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Pimavanserin for the Treatment of Irritability Associated With Autism Spectrum Disorder

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ClinicalTrials.gov Identifier: NCT05523895
Recruitment Status : Recruiting
First Posted : August 31, 2022
Last Update Posted : May 9, 2024
Sponsor:
Information provided by (Responsible Party):
ACADIA Pharmaceuticals Inc.

Study Description
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Brief Summary:
6-week, randomized, double-blind, fixed-dose, placebo-controlled, parallel group study in children and adolescents (aged 5 to17 years) with autism spectrum disorder (ASD) with irritability, agitation, or self-injurious behaviors to study the efficacy and safety of pimavanserin

Condition or disease Intervention/treatment Phase
Irritability Associated With Autism Spectrum Disorder Drug: Pimavanserin Drug: Placebo Phase 2 Phase 3

Detailed Description:
This study will be conducted as a 6-week, randomized, double-blind, fixed-dose, placebo-controlled, parallel group study in children and adolescents (5 through 17 years of age) with ASD with irritability, agitation, or self-injurious behaviors.
Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 228 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 2, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Pimavanserin for the Treatment of Irritability Associated With Autism Spectrum Disorder
Actual Study Start Date : August 9, 2022
Estimated Primary Completion Date : September 2024
Estimated Study Completion Date : September 2024

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Placebo Comparator: Placebo
Placebo given once daily, as one capsule matching in size and color the respective pimavanserin treatment
 Drug: Placebo
Pimavanserin matching placebo
Experimental: Pimavanserin low dose

Patients aged 5 to 12 years: 10 mg/day pimavanserin Patients aged 13 to 17 years: 20 mg/day pimavanserin

Pimavanserin given once daily, as capsule of 10 or 20 mg dose strength, respectively, according to the patient's age

 Drug: Pimavanserin
Pimavanserin
Other Name: Nuplazid
Experimental: Pimavanserin high dose

Patients aged 5 to 12 years: 20 mg/day pimavanserin Patients aged 13 to 17 years: 34 mg/day pimavanserin

Pimavanserin given once daily, as capsule of 20 or 34 mg dose strength, respectively, according to the patient's age

 Drug: Pimavanserin
Pimavanserin
Other Name: Nuplazid


Outcome Measures
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Primary Outcome Measures :
  1. Change from Baseline at Week 6 in caregiver-rated Aberrant Behavior Checklist (ABC) Irritability subscale score [ Time Frame: 6 weeks ]
    The Aberrant Behavior Checklist (ABC) is a caregiver-rated scale comprised of five empirically-derived subscales encompassing 58 items that describe various behavior problems It measures domains of irritability Lethargy/Social Withdrawal, Stereotypic Behavior, Hyperactivity/noncompliance, and Inappropriate Speech. ABC-Irritability is one of the subscales and comprises of 15 items. Minimum score is 0, maximum is 45. The ABC will be administered at Screening and Baseline, and at all post-Baseline visits from Week 1 through Week 6. A score for each item ranges from 0 indicating "not at all a problem" to 3 indicating "the problem is severe in degree". Subscale scores are calculated by summing the items within that subscale. Higher scores indicate greater impairment.


Secondary Outcome Measures :
  1. Change from Baseline at Week 6 in caregiver-rated ABC subscale scores: Stereotypic Behavior; Lethargy; Hyperactivity; Inappropriate speech [ Time Frame: 6 weeks ]
  2. Change from Baseline at Week 6 in Clinical Global Impression-Severity (CGI-S) of Irritability score [ Time Frame: 6 weeks ]
  3. Clinical Global Impression-Improvement (CGI-I) of irritability score at Week 6 [ Time Frame: 6 weeks ]
  4. Change from Baseline at Week 6 in Repetitive Behavior Scale-Revised (RBS-R) scores [ Time Frame: 6 weeks ]
  5. Change from Baseline at Week 6 in Vineland Adaptive Behavior Scales (VABS)-Socialization subscale score [ Time Frame: 6 weeks ]
  6. Change from Baseline at Week 6 in Caregiver Strain Questionnaire (CGSQ) scores [ Time Frame: 6 weeks ]
  7. Proportion of patients with at least 25% reduction from Baseline in ABC-Irritability subscale score at Week 6 [ Time Frame: 6 weeks ]
  8. Proportion of patients with CGI-I of irritability score of 1 (very much improved) or 2 (much improved) at Week 6 [ Time Frame: 6 weeks ]
  9. Proportion of patients with at least 25% reduction from Baseline in ABC-Irritability subscale score and CGI-I of irritability score of 1 or 2 at Week 6 [ Time Frame: 6 weeks ]

Eligibility Criteria
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Ages Eligible for Study:   5 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

INCLUSION CRITERIA:

