A Study to Evaluate Mavacamten in Adolescents With Symptomatic Obstructive Hypertrophic Cardiomyopathy

NCT06253221 | Active Not Recruiting Phase 3 DMC FDA Drug

• 3 other identifiers: CV027-0102023-505650-17-00U1111-1290-1175
• Study Type: interventional
• Target: 44
• Locations: 9 countries
• Sites: 47

Brief Summary

The purpose of this study is to evaluate the efficacy, safety, and pharmacokinetics of mavacamten in adolescent patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM).

Conditions

Cardiomyopathy, Hypertrophic

Keywords

HCM; mavacamten; Obstructive Hypertrophic Cardiomyopathy; SCOUT; SCOUT-HCM; Pediatric HCM; Pediatric hypertrophic cardiomyopathy; Symptomatic HCM; oHCM

Outcome Measures

Primary Outcomes (1)

• Change from baseline in Valsalva left ventricular outflow tract (LVOT) (VLVOT) gradient

  At Week 28

Secondary Outcomes (19)

• Change from baseline in resting LVOT gradient

  At Week 28

• Change from baseline in post-exercise peak LVOT gradient

  At Week 28

• Change from baseline in maximal wall thickness

  At Week 28

• Change from baseline in ratio between early mitral inflow velocity and mitral annular early diastolic velocity (E/e')

  At Week 28

• Proportion of participants achieving an increase from baseline to Week 28 in peak oxygen uptake test (pVO2)

  From baseline up to Week 28

Study Arms (2)

Mavacamten

EXPERIMENTAL

Participants assigned to this arm will receive mavacamten (1 mg to 15 mg) from day 1 to end of treatment at week 200.

Drug: Mavacamten

Placebo

EXPERIMENTAL

Participants assigned to this arm will receive mavacamten (1 mg to 15 mg) from week 28 to end of treatment at week 200.

Drug: Placebo

Interventions

Mavacamten DRUG

Specified dose on specified days

Also known as: BMS-986427

Mavacamten

Placebo DRUG

Specified dose on specified days

Placebo

Eligibility Criteria

• Age: 12 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Diagnosis of HCM
• Presence of LVOT obstruction
• Presence of symptoms

You may not qualify if:

• 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)

Sponsors & Collaborators

• Bristol-Myers Squibb: lead

Study Sites (47)

