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New drug could help teens with rare heart disease

NCT ID NCT06253221

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

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.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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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