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

Status: Recruiting

The purpose of this study is to test a new drug, called ST-920, to see if it is safe and if it works to treat Fabry disease. ST-920 is a gene therapy treatment, which means that ST-920 replaces the missing or broken gene you have because you have Fabry disease, with a version or copy that works.

I'm interested

Sex: Male or Female
Age Group: 18 years and over
This study is NOT accepting healthy volunteers
Inclusion Criteria:

• at least 18 years of age
• diagnosis of Fabry disease
• one or more of the following symptoms: i) cornea verticillata, ii) acroparesthesia, iii) anhidrosis, iv) angiokeratoma
• fully vaccinated for COVID- 19 per CDC guidance
• additional requirements apply for cardiac and renal groups (study staff will review)
Exclusion Criteria:

• history of liver disease
• current or history of use in the last six months of systemic steroids
• other significant medical & mental health diagnosis (study staff will review)
Interventions:

Biological: ST-920

Conditions:

Rare Diseases

Keywords:

Fabry Disease

Study Contact: Brenda Diethelm-Okita - dieth001@umn.edu
Principal Investigator: Chester Whitley, MD, PhD
Phase: PHASE1
IRB Number: STUDY00007094
See this study on ClinicalTrials.gov

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