Actively Recruiting
An Open-Label, Phase 1/2a Trial of Gene Therapy 4D-310 in Adults With Fabry Disease and Cardiac Involvement
Led by 4D Molecular Therapeutics · Updated on 2025-02-21
18
Participants Needed
4
Research Sites
208 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety, tolerability, and pharmacodynamics of 4D-310, a gene therapy, in adults with Fabry Disease who have heart involvement. This open-label, dose-escalation trial includes adult males and females aged 18 to 65 years and aims to understand how the treatment works after a single intravenous dose. Fabry Disease is a condition that affects multiple organs, including the heart, and requires new treatment approaches. Participants receive one single intravenous administration of 4D-310 at different dose levels as part of the study. The trial includes several dose groups, with some dose levels no longer enrolling. The study is conducted across multiple centers and is designed to carefully monitor responses to the gene therapy over time. During the study, participants will be regularly assessed for safety by tracking any adverse events for one year following treatment. Researchers will also monitor how the body responds to the therapy through various evaluations. The total participation time includes screening, treatment, and follow-up visits to ensure thorough observation of treatment effects and safety.
CONDITIONS
Brief Title
4D-310 in Adults With Fabry Disease and Cardiac Involvement
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male or female aged 18 to 65 years
- Pathogenic GLA mutation consistent with Fabry Disease
- Confirmed diagnosis of classic or late-onset Fabry Disease with cardiac involvement
- Intolerant of enzyme replacement therapy (ERT), unable or unwilling to receive ERT, or progressive disease despite ERT or migalastat
- If receiving ERT, must be on a stable dose for at least 6 months
- Agree to use highly effective contraception
You will not qualify if you...
- Presence of pre-existing antibodies to 4D-310 capsid or to AGA
- Estimated glomerular filtration rate (eGFR) less than 65 mL/min/1.73 m2
- History of kidney transplantation or currently on hemodialysis or peritoneal dialysis
- HIV infection or active/chronic hepatitis B or C
- Evidence of liver disease, severe lung disease, or poorly controlled diabetes
- Stroke or transient ischemic attack within the last 12 months or significant thromboembolic disease history
- Contraindication to systemic corticosteroid or immunosuppressive therapy
- Chronic steroid use (3 months or more oral corticosteroid use within last 12 months)
- Moderately severe to severe cardiovascular disease or uncontrolled hypertension
- Left ventricular ejection fraction below 45% on echocardiogram
- Currently receiving any investigational drug, device, or therapy or prior gene therapy
- History of infusion-related response or adverse reaction to ERT leading to discontinuation
- History of cancer within 2 years (except non-melanoma skin cancer or localized prostate cancer treated with curative intent)
- Pregnant or breastfeeding
- Other inclusion and exclusion criteria may apply
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - Participants are followed for 1 year after administration to assess safety and tolerability.
Participants receive a single intravenous administration of the gene therapy 4D-310 at different dose levels.
Trial Site Locations
Total: 4 locations
1
Royal Melbourne Hospital
Melbourne, Australia, 3050
Active, Not Recruiting
2
Royal Perth Hospital
Perth, Australia, 6000
Active, Not Recruiting
3
Westmead Hospital
Westmead, Australia, 2145
Not Yet Recruiting
4
Taipei Veterans General Hospital
Taipei, Taiwan, 11217
Actively Recruiting
Research Team
4
4DMT Patient Advocacy
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
4
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