Brexpiprazole in Treatment of Children and Adolescents With Irritability Associated With Autism Spectrum Disorder

(Anchor Trial)

Overseen By OPDC Call Center

Age: < 18

Sex: Any

Trial Phase: Phase 3

Sponsor: Otsuka Pharmaceutical Development & Commercialization, Inc.

Disqualifiers: Bipolar, Schizophrenia, Epilepsy, Diabetes, 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 6 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 8 weeks

119 Participants Needed

Trial Summary

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).

How does the drug Brexpiprazole differ from other treatments for its condition?

Brexpiprazole is unique because it acts as a serotonin-dopamine activity modulator, which means it helps balance certain chemicals in the brain differently than other treatments. This mechanism can make it effective for conditions like depression and schizophrenia, where other treatments might not work as well. ^{1 2 3 4 5}

What is the purpose of this trial?

This trial is testing brexpiprazole, a medication, to see if it can help reduce irritability in children and teenagers aged 5 to 17 with autism. The medication works by balancing brain chemicals to improve mood and reduce irritability.

Eligibility Criteria

Inclusion Criteria

Primary DSM-5 diagnosis of Autism Spectrum Disorder

ABC-I subscale score of ≥ 18

CGI-S scale score pertaining to irritability ≥ 4

Timeline

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

Treatment Details

Interventions

• Brexpiprazole

Participant Groups

Group I: Brexpiprazole Experimental Treatment 1 Intervention

Participants received flexible doses of brexpiprazole 0.25 to 3 milligram per day (mg/day), orally, once daily (QD) up to Week 8. For participants with body weight \< 50 kilograms (kg) the dose was titrated up from 0.25 mg/day on Days 1 to 3, followed by 0.5 mg on Days 4 to 7, and to 1 mg on Days 8 to 14. Based on the investigator's judgment the dose was increased to 1.5 mg/day after Day 15. The dose was fixed after Week 6 and administration continued for another 2 weeks until Week 8. For participants with body weight ≥ 50 kg the dose was titrated up from 0.5 mg/day on Days 1 to 3, followed by 1.5 mg on Days 4 to 7, and to 2 mg on Days 8 to 14. Based on the investigator's judgment the dose was increased to 3 mg/day after Day 15. The dose was fixed after Week 6 and administration continued for another 2 weeks until Week 8.

Group II: Placebo Placebo Group 1 Intervention

Participants received brexpiprazole matching placebo orally, QD, in the same way as brexpiprazole up to Week 8.

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

Approved in United States as Rexulti for:

• Major depressive disorder (as an adjunctive therapy to antidepressants)
• Schizophrenia
• Agitation associated with dementia due to Alzheimer's disease

Approved in Canada as Rexulti for:

• Major depressive disorder (as an adjunctive therapy to antidepressants)
• Schizophrenia

Approved in European Union as Rexulti for:

• Schizophrenia

Approved in Japan as Rexulti for:

• Schizophrenia

Approved in Brazil as Rexulti for:

• Major depressive disorder (as an adjunctive therapy to antidepressants)

Who Is Running the Clinical Trial?

Otsuka Pharmaceutical Development & Commercialization, Inc.

Lead Sponsor

Trials

271

Recruited

170,000+

John Kraus

Otsuka Pharmaceutical Development & Commercialization, Inc.

Chief Medical Officer since 2023

MD, PhD

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

H. Lundbeck A/S

Chief Executive Officer since 2023

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

Johan Luthman

H. Lundbeck A/S

Chief Medical Officer since 2019

MD from the University of Gothenburg, Sweden

Findings from Research

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.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

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.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Treatment of antidepressant-resistant bulimia with naltrexone. Jonas, JM., Gold, MS.[2019]

References

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

A pilot randomized controlled trial of liraglutide 3.0 mg for binge eating disorder. [2023]

2. England pubmed.ncbi.nlm.nih.gov

Separation of emetic and anorexic responses of exendin-4, a GLP-1 receptor agonist in Suncus murinus (house musk shrew). [2022]

3. Netherlands pubmed.ncbi.nlm.nih.gov

GLP-1 receptors are involved in the GLP-1 (7-36) amide-induced modulation of glucose homoeostasis, emesis and feeding in Suncus murinus (house musk shrew). [2021]

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

Treatment of antidepressant-resistant bulimia with naltrexone. [2019]

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

Acamprosate in the treatment of binge eating disorder: a placebo-controlled trial. [2022]