RecruitingPhase 1Phase 2NCT05629559

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

An Open-Label, Phase 1/2a Trial of Gene Therapy 4D-310 in Adults With Fabry Disease and Cardiac Involvement

Sponsor

4D Molecular Therapeutics

Enrollment

18 participants

Start Date

Oct 16, 2022

Study Type

INTERVENTIONAL

Conditions

Fabry Disease

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 comprised of adult males and females with Fabry Disease and cardiac involvement

Eligibility

Min Age: 18 YearsMax Age: 65 Years

Inclusion Criteria6

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

Exclusion Criteria14

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