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New drug could help teens with rare heart disease

NCT ID NCT06253221

Disease control Sponsor: Bristol-Myers Squibb Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tests a drug called mavacamten in 44 teenagers aged 12 to 18 who have a condition where the heart muscle is too thick and blocks blood flow. The goal is to see if the drug can reduce the blockage and improve symptoms. Participants are randomly assigned to receive either mavacamten or a placebo, and neither they nor their doctors know which they get.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
mavacamten
What this could lead to
If successful, mavacamten could become a new treatment option for teenagers with this heart condition, potentially improving their symptoms and daily life.
What could go wrong
This is a small, early-phase trial with only 44 participants, so results may not apply to all teens. The drug may cause side effects like reduced heart pumping function.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

44 people

The number who actually took part.

Started

Apr 2024

Expected to finish

Mar 2031

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

12 to 17 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Diagnosis of HCM * Presence of LVOT obstruction * Presence of symptoms Exclusion Criteria: * Phenocopy diseases resulting in myocardial hypertrophy not related to sarcomere dysfunction * Evidence of LVEF \<50% in prior 6 months * Planned escalation in HCM therapy or upcoming intervention (eg, major cardiac surgery, HCM medication dose increase) Other protocol-defined Inclusion/Exclusion criteria apply.

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Conditions

The condition(s) this trial relates to.

hypertrophic cardiomyopathy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Cardiomyopathy, hypertrophic

Contacts and locations

Locations

  • Local Institution - 0001

    Cleveland, Ohio, 44195-0001, United States

  • Local Institution - 0002

    Ann Arbor, Michigan, 48109, United States

  • Local Institution - 0003

    Salt Lake City, Utah, 84113-1103, United States

  • Local Institution - 0005

    Pittsburgh, Pennsylvania, 15224-1334, United States

  • Local Institution - 0006

    Munich, Bavaria, 80636, Germany

  • Local Institution - 0007

    London, WC1N 3JH, United Kingdom

  • Local Institution - 0008

    Los Angeles, California, 90095-8344, United States

  • Local Institution - 0009

    Indianapolis, Indiana, 46202-5272, United States

  • Local Institution - 0010

    New Hyde Park, New York, 11042, United States

  • Local Institution - 0012

    Charlottesville, Virginia, 22908-0816, United States

  • Local Institution - 0013

    Chicago, Illinois, 60637-1447, United States

  • Local Institution - 0014

    Barcelona, B, 8950, Spain

  • Local Institution - 0015

    Charlotte, North Carolina, 28204, United States

  • Local Institution - 0016

    Florence, FI, 50100, Italy

  • Local Institution - 0017

    Birmingham, Alabama, 35294-0004, United States

  • Local Institution - 0018

    Berlin, State of Berlin, 13353, Germany

  • Local Institution - 0019

    Cincinnati, Ohio, 45229, United States

  • Local Institution - 0020

    Sydney, New South Wales, 2145, Australia

  • Local Institution - 0022

    Paris, 75015, France

  • Local Institution - 0024

    New York, New York, 10029-6574, United States

  • Local Institution - 0025

    Madrid, 28046, Spain

  • Local Institution - 0026

    Pessac, 33604, France

  • Local Institution - 0027

    Naples, 80131, Italy

  • Local Institution - 0029

    Columbus, Ohio, 43205, United States

  • Local Institution - 0030

    Philadelphia, Pennsylvania, 19104-4319, United States

  • Local Institution - 0031

    St. Petersburg, Florida, 33701-4634, United States

  • Local Institution - 0032

    Los Angeles, California, 90027-6062, United States

  • Local Institution - 0033

    Palo Alto, California, 94304-1601, United States

  • Local Institution - 0034

    Memphis, Tennessee, 38105, United States

  • Local Institution - 0036

    New York, New York, 10032-1559, United States

  • Local Institution - 0037

    St Louis, Missouri, 63110, United States

  • Local Institution - 0038

    Aurora, Colorado, 80045-7106, United States

  • Local Institution - 0039

    Durham, North Carolina, 27705, United States

  • Local Institution - 0040

    The Bronx, New York, 10467-2403, United States

  • Local Institution - 0041

    Edmonton, Alberta, T6G 2R7, Canada

  • Local Institution - 0042

    Clayton, Victoria, 3168, Australia

  • Local Institution - 0043

    Boston, Massachusetts, 02115-5724, United States

  • Local Institution - 0044

    San Diego, California, 92123-4223, United States

  • Local Institution - 0045

    Austin, Texas, 78712, United States

  • Local Institution - 0046

    Toronto, Ontario, M5G 1X8, Canada

  • Local Institution - 0047

    Dublin 12, D12 N512, D12 N512, Ireland

  • Local Institution - 0049

    Belfast, BFS, BT12 6BA, United Kingdom

  • Local Institution - 0050

    Phoenix, Arizona, 85016-7710, United States

  • Local Institution - 0051

    Genova, GE, 16147, Italy

  • Local Institution - 0052

    Morristown, New Jersey, 07960, United States

  • Local Institution - 0053

    Atlanta, Georgia, 30329-3117, United States

  • Local Institution - 0054

    Houston, Texas, 77030, United States

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