Breakthrough heart drug shows promise for debilitating genetic condition

NCT ID NCT05582395

Summary

This Phase 3 trial tested whether the drug mavacamten could improve symptoms and exercise capacity in adults with symptomatic non-obstructive hypertrophic cardiomyopathy (nHCM), a genetic heart condition that causes the heart muscle to thicken. The study involved 580 participants who received either the drug or a placebo for 48 weeks. Researchers measured changes in quality of life, exercise capacity, and heart-related symptoms to determine if the treatment was effective.

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes NO responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

Locations

  • Local Institution - 0001

    Nice, Alpes-Maritimes, 06000, France

  • Local Institution - 0003

    Chambray-lès-Tours, Indre-et-Loire, 37044, France

  • Local Institution - 0004

    Paris, 75013, France

  • Local Institution - 0005

    Toulouse, Haute-Garonne, 31400, France

  • Local Institution - 0006

    Paris, 75015, France

  • Local Institution - 0007

    Stony Brook, New York, 11790-3407, United States

  • Local Institution - 0008

    Rennes, 35000, France

  • Local Institution - 0009

    Nantes, 44093, France

  • Local Institution - 0010

    Los Angeles, California, 90048-3311, United States

  • Local Institution - 0012

    Seoul, Seoul Teugbyeolsi, 03722, South Korea

  • Local Institution - 0013

    Bad Nauheim, Hesse, 61231, Germany

  • Local Institution - 0014

    Vila Nova de Gaia, 4434-502, Portugal

  • Local Institution - 0015

    Almada, 2805-267, Portugal

  • Local Institution - 0016

    Jena, Thuringia, 07747, Germany

  • Local Institution - 0017

    Katowice, Silesian Voivodeship, 40-555, Poland

  • Local Institution - 0018

    Barcelona, 08035, Spain

  • Local Institution - 0019

    Aarhus N, 8200, Denmark

  • Local Institution - 0021

    Majadahonda, Madrid, 28222, Spain

  • Local Institution - 0022

    München, Bavaria, 81377, Germany

  • Local Institution - 0023

    Lisbon, 1500-650, Portugal

  • Local Institution - 0024

    Lisbon, 1649-035, Portugal

  • Local Institution - 0025

    Jerusalem, Jerusalem, 90000, Israel

  • Local Institution - 0026

    Ashkelon, Tel Aviv, 7830604, Israel

  • Local Institution - 0027

    Valencia, 46026, Spain

  • Local Institution - 0028

    Roma, 00189, Italy

  • Local Institution - 0030

    Bialystok, 15-276, Poland

  • Local Institution - 0031

    Salamanca, 37007, Spain

  • Local Institution - 0032

    Hamburg, 20246, Germany

  • Local Institution - 0033

    Prague, Praha, Hlavní Mesto, 140 21, Czechia

  • Local Institution - 0034

    Anderlecht, Brussels Capital, 1070, Belgium

  • Local Institution - 0035

    Budapest, 1122, Hungary

  • Local Institution - 0037

    Tel Aviv, Tel Aviv, 6423906, Israel

  • Local Institution - 0038

    Wroclaw, Lower Silesian Voivodeship, 50-556, Poland

  • Local Institution - 0039

    Budapest, 1097, Hungary

  • Local Institution - 0040

    Palma de Mallorca, Balearic Islands, 07198, Spain

  • Local Institution - 0041

    Málaga, 29010, Spain

  • Local Institution - 0042

    Krakow, Lesser Poland Voivodeship, 31-202, Poland

  • Local Institution - 0043

    A Coruña, 15006, Spain

  • Local Institution - 0044

    Guimarães, Braga District, 4835-044, Portugal

  • Local Institution - 0045

    Aalborg, North Denmark, DK-9000, Denmark

  • Local Institution - 0046

    Münster, 48149, Germany

  • Local Institution - 0047

    Hershey, Pennsylvania, 17033, United States

  • Local Institution - 0048

    Budapest, 1096, Hungary

  • Local Institution - 0049

    Seoul, 05505, South Korea

  • Local Institution - 0050

    Bedford Park, South Australia, 5042, Australia

  • Local Institution - 0051

    Seville, 41009, Spain

  • Local Institution - 0052

    Prague, Praha, Hlavní Mesto, 128 21, Czechia

  • Local Institution - 0053

    Pessac, 33604, France

  • Local Institution - 0054

    Rehovot, 76100, Israel

  • Local Institution - 0055

    Seoul, Seoul Teugbyeolsi, 6351, South Korea

  • Local Institution - 0056

    Barcelona, 08036, Spain

  • Local Institution - 0057