  • Male or female and 5 through 17 years of age
  • Informed consent prior to the conduct of any study procedures
  • Patients (to the best of his/her ability), parent/legally accepted representative, and designated caregiver (if applicable) are able to understand the nature of the study, follow protocol requirements, and be willing to comply with study drug administration requirements
  • Able to swallow a test placebo capsule without difficulty
  • Meets Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (DSM-5) criteria for a primary diagnosis of ASD (APA 2013) and diagnosis is confirmed by the Autism Diagnostic Interview-Revised (ADI-R)
  • Score ≥18 on the Irritability subscale of the Aberrant Behavior Checklist (ABC)
  • Score ≥4 (moderate or greater severity) on the Clinical Global Impression-Severity (CGI-S) of irritability score
  • No current comorbid psychiatric disorder other than attention-deficit hyperactivity disorder (ADHD) or anxiety disorder
  • Drug-naïve to antipsychotic treatment (or <2 weeks antipsychotic treatment for any reason), or prior lack of tolerability to adequate dose of any duration of antipsychotic confirmed by caregiver and medical records review
  • If patient is undergoing concurrent behavioral therapy for autism related symptoms or behaviors, this non-pharmacological treatment regimen has been stable for at least 4 weeks, and will be consistent throughout the study
  • For female patients only: unable to become pregnant or agree to use a highly effective non-hormonal method of contraception. Females of childbearing potential must have a negative pregnancy test

EXCLUSION CRITERIA:

  • Requires treatment with a medication prohibited by the protocol, including concomitant psychotropic drugs targeting irritability, including those used off-label (clonidine, guanfacine, and propranolol; lithium, valproate), medications that prolong the QT interval, and strong cytochrome P450 (CYP) 3A4 enzyme (CYP3A4) inhibitors and inducers
  • Changes in medications or medication doses (for medical and allowed comorbid psychiatric conditions) in the last 4 weeks
  • Any known history of angioedema, serotonin or neuroleptic malignant syndromes, dystonic reaction, or tardive dyskinesia, due to an antipsychotic or psychotropic medication
  • At a significant risk of suicide, or is a danger to self or others
  • At risk of significant violent behavior to the extent that participation would pose an undue risk to other patients, caregivers, or others
  • Positive urine drug test
  • Met DSM-5 criteria for substance use disorders within the last 6 months
  • Confirmed genetic disorder associated with ASD, a cognitive and/ or behavioral disturbance or profound intellectual disability (IQ ≤50)
  • History of seizures, unless seizure-free and off epileptic drugs for at least 6 months
  • Any condition that, in the opinion of the Investigator, would interfere with the ability to comply with study instructions, or that might confound the interpretation of the study results or put the subject at undue risk
  • Current evidence, or history within the last 12 weeks, of a serious and/or unstable psychiatric, neurologic, cardiovascular, respiratory, gastrointestinal, renal, hepatic, hematologic, or other medical disorder, including cancer or malignancies
  • Weight <15 kg
  • History or presence on at least one ECG of protocol-defined cardiac conduction abnormalities
  • Known family or personal history or symptoms of long QT syndrome or history of cardiac arrhythmias or risk factors for torsade de pointes and/or sudden death, including symptomatic bradycardia, hypokalemia or hypomagnesemia, and presence of congenital prolongation of the QT interval
  • Any member of the household has suffered from COVID-19 or had a COVID-19 (PCR or immunoglobulin) positive test in the last 4 weeks
  • One or more clinical laboratory test value outside of protocol-defined limits
  • Breastfeeding or lactating, or has a positive pregnancy test result (for patients of childbearing potential)
  • Sensitivity to pimavanserin or any of the excipients
  • Participating in another clinical study of any investigational drug, device, or intervention
  • Participated in greater than 2 interventional pharmaceutical clinical research studies in the last 6 months
  • Additional inclusion/exclusion criteria apply. Subjects will be evaluated at screening to ensure that all criteria for study participation are met.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05523895


Contacts
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Contact: Sharon Ortiz 646-397-7336 Sharon.Ortiz@acadia-pharm.com
Contact: Robert Hofbauer 609-250-6221 rhofbauer@acadia-pharm.com

Locations
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Sponsors and Collaborators
ACADIA Pharmaceuticals Inc.
More Information
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Responsible Party: ACADIA Pharmaceuticals Inc.
ClinicalTrials.gov Identifier: NCT05523895    
Other Study ID Numbers: ACP-103-069
First Posted: August 31, 2022    Key Record Dates
Last Update Posted: May 9, 2024
Last Verified: May 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by ACADIA Pharmaceuticals Inc.:
Randomized
Placebo-controlled
Additional relevant MeSH terms:
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Antiparkinson Agents
Anti-Dyskinesia Agents
Antipsychotic Agents
Tranquilizing Agents
Physiological Effects of Drugs
Psychotropic Drugs
Serotonin Agents
Neurotransmitter Agents
Autistic Disorder
 Autism Spectrum Disorder
Child Development Disorders, Pervasive
Neurodevelopmental Disorders
Mental Disorders
Pimavanserin
Central Nervous System Depressants
Serotonin 5-HT2 Receptor Antagonists
Serotonin Antagonists
Molecular Mechanisms of Pharmacological Action