4D-310 in Adults With Fabry Disease and Cardiac Involvement
ConditionFabry Disease
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18-65
Sponsor4D Molecular Therapeutics
About this trial
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
Eligibility criteria
Qualifiers
Male or female ≥ 18 to ≤65 years of age
Pathogenic GLA mutation consistent with Fabry Disease
Confirmed diagnosis of classic or late-onset Fabry disease with cardiac involvement
Intolerant of ERT, unable or unwilling to receive ERT, or progressive disease despite ERT or migalastat
Disqualifiers
Presence of pre-existing antibodies to 4D-310 capsid or to AGA
eGFR <65 mL/min/1.73 m2
Undergone kidney transplantation or currently on hemodialysis or peritoneal dialysis
HIV, active or chronic hepatitis B or C,
Trial design
Treatments tested in this trial
- 4D-310
Treatment groups
18 Participants
are divided into 4 treatment groupsSponsors and collaborators
Source ClinicalTrials.gov