Irritability Associated With Autism Spectrum DisorderPhase 2NCT05733390
A Study of JZP541 in Adults With Irritability Associated With Autism Spectrum Disorder
A Phase 2 study testing JZP541 in people with Irritability Associated With Autism Spectrum Disorder and Autism Spectrum Disorder, run by Jazz Pharmaceuticals at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 45 years
- Conditions the study is for
- Irritability Associated With Autism Spectrum Disorder, Autism Spectrum Disorder, ASD
- What is being tested?
- JZP541
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- 30 Jun 2023to 9 Nov 2023
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 45 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 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
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- 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
- Male participants must agree to refrain from donating sperm, or be abstinent, or agree to use contraception during the Study Intervention Period and for at least 12 weeks after the last dose of study intervention
- Female participants are eligible if she is not pregnant or breastfeeding as specified
- Women of childbearing potential (WOCBP) must have a negative serum pregnancy test within 14 days before the first dose of study intervention
- Participant (or study partner) is willing and able to give informed consent or assent
Who cannot join
- 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
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- 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
- Has any other significant disease or disorder which, in the opinion of the investigator, may either put the participant at risk because of participation in the study, may influence the result of the study, or may affect the participant's ability to take part in the study
- Had in the 2 weeks prior to screening or up to randomization any condition that might affect baseline assessments
- Has received administration of strong inducers of CYP3A4 ≤ 14 days prior to first doses of study intervention or have ongoing requirements for these medications
- Is currently using or has used within the 12 weeks prior to screening recreational or medicinal cannabis, cannabinoid-based medications (botanical or synthetic; eg, Sativex, Epidiolex/Epidyolex, Nabilone, Dronabinol) and/or is unwilling to abstain for the duration of the study
- Is currently taking bupropion
- Has received another investigational product within the 12 weeks prior to the Screening Visit
- Has any known or suspected hypersensitivity to cannabinoids or any of the excipients of the study intervention
- Has been previously randomized into this study
- Has impaired liver function at screening, as reflected by serum alanine aminotransferase or aspartate aminotransferase > 1.5 × ULN, or total bilirubin > ULN
- Female participant who is pregnant (positive pregnancy test), lactating, or planning pregnancy during the study or within 3 months thereafter
- Has any history of suicidal behavior or any suicidal ideation (with intent per C-SSRS questions 4 and 5) in the last month at screening (last month) or Visit 2 (randomization)
- Has any known or suspected history of alcohol or substance abuse
- Has positive drug abuse test at screening
- Is living in the same household as another active participant of this study or nominated study partner is actively serving as a study partner for another participant of this study (Note: Same household does not apply to professional care-home environment)
Trial locations and contacts
1 site in 1 country.
United States1 site
Albert Einstein College of Medicine
The Bronx, New York, 10461
About this study
Official title: 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
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.
Read the 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.
- Being tested
- JZP541, Placebo
- Keywords
- Irritability, Autism Spectrum Disorder, ASD, JZP541
- Medical subject headings
- Autism Spectrum Disorder
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
JZP541Drug
Oral suspension
PlaceboOther
Oral suspension
| Study group | Role | What they receive |
|---|---|---|
| JZP541 | Experimental | Participants who will be randomized to receive JZP541. Receives: Drug: JZP541 |
| Placebo | Placebo comparator | Participants who will be randomized to receive placebo. Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Mean Change From Baseline to Week 12 in the Aberrant Behavior Checklist-Irritability (ABC-I) Subscale Score
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.
Measured: Baseline to Week 12
Secondary outcomes (7)
Number of Participants With Treatment-emergent Adverse Events
Measured: Day 1 up until end of study or discontinuation (whichever occurs first), up to approximately 2 years
Percentage of Participants with ≥ 25% Reduction in Aberrant Behavior Checklist-Irritability (ABC-I) Subscale Score From Baseline
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.
Measured: 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
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.
