Cardiomyopathy, Hypertrophic Clinical Trial
Official title:
A Randomized, Double-blind, Placebo-controlled Clinical Study to Evaluate Mavacamten in Adolescents (Age 12 Years to < 18 Years) With Symptomatic Obstructive Hypertrophic Cardiomyopathy
The purpose of this study is to evaluate the efficacy, safety, and pharmacokinetics of mavacamten in adolescent patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM).
Status | Recruiting |
Enrollment | 40 |
Est. completion date | March 29, 2030 |
Est. primary completion date | February 15, 2028 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 12 Years to 17 Years |
Eligibility | Inclusion Criteria: - Diagnosis of HCM - Presence of LVOT obstruction - Presence of symptoms Exclusion Criteria: - Phenocopy diseases resulting in myocardial hypertrophy not related to sarcomere dysfunction - Evidence of LVEF <50% in prior 6 months - Planned escalation in HCM therapy or upcoming intervention (eg, major cardiac surgery, HCM medication dose increase) Other protocol-defined Inclusion/Exclusion criteria apply. |
Country | Name | City | State |
---|---|---|---|
Australia | Local Institution - 0042 | Clayton | Victoria |
Australia | Local Institution - 0028 | Parkville | Victoria |
Australia | Local Institution - 0020 | Sydney | New South Wales |
Canada | Local Institution - 0041 | Edmonton | Alberta |
Canada | Local Institution - 0046 | Toronto | Ontario |
France | Local Institution - 0022 | Paris | |
France | Local Institution - 0026 | Pessac | |
Germany | Local Institution - 0018 | Berlin | BE |
Germany | Local Institution - 0006 | Munich | BY |
Ireland | Local Institution - 0047 | Drimnagh | D |
Italy | Local Institution - 0016 | Firenze | FI |
Italy | Local Institution - 0051 | Genova | GE |
Italy | Local Institution - 0027 | Napoli | |
Spain | Local Institution - 0011 | A Coruna | |
Spain | Local Institution - 0014 | Barcelona | B |
Spain | Local Institution - 0021 | Granada | GR |
Spain | Local Institution - 0023 | Madrid | |
Spain | Local Institution - 0025 | Madrid | |
Spain | Local Institution - 0048 | Madrid | M |
United Kingdom | Local Institution - 0049 | Belfast | BFS |
United Kingdom | Local Institution - 0007 | London | |
United States | University of Michigan - C.S. Mott Children's Hospital | Ann Arbor | Michigan |
United States | Children's Healthcare of Atlanta Foundation, Inc. | Atlanta | Georgia |
United States | Local Institution - 0038 | Aurora | Colorado |
United States | Local Institution - 0045 | Austin | Texas |
United States | Local Institution - 0017 | Birmingham | Alabama |
United States | Local Institution - 0043 | Boston | Massachusetts |
United States | Local Institution - 0040 | Bronx | New York |
United States | Local Institution - 0015 | Charlotte | North Carolina |
United States | University Of Virginia School of Medicine | Charlottesville | Virginia |
United States | Local Institution - 0013 | Chicago | Illinois |
United States | Local Institution - 0019 | Cincinnati | Ohio |
United States | Local Institution - 0001 | Cleveland | Ohio |
United States | Local Institution - 0029 | Columbus | Ohio |
United States | Local Institution - 0055 | Dallas | Texas |
United States | Local Institution - 0039 | Durham | North Carolina |
United States | Local Institution - 0054 | Houston | Texas |
United States | Riley Hospital for Children - Pediatric Cardiology | Indianapolis | Indiana |
United States | Local Institution - 0008 | Los Angeles | California |
United States | Local Institution - 0032 | Los Angeles | California |
United States | Local Institution - 0035 | Madison | Wisconsin |
United States | Le Bonheur Children's Hospital | Memphis | Tennessee |
United States | Local Institution - 0052 | Morristown | New Jersey |
United States | Cohen Children's Medical Center of New York | New Hyde Park | New York |
United States | Icahn School of Medicine at Mount Sinai | New York | New York |
United States | Local Institution - 0036 | New York | New York |
United States | Local Institution - 0004 | Oakland | California |
United States | Local Institution - 0033 | Palo Alto | California |
United States | The Children's Hospital of Philadelphia (CHOP) - Lipid Heart Clinic - Main Campus | Philadelphia | Pennsylvania |
United States | Phoenix Children's Cardiology | Phoenix | Arizona |
United States | UPMC Childrens Hospital of Pittsburgh | Pittsburgh | Pennsylvania |
United States | Washington University School of Medicine in St. Louis - St. Louis Children's Hospital | Saint Louis | Missouri |
United States | Local Institution - 0031 | Saint Petersburg | Florida |
United States | Local Institution - 0003 | Salt Lake City | Utah |
United States | Local Institution - 0044 | San Diego | California |
Lead Sponsor | Collaborator |
---|---|
Bristol-Myers Squibb |
United States, Australia, Canada, France, Germany, Ireland, Italy, Spain, United Kingdom,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Change from baseline in Valsalva left ventricular outflow tract (LVOT) (VLVOT) gradient | At Week 28 | ||
Secondary | Change from baseline in resting LVOT gradient | At Week 28 | ||
Secondary | Change from baseline in post-exercise peak LVOT gradient | At Week 28 | ||
Secondary | Change from baseline in maximal wall thickness | At Week 28 | ||
Secondary | Change from baseline in ratio between early mitral inflow velocity and mitral annular early diastolic velocity (E/e') | At Week 28 | ||
Secondary | Proportion of participants achieving an increase from baseline to Week 28 in peak oxygen uptake test (pVO2) | From baseline up to Week 28 | ||
Secondary | Proportion of participants achieving a reduction from baseline to Week 28 in maximal LVOT gradient to < 30 mmHg | From baseline up to Week 28 | ||
Secondary | Proportion of participants with at least 1 class improvement in New York Heart Association (NYHA) class from baseline to Week 28 | From baseline up to Week 28 | ||
Secondary | Proportion of participants with at least 1 grade improvement in mitral regurgitation at Week 28 | From baseline up to Week 28 | ||
Secondary | Number of participants with treatment-emergent adverse events (TEAEs) | Up to Week 74 | ||
Secondary | Number of participants with treatment-emergent serious adverse events (TESAEs) | Up to Week 74 | ||
Secondary | Change from baseline in electrocardiogram (ECG) (QT interval) | At Week 28 | ||
Secondary | Number of participants with left ventricular ejection fraction (LVEF) = 30% | Up to Week 56 | ||
Secondary | Number of participants with LVEF = 50% | Up to Week 56 | ||
Secondary | Trough observed plasma concentration (Ctrough) | Up to Week 56 | ||
Secondary | Post-dose plasma concentration of mavacamten | Up to Week 56 | ||
Secondary | Maximum observed concentration (Cmax) | Up to Week 56 | ||
Secondary | Area under the concentration-time curve (AUC) | Up to Week 56 | ||
Secondary | Proportion of participants who evaluate taste and swallowability as neutral or better using taste and swallowability scales | At Day 1 and Week 11 | ||
Secondary | Change from baseline in the Hypertrophic Cardiomyopathy Symptom Questionnaire - Shortness of Breath (HCMSQ SoB) domain | At Week 28 |
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