Brexpiprazole for Autism

(Anchor Trial)

OC
Overseen ByOPDC Call Center
Age: < 18
Sex: Any
Trial Phase: Phase 3
Sponsor: Otsuka Pharmaceutical Development & Commercialization, Inc.
Pivotal Trial (Near Approval)This treatment is in the last trial phase before FDA approval
Prior Safety DataThis treatment has passed at least one previous human trial
Approved in 6 JurisdictionsThis treatment is already approved in other countries

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores the potential benefits and safety of brexpiprazole, an antipsychotic, for children and teens with irritability linked to autism spectrum disorder (ASD). Researchers aim to determine if this treatment can reduce irritability in participants aged 5 to 17. The study compares brexpiprazole's effects to a placebo, which contains no active medication. Children and teens with an ASD diagnosis and noticeable irritability might be suitable for this study. Participants must be able to swallow tablets and follow trial procedures with a caregiver's assistance. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking treatment.

Will I have to stop taking my current medications?

Yes, you will need to stop taking certain medications before and during the trial, as the study requires a washout period (time without taking certain medications).

Is there any evidence suggesting that brexpiprazole is likely to be safe for children and adolescents with irritability associated with autism spectrum disorder?

Research has shown that brexpiprazole has been tested for safety in over 12,550 adults across various clinical trials for conditions like depression. These studies indicate it is generally well-tolerated. Common side effects include weight gain and sleepiness, while serious issues remain rare.

In long-term studies involving adults with schizophrenia, brexpiprazole was used safely for up to a year, with regular safety checks. Although this trial focuses on children and teens with autism, the existing data in adults offers some reassurance about its safety.

Brexpiprazole already has FDA approval for treating other conditions, indicating it has met certain safety standards. However, effects in children might differ, so ongoing trials are crucial to confirm safety for this group.12345

Why do researchers think this study treatment might be promising?

Brexpiprazole is unique because it offers a new approach to managing irritability associated with Autism Spectrum Disorder (ASD) in children and adolescents. Unlike standard treatments such as risperidone or aripiprazole, which can have side effects like weight gain and sedation, brexpiprazole is designed to be a more tolerable option. It works by balancing dopamine and serotonin in the brain, which may help reduce irritability with fewer side effects. Researchers are excited about brexpiprazole because it could provide a more effective and safer alternative for young people with ASD.

What evidence suggests that brexpiprazole might be an effective treatment for irritability associated with autism?

Research has shown that brexpiprazole, which participants in this trial may receive, can help reduce irritability in conditions similar to autism. One study found that participants experienced a noticeable decrease in irritability. Additionally, it proved effective in calming agitation in people with Alzheimer's. These results suggest that brexpiprazole might help manage irritability in children and teenagers with autism spectrum disorder.46789

Are You a Good Fit for This Trial?

Inclusion Criteria

Primary DSM-5 diagnosis of Autism Spectrum Disorder
ABC-I subscale score of ≥ 18
CGI-S scale score pertaining to irritability ≥ 4
See 4 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive flexible doses of brexpiprazole or placebo up to Week 8

8 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

  • Brexpiprazole
How Is the Trial Designed?
2Treatment groups
Experimental Treatment
Placebo Group
Group I: BrexpiprazoleExperimental Treatment1 Intervention
Group II: PlaceboPlacebo Group1 Intervention

Brexpiprazole is already approved in United States, Canada, European Union, Japan, Brazil for the following indications:

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Approved in United States as Rexulti for:
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Approved in Canada as Rexulti for:
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Approved in European Union as Rexulti for:
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Approved in Japan as Rexulti for:
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Approved in Brazil as Rexulti for:

Find a Clinic Near You

Who Is Running the Clinical Trial?

Otsuka Pharmaceutical Development & Commercialization, Inc.

Lead Sponsor

Trials
271
Recruited
170,000+
John Kraus profile image

John Kraus

Otsuka Pharmaceutical Development & Commercialization, Inc.

Chief Medical Officer since 2023

MD, PhD

Tarek Rabah profile image

Tarek Rabah

Otsuka Pharmaceutical Development & Commercialization, Inc.

Chief Executive Officer since 2022

BS in Biology and BA in Business from the American University of Beirut, MBA from McGill University

H. Lundbeck A/S

Industry Sponsor

Trials
332
Recruited
78,300+
Charl van Zyl profile image

Charl van Zyl

H. Lundbeck A/S

Chief Executive Officer since 2023

Degree in Medical Biochemistry from the University of Cape Town, South Africa

Johan Luthman profile image

Johan Luthman

H. Lundbeck A/S

Chief Medical Officer since 2019

MD from the University of Gothenburg, Sweden

Published Research Related to This Trial

In a 17-week pilot study involving 27 adults with binge eating disorder (BED), liraglutide 3.0 mg/day led to a significant weight loss of 5.2% compared to 0.9% in the placebo group, indicating its potential efficacy for weight management in BED.
While both liraglutide and placebo groups experienced reductions in binge eating episodes, the difference in the number of objective binge episodes per week was not statistically significant, suggesting that while liraglutide may aid in weight loss, its effect on binge eating frequency needs further investigation.
A pilot randomized controlled trial of liraglutide 3.0 mg for binge eating disorder.Allison, KC., Chao, AM., Bruzas, MB., et al.[2023]
In a small study of 10 individuals with bulimia who did not respond to traditional antidepressants, treatment with the long-acting opiate antagonist naltrexone led to a significant reduction in bulimic symptoms for 7 participants, with improvements lasting for 3 to 5 months.
These findings suggest that naltrexone could be a promising alternative treatment for bulimia, particularly for those resistant to standard therapies, and may help researchers understand the role of opioids in eating disorders.
Treatment of antidepressant-resistant bulimia with naltrexone.Jonas, JM., Gold, MS.[2019]

Citations

Efficacy and Safety of Brexpiprazole (OPC-34712) as ...Brexpiprazole was found to be an efficacious maintenance therapy over the course of 1 year, reducing the risk of impending relapse by 71% vs placebo, and with ...
Study Details | NCT01668797 | Efficacy, Safety, and ...The purpose of this study is to evaluate the efficacy of brexpiprazole compared with placebo as maintenance treatment in adults with schizophrenia. Detailed ...
Brexpiprazole for the Treatment of Agitation in Alzheimer ...Brexpiprazole, 2 or 3 mg, reduced agitation in Alzheimer dementia and was generally well tolerated over 12 weeks.
JAMA Neurology Publishes Complete Results of Positive ...In Study 213, brexpiprazole demonstrated significant efficacy as measured by a 22.6 point reduction in participants' mean CMAI total scores.
Real-World Evidence of Brexpiprazole Use and 6-Month ...Specifically, incident brexpiprazole users were about 4 percentage points less likely to die within 6 months of use, with no significant ...
6.rexultihcp.comrexultihcp.com/sz/safety
SZ | Safety Profile - REXULTI® (brexpiprazole)Safety data were collected at regular intervals throughout the 52 weeks. The overall N was 813; n values for individual parameters varied.6. bSD for mean ...
Reference ID: 5588438 - accessdata.fda.govBrexpiprazole has been evaluated for safety in 12,550 adult patients who participated in multiple-dose clinical trials for major depressive disorder, ...
8.rexulti.comrexulti.com/
REXULTI® (brexpiprazole) | Patient Information WebsiteMedicines like REXULTI can raise the risk of death in elderly people who have lost touch with reality (psychosis) due to confusion and memory loss (dementia).
RXULTI, INN-brexpiprazole - EMA5.3 Preclinical safety data. Effects observed in repeated-dose toxicity studies in rats and monkeys were mainly related to the exaggerated pharmacological ...
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