    Jerusalem, Jerusalem, 9103102, Israel

  • Local Institution - 0059

    Lodz, Łódź Voivodeship, 91-347, Poland

  • Local Institution - 0060

    Granada, 18014, Spain

  • Local Institution - 0061

    Utrecht, 3584 CX, Netherlands

  • Local Institution - 0062

    Graz, 8036, Austria

  • Local Institution - 0063

    Concord, New South Wales, 2139, Australia

  • Local Institution - 0064

    Detroit, Michigan, 48202-2608, United States

  • Local Institution - 0065

    Durham, North Carolina, 27710, United States

  • Local Institution - 0066

    Lübeck, Schleswig-Holstein, 23538, Germany

  • Local Institution - 0067

    Bron, Rhône, 69500, France

  • Local Institution - 0069

    Braunau am Inn, Upper Austria (Oberösterreich), 5280, Austria

  • Local Institution - 0070

    Iowa City, Iowa, 52242-1009, United States

  • Local Institution - 0071

    Freiburg im Breisgau, 79106, Germany

  • Local Institution - 0072

    Rzeszów, Podkarpackie Voivodeship, 35-055, Poland

  • Local Institution - 0073

    Glasgow, Lanarkshire, G51 4TF, United Kingdom

  • Local Institution - 0074

    Ramat Gan, 5265601, Israel

  • Local Institution - 0075

    Linz, Upper Austria, 4020, Austria

  • Local Institution - 0076

    Murdoch, Western Australia, 6150, Australia

  • Local Institution - 0077

    Heidelberg, 69120, Germany

  • Local Institution - 0078

    Milton, Queensland, 4064, Australia

  • Local Institution - 0079

    Stanford, California, 94305-2200, United States

  • Local Institution - 0080

    Chermside, Queensland, 4032, Australia

  • Local Institution - 0081

    Pittsburgh, Pennsylvania, 15213-2536, United States

  • Local Institution - 0082

    Birmingham, Alabama, 35233-1900, United States

  • Local Institution - 0083

    Cleveland, Ohio, 44195, United States

  • Local Institution - 0085

    Darlinghurst, New South Wales, 2010, Australia

  • Local Institution - 0086

    Broumov, Královéhradecký kraj, 550 01, Czechia

  • Local Institution - 0087

    Alicante, 03010, Spain

  • Local Institution - 0089

    Dallas, Texas, 75246, United States

  • Local Institution - 0090

    Innsbruck, Tyrol, 6020, Austria

  • Local Institution - 0091

    Wroclaw, Lower Silesian Voivodeship, 50-981, Poland

  • Local Institution - 0092

    Atlanta, Georgia, 30322, United States

  • Local Institution - 0093

    Québec, Quebec, G1V 4G5, Canada

  • Local Institution - 0094

    Charlotte, North Carolina, 28204-2826, United States

  • Local Institution - 0095

    Richmond, Virginia, 23298-5024, United States

  • Local Institution - 0107

    Yokohama, Kanagawa, 227-8501, Japan

  • Local Institution - 0119

    Nankoku-shi, Kochi, 783-8505, Japan

  • Local Institution - 0127

    Baltimore, Maryland, 21287, United States

  • Local Institution - 0168

    Hamamatsu, Shizuoka, 431-3192, Japan

  • Local Institution - 0171

    Pisa, 56124, Italy

  • Local Institution - 0174

    Bielefeld, North Rhine-Westphalia, 33604, Germany

  • Local Institution - 0188

    Brno, 656 91, Czechia

  • Local Institution - 0197

    Amsterdam-Zuidoost, North Holland, 1105 AZ, Netherlands

  • Local Institution - 0203

    Warsaw, Masovian Voivodeship, 02-507, Poland

  • Local Institution - 0207

    Germantown, Tennessee, 38138-1727, United States

  • Local Institution - 0208

    Himeji-Shi, Hyōgo, 672-8044, Japan

  • Local Institution - 0213

    Rotterdam, South Holland, 3015 GD, Netherlands

  • Local Institution - 0217

    Seoul, 03080, South Korea

  • Local Institution - 0224

    Itabashi-Ku, Tokyo, 173-0003, Japan

  • Local Institution - 0237

    El Palmar, Murcia, 30120, Spain

  • Local Institution - 0253

    Safed, 13100, Israel

  • Local Institution - 0263

    Philadelphia, Pennsylvania, 19104, United States

  • Local Institution - 0273

    Cologne, North Rhine Wesrphalia, 50924, Germany

  • Local Institution - 0281

    Tampa, Florida, 33606-3603, United States

  • Local Institution - 0299

    L'Hospitalet de Llobregat, Barcelona, 08907, Spain

  • Local Institution - 0314

    Shinjuku-ku, Tokyo, 160-8582, Japan

  • Local Institution - 0315

    Suita, Osaka, 564-8565, Japan

