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Search / Trial NCT05629559

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

Launched by 4D MOLECULAR THERAPEUTICS · Nov 17, 2022

Trial Information

Current as of July 22, 2025

Recruiting

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

ClinConnect Summary

This clinical trial, called 4D-310 in Adults With Fabry Disease and Cardiac Involvement, is exploring a new treatment for adults with Fabry Disease, particularly those experiencing issues related to their heart. Fabry Disease is a genetic condition that can affect various organs, and this trial aims to find out if a single intravenous (IV) dose of the study drug, 4D-310, is safe and how it works in the body. The trial is currently recruiting participants aged 18 to 65 who have a confirmed diagnosis of Fabry Disease with heart involvement and may have difficulty with existing treatments.

To be eligible, participants need to have a specific genetic mutation linked to Fabry Disease and should either be intolerant to enzyme replacement therapy (ERT), unable to take it, or experiencing worsening symptoms even with treatment. Participants will receive the study drug and will be monitored for any side effects. It's important to note that individuals with certain health conditions, such as severe heart disease or those who have received gene therapy in the past, cannot participate. Overall, this trial hopes to provide valuable information that could lead to better treatment options for people living with Fabry Disease.

Gender

ALL

Eligibility criteria

  • 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

About 4d Molecular Therapeutics

4D Molecular Therapeutics is a pioneering biotechnology company specializing in the development of innovative gene therapies to address rare and complex diseases. With a strong emphasis on harnessing the power of its proprietary 4D genome platform, the company focuses on creating targeted and effective therapeutic solutions that enhance the delivery and expression of genetic material. Committed to advancing the field of molecular medicine, 4D Molecular Therapeutics collaborates with leading research institutions and stakeholders to drive clinical development and improve patient outcomes through cutting-edge science and technology.

Locations

Westmead, New South Wales, Australia

Perth, , Australia

Melbourne, , Australia

Taipei, , Taiwan

Westmead, , Australia

Patients applied

0 patients applied

Trial Officials

Alan H Cohen, MD

Study Director

4D Molecular Therapeutics

Timeline

First submit

Trial launched

Trial updated

Estimated completion

Not reported

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