Pimavanserin for Autism Spectrum Disorder

Recruiting at 76 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

237 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing pimavanserin, a medication that may help reduce irritability, agitation, and self-harm in young individuals with autism. The study focuses on those who struggle with these challenging behaviors. Pimavanserin works by balancing brain chemicals to improve mood and behavior.

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.

How is the drug Pimavanserin unique for treating Autism Spectrum Disorder?

Pimavanserin is unique because it is a selective serotonin 2A receptor inverse agonist, originally approved for treating hallucinations and delusions in Parkinson's disease psychosis, and it may offer a novel approach for Autism Spectrum Disorder by targeting serotonin pathways differently than other treatments. ^{1 2 3 4 5}

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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)

Findings from Research

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 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]

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]

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]

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]

References

1. United States pubmed.ncbi.nlm.nih.gov

Pilot Study to Evaluate Pimavanserin for the Treatment of Motor and Behavioral Symptoms of Tourette Syndrome. [2022]

2. United States pubmed.ncbi.nlm.nih.gov

Pimavanserin: A Novel Drug Approved to Treat Parkinson's Disease Psychosis. [2021]

3. New Zealand pubmed.ncbi.nlm.nih.gov

Pimavanserin: First Global Approval. [2022]

4. United States pubmed.ncbi.nlm.nih.gov

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

5. England pubmed.ncbi.nlm.nih.gov

Pimavanserin: novel pharmacotherapy for Parkinson's disease psychosis. [2018]