Trial Outcomes & Findings for A Fabry Disease Gene Therapy Study (NCT NCT04040049)

NCT ID: NCT04040049

Last Updated: 2026-08-06

Results Overview

To investigate the safety of systemic administration of FLT190.

Recruitment status

TERMINATED

Study phase

PHASE1/PHASE2

Target enrollment

3 participants

Primary outcome timeframe

From screening to W38

Results posted on

2026-08-06

Participant Flow

This was a multicenter study with 7 study sites in Germany, UK, Italy, Austria, and Denmark. A total of 11 participants were screened, with 3 dosed with FLT190 at 2 study sites (1 Germany, 1 UK).

Participants were to undergo screening assessments for up to 18 weeks prior to Day 1 (gene therapy infusion). Treatment-eligible participants reported to the infusion trial site on the day prior to receiving the gene therapy infusion (Day -1).

Participant milestones

Participant milestones
Measure	0.75 × 1012 Vector Genomes (vg)/kg FLT190 (Cohort 1) 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).	1.5 × 1012 vg/kg FLT190 (Cohort 2) 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).
Overall Study STARTED	2	1
Overall Study COMPLETED	2	1
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

A Fabry Disease Gene Therapy Study

Baseline characteristics by cohort

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).	FLT190-01: 1.5 × 1012 vg/kg 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).	Total n=3 Participants Total of all reporting groups
Age, Continuous	37 years n=20 Participants	46 years n=20 Participants	40 years n=40 Participants
Sex: Female, Male Female	0 Participants n=20 Participants	0 Participants n=20 Participants	0 Participants n=40 Participants
Sex: Female, Male Male	2 Participants n=20 Participants	1 Participants n=20 Participants	3 Participants n=40 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=20 Participants	0 Participants n=20 Participants	0 Participants n=40 Participants
Race (NIH/OMB) Asian	0 Participants n=20 Participants	0 Participants n=20 Participants	0 Participants n=40 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=20 Participants	0 Participants n=20 Participants	0 Participants n=40 Participants
Race (NIH/OMB) Black or African American	0 Participants n=20 Participants	0 Participants n=20 Participants	0 Participants n=40 Participants
Race (NIH/OMB) White	2 Participants n=20 Participants	1 Participants n=20 Participants	3 Participants n=40 Participants
Race (NIH/OMB) More than one race	0 Participants n=20 Participants	0 Participants n=20 Participants	0 Participants n=40 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=20 Participants	0 Participants n=20 Participants	0 Participants n=40 Participants
Region of Enrollment United Kingdom	2 participants n=20 Participants	1 participants n=20 Participants	3 participants n=40 Participants
Region of Enrollment Germany	2 participants n=20 Participants	1 participants n=20 Participants	3 participants n=40 Participants

PRIMARY outcome

Timeframe: From screening to W38

Population: Three participants were treated, which included: 2 participants treated with 0.75 × 1012 vg/kg (Cohort 1) and 1 participant treated with 1.5 × 1012 vg/kg (Cohort 2). 8 remaining participants were withdrawn due to screening failure. One of the screen failures was withdrawn due to a protocol violation (screening phase was too long) and was subsequently re-screened under a new participant ID, becoming one of the 3 treated participants.

To investigate the safety of systemic administration of FLT190.

Outcome measures

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).
Frequency of Treatment-emergent Adverse Events (AEs) Number of Participants With any Adverse Event	2 participants	1 participants
Frequency of Treatment-emergent Adverse Events (AEs) Any TEAE	2 participants	1 participants
Frequency of Treatment-emergent Adverse Events (AEs) Any serious TEAE	1 participants	1 participants
Frequency of Treatment-emergent Adverse Events (AEs) Any IMP-related TEAE	1 participants	1 participants
Frequency of Treatment-emergent Adverse Events (AEs) Any TEAE leading to death	0 participants	0 participants

