Clinical trial NCT05582395: A Study of Mavacamten in Non-Obstructive Hypertrophic Cardiomyopathy
Official title: A Randomized, Double-blind, Placebo-controlled Clinical Study to Evaluate Mavacamten in Adults With Symptomatic Non-obstructive Hypertrophic Cardiomyopathy
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Cardiomyopathy, Hypertrophic
Keywords: Mavacamten
non-obstructive HCM
non-obstructive hypertrophic cardiomyopathy (nHCM)
Lead sponsor: Bristol-Myers Squibb
Sponsor class: INDUSTRY
First posted: 2022-10-17
Enrollment: 580
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to evaluate the safety, tolerability, and efficacy of mavacamten compared with placebo in participants with symptomatic non-obstructive hypertrophic cardiomyopathy (nHCM).


Eligibility criteria: Inclusion Criteria

* Diagnosis of HCM consistent with current American College of Cardiology Foundation/American Heart Association and European Society of Cardiology guidelines: unexplained left-ventricular hypertrophy with non-dilated ventricular chambers in the absence of other cardiac or systemic disease which can produce the required magnitude of hypertrophy of a maximal left ventricular (LV) wall thickness ≥ 15 millimeters (mm) (or ≥ 13 mm with positive family history of hypertrophic cardiomyopathy \[HCM\]) as determined by core laboratory interpretation.
* Peak left ventricular outflow tract (LVOT) pressure gradient \< 30 millimeters mercury (mm Hg) at rest and \< 50 mm Hg with provocation (Valsalva maneuver and stress echocardiography).
* New York Heart Association (NYHA) Class II or III.

Exclusion Criteria

* Known infiltrative or storage disorder causing cardiac hypertrophy that mimics non-obstructive hypertrophic cardiomyopathy (nHCM) such as Fabry disease, amyloidosis, or Noonan syndrome with LV hypertrophy.
* History of unexplained syncope within 6 months prior to screening.
* History of sustained ventricular tachyarrhythmia (\> 30 seconds) within 6 months prior to screening.
* Other protocol-defined Inclusion/Exclusion criteria apply.

Primary outcomes:
measure: Change from baseline in Kansas City Cardiomyopathy Questionnaire (23-item) Clinical Summary Score (KCCQ-23 CSS) at Week 48
timeFrame: Up to Week 48
measure: Change from baseline in peak oxygen consumption (pVO2) at Week 48
timeFrame: Up to Week 48
Secondary outcomes:
measure: Change from baseline in ventilatory efficiency (VE/VCO2) slope to Week 48
timeFrame: Up to Week 48
measure: Proportion of participants with at least 1 class of New York Heart Association (NYHA) improvement from baseline to Week 48
timeFrame: Up to Week 48
measure: Change from baseline in N-terminal pro B-type natriuretic peptide (NT-proBNP) to Week 48
timeFrame: Up to Week 48
measure: Change from baseline in hypertrophic cardiomyopathy symptom questionnaire-shortness of breath (HCMSQ-SoB) domain to Week 48
timeFrame: Up to Week 48
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: Local Institution - 0082
state: Alabama
city: Phoenix
country: United States
facility: Local Institution - 0458
state: Arizona
city: La Jolla
country: United States
facility: Local Institution - 0409
state: California
city: Los Angeles
country: United States
facility: Local Institution - 0010
state: California
city: San Francisco
country: United States
facility: Local Institution - 0406
state: California
city: Stanford
country: United States
facility: Local Institution - 0079
state: California
city: Gainesville
country: United States
facility: Local Institution - 0468
state: Florida
city: Tampa
country: United States
facility: Local Institution - 0281
state: Florida
city: Atlanta
country: United States
facility: Local Institution - 0092
state: Georgia
city: Indianapolis
country: United States
facility: Local Institution - 0424
state: Indiana
city: Iowa City
country: United States
facility: Local Institution - 0070
state: Iowa
city: Kansas City
country: United States
facility: Local Institution - 0410
state: Kansas
city: Louisville
country: United States
facility: Local Institution - 0484
state: Kentucky
city: Baltimore
country: United States
facility: Local Institution - 0127
state: Maryland
city: Boston
country: United States
facility: Local Institution - 0479
