Fabry Disease, Fabry Disease, Cardiac Variant
Conditions
Keywords
Fabry Disease, Gene Therapy, ST-920, Rare, Genetic, DNA, Sangamo, Long Term
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.
Interventions
BIOLOGICALST-920
No study drug is administered in this study. Subjects who received ST-920 in a separate parent trial will be evaluated in this trial for long-term safety.
Sponsors
Sangamo Therapeutics
Study design
Observational model
CASE_ONLY
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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) |
Countries
Australia, Canada, Germany, United Kingdom, United States
Contacts
STUDY_DIRECTORMedical Monitor
Sangamo Therapeutics, Inc.
Outcome results
None listed