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

April 3, 2024 updated by: Bristol-Myers Squibb

A Randomized, Double-blind, Placebo-controlled Clinical Study to Evaluate Mavacamten in Adolescents (Age 12 Years to < 18 Years) With Symptomatic Obstructive Hypertrophic Cardiomyopathy

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

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

40

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

  • Name: BMS Study Connect Contact Center www.BMSStudyConnect.com
  • Phone Number: 855-907-3286
  • Email: Clinical.Trials@bms.com

Study Contact Backup

  • Name: First line of the email MUST contain the NCT# and Site #.

Study Locations

  • Australia
    • New South Wales
      • Sydney, New South Wales, Australia, 2145
        • Not yet recruiting
        • Local Institution - 0020
        • Contact:
          • Site 0020
    • Victoria
      • Clayton, Victoria, Australia, 3168
        • Not yet recruiting
        • Local Institution - 0042
        • Contact:
          • Site 0042
      • Parkville, Victoria, Australia, 3052
        • Withdrawn
        • Local Institution - 0028
  • Canada
    • Alberta
      • Edmonton, Alberta, Canada, T6G 2R7
        • Not yet recruiting
        • Local Institution - 0041
        • Contact:
          • Site 0041
    • Ontario
      • Toronto, Ontario, Canada, M5G 1X8
        • Not yet recruiting
        • Local Institution - 0046
        • Contact:
          • Site 0046
  • France
      • Paris, France, 75015
        • Not yet recruiting
        • Local Institution - 0022
        • Contact:
          • Site 0022
      • Pessac, France, 33604
        • Not yet recruiting
        • Local Institution - 0026
        • Contact:
          • Site 0026
  • Germany
    • BE
      • Berlin, BE, Germany, 13353
        • Not yet recruiting
        • Local Institution - 0018
        • Contact:
          • Site 0018
    • BY
      • Munich, BY, Germany, 80636
        • Not yet recruiting
        • Local Institution - 0006
        • Contact:
          • Site 0006
  • Ireland
    • D
      • Drimnagh, D, Ireland, D12 V443
        • Not yet recruiting
        • Local Institution - 0047
        • Contact:
          • Site 0047
  • Italy
      • Napoli, Italy, 80131
        • Not yet recruiting
        • Local Institution - 0027
        • Contact:
          • Site 0027
    • FI
      • Firenze, FI, Italy, 50100
        • Not yet recruiting
        • Local Institution - 0016
        • Contact:
          • Site 0016
    • GE
      • Genova, GE, Italy, 16147
        • Not yet recruiting
        • Local Institution - 0051
        • Contact:
          • Site 0051
  • Spain
      • A Coruna, Spain, 15009
        • Active, not recruiting
        • Local Institution - 0011
      • Madrid, Spain, 28041
        • Active, not recruiting
        • Local Institution - 0023
      • Madrid, Spain, 28046
        • Not yet recruiting
        • Local Institution - 0025
        • Contact:
          • Site 0025
    • B
      • Barcelona, B, Spain, 8950
        • Not yet recruiting
        • Local Institution - 0014
        • Contact:
          • Site 0014
    • GR
      • Granada, GR, Spain, 18014
        • Active, not recruiting
        • Local Institution - 0021
    • M
      • Madrid, M, Spain, 28007
        • Not yet recruiting
        • Local Institution - 0048
        • Contact:
          • Site 0048
  • United Kingdom
      • London, United Kingdom, WC1N 3JH
        • Not yet recruiting
        • Local Institution - 0007
        • Contact:
          • Site 0007
    • BFS
      • Belfast, BFS, United Kingdom, BT12 6BA
        • Not yet recruiting
        • Local Institution - 0049
        • Contact:
          • Site 0049
  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35294
        • Not yet recruiting
        • Local Institution - 0017
        • Contact:
          • Site 0017
    • Arizona
      • Phoenix, Arizona, United States, 85016-7710
        • Not yet recruiting
        • Local Institution - 0050
        • Contact:
          • Site 0050
    • California
      • Los Angeles, California, United States, 90027-6062
        • Not yet recruiting
        • Local Institution - 0032
        • Contact:
          • Site 0032
      • Los Angeles, California, United States, 90095-8344
        • Not yet recruiting
        • Local Institution - 0008
        • Contact:
          • Site 0008
      • Oakland, California, United States, 94609
        • Not yet recruiting
        • Local Institution - 0004
        • Contact:
          • Site 0004
      • Palo Alto, California, United States, 94304-1601
        • Not yet recruiting
        • Local Institution - 0033
        • Contact:
          • Site 0033
      • San Diego, California, United States, 92123-4223
        • Not yet recruiting
        • Local Institution - 0044
        • Contact:
          • Site 0044
    • Colorado
      • Aurora, Colorado, United States, 80045-7106
        • Not yet recruiting
        • Local Institution - 0038
        • Contact:
          • Site 0038
    • Florida
      • Saint Petersburg, Florida, United States, 33701-4634
        • Not yet recruiting
        • Local Institution - 0031
        • Contact:
          • Site 0031
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Not yet recruiting
        • Local Institution - 0053
        • Contact:
          • Site 0053
    • Illinois
      • Chicago, Illinois, United States, 60637-1447
        • Not yet recruiting
        • Local Institution - 0013
        • Contact:
          • Site 0013
    • Indiana
      • Indianapolis, Indiana, United States, 46202-5272
        • Recruiting
        • Riley Hospital for Children - Pediatric Cardiology
        • Contact:
          • John Parent, Site 0009
          • Phone Number: 317-908-4944
    • Massachusetts
      • Boston, Massachusetts, United States, 02115-5724
        • Not yet recruiting
        • Local Institution - 0043
        • Contact:
          • Site 0043
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • Recruiting
        • University of Michigan - C.S. Mott Children's Hospital
        • Contact:
          • Mark Russell, Site 0002
          • Phone Number: 734-660-3431
    • Missouri
      • Saint Louis, Missouri, United States, 63110
        • Not yet recruiting
        • Local Institution - 0037
        • Contact:
          • Site 0037
    • New Jersey
      • Morristown, New Jersey, United States, 07960-6083
        • Not yet recruiting
        • Local Institution - 0052
        • Contact:
          • Site 0052
    • New York
      • Bronx, New York, United States, 10467-2403
        • Not yet recruiting
        • Local Institution - 0040
        • Contact:
          • Site 0040
      • New Hyde Park, New York, United States, 11042
        • Recruiting
        • Cohen Children's Medical Center of New York
        • Contact:
          • Jeffrey Gossett, Site 0010
          • Phone Number: 516-601-7291
      • New York, New York, United States, 10029-6574
        • Not yet recruiting
        • Local Institution - 0024
        • Contact:
          • Site 0024
      • New York, New York, United States, 10032-1559
        • Not yet recruiting
        • Local Institution - 0036
        • Contact:
          • Site 0036
    • North Carolina
      • Charlotte, North Carolina, United States, 28204
        • Not yet recruiting
        • Local Institution - 0015
        • Contact:
          • Site 0015
      • Durham, North Carolina, United States, 27705
        • Not yet recruiting
        • Local Institution - 0039
        • Contact:
          • Site 0039
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Not yet recruiting
        • Local Institution - 0019
        • Contact:
          • Site 0019
      • Cleveland, Ohio, United States, 44195-0001
        • Not yet recruiting
        • Local Institution - 0001
        • Contact:
          • Site 0001
      • Columbus, Ohio, United States, 43205
        • Not yet recruiting
        • Local Institution - 0029
        • Contact:
          • Site 0029
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104-4319
        • Not yet recruiting
        • Local Institution - 0030
        • Contact:
          • Site 0030
      • Pittsburgh, Pennsylvania, United States, 15224-1334
        • Not yet recruiting
        • Local Institution - 0005
        • Contact:
          • Site 0005
    • Tennessee
      • Memphis, Tennessee, United States, 38104
        • Not yet recruiting
        • Local Institution - 0034
        • Contact:
          • Site 0034
    • Texas
      • Austin, Texas, United States, 78712
        • Not yet recruiting
        • Local Institution - 0045
        • Contact:
          • Site 0045
      • Houston, Texas, United States, 77030
        • Not yet recruiting
        • Local Institution - 0054
        • Contact:
          • Site 0054
    • Utah
      • Salt Lake City, Utah, United States, 84113-1103
        • Not yet recruiting
        • Local Institution - 0003
        • Contact:
          • Site 0003
    • Virginia
      • Charlottesville, Virginia, United States, 22908-0816
        • Not yet recruiting
        • Local Institution - 0012
        • Contact:
          • Site 0012
    • Wisconsin
      • Madison, Wisconsin, United States, 53792-0002
        • Not yet recruiting
        • Local Institution - 0035
        • Contact:
          • Site 0035