state: Massachusetts
city: Boston
country: United States
facility: Local Institution - 0500
state: Massachusetts
city: Ann Arbor
country: United States
facility: Local Institution - 0464
state: Michigan
city: Detroit
country: United States
facility: Local Institution - 0064
state: Michigan
city: Grand Rapids
country: United States
facility: Local Institution - 0455
state: Michigan
city: Rochester
country: United States
facility: Local Institution - 0431
state: Minnesota
city: Saint Louis
country: United States
facility: Local Institution - 0461
state: Missouri
city: Omaha
country: United States
facility: Local Institution - 0435
state: Nebraska
city: Morristown
country: United States
facility: Local Institution - 0490
state: New Jersey
city: Manhasset
country: United States
facility: Local Institution - 0449
state: New York
city: New York
country: United States
facility: Local Institution - 0486
state: New York
city: Stony Brook
country: United States
facility: Local Institution - 0007
state: New York
city: Valhalla
country: United States
facility: Local Institution - 0426
state: New York
city: Charlotte
country: United States
facility: Local Institution - 0094
state: North Carolina
city: Durham
country: United States
facility: Local Institution - 0065
state: North Carolina
city: Cleveland
country: United States
facility: Local Institution - 0083
state: Ohio
city: Hershey
country: United States
facility: Local Institution - 0047
state: Pennsylvania
city: Lancaster
country: United States
facility: Local Institution - 0444
state: Pennsylvania
city: Philadelphia
country: United States
facility: Local Institution - 0263
state: Pennsylvania
city: Pittsburgh
country: United States
facility: Local Institution - 0081
state: Pennsylvania
city: Germantown
country: United States
facility: Local Institution - 0207
state: Tennessee
city: Dallas
country: United States
facility: Local Institution - 0089
state: Texas
city: Dallas
country: United States
facility: Local Institution - 0365
state: Texas
city: Plano
country: United States
facility: Local Institution - 0492
state: Texas
city: Salt Lake City
country: United States
facility: Local Institution - 0402
state: Utah
city: Charlottesville
country: United States
facility: Local Institution - 0478
state: Virginia
city: Richmond
country: United States
facility: Local Institution - 0095
state: Virginia
city: Concord
country: Australia
facility: Local Institution - 0063
state: New South Wales
city: Darlinghurst
country: Australia
facility: Local Institution - 0085
state: New South Wales
city: Sydney
country: Australia
facility: Local Institution - 0369
state: New South Wales
city: Chermside
country: Australia
facility: Local Institution - 0080
state: Queensland
city: Milton
country: Australia
facility: Local Institution - 0078
state: Queensland
city: Bedford Park
country: Australia
facility: Local Institution - 0050
state: South Australia
city: Murdoch
country: Australia
facility: Local Institution - 0076
state: Western Australia
city: Linz
country: Austria
facility: Local Institution - 0075
state: Oberösterreich
city: Innsbruck
country: Austria
facility: Local Institution - 0090
state: Tirol
city: Braunau am Inn
country: Austria
facility: Local Institution - 0069
state: Upper Austria (Oberösterreich)
city: Graz
country: Austria
facility: Local Institution - 0062
city: Wien
country: Austria
facility: Local Institution - 0429
city: Edegem
country: Belgium
facility: Local Institution - 0462
state: Antwerpen
city: Anderlecht
country: Belgium
facility: Local Institution - 0034
state: Brussels
city: Genk
country: Belgium
facility: Local Institution - 0476
state: Limburg
city: Leuven
country: Belgium
facility: Local Institution - 0319
state: Vlaams Brabant
city: Salvador
country: Brazil
facility: Local Institution - 0413
state: Bahia
city: Brasília
country: Brazil
facility: Local Institution - 0438
state: Distrito Federal
city: Belo Horizonte
country: Brazil
facility: Local Institution - 0488
state: MG
city: Porto Alegre
country: Brazil
facility: Local Institution - 0447
state: RIO Grande DO SUL
city: Porto Alegre
country: Brazil
facility: Local Institution - 0450
state: RIO Grande DO SUL
city: Bragança Paulista
country: Brazil
