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Clinical Study to Evaluate Mavacamten (MYK-461) in Adults With Symptomatic Obstructive Hypertrophic Cardiomyopathy (EXPLORER-HCM)

Primary Purpose

Obstructive Hypertrophic Cardiomyopathy

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
mavacamten
Placebo
Sponsored by
MyoKardia, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Obstructive Hypertrophic Cardiomyopathy focused on measuring Symptomatic, left ventricular outflow tract gradient

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Key Inclusion Criteria:

  • Age 18 and greater, body weight ≥ 45kg
  • Has adequate acoustic windows to enable accurate transthoracic echocardiograms (TTEs)
  • Diagnosed with oHCM consistent with current American College of Cardiology Foundation/American Heart Association and European Society of Cardiology guidelines and satisfy both criteria:
  • Has documented left ventricular ejection fraction (LVEF) ≥55%
  • NYHA Class II or III
  • Has documented oxygen saturation at rest ≥90% at Screening
  • Is able to perform an upright CPET and has a respiratory exchange ratio (RER) ≥1.0 at Screening per central reading

Key Exclusion Criteria:

  • Known infiltrative or storage disorder causing cardiac hypertrophy that mimics oHCM, such as Fabry disease, amyloidosis, or Noonan syndrome with LV hypertrophy
  • History of syncope or sustained ventricular tachyarrhythmia with exercise within 6 months prior to Screening
  • History of resuscitated sudden cardiac arrest (at any time) or known history of appropriate implantable cardioverter defibrillator (ICD) discharge for life-threatening ventricular arrhythmia within 6 months prior to Screening
  • Paroxysmal, intermittent atrial fibrillation with atrial fibrillation present at Screening
  • Persistent or permanent atrial fibrillation not on anticoagulation for at least 4 weeks prior to Screening and/or not adequately rate controlled within 6 months prior to Screening
  • Treatment (within 14 days prior to Screening) or planned treatment during the study with disopyramide or ranolazine
  • Treatment (within 14 days prior to Screening) or planned treatment during the study with a combination of β-blockers and calcium channel blockers
  • LVOT gradient with Valsalva maneuver <30 mmHg at Screening
  • Has been successfully treated with invasive septal reduction (surgical myectomy or percutaneous alcohol septal ablation [ASA]) within 6 months prior to Screening or plans to have either of these treatments during the study
  • ICD placement within 2 months prior to Screening or planned ICD placement during the study
  • Has a history or evidence of any other clinically significant disorder, condition, or disease that, in the opinion of the investigator, would pose a risk to participant safety or interfere with the study evaluation, procedures, or completion
  • Prior treatment with cardiotoxic agents such as doxorubicin or similar

Sites / Locations

  • Mayo Clinic Arizona
  • Cedars-Sinai Medical Center (Smidt Heart Institute)
  • UCSF School of Medicine
  • Stanford University
  • Yale New Haven Hospital
  • Mayo Clinic Jacksonville - PPDS
  • Northwestern University
  • University Of Iowa Hospitals And Clinics
  • Brigham and Women's Hospital
  • University of Michigan
  • Spectrum Health
  • Washington University School of Medicine
  • NYU Langone Medical Center
  • Columbia University Medical Center
  • Carolinas Medical Center
  • Duke Cardiology at Southpoint
  • Cincinnati Children's Hospital Medical Center
  • Oregon Health & Science University
  • St. Luke's Cardiology Associates
  • University of Pennsylvania (Penn Heart and Vascular Center)
  • University of Pittsburgh Medical Center Presbyterian
  • Methodist University Hospital
  • University of Texas Southwestern Medical Center
  • Houston Methodist Hospital
  • University of Texas Houston Medical School
  • Intermountain Medical Center
  • University of Utah
  • University of Virginia Health System
  • University of Washington Medical Center
  • UZ Antwerpen
  • Onze-Lieve-Vrouwziekenhuis
  • Hôpital Erasme
  • Institut Klinicke a Experimentalni Mediciny
  • Vseobecna fakultni nemocnice v Praze
  • Aarhus Universitetshospital
  • Bispebjerg Hospital
  • Odense Universitetshospital
  • CHRU Nantes
  • Groupe Hospitalier Pitié Salpétrière
  • Hôpital Européen Georges Pompidou
  • Hôpital de Rangueil
  • University Medicine Göttingen
  • Kerckhoff-Klinik-Forschungs-GmbH
  • Charité Campus Buch - Experimental and Clinical Research Center
  • Charité - Universitätsmedizin Berlin
  • Cardiologicum Dresden und Pirna
  • University Clinic Heidelberg - PPDS
  • Universitatsklinikum Schleswig-Holstein
  • Tel Aviv Sourasky Medical Center
  • Barzilai Medical Center
  • Hadassah Medical Center PPDS -
  • Rabin Medical Center - PPDS
  • The Chaim Sheba Medical Center - The Edmond and Lily Safra Children's Hospital
  • Kaplan Medical Center
  • ZIV Medical Center
  • Azienda Ospedaliera Universitaria Careggi
  • Maastricht University Medical Center
  • Erasmus MC
  • Collegium Medicum Uniwersytetu Jagiellonskiego
  • Kardio Klinika Brynów
  • Szpital Kliniczny Przemienienia Panskiego Uniwesytetu Medycznego im. Karola Marcinkowskiego
  • Instytut Kardiologii im Prymasa Tysiaclecia Kardynala Stefana Wyszynskiego
  • Hospital Garcia de Orta
  • Hospital da Luz
  • Hospital Universitario Virgen de La Arrixaca
  • Hospital Universitario A Coruña
  • Hospital Universitario Puerta de Hierro - Majadahonda
  • Hospital Universitario Ramon y Cajal
  • Hospital Universitario Virgen Macarena
  • University Hospital of Wales
  • St Bartholomew's Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Placebo Comparator

