Gene therapy 4D-310 for adults with Fabry disease affecting the heart
Tests a one-time gene therapy infusion in adults with Fabry disease affecting the heart.
Plain-language title generated from the registry record
Registry title: 4D-310 in Adults With Fabry Disease and Cardiac Involvement
Key facts
- Where
- Melbourne, Perth, and Westmead, AustraliaSee the 3 sites
- Phase
- Phase 1/2
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 18 and 65 years old?
Source: NCT05629559, who can take part
Where in Australia
- Royal Melbourne HospitalMelbourne, AustraliaNo longer taking part
- Royal Perth HospitalPerth, AustraliaNo longer taking part
- Westmead HospitalWestmead, AustraliaNot yet recruiting
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- 4D Molecular Therapeutics
- Trial code
- NCT05629559
- Record updated
- 21 February 2025
- Original description
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 involvementRead full original descriptionShow less
An Open-Label, Phase 1/2a Trial of Gene Therapy 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
Inclusion: 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: Presence of pre-existing antibodies to 4D-310 capsid or to AGA; Undergone kidney transplantation or currently on hemodialysis or peritoneal dialysis; Contraindication to systemic corticosteroid therapy or immunosuppressive therapy; Chronic steroid use, defined as ≥ 3 months of oral corticosteroid use within the last 12 months; Currently receiving investigational drug, device or therapy or having ever received gene therapy; 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); Moderately severe to severe cardiovascular disease or uncontrolled hypertension; 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); eGFR <65 mL/min/1.73 m2; Left ventricular ejection fraction of <45% on echocardiogram (ECHO); Pregnant or breast-feeding; Note: Other inclusion and exclusion criteria apply.