Clinical trial NCT05039866: Long-Term Follow-up of Subjects Who Were Treated With ST-920
Official title: Long-Term Follow-up of Fabry Disease Subjects Who Were Treated With ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy
Study type: OBSERVATIONAL
Overall status: ENROLLING_BY_INVITATION
Conditions: Fabry Disease
Fabry Disease, Cardiac Variant
Keywords: Fabry Disease
Gene Therapy
ST-920
Rare
Genetic
DNA
Sangamo
Long Term
Lead sponsor: Sangamo Therapeutics
Sponsor class: INDUSTRY
First posted: 2021-09-10
Enrollment: 48
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: All subjects who have received ST-920 in separate parent trial and who have consented to participate in this Long Term Follow-up study.

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.

Eligibility criteria: 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

Primary outcomes:
measure: To evaluate long-term safety of ST-920
timeFrame: 4 years
Secondary outcomes:
Other outcomes:
Locations:
city: Irvine
country: United States
facility: University of California, Irvine
state: California
city: Tampa
country: United States
facility: University of South Florida
state: Florida
city: Atlanta
country: United States
facility: Emory University School of Medicine
state: Georgia
city: Iowa City
country: United States
facility: University of Iowa Hospital and Clinics
state: Iowa
city: Minneapolis
country: United States
facility: University of Minnesota Medical Center
state: Minnesota
city: New York
country: United States
facility: Mt. Sinai Hospital
state: New York
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Fairfax
country: United States
facility: Lysosomal & Rare Disorders Research & Treatment Center (LDRTC)
state: Virginia
city: Parkville
country: Australia
facility: The Royal Melbourne Hospital
state: Victoria
city: Calgary
country: Canada
facility: M.A.G.I.C. Clinic Ltd.
state: Alberta
city: Würzburg
country: Germany
facility: University Hospital of Würzburg
city: Cambridge
country: United Kingdom
facility: Addenbrooke's Hospital
city: London
country: United Kingdom
facility: Royal Free Hospital
Study officials:
affiliation: Sangamo Therapeutics, Inc.
name: Medical Monitor
role: STUDY_DIRECTOR
References: