KarXT + KarX-EC for Autism Spectrum Disorder

Not yet recruiting at 52 trial locations

Overseen By BMS Study Connect www.BMSStudyConnect.com

Age: < 18

Sex: Any

Trial Phase: Phase 3

Sponsor: Bristol-Myers Squibb

Disqualifiers: Bipolar, Schizophrenia, PTSD, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

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

Compensation: Varies

Number of Visits: Unknown

Duration: 8 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to test a new treatment, KarXT + KarX-EC (a combination therapy), for managing irritability in children and adolescents with autism. The study will compare the effects of this treatment to a placebo, which is a harmless pill resembling the real treatment but containing no active ingredients. Children and teens with autism who frequently experience irritability may be suitable candidates for this trial. Participants should not have conditions such as bipolar disorder or schizophrenia, and their irritability must be confirmed by a specific diagnostic tool. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants the opportunity to access a potentially groundbreaking treatment.

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. It's best to discuss this with the trial team or your doctor.

Is there any evidence suggesting that KarXT + KarX-EC is likely to be safe for humans?

Research shows that KarXT + KarX-EC is being tested to help with irritability in children and teens with autism. In earlier studies, this treatment appeared safe. Most participants experienced mild to moderate side effects, such as dry mouth and nausea, which were generally manageable and did not require stopping the treatment.

Since this is a Phase 3 study, KarXT + KarX-EC has already passed earlier safety tests. These earlier phases primarily focus on safety, so by the time a treatment reaches this stage, it is usually considered quite safe. However, like any medication, some risks remain, so discussing any concerns with a doctor is important. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for autism?

Most treatments for Autism Spectrum Disorder (ASD) focus on managing symptoms with behavioral therapies or medications like antipsychotics and stimulants, which can come with significant side effects. Unlike these standard options, KarXT + KarX-EC is unique because it targets the muscarinic receptors in the brain, which may help balance neurotransmitters more effectively. This novel mechanism of action could potentially reduce core symptoms of ASD with fewer side effects. Researchers are excited about this treatment because it offers a targeted approach that could improve quality of life for those with ASD.

What evidence suggests that KarXT + KarX-EC might be an effective treatment for irritability in autism?

Research suggests that KarXT + KarX-EC, which participants in this trial may receive, might help reduce irritability in children and teenagers with autism. Earlier studies have shown promising results in managing symptoms related to autism. Although specific data on this combination's effectiveness for irritability in autism is limited, KarXT has been studied for other conditions like schizophrenia, showing positive effects on symptoms. KarXT combines two components that affect the brain's chemical systems, believed to influence mood and behavior. This combination aims to reduce irritability by balancing these brain chemicals, potentially improving behavior in children with autism. ^{1 2 3 4 6}

Who Is on the Research Team?

Bristol-Myers Squibb

Principal Investigator

Bristol-Myers Squibb

Are You a Good Fit for This Trial?

This trial is for children and adolescents with Autism Spectrum Disorder who experience irritability. Specific eligibility details are not provided, but typically participants must meet diagnostic criteria for ASD and have a certain level of irritability.

Inclusion Criteria

I have been diagnosed with ASD and often feel irritable.

Participants must have ABC-I ≥18 (C18 on the Irritability subscale of the ABC-I) and CGIS specific to irritability ≥4, at screening and baseline (Day 1)

Exclusion Criteria

Other protocol-defined Inclusion/Exclusion criteria may apply

Exception Include: Participants with comorbid ADHD, provided that attention deficit/hyperactivity disorder (ADHD) is not the primary disorder, the participant is adequately treated and based on the investigator judgment the disorder is clinically stable

I don't have any serious health issues that could risk my safety or affect the study's results.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive KarXT + KarX-EC or placebo for the treatment of irritability associated with autism

8 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• KarXT + KarX-EC

Trial Overview

The study is testing the effectiveness of KarXT alone or combined with KarX-EC compared to a placebo in reducing irritability in young individuals with Autism Spectrum Disorder.

How Is the Trial Designed?

Group I: Placebo Experimental Treatment 1 Intervention

Group II: KarXT + KarX-EC Arm Experimental Treatment 2 Interventions

Who Is Running the Clinical Trial?

Bristol-Myers Squibb

Lead Sponsor

Trials

2,731

Recruited

4,127,000+

Headquarters

New York City, USA

Known For

Oncology & Cardiovascular

Top Products

Eliquis, Opdivo, Revlimid, Orencia

Christopher Boerner

Bristol-Myers Squibb

Chief Executive Officer since 2023

PhD in Business Administration from the Haas School of Business, University of California, Berkeley; BA in Economics and History from Washington University in St. Louis

Deepak L. Bhatt

Bristol-Myers Squibb

Chief Medical Officer since 2024

MD from Yale University; MSc in Clinical Epidemiology from the University of Pennsylvania

Citations

1.

clinicaltrials.gov

clinicaltrials.gov/study/NCT07284745?aggFilters=status%3A

A Study of KarXT + KarX-EC for Treatment of Irritability in ...

The purpose of this study is to assess KarXT + KarX-EC for the treatment of irritability associated with autism in children and adolescents.

2.

clinicaltrials.gov

clinicaltrials.gov/study/NCT07285798

A Study of KarXT + KarX-EC for Treatment of Irritability in ...

The purpose of this study is to assess KarXT + KarX-EC for the treatment of irritability associated with autism in children and adolescents. Official Title. A ...

3.

isrctn.com

isrctn.com/pdf/26415952

A Study to evaluate novel KarX and KarT Prototypes versus ...

The new form of the test medicine is a potential treatment for autism which is a neuropsychiatric condition (mental disorder affecting the ...

4.

bmsstudyconnect.com

bmsstudyconnect.com/se/en/clinical-trials/NCT07285798.html

A Study of KarXT + KarX-EC for Treatment of Irritability in ...

The purpose of this study is to assess KarXT + KarX-EC for the treatment of irritability associated with autism in children and adolescents.

5.

magazine.ingentium.com

magazine.ingentium.com/2025/12/18/a-phase-3-multicenter-randomized-double-blind-placebo-controlled-study-to-assess-the-efficacy-safety-and-tolerability-of-karxt-karx-ec-in-children-and-adolescents-5-to-17-years-of-age-with-i-2/

A Phase 3, Multicenter, Randomized, Double-blind, Placebo ...

Publication date: Dec 09, 2025. The purpose of this study is to assess KarXT + KarX-EC for the treatment of irritability associated with ...

6.

isrctn.com

isrctn.com/ISRCTN26415952

A Study to evaluate novel KarX and KarT Prototypes versus ...

A Study to evaluate novel KarX and KarT Prototypes versus the KarXT and KarX-EC reference following single doses, and to explore the effect of ...