Overview
Sponsor-declared trial summary
Phase
Phase III and Phase IV (Integrated)
Status
Ended
Participants planned
175
Countries
11
Sites
50
Symptomatic Obstructive Hypertrophic Cardiomyopathy
The purpose of this study is to see if mavacamten treatment reduces the heart wall (ventricle) thickness and size of the upper heart chamber (atria) on imaging studies by cardiac magnetic resonance (CMR).
Key facts
- Sponsor
- Myokardia Inc.
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Cardiovascular Diseases [C14]
- Trial duration
- completed 15 Mar 2024
- Decision date (initial)
- 2024-02-12
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- No
- Vulnerable population
- Yes
- Funding sources
- Myokardia, Inc, A Wholly Owned Susidiary of Bristol Myers Squibb Company.
External identifiers
- EU CT number
- 2022-502316-36-00
- WHO UTN
- U1111-1284-1414
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Efficacy
The purpose of this study is to see if mavacamten treatment reduces the heart wall (ventricle) thickness and size of the upper heart chamber (atria) on imaging studies by cardiac magnetic resonance (CMR).
Secondary objectives 1
- To explore the effect of mavacamten on the NYHA functional classification as well as safety events during the study period.
Conditions and MedDRA coding
Symptomatic Obstructive Hypertrophic Cardiomyopathy
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 20.0 | PT | 10020871 | Hypertrophic cardiomyopathy | 100000004850 |
Regulatory references
| EU CT number | Title | Sponsor |
|---|---|---|
| 2022-502858-14-00 | A Long-term Safety Extension Study of Mavacamten (MYK-461) in Adults with Hypertrophic Cardiomyopathy Who Have Completed the MAVERICKHCM (MYK-461-006) or EXPLORER-HCM (MYK-461-005) Trials (MAVA-LTE) | Myokardia Inc. |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 1
- This trial will assess elegible adults with symptomatic oHCM (NYHA Funtional Class II or III)
Exclusion criteria 1
- Adults with no symptomatic oHCM (NYHA Funtional Class II or III)
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- To evaluate the efficacy of mavacamten on both the reduction in heart wall thickness and upper chamber size at week 48 on cardiac magnetic resonance (CMR).
Secondary endpoints 1
- To evaluate the impact of mavacamten treatment: on improvement of heart failure class in symptomatic oHCM (New York Heart Association [NYHA] Functional Class) as well as monitor for other cardiac events as a safety assessment.
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 4
PRD10116939 · Product
- Active substance
- Mavacamten
- Substance synonyms
- MYK-461, 6-{[(1S)-1-phenylethyl]amino}-3-(propan-2-yl)-1,2,3,4 tetrahydropyrimidine-2,4-dione, SAR439152, 3-(1-methylethyl)-6-[[(1S)-1-phenylethyl]amino]-2,4(1H,3H)-pyrimidinedione
- Other product name
- 10 mg per capsule
- Pharmaceutical form
- CAPSULE
- Route of administration
- ORAL
- Max daily dose
- 15 mg milligram(s)
- Max total dose
- 15 mg milligram(s)
- Max treatment duration
- 96 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- BRISTOL-MYERS SQUIBB INTERNATIONAL CORPORATION
- Paediatric formulation
- No
- Orphan designation
- No
PRD10116938 · Product
- Active substance
- Mavacamten
- Substance synonyms
- MYK-461, 6-{[(1S)-1-phenylethyl]amino}-3-(propan-2-yl)-1,2,3,4 tetrahydropyrimidine-2,4-dione, SAR439152, 3-(1-methylethyl)-6-[[(1S)-1-phenylethyl]amino]-2,4(1H,3H)-pyrimidinedione
- Other product name
- 5 mg per capsule
- Pharmaceutical form
- CAPSULE
- Route of administration
- ORAL
- Max daily dose
- 15 mg milligram(s)
- Max total dose
- 15 mg milligram(s)
- Max treatment duration
- 96 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- BRISTOL-MYERS SQUIBB INTERNATIONAL CORPORATION
- Paediatric formulation
- No
- Orphan designation
- No
PRD10116937 · Product
- Active substance
- Mavacamten
- Substance synonyms
- MYK-461, 6-{[(1S)-1-phenylethyl]amino}-3-(propan-2-yl)-1,2,3,4 tetrahydropyrimidine-2,4-dione, SAR439152, 3-(1-methylethyl)-6-[[(1S)-1-phenylethyl]amino]-2,4(1H,3H)-pyrimidinedione
- Other product name
- 2.5 mg per capsule
- Pharmaceutical form
- CAPSULE
- Route of administration
- ORAL
- Max daily dose
- 15 mg milligram(s)
- Max total dose
- 15 mg milligram(s)
- Max treatment duration
- 96 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- BRISTOL-MYERS SQUIBB INTERNATIONAL CORPORATION
- Paediatric formulation
- No
- Orphan designation
- No
PRD10116941 · Product
- Active substance
- Mavacamten
- Substance synonyms
- MYK-461, 6-{[(1S)-1-phenylethyl]amino}-3-(propan-2-yl)-1,2,3,4 tetrahydropyrimidine-2,4-dione, SAR439152, 3-(1-methylethyl)-6-[[(1S)-1-phenylethyl]amino]-2,4(1H,3H)-pyrimidinedione
- Other product name
- 15 mg per capsule
- Pharmaceutical form
- CAPSULE
- Route of administration
- ORAL
- Max daily dose
- 15 mg milligram(s)
- Max total dose
- 15 mg milligram(s)
- Max treatment duration
- 96 Week(s)
- Authorisation status
- Not Authorised
- MA holder
- BRISTOL-MYERS SQUIBB INTERNATIONAL CORPORATION
- Paediatric formulation
- No
- Orphan designation
- No
Placebo 1
N/A · Product
- Other product name
- N/A
- Pharmaceutical form
- N/A
- ATC code
- N/A — N/A
- Marketing authorisation
- N/A
- MA holder
- N/A
- MA country
- Iceland
- Paediatric formulation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Myokardia Inc.
- Sponsor organisation
- Myokardia Inc.
- Address
- 1000 Sierra Point Pky
- City
- Brisbane
- Postcode
- 94005
- Country
- United States
Scientific contact point
- Organisation
- Myokardia Inc.
- Contact name
- GSM-CT
Public contact point
- Organisation
- Myokardia Inc.
- Contact name
- GSM-CT
Third parties 8
| Organisation | City, country | Duties |
|---|---|---|
| Signant Health Global LLC ORG-100040604
|
Blue Bell, United States | Other |
| Perceptive Informatics Inc. ORG-100013171
|
Billerica, United States | Code 13 |
| Pharmaceutical Product Development LLC ORG-100016999
|
Wilmington, United States | On site monitoring, Code 10, Code 11, Code 12, Code 2, Data management, Code 8, Code 9 |
| RWS Life Sciences Inc. ORG-100042348
|
East Hartford, United States | Other |
| Yprime LLC ORG-100042888
|
Malvern, United States | Interactive response technologies (IRT) |
| PPD Global Ltd. ORG-100007531
|
Marousi, Greece | On site monitoring, Code 12, Code 2, Code 8 |
| Greenphire LLC ORG-100041621
|
King Of Prussia, United States | Other |
| Labcorp Central Laboratory Services LP ORG-100032236
|
Indianapolis, United States | Laboratory analysis |
Locations
11 EU/EEA countries · 50 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Austria | Ended | 11 | 5 |
| Belgium | Ended | 10 | 5 |
| Czechia | Ended | 7 | 2 |
| Finland | Ended | 5 | 2 |
| France | Ended | 7 | 4 |
| Germany | Ended | 7 | 4 |
| Greece | Ended | 12 | 6 |
| Hungary | Ended | 12 | 6 |
| Italy | Ended | 11 | 6 |
| Poland | Not authorised | 11 | 3 |
| Spain | Ended | 12 | 7 |
| Rest of world
Argentina, United States, Australia, Switzerland, United Kingdom, Canada
|
— | 70 | — |
Investigational sites
Medical University Of Graz
Department of Internal Medicine Division of Cardiology, Neue Stiftingtalstrasse 6, 8010, Graz
Medical University of Vienna
Department of Internal Medicine II Division of Cardiology, Waehringer Guertel 18-20, Alsergrund, Vienna
Landeskrankenanstalten-Betriebsgesellschaft Kabeg
N/A, Feschnigstrasse 11, Klagenfurt,09.Bez.:Annabichl, Klagenfurt Am Woerthersee
A.O. Krankenhaus St. Josef Braunau GmbH
N/A, Ringstrasse 60, 5280, Braunau Am Inn
Klinik Favoriten
5th Medical Department/Cardiology, Kundratstrasse 3, Favoriten, Vienna
Hopital Erasme
Cardiology, Lennikse Baan 808, 1070, Anderlecht
Algemeen Ziekenhuis Delta
Cardiology, Deltalaan 1, 8800, Roeselare
UZ Brussel
Cardiology, Laarbeeklaan 101, 1090, Jette
Cliniques Universitaires Saint-Luc
Cardiology, Hippokrateslaan 10, Batiment 54, Sint-Lambrechts-Woluwe
UZ Leuven
Cardiology, Herestraat 49, 3000, Leuven
Fakultni Nemocnice U Sv Anny V Brne
I. Interní kardioangiologická klinika, Pekarska 53, Stare Brno, Brno-Stred
Nemocnice AGEL Trinec-Podlesi a.s.
