Trial Outcomes & Findings for A Long Term Follow-Up Study of Fabry Disease Subjects Treated With FLT190 (NCT NCT04455230)
NCT ID: NCT04455230
Last Updated: 2026-09-03
Results Overview
Recruitment status
TERMINATED
Study phase
PHASE1/PHASE2
Target enrollment
3 participants
Primary outcome timeframe
Mean: 1.72 years; range: 0.43- 3.10 years (From enrollment in LTFU)
Results posted on
2026-09-03
Participant Flow
This was a long-term follow up study of patients from FLT190-01.
Only patients having been dosed in FLT190-01 were enrolled.
Participant milestones
| Measure |
FLT190: 0.75 × 1012 vg/kg
All treated patients received FLT190. Three participants were treated, which included: 2 participants treated with 0.75 × 1012 vector genomes (vg)/kg FLT190 (Cohort 1) and 1 participant treated with 1.5 × 1012 vg/kg FLT190 (Cohort 2). All 3 treated participants entered the long-term follow up study (FLT190-02) and were subsequently discontinued due to the Sponsor's early termination of the trial.
|
FLT190-01: 1.5 × 1012 vg/kg FLT190
All treated patients received FLT190. Three participants were treated, which included: 2 participants treated with 0.75 × 1012 vector genomes (vg)/kg FLT190 (Cohort 1) and 1 participant treated with 1.5 × 1012 vg/kg FLT190 (Cohort 2). All 3 treated participants entered the long-term follow up study (FLT190-02) and were subsequently discontinued due to the Sponsor's early termination of the trial.
|
|---|---|---|
|
Overall Study
STARTED
|
2
|
1
|
|
Overall Study
COMPLETED
|
0
|
0
|
|
Overall Study
NOT COMPLETED
|
2
|
1
|
Reasons for withdrawal
| Measure |
FLT190: 0.75 × 1012 vg/kg
All treated patients received FLT190. Three participants were treated, which included: 2 participants treated with 0.75 × 1012 vector genomes (vg)/kg FLT190 (Cohort 1) and 1 participant treated with 1.5 × 1012 vg/kg FLT190 (Cohort 2). All 3 treated participants entered the long-term follow up study (FLT190-02) and were subsequently discontinued due to the Sponsor's early termination of the trial.
|
FLT190-01: 1.5 × 1012 vg/kg FLT190
All treated patients received FLT190. Three participants were treated, which included: 2 participants treated with 0.75 × 1012 vector genomes (vg)/kg FLT190 (Cohort 1) and 1 participant treated with 1.5 × 1012 vg/kg FLT190 (Cohort 2). All 3 treated participants entered the long-term follow up study (FLT190-02) and were subsequently discontinued due to the Sponsor's early termination of the trial.
|
|---|---|---|
|
Overall Study
Early closure of study
|
2
|
1
|
Baseline Characteristics
Number of participants by region
Baseline characteristics by cohort
| Measure |
FLT190: 0.75 × 1012 vg/kg
n=2 Participants
All treated patients received FLT190. Three participants were treated, which included: 2 participants treated with 0.75 × 1012 vector genomes (vg)/kg FLT190 (Cohort 1) and 1 participant treated with 1.5 × 1012 vg/kg FLT190 (Cohort 2). All 3 treated participants entered the long-term follow up study (FLT190-02) and were subsequently discontinued due to the Sponsor's early termination of the trial
|
FLT190-01: 1.5 × 1012 vg/kg FLT190
n=1 Participants
All treated patients received FLT190. Three participants were treated, which included: 2 participants treated with 0.75 × 1012 vector genomes (vg)/kg FLT190 (Cohort 1) and 1 participant treated with 1.5 × 1012 vg/kg FLT190 (Cohort 2). All 3 treated participants entered the long-term follow up study (FLT190-02) and were subsequently discontinued due to the Sponsor's early termination of the trial.
