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

Cardiomyopathy, Hypertrophic Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06253221
• Other study ID #: CV027-010
• Secondary ID: 2023-505650-17-0
• Status: Recruiting
• Phase: Phase 3
• Start date: April 17, 2024
• Est. completion date: March 29, 2030

Study information

• Verified date: May 2024
• Source: Bristol-Myers Squibb
• Contact: BMS Study Connect Contact Center www.BMSStudyConnect.com
• Phone: 855-907-3286
• Email: Clinical.Trials[@]bms.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 40
• Est. completion date: March 29, 2030
• Est. primary completion date: February 15, 2028
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years to 17 Years

Eligibility

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.

Related Conditions & MeSH terms

• Cardiomyopathies
• Cardiomyopathy, Hypertrophic
• Hypertrophy

Intervention

Drug:
• Mavacamten
Specified dose on specified days
• Placebo
Specified dose on specified days

Locations

Country	Name	City	State
Australia	Local Institution - 0042	Clayton	Victoria
Australia	Local Institution - 0028	Parkville	Victoria
Australia	Local Institution - 0020	Sydney	New South Wales
Canada	Local Institution - 0041	Edmonton	Alberta
Canada	Local Institution - 0046	Toronto	Ontario
France	Local Institution - 0022	Paris
France	Local Institution - 0026	Pessac
Germany	Local Institution - 0018	Berlin	BE
Germany	Local Institution - 0006	Munich	BY
Ireland	Local Institution - 0047	Drimnagh	D
Italy	Local Institution - 0016	Firenze	FI
Italy	Local Institution - 0051	Genova	GE
Italy	Local Institution - 0027	Napoli
Spain	Local Institution - 0011	A Coruna
Spain	Local Institution - 0014	Barcelona	B
Spain	Local Institution - 0021	Granada	GR
Spain	Local Institution - 0023	Madrid
Spain	Local Institution - 0025	Madrid
Spain	Local Institution - 0048	Madrid	M
United Kingdom	Local Institution - 0049	Belfast	BFS
United Kingdom	Local Institution - 0007	London
United States	University of Michigan - C.S. Mott Children's Hospital	Ann Arbor	Michigan
United States	Local Institution - 0053	Atlanta	Georgia
United States	Local Institution - 0038	Aurora	Colorado
United States	Local Institution - 0045	Austin	Texas
United States	Local Institution - 0017	Birmingham	Alabama
United States	Local Institution - 0043	Boston	Massachusetts
United States	Local Institution - 0040	Bronx	New York
United States	Local Institution - 0015	Charlotte	North Carolina
United States	University Of Virginia School of Medicine	Charlottesville	Virginia
United States	Local Institution - 0013	Chicago	Illinois
United States	Local Institution - 0019	Cincinnati	Ohio
United States	Local Institution - 0001	Cleveland	Ohio
United States	Local Institution - 0029	Columbus	Ohio
United States	Local Institution - 0039	Durham	North Carolina
United States	Local Institution - 0054	Houston	Texas
United States	Riley Hospital for Children - Pediatric Cardiology	Indianapolis	Indiana
United States	Local Institution - 0008	Los Angeles	California
United States	Local Institution - 0032	Los Angeles	California
United States	Local Institution - 0035	Madison	Wisconsin
United States	Local Institution - 0034	Memphis	Tennessee
United States	Local Institution - 0052	Morristown	New Jersey
United States	Cohen Children's Medical Center of New York	New Hyde Park	New York
United States	Local Institution - 0024	New York	New York
United States	Local Institution - 0036	New York	New York
United States	Local Institution - 0004	Oakland	California
United States	Local Institution - 0033	Palo Alto	California
United States	Local Institution - 0030	Philadelphia	Pennsylvania
United States	Local Institution - 0050	Phoenix	Arizona
United States	UPMC Childrens Hospital of Pittsburgh	Pittsburgh	Pennsylvania
United States	Washington University School of Medicine in St. Louis - St. Louis Children's Hospital	Saint Louis	Missouri
United States	Local Institution - 0031	Saint Petersburg	Florida
United States	Local Institution - 0003	Salt Lake City	Utah
United States	Local Institution - 0044	San Diego	California

Sponsors (1)

Lead Sponsor	Collaborator
Bristol-Myers Squibb

Countries where clinical trial is conducted

United States,  Australia,  Canada,  France,  Germany,  Ireland,  Italy,  Spain,  United Kingdom, 

Outcome

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

External Links

•   BMS Clinical Trial Information
•   BMS Clinical Trial Patient Recruiting