NCT05629559

4D-310 in Adults With Fabry Disease and Cardiac Involvement

An Open-Label, Phase 1/2a Trial of Gene Therapy 4D-310 in Adults With Fabry Disease and Cardiac Involvement

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 18 (estimated)
• Sponsor: 4D Molecular Therapeutics · Industry
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

Summary

This is a prospective multicenter, open-label, dose-escalation trial to assess the safety, tolerability, and pharmacodynamics of 4D-310 following a single IV administration. The study population is comprised of adult males and females with Fabry Disease and cardiac involvement

Detailed description

This is a prospective multicenter, open-label, dose-escalation trial to assess the safety, tolerability, and pharmacodynamics of 4D-310 following a single IV administration. The study population is comprised of adult males and females with Fabry Disease and cardiac involvement

Conditions

• Fabry Disease

Interventions

Type	Name	Description
BIOLOGICAL	4D-310	Single IV administration of 4D-310

Timeline

• Start date: 2022-10-16
• Primary completion: 2026-06-01
• Completion: 2030-06-01
• First posted: 2022-11-29
• Last updated: 2025-02-21

Locations

4 sites across 2 countries: Australia, Taiwan

Regulatory

• FDA-regulated drug study