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Mavacamten
Participants assigned to this arm will receive mavacamten from day 1 to end of treatment at week 56.
Drug: Mavacamten
Specified dose on specified days
Other Names:
  • BMS-986427
Experimental: Placebo
Participants assigned to this arm will receive mavacamten from week 28 to end of treatment at week 56.
Drug: Placebo
Specified dose on specified days

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Change from baseline in Valsalva left ventricular outflow tract (LVOT) (VLVOT) gradient
Time Frame: At Week 28
At Week 28

Secondary Outcome Measures

Outcome Measure
Time Frame
Change from baseline in resting LVOT gradient
Time Frame: At Week 28
At Week 28
Change from baseline in post-exercise peak LVOT gradient
Time Frame: At Week 28
At Week 28
Change from baseline in maximal wall thickness
Time Frame: At Week 28
At Week 28
Change from baseline in ratio between early mitral inflow velocity and mitral annular early diastolic velocity (E/e')
Time Frame: At Week 28
At Week 28
Proportion of participants achieving an increase from baseline to Week 28 in peak oxygen uptake test (pVO2)
Time Frame: From baseline up to Week 28
From baseline up to Week 28
Proportion of participants achieving a reduction from baseline to Week 28 in maximal LVOT gradient to < 30 mmHg
Time Frame: From baseline up to Week 28
From baseline up to Week 28
Proportion of participants with at least 1 class improvement in New York Heart Association (NYHA) class from baseline to Week 28
Time Frame: From baseline up to Week 28
From baseline up to Week 28
Proportion of participants with at least 1 grade improvement in mitral regurgitation at Week 28
Time Frame: From baseline up to Week 28
From baseline up to Week 28
Number of participants with treatment-emergent adverse events (TEAEs)
Time Frame: Up to Week 74
Up to Week 74
Number of participants with treatment-emergent serious adverse events (TESAEs)
Time Frame: Up to Week 74
Up to Week 74
Change from baseline in electrocardiogram (ECG) (QT interval)
Time Frame: At Week 28
At Week 28
Number of participants with left ventricular ejection fraction (LVEF) ≤ 30%
Time Frame: Up to Week 56
Up to Week 56
Number of participants with LVEF ≤ 50%
Time Frame: Up to Week 56
Up to Week 56
Trough observed plasma concentration (Ctrough)
Time Frame: Up to Week 56
Up to Week 56
Post-dose plasma concentration of mavacamten
Time Frame: Up to Week 56
Up to Week 56
Maximum observed concentration (Cmax)
Time Frame: Up to Week 56
Up to Week 56
Area under the concentration-time curve (AUC)
Time Frame: Up to Week 56
Up to Week 56
Proportion of participants who evaluate taste and swallowability as neutral or better using taste and swallowability scales
Time Frame: At Day 1 and Week 11
At Day 1 and Week 11
Change from baseline in the Hypertrophic Cardiomyopathy Symptom Questionnaire - Shortness of Breath (HCMSQ SoB) domain
Time Frame: At Week 28
At Week 28

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Bristol-Myers Squibb

Investigators

  • Study Director: Bristol-Myers Squibb, Bristol-Myers Squibb

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Estimated)

March 29, 2024

Primary Completion (Estimated)

February 15, 2028

Study Completion (Estimated)

March 29, 2030

Study Registration Dates

First Submitted

February 2, 2024

First Submitted That Met QC Criteria

February 2, 2024

First Posted (Actual)

February 12, 2024

Study Record Updates

Last Update Posted (Actual)

April 4, 2024

Last Update Submitted That Met QC Criteria

April 3, 2024

Last Verified

April 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CV027-010
  • 2023-505650-17-00 (Other Identifier: EU CTR)
  • U1111-1290-1175 (Other Identifier: WHO)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

BMS will provide access to individual anonymized participant data upon request from qualified researchers, and subject to certain criteria. Additional information regarding Bristol Myer Squibb's data sharing policy and process can be found at: https://www.bms.com/researchers-and-partners/clinical-trials-and-research/disclosure-commitment.html

IPD Sharing Time Frame

See plan description

IPD Sharing Access Criteria

See plan description

IPD Sharing Supporting Information Type

  • STUDY_PROTOCOL
  • SAP
  • CSR

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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