An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry Disease

Running, not enrolling · 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.

Key facts

Study ID

NCT04519749

Run by

4D Molecular Therapeutics

People needed

18

Starts

2020-09-01

Expected to finish

2030-06-01

Last updated by the study team

2024-04-08

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Male or female ≥ 18 years of age
• Pathogenic GLA mutation consistent with Fabry Disease
• Confirmed diagnosis of classic or late-onset Fabry disease
• Individuals on ERT must be on a stable dose for at least 6 months (and a minimum of 12 months total exposure) prior to study enrollment
• Agree to use highly effective contraception

You may not qualify if…

• Presence of high titer neutralizing antibody to 4D-310 capsid, or presence of high antibody titer to AGA
• eGFR <45 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

Where it is running

• University of California at San Diego — La Jolla, California, United States
• Emory University — Atlanta, Georgia, United States
• Children's Hospital of Pittsburgh of UPMC — Pittsburgh, Pennsylvania, United States
• Lysosomal & Rare Disorders Research & Treatment Center, Inc — Fairfax, Virginia, United States

Full record on ClinicalTrials.gov

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