NCT07486752

BAG3-DCM Natural History Study

An Observational Study of Patients With Dilated Cardiomyopathy (DCM) Associated With Pathogenic BAG3 Variants

• Status: Not Yet Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 30 (estimated)
• Sponsor: Rocket Pharmaceuticals Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The goal of this international observational study is to learn about the natural history of Dilated Cardiomyopathy (DCM) arising from pathogenic BAG3 variants in adult patients ≥18 years of age.

Detailed description

This is an observational study with both retrospective and prospective data collection. The study is designed to describe the natural history of BAG3-DCM including the signs and symptoms, key clinical events, and impact of the disease on quality of life as managed with the current standard of care. A hybrid (retrospective and prospective data collection) approach is being used to generate robust and longitudinal data.

Conditions

• Dilated Cardiomyopathy (DCM)
• Cardiovascular Diseases
• Heart Diseases
• Genetic Diseases

Timeline

• Start date: 2026-05-01
• Primary completion: 2032-12-01
• Completion: 2033-03-01
• First posted: 2026-03-20
• Last updated: 2026-03-20