|
Total
n=3 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
37 Years
n=136 Participants
|
46 Years
n=136 Participants
|
40 Years
n=272 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=136 Participants
|
0 Participants
n=136 Participants
|
0 Participants
n=272 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=136 Participants
|
1 Participants
n=136 Participants
|
3 Participants
n=272 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=136 Participants
|
0 Participants
n=136 Participants
|
0 Participants
n=272 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=136 Participants
|
0 Participants
n=136 Participants
|
0 Participants
n=272 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=136 Participants
|
0 Participants
n=136 Participants
|
0 Participants
n=272 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=136 Participants
|
0 Participants
n=136 Participants
|
0 Participants
n=272 Participants
|
|
Race (NIH/OMB)
White
|
2 Participants
n=136 Participants
|
1 Participants
n=136 Participants
|
3 Participants
n=272 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=136 Participants
|
0 Participants
n=136 Participants
|
0 Participants
n=272 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=136 Participants
|
0 Participants
n=136 Participants
|
0 Participants
n=272 Participants
|
|
Region of Enrollment
Germany
|
1 Participants
n=136 Participants • Number of participants by region
|
0 Participants
n=136 Participants • Number of participants by region
|
1 Participants
n=272 Participants • Number of participants by region
|
|
Region of Enrollment
United Kingdom
|
1 Participants
n=136 Participants • Number of participants by region
|
1 Participants
n=136 Participants • Number of participants by region
|
2 Participants
n=272 Participants • Number of participants by region
|
PRIMARY outcome
Timeframe: Mean: 1.72 years; range: 0.43- 3.10 years (From enrollment in LTFU)Outcome measures
| Measure |
FLT190: 0.75 × 1012 vg/kg
n=2 Participants
2 participants treated with 0.75 × 1012 vg/kg FLT190 (Cohort 1)
|
FLT190:1.5 × 1012 vg/kg
n=1 Participants
1 participant treated with 1.5 × 1012 vg/kg FLT190 (Cohort 2)
|
|---|---|---|
|
Safety Endpoint Will be Assessed by the Reporting of Adverse Events (AEs) According to Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0.
Number of Participants With any Adverse Event
|
2 Participants
|
0 Participants
|
|
Safety Endpoint Will be Assessed by the Reporting of Adverse Events (AEs) According to Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0.
Any TEAE
|
2 Participants
|
0 Participants
|
|
Safety Endpoint Will be Assessed by the Reporting of Adverse Events (AEs) According to Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0.
Any serious TEAE
|
0 Participants
|
0 Participants
|
|
Safety Endpoint Will be Assessed by the Reporting of Adverse Events (AEs) According to Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0.
Any IMP-related TEAE
|
0 Participants
|
0 Participants
|
|
Safety Endpoint Will be Assessed by the Reporting of Adverse Events (AEs) According to Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0.
Any TEAE leading to death
|
0 Participants
|
0 Participants
|
Adverse Events
FLT190: 0.75 × 1012 vg/kg
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
FLT190:1.5 × 1012 vg/kg
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
FLT190: 0.75 × 1012 vg/kg
n=2 participants at risk
2 participants treated with 0.75 × 1012 vg/kg FLT190 (Cohort 1)
|
FLT190:1.5 × 1012 vg/kg
n=1 participants at risk
1 participant treated with 1.5 × 1012 vg/kg FLT190 (Cohort 2).
|
|---|---|---|
|
Infections and infestations
COVID-19
|
50.0%
1/2 • Number of events 1 • Mean: 1.72 years; range: 0.43- 3.10 years (From enrollment in LTFU)
|
0.00%
0/1 • Mean: 1.72 years; range: 0.43- 3.10 years (From enrollment in LTFU)
|
|
Metabolism and nutrition disorders
Polydypsia
|
50.0%
1/2 • Number of events 1 • Mean: 1.72 years; range: 0.43- 3.10 years (From enrollment in LTFU)
|
0.00%
0/1 • Mean: 1.72 years; range: 0.43- 3.10 years (From enrollment in LTFU)
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
50.0%
1/2 • Number of events 1 • Mean: 1.72 years; range: 0.43- 3.10 years (From enrollment in LTFU)
|
0.00%
0/1 • Mean: 1.72 years; range: 0.43- 3.10 years (From enrollment in LTFU)
|
|
Psychiatric disorders
Attention deficit hyperactivity disorder
|
50.0%
1/2 • Number of events 1 • Mean: 1.72 years; range: 0.43- 3.10 years (From enrollment in LTFU)
|
0.00%
0/1 • Mean: 1.72 years; range: 0.43- 3.10 years (From enrollment in LTFU)
|
|
Infections and infestations
Respiratory tract infection
|
50.0%
1/2 • Number of events 1 • Mean: 1.72 years; range: 0.43- 3.10 years (From enrollment in LTFU)
|
0.00%
0/1 • Mean: 1.72 years; range: 0.43- 3.10 years (From enrollment in LTFU)
|
|
Infections and infestations
Borrelia infection
|
50.0%
1/2 • Number of events 1 • Mean: 1.72 years; range: 0.43- 3.10 years (From enrollment in LTFU)
|
0.00%
0/1 • Mean: 1.72 years; range: 0.43- 3.10 years (From enrollment in LTFU)
|
Additional Information
Spur Clinical Trials Contact
Spur Theraputics (formerly known as Freeline Therapeutics Ltd)
Phone: +44 (0) 1438 906870
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place