Clinical trial NCT06253221: A Study to Evaluate Mavacamten in Adolescents With Symptomatic Obstructive Hypertrophic Cardiomyopathy
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
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Cardiomyopathy, Hypertrophic
Keywords: HCM
mavacamten
Obstructive Hypertrophic Cardiomyopathy
SCOUT
SCOUT-HCM
Pediatric HCM
Pediatric hypertrophic cardiomyopathy
Symptomatic HCM
oHCM
Lead sponsor: Bristol-Myers Squibb
Sponsor class: INDUSTRY
First posted: 2024-02-12
Enrollment: 40
Sex: ALL
Minimum age (years): 12
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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).


Eligibility criteria: 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.

Primary outcomes:
measure: Change from baseline in Valsalva left ventricular outflow tract (LVOT) (VLVOT) gradient
timeFrame: At Week 28
Secondary outcomes:
measure: Change from baseline in resting LVOT gradient
timeFrame: At Week 28
measure: Change from baseline in post-exercise peak LVOT gradient
timeFrame: At Week 28
measure: Change from baseline in maximal wall thickness
timeFrame: At Week 28
measure: Change from baseline in ratio between early mitral inflow velocity and mitral annular early diastolic velocity (E/e')
timeFrame: At Week 28
measure: Proportion of participants achieving an increase from baseline to Week 28 in peak oxygen uptake test (pVO2)
timeFrame: From baseline up to Week 28
measure: Proportion of participants achieving a reduction from baseline to Week 28 in maximal LVOT gradient to < 30 mmHg
timeFrame: From baseline up to Week 28
measure: Proportion of participants with at least 1 class improvement in New York Heart Association (NYHA) class from baseline to Week 28
timeFrame: From baseline up to Week 28
measure: Proportion of participants with at least 1 grade improvement in mitral regurgitation at Week 28
timeFrame: From baseline up to Week 28
measure: Number of participants with treatment-emergent adverse events (TEAEs)
timeFrame: Up to Week 218
measure: Number of participants with treatment-emergent serious adverse events (TESAEs)
timeFrame: Up to Week 218
measure: Change from baseline in electrocardiogram (ECG) (QT interval)
timeFrame: At Week 28
measure: Number of participants with left ventricular ejection fraction (LVEF) ≤ 30%
timeFrame: Up to Week 200
measure: Number of participants with LVEF < 50%
timeFrame: Up to Week 200
measure: Trough observed plasma concentration (Ctrough)
timeFrame: Up to Week 200
measure: Post-dose plasma concentration of mavacamten
timeFrame: Up to Week 200
measure: Maximum observed concentration (Cmax)
timeFrame: Up to Week 200
measure: Area under the concentration-time curve (AUC)
timeFrame: Up to Week 200
measure: Proportion of participants who evaluate taste and swallowability as neutral or better using taste and swallowability scales
timeFrame: At Day 1 and Week 11
measure: Change from baseline in the Hypertrophic Cardiomyopathy Symptom Questionnaire - Shortness of Breath (HCMSQ SoB) domain
timeFrame: At Week 28
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: Local Institution - 0017
state: Alabama
city: Phoenix
country: United States
facility: Local Institution - 0050
state: Arizona
city: Los Angeles
country: United States
facility: Local Institution - 0032
state: California
city: Los Angeles
country: United States
facility: Local Institution - 0008
state: California
city: Palo Alto
country: United States
facility: Local Institution - 0033
state: California
city: San Diego
country: United States
facility: Local Institution - 0044
state: California
city: Aurora
country: United States
facility: Local Institution - 0038
state: Colorado
city: Saint Petersburg
country: United States
facility: Local Institution - 0031
state: Florida
city: Atlanta
country: United States
facility: Local Institution - 0053
state: Georgia
city: Chicago
country: United States
facility: Local Institution - 0013
state: Illinois
city: Indianapolis
country: United States
facility: Local Institution - 0009
state: Indiana
city: Boston
country: United States
facility: Local Institution - 0043
state: Massachusetts
city: Ann Arbor
country: United States
facility: Local Institution - 0002
state: Michigan
city: Saint Louis
country: United States
facility: Local Institution - 0037
state: Missouri
city: Morristown
country: United States
facility: Local Institution - 0052
state: New Jersey
city: Bronx
country: United States
facility: Local Institution - 0040
state: New York
city: New Hyde Park
country: United States
facility: Local Institution - 0010
state: New York
city: New York
country: United States
facility: Local Institution - 0024
state: New York
city: New York
country: United States
facility: Local Institution - 0036
state: New York
city: Charlotte
country: United States
facility: Local Institution - 0015
state: North Carolina
city: Durham
country: United States
facility: Local Institution - 0039
state: North Carolina
city: Cincinnati
country: United States
facility: Local Institution - 0019
state: Ohio
city: Cleveland
country: United States
facility: Local Institution - 0001
state: Ohio
city: Columbus
country: United States
facility: Local Institution - 0029
state: Ohio
city: Philadelphia
country: United States
facility: Local Institution - 0030
state: Pennsylvania
city: Pittsburgh
country: United States
facility: Local Institution - 0005
state: Pennsylvania
city: Memphis
country: United States
facility: Local Institution - 0034
state: Tennessee
city: Austin
country: United States
facility: Local Institution - 0045
state: Texas
city: Houston
country: United States
facility: Local Institution - 0054
state: Texas
city: Salt Lake City
country: United States
facility: Local Institution - 0003
state: Utah
city: Charlottesville
country: United States
facility: Local Institution - 0012
state: Virginia
city: Sydney
country: Australia
facility: Local Institution - 0020
state: New South Wales
city: Clayton
country: Australia
facility: Local Institution - 0042
state: Victoria
city: Edmonton
country: Canada
facility: Local Institution - 0041
state: Alberta
city: Toronto
country: Canada
facility: Local Institution - 0046
state: Ontario
city: Paris
country: France
facility: Local Institution - 0022
city: Pessac
country: France
facility: Local Institution - 0026
city: Berlin
country: Germany
facility: Local Institution - 0018
state: BE
city: Munich
country: Germany
facility: Local Institution - 0006
state: BY
city: Dublin 12, D12 N512
country: Ireland
facility: Local Institution - 0047
city: Firenze
country: Italy
facility: Local Institution - 0016
state: FI
city: Genova
country: Italy
facility: Local Institution - 0051
state: GE
city: Napoli
country: Italy
facility: Local Institution - 0027
city: Barcelona
country: Spain
facility: Local Institution - 0014
state: B
city: Madrid
country: Spain
facility: Local Institution - 0025
city: Belfast
country: United Kingdom
facility: Local Institution - 0049
state: BFS
city: London
country: United Kingdom
facility: Local Institution - 0007
Study officials:
affiliation: Bristol-Myers Squibb
name: Bristol-Myers Squibb
role: STUDY_DIRECTOR
References: