A Study of Mavacamten in Adolescent Participants with Symptomatic Obstructive Hypertrophic Cardiomyopathy

Phase 3

Active, not recruiting

• Conditions: Symptomatic Obstructive Hypertrophic Cardiomyopathy

• Registration Number: 2023-505650-17-00
• Lead Sponsor: Bristol-Myers Squibb Services Unlimited Company

Brief Summary

To evaluate the effect of mavacamten compared to placebo on left ventricular outflow tract (LVOT) obstruction in adolescents with symptomatic obstructive HCM

Detailed Description

Not available

Study Timeline

• Study First Posted: 2024-04-25
• Last Update Posted: 2024-12-19

Recruitment & Eligibility

• Status: Ongoing, recruitment ended
• Sex: Not specified
• Target Recruitment: 11

Inclusion Criteria

Adolescents aged 12 to <18 at the time of agreeing to participate

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)

Study & Design

• Study Type: Not specified
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Change from baseline in Valsalva LVOT gradient at Week 28		Change from baseline in Valsalva LVOT gradient at Week 28

Secondary Outcome Measures

Name	Time	Method
Change from baseline in the following at Week 28: 1) Resting LVOT gradient 2) Post-exercise peak LVOT gradient 3) Maximal wall thickness 4) Ratio between early mitral inflow velocity and mitral annular early diastolic velocity (E/e’)		Change from baseline in the following at Week 28: 1) Resting LVOT gradient 2) Post-exercise peak LVOT gradient 3) Maximal wall thickness 4) Ratio between early mitral inflow velocity and mitral annular early diastolic velocity (E/e’)
Proportion of participants achieving an increase from baseline to Week 28 in peak oxygen uptake test (pVO2)		Proportion of participants achieving an increase from baseline to Week 28 in peak oxygen uptake test (pVO2)
Proportion of participants achieving a reduction from baseline to Week 28 in maximal LVOT gradient to < 30 mmHg		Proportion of participants achieving a reduction from baseline to Week 28 in maximal LVOT gradient to < 30 mmHg
Proportion of participants with at least 1 class improvement in New York Heart Association (NYHA) class from baseline to Week 28		Proportion of participants with at least 1 class improvement in New York Heart Association (NYHA) class from baseline to Week 28
Proportion of participants with at least 1 grade improvement in mitral regurgitation at Week 28 Incidence of treatment emergent adverse events (TEAEs) and treatment emergent serious adverse events (TESAEs)		Proportion of participants with at least 1 grade improvement in mitral regurgitation at Week 28 Incidence of treatment emergent adverse events (TEAEs) and treatment emergent serious adverse events (TESAEs)
Change from baseline in QT interval on electrocardiogram at Week 28		Change from baseline in QT interval on electrocardiogram at Week 28
Incidence of left ventricular ejection fraction (LVEF) ≤ 30%		Incidence of left ventricular ejection fraction (LVEF) ≤ 30%
Incidence of LVEF < 50%		Incidence of LVEF < 50%
Summary of plasma concentrations by visit (Ctrough and post dose)		Summary of plasma concentrations by visit (Ctrough and post dose)
Cmax and AUC using sparse sampling utilizing population PK approach		Cmax and AUC using sparse sampling utilizing population PK approach
Proportion of participants who evaluate taste and swallowability as neutral or better using taste and swallowability scales at Day 1 and Week 11		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 HCMSQ SoB domain at Week 28		Change from baseline in the HCMSQ SoB domain at Week 28

Trial Locations

Locations (11)

Hospital General Universitario Gregorio Maranon; 🇪🇸 Madrid, Spain

Hospital Universitario La Paz; 🇪🇸 Madrid, Spain

Sant Joan De Deu Barcelona Hospital; 🇪🇸 Esplugues De Llobregat, Spain

Deutsches Herzzentrum Muenchen Des Freistaates Bayern Klinik An Der Technischen Universitaet Muenchen; 🇩🇪 Munich, Germany

Charite Universitaetsmedizin Berlin KöR; 🇩🇪 Berlin, Germany

Children's Health Ireland; 🇮🇪 Dublin, Ireland

Giannina Gaslini Institute For Scientific Hospitalization And Care; 🇮🇹 Genoa, Italy

Azienda Ospedaliera Dei Colli; 🇮🇹 Naples, Italy

Azienda Ospedaliera Universitaria Meyer IRCCS; 🇮🇹 Florence, Italy

Centre Hospitalier Universitaire De Bordeaux; 🇫🇷 Pessac, France

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Hospital General Universitario Gregorio Maranon

🇪🇸Madrid, Spain

Patricia Alonso Rivero

Site contact

34695140034

palonsori@gmail.com