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
This trial does not accept healthy volunteersQualifiers
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
Design model
Sequential
Treatments tested in this trial
4D-310
Biological/VaccineSingle IV administration of 4D-310
Treatment groups
18 Participants
are divided into 4 treatment groupsGroup A: 4D-310 Dose Level -1Experimental treatment 1 intervention
Group B: 4D-310 Dose Level 2Experimental treatment 1 intervention
Group C: 4D-310 Dose Level 3Experimental treatment 1 intervention
Group D: 4D-310 Dose Level 1 (No longer enrolling)Experimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Incidence and severity of adverse events
Incidence and severity of adverse events following a single IV dose of 4D-310intravenous (IV) dose
Time frame
1 yearSponsors and contacts
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Source ClinicalTrials.gov