An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adult Males With Fabry Disease

  • STATUS
    Recruiting
  • End date
    Apr 24, 2027
  • participants needed
    18
  • sponsor
    4D Molecular Therapeutics
Updated on 24 April 2022
gene therapy
hypertrophy
ventricular hypertrophy
cornea verticillata
acroparesthesia
angiokeratoma

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 with Fabry Disease.

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 with Fabry Disease.

Details
Condition Fabry Disease
Treatment 4D-310
Clinical Study IdentifierNCT04519749
Sponsor4D Molecular Therapeutics
Last Modified on24 April 2022

Eligibility

Yes No Not Sure

Inclusion Criteria

Male ≥ 18 years of age
Pathogenic GLA mutation consistent with Fabry Disease
Confirmed diagnosis of classic Fabry disease with one or more clinical characteristics: acroparesthesia, hypohidrosis, angiokeratoma or cornea verticillata, or late-onset Fabry disease with left ventricular hypertrophy
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

Exclusion Criteria

Presence of high titer NAb to 4D-310 capsid
eGFR <45 mL/min/1.73 m2
Presence of high antibody titer to AGA
Undergone kidney transplantation or currently on hemodialysis or peritoneal dialysis
Either history of, or a positive serology test at Screening for hepatitis B surface antigen (HBsAg), hepatitis C antibody (HCAb), or human immunodeficiency virus (HIV)
Evidence of liver disease
Severe pulmonary disease
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)
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
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)
Presence of intercurrent illness or other extenuating circumstance including active infection, active illicit drug or alcohol abuse; that might limit compliance with study requirements; morbid obesity with a BMI of >=35; or in the Investigator's assessment would place the subject at an unacceptable risk or confound interpretation of results
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