Clinical trial NCT03723655: A Long-Term Safety Extension Study of Mavacamten in Adults Who Have Completed MAVERICK-HCM or EXPLORER-HCM
Official title: A Long-Term Safety Extension Study of Mavacamten (MYK-461) in Adults With Hypertrophic Cardiomyopathy Who Have Completed the MAVERICK-HCM (MYK-461-006) or EXPLORER-HCM (MYK-461-005) Trials (MAVA-LTE)
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE2
PHASE3
Conditions: Hypertrophic Cardiomyopathy
Obstructive Hypertrophic Cardiomyopathy
Non-obstructive Hypertrophic Cardiomyopathy
Keywords: Symptomatic, left ventricular outflow tract gradient
Lead sponsor: Bristol-Myers Squibb
Sponsor class: INDUSTRY
First posted: 2018-10-29
Enrollment: 282
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: Approximately 30 sites that enrolled participants in the MAVERICK-HCM (MYK-461-006) study in the United States (US) will initiate this study.

Approximately 90 sites that enrolled participants in the EXPLORER-HCM (MYK-461-005) study in the US, Europe, and Israel will initiate this study.

Note: Approximately 30 centers overlap between MAVERICK and EXPLORER.


Eligibility criteria: Key Inclusion Criteria:

* Has completed the Parent Study through to the EOS Visit within 90 days of consent. (Participants who are beyond the 90-day window from EOS Visit may be included in this study pending MyoKardia Medical Monitoring approval) Participants who prematurely discontinued from the Parent Study or the MAVA-LTE study may be considered for inclusion.
* Has a body weight greater than 45 kg at the Screening Visit
* Has adequate acoustic windows to enable accurate TTEs.
* Has documented LVEF ≥ 50% by echocardiography core laboratory read of screening TTE at rest.
* Has safety laboratory parameters (chemistry, hematology, coagulation, and urinalysis) within normal limits (according to the central laboratory reference range).
* Female participants must not be pregnant or lactating and, if sexually active, must use one of the following highly effective birth control methods from the Screening Visit through 90 days after the last dose of investigational medicinal product (IMP).

In addition to the above contraceptive requirements for female participants, male partners must also use a contraceptive (eg. barrier, condom, or vasectomy).

Key Exclusion Criteria:

* Has any ECG abnormality considered by the investigator to pose a risk to participant safety (eg. second degree atrioventricular block type II).
* Has a history of syncope or a history of sustained ventricular tachyarrhythmia with exercise between Parent Study EOS Visit and Screening Visit.
* Has a history of resuscitated sudden cardiac arrest or known history of appropriate implantable cardioverter-defibrillator (ICD) discharge for life-threatening ventricular arrhythmia between Parent Study EOS Visit and Screening Visit. (Note: history of anti-tachycardia pacing (ATP) is allowed).• Currently treated with disopyramide or ranolazine (within 14 days prior to Screening) or treatment with disopyramide or ranolazine is planned during the study.
* Has any acute or serious comorbid condition (eg. major infection or hematologic, renal, metabolic, gastrointestinal, or endocrine dysfunction) that, in the judgment of the Investigator, could lead to premature termination of study participation or interfere with the measurement or interpretation of the efficacy and safety assessments in the study.
* History of clinically significant malignant disease that developed since enrollment in the Parent Study.
* Is unable to comply with the study requirements, including the number of required visits to the clinical site.
* Has participated in a clinical trial in which the participant received any investigational drug (or is currently using an investigational device) within 30 days prior to Screening, or at least 5 times the respective elimination half-life (whichever is longer), except for participation in MAVERICK-HCM or EXPLORER-HCM. Prior participation in a non-interventional observational study is allowed.

Primary outcomes:
measure: Frequency and severity of treatment-emergent adverse events and serious adverse events
timeFrame: 252 weeks
Secondary outcomes:
Other outcomes:
Locations:
city: Scottsdale
country: United States
facility: Local Institution - 0045
state: Arizona
city: Los Angeles
country: United States
facility: Local Institution - 0058
state: California
city: San Francisco
country: United States
facility: Local Institution - 0066
state: California
city: Stanford
country: United States
facility: Local Institution - 0063
state: California
city: New Haven
country: United States
facility: Local Institution - 0043
state: Connecticut
city: Jacksonville
country: United States
facility: Local Institution - 0067
state: Florida
city: Chicago
country: United States
facility: Local Institution - 0050
state: Illinois
city: Indianapolis
country: United States
facility: Local Institution - 0070
state: Indiana
city: Iowa City
country: United States
facility: Local Institution - 0049
state: Iowa
city: Boston
country: United States
facility: Local Institution - 0054
state: Massachusetts
city: Ann Arbor
country: United States
facility: Local Institution - 0051
state: Michigan
city: Grand Rapids
country: United States
facility: Local Institution - 0074
state: Michigan
city: Saint Louis
country: United States
facility: Local Institution - 0048
state: Missouri
city: New York
country: United States
facility: Local Institution - 0060
state: New York
city: New York
country: United States
facility: Local Institution - 0056
state: New York
city: Charlotte
country: United States
facility: Local Institution - 0071
state: North Carolina
city: Durham
country: United States
facility: Local Institution - 0047
state: North Carolina
city: Cincinnati
country: United States
facility: Local Institution - 0052
state: Ohio
city: Portland
country: United States
facility: Local Institution - 0044
state: Oregon
city: Bethlehem
country: United States
facility: Local Institution - 0068
state: Pennsylvania
city: Philadelphia
country: United States
facility: Local Institution - 0046
state: Pennsylvania
city: Pittsburgh
country: United States
facility: Local Institution - 0064
state: Pennsylvania
city: Memphis
country: United States
facility: Local Institution - 0073
state: Tennessee
city: Dallas
country: United States
facility: Local Institution - 0061
state: Texas
city: Houston
country: United States
facility: Local Institution - 0055
state: Texas
city: Houston
country: United States
facility: Local Institution - 0057
state: Texas
city: Murray
country: United States
facility: Local Institution - 0072
state: Utah
city: Salt Lake City
country: United States
facility: Local Institution - 0062
state: Utah
city: Charlottesville
country: United States
facility: Local Institution - 0053
state: Virginia
city: Richmond
country: United States
facility: Local Institution - 0059
state: Virginia
city: Seattle
country: United States
facility: Local Institution - 0065
state: Washington
city: Aalst
country: Belgium
facility: Local Institution - 0002
city: Brussels
country: Belgium
facility: Local Institution - 0001
city: Edegem
country: Belgium
facility: Local Institution - 0003
city: Prague
country: Czechia
facility: Local Institution - 0009
city: Praha 2
country: Czechia
facility: Local Institution - 0010
city: Aarhus
country: Denmark
facility: Local Institution - 0011
city: Frederiksberg
country: Denmark
facility: Local Institution - 0012
city: Odense
country: Denmark
facility: Local Institution - 0013
city: Nantes
country: France
facility: Local Institution - 0014
city: Paris
country: France
facility: Local Institution - 0017
city: Paris
country: France
facility: Local Institution - 0016
city: Toulouse
country: France
facility: Local Institution - 0015
city: Bad Nauheim
country: Germany
facility: Local Institution - 0020
city: Dresden
country: Germany
facility: Local Institution - 0018
city: Goettingen
country: Germany
facility: Local Institution - 0019
city: Heidelberg
country: Germany
facility: Local Institution - 0021
city: Jerusalem
country: Israel
facility: Local Institution - 0027
city: Petah Tikva
country: Israel
facility: Local Institution - 0024
city: Ramat-gan
country: Israel
facility: Local Institution - 0022
city: Rehovot
country: Israel
facility: Local Institution - 0025
city: Safed
country: Israel
facility: Local Institution - 0023
city: Tel Aviv
country: Israel
facility: Local Institution - 0026
city: Firenze
country: Italy
facility: Local Institution - 0028
city: Maastricht
country: Netherlands
facility: Local Institution - 0029
city: Rotterdam
country: Netherlands
facility: Local Institution - 0030
city: Katowice
country: Poland
facility: Local Institution - 0033
city: Krakow
country: Poland
facility: Local Institution - 0034
city: Poznan
country: Poland
facility: Local Institution - 0031
city: Warsaw
country: Poland
facility: Local Institution - 0032
city: Almada
country: Portugal
facility: Local Institution - 0036
city: Lisboa
country: Portugal
facility: Local Institution - 0035
city: A Coruña
country: Spain
facility: Local Institution - 0038
city: El Palmar
country: Spain
facility: Local Institution - 0037
city: Madrid
country: Spain
facility: Local Institution - 0040
city: Majadahonda
country: Spain
facility: Local Institution - 0039
city: Sevilla
country: Spain
facility: Local Institution - 0041
city: London
country: United Kingdom
facility: Local Institution - 0042
Study officials:
affiliation: Bristol-Myers Squibb
name: Bristol-Myers Squibb
role: STUDY_DIRECTOR
References: