Fabry DiseasePhase 1/2NCT05629559
4D-310 in Adults With Fabry Disease and Cardiac Involvement
A Phase 1/2 study testing 4D-310 in people with Fabry Disease, run by 4D Molecular Therapeutics at 4 sites in 2 countries.
Trial 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 Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Fabry Disease
- What is being tested?
- 4D-310
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 4 sitesin 2 countries
- Study dates
- 16 Oct 2022to Jun 2030
- Phase
- Phase 1/2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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)
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- 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
Trial locations and contacts
4 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
4 sites in 2 countries
Australia3 sites, 1 recruiting
- Active, not recruiting
Royal Melbourne Hospital
Melbourne, 3050
- Active, not recruiting
Royal Perth Hospital
Perth, 6000
- Not yet recruiting
Westmead Hospital
Westmead, 2145
4DMT Patient Advocacy, (888) 748-8881, clinicaltrials@4DMT.com
Taiwan1 site, 1 recruiting
- Recruiting
Taipei Veterans General Hospital
Taipei, 11217
4DMT Patient Advocacy, (888) 748-8881, clinicaltrials@4DMT.com
About this study
Official title: An Open-Label, Phase 1/2a Trial of Gene Therapy 4D-310 in Adults With Fabry Disease and Cardiac Involvement
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
Read the detailed description
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
- Conditions
- Fabry Disease
- Being tested
- 4D-310
- Keywords
- Lysosomal Storage Diseases, Nervous System Brain Diseases, Metabolic, Inborn Brain Diseases, Central Nervous System Diseases, Nervous System Diseases, Cerebral Small Vessel Diseases, Cerebrovascular Disorders, Vascular Diseases, Cardiovascular Diseases, Genetic Diseases, X-Linked, Inborn, Metabolic Diseases, Lipid Metabolism Disorders, Sphingolipidoses, Metabolism, Inborn Errors, Lipidoses, Lipid Metabolism
- Medical subject headings
- Fabry Disease, Lysosomal Storage Diseases, Central Nervous System Diseases, Nervous System Diseases, Cerebral Small Vessel Diseases, Cerebrovascular Disorders, Vascular Diseases, Cardiovascular Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
4D-310Biological
Single IV administration of 4D-310
| Study group | Role | What they receive |
|---|---|---|
| 4D-310 Dose Level -1 | Experimental | Single IV administration of 4D-310 Dose Level -1 Receives: Biological: 4D-310 |
| 4D-310 Dose Level 2 | Experimental | Single IV administration of 4D-310 Dose Level 2 Receives: Biological: 4D-310 |
| 4D-310 Dose Level 3 | Experimental | Single IV administration of 4D-310 at Dose Level 3 Receives: Biological: 4D-310 |
| 4D-310 Dose Level 1 (No longer enrolling) | Experimental | No longer enrolling - Single IV administration of 4D-310 at Dose Level 1 Receives: Biological: 4D-310 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence and severity of adverse events
Incidence and severity of adverse events following a single IV dose of 4D-310intravenous (IV) dose
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Nov 2022
- Study start16 Oct 2022
- Primary completionJun 2026 (estimated)
- Study completionJun 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fabry DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 18 participants (estimated)
Sponsor and investigators
- Lead sponsor
- 4D Molecular Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Alan H Cohen, MD, Study director, 4D Molecular Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05629559
- Sponsor's protocol ID
- 4D-310-C002
- Status last verified
- Feb 2025
- First submitted
- 4 Nov 2022
- Last updated
- 21 Feb 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No