Actively Recruiting
4D-310 in Adults With Fabry Disease and Cardiac Involvement
Led by 4D Molecular Therapeutics · Updated on 2025-02-21
18
Participants Needed
4
Research Sites
397 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
Official Title
4D-310 in Adults With Fabry Disease and Cardiac Involvement
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male or female aged 18 to 65 years
- Pathogenic GLA mutation consistent with Fabry Disease
- Confirmed diagnosis of classic or late-onset Fabry Disease with cardiac involvement
- Intolerant of enzyme replacement therapy (ERT), unable or unwilling to receive ERT, or progressive disease despite ERT or migalastat
- If receiving ERT, must be on stable dose for at least 6 months
- Agree to use highly effective contraception
You will not qualify if you...
- Presence of pre-existing antibodies to 4D-310 capsid or to alpha-galactosidase A (AGA)
- Estimated glomerular filtration rate (eGFR) less than 65 mL/min/1.73 m2
- History of kidney transplantation or currently on hemodialysis or peritoneal dialysis
- HIV infection, active or chronic hepatitis B or C
- Evidence of liver disease, severe lung disease, or diabetes with poor blood sugar control
- Stroke or transient ischemic attack within the last 12 months or significant blood clot history
- Contraindication to systemic corticosteroids or immunosuppressive therapy
- Chronic steroid use of 3 months or more in the past year
- Moderately severe to severe heart disease or uncontrolled high blood pressure
- Left ventricular ejection fraction below 45% on echocardiogram
- Currently receiving investigational drugs, devices, or gene therapy
- History of infusion reactions or adverse events leading to stopping ERT
- Cancer history within 2 years except for certain skin and prostate cancers
- Pregnant or breastfeeding
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 4 locations
1
Royal Melbourne Hospital
Melbourne, Australia, 3050
Active, Not Recruiting
2
Royal Perth Hospital
Perth, Australia, 6000
Active, Not Recruiting
3
Westmead Hospital
Westmead, Australia, 2145
Not Yet Recruiting
4
Taipei Veterans General Hospital
Taipei, Taiwan, 11217
Actively Recruiting
Research Team
4
4DMT Patient Advocacy
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
4
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