facility: Local Institution - 0430
state: SAO Paulo
city: Ribeirão Preto
country: Brazil
facility: Local Institution - 0422
state: SAO Paulo
city: São Paulo
country: Brazil
facility: Local Institution - 0412
state: SAO Paulo
city: Rio De Janeiro
country: Brazil
facility: Local Institution - 0446
city: Rio de Janeiro
country: Brazil
facility: Local Institution - 0451
city: Sao Paulo
country: Brazil
facility: Local Institution - 0418
city: Calgary
country: Canada
facility: Local Institution - 0381
state: Alberta
city: Vancouver
country: Canada
facility: Local Institution - 0467
state: British Columbia
city: Hamilton
country: Canada
facility: Local Institution - 0414
state: Ontario
city: London
country: Canada
facility: Local Institution - 0411
state: Ontario
city: Quebec
country: Canada
facility: Local Institution - 0093
city: Beijing
country: China
facility: Local Institution - 0535
state: Beijing
city: Beijing
country: China
facility: Local Institution - 0534
state: Beijing
city: Chongqing
country: China
facility: Local Institution - 0503
state: Chongqing
city: Chongqing
country: China
facility: Local Institution - 0541
state: Chongqing
city: Guangzhou
country: China
facility: Local Institution - 0539
state: Guangdong
city: Guangzhou
country: China
facility: Local Institution - 0543
state: Guangdong
city: Zhengzhou
country: China
facility: Local Institution - 0549
state: Henan
city: Wuhan
country: China
facility: Local Institution - 0542
state: Hubei
city: Changsha
country: China
facility: Local Institution - 0548
state: Hunan
city: Suzhou
country: China
facility: Local Institution - 0545
state: Jiangsu
city: Nanchang
country: China
facility: Local Institution - 0550
state: Jiangxi
city: Changchun
country: China
facility: Local Institution - 0544
state: Jilin
city: Dalian
country: China
facility: Local Institution - 0540
state: Liaoning
city: Shenyang
country: China
facility: Local Institution - 0501
state: Liaoning
city: Shenyang
country: China
facility: Local Institution - 0538
state: Liaoning
city: Xian
country: China
facility: Local Institution - 0547
state: Shan1xi
city: Jinan
country: China
facility: Local Institution - 0546
state: Shandong
city: Shanghai
country: China
facility: Local Institution - 0537
state: Shanghai
city: Chengdu
country: China
facility: Local Institution - 0533
state: Sichuan
city: Chengdu
country: China
facility: Local Institution - 0552
state: Sichuan
city: Wenzhou
country: China
facility: Local Institution - 0536
state: Zhejiang
city: Beijing
country: China
facility: Local Institution - 0502
city: Beijing
country: China
facility: Local Institution - 0553
city: Broumov
country: Czechia
facility: Local Institution - 0086
state: Královéhradecký Kraj
city: Prague
country: Czechia
facility: Local Institution - 0033
state: Praha, Hlavní Mesto
city: Praha 2
country: Czechia
facility: Local Institution - 0052
state: Praha, Hlavní Mesto
city: Brno
country: Czechia
facility: Local Institution - 0188
city: Ålborg
country: Denmark
facility: Local Institution - 0045
state: Nordjylland
city: Aabenraa
country: Denmark
facility: Local Institution - 0428
state: Sønderjylland
city: Aarhus N
country: Denmark
facility: Local Institution - 0019
city: Nice
country: France
facility: Local Institution - 0001
state: Alpes-Maritimes
city: Brest
country: France
facility: Local Institution - 0463
state: Finistère
city: Toulouse
country: France
facility: Local Institution - 0005
state: Haute-Garonne
city: Chambray-lès-tours
country: France
facility: Local Institution - 0003
state: Indre-et-Loire
city: La Tronche
country: France
facility: Local Institution - 0489
state: Isère
city: Bron
country: France
facility: Local Institution - 0067
state: Rhône
city: Nantes Cedex 01
country: France
facility: Local Institution - 0009
city: Paris
country: France
facility: Local Institution - 0004
city: Paris
country: France
facility: Local Institution - 0006
city: Pessac
country: France
facility: Local Institution - 0053
city: Rennes
country: France
facility: Local Institution - 0008
city: Ulm
country: Germany
facility: Local Institution - 0404
state: Baden-Württemberg
city: München
country: Germany
facility: Local Institution - 0022
state: Bayern
city: Regensburg
country: Germany
facility: Local Institution - 0440
state: Bayern
city: Bad Nauheim
country: Germany
facility: Local Institution - 0013
state: Hessen
city: Göttingen
country: Germany
facility: Local Institution - 0496
state: Niedersachsen
city: Hannover
country: Germany
facility: Local Institution - 0448
state: Niedersachsen
city: Bad Oeynhausen
country: Germany
facility: Local Institution - 0439
state: Nordrhein-Westfalen
city: Bielefeld
country: Germany
facility: Local Institution - 0174
state: Nordrhein-Westfalen
city: Köln
country: Germany
facility: Local Institution - 0273
state: North Rhine Wesrphalia
city: Mainz
country: Germany
facility: Local Institution - 0420
state: Rheinland-Pfalz
city: Luebeck
country: Germany
facility: Local Institution - 0066
state: Schleswig-Holstein
city: Jena
country: Germany
facility: Local Institution - 0016
state: Thüringen
city: Berlin
country: Germany
facility: Local Institution - 0403
city: Freiburg
country: Germany
facility: Local Institution - 0071
city: Hamburg
country: Germany
facility: Local Institution - 0032
city: Heidelberg
country: Germany
facility: Local Institution - 0077
city: München
country: Germany
facility: Local Institution - 0454
city: Münster
country: Germany
facility: Local Institution - 0046
city: Balatonfüred
country: Hungary
facility: Local Institution - 0423
state: Veszprém
city: Budapest
country: Hungary
facility: Local Institution - 0048
city: Budapest
country: Hungary
facility: Local Institution - 0039
city: Budapest
country: Hungary
facility: Local Institution - 0035
city: Szeged
country: Hungary
facility: Local Institution - 0417
city: Ahmedabad
country: India
facility: Local Institution - 0482
state: Gujarat
city: Bengaluru
country: India
facility: Local Institution - 0480
state: Karnataka
city: Kochi
country: India
facility: Local Institution - 0481
state: Kerala
city: Chandigarh
country: India
facility: Local Institution - 0483
city: Gurgaon
country: India
facility: Local Institution - 0330
city: Ashkelon
country: Israel
facility: Local Institution - 0026
state: Tel-Aviv
city: Tel Aviv
country: Israel
facility: Local Institution - 0037
state: Tel-Aviv
city: Jerusalem
country: Israel
facility: Local Institution - 0025
state: Yerushalayim
city: Jerusalem
country: Israel
facility: Local Institution - 0057
state: Yerushalayim
city: Haifa
country: Israel
facility: Local Institution - 0419
city: Petah Tikva
country: Israel
facility: Local Institution - 0427
city: Ramat -Gan
country: Israel
facility: Local Institution - 0074
city: Rehovot
country: Israel
facility: Local Institution - 0054
city: Safed
country: Israel
facility: Local Institution - 0253
city: Firenze
country: Italy
facility: Local Institution - 0373
state: Toscana
city: Pisa
country: Italy
facility: Local Institution - 0171
city: Roma
country: Italy
facility: Local Institution - 0028
city: Uwajima
country: Japan
facility: Local Institution - 0370
state: Ehime
city: Himeji-Shi
country: Japan
facility: Local Institution - 0208
state: Hyogo
city: Kanazawa-shi
country: Japan
facility: Local Institution - 0457
state: Ishikawa
city: Yokohama
country: Japan
facility: Local Institution - 0107
state: Kanagawa
city: Nankoku-shi
country: Japan
facility: Local Institution - 0119
state: Kochi
city: Tsu
country: Japan
facility: Local Institution - 0453
state: MIE
city: Sendai
country: Japan
facility: Local Institution - 0456
state: Miyagi
city: Osaka-Shi
country: Japan
facility: Local Institution - 0452
state: Osaka
city: Suita
country: Japan
facility: Local Institution - 0315
state: Osaka
city: Takatsuki-Shi
country: Japan
facility: Local Institution - 0471
state: Osaka
city: Hamamatsu-city
country: Japan
facility: Local Institution - 0168
state: Shizuoka
city: Bunkyo-ku
country: Japan
facility: Local Institution - 0477
state: Tokyo
city: Chuo-Ku
country: Japan
facility: Local Institution - 0505
state: Tokyo
city: Itabashi-Ku
country: Japan
facility: Local Institution - 0224
state: Tokyo
city: Koto-Ku
country: Japan
facility: Local Institution - 0466
state: Tokyo
city: Shinjuku-ku
country: Japan
facility: Local Institution - 0314
state: Tokyo
city: Shinjuku-ku
country: Japan
facility: Local Institution - 0475
state: Tokyo
city: Fukuoka
