A Study to Evaluate Mavacamten in Adolescents With Symptomatic Obstructive Hypertrophic Cardiomyopathy
- Registration Number
- NCT06253221
- Lead Sponsor
- Bristol-Myers Squibb
- Brief Summary
The purpose of this study is to evaluate the efficacy, safety, and pharmacokinetics of mavacamten in adolescent patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 40
- Diagnosis of HCM
- Presence of LVOT obstruction
- Presence of symptoms
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Placebo Placebo Participants assigned to this arm will receive mavacamten (1 mg to 15 mg) from week 28 to end of treatment at week 200. Mavacamten Mavacamten Participants assigned to this arm will receive mavacamten (1 mg to 15 mg) from day 1 to end of treatment at week 200.
- Primary Outcome Measures
Name Time Method Change from baseline in Valsalva left ventricular outflow tract (LVOT) (VLVOT) gradient At Week 28
- Secondary Outcome Measures
Name Time Method Change from baseline in resting LVOT gradient At Week 28 Change from baseline in post-exercise peak LVOT gradient At Week 28 Change from baseline in maximal wall thickness At Week 28 Change from baseline in ratio between early mitral inflow velocity and mitral annular early diastolic velocity (E/e') At Week 28 Proportion of participants achieving an increase from baseline to Week 28 in peak oxygen uptake test (pVO2) From baseline up to Week 28 Proportion of participants achieving a reduction from baseline to Week 28 in maximal LVOT gradient to < 30 mmHg From baseline up to Week 28 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 Proportion of participants with at least 1 grade improvement in mitral regurgitation at Week 28 From baseline up to Week 28 Number of participants with treatment-emergent adverse events (TEAEs) Up to Week 218 Number of participants with treatment-emergent serious adverse events (TESAEs) Up to Week 218 Change from baseline in electrocardiogram (ECG) (QT interval) At Week 28 Number of participants with left ventricular ejection fraction (LVEF) ≤ 30% Up to Week 200 Number of participants with LVEF < 50% Up to Week 200 Trough observed plasma concentration (Ctrough) Up to Week 200 Post-dose plasma concentration of mavacamten Up to Week 200 Maximum observed concentration (Cmax) Up to Week 200 Area under the concentration-time curve (AUC) Up to Week 200 Proportion of participants who evaluate taste and swallowability as neutral or better using taste and swallowability scales At Day 1 and Week 11 Change from baseline in the Hypertrophic Cardiomyopathy Symptom Questionnaire - Shortness of Breath (HCMSQ SoB) domain At Week 28
Trial Locations
- Locations (55)
Rady Children's Hospital-San Diego
🇺🇸San Diego, California, United States
Children's Hospital of Philadelphia-Center for Childhood Cancer Research
🇺🇸Philadelphia, Pennsylvania, United States
The Hospital for Sick Children (SickKids)
🇨🇦Toronto, Ontario, Canada
Royal Belfast Hospital for Sick Children
🇬🇧Belfast, BFS, United Kingdom
The University of Alabama at Birmingham
🇺🇸Birmingham, Alabama, United States
Phoenix Children's Cardiology
🇺🇸Phoenix, Arizona, United States
Children's Hospital Los Angeles
🇺🇸Los Angeles, California, United States
University of California Los Angeles (UCLA) - Mattel Children's Hospital
🇺🇸Los Angeles, California, United States
Local Institution - 0004
🇺🇸Oakland, California, United States
Lucile Packard Children's Hospital (LPCH)
🇺🇸Palo Alto, California, United States
Children's Hospital Colorado
🇺🇸Aurora, Colorado, United States
Johns Hopkins All Children's Hospital
🇺🇸Saint Petersburg, Florida, United States
Children's Healthcare of Atlanta - Arthur M. Blank Hospital
🇺🇸Atlanta, Georgia, United States
The University of Chicago Medical Center
🇺🇸Chicago, Illinois, United States
Riley Hospital for Children - Pediatric Cardiology
🇺🇸Indianapolis, Indiana, United States
Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
University of Michigan - C.S. Mott Children's Hospital
🇺🇸Ann Arbor, Michigan, United States
Washington University School of Medicine in St. Louis - St. Louis Children's Hospital
🇺🇸Saint Louis, Missouri, United States
Atlantic Medical Group Hypertrophic Cardiomyopathy Associates
🇺🇸Morristown, New Jersey, United States
The Children's Hospital at Montefiore
🇺🇸Bronx, New York, 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
Morgan Stanley Children's Hospital of NewYork-Presbyterian
🇺🇸New York, New York, United States
Sanger Heart & Vascular Institute Heart Failure & Transplant Kenilworth
🇺🇸Charlotte, North Carolina, United States
Duke University Hospital
🇺🇸Durham, North Carolina, United States
Cincinnati Children's Hospital Medical Center
🇺🇸Cincinnati, Ohio, United States
Cleveland Clinic
🇺🇸Cleveland, Ohio, United States
Nationwide Children's Hospital
🇺🇸Columbus, Ohio, United States
UPMC Childrens Hospital of Pittsburgh
🇺🇸Pittsburgh, Pennsylvania, United States
Le Bonheur Children's Hospital
🇺🇸Memphis, Tennessee, United States
Dell Children's Medical Center of Central Texas
🇺🇸Austin, Texas, United States
Local Institution - 0055
🇺🇸Dallas, Texas, United States
Texas Children's Hospital
🇺🇸Houston, Texas, United States
Primary Children's Hospital
🇺🇸Salt Lake City, Utah, United States
University Of Virginia School of Medicine
🇺🇸Charlottesville, Virginia, United States
Local Institution - 0035
🇺🇸Madison, Wisconsin, United States
The Children's Hospital at Westmead
🇦🇺Sydney, New South Wales, Australia
Monash Medical Centre Clayton
🇦🇺Clayton, Victoria, Australia
Local Institution - 0028
🇦🇺Parkville, Victoria, Australia
Stollery Children's Hospital
🇨🇦Edmonton, Alberta, Canada
Hôpital Necker - Enfants Malades
🇫🇷Paris, France
CHU Hopitaux de Bordeaux - Hopital Haut-Leveque
🇫🇷Pessac, France
Charite - UB - CVK - Medizinische Klinik
🇩🇪Berlin, BE, Germany
Herzzentrum München
🇩🇪Munich, BY, Germany
Children's Health Ireland
🇮🇪Dublin 12, D12 N512, Ireland
Azienda Ospedaliero Universitaria Meyer
🇮🇹Firenze, FI, Italy
Istituto Giannina Gaslini
🇮🇹Genova, GE, Italy
AORN - Ospedali dei Colli Ospedale V. Monaldi
🇮🇹Napoli, Italy
Hospital Sant Joan de Déu Barcelona
🇪🇸Barcelona, B, Spain
Local Institution - 0021
🇪🇸Granada, GR, Spain
Local Institution - 0048
🇪🇸Madrid, M, Spain
Local Institution - 0011
🇪🇸A Coruna, Spain
Local Institution - 0023
🇪🇸Madrid, Spain
Hospital Universitario La Paz
🇪🇸Madrid, Spain
Great Ormond Street Hospital for Children NHS Foundation Trust - Great Ormond Street Hospital
🇬🇧London, United Kingdom