Clinical Trials in Australia / NCT05629559
Recruiting
Phase 1/2
4D-310 in Adults With Fabry Disease and Cardiac Involvement
NCT05629559 · tracked via the Priya Life Science Australia tracker
Condition(s) studied
Fabry Disease
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
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)
| Facility | City | Region | Status |
|---|---|---|---|
| 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 |
More 4D Molecular Therapeutics trials in Australia
Other trials for the same condition
Arrhythmia Burden, Risk of Sudden Cardiac Death and Stroke in Patients With Fabry Disease
Active, not recruiting
A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease
Active, not recruiting
Long-Term Follow-up of Subjects Who Were Treated With ST-920
Active, not recruiting
A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease
Active, not 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