Cariprazine for Autism Spectrum Disorder

Recruiting at 43 trial locations

Overseen By ABBVIE CALL CENTER

Age: < 18

Sex: Any

Trial Phase: Phase 3

Sponsor: AbbVie

Disqualifiers: Intellectual disability, Major depressive disorder, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

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

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 8 weeks

161 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the study doctors to understand how your current medications might interact with the trial.

What data supports the effectiveness of the drug Cariprazine for Autism Spectrum Disorder?

Cariprazine, a drug approved for schizophrenia and bipolar disorder, showed numerical improvement in behavior assessments in a study with children with autism, suggesting potential benefits for autism symptoms. ^{1 2 3 4 5}

Is Cariprazine safe for use in humans?

Cariprazine has been studied for safety in both adults and children, showing that it is generally well tolerated. Common mild or moderate side effects include weight gain, increased appetite, dizziness, and nasal congestion, with some reports of movement-related symptoms that resolved without stopping the treatment. ^{2 3 4 5 6}

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

Cariprazine is unique because it is a dopamine D3-preferring D3/D2 receptor partial agonist, which means it works by balancing certain brain chemicals differently than other treatments. It is being explored for Autism Spectrum Disorder (ASD) as there are currently no approved medications specifically for the core social and behavioral deficits in ASD. ^{1 2 3 4 5}

What is the purpose of this trial?

This trial is testing a new drug called cariprazine to see if it can help reduce irritability in children and teenagers with autism. The study involves taking the drug daily for a period of time and includes regular check-ups to monitor safety and effectiveness. The goal is to find out if cariprazine can improve behavior in young people with autism. Cariprazine has shown potential in alleviating core behavioral deficits and hyperactivity in animal models of autism spectrum disorder.

Research Team

ABBVIE INC.

Principal Investigator

AbbVie

Eligibility Criteria

This trial is for children aged 5-17 with Autism Spectrum Disorder (ASD) who show significant irritability. They must have specific scores on clinical assessments that measure severity of irritability and confirm an ASD diagnosis. Participants will be randomly assigned to receive either the drug Cariprazine or a placebo.

Inclusion Criteria

You have a score of 4 or more on the CGI-S Irritability subscale at screening.

Participants at the time of screening must have a Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition (DSM-5) diagnosis of ASD, confirmed by Kiddie Schedule for Affective Disorders and Schizophrenia-Present and Lifetime (K-SADS-PL) administered at screening (Visit 1) by a trained clinician.

Participants must have an Aberrant Behavior Checklist, 2nd edition - Community Version - Irritability (ABC-I) subscale score ≥ 18 (Visits 1 and 2).

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive oral capsules or oral solution of cariprazine or placebo once daily for 8 weeks

8 weeks

Regular visits at a hospital or clinic

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Cariprazine
• Placebo

Trial Overview The study tests Cariprazine's safety and effectiveness in reducing irritability due to ASD compared to a placebo, over an 8-week treatment period followed by a 12-week safety follow-up. This double-blind trial means neither participants nor doctors know who receives the actual drug or placebo.

Participant Groups

Group I: Cariprazine Experimental Treatment 1 Intervention

Participants will receive age-and weight dependent flexible doses of cariprazine once daily for 8-weeks.

Group II: Placebo Placebo Group 1 Intervention

Participants will receive placebo once daily for 8-weeks.

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

Approved in United States as Vraylar for:

• Schizophrenia
• Acute manic or mixed episodes associated with bipolar I disorder
• Depressive episodes associated with bipolar I disorder (bipolar depression)
• Adjunctive treatment with an antidepressant therapy (ADT) for major depressive disorder (MDD)

Who Is Running the Clinical Trial?

AbbVie

Lead Sponsor

Trials

1,079

Recruited

535,000+

Founded

2013

Headquarters

North Chicago, USA

Known For

Immunology treatments

Top Products

Humira (adalimumab), Skyrizi (risankizumab), Rinvoq (upadacitinib)

Dr. Roopal Thakkar

AbbVie

Chief Medical Officer since 2023

MD from Wayne State University School of Medicine

Robert A. Michael

AbbVie

Chief Executive Officer

Bachelor's degree in Finance from the University of Illinois

Findings from Research

In clinical trials, cariprazine significantly improved schizophrenia symptoms compared to placebo, as measured by the Positive and Negative Syndrome Scale (PANSS), and showed a longer time to relapse in a long-term study.

Cariprazine was more effective than risperidone in treating negative symptoms of schizophrenia, which are often challenging to address, and it was generally well tolerated with mostly mild to moderate side effects.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Cariprazine: A Review in Schizophrenia. Garnock-Jones, KP.[2018]

Cariprazine (Vraylar) is an atypical antipsychotic that acts as a partial agonist at dopamine D3 and D2 receptors, with a preference for D3, and also affects serotonin 5-HT1A receptors, which contributes to its therapeutic effects.

Approved in the USA in September 2015, cariprazine is used to treat schizophrenia and acute manic or mixed episodes in bipolar I disorder, and is being explored for additional uses in treating negative symptoms of schizophrenia and bipolar depression.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Cariprazine: First Global Approval. McCormack, PL.[2018]

Cariprazine is effective for treating schizophrenia and bipolar I disorder, showing a favorable side effect profile due to its selective affinity for D3 receptors, which helps address a range of symptoms including negative and cognitive symptoms.

It has a longer half-life and a delayed relapse time compared to other second-generation antipsychotics, making it a potentially better option for patients with high rates of non-adherence and frequent relapses.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Treatment of Symptom Clusters in Schizophrenia, Bipolar Disorder and Major Depressive Disorder With the Dopamine D3/D2 Preferring Partial Agonist Cariprazine. Batinic, B., Ristic, I., Zugic, M., et al.[2021]

Cariprazine demonstrated dose-dependent efficacy in reversing core behavioral deficits associated with autism spectrum disorder (ASD) in a rat model, showing effectiveness in social play and hyperactivity.

Compared to risperidone and aripiprazole, cariprazine was uniquely effective in the social play test, suggesting it may offer a distinct therapeutic benefit for treating social communication deficits in ASD.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Cariprazine alleviates core behavioral deficits in the prenatal valproic acid exposure model of autism spectrum disorder. Román, V., Adham, N., Foley, AG., et al.[2022]

References

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

Cariprazine: A Review in Schizophrenia. [2018]

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

Cariprazine: First Global Approval. [2018]

3. Switzerland pubmed.ncbi.nlm.nih.gov

Treatment of Symptom Clusters in Schizophrenia, Bipolar Disorder and Major Depressive Disorder With the Dopamine D3/D2 Preferring Partial Agonist Cariprazine. [2021]

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

Cariprazine in Pediatric Patients with Autism Spectrum Disorder: Results of a Pharmacokinetic, Safety and Tolerability Study. [2023]

5. Germany pubmed.ncbi.nlm.nih.gov

Cariprazine alleviates core behavioral deficits in the prenatal valproic acid exposure model of autism spectrum disorder. [2022]

6. United Arab Emirates pubmed.ncbi.nlm.nih.gov

Cariprazine in Bipolar Depression and Mania: State of the Art. [2019]