Long-Term Follow-up of Subjects Who Were Treated with ST-920

• Conditions: Fabry Disease; Fabry Disease, Cardiac Variant
• Interventions: Biological: ST-920

• Registration Number: NCT05039866
• Lead Sponsor: Sangamo Therapeutics

Brief Summary

Long-term follow-up of subjects who received ST-920 in a previous trial (ST-920-201) and completed at least 52 weeks post-infusion follow-up in their primary protocol. Enrolled subjects will be followed for a total of up to 5 years following ST-920 infusion.

Detailed Description

Non-interventional, multi-center, long-term follow-up (LTFU) study of subjects dosed with ST-920 in the clinical study ST-920-201. All subjects dosed in the study who completed at least 52 weeks post-infusion follow-up in their primary protocol will be offered to participate. Subjects who enroll will be monitored for a total of up to 5 years following ST-920 infusion.

Study Timeline

• Study Start: 2021-08-16
• Study First Submitted: 2021-08-20
• Study First Submitted QC: 2021-09-01
• Study First Posted: 2021-09-10
• Status Verified: 2025-01
• Last Update Submitted: 2025-01-27
• Last Update Posted: 2025-01-29
• Primary Completion: 2029-03
• Study Completion: 2039-03

Recruitment & Eligibility

• Status: ENROLLING_BY_INVITATION
• Sex: All
• Target Recruitment: 48

Inclusion Criteria

• Subjects who received ST-920 therapy in a separate parent trial
• Subjects who have consented to participate in this LTFU study.

Exclusion Criteria

-This study has no exclusion criteria

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Subjects who received ST-920	ST-920	Subjects who received ST-920 in a separate parent trial

Primary Outcome Measures

Name	Time	Method
To evaluate long-term safety of ST-920	4 years	To evaluate long-term safety of ST-920 by assessment of incidence and severity of adverse events (AEs)

Trial Locations

Locations (11)

The Royal Melbourne Hospital; 🇦🇺 Parkville, Victoria, Australia

Addenbrooke's Hospital; 🇬🇧 Cambridge, United Kingdom

M.A.G.I.C. Clinic Ltd.; 🇨🇦 Calgary, Alberta, Canada

University of South Florida; 🇺🇸 Tampa, Florida, United States

University of California, Irvine; 🇺🇸 Irvine, California, United States

Emory University School of Medicine; 🇺🇸 Atlanta, Georgia, United States

University of Iowa Hospital and Clinics; 🇺🇸 Iowa City, Iowa, United States

University of Minnesota Medical Center; 🇺🇸 Minneapolis, Minnesota, United States

Mt. Sinai Hospital; 🇺🇸 New York, New York, United States

Cincinnati Children's Hospital Medical Center; 🇺🇸 Cincinnati, Ohio, United States

Lysosomal & Rare Disorders Research & Treatment Center (LDRTC); 🇺🇸 Fairfax, Virginia, United States