHg or diastolic blood pressure ≥ 10 mmHg, with or without symptoms, at screening or baseline (prior to randomization)
•Has a QTcF interval \> 450 ms or a history of additional risk factors for Torsades de pointes

Arms & Interventions

JZP541

Participants who will be randomized to receive JZP541.

Intervention: JZP541

Placebo

Participants who will be randomized to receive placebo.

Intervention: Placebo

Outcomes

Primary Outcomes

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

Time Frame: Baseline to Week 12

The ABC-I subscale contains 15 items and its score is used to rate a person's irritability over the past 7 days on a 4-point scale, where 0 indicates no irritability and 3 indicates severe irritability. Higher scores indicate worse outcome.

Secondary Outcomes

Mean Change From Baseline to Week 12 in Aberrant Behavior Checklist (ABC) Modified Social Withdrawal Subscale Score(Baseline to Week 12)
Mean Change From Baseline to Week 12 in Aberrant Behavior Checklist (ABC) Stereotypic Behavior Subscale Score(Baseline to Week 12)
Mean Change From Baseline to Week 12 in Repetitive Behavior Scale - Revised (RBS-R) Subscale Scores(Baseline to Week 12)
Percentage of Participants with ≥ 25% Reduction in Aberrant Behavior Checklist-Irritability (ABC-I) Subscale Score From Baseline(Baseline up until end of study or discontinuation (whichever occurs first), up to approximately 2 years)
Mean Change in ABC-I Score After Treatment Discontinuation(Date of treatment discontinuation up until end of study or discontinuation (whichever occurs first), up to approximately 2 years)
Mean Change From Baseline in the Caregiver Top 3 ABC-I Items of Concern(Baseline up until end of study or discontinuation (whichever occurs first), up to approximately 2 years)
Number of Participants With Treatment-emergent Adverse Events(Day 1 up until end of study or discontinuation (whichever occurs first), up to approximately 2 years)