Pimavanserin for Rigid-compulsive Symptoms in Autism Spectrum Disorder

NCT05999240 | Recruiting Phase 2 FDA Drug

• 1 other identifier: 8449
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 2

Brief Summary

This Phase 2 study examines the safety, tolerability, and preliminary efficacy of pimavanserin in individuals with Autism Spectrum Disorder. Male or female participants aged 12 to 40 years of age will be randomized to receive single doses of either placebo or pimavanserin in this randomized, placebo-controlled, cross-over designed study, followed by open label extension.

Conditions

Autism Spectrum Disorder

Keywords

Pimavanserin; Nuplazid; Antiparkinson Agents; Antipsychotic Agents; Serotonin 5-HT2 Receptor Antagonist; Physiological Effects of Drugs; Serotonin Agents; Autism Spectrum Disorder; Autistic Disorder

Outcome Measures

Primary Outcomes (2)

• Evaluate the impact of a single dose of pimavanserin 34 mg versus placebo

  Assessed using cognitive neuroscience task, comparison between pimavanserin and placebo within participants in single-dose cross-over experiment.

  Post Intervention, 30 days after study completion

• Evaluate pimavanserin for treatment of restricted/repetitive behavior in ASD

  Assessed using the Repetitive Behavior Scale - Revised (RBS-R) rated at baseline, 3, and 6 weeks. The RBS-R is a 44-item self-report form used to measure the wide range of repetitive behavior in children, adolescents, and adults with Autism Spectrum disorders. The scale consists of six subscales with questions related to Stereotyped Behavior, Self-injurious Behavior, Compulsive Behavior, Routine Behavior, Sameness Behavior, and Restricted Behavior rated between 0 to 3 on severity of each item.

  6 weeks of Open-Label treatment

Secondary Outcomes (1)

• Explore the effects of pimavanserin on an electroencephalogram event-related potential (EEG/ERP) reversal learning test

  Post 6-week treatment

Study Arms (2)

Pimavanserin tartrate

EXPERIMENTAL

Pimavanserin 34 mg white to off-white capsules.

Drug: Pimavanserin Tartrate 34 Mg ORAL CAPSULE [NUPLAZID]

Placebo

PLACEBO COMPARATOR

Placebo to Pimavanserin tartrate

Drug: Placebo

Interventions

Pimavanserin Tartrate 34 Mg ORAL CAPSULE [NUPLAZID] DRUG

Participants will be randomized to take a single dose of either pimavanserin 34 mg or placebo at Visit 2.

Also known as: Nuzplazid

Pimavanserin tartrate

Placebo DRUG

Placebo for Pimavanserin Tartrate 34 Mg ORAL CAPSILE \[NUPLAZID\]

Placebo

Eligibility Criteria

• Age: 12 Years - 40 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Participant must be at least 12 years old and no greater than 40 years old.
• Participants 18 years of age or older or a legally acceptable representative must be able and willing to sign an informed consent document indicating understanding of the protocol and procedures and willingness to participate in full. For a participant who is under 18 years of age, a parent or guardian must sign an informed consent document indicating understanding the protocol and procedures and willingness to participate in full. When appropriate to the participants developmental level and age, assent will be used to indicate understanding and willingness to participant. If the participant is unable to sign the assent form, the participant must provide verbal or non-verbal confirmation of willingness to continue with study procedures. Each site will abide by their respective institutional requirements regarding consent of adult informants. For adult participants at the New York site only, a parent, caregiver, or other adult informant must sign an informed consent document indicating understanding the requirements for completing their portion of the study and their willingness to participate, due to institutional human subjects requirements.
• Participant must have a diagnosis of Autism Spectrum Disorder, according to DSM-5 criteria, made by a licensed study psychiatrist or psychologist and supported by the Autism Diagnostic Observation Schedule 2 (ADOS-2) completed at screening or within the past 12 months prior to screening by an appropriately trained professional.
• Participant must have a Clinical Global Impression of Severity for Repetitive Behavior of 4 or greater, as rated by the study psychiatrist or psychologist at Screening and at Baseline
• Participant must have a non-verbal IQ of greater than or equal to 70 as determined by the 4-subtest Wechsler Abbreviated Scale of Intelligence.
• Participant must be able to speak and understand English in order to complete study measures.
• Participant must live with a parent, primary caregiver, or other adult informant who can complete study measures on the basis of spending an average of at least 4 hours per day with the participant
• Parent, primary caregiver, or other adult informant must speak and understand English in order to complete study measures.
• Participant must be able to self-administer study medication or have parent/caregiver be able to administer study medication.
• Participant must be able to swallow study medication whole with liquid.
• Participant or legally acceptable representative must be willing to continue current medication(s) and behavioral intervention(s) and to not add or change medication(s) or behavioral intervention(s) over the full course of the study.

