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
- Are there results?
- Not yet: the study is under way
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
Study at a glance
From the registry record, last updated by the sponsor on 21 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label, Phase 1/2a Trial of Gene Therapy 4D-310 in Adults With Fabry Disease and Cardiac Involvement
- ClinicalTrials.gov ID
- NCT05629559
- Acronym / sponsor ID
- 4D-310-C002
- Conditions
- Fabry Disease
- 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
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 18 participants (estimated)
- Lead sponsor
- 4D Molecular TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 16 Oct 2022 (actual)
- Primary completion
- Jun 2026 (estimated)
- Study completion
- Jun 2030 (estimated)
- First posted
- 29 Nov 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
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
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| 4D-310 Dose Level -1 | Experimental | Biological: 4D-310Single IV administration of 4D-310 Dose Level -1 |
| 4D-310 Dose Level 2 | Experimental | Biological: 4D-310Single IV administration of 4D-310 Dose Level 2 |
| 4D-310 Dose Level 3 | Experimental | Biological: 4D-310Single IV administration of 4D-310 at Dose Level 3 |
| 4D-310 Dose Level 1 (No longer enrolling) | Experimental | Biological: 4D-310No longer enrolling - Single IV administration of 4D-310 at Dose Level 1 |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (15 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 4D-310Single IV administration of 4D-310 | Biological | 4D-310 Dose Level -1, 4D-310 Dose Level 1 (No longer enrolling), 4D-310 Dose Level 2, 4D-310 Dose Level 3 |
Primary outcome
Incidence and severity of adverse events
Time frame: 1 year
Incidence and severity of adverse events following a single IV dose of 4D-310intravenous (IV) dose
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Feb 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Alan H Cohen, MD study director
4D Molecular Therapeutics
4 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing 4D-310 in people with Fabry Disease, run by 4D Molecular Therapeutics at 4 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 21 Feb 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in Australia, Taiwan. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact 4DMT Patient Advocacy ((888) 748-8881, clinicaltrials@4DMT.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence and severity of adverse events (time frame: 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 Feb 2025.