Arm Label

mavacamten (MYK-461)

Placebo

Arm Description

Outcomes

Primary Outcome Measures

Percentage of Participants Achieving A Clinical Response
A positive clinical response (value="YES") is defined as having achieved either an improvement of at least 1.5 mL/kg/min in peak oxygen consumption (pVO2) as determined by cardiopulmonary exercise testing (CPET) and a reduction of one or more class in New York Heart Association (NYHA) functional classification (e.g.I, II, III, or IV) -OR- an improvement of 3.0 mL/kg/min or more in pVO2 with no worsening in NYHA Functional Class.

Secondary Outcome Measures

Changes From Baseline to Week 30 in Post Exercise in LVOT Peak Gradient.
The post-exercise LVOT gradient was measured from echocardiograms obtained at baseline and week 30 following a study-specified exercise protocol and read by the Cardiovascular Imaging Core Laboratory (CICL, Boston MA). Change from baseline was determined as per the study statistical analysis plan and compared between treatment arms.
Change From Baseline to Week 30 in pVO2 as Assessed by CPET
Cardiopulmonary exercise testing (CPET) was performed at baseline and week 30 following a study-specified protocol and peak oxygen consumption (pVO2) was determined by the Cardiovascular Metabolic Disease Research Institute (CMDRI, Palo Alto, CA). Change from baseline was determined as per the study statistical analysis plan and compared between treatment arms.
Proportion of Participants With at Least 1 Class Improvement in NYHA Functional Class From Baseline to Week 30
New York Heart Association (NYHA) functional classification was determined by the principal investigator at baseline and at specified timepoints in the study. At baseline, all subjects were NYHA Class II or III. For the secondary outcome, NYHA class at Week 30 was compared to baseline and the proportion of subjects with an improvement of at least one class was determined, and the difference between treatment groups was analyzed. The proportion was also multiplied by 100 to provide the result as a percent.
Change From Baseline to Week 30 in Participant-reported Health-related Quality of Life as Assessed by the KCCQ Score
The Kansas City Cardiomyopathy Questionnaire (KCCQ) is a patient reported outcome instrument with minimum score = 0 and maximum score = 100 where higher score indicates better health status. There are no units to the score. The instrument utilizes a recall period of 2 weeks over which patients describe the frequency and severity of their symptoms, their physical and social limitations, and how they perceive their heart failure symptoms to affect their quality of life. The KCCQ clinical summary (KCCQ-CS) score, a prespecified secondary outcome of EXPLORER-HCM, combines the physical limitation and total symptom scores.
Change From Baseline to Week 30 in Participant-reported Severity of HCM Symptoms as Assessed by the HCMSQ Score
The Hypertrophic Cardiomyopathy Symptom Questionnaire (HCMSQ) is a patient reported outcome instrument that is a daily self-administered 11-item questionnaire. The HCMSQ assesses the core symptoms of HCM (tiredness/fatigue, heart palpitations, chest pain, dizziness, and shortness of breath). The Shortness of Breath domain score, a pre-specified secondary outcome of EXPLORER-HCM, assesses the frequency and severity of shortness of breath. The minimum score = 0 and maximum score = 18 where lower score indicates better health status. There are no units to the score.

Full Information

First Posted
March 12, 2018
Last Updated
September 7, 2021
Sponsor
MyoKardia, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT03470545
Brief Title
Clinical Study to Evaluate Mavacamten (MYK-461) in Adults With Symptomatic Obstructive Hypertrophic Cardiomyopathy
Acronym
EXPLORER-HCM
Official Title
A Randomized, Double Blind, Placebo Controlled Clinical Study to Evaluate Mavacamten (MYK-461) in Adults With Symptomatic Obstructive Hypertrophic Cardiomyopathy
Study Type
Interventional

2. Study Status

Record Verification Date
May 2020
Overall Recruitment Status
Completed
Study Start Date
May 29, 2018 (Actual)
Primary Completion Date
March 14, 2020 (Actual)
Study Completion Date
May 6, 2020 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
MyoKardia, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
This is a multicenter, international, double-blind study of the administration of mavacamten in participants with symptomatic obstructive HCM (oHCM). Approximately 220 participants will be randomized to receive placebo or mavacamten.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Obstructive Hypertrophic Cardiomyopathy
Keywords
Symptomatic, left ventricular outflow tract gradient

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
251 (Actual)

8. Arms, Groups, and Interventions

Arm Title
mavacamten (MYK-461)
Arm Type
Experimental
Arm Title
Placebo
Arm Type
Placebo Comparator
Intervention Type
Drug
Intervention Name(s)
mavacamten
Other Intervention Name(s)
MYK-461
Intervention Description
mavacamten capsules
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
placebo oral capsule
Primary Outcome Measure Information:
Title
Percentage of Participants Achieving A Clinical Response
Description
A positive clinical response (value="YES") is defined as having achieved either an improvement of at least 1.5 mL/kg/min in peak oxygen consumption (pVO2) as determined by cardiopulmonary exercise testing (CPET) and a reduction of one or more class in New York Heart Association (NYHA) functional classification (e.g.I, II, III, or IV) -OR- an improvement of 3.0 mL/kg/min or more in pVO2 with no worsening in NYHA Functional Class.
Time Frame
30 weeks
Secondary Outcome Measure Information:
Title
Changes From Baseline to Week 30 in Post Exercise in LVOT Peak Gradient.
Description
The post-exercise LVOT gradient was measured from echocardiograms obtained at baseline and week 30 following a study-specified exercise protocol and read by the Cardiovascular Imaging Core Laboratory (CICL, Boston MA). Change from baseline was determined as per the study statistical analysis plan and compared between treatment arms.
Time Frame
30 weeks
Title
Change From Baseline to Week 30 in pVO2 as Assessed by CPET
Description
Cardiopulmonary exercise testing (CPET) was performed at baseline and week 30 following a study-specified protocol and peak oxygen consumption (pVO2) was determined by the Cardiovascular Metabolic Disease Research Institute (CMDRI, Palo Alto, CA). Change from baseline was determined as per the study statistical analysis plan and compared between treatment arms.
Time Frame
30 weeks
Title
Proportion of Participants With at Least 1 Class Improvement in NYHA Functional Class From Baseline to Week 30
Description
New York Heart Association (NYHA) functional classification was determined by the principal investigator at baseline and at specified timepoints in the study. At baseline, all subjects were NYHA Class II or III. For the secondary outcome, NYHA class at Week 30 was compared to baseline and the proportion of subjects with an improvement of at least one class was determined, and the difference between treatment groups was analyzed. The proportion was also multiplied by 100 to provide the result as a percent.
Time Frame
30 weeks
Title
Change From Baseline to Week 30 in Participant-reported Health-related Quality of Life as Assessed by the KCCQ Score
Description
