Pimavanserin for Autism Spectrum Disorder

No longer recruiting at 83 trial locations

Overseen By Jennifer Nicole Tomer

Age: < 18

Sex: Any

Travel: May Be Covered

Trial Phase: Phase 2 & 3

Sponsor: ACADIA Pharmaceuticals Inc.

Must not be taking: Psychotropic drugs, QT prolonging, CYP3A4 inhibitors

Disqualifiers: Substance use, Seizures, Genetic disorder, others

Prior Safety Data This treatment has passed at least one previous human trial

Breakthrough Therapy This drug has been fast-tracked for approval by the FDA given its high promise

Compensation: Varies

Number of Visits: Unknown

Duration: 6 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores the effectiveness and safety of pimavanserin for children and teens with autism who experience irritability, agitation, or self-injury. Participants will receive either varying doses of pimavanserin or a placebo (a look-alike pill with no active medication) for comparison. It is ideal for those diagnosed with autism spectrum disorder who frequently encounter these challenging behaviors. Participants should not have other psychiatric disorders, except for ADHD or anxiety, and should avoid certain medications. As a Phase 2, Phase 3 trial, this study is crucial for determining pimavanserin's efficacy and is in the final step before potential FDA approval, offering participants a chance to contribute to significant advancements in autism treatment.

Will I have to stop taking my current medications?

The trial requires stopping certain medications, including those that target irritability, medications that affect heart rhythm, and certain enzyme inhibitors and inducers. If you're on these, you may need to stop them before joining the trial.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that pimavanserin is generally safe for use. It is primarily approved for treating hallucinations and delusions in Parkinson's disease, indicating its safety. In past studies on autism spectrum disorder (ASD), researchers tested pimavanserin for safety in children and teenagers. These studies found that most patients did not experience serious side effects.

However, like any treatment, some unwanted effects may occur. Common issues include nausea and fatigue. It's important to remember that while the treatment appears promising, individual reactions can vary. Always consult a healthcare provider about possible risks before starting any new medication. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for autism?

Most treatments for Autism Spectrum Disorder, like behavioral therapies and medications such as antipsychotics, focus on managing symptoms rather than addressing underlying causes. Pimavanserin is unique because it targets the serotonin 5-HT2A receptor, a different mechanism of action from typical antipsychotics, which often work on dopamine pathways. This distinct approach may offer new benefits, potentially improving social behaviors and communication in individuals with autism without the common side effects associated with current medications. Researchers are excited about pimavanserin because it could provide a more effective and safer alternative for managing core symptoms of autism.

What evidence suggests that pimavanserin might be an effective treatment for autism spectrum disorder?

Research suggests that pimavanserin might help with symptoms of autism spectrum disorder (ASD), such as irritability and agitation. In this trial, participants will receive either a high or low dose of pimavanserin, or a placebo. Studies have shown that pimavanserin effectively manages similar symptoms in other conditions, like Parkinson's disease psychosis, where it reduces hallucinations and delusions without worsening movement problems. Early trials with people who have ASD showed that pimavanserin was safe and well-tolerated, and it may help reduce irritability and other challenging behaviors. This effect likely occurs because pimavanserin targets certain brain areas that influence mood and perception. Although more research is needed to confirm these findings, the initial results are promising. ^{1 4 5 6 7}

Are You a Good Fit for This Trial?

This trial is for children and adolescents aged 5 to 17 with Autism Spectrum Disorder (ASD) who show irritability, agitation, or self-injurious behaviors. They must be able to swallow pills, have not used antipsychotic drugs before or had issues with them, and meet specific criteria for ASD diagnosis. Girls must use non-hormonal contraception if of childbearing age.

Inclusion Criteria

I, or my caregiver, understand the study and can follow its requirements.

I have either never taken antipsychotic drugs or only did for less than 2 weeks.

I am a woman who cannot get pregnant or will use effective birth control.

Exclusion Criteria

You are at risk of hurting yourself or others, including having suicidal thoughts.

I have been seizure-free and not taken epilepsy medication for over 6 months.

Someone in my home had COVID-19 or tested positive for it in the last month.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive pimavanserin or placebo for 6 weeks to evaluate efficacy and safety for irritability associated with ASD

6 weeks

Weekly visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Pimavanserin
• Placebo

Trial Overview

The study tests the effectiveness and safety of Pimavanserin compared to a placebo over six weeks in managing irritability associated with ASD. Participants are randomly assigned to either the medication or placebo group in a double-blind manner so neither they nor the researchers know who's receiving which treatment.

How Is the Trial Designed?

Group I: Pimavanserin low dose Experimental Treatment 1 Intervention

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

Group II: Pimavanserin high dose Experimental Treatment 1 Intervention

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

Group III: Placebo Placebo Group 1 Intervention

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

Pimavanserin is already approved in United States for the following indications:

Approved in United States as Nuplazid for:

• Hallucinations and delusions associated with Parkinson's disease psychosis

Who Is Running the Clinical Trial?

ACADIA Pharmaceuticals Inc.