You may not qualify if:

• Participant is judged by the Investigator to be unable to perform or comply with all study specific requirements.
• Participant is an employee of an investigator with direct involvement in the proposed study or other studies under the direction of a study investigator, or is a family member of an investigator.
• Participant has a current or recent history of clinically significant suicidal ideation within the past 6 months, corresponding to a score of 3 or higher (active suicidal ideation with some intent to act, without specific plan) on the Columbia Suicide Severity Rating Scale (C-SSRS), or a history of suicidal behavior within the past year, as validated by the C-SSRS at screening or Day 1.
• Participant has met DSM-5 criteria for a substance abuse disorder within the last 6 months prior to Screening, except for disorders related to caffeine or nicotine.
• Participant has a positive test for an illicit drug or cannabis at Screening or Baseline. Participants who test positive for cannabis and who have a valid prescription may be rescreened if they agree to abstain from the cannabis for the length of their participation in the study. The repeat test must be negative for them to participate in the study.
• Participant is taking a serotonin reuptake inhibitor or antipsychotic medication.
• Participant has had a change to psychotropic medications within the last 2 months
• Participant has received electroconvulsive therapy (ECT) in the last 6 months.
• Participant has received new-onset psychotherapy or has had a change in the intensity of psychotherapy within the 2 months prior to Screening.
• Participant has known allergies, hypersensitivity, or intolerance to Pimavanserin or its excipients.
• Participant has received an investigational drug or used an investigational medical device within 3 months before the planned start of study or is currently enrolled in an investigational study.
• Participant has a body mass index (BMI) \<17 or \>40 at Screening.
• Participant has a known history of long QT syndrome or family history of sudden death.
• Participant has a history of myocardial infarction, unstable angina, acute coronary syndrome, or cerebrovascular accident (CVA) within the last 4 months. Has greater than NYHA Class 2 congestive heart failure or Class 2 angina pectoris, sustained ventricular tachycardia (VT), ventricular fibrillation, torsade de pointes, or syncope due to an arrhythmia.
• Participant has a history of neuroleptic malignant syndrome/serotonin syndrome.

Sponsors & Collaborators

• ACADIA Pharmaceuticals Inc.: collaborator
• University of Kansas Medical Center: collaborator
• Research Foundation for Mental Hygiene, Inc.: collaborator
• Weill Medical College of Cornell University: collaborator
• New York State Psychiatric Institute: lead

Study Sites (2)

University of Kansas, Center for Research

Lawrence, Kansas, 66045, United States

RECRUITING

Center for Autism and the Developing Brain

White Plains, New York, 10605, United States

RECRUITING

MeSH Terms

Conditions

Autism Spectrum Disorder; Autistic Disorder

Interventions

pimavanserin

Condition Hierarchy (Ancestors)

Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Study Officials

• Jeremy Veenstra-VanderWeele, MD

  New York State Psychiatric Institute

  PRINCIPAL INVESTIGATOR

• Matthew Mosconi, PhD

  University of Kansas Center for Autism Research and Training

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Alyssa Verdes, BA

CONTACT

914-997-5532; alyssa.verdes@nyspi.columbia.edu

Suvekcha Bhattachan, MA

CONTACT

914-997-5587; suvekcha.bhattachan@nyspi.columbia.edu

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Masking Details: Information regarding Masking will be communicated to the investigational pharmacy only - all other staff will remain blind to group assignment throughout the study.
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: MD

Model Details: This study plans to enroll 30 participants between the ages of 12-40 years old with a diagnosis of autism spectrum disorder to participate in a randomized, placebo-controlled, cross-over designed study. Participants will be randomized to take a single dose of either pimavanserin 34 mg or placebo (Visit 2) three hours before beginning a cognitive neuroscience battery. One week later (Visit 3), participants will take the other medication (placebo or pimavanserin 34 mg) three hours before beginning the cognitive neuroscience battery. After completing the second experimental day, participants will immediately enter the Open Label Trial, where they will take pimavanserin 34 mg for six weeks total, with study visits at 3 weeks (Visit 4) and 6 weeks (Visit 5) to complete safety and primary/secondary outcome measures, followed by a follow-up phone call within 30 days after completing the open label trial.

Study Record Dates

• First Submitted: August 13, 2023
• First Posted: August 21, 2023
• Study Start: October 1, 2025
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): December 31, 2026
• Last Updated: April 1, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)