Kardiologické oddělení, Konska 453, 739 61, Konska
HUS Helsinki University Hospital
Cardiovascular laboratory, Haartmaninkatu 4, 00290, Helsinki
Turku University Hospital
Heart Center, Hameentie 11, 20520, Turku
Centre Hospitalier Universitaire De Rennes
N/A, 2 Rue Henri Le Guilloux, 35000, Rennes
Centre Hospitalier Universitaire Grenoble Alpes
N/A, Boulevard De La Chantourne, Cs 10217, Grenoble Cedex 9
Assistance Publique Hopitaux De Paris
N/A, 20 Rue Leblanc, 75015, Paris
Centre Hospitalier Universitaire De Bordeaux
N/A, Avenue De Magellan, 33600, Pessac
Charite Universitaetsmedizin Berlin KöR
Deutsches Herzzentrum der Charité Klinik für Kardiologie, Angiologie und Intensivmedizin, Hindenburgdamm 30, Lichterfelde, Berlin
Herz Und Diabeteszentrum NRW Bad Oeynhausen Universitaetsklinik Der Ruhr-Universitaet Bochum
Herz- und Diabeteszentrum Nordrhein-Westfalen, Georgstrasse 11, Innenstadt, Bad Oeynhausen
Universitaetsmedizin Goettingen
Klinik für Kardiologie und Pneumologie, Robert-Koch-Strasse 40, Weende, Goettingen
Justus-Liebig-Universitaet Giessen
Medizinische Klinik I Abteilung für Kardiologie/Angiologie, Klinikstrasse 33, 35392, Giessen
Hippokration Hospital
1 st Department of Cardiology, Vassilissas Sofias Avenue 114, 115 27, Athens
University General Hospital Of Thessaloniki Ahepa
1 st Department of Cardiology, 1st St Kiriakidis Str, 546 36, Thessaloniki
Hippokration Hospital
3rd Department of Cardiology, Konstadinoupoleos 49, 546 42, Thessaloniki
University General Hospital Of Heraklion
Department of Cardiology, Stavrakia And Voutes, 715 00, Heraklion
Onassis Cardiac Surgery Center
Unit of Inherited and Rare Cardiovascular Diseases, Leoforos Andrea Siggrou 356, 176 74, Kallithea
University General Hospital Attikon
2nd Department of Cardiology, Rimini Street 1, 124 62, Athens
University Of Pecs
Heart Institute, Ifjusag Utja 13, 7624, Pecs
Central Hospital Of Northern Pest Military Hospital
Cardiology, Robert Karoly Korut 44, 1134, Budapest XIII
Semmelweis University
Heart and Vascular Center, Varosmajor Utca 68, Kerulet, Budapest XII
Gottsegen National Cardiovascular Center
Adult cardiology department, Kerulet, Haller Utca 29/IX., Budapest
University Of Szeged
2nd department of Internal medicine and Cardiology Center, Semmelweis Utca 8, 6725, Szeged
Balatonfueredi Allami Szivkorhaz
Cardiology Department I., Gyogy Ter 2, 8230, Balatonfured
Istituto Auxologico Italiano
Department of Cardiology, Cardiological, Piazzale Brescia 20, 20149, Milan
Careggi University Hospital
Clinica medica di cardiologia, Largo Giovanni Alessandro Brambilla 3, 50134, Florence
Fondazione IRCCS Policlinico San Matteo
Cardiology Unit 1, Viale Camillo Golgi 19, 27100, Pavia
Fondazione Toscana Gabriele Monasterio
Cardio-Thoracic-UOC Cardiology and cardiovascular Medicine, Via Giuseppe Moruzzi 1, 56124, Pisa
Azienda USL Toscana Sud Est
Cardiology department, Ospedale Area Aretina Nord, Via Pietro Nenni 20/22, Arezzo
ASST Grande Ospedale Metropolitano Niguarda
Cardio-toracico-vascolare, Piazza Dell'ospedale Maggiore 3, 20162, Milan
Panstwowy Instytut Medyczny Ministerstwa Spraw Wewnetrznych I Administracji
Klinika Kardiologii, Ul. Woloska 137, 02-507, Warsaw
Slaskie Centrum Chorob Serca W Zabrzu
II Oddział Kardiologii i Angiologii, Ul. Marii Curie-Sklodowskiej 9, 41-800, Zabrze
Kardio Brynow Sp. z o.o.
N/A, Ul. Rolna 17/4-5, 40-555, Katowice
University Hospital Virgen Del Rocio S.L.
Cardiology department, Avenida De Manuel Siurot S/n, 41013, Sevilla
Hospital Universitario Ramon Y Cajal
Cardiology department, Carretera Del Colmenar Viejo Km 9 100, Por El Pardo, Madrid
Hospital Universitari Vall D Hebron
Cardiology department, Edificio Materno-Infantil, Passeig De La Vall D'hebron 119-129, Barcelona
Area De Salud De Salamanca
Cardiology Department, Paseo De San Vicente 55-182, 37007, Salamanca
Hospital Universitario Puerta De Hierro De Majadahonda
Cardiology department, Calle De Joaquin Rodrigo 2, 28222, Majadahonda
Hospital Son Llatzer
Department of cardiology, Carretera De Manacor Km 4, 07198, Palma
Hospital Universitario Virgen De La Victoria
Cardilology department, Calle Del Arroyo Teatinos Sn, 29010, Malaga
Application history
1 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2023-09-28 | Austria | Acceptable with conditions 2024-02-05
|
2024-02-06 |