Extension Study of Pimavanserin in Irritability Associated With Autism Spectrum Disorder

NCT05555615 | Terminated Phase 2 Results DMC FDA Drug

• 1 other identifier: ACP-103-070
• Study Type: interventional
• Target: 209
• Locations: 8 countries
• Sites: 48

Brief Summary

52-week, open-label extension study of double-blind study ACP-103-069 to determine the long-term safety and tolerability of pimavanserin for the treatment of irritability associated with ASD in children and adolescents (aged 5 to 17 years). ACP-103-069 is a 6-week, randomized, double-blind, fixed-dose, placebo controlled, parallel group study of pimavanserin in children and adolescents with irritability associated with autism spectrum disorder (ASD).

Conditions

Irritability Associated With Autism Spectrum Disorder

Outcome Measures

Primary Outcomes (1)

• Treatment-emergent Adverse Events

  Number (%) of patients with treatment emergent adverse events

  52 weeks

Secondary Outcomes (1)

• Aberrant Behavior Checklist-Irritability (ABC-I) and Clinical Global Impression-Improvement (CGI-I) Response Rate

  52 weeks

Study Arms (1)

Pimavanserin

EXPERIMENTAL

Pimavanserin once daily. All patients will receive the pimavanserin low dose (patients aged 5 to 12 years: 10 mg/day pimavanserin; patients aged 13 to 17 years: 20 mg/day) the first 2 weeks of the study. Thereafter, the dose may be increased to the high dose (5 to 12 years: 20 mg/day; 13 to 17 years: 34 mg/day) based on the Investigator's assessment of clinical response. After Week 2 and up to Week 20, dose adjustments are allowed at any clinic visit based on the Investigator's assessment of clinical response and tolerability. No further dose adjustments are allowed after Week 20.

Drug: Pimavanserin

Interventions

Pimavanserin DRUG

Pimavanserin given once daily, as capsule of 10, 20, or 34 mg dose strength, respectively, according to the patient's age

Also known as: Nuplazid

Pimavanserin

Eligibility Criteria

• Age: 5 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Has completed the treatment period of study ACP-103-069
• Informed consent prior to the conduct of any study procedures
• Continues to be both clinically stable and not at imminent risk of suicide or injury to self, others, or property
• Continues to be medically stable at enrollment
• 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:

• Patient or parent/legally accepted representative is judged by the Investigator to be inappropriate for the study
• 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
• 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
• Serious and/or unstable psychiatric, neurologic, cardiovascular, respiratory, gastrointestinal, renal, hepatic, hematologic, or other medical disorder, including cancer or malignancies
• Any change in medical or treatment status that may increase the risk associated with taking pimavanserin, would interfere with safety assessments, or would confound the interpretation of study results
• Clinically significant abnormal ECG of protocol-defined cardiac conduction abnormalities
• Weight \<15 kg

Sponsors & Collaborators

• ACADIA Pharmaceuticals Inc.: lead

Study Sites (48)

Southwest Autism Research and Resource Center

Phoenix, Arizona, 85006, United States

Location

Cortica Inc. (Glendale)

Glendale, California, 91361, United States

Location

Cortica Inc.

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, 32803-3809, 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, Queensland, 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

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

La Nostra Famiglia - Scientifica IRCCS Eugenio Medea

Bosisio Parini, LC, 23842, Italy

Location

Policlinico Riuniti - Azienda Ospedaliero Universitaria

Foggia, 71122, Italy

Location

Azienda Ospedaliera Universitaria "Federico II"

Naples, 80131, Italy

Location

Fondazione Istituto Neurologico Nazionale Casimiro Mondino - IRCCS

Pavia, 27100, Italy

Location

Fondazione PTV - Policlinico Tor Vergata

Rome, 00133, 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

MeSH Terms

Interventions

pimavanserin

Limitations and Caveats

The study was terminated early by the Sponsor due to lack of efficacy in the antecedent double-blind study (ACP-103-069).

Results Point of Contact

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

medicalinformation@acadia-pharm.com

844-422-2342

Publication Agreements

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

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 30, 2022
• First Posted: September 27, 2022
• Study Start: November 2, 2022
• Primary Completion: February 14, 2025
• Study Completion: February 14, 2025
• Last Updated: December 3, 2025
• Results First Posted: December 3, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will not share

Locations

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