The Kansas City Cardiomyopathy Questionnaire (KCCQ) is a patient reported outcome instrument with minimum score = 0 and maximum score = 100 where higher score indicates better health status. There are no units to the score. The instrument utilizes a recall period of 2 weeks over which patients describe the frequency and severity of their symptoms, their physical and social limitations, and how they perceive their heart failure symptoms to affect their quality of life. The KCCQ clinical summary (KCCQ-CS) score, a prespecified secondary outcome of EXPLORER-HCM, combines the physical limitation and total symptom scores.
Time Frame
30 weeks
Title
Change From Baseline to Week 30 in Participant-reported Severity of HCM Symptoms as Assessed by the HCMSQ Score
Description
The Hypertrophic Cardiomyopathy Symptom Questionnaire (HCMSQ) is a patient reported outcome instrument that is a daily self-administered 11-item questionnaire. The HCMSQ assesses the core symptoms of HCM (tiredness/fatigue, heart palpitations, chest pain, dizziness, and shortness of breath). The Shortness of Breath domain score, a pre-specified secondary outcome of EXPLORER-HCM, assesses the frequency and severity of shortness of breath. The minimum score = 0 and maximum score = 18 where lower score indicates better health status. There are no units to the score.
Time Frame
30 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Key Inclusion Criteria: Age 18 and greater, body weight ≥ 45kg Has adequate acoustic windows to enable accurate transthoracic echocardiograms (TTEs) Diagnosed with oHCM consistent with current American College of Cardiology Foundation/American Heart Association and European Society of Cardiology guidelines and satisfy both criteria: Has documented left ventricular ejection fraction (LVEF) ≥55% NYHA Class II or III Has documented oxygen saturation at rest ≥90% at Screening Is able to perform an upright CPET and has a respiratory exchange ratio (RER) ≥1.0 at Screening per central reading Key Exclusion Criteria: Known infiltrative or storage disorder causing cardiac hypertrophy that mimics oHCM, such as Fabry disease, amyloidosis, or Noonan syndrome with LV hypertrophy History of syncope or sustained ventricular tachyarrhythmia with exercise within 6 months prior to Screening History of resuscitated sudden cardiac arrest (at any time) or known history of appropriate implantable cardioverter defibrillator (ICD) discharge for life-threatening ventricular arrhythmia within 6 months prior to Screening Paroxysmal, intermittent atrial fibrillation with atrial fibrillation present at Screening Persistent or permanent atrial fibrillation not on anticoagulation for at least 4 weeks prior to Screening and/or not adequately rate controlled within 6 months prior to Screening Treatment (within 14 days prior to Screening) or planned treatment during the study with disopyramide or ranolazine Treatment (within 14 days prior to Screening) or planned treatment during the study with a combination of β-blockers and calcium channel blockers LVOT gradient with Valsalva maneuver <30 mmHg at Screening Has been successfully treated with invasive septal reduction (surgical myectomy or percutaneous alcohol septal ablation [ASA]) within 6 months prior to Screening or plans to have either of these treatments during the study ICD placement within 2 months prior to Screening or planned ICD placement during the study Has a history or evidence of any other clinically significant disorder, condition, or disease that, in the opinion of the investigator, would pose a risk to participant safety or interfere with the study evaluation, procedures, or completion Prior treatment with cardiotoxic agents such as doxorubicin or similar
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Information Team
Organizational Affiliation
MyoKardia, Inc.
Official's Role
Study Director
Facility Information:
Facility Name
Mayo Clinic Arizona
City
Scottsdale
State/Province
Arizona
ZIP/Postal Code
85259
Country
United States
Facility Name
Cedars-Sinai Medical Center (Smidt Heart Institute)
City
Los Angeles
State/Province
California
ZIP/Postal Code
90048
Country
United States
Facility Name
UCSF School of Medicine
City
San Francisco
State/Province
California
ZIP/Postal Code
94143
Country
United States
Facility Name
Stanford University
City
Stanford
State/Province
California
ZIP/Postal Code
94305
Country
United States
Facility Name
Yale New Haven Hospital
City
New Haven
State/Province
Connecticut
ZIP/Postal Code
16511
Country
United States
Facility Name
Mayo Clinic Jacksonville - PPDS
City
Jacksonville
State/Province
Florida
ZIP/Postal Code
32224
Country
United States
Facility Name
Northwestern University
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Facility Name
University Of Iowa Hospitals And Clinics
City
Iowa City
State/Province
Iowa
ZIP/Postal Code
52242
Country
United States
Facility Name
Brigham and Women's Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
University of Michigan
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48109
Country
United States
Facility Name
Spectrum Health
City
Grand Rapids
State/Province
Michigan
ZIP/Postal Code
49512
Country
United States
Facility Name
Washington University School of Medicine
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
NYU Langone Medical Center
City
New York
State/Province
New York
ZIP/Postal Code
10017
Country
United States
Facility Name
Columbia University Medical Center
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Facility Name
Carolinas Medical Center
City
Charlotte
State/Province
North Carolina
ZIP/Postal Code
28203
Country
United States
Facility Name
Duke Cardiology at Southpoint
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Facility Name
Cincinnati Children's Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Facility Name
Oregon Health & Science University
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Facility Name
St. Luke's Cardiology Associates
City
Bethlehem
State/Province
Pennsylvania
ZIP/Postal Code
18018
Country
United States
Facility Name
University of Pennsylvania (Penn Heart and Vascular Center)
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
University of Pittsburgh Medical Center Presbyterian
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15213
Country
United States
Facility Name
Methodist University Hospital
City
Memphis
State/Province
Tennessee
ZIP/Postal Code
38163
Country
United States
Facility Name
University of Texas Southwestern Medical Center
City
Dallas
State/Province
Texas
ZIP/Postal Code
75235
Country
United States
Facility Name
Houston Methodist Hospital
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
University of Texas Houston Medical School
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
Intermountain Medical Center
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84107
Country
United States
Facility Name
University of Utah
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84132
Country
United States
Facility Name
University of Virginia Health System
City
Charlottesville
State/Province
Virginia
ZIP/Postal Code
22903
Country
United States
Facility Name
University of Washington Medical Center
City
Seattle
State/Province
Washington
ZIP/Postal Code
98195
Country
United States
Facility Name
UZ Antwerpen
City
Edegem
State/Province
Antwerpen
ZIP/Postal Code
2650
Country
Belgium
Facility Name
Onze-Lieve-Vrouwziekenhuis
City
Aalst
State/Province
Oost-Vlaanderen
ZIP/Postal Code
9300
Country
Belgium
Facility Name
Hôpital Erasme
City
Brussels
ZIP/Postal Code
1070
Country
Belgium
Facility Name
Institut Klinicke a Experimentalni Mediciny
City
Prague
Country
Czechia
Facility Name
Vseobecna fakultni nemocnice v Praze
City
Praha 2
ZIP/Postal Code
128 08
Country
Czechia
Facility Name
Aarhus Universitetshospital
City
Aarhus N
ZIP/Postal Code
8200
Country
Denmark
Facility Name
Bispebjerg Hospital
City
København NV
ZIP/Postal Code
2400
Country
Denmark
Facility Name
Odense Universitetshospital
City
Odense
ZIP/Postal Code
5000
Country
Denmark
Facility Name
CHRU Nantes
City
Nantes
State/Province
Loire-Atlantique
ZIP/Postal Code
44805
Country
France
Facility Name
Groupe Hospitalier Pitié Salpétrière
City
Paris
ZIP/Postal Code
75013
Country
France
Facility Name
Hôpital Européen Georges Pompidou
City
Paris
ZIP/Postal Code
75015
Country
France
Facility Name
Hôpital de Rangueil
City
Toulouse
ZIP/Postal Code
31403
Country
France
Facility Name
University Medicine Göttingen
City
Göttingen
State/Province
Neidersachsen
Country
Germany
Facility Name
Kerckhoff-Klinik-Forschungs-GmbH
City
Bad Nauheim
ZIP/Postal Code
61231
Country
Germany
Facility Name
Charité Campus Buch - Experimental and Clinical Research Center
City
Berlin
ZIP/Postal Code
13125
Country
Germany
Facility Name
Charité - Universitätsmedizin Berlin
City
Berlin
ZIP/Postal Code
13353
Country
Germany
Facility Name
Cardiologicum Dresden und Pirna
City
Dresden
ZIP/Postal Code
01277
Country
Germany
Facility Name
University Clinic Heidelberg - PPDS
City
Heidelberg
ZIP/Postal Code
69120
Country
Germany
Facility Name
Universitatsklinikum Schleswig-Holstein
City
Kiel
ZIP/Postal Code
24105
Country
Germany
Facility Name
Tel Aviv Sourasky Medical Center
City
Tel Aviv
State/Province
Tel-Aviv
ZIP/Postal Code
62431
Country
Israel
Facility Name
Barzilai Medical Center
City
Ashkelon
ZIP/Postal Code
78278
Country
Israel
Facility Name
Hadassah Medical Center PPDS -
City
Jerusalem
ZIP/Postal Code
91120
Country
Israel
Facility Name
Rabin Medical Center - PPDS
City
Petach Tikva
ZIP/Postal Code
49100
Country
Israel
Facility Name
The Chaim Sheba Medical Center - The Edmond and Lily Safra Children's Hospital
City
Ramat-Gan
ZIP/Postal Code
52621
Country
Israel
Facility Name
Kaplan Medical Center
City
Reẖovot
ZIP/Postal Code
76100
Country
Israel
Facility Name
ZIV Medical Center
City
Safed
ZIP/Postal Code
13100
Country
Israel
Facility Name
Azienda Ospedaliera Universitaria Careggi
City
Firenze
Country
Italy
Facility Name
Maastricht University Medical Center
City
Maastricht
State/Province
Limburg
ZIP/Postal Code
6229 HX
Country
Netherlands
Facility Name
Erasmus MC
City
Rotterdam
State/Province
Zuid-Holland
ZIP/Postal Code
3015GD
Country
Netherlands
Facility Name
Collegium Medicum Uniwersytetu Jagiellonskiego
City
Kraków
State/Province
Malopolskie
ZIP/Postal Code
31-501
Country
Poland
Facility Name
Kardio Klinika Brynów
City
Katowice
State/Province
Slaskie
Country
Poland
Facility Name
Szpital Kliniczny Przemienienia Panskiego Uniwesytetu Medycznego im. Karola Marcinkowskiego
City
Poznań
ZIP/Postal Code
61-848
Country
Poland
Facility Name
Instytut Kardiologii im Prymasa Tysiaclecia Kardynala Stefana Wyszynskiego
City
Warsaw
ZIP/Postal Code
04-628
Country
Poland
Facility Name
Hospital Garcia de Orta
City
Almada
ZIP/Postal Code
2805-267
Country
Portugal
Facility Name
Hospital da Luz
City
Lisboa
ZIP/Postal Code
1500-650
Country
Portugal
Facility Name
Hospital Universitario Virgen de La Arrixaca
City
El Palmar
State/Province
Murcia
ZIP/Postal Code
30120
Country
Spain
Facility Name
Hospital Universitario A Coruña
City
La Coruña
Country
Spain
Facility Name
Hospital Universitario Puerta de Hierro - Majadahonda
City
Madrid
Country
Spain
Facility Name
Hospital Universitario Ramon y Cajal
City
Madrid
Country
Spain
Facility Name
Hospital Universitario Virgen Macarena
City
Sevilla
ZIP/Postal Code
41009
Country
Spain
Facility Name
University Hospital of Wales
City
Cardiff
State/Province
South Glamergon
ZIP/Postal Code
CF14 4XW
Country
United Kingdom
Facility Name
St Bartholomew's Hospital
City
London
ZIP/Postal Code
W1G 8PH
Country
United Kingdom

12. IPD Sharing Statement

Citations:
PubMed Identifier
35902155
Citation
Nassif M, Fine JT, Dolan C, Reaney M, Addepalli P, Allen VD, Sehnert AJ, Gosch K, Spertus JA. Validation of the Kansas City Cardiomyopathy Questionnaire in Symptomatic Obstructive Hypertrophic Cardiomyopathy. JACC Heart Fail. 2022 Aug;10(8):531-539. doi: 10.1016/j.jchf.2022.03.002. Epub 2022 May 4.
Results Reference
derived
PubMed Identifier
35718845
Citation
Reaney M, Addepalli P, Allen V, Spertus JA, Dolan C, Sehnert AJ, Fine JT. Longitudinal Psychometric Analysis of the Hypertrophic Cardiomyopathy Symptom Questionnaire (HCMSQ) Using Outcomes from the Phase III EXPLORER-HCM Trial. Pharmacoecon Open. 2022 Jul;6(4):575-586. doi: 10.1007/s41669-022-00340-8. Epub 2022 Jun 20.
Results Reference
derived
PubMed Identifier
34915982
Citation
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Clinical Study to Evaluate Mavacamten (MYK-461) in Adults With Symptomatic Obstructive Hypertrophic Cardiomyopathy

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