Summary

Eligibility
for people ages 18 years and up (full criteria)
Location
at La Jolla, California and other locations
Dates
study started
estimated completion
Principal Investigator
by Eric Adler, MD
Headshot of Eric Adler
Eric Adler

Description

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.

Details

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.

Keywords

Fabry Disease, Lysosomal Storage Diseases, Nervous System Brain Diseases, Metabolic, Inborn Brain Diseases, Metabolic 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, Lipodoses, Lipid Metabolism

Eligibility

You can join if…

Open to people ages 18 years and up

  1. Male or female ≥ 18 years of age
  2. Pathogenic GLA mutation consistent with Fabry Disease
  3. Confirmed diagnosis of classic or late-onset Fabry disease
  4. Individuals on ERT must be on a stable dose for at least 6 months (and a minimum of 12 months total exposure) prior to study enrollment
  5. Agree to use highly effective contraception

You CAN'T join if...

  1. Presence of high titer neutralizing antibody to 4D-310 capsid, or presence of high antibody titer to AGA
  2. eGFR <45 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

Locations

  • University of California at San Diego accepting new patients
    La Jolla California 92037 United States
  • University of California at Los Angeles not yet accepting patients
    Los Angeles California 90025 United States

Lead Scientist at UCSD

  • Eric Adler, MD
    Professor Of Clinical, Medicine, Vc-health Sciences-schools

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
4D Molecular Therapeutics
Links
Study Website
ID
NCT04519749
Phase
Phase 1/2 Fabry Disease Research Study
Study Type
Interventional
Participants
Expecting 18 study participants
Last Updated