Mavacamten / Non-mavacamten symptomatic oHCM therapy / Obstructive Hypertrophic Cardiomyopathy · Observational Study

A Prospective Registry Study to Assess Real-world Patient Characteristics, Treatment Patterns, and Longitudinal Outcomes in Patients Receiving Mavacamten and Other Treatments for Symptomatic Obstructive Hypertrophic Cardiomyopathy (Obstructive-HCM)

ClinicalTrials.gov · August 19, 2026

Preliminary Why this matters to you Expert discussion

Early or partial results. Treat as a signal, not a conclusion.

AI Summary

This is a prospective observational registry study currently recruiting patients with symptomatic obstructive HCM to characterize real-world use of mavacamten and other treatments. No results are posted in this registry record; the study aims to evaluate safety (incidence of new or worsening heart failure with systolic dysfunction) and effectiveness (change in LVOT gradient) over up to 5 years of follow-up.

What was studied

Observational. Obstructive Hypertrophic Cardiomyopathy; age from 18 Years. Intervention: Mavacamten; Beta-blocker (BB) / non-dihydropyridine (non-DHP) calcium c…. Compared with: Non-mavacamten symptomatic oHCM therapy and no oHCM treatment. n = 1,700. 101 sites across 9 countries.

Key findings

Safety

Safety was not reported in the material analysed. Check the source before drawing any conclusion about harm.

Who this may help

When results become available, this registry will provide real-world evidence on safety and effectiveness of mavacamten compared to other treatments in symptomatic obstructive HCM. Clinicians should await completion and publication before drawing conclusions.

Plain-language summary

This is an ongoing observational registry study with no results yet reported; it is designed to generate real-world safety and effectiveness data but carries no outcome evidence at this stage.

Study design

As stated by the source record.

MethodsCohort model. Prospective time perspective. Primary outcome measures: Incidence rate of new or worsening heart failure due to systolic dysfunction among participants with symptomatic obstructive HCM during periods of exposure to mavacamten and other… (assessed at Up to 5 Years); Change in resting left ventricular outflow tract (LVOT) gradient from baseline (assessed at Up to 18 months)

DesignObservational

PopulationObstructive Hypertrophic Cardiomyopathy; age from 18 Years

Sample size1,700

InterventionMavacamten; Beta-blocker (BB) / non-dihydropyridine (non-DHP) calcium c…

ComparatorNon-mavacamten symptomatic oHCM therapy and no oHCM treatment

Setting101 sites across 9 countries

Reported figures

Quoted from the source exactly as published.

Planned enrollment1700

Primary outcome 1Incidence rate of new or worsening heart failure due to systolic dysfunction, up to 5 years

Primary outcome 2Change in resting LVOT gradient from baseline, up to 18 months

Clinical bottom line

Caveats

This is a registry record only; no results are posted or reported here.
No sample size breakdown by treatment group is provided.
Study is still recruiting; completion date and results timeline are not stated.
Observational design limits causal inference; real-world confounding and selection bias cannot be ruled out from this record.

Evidence assessment

Graded across the dimensions that decide whether you should act, each from what the source actually supports. There is no single score, and where a dimension was not assessed it says so.

Study design

moderate

Prospective observational registry; no randomization or blinding; real-world setting.

Directness

strong

Directly measures safety (heart failure incidence) and effectiveness (LVOT gradient change) in symptomatic obstructive HCM.

Consistency

insufficient

No results reported; consistency cannot be assessed from a registry record.

Risk of bias

limited

Observational design; no control for confounding; treatment selection is non-randomized and real-world.

Sample adequacy

moderate

Planned enrollment 1700 participants across United States and Europe; adequate for real-world registry but study is still recruiting.

What is missing. This record has no key findings. That is a gap in the analysis, not a judgement about the study.

Source abstract

Registry record from ClinicalTrials.gov (NCT05489705). This is a study registration, not published results. Lead sponsor: Bristol-Myers Squibb. Recruitment status: RECRUITING. Study type: OBSERVATIONAL. Enrollment: 1700 participants (ESTIMATED). Conditions: Obstructive Hypertrophic Cardiomyopathy. Interventions: DRUG: Mavacamten; DRUG: Non-mavacamten symptomatic oHCM therapy. Primary outcome measures: Incidence rate of new or worsening heart failure due to systolic dysfunction among participants with symptomatic obstructive HCM during periods of exposure to mavacamten and other oHCM treatment , Up to 5 Years; Change in resting left ventricular outflow tract (LVOT) gradient from baseline , Up to 18 months. Brief summary: This registry evaluates patient characteristics, real-world treatment patterns, and short- and long-term outcomes in a population of patients in the United States and Europe with symptomatic obstructive hypertrophic cardiomyopathy (HCM) who are receiving mavacamten, receiving other treatment for obstructive HCM, or not receiving treatment for obstructive HCM due to intolerance or failure of prior treatment. United States Sub-Study: The purpose of this study is to evaluate the safety of mavacamten in patients with symptomatic obstructive HCM in the real-world setting. Europe Sub-Study: The purpose of this study is to evaluate the effectiveness and safety of mavacamten in patients with symptomatic obstructive HCM in the real-world setting.

Follow from this

Taken from the source record, never inferred. Follow any of these and new work involving them reaches your briefing.

MavacamtenNon-mavacamten symptomatic oHCM therapyObstructive Hypertrophic CardiomyopathyNCT05489705Bristol-Myers Squibb