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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT05523895




Registration number
NCT05523895
Ethics application status
Date submitted
29/08/2022
Date registered
31/08/2022
Date last updated
10/06/2024

Titles & IDs
Public title
Pimavanserin for the Treatment of Irritability Associated With Autism Spectrum Disorder
Scientific 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
Secondary ID [1] 0 0
ACP-103-069
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Irritability Associated With Autism Spectrum Disorder 0 0
Condition category
Condition code
Mental Health 0 0 0 0
Autistic spectrum disorders
Mental Health 0 0 0 0
Other mental health disorders
Neurological 0 0 0 0
Other neurological disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Pimavanserin
Treatment: Drugs - Placebo

Placebo Comparator: Placebo - Placebo given once daily, as one capsule matching in size and color the respective pimavanserin treatment

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

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


Treatment: Drugs: Pimavanserin
Pimavanserin

Treatment: Drugs: Placebo
Pimavanserin matching placebo
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change from Baseline at Week 6 in caregiver-rated Aberrant Behavior Checklist (ABC) Irritability subscale score
Timepoint [1] 0 0
6 weeks
Secondary outcome [1] 0 0
Change from Baseline at Week 6 in caregiver-rated ABC subscale scores: Stereotypic Behavior; Lethargy; Hyperactivity; Inappropriate speech
Timepoint [1] 0 0
6 weeks
Secondary outcome [2] 0 0
Change from Baseline at Week 6 in Clinical Global Impression-Severity (CGI-S) of Irritability score
Timepoint [2] 0 0
6 weeks
Secondary outcome [3] 0 0
Clinical Global Impression-Improvement (CGI-I) of irritability score at Week 6
Timepoint [3] 0 0
6 weeks
Secondary outcome [4] 0 0
Change from Baseline at Week 6 in Repetitive Behavior Scale-Revised (RBS-R) scores
Timepoint [4] 0 0
6 weeks
Secondary outcome [5] 0 0
Change from Baseline at Week 6 in Vineland Adaptive Behavior Scales (VABS)-Socialization subscale score
Timepoint [5] 0 0
6 weeks
Secondary outcome [6] 0 0
Change from Baseline at Week 6 in Caregiver Strain Questionnaire (CGSQ) scores
Timepoint [6] 0 0
6 weeks
Secondary outcome [7] 0 0
Proportion of patients with at least 25% reduction from Baseline in ABC-Irritability subscale score at Week 6
Timepoint [7] 0 0
6 weeks
Secondary outcome [8] 0 0
Proportion of patients with CGI-I of irritability score of 1 (very much improved) or 2 (much improved) at Week 6
Timepoint [8] 0 0
6 weeks
Secondary outcome [9] 0 0
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
Timepoint [9] 0 0
6 weeks

Eligibility
Key inclusion 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.
Minimum age
5 Years
Maximum age
17 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2/Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
9/08/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/09/2024
Actual
Sample size
Target
228
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
QDLVIC
Recruitment hospital [1] 0 0
Children's Health Queensland Hospital and Health Service - South Brisbane
Recruitment hospital [2] 0 0
Monash Health - Clayton
Recruitment hospital [3] 0 0
Murdoch Children's Research Institute - Parkville
Recruitment postcode(s) [1] 0 0
4101 - South Brisbane
Recruitment postcode(s) [2] 0 0
3168 - Clayton
Recruitment postcode(s) [3] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
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Arizona
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United States of America
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California
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United States of America
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Colorado
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United States of America
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District of Columbia
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United States of America
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Florida
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United States of America
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Illinois
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Michigan
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Nevada
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New Jersey
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New York
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Ohio
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Pennsylvania
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South Carolina
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Texas
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Utah
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Washington
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France
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Bordeaux
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France
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Bron
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France
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Nantes
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France
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Paris
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France
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Rouen
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Hungary
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Budapest
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Hungary
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Gyula
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Hungary
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Szeged
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Italy
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Bari
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Italy
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Bosisio Parini
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Italy
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Cagliari
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Foggia
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Genova
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Italy
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Napoli
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Italy
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Pavia
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Italy
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Roma
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Italy
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Rome
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Italy
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Siena
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Italy
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Verona
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Poland
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Gdansk
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Poland
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Wroclaw
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Poland
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Lódz
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Serbia
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Belgrade
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Serbia
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Kragujevac
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Serbia
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Nis
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Serbia
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Novi Sad
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Spain
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Alicante
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Spain
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Barcelona
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Burgos
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Madrid
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Spain
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Zamora

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
ACADIA Pharmaceuticals Inc.
Address
Country

Ethics approval
Ethics application status

Summary
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
Trial website
https://clinicaltrials.gov/ct2/show/NCT05523895
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Sharon Ortiz
Address 0 0
Country 0 0
Phone 0 0
646-397-7336
Fax 0 0
Email 0 0
Sharon.Ortiz@acadia-pharm.com
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT05523895