  • Local Institution - 0319

    Leuven, Vlaams Brabant, 3000, Belgium

  • Local Institution - 0330

    Gūrgaon, 122 001, India

  • Local Institution - 0338

    Warsaw, Masovian Voivodeship, 02-097, Poland

  • Local Institution - 0365

    Dallas, Texas, 75390, United States

  • Local Institution - 0369

    Sydney, New South Wales, 2170, Australia

  • Local Institution - 0370

    Uwajima, Ehime, 798-8510, Japan

  • Local Institution - 0373

    Florence, Tuscany, 50139, Italy

  • Local Institution - 0381

    Calgary, Alberta, T2N 1N4, Canada

  • Local Institution - 0402

    Salt Lake City, Utah, 84132-0001, United States

  • Local Institution - 0403

    Berlin, 13353, Germany

  • Local Institution - 0404

    Ulm, Baden-Wurttemberg, 89081, Germany

  • Local Institution - 0406

    San Francisco, California, 94143-2204, United States

  • Local Institution - 0407

    Birmingham, B15 2TH, United Kingdom

  • Local Institution - 0409

    La Jolla, California, 92037-1337, United States

  • Local Institution - 0410

    Kansas City, Kansas, 66160-8500, United States

  • Local Institution - 0411

    London, Ontario, N6A 5A5, Canada

  • Local Institution - 0412

    São Paulo, São Paulo, 05403-000, Brazil

  • Local Institution - 0413

    Salvador, Estado de Bahia, 40170-130, Brazil

  • Local Institution - 0414

    Hamilton, Ontario, L8L 2X2, Canada

  • Local Institution - 0415

    Zabrze, Silesian Voivodeship, 41-800, Poland

  • Local Institution - 0416

    Lódz, 93-338, Poland

  • Local Institution - 0417

    Szeged, 6720, Hungary

  • Local Institution - 0418

    São Paulo, 04012-180, Brazil

  • Local Institution - 0419

    Haifa, 3109601, Israel

  • Local Institution - 0420

    Mainz, Rhineland-Palatinate, 55131, Germany

  • Local Institution - 0421

    Madrid, 28034, Spain

  • Local Institution - 0422

    Ribeirão Preto, São Paulo, 14026-900, Brazil

  • Local Institution - 0423

    Balatonfüred, Veszprém megye, 8230, Hungary

  • Local Institution - 0424

    Indianapolis, Indiana, 46202-1243, United States

  • Local Institution - 0426

    Valhalla, New York, 10595, United States

  • Local Institution - 0427

    Petah Tikva, 49100, Israel

  • Local Institution - 0428

    Aabenraa, Sønderjylland, 6200, Denmark

  • Local Institution - 0429

    Vienna, 1090, Austria

  • Local Institution - 0430

    Bragança Paulista, São Paulo, 12916-542, Brazil

  • Local Institution - 0431

    Rochester, Minnesota, 55905, United States

  • Local Institution - 0433

    Leiria, 2410-197, Portugal

  • Local Institution - 0434

    Maastricht, Limburg, 6229 HX, Netherlands

  • Local Institution - 0435

    Omaha, Nebraska, 68198-2265, United States

  • Local Institution - 0438

    Brasília, Federal District, 70710-140, Brazil

  • Local Institution - 0439

    Bad Oeynhausen, North Rhine-Westphalia, 32545, Germany

  • Local Institution - 0440

    Regensburg, Bavaria, 93053, Germany

  • Local Institution - 0441

    Oslo, 0424, Norway

  • Local Institution - 0442

    Nijmegen, Gelderland, 6525 GA, Netherlands

  • Local Institution - 0443

    Lørenskog, 1478, Norway

  • Local Institution - 0444

    Lancaster, Pennsylvania, 17602-2250, United States

  • Local Institution - 0445

    London, Greater London, SW3 6NP, United Kingdom

  • Local Institution - 0446

    Rio de Janeiro, 20551-030, Brazil

  • Local Institution - 0447

    Porto Alegre, Rio Grande do Sul, 90560-030, Brazil

  • Local Institution - 0448

    Hanover, Lower Saxony, 30625, Germany

  • Local Institution - 0449

    Manhasset, New York, 11030-3816, United States

  • Local Institution - 0450

    Porto Alegre, Rio Grande do Sul, 90620-001, Brazil

  • Local Institution - 0451

    Rio de Janeiro, 22061-080, Brazil

  • Local Institution - 0452

    Osaka, Osaka, 545-0051, Japan

  • Local Institution - 0453

    Tsu, Mie-ken, 514-8507, Japan

  • Local Institution - 0454

    München, 80636, Germany

  • Local Institution - 0455

    Grand Rapids, Michigan, 49525, United States

  • Local Institution - 0456

    Sendai, Miyagi, 980-8574, Japan

  • Local Institution - 0457

    Kanazawa, Ishikawa-ken, 920-8641, Japan

