A Study to Assess the Real-World Effectiveness of Mavacamten in Adult Patients With Obstructive Hypertrophic Cardiomyopathy in China

Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age18+
SponsorBristol-Myers Squibb

About this trial

The purpose of this study is to assess the effectiveness of mavacamten treatment in Chinese adults with symptomatic obstructive hypertrophic cardiomyopathy (HCM) in real-world clinical practice

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Participants aged ≥ 18 years (participants enrolled retrospectively: at the time of initial mavacamten prescription), irrespective of gender.

Participants who have initiated mavacamten (for whom enrolled retrospectively) or are scheduled to initiate mavacamten (for whom enrolled prospectively) based on clinical therapeutic needs.

Has a documented diagnosis of HCM prior to enrollment, and

Peak left ventricular outflow tract (LVOT) gradient ≥ 30 mmHg at rest or with provocation in the most recent medical record within 3 months prior to enrollment as assessed by echocardiography.

Disqualifiers

Known HCM phenocopy disease (e.g., Fabry disease, amyloidosis).

Participants who are expected to undergo major cardiac surgery during the study.

Prior treatment of obstructive HCM with invasive septal reduction (surgical myectomy or percutaneous alcohol septal ablation [ASA] or septal radiofrequency ablation) within 6 months prior to enrollment (participants enrolled retrospectively: within 6 months prior to initial mavacamten prescription); participants with an unsuccessful myectomy or percutaneous ASA or septal radiofrequency ablation performed >6 months prior to enrollment (participants enrolled retrospectively: within 6 months prior to initial mavacamten prescription) may be enrolled.

Currently treated with disopyramide or ranolazine (within 14 days prior to enrollment [participants enrolled retrospectively: within 14 days prior to initial mavacamten prescription]) or participants who are expected to be taking disopyramide, ranolazine, verapamil in combination with β-receptor blockers, or diltiazem in combination with β-receptor blockers during the study.

Trial population

The study population will consist of adults diagnosed with symptomatic obstructive hypertrophic cardiomyopathy that have received mavacamten treatment in China

Trial design

Design model

Cohort

Time perspective

Other

Treatments tested in this trial

  • Mavacamten

    Drug

    According to the product label

Treatment groups

500 Participants
are divided into 1 treatment group
Group A: Cohort 11 intervention

Trial outcomes

Primary outcomes

1

Change from baseline in Valsalva left ventricular outflow tract (LVOT) gradient

Time frame
Week 48, Week 96
2

Change from baseline in resting valsalva left ventricular outflow tract (LVOT) gradient

Time frame
Week 48, Week 96

Secondary outcomes

1

Change from baseline in New York Heart Association (NYHA) class

Time frame
Week 4, Week 8, Week 12, Week 24, Week 36, Week 48, Week 60, Week 72, Week 84, and Week 96
2

Number and proportion of participants with a resting/provoked left ventricular outflow tract (LVOT) gradient < 30/50 mmHg

Time frame
Week 12, Week 36, Week 48, Week 72, and Week 96
3

Number and proportion of participants achieving complete response (defined as all left ventricular outflow tract (LVOT) gradients < 30 mmHg and New York Heart Association (NYHA) Class I)

Time frame
Week 12, Week 36, Week 48, Week 72, and Week 96

Other outcomes

Sponsors and contacts

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