What side effects are associated with this type of intervention?

Common treatments for Autism Spectrum Disorder (ASD) that are similar to Pimavanserin include selective serotonin reuptake inhibitors (SSRIs) and atypical antipsychotics. SSRIs, such as fluoxetine and sertraline, can cause side effects like gastrointestinal disturbances, sleep issues, and increased agitation. Atypical antipsychotics, such as risperidone and aripiprazole, may lead to weight gain, metabolic changes, sedation, and extrapyramidal symptoms. It is important to monitor these side effects closely and discuss them with a healthcare provider.

What prior FDA approvals exist?

The FDA has approved two antipsychotic medications for the treatment of irritability associated with Autism Spectrum Disorder (ASD): risperidone and aripiprazole. Both drugs are atypical antipsychotics that work by modulating serotonin and dopamine receptors, similar to the mechanism of Pimavanserin, which targets 5-HT2A receptors. These medications help manage symptoms such as aggression, self-injury, and severe tantrums in children with ASD.

What other types of research exist?

Promising novel alternatives to Pimavanserin for treating irritability and agitation in Autism Spectrum Disorder patients include Aripiprazole and Risperidone. Both are second-generation antipsychotics that have been shown to be effective in managing these symptoms in ASD. Additionally, ongoing research into other pharmacological agents and personalized medicine approaches may offer new options in the near future.

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Serotonin Inverse Agonist

Pimavanserin for Autism Spectrum Disorder

Phase 2 & 3

Waitlist Available

Research Sponsored by ACADIA Pharmaceuticals Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Male or female and 5 through 17 years of age

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

Must not have

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; stimulants, and non-stimulant medications), medications that prolong the QT interval, and strong cytochrome P450 (CYP) 3A4 enzyme (CYP3A4) inhibitors and inducers

Any known history of angioedema, serotonin or neuroleptic malignant syndromes, dystonic reaction, or tardive dyskinesia, due to an antipsychotic or psychotropic medication

Timeline

Screening 28 days

Treatment 6 weeks

Follow Up 1 week

Summary

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.

Who is the study for?

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.

What is being tested?

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.

What are the potential side effects?

Potential side effects of Pimavanserin may include changes in heart rhythm, upset stomach, mood alterations like anxiety or depression, weight changes, fatigue, and possibly allergic reactions. The exact side effects will be monitored throughout the trial.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I am between 5 and 17 years old.

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

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My child is between 5 and 17 years old.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am not taking any medication that is not allowed in the study, including certain drugs for mood, heart rhythm, or those affecting liver enzymes.

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I have had severe reactions to mental health medications in the past.

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I have a genetic disorder linked to autism, behavioral issues, or severe intellectual disability.

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

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I weigh less than 15 kg.

Timeline

Screening ~ 28 days1 visit

Treatment ~ 6 weeks10 visits

Follow Up ~ 1 week1 visit

Screening ~ 28 days

Treatment ~ 6 weeks

Follow Up ~1 week

This trial's timeline: 28 days for screening, 6 weeks for treatment, and 1 week for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Change from Baseline at Week 6 in caregiver-rated Aberrant Behavior Checklist (ABC) Irritability subscale score

Side effects data

From 2021 Phase 4 trial • 6 Patients • NCT04188392

33%

sleepiness

17%

skin irritation

17%

diarrhea

17%

headache

17%

weight gain

17%

arthralgia

17%

dry mouth

100%

80%

60%

40%

20%

Study treatment Arm

Open-label Treatment

Trial Design

3Treatment groups

Experimental Treatment

Placebo Group

Group I: Pimavanserin low doseExperimental Treatment1 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 doseExperimental Treatment1 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: PlaceboPlacebo Group1 Intervention

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

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Pimavanserin

2018

Completed Phase 4

~3250

0 / 8Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for Autism Spectrum Disorder (ASD) often target neurotransmitter systems to alleviate symptoms such as irritability, agitation, and self-injurious behaviors. Pimavanserin, a selective serotonin inverse agonist and antagonist at 5-HT2A receptors, works by modulating serotonin activity, which is crucial for mood regulation and social behavior. This mechanism is particularly relevant for ASD patients as it can help manage behavioral symptoms without the side effects commonly associated with traditional antipsychotics. Other treatments may include SSRIs, which increase serotonin levels to improve mood and reduce anxiety, and atypical antipsychotics, which target multiple neurotransmitter receptors to address a range of symptoms. Understanding these mechanisms is essential for tailoring treatments to individual needs and improving overall quality of life for ASD patients.

Common Defects of Spine Dynamics and Circuit Function in Neurodevelopmental Disorders: A Systematic Review of Findings From in Vivo Optical Imaging of Mouse Models.