4D-310 in Adults With Fabry Disease and Cardiac Involvement
NCT ID: NCT05629559
Last Updated: 2025-02-21
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE1/PHASE2
18 participants
INTERVENTIONAL
2022-10-16
2030-06-30
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NON_RANDOMIZED
SEQUENTIAL
TREATMENT
NONE
Study Groups
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4D-310 Dose Level -1
Single IV administration of 4D-310 Dose Level -1
4D-310
Single IV administration of 4D-310
4D-310 Dose Level 2
Single IV administration of 4D-310 Dose Level 2
4D-310
Single IV administration of 4D-310
4D-310 Dose Level 3
Single IV administration of 4D-310 at Dose Level 3
4D-310
Single IV administration of 4D-310
4D-310 Dose Level 1 (No longer enrolling)
No longer enrolling - Single IV administration of 4D-310 at Dose Level 1
4D-310
Single IV administration of 4D-310
Interventions
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4D-310
Single IV administration of 4D-310
Eligibility Criteria
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Inclusion Criteria
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
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
18 Years
65 Years
ALL
No
Sponsors
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4D Molecular Therapeutics
INDUSTRY
Responsible Party
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Principal Investigators
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Alan H Cohen, MD
Role: STUDY_DIRECTOR
4D Molecular Therapeutics
Locations
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Royal Melbourne Hospital
Melbourne, , Australia
Royal Perth Hospital
Perth, , Australia
Westmead Hospital
Westmead, , Australia
Taipei Veterans General Hospital
Taipei, , Taiwan
Countries
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Central Contacts
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Facility Contacts
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Other Identifiers
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4D-310-C002
Identifier Type: -
Identifier Source: org_study_id
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