Measured: 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
Measured: Baseline up until end of study or discontinuation (whichever occurs first), up to approximately 2 years
Mean Change From Baseline to Week 12 in Aberrant Behavior Checklist (ABC) Modified Social Withdrawal Subscale Score
The ABC Modified Social Withdrawal subscale contains 13 items and its score is used to rate a person's social withdrawal over the past 7 days on a 4-point scale, where 0 indicates no withdrawal and 3 indicates severe/extreme social withdrawal. Higher scores indicate worse outcome.
Measured: Baseline to Week 12
Mean Change From Baseline to Week 12 in Aberrant Behavior Checklist (ABC) Stereotypic Behavior Subscale Score
The ABC Stereotypic Behavior subscale contains 7 items and its score is used to rate a person's stereotypic behavior over the past 7 days on a 4-point scale, where 0 indicates no stereotypic behavior and 3 indicates severe stereotypic behavior. Higher scores indicate worse outcome.
Measured: Baseline to Week 12
Mean Change From Baseline to Week 12 in Repetitive Behavior Scale - Revised (RBS-R) Subscale Scores
The RBS-R subscale score is used to rate a person's behavior over the past 7 days on a 4-point scale, where 0 indicates that the behavior does not occur and 3 indicates that the behavior occurs and is a serious problem. Higher scores indicate worse behavior.
Measured: Baseline to Week 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Feb 2023
- Study start30 Jun 2023
- Primary completion9 Nov 2023
- Study completion9 Nov 2023
Why it stopped: A decision has been made to prioritize other pipeline programs and terminate this study.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Irritability Associated With Autism Spectrum DisorderStudies of the same condition, open and under way.
- More trials run by Jazz PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Jazz Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- GW Research
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05733390
- Sponsor's protocol ID
- JZP541-201
- Other IDs
- 2022-502355-61-00
- Status last verified
- Nov 2024
- First submitted
- 8 Feb 2023
- Last updated
- 8 Nov 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Nov 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT05733390
- Acronym / sponsor ID
- JZP541-201
- Keywords
- Irritability, Autism Spectrum Disorder, ASD, JZP541
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: A decision has been made to prioritize other pipeline programs and terminate this study.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Jazz PharmaceuticalsIndustry (pharma, biotech, devices)
- Collaborators
- GW Research
- Study start
- 30 Jun 2023 (actual)
- Primary completion
- 9 Nov 2023 (actual)
- Study completion
- 9 Nov 2023 (actual)
- First posted
- 17 Feb 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| JZP541 | Experimental | Drug: JZP541Participants who will be randomized to receive JZP541. |
| Placebo | Placebo comparator | Other: PlaceboParticipants who will be randomized to receive placebo. |
Who may qualify
- Ages
- 18 to 45 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (14 listed)
- 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
Key exclusion criteria (24 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| JZP541Oral suspension | Drug | JZP541 |
| PlaceboOral suspension | Other | Placebo |
Primary outcome
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.
7 secondary outcomes
Number of Participants With Treatment-emergent Adverse Events
Time frame: Day 1 up until end of study or discontinuation (whichever occurs first), up to approximately 2 years
Percentage of Participants with ≥ 25% Reduction in Aberrant Behavior Checklist-Irritability (ABC-I) Subscale Score From Baseline
Time frame: 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
Time frame: 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
Time frame: Baseline up until end of study or discontinuation (whichever occurs first), up to approximately 2 years
Mean Change From Baseline to Week 12 in Aberrant Behavior Checklist (ABC) Modified Social Withdrawal Subscale Score
Time frame: Baseline to Week 12
Mean Change From Baseline to Week 12 in Aberrant Behavior Checklist (ABC) Stereotypic Behavior Subscale Score
Time frame: Baseline to Week 12
Mean Change From Baseline to Week 12 in Repetitive Behavior Scale - Revised (RBS-R) Subscale Scores
Time frame: Baseline to Week 12
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Nov 2024.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing JZP541 in people with Irritability Associated With Autism Spectrum Disorder and Autism Spectrum Disorder, run by Jazz Pharmaceuticals at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 14 inclusion and 24 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 8 Nov 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Mean Change From Baseline to Week 12 in the Aberrant Behavior Checklist-Irritability (ABC-I) Subscale Score (time frame: Baseline to Week 12). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Nov 2024.