Local Institution - 0017

Birmingham, Alabama, 35294-0004, United States

Location

Local Institution - 0050

Phoenix, Arizona, 85016-7710, United States

Location

Local Institution - 0032

Los Angeles, California, 90027-6062, United States

Location

Local Institution - 0008

Los Angeles, California, 90095-8344, United States

Location

Local Institution - 0033

Palo Alto, California, 94304-1601, United States

Location

Local Institution - 0044

San Diego, California, 92123-4223, United States

Location

Local Institution - 0038

Aurora, Colorado, 80045-7106, United States

Location

Local Institution - 0031

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

Location

Local Institution - 0053

Atlanta, Georgia, 30329-3117, United States

Location

Local Institution - 0013

Chicago, Illinois, 60637-1447, United States

Location

Local Institution - 0009

Indianapolis, Indiana, 46202-5272, United States

Location

Local Institution - 0043

Boston, Massachusetts, 02115-5724, United States

Location

Local Institution - 0002

Ann Arbor, Michigan, 48109, United States

Location

Local Institution - 0037

St Louis, Missouri, 63110, United States

Location

Local Institution - 0052

Morristown, New Jersey, 07960, United States

Location

Local Institution - 0010

New Hyde Park, New York, 11042, United States

Location

Local Institution - 0024

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

Location

Local Institution - 0036

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

Location

Local Institution - 0040

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

Location

Local Institution - 0015

Charlotte, North Carolina, 28204, United States

Location

Local Institution - 0039

Durham, North Carolina, 27705, United States

Location

Local Institution - 0019

Cincinnati, Ohio, 45229, United States

Location

Local Institution - 0001

Cleveland, Ohio, 44195-0001, United States

Location

Local Institution - 0029

Columbus, Ohio, 43205, United States

Location

Local Institution - 0030

Philadelphia, Pennsylvania, 19104-4319, United States

Location

Local Institution - 0005

Pittsburgh, Pennsylvania, 15224-1334, United States

Location

Local Institution - 0034

Memphis, Tennessee, 38105, United States

Location

Local Institution - 0045

Austin, Texas, 78712, United States

Location

Local Institution - 0054

Houston, Texas, 77030, United States

Location

Local Institution - 0003

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

Location

Local Institution - 0012

Charlottesville, Virginia, 22908-0816, United States

Location

Local Institution - 0020

Sydney, New South Wales, 2145, Australia

Location

Local Institution - 0042

Clayton, Victoria, 3168, Australia

Location

Local Institution - 0041

Edmonton, Alberta, T6G 2R7, Canada

Location

Local Institution - 0046

Toronto, Ontario, M5G 1X8, Canada

Location

Local Institution - 0022

Paris, 75015, France

Location

Local Institution - 0026

Pessac, 33604, France

Location

Local Institution - 0006

Munich, Bavaria, 80636, Germany

Location

Local Institution - 0018

Berlin, State of Berlin, 13353, Germany

Location

Local Institution - 0047

Dublin 12, D12 N512, D12 N512, Ireland

Location

Local Institution - 0016

Florence, FI, 50100, Italy

Location

Local Institution - 0051

Genova, GE, 16147, Italy

Location

Local Institution - 0027

Naples, 80131, Italy

Location

Local Institution - 0014

Barcelona, B, 8950, Spain

Location

Local Institution - 0025

Madrid, 28046, Spain

Location

Local Institution - 0049

Belfast, BFS, BT12 6BA, United Kingdom

Location

Local Institution - 0007

London, WC1N 3JH, United Kingdom

Location

Related Publications (2)

• Rossano JW, Canter C, Wolf CM, Papez A, Gambra M, Bryant RM, Alejos J, McCulloch M, Sarquella Brugada G, Bock MJ, Jeewa A, Pearce FB, Desai MY, Favatella N, Javidialsaadi A, Phung V, Rano T, Zhu L, Dyme JL, Mital S; SCOUT-HCM Investigators. Mavacamten in Adolescents with Obstructive Hypertrophic Cardiomyopathy. N Engl J Med. 2026 Mar 29. doi: 10.1056/NEJMoa2601103. Online ahead of print.

  PMID: 41910394 DERIVED

• Rossano J, Canter C, Wolf C, Favatella N, Lockman J, Puli S, Javidialsaadi A, Dyme J, Crevar C, Mital S. Mavacamten in symptomatic adolescent patients with obstructive hypertrophic cardiomyopathy: design of the phase 3 SCOUT-HCM trial. Am Heart J. 2026 Feb;292:107283. doi: 10.1016/j.ahj.2025.107283. Epub 2025 Sep 30.

  PMID: 41038603 DERIVED

Related Links

• BMS Clinical Trial Information
• BMS Clinical Trial Patient Recruiting

MeSH Terms

Conditions

Cardiomyopathy, Hypertrophic

Interventions

MYK-461

Condition Hierarchy (Ancestors)

Cardiomyopathies; Heart Diseases; Cardiovascular Diseases; Aortic Stenosis, Subvalvular; Aortic Valve Stenosis; Aortic Valve Disease; Heart Valve Diseases

Study Officials

• Bristol-Myers Squibb

  Bristol-Myers Squibb

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 2, 2024
• First Posted: February 12, 2024
• Study Start: April 17, 2024
• Primary Completion: November 25, 2025
• Study Completion (Estimated): March 28, 2031
• Last Updated: December 19, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, CSR
• Time Frame: See plan description
• Access Criteria: See plan description

Locations

CA (2); AU (2); IE (1); FR (2); GB (2); DE (2); ES (2); IT (3); US (31)