Actively Recruiting
Phase 12a Open-Label Study of 4D-310 Gene Therapy in Adults with Fabry Disease Affecting the Heart
Led by 4D Molecular Therapeutics · Updated on 2025-02-21
18
Participants Needed
4
Research Sites
208 weeks
Total Duration
AI-Summary
What this Trial Is About
Researchers are evaluating the safety, tolerability, and pharmacodynamics of 4D-310, a gene therapy, in adults with Fabry Disease who have heart involvement. This open-label, dose-escalation trial includes adult males and females aged 18 to 65 years and aims to understand how the treatment works after a single intravenous dose. Fabry Disease is a condition that affects multiple organs, including the heart, and requires new treatment approaches. Participants receive one single intravenous administration of 4D-310 at different dose levels as part of the study. The trial includes several dose groups, with some dose levels no longer enrolling. The study is conducted across multiple centers and is designed to carefully monitor responses to the gene therapy over time. During the study, participants will be regularly assessed for safety by tracking any adverse events for one year following treatment. Researchers will also monitor how the body responds to the therapy through various evaluations. The total participation time includes screening, treatment, and follow-up visits to ensure thorough observation of treatment effects and safety.
CONDITIONS
Brief Title
4D-310 in Adults With Fabry Disease and Cardiac Involvement
Research Team
4
4DMT Patient Advocacy
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