Who may be eligible for the 4D-310 in Adults With Fabry Disease and Cardiac Involvement trial?

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

ClinicalTrials.gov record NCT05629559

4D-310 in Adults With Fabry Disease and Cardiac Involvement

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 17, 2026

An Open-Label, Phase 1/2a Trial of Gene Therapy 4D-310 in Adults With Fabry Disease and Cardiac Involvement

About this page. This page shows the study's official ClinicalTrials.gov record ↗, with added help to make it easier to understand. We explain medical terms, highlight key details in plain language, and suggest questions you may want to ask the study team.

The record is periodically copied from ClinicalTrials.gov and may lag behind changes. AI summaries are checked by software, not a clinician.Read moreShow less

The official record is written by the study sponsor and published on ClinicalTrials.gov. It may not always reflect the latest changes. Before making any decisions, please confirm important information with the study team and your own healthcare team.

Where you see "AI summary", AI was used to create a plain-language explanation and software checked it against the source. It has not been reviewed by a clinician, so it may contain mistakes or leave out important details.

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.

Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.

PhasePhase 1/2A study that combines safety and dose-finding (phase 1) with a first look at whether the treatment has an effect (phase 2).

Age18 years old to 65 years old

SexAll

LocationAustralia, Taiwan

AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This is a prospective, open-label, dose-escalation trial of a single intravenous dose of 4D-310. It enrolls adult males and females with Fabry disease who have cardiac involvement. The study measures safety, tolerability, and pharmacodynamics, with monitoring for adverse events up to one year.

What it is studying. The study tests the safety, tolerability, and pharmacodynamic effects after a single IV administration of the gene therapy 4D-310 in adults with Fabry disease and cardiac involvement.

What the researchers hope to learn. To assess the safety, tolerability, and pharmacodynamics of a single intravenous dose of 4D-310 in adults with Fabry disease and cardiac involvement.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Biological: 4D-310

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion 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

Exclusion criteria

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

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Detailed description from the registry

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants receive a single intravenous (IV) infusion of 4D-310 at a study dose as part of a sequential dose‑escalation, open‑label trial. After the infusion they will have scheduled clinic visits and tests to monitor safety, tolerability, and pharmacodynamic effects; the main safety follow-up in the registry is reported for one year. The study team will record any adverse events. The registry record does not list full visit or test details.

How the study is structured. Interventional, non-randomized, open-label dose-escalation study. Participants receive a single IV dose of 4D-310 at one of several dose levels (sequential enrollment by dose). Safety and adverse events are tracked, with key measurements reported through one year.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

This is an early-phase study, mainly about safety and dose. What is already known about this treatment?
The listed sites are in Australia, Taiwan. Is any of them reachable for me, and does the study help with travel?
Which of the eligibility criteria would need to be checked with tests before I could join?

More on Fabry disease

Clinical trials47 trials listed for Fabry diseaseEvery trial the registry lists for Fabry disease, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in Fabry disease researchLatest monthly update: September 2026. Includes: SGLT2 outcome link. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start: Oct 16, 2022
Primary completion: June 2026 (estimated)
Study completion: June 2030 (estimated)
Target enrollment: 18 participants (estimated)
Last updated on registry: Feb 21, 2025
Data verified: Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor: 4D Molecular Therapeutics

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

4DMT Patient Advocacy 📞 (888) 748-8881 ✉ clinicaltrials@4DMT.com

Participating sites (4)

Filter by country:

Royal Melbourne HospitalActive Not Recruiting
Melbourne, 3050 · Australia
Royal Perth HospitalActive Not Recruiting
Perth, 6000 · Australia
Westmead HospitalNot Yet Recruiting
Westmead, 2145 · Australia
- 4DMT Patient Advocacy · (888) 748-8881 · clinicaltrials@4DMT.com
Taipei Veterans General HospitalRecruiting
Taipei, 11217 · Taiwan
- 4DMT Patient Advocacy · (888) 748-8881 · clinicaltrials@4DMT.com

Terms used on this page · explanations by MedicalUpdates.ai

open-label: No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
dose-escalation: Groups of participants receive increasing doses of a treatment, one group after another, to find a dose that can be given safely.
pharmacodynamics: What a drug does to the body, for example how it changes a blood marker or a symptom, and how that relates to the dose.
stable dose: A dose that has not changed for a defined period before the study starts. Studies may require this so that background treatment is relatively stable when study measurements begin.
highly effective contraception: Studies often ask participants who could become pregnant, or could father a child, to use contraception during the study and for a period afterwards, because the effect of the treatment on a pregnancy is unknown.
eGFR: A blood-test estimate of how well the kidneys are filtering. Lower numbers mean reduced kidney function; many studies set a minimum value.
Contraindication: A reason a particular test or treatment should not be used for a person, for example a metal implant that rules out an MRI scan.
corticosteroid: Steroid medicines, such as prednisone, that reduce inflammation. Studies often require a stable dose at entry or measure whether the study treatment allows the dose to be reduced.
immunosuppressive: A medicine that reduces the activity of the immune system. Studies of autoimmune conditions often require a stable dose, a prior failure, or a washout of such medicines.
echocardiogram: An ultrasound scan of the heart. A transesophageal echocardiogram is done through a probe passed into the food pipe for a closer view.
investigational drug: A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
exclusion criteria: The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
Inclusion Criteria: The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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