Lead Sponsor

Trials

49

Recruited

11,700+

Founded

1993

Headquarters

San Diego, USA

Known For

Neurological Disorders

Top Products

Nuplazid (pimavanserin), Daybue (trofinetide)

Published Research Related to This Trial

Pimavanserin is the first FDA-approved drug specifically for treating hallucinations and delusions in Parkinson's disease psychosis, showing significant improvement in symptoms compared to placebo in a pivotal phase III clinical trial.

The drug works as a potent inverse agonist at serotonin 5-HT2A receptors, with a long half-life of 57 hours, allowing for once-daily dosing and demonstrating a favorable safety profile without impairing motor functions.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pimavanserin: novel pharmacotherapy for Parkinson's disease psychosis. Sahli, ZT., Tarazi, FI.[2018]

In a chart review of 26 patients with Parkinson's disease and related conditions, nearly 50% found Pimavanserin (Pim) helpful for managing psychotic symptoms, with 12 patients continuing treatment after 6 weeks.

While some patients experienced adverse effects leading to discontinuation, the overall safety profile suggests that intolerance was not directly related to the medication itself.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pimavanserin for Psychotic Symptoms in People With Parkinsonism: A Second Chart Review. Friedman, JH.[2018]

In a study involving 12 adults with Tourette syndrome, pimavanserin treatment led to a statistically significant reduction in tic severity, with a 12% decrease in the Yale Global Tic Severity Scale score after 8 weeks, although this improvement may not be clinically significant.

Pimavanserin was found to be safe, with no serious adverse events reported, and it also showed positive effects on non-motor symptoms and quality of life, suggesting potential benefits that merit further investigation in larger trials.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pilot Study to Evaluate Pimavanserin for the Treatment of Motor and Behavioral Symptoms of Tourette Syndrome. Billnitzer, A., Jankovic, J.[2022]

Pimavanserin is a selective serotonin 2A receptor inverse agonist that has been approved in the US specifically for treating hallucinations and delusions in patients with Parkinson's disease psychosis, a condition affecting up to 60% of Parkinson's patients.

The approval of pimavanserin is significant as it provides a treatment option for a condition that has limited therapeutic alternatives and is associated with increased morbidity and mortality.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pimavanserin: First Global Approval. Markham, A.[2022]

Pimavanserin is the first FDA-approved treatment for Parkinson's disease psychosis (PDP) and has been shown to be well-tolerated with minimal adverse effects and no worsening of motor symptoms, based on four randomized controlled trials.

A Phase III trial demonstrated that pimavanserin 40mg significantly improved PDP symptoms compared to placebo, with notable improvements in secondary outcomes such as caregiver burden and sleep quality, highlighting its efficacy as a treatment option for PDP.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pimavanserin: A Novel Drug Approved to Treat Parkinson's Disease Psychosis. Kitten, AK., Hallowell, SA., Saklad, SR., et al.[2021]

Citations

1.

clinicaltrials.gov

clinicaltrials.gov/study/NCT05523895

Study Details | NCT05523895 | Pimavanserin for the ...

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 ...

2.

clinicaltrials.gov

clinicaltrials.gov/study/NCT05999240

Pimavanserin for Rigid-compulsive Symptoms in Autism ...

This Phase 2 study examines the safety, tolerability, and preliminary efficacy of pimavanserin in individuals with Autism Spectrum Disorder.

3.

ctv.veeva.com

ctv.veeva.com/study/pimavanserin-for-the-treatment-of-irritability-associated-with-autism-spectrum-disorder

Pimavanserin for the Treatment of Irritability Associated With ...

6-week, randomized, double-blind, fixed-dose, placebo-controlled, parallel group study in children and adolescents (aged 5 to17 years) with autism spectrum ...

4.

clinicalresearch.com

clinicalresearch.com/en/find-trials/Study/NCT05555615

NCT05555615

ACP-103-069 is a 6-week, randomized, double-blind, fixed-dose, placebo controlled, parallel group study of pimavanserin in children and adolescents with ...

5.

centerwatch.com

centerwatch.com/clinical-trials/listings/NCT05523895/pimavanserin-for-the-treatment-of-irritability-associated-with-autism-spectrum-disorder

Pimavanserin for the Treatment of Irritability Associated ...

This study will be conducted as a 6-week, randomized, double-blind, fixed-dose, placebo-controlled, parallel group study in children and ...

6.

nuplazidhcp.com

nuplazidhcp.com/safety-tolerability-data

Safety and Tolerability Data | NUPLAZID® (pimavanserin)

Learn about NUPLAZID® safety data. See Important Safety Information and Full Prescribing Information, including Boxed WARNING.

7.

clinicaltrials.eu

clinicaltrials.eu/trial/study-on-the-long-term-safety-of-pimavanserin-for-treating-irritability-in-children-and-adolescents-with-autism-spectrum-disorder/

Study on the Long-Term Safety of Pimavanserin for ...

Pimavanserin is being studied for its long-term safety and tolerability in children and adolescents with irritability associated with Autism Spectrum Disorder ( ...