The company 4D Molecular Therapeutics is enrolling patients into the clinical trial investigating 4D-310 in Adults With Fabry Disease and Cardiac Involvement.
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
The trial is designed to enroll male and female 18 Years and older and is being conducted in the Royal Melbourne Hospital, Melbourne, Australia; Royal Perth Hospital, Perth, Australia; Taipei Veterans General Hospital, Taipei, Taiwan. The study started in October, 2022.
Included can be adult males and females with athogenic GLA mutation consistent with Fabry Disease; with confirmed diagnosis of classic or late-onset Fabry disease with cardiac involvement; 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; must agree to use highly effective contraception.
Whereas the following population cannot be enrolled into this study: patients with presence of high titer neutralizing antibody to 4D-310 capsid, or presence of high antibody titer to AGA 2. eGFR <45 mL/min/1.73 m2; undergone kidney transplantation or currently on hemodialysis or peritoneal dialysis; have the diagnised HIV, active or chronic hepatitis B or C; with evidence of liver disease, severe pulmonary disease or diabetes with poor glycemic control; with history of stroke or transient ischemic attack within the last 12 months, or other significant thromboembolic disease history (e.g. pulmonary embolism); have contraindication to systemic corticosteroid therapy or immunosuppressive therapy; with chronic steroid use, defined as ≥ 3 months of oral corticosteroid use within the last 12 months; have moderately severe to severe cardiovascular disease or uncontrolled hypertension; have left ventricular ejection fraction of <45% on echocardiogram (ECHO); are currently receiving investigational drug, device or therapy or having ever received gene therapy; have history of infusion related response to ERT or any adverse reaction leading to ERT discontinuation; with history of cancer within 2 years (exceptions include non-melanoma skin cancer, localized prostate cancer treated with curative intent); are pregnant or breast-feeding.
This page provides a more detailed overview of this clinical trial: https://ichgcp.net/clinical-trials-registry/NCT05629559