NCT05629559 4D-310 in Adults With Fabry Disease and Cardiac Involvement
| NCT ID | NCT05629559 |
| Status | Recruiting |
| Phase | Phase 1, Phase 2 |
| Sponsor | 4D Molecular Therapeutics |
| Condition | Fabry Disease |
| Study Type | INTERVENTIONAL |
| Enrollment | 18 participants |
| Start Date | 2022-10-16 |
| Primary Completion | 2026-06 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 18 participants in total. It began in 2022-10-16 with a primary completion date of 2026-06.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief 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 Criteria
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
Contact & Investigator
Alan H Cohen, MD
STUDY DIRECTOR
4D Molecular Therapeutics
Frequently Asked Questions
Who can join the NCT05629559 clinical trial?
This trial is open to participants of all sexes, aged 18 Years or older, up to 65 Years, studying Fabry Disease. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT05629559 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT05629559 currently recruiting?
Yes, NCT05629559 is actively recruiting participants. Contact the research team at clinicaltrials@4DMT.com for enrollment information.
Where is the NCT05629559 trial being conducted?
This trial is being conducted at Melbourne, Australia, Perth, Australia, Westmead, Australia, Taipei, Taiwan.
Who is sponsoring the NCT05629559 clinical trial?
NCT05629559 is sponsored by 4D Molecular Therapeutics. The principal investigator is Alan H Cohen, MD at 4D Molecular Therapeutics. The trial plans to enroll 18 participants.