Adverse Events

FLT190: 0.75 × 1012 vg/kg

Serious events: 1 serious events

Other events: 2 other events

Deaths: 0 deaths

FLT190:1.5 × 1012 vg/kg

Serious events: 1 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Serious 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).
Cardiac disorders Myocarditis	50.0% 1/2 • Number of events 1 • Adverse events were collected over a 38 week study period.	0.00% 0/1 • Adverse events were collected over a 38 week study period.
Investigations Transaminase increase	50.0% 1/2 • Number of events 1 • Adverse events were collected over a 38 week study period.	100.0% 1/1 • Number of events 1 • Adverse events were collected over a 38 week study period.
General disorders Pyrexia	0.00% 0/2 • Adverse events were collected over a 38 week study period.	100.0% 1/1 • Number of events 1 • Adverse events were collected over a 38 week study period.
Infections and infestations Urinary Tract Infection	0.00% 0/2 • Adverse events were collected over a 38 week study period.	100.0% 1/1 • Number of events 1 • Adverse events were collected over a 38 week study period.

Other adverse events

Additional Information

Spur Clinical Trials Contact

Spur Therapeutics (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

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).
Eye disorders Corneal clouding	50.0% 1/2 • Number of events 1 • Adverse events were collected over a 38 week study period.	0.00% 0/1 • Adverse events were collected over a 38 week study period.
Gastrointestinal disorders Bloating	50.0% 1/2 • Number of events 1 • Adverse events were collected over a 38 week study period.	0.00% 0/1 • Adverse events were collected over a 38 week study period.
Gastrointestinal disorders Nausea	50.0% 1/2 • Number of events 1 • Adverse events were collected over a 38 week study period.	0.00% 0/1 • Adverse events were collected over a 38 week study period.
Gastrointestinal disorders Diarrhea	50.0% 1/2 • Number of events 2 • Adverse events were collected over a 38 week study period.	0.00% 0/1 • Adverse events were collected over a 38 week study period.
Gastrointestinal disorders Abdominal pain	50.0% 1/2 • Number of events 1 • Adverse events were collected over a 38 week study period.	0.00% 0/1 • Adverse events were collected over a 38 week study period.
Gastrointestinal disorders Heartburn	50.0% 1/2 • Number of events 1 • Adverse events were collected over a 38 week study period.	0.00% 0/1 • Adverse events were collected over a 38 week study period.
General disorders Flu-like symptoms	50.0% 1/2 • Number of events 1 • Adverse events were collected over a 38 week study period.	0.00% 0/1 • Adverse events were collected over a 38 week study period.
General disorders Pain	50.0% 1/2 • Number of events 1 • Adverse events were collected over a 38 week study period.	0.00% 0/1 • Adverse events were collected over a 38 week study period.
General disorders Fatigue	100.0% 2/2 • Number of events 2 • Adverse events were collected over a 38 week study period.	0.00% 0/1 • Adverse events were collected over a 38 week study period.
General disorders Pain at blood sampling site	50.0% 1/2 • Number of events 1 • Adverse events were collected over a 38 week study period.	0.00% 0/1 • Adverse events were collected over a 38 week study period.
Immune system disorders Food allergy	50.0% 1/2 • Number of events 1 • Adverse events were collected over a 38 week study period.	0.00% 0/1 • Adverse events were collected over a 38 week study period.
Infections and infestations Rhinitis	50.0% 1/2 • Number of events 1 • Adverse events were collected over a 38 week study period.	100.0% 1/1 • Number of events 1 • Adverse events were collected over a 38 week study period.
Infections and infestations Folliculitis	50.0% 1/2 • Number of events 1 • Adverse events were collected over a 38 week study period.	0.00% 0/1 • Adverse events were collected over a 38 week study period.
Investigations ST elevation	50.0% 1/2 • Number of events 1 • Adverse events were collected over a 38 week study period.	0.00% 0/1 • Adverse events were collected over a 38 week study period.
