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

Recruiting now · Phase 1/Phase 2

Conditions studied: Fabry Disease

In brief

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

Key facts

Study ID

NCT05629559

Run by

4D Molecular Therapeutics

People needed

18

Starts

2022-10-16

Expected to finish

2030-06-01

Last updated by the study team

2025-02-21

Who can join

Age: 18 and older, up to 65. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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
• Individuals receiving ERT, must be on a stable dose for at least 6 months
• Agree to use highly effective contraception

You may not qualify if…

• 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,
• Evidence of liver disease, severe pulmonary disease or diabetes with poor glycemic control
• History of stroke or transient ischemic attack within the last 12 months, or other significant thromboembolic disease history (e.g. pulmonary embolism)
• Contraindication to systemic corticosteroid therapy or immunosuppressive therapy
• Chronic steroid use, defined as ≥ 3 months of oral corticosteroid use within the last 12 months.
• Moderately severe to severe cardiovascular disease or uncontrolled hypertension
• Left ventricular ejection fraction of <45% on echocardiogram (ECHO)
• Currently receiving investigational drug, device or therapy or having ever received gene therapy
• History of infusion related response to ERT or any adverse reaction leading to ERT discontinuation
• History of cancer within 2 years (exceptions include non-melanoma skin cancer, localized prostate cancer treated with curative intent)
• Pregnant or breast-feeding
• Note: Other inclusion and exclusion criteria apply

Where it is running

• Taipei Veterans General Hospital — Taipei, Taiwan (enrolling)
• Royal Melbourne Hospital — Melbourne, Australia
• Royal Perth Hospital — Perth, Australia
• Westmead Hospital — Westmead, Australia

Full record on ClinicalTrials.gov

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