Status:
ACTIVE_NOT_RECRUITING
An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry Disease
Lead Sponsor:
4D Molecular Therapeutics
Conditions:
Fabry Disease
Eligibility:
All Genders
18+ years
Phase:
PHASE1
PHASE2
Brief Summary
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 co...
Detailed Description
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 co...
Eligibility Criteria
Inclusion
- 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
Exclusion
- 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
Key Trial Info
Start Date :
September 1 2020
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
June 1 2030
Estimated Enrollment :
18 Patients enrolled
Trial Details
Trial ID
NCT04519749
Start Date
September 1 2020
End Date
June 1 2030
Last Update
April 8 2024
Active Locations (4)
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1
University of California at San Diego
La Jolla, California, United States, 92037
2
Emory University
Atlanta, Georgia, United States, 30322
3
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States, 15224
4
Lysosomal & Rare Disorders Research & Treatment Center, Inc
Fairfax, Virginia, United States, 22030