Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease

  • STATUS
    Recruiting
  • End date
    Feb 29, 2024
  • participants needed
    48
  • sponsor
    Sangamo Therapeutics
Updated on 29 April 2022
gene therapy
alpha-galactosidase
cornea verticillata
acroparesthesia
angiokeratoma
anhidrosis
lyso-gb3

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.

Details
Condition Fabry Disease
Treatment ST-920
Clinical Study IdentifierNCT04046224
SponsorSangamo Therapeutics
Last Modified on29 April 2022

Eligibility

Yes No Not Sure

Inclusion Criteria

≥ 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

Exclusion Criteria

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)
History of malignancy except for non-melanoma skin cancer
Contraindication to use of corticosteroids
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
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