Summary

Eligibility
for people ages 18 years and up (full criteria)
Location
at Irvine, California and other locations
Dates
study started
estimated completion
Principal Investigator
by Madeleine Pahl

Description

Summary

This is the first in human treatment with ST-920, a recombinant AAV2/6 vector encoding the cDNA for human a-Gal A. The purpose of this study is to evaluate the safety and tolerability of ascending doses of ST-920. ST-920 aims to provide stable, long-term production of α-Gal A at therapeutic levels in subjects with Fabry disease. The constant production of α-Gal A in humans should, importantly, enable reduction and potentially clearance of Fabry disease substrates Gb3 and lyso-Gb3. On Day 1, patients will be infused intravenously with a single dose of ST-920 and followed for a period of 52 weeks.

Official Title

A Phase I/II, Multicenter, Open-Label, Single-Dose, Dose-Ranging Study to Assess the Safety and Tolerability of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy, in Subjects With Fabry Disease

Keywords

Fabry Disease Sangamo Rare Lysosomal Storage Disease Gene Therapy

Eligibility

You can join if…

Open to people ages 18 years and up

  • ≥ 18 years of age
  • Documented diagnosis of Fabry disease
  • One or more of the following symptoms: i) cornea verticillata, ii) acroparesthesia, iii) anhidrosis, iv) angiokeratoma

You CAN'T join if...

  • Known to be unresponsive to ERT
  • Neutralizing antibodies to AAV2/6
  • Currently receiving migalastat (Galafold™)
  • eGFR ≤ 40 ml/min/1.73m2
  • New York Heart Association Class III or higher
  • Active infection with hepatitis A, B or C, HIV or TB
  • History of liver disease such as secondary steatosis, non-alcoholic steatohepatitis (NASH) and cirrhosis, cholangitis or biliary disease within 6 months of informed consent; except for Gilbert's syndrome
  • Elevated circulating serum AFP
  • Recent or recurrent hypersensitivity response to ERT within previous 6 months
  • Current or history of systemic (IV or oral) immunomodulatory agents, or biologics or steroid use in the past 6 months (topical treatment and inhaled allowed).
  • Contraindication to use of corticosteroids
  • History of malignancy except for non-melanoma skin cancer
  • Recent history of alcohol or substance abuse
  • Participation in prior investigational interventional drug or medical device study within previous 3 months
  • Prior treatment with a gene therapy product
  • Known hypersensitivity to components of ST-920 formulation
  • Any other reason that, in the opinion of the Site Investigator or Medical Monitor, would render the subject unsuitable for participation in the study including but not limited to risk of COVID-19 infection

Locations

  • University of California, Irvine accepting new patients
    Irvine California 92697 United States
  • University of Minnesota Medical Center accepting new patients
    Minneapolis Minnesota 55455 United States

Lead Scientist at UC Irvine

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Sangamo Therapeutics
ID
NCT04046224
Phase
Phase 1/2 Fabry Disease Research Study
Study Type
Interventional
Participants
Expecting 48 study participants
Last Updated