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Recruiting Phase 1/2

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

NCT05629559 · tracked via the Priya Life Science Australia tracker
Phase
Phase 1/2
Started
2022-10-16
Last updated
2025-02-21

Condition(s) studied

Fabry Disease

Investigational drug(s) / intervention(s)

4D-310 →

4D-310: Single IV administration of 4D-310

Study 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

Sex
ALL
Min age
18 Years
Max age
65 Years
Healthy volunteers
No
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

Primary outcome measure(s)

  • Incidence and severity of adverse events — 1 year
    Incidence and severity of adverse events following a single IV dose of 4D-310intravenous (IV) dose

Trial sites (4)

FacilityCityRegionStatus
Royal Melbourne Hospital Melbourne Australia Active Not Recruiting
Royal Perth Hospital Perth Australia Active Not Recruiting
Westmead Hospital Westmead Australia Not Yet Recruiting
Taipei Veterans General Hospital Taipei Taiwan Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05629559 on ClinicalTrials.gov ↗ ← All trials in Australia