Pimavanserin for the Treatment of Irritability Associated With Autism Spectrum Disorder

NCT05523895 | Completed Phase 2 Results DMC FDA Drug

• 1 other identifier: ACP-103-069
• Study Type: interventional
• Target: 237
• Locations: 8 countries
• Sites: 57

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

Conditions

Irritability Associated With Autism Spectrum Disorder

Keywords

Randomized; Placebo-controlled

Outcome Measures

Primary Outcomes (1)

• Change From Baseline at Week 6 in Caregiver-rated Aberrant Behavior Checklist Irritability (ABC-I) Subscale Score

  The Aberrant Behavior Checklist (ABC) is a caregiver-rated scale comprised of 5 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-I is one of the subscales and comprises of 15 items. Minimum score is 0, maximum is 45. 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.

  6 weeks

Study Arms (3)

Placebo

PLACEBO COMPARATOR

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

Drug: Placebo

Pimavanserin low dose

EXPERIMENTAL

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 high dose

EXPERIMENTAL

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

Interventions

Pimavanserin DRUG

Pimavanserin

Also known as: Nuplazid

Pimavanserin high dose; Pimavanserin low dose

Placebo DRUG

Pimavanserin matching placebo

Placebo

Eligibility Criteria

• Age: 5 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• ACADIA Pharmaceuticals Inc.: lead

Study Sites (57)

Southwest Autism Research & Resource Center

Phoenix, Arizona, 85006, United States

Location

Cortica Inc. (Glendale)

Glendale, California, 91361, United States

Location

Cortica Inc. (San Rafael)

San Rafael, California, 94903, United States

Location

1st Allergy and Clinical Research Group, d/b/a IMUNOe Research Centers

Centennial, Colorado, 80112, United States

Location

Children's Research Institute

Washington D.C., District of Columbia, 20010, United States

Location

The EHS Medical Practice, PA, D/B/A Sarkis Clinical Trials

Gainesville, Florida, 32607, United States

Location

APG Research, LLC

Orlando, Florida, 32803, United States

Location

AMR Baber Research Incorporated

Naperville, Illinois, 60563, United States

Location

Clinical Research of Southern Nevada, LLC

Las Vegas, Nevada, 89128, United States

Location

ERG Clinical Research - New York, PLLC DBA Richmond Behavioral Associates

Staten Island, New York, 10314, United States

Location

Quest Therapeutics of Avon Lake dba Haidar Almhana Nieding LLC

Avon Lake, Ohio, 44012, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19146, United States

Location

Medical University of South Carolina

Charleston, South Carolina, 29425, United States

Location

Relaro Medical Trials, LLC

Dallas, Texas, 75243, United States

Location

Red Oak Psychiatry Associates, PA

Houston, Texas, 77090, United States

Location

AIM Trials, LLC

Plano, Texas, 75093, United States

Location

Eastside Therapeutic Resource, Inc. dba Core Clinical Research

Everett, Washington, 98201, United States

Location

Children's Health Queensland Hospital and Health Service

South Brisbane, QDL, 4101, Australia

Location

Monash Health

Clayton, Victoria, 3168, Australia

Location

Murdoch Children's Research Institute

Parkville, Victoria, 3052, Australia

Location

Centre Hospitalier Charles Perrens

Bordeaux, 33076, France

Location

Centre de Ressources Autisme Rhône-Alpes - Center Hospitalier le Vinatier

Bron, 69678, France

Location

CHU de Nantes

Nantes, 44093, France

Location

L'Assistance Publique - Hôpitaux de Paris, labélisé Institut Carnot

Paris, 75019, France

Location

Vadaskert Hospital

Budapest, H-1021, Hungary

Location

Magyarországi Református Egyház Bethesda Gyermekkórháza

Budapest, H-1146, Hungary

Location

Békés Megyei Központi Kórház

Gyula, H-5700, Hungary

Location

Szegedi Tudományegyetem

Szeged, H-6725, Hungary

Location

Azienda Ospedaliero Universitaria (AOU) Consorziale Policlinico

Bari, 70124, Italy

Location

La Nostra Famiglia - Scientifica IRCCS Eugenio Medea

Bosisio Parini, 23842, Italy

Location

The Childhood and Adolescence Neuropsychiatry Clinic - University of Cagliari and Pediatric Hospital "A. Cao" - ASL 8 of Cagliari

Cagliari, 09134, Italy

Location

Policlinico Riuniti - Azienda Ospedaliero Universitaria

Foggia, 71122, Italy

Location

IRCCS Istituto Giannina Gaslini

Genova, 16147, Italy

Location

Azienda Ospedaliera Universitaria "Federico II"

Napoli, 80131, Italy

Location

Fondazione Istituto Neurologico Nazionale Casimiro Mondino - IRCCS

Pavia, 27100, Italy

Location

Fondazione PTV - Policlinico Tor Vergata

Roma, 00133, Italy

Location

IRCCS Ospedale Pediatrico Bambino Gesù

Rome, 00165, Italy

Location

Azienda Ospedaliera-Universitaria Senese

Siena, 53100, Italy

Location

Azienda Ospedaliera Universitaria Integrata di Verona (AOUI)

Verona, 37126, Italy

Location

Gdańskie Centrum Zdrowia Sp. z o.o.

Gdansk, 80-542, Poland

Location

Centrum Badań Klinicznych PI-House Sp. z o.o.

Gdansk, 80-546, Poland

Location

NAVICULA - Centrum Diagnozy i Terapii Autyzmu

Lodz, 91-129, Poland

Location

Ginemedica Sp. Zoo, S.K.

Wroclaw, 50-414, Poland

Location

Centrum Neuropsychiatrii NEUROMED SP ZOZ

Wroclaw, 54-238, Poland

Location

MedicMental Indywidualna Specjalistyczna Praktyka Lekarska Monika Szewczuk-Boguslawska

Wroclaw, 54-617, Poland

Location

Institute of Mental Health

Belgrade, 11000, Serbia

Location

University Clinical Center Kragujevac, Clinic for Psychiatry

Kragujevac, 34000, Serbia

Location

University Clinical Center Nis, Center for Mental Health

Niš, 18000, Serbia

Location

Clinical Center of Vojvodina, Clinic for Psychiatry

Novi Sad, 21000, Serbia

Location

Hospital General de Alicante

Alicante, 03010, Spain

Location

Institut Global d´Atenció Integral del Neurodesenvolupament (IGAIN)

Barcelona, 08007, Spain

Location

Hospital Universitari Vall d'Hebron

Barcelona, 08035, Spain

Location

Hospital Clínic de Barcelona

Barcelona, 08036, Spain

Location

Complejo Asistencial Universitario de Burgos

Burgos, 09006, Spain

Location

Hospital Universitario Infanta Leonor

Madrid, 28031, Spain

Location

Complejo Asistencial de Zamora

Zamora, 49021, Spain

Location

MeSH Terms

Interventions

pimavanserin

Results Point of Contact

• Title: Sr. Dir. Medical Information and Medical Communications
• Organization: Acadia Pharmaceuticals Inc.

medicalinformation@acadia-pharm.com

858-261-2289

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 29, 2022
• First Posted: August 31, 2022
• Study Start: August 9, 2022
• Primary Completion: September 27, 2024
• Study Completion: September 27, 2024
• Last Updated: August 24, 2025
• Results First Posted: August 24, 2025

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will not share

Locations

IT (11); SE (4); US (18); ES (7); HU (4); AU (3); PL (6); FR (4)