Treatment for adults with Fabry disease and heart issues
An Open-Label, Phase 1/2a Trial of Gene Therapy 4D-310 in Adults With Fabry Disease and Cardiac Involvement
This study is testing a new treatment for adults with Fabry disease and heart problems to see if it is safe and how well it works.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 18 (estimated) |
| Ages | 18 Years to 65 Years |
| Sex | All |
| Sponsor | 4D Molecular Therapeutics Industry-sponsored |
| Locations | 4 sites (Melbourne and 3 other locations) |
| Trial ID | NCT05629559 on ClinicalTrials.gov |
What this trial studies
This is a multicenter, open-label trial designed to evaluate the safety, tolerability, and pharmacodynamics of a single intravenous administration of 4D-310 in adults diagnosed with Fabry disease who also have cardiac involvement. The trial will involve dose escalation to determine the optimal dosage while monitoring participants for any adverse effects. The study population includes both male and female adults aged 18 to 65 years with specific genetic mutations associated with Fabry disease.
Who should consider this trial
Good fit: Ideal candidates are adults aged 18 to 65 with a confirmed diagnosis of Fabry disease and cardiac involvement who are intolerant to or have had inadequate responses to existing enzyme replacement therapies.
Not a fit: Patients with severe kidney impairment, recent history of significant thromboembolic events, or those with contraindications to corticosteroid therapy may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with Fabry disease who have cardiac complications.
How similar studies have performed: While there have been studies on Fabry disease treatments, the specific approach of using 4D-310 in this context is novel and has not been extensively tested in prior trials.
Eligibility criteria
Show full inclusion / exclusion 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
Where this trial is running
Melbourne and 3 other locations
- 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)
Study contacts
- Study coordinator: 4DMT Patient Advocacy
- Email: clinicaltrials@4DMT.com
- Phone: (888) 748-8881
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.