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

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Updated on 29 April 2022

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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 Identifier	NCT04046224
Sponsor	Sangamo Therapeutics
Last Modified on	29 April 2022

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