Treatment for adults with Fabry disease and heart issues

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This is a multicenter, open-label trial designed to evaluate the safety, tolerability, and pharmacodynamics of a single intravenous administration of 4D-310 in adults diagnosed with Fabry disease who also have cardiac involvement. The trial will involve dose escalation to determine the optimal dosage while monitoring participants for any adverse effects. The study population includes both male and female adults aged 18 to 65 years with specific genetic mutations associated with Fabry disease.

Why it matters

Who can join: the full rules

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

Where it's running

• Taipei Veterans General Hospital
  Taipei, Taiwan Recruiting
• Royal Melbourne Hospital
  Melbourne, Australia Active not recruiting
• Royal Perth Hospital
  Perth, Australia Active not recruiting
• Westmead Hospital
  Westmead, Australia Not yet recruiting

Study contacts

• Study coordinator: 4DMT Patient Advocacy
• Email: clinicaltrials@4DMT.com
• Phone: (888) 748-8881

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Lucerastat in untreated adult men with Fabry disease shows how well it works and safety

  This study tests lucerastat to see if it can reduce Gb3 buildup and be safe in untreated adult men with Fabry disease.

  Recruiting Phase 3 Idorsia Pharmaceuticals Ltd. · Birmingham, Alabama and 15 other locations · NCT07778667

• Periodontal Health in Rare Kidney Conditions and Controls

  This study tests how common periodontitis is in people with rare kidney disorders and compares it to CKD patients and people without kidney disease.

  Recruiting Institutul Clinic Fundeni · Bucharest · NCT07575347

• Anderson-Fabry disease fitness improvement program

  This study tests whether a 12-week physiotherapy-led exercise program can improve function, fitness, strength, and quality of life in adults with Fabry disease.

  Recruiting General University Hospital, Prague · Prague · NCT07495410

• Using advanced heart MRI to improve diagnosis and risk prediction for rare cardiomyopathies

  This project tests whether advanced cardiac MRI techniques can help diagnose and predict future risk for people with rare heart muscle diseases like Danon, Fabry, cardiac amyloidosis, Noonan syndrome, and cardiac sarcoidosis.

  Recruiting Chinese Academy of Medical Sciences, Fuwai Hospital · Beijing · NCT07336394

• Quality of life for people 65+ with Fabry disease, treated or untreated

  This project will see if starting or continuing Fabry disease treatment changes quality of life over two and five years in people aged 65 and older.

  Recruiting Groupe Hospitalier Diaconesses Croix Saint-Simon · Paris, France · NCT07277361

• Agalsidase alfa for heart inflammation in Fabry disease measured by PET‑CMR

  This study will test whether agalsidase alfa reduces heart inflammation in people with Fabry disease by comparing PET‑CMR scans before and after 12 months of treatment.

  Recruiting Yonsei University · Seoul · NCT07235709

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.