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RECRUITINGPhase 1 / Phase 2INTERVENTIONAL

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

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

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

Who May Be Eligible (Plain English)

Who May Qualify: 1. Male or female ≥ 18 to ≤65 years of age 2. Pathogenic GLA mutation consistent with Fabry Disease 3. Confirmed diagnosis of classic or late-onset Fabry disease with cardiac involvement 4. Intolerant of ERT, unable or unwilling to receive ERT, or progressive disease despite ERT or migalastat 5. Individuals receiving ERT, must be on a stable dose for at least 6 months 6. Agree to use highly effective contraception Who Should NOT Join This Trial: 1. Presence of pre-existing antibodies to 4D-310 capsid or to AGA 2. eGFR \<65 mL/min/1.73 m2 3. Undergone kidney transplantation or currently on hemodialysis or peritoneal dialysis 4. HIV, active or chronic hepatitis B or C, 5. Evidence of liver disease, severe pulmonary disease or diabetes with poor glycemic control 6. History of stroke or transient ischemic attack within the last 12 months, or other significant thromboembolic disease history (e.g. pulmonary embolism) 7. Contraindication to systemic corticosteroid therapy or immunosuppressive therapy 8. Chronic steroid use, defined as ≥ 3 months of oral corticosteroid use within the last 12 months. 9. Moderately severe to severe cardiovascular disease or uncontrolled hypertension 10. Left ventricular ejection fraction of \<45% on echocardiogram (ECHO) 11. Currently receiving investigational drug, device or therapy or having ever received gene therapy 12. History of infusion related response to ERT or any adverse reaction leading to ERT discontinuation 13. History of cancer within 2 years (exceptions include non-melanoma skin cancer, localized prostate cancer treated with curative intent) 14. Pregnant or breast-feeding Note: Other inclusion and exclusion criteria apply Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
Inclusion Criteria: 1. Male or female ≥ 18 to ≤65 years of age 2. Pathogenic GLA mutation consistent with Fabry Disease 3. Confirmed diagnosis of classic or late-onset Fabry disease with cardiac involvement 4. Intolerant of ERT, unable or unwilling to receive ERT, or progressive disease despite ERT or migalastat 5. Individuals receiving ERT, must be on a stable dose for at least 6 months 6. Agree to use highly effective contraception Exclusion Criteria: 1. Presence of pre-existing antibodies to 4D-310 capsid or to AGA 2. eGFR \<65 mL/min/1.73 m2 3. Undergone kidney transplantation or currently on hemodialysis or peritoneal dialysis 4. HIV, active or chronic hepatitis B or C, 5. Evidence of liver disease, severe pulmonary disease or diabetes with poor glycemic control 6. History of stroke or transient ischemic attack within the last 12 months, or other significant thromboembolic disease history (e.g. pulmonary embolism) 7. Contraindication to systemic corticosteroid therapy or immunosuppressive therapy 8. Chronic steroid use, defined as ≥ 3 months of oral corticosteroid use within the last 12 months. 9. Moderately severe to severe cardiovascular disease or uncontrolled hypertension 10. Left ventricular ejection fraction of \<45% on echocardiogram (ECHO) 11. Currently receiving investigational drug, device or therapy or having ever received gene therapy 12. History of infusion related response to ERT or any adverse reaction leading to ERT discontinuation 13. History of cancer within 2 years (exceptions include non-melanoma skin cancer, localized prostate cancer treated with curative intent) 14. Pregnant or breast-feeding Note: Other inclusion and exclusion criteria apply

Treatments Being Tested

BIOLOGICAL

4D-310

Single IV administration of 4D-310

Locations (4)

Royal Melbourne Hospital
Melbourne, Australia
Royal Perth Hospital
Perth, Australia
Westmead Hospital
Westmead, Australia
Taipei Veterans General Hospital
Taipei, Taiwan