  • Local Institution - 0458

    Phoenix, Arizona, 85054, United States

  • Local Institution - 0460

    Seoul, Seoul Teugbyeolsi, 6591, South Korea

  • Local Institution - 0461

    St Louis, Missouri, 63110, United States

  • Local Institution - 0462

    Edegem, Antwerpen, 2650, Belgium

  • Local Institution - 0463

    Brest, Finistère, 29200, France

  • Local Institution - 0464

    Ann Arbor, Michigan, 48109, United States

  • Local Institution - 0466

    Koto-Ku, Tokyo, 135-0061, Japan

  • Local Institution - 0467

    Vancouver, British Columbia, V6E 1M7, Canada

  • Local Institution - 0468

    Gainesville, Florida, 32608-1134, United States

  • Local Institution - 0471

    Takatsuki-Shi, Osaka, 569-8686, Japan

  • Local Institution - 0472

    London, EC1A 7BE, United Kingdom

  • Local Institution - 0473

    Trondheim, 7030, Norway

  • Local Institution - 0475

    Shinjuku-ku, Tokyo, 162-8666, Japan

  • Local Institution - 0476

    Genk, Limburg, 3600, Belgium

  • Local Institution - 0477

    Bunkyo-ku, Tokyo, 1138603, Japan

  • Local Institution - 0478

    Charlottesville, Virginia, 22908-0001, United States

  • Local Institution - 0479

    Boston, Massachusetts, 02114, United States

  • Local Institution - 0480

    Bengaluru, Karnataka, 560099, India

  • Local Institution - 0481

    Kochi, Kerala, 682001, India

  • Local Institution - 0482

    Ahmedabad, Gujarat, 380060, India

  • Local Institution - 0483

    Chandigarh, 160012, India

  • Local Institution - 0484

    Louisville, Kentucky, 40217-1363, United States

  • Local Institution - 0486

    New York, New York, 10019-1147, United States

  • Local Institution - 0488

    Belo Horizonte, Minas Gerais, 30220-140, Brazil

  • Local Institution - 0489

    La Tronche, Isère, 38700, France

  • Local Institution - 0490

    Morristown, New Jersey, 07962, United States

  • Local Institution - 0492

    Plano, Texas, 75093, United States

  • Local Institution - 0496

    Göttingen, Lower Saxony, 37075, Germany

  • Local Institution - 0500

    Boston, Massachusetts, 02118-2908, United States

  • Local Institution - 0501

    Shenyang, Liaoning, 110016, China

  • Local Institution - 0502

    Beijing, 100730, China

  • Local Institution - 0503

    Chongqing, Chongqing Municipality, 400016, China

  • Local Institution - 0505

    Chuo-Ku, Tokyo, 104-0044, Japan

  • Local Institution - 0508

    Seongnam-si, 0, South Korea

  • Local Institution - 0516

    Daegu, Daegu Gwang'yeogsi, 42601, South Korea

  • Local Institution - 0517

    Busan, 49241, South Korea

  • Local Institution - 0518

    Incheon, Incheon Gwang'yeogsi, 22332, South Korea

  • Local Institution - 0521

    Fukuoka, 810-0001, Japan

  • Local Institution - 0533

    Chengdu, Sichuan, 610000, China

  • Local Institution - 0534

    Beijing, Beijing Municipality, 100730, China

  • Local Institution - 0535

    Beijing, Beijing Municipality, 100083, China

  • Local Institution - 0536

    Wenzhou, Zhejiang, 325000, China

  • Local Institution - 0537

    Shanghai, Shanghai Municipality, 200127, China

  • Local Institution - 0538

    Shenyang, Liaoning, 110016, China

  • Local Institution - 0539

    Guangzhou, Guangdong, 510120, China

  • Local Institution - 0540

    Dalian, Liaoning, 116011, China

  • Local Institution - 0541

    Chongqing, Chongqing Municipality, 400016, China

  • Local Institution - 0542

    Wuhan, Hubei, 430060, China

  • Local Institution - 0543

    Guangzhou, Guangdong, 510515, China

  • Local Institution - 0544

    Changchun, Jilin, 130041, China

  • Local Institution - 0545

    Suzhou, Jiangsu, 215006, China

  • Local Institution - 0546

    Jinan, Shandong, 250021, China

  • Local Institution - 0547

    Xi'an, Shan1xi, 710061, China

  • Local Institution - 0548

    Changsha, Hunan, 410011, China

  • Local Institution - 0549

    Zhengzhou, Henan, 450003, China

  • Local Institution - 0550

    Nanchang, Jiangxi, 330008, China

  • Local Institution - 0552

    Chengdu, Sichuan, 610015, China

  • Local Institution - 0553

    Beijing, 102218, China

Conditions

Explore the condition pages connected to this study.