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New treatment option for Fabry Disease
Official title 4D-310 in Adults With Fabry Disease and Cardiac Involvement
ClinicalTrials.gov ID: NCT05629559
What this study is testing
What is 4D-310?
4D-310 is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for fabry disease.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 65
You may be able to join if
- 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
You likely can't join if
- Presence of pre-existing antibodies to 4D-310 capsid or to AGA
- eGFR \<65 mL/min/1.73 m2
- Undergone kidney transplantation or currently on hemodialysis or peritoneal dialysis
- 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...
See the full eligibility criteria
- 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
- Presence of pre-existing antibodies to 4D-310 capsid or to AGA
- eGFR \<65 mL/min/1.73 m2
- Undergone kidney transplantation or currently on hemodialysis or peritoneal dialysis
- 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)
- Contraindication to systemic corticosteroid therapy or immunosuppressive therapy
- Chronic steroid use, defined as ≥ 3 months of oral corticosteroid use within the last 12 months.
- Moderately severe to severe cardiovascular disease or uncontrolled hypertension
- Left ventricular ejection fraction of \<45% on echocardiogram (ECHO)
- Currently receiving investigational drug, device or therapy or having ever received gene therapy
- 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)
- Pregnant or breast-feeding Note: Other inclusion and exclusion criteria apply
The study team makes the final eligibility decision.
Where it's taking place
- Melbourne, Australia
- Perth, Australia
- Westmead, Australia
- Taipei, Taiwan
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 65 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Melbourne, Australia; Perth, Australia; Westmead, Australia; Taipei, Taiwan. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.