Investigations Transaminase increase	50.0% 1/2 • Number of events 2 • Adverse events were collected over a 38 week study period.	100.0% 1/1 • Number of events 1 • Adverse events were collected over a 38 week study period.
Musculoskeletal and connective tissue disorders Pain in lower extremity	50.0% 1/2 • Number of events 3 • Adverse events were collected over a 38 week study period.	100.0% 1/1 • Number of events 1 • Adverse events were collected over a 38 week study period.
Musculoskeletal and connective tissue disorders Joint pain	50.0% 1/2 • Number of events 2 • Adverse events were collected over a 38 week study period.	0.00% 0/1 • Adverse events were collected over a 38 week study period.
Musculoskeletal and connective tissue disorders Back pain	50.0% 1/2 • Number of events 1 • Adverse events were collected over a 38 week study period.	0.00% 0/1 • Adverse events were collected over a 38 week study period.
Nervous system disorders Paraesthesia	50.0% 1/2 • Number of events 1 • Adverse events were collected over a 38 week study period.	0.00% 0/1 • Adverse events were collected over a 38 week study period.
Nervous system disorders Headache	50.0% 1/2 • Number of events 7 • Adverse events were collected over a 38 week study period.	0.00% 0/1 • Adverse events were collected over a 38 week study period.
Nervous system disorders Neuralgia	50.0% 1/2 • Number of events 1 • Adverse events were collected over a 38 week study period.	0.00% 0/1 • Adverse events were collected over a 38 week study period.
Respiratory, thoracic and mediastinal disorders Exertional dyspnea	50.0% 1/2 • Number of events 1 • Adverse events were collected over a 38 week study period.	0.00% 0/1 • Adverse events were collected over a 38 week study period.
Respiratory, thoracic and mediastinal disorders Sore throat	50.0% 1/2 • Number of events 3 • Adverse events were collected over a 38 week study period.	0.00% 0/1 • Adverse events were collected over a 38 week study period.
Skin and subcutaneous tissue disorders Acne	50.0% 1/2 • Number of events 1 • Adverse events were collected over a 38 week study period.	0.00% 0/1 • Adverse events were collected over a 38 week study period.
Cardiac disorders Atrial fibrillation	50.0% 1/2 • Number of events 1 • Adverse events were collected over a 38 week study period.	0.00% 0/1 • Adverse events were collected over a 38 week study period.
Injury, poisoning and procedural complications Vein scarring	50.0% 1/2 • Number of events 1 • Adverse events were collected over a 38 week study period.	0.00% 0/1 • Adverse events were collected over a 38 week study period.
Investigations Troponin increase	50.0% 1/2 • Number of events 1 • Adverse events were collected over a 38 week study period.	0.00% 0/1 • Adverse events were collected over a 38 week study period.
Musculoskeletal and connective tissue disorders Muscle atrophy	50.0% 1/2 • Number of events 1 • Adverse events were collected over a 38 week study period.	0.00% 0/1 • Adverse events were collected over a 38 week study period.
General disorders Non-cardiac chest pain	0.00% 0/2 • Adverse events were collected over a 38 week study period.	100.0% 1/1 • Number of events 1 • Adverse events were collected over a 38 week study period.
Infections and infestations UTI	0.00% 0/2 • Adverse events were collected over a 38 week study period.	100.0% 1/1 • Number of events 1 • Adverse events were collected over a 38 week study period.
Investigations Elevated proBNP	0.00% 0/2 • Adverse events were collected over a 38 week study period.	100.0% 1/1 • Number of events 1 • Adverse events were collected over a 38 week study period.
Skin and subcutaneous tissue disorders Angiokeratoma	0.00% 0/2 • Adverse events were collected over a 38 week study period.	100.0% 1/1 • Number of events 1 • Adverse events were collected over a 38 week study period.
Skin and subcutaneous tissue disorders Rash	0.00% 0/2 • Adverse events were collected over a 38 week study period.	100.0% 1/1 • Number of events 1 • Adverse events were collected over a 38 week study period.