country: Japan
facility: Local Institution - 0521
city: Daegu
country: Korea, Republic of
facility: Local Institution - 0516
state: Daegu Gwang'yeogsi
city: Incheon
country: Korea, Republic of
facility: Local Institution - 0518
state: Incheon Gwang'yeogsi
city: Seoul
country: Korea, Republic of
facility: Local Institution - 0012
state: Seoul Teugbyeolsi
city: Seoul
country: Korea, Republic of
facility: Local Institution - 0055
state: Seoul Teugbyeolsi
city: Seoul
country: Korea, Republic of
facility: Local Institution - 0460
state: Seoul Teugbyeolsi
city: Busan
country: Korea, Republic of
facility: Local Institution - 0517
city: Seongnam-si
country: Korea, Republic of
facility: Local Institution - 0508
city: Seoul
country: Korea, Republic of
facility: Local Institution - 0217
city: Seoul
country: Korea, Republic of
facility: Local Institution - 0049
city: Nijmegen
country: Netherlands
facility: Local Institution - 0442
state: Gelderland
city: Maastricht
country: Netherlands
facility: Local Institution - 0434
state: Limburg
city: Amsterdam Zuidoost
country: Netherlands
facility: Local Institution - 0197
state: Noord-Holland
city: Rotterdam
country: Netherlands
facility: Local Institution - 0213
state: Zuid-Holland
city: Utrecht
country: Netherlands
facility: Local Institution - 0061
city: Lørenskog
country: Norway
facility: Local Institution - 0443
city: Oslo
country: Norway
facility: Local Institution - 0441
city: Trondheim
country: Norway
facility: Local Institution - 0473
city: Wroclaw
country: Poland
facility: Local Institution - 0038
state: Dolnoslaskie
city: Wroclaw
country: Poland
facility: Local Institution - 0091
state: Dolnoslaskie
city: Lodz
country: Poland
facility: Local Institution - 0059
state: Lodzkie
city: Kraków
country: Poland
facility: Local Institution - 0042
state: Malopolskie
city: Warszawa
country: Poland
facility: Local Institution - 0338
state: Mazowieckie
city: Warszawa
country: Poland
facility: Local Institution - 0203
state: Mazowieckie
city: Rzeszów
country: Poland
facility: Local Institution - 0072
state: Podkarpackie
city: Katowice
country: Poland
facility: Local Institution - 0017
state: Slaskie
city: Zabrze
country: Poland
facility: Local Institution - 0415
state: Slaskie
city: Bialystok
country: Poland
facility: Local Institution - 0030
city: Lódz
country: Poland
facility: Local Institution - 0416
city: Guimarães
country: Portugal
facility: Local Institution - 0044
state: Braga
city: Almada
country: Portugal
facility: Local Institution - 0015
city: Leiria
country: Portugal
facility: Local Institution - 0433
city: Lisboa
country: Portugal
facility: Local Institution - 0023
city: Lisboa
country: Portugal
facility: Local Institution - 0024
city: Vila Nova de Gaia
country: Portugal
facility: Local Institution - 0014
city: Palma de Mallorca
country: Spain
facility: Local Institution - 0040
state: Baleares
city: Hospitalet de Llobregat
country: Spain
facility: Local Institution - 0299
state: Barcelona
city: Majadahonda
country: Spain
facility: Local Institution - 0021
state: Madrid
city: El Palmar
country: Spain
facility: Local Institution - 0237
state: Murcia
city: A Coruna
country: Spain
facility: Local Institution - 0043
city: Alicante
country: Spain
facility: Local Institution - 0087
city: Barcelona
country: Spain
facility: Local Institution - 0018
city: Barcelona
country: Spain
facility: Local Institution - 0056
city: Granada
country: Spain
facility: Local Institution - 0060
city: Madrid
country: Spain
facility: Local Institution - 0421
city: Malaga
country: Spain
facility: Local Institution - 0041
city: Salamanca
country: Spain
facility: Local Institution - 0031
city: Sevilla
country: Spain
facility: Local Institution - 0051
city: Valencia
country: Spain
facility: Local Institution - 0027
city: London
country: United Kingdom
facility: Local Institution - 0445
state: Greater London
city: Glasgow
country: United Kingdom
facility: Local Institution - 0073
state: Lanarkshire
city: Birmingham
country: United Kingdom
facility: Local Institution - 0407
city: London
country: United Kingdom
facility: Local Institution - 0472
Study officials:
affiliation: Bristol-Myers Squibb
name: Bristol-Myers Squibb
role: STUDY_DIRECTOR
References: