Fabry Disease, Lysosomal Storage Diseases
Conditions
Keywords
Gene Therapy
Brief summary
Fabry disease is a rare, X-linked inborn error of glycosphingolipid metabolism caused by an abnormal gene encoding the α-galactosidase A (αGLA) enzyme. The αGLA enzyme is ubiquitously expressed throughout the body and is responsible for the breakdown of glycosphingolipids, deficiency of which results in the accumulation of specific glycosphingolipids that are associated with the pathophysiology of the disease. Current treatment for Fabry disease is limited to the symptomatic management of pain, conventional management of complications, and methods to increase the availability of functional αGLA. This clinical study aims to investigate the long-term safety and durability of αGLA in patients who have been dosed with a new gene therapy product (FLT190) in earlier clinical studies.
Interventions
GENETICFLT190
FLT190 is a recombinant adeno-associated viral (AAV) vector (AAVS3) containing the human αGLA gene as a single stranded (ss) deoxyribonucleic acid (DNA).
Sponsors
Spur Therapeutics
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
OTHER
Masking
NONE
Intervention model description
A Multicenter, Long-term, Follow-up Study to Investigate the Safety and Durability of Response Following Dosing of an Adeno-associated Viral Vector (FLT190) in Subjects with Fabry Disease
Eligibility
Sex/Gender
MALE
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Subjects who have previously received FLT190 * Provision of full informed consent and able to comply with all requirements of the study including long-term follow-up for 60 months (5 years) post-treatment.
Exclusion criteria
* N/A
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Safety Endpoint Will be Assessed by the Reporting of Adverse Events (AEs) According to Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0. | Mean: 1.72 years; range: 0.43- 3.10 years (From enrollment in LTFU) |
Countries
Germany, United Kingdom
Participant flow
Recruitment details
This was a long-term follow up study of patients from FLT190-01.
Pre-assignment details
Only patients having been dosed in FLT190-01 were enrolled.
Baseline characteristics
| Characteristic | — |
|---|---|
| Age, Continuous | 40 Years |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 3 Participants |
| Region of Enrollment Germany | 0 Participants |
| Region of Enrollment United Kingdom | 1 Participants |
| Sex: Female, Male Female | 0 Participants |
| Sex: Female, Male Male | 2 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 2 | 0 / 1 |
| other Total, other adverse events | 2 / 2 | 0 / 1 |
| serious Total, serious adverse events | 0 / 2 | 0 / 1 |
Outcome results
None listed