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Trial Outcomes & Findings for A Gene Therapy Study in Patients With Gaucher Disease Type 1 (NCT NCT05324943)

NCT ID: NCT05324943

Last Updated: 2026-06-02

Results Overview

Treatment-emergent adverse events (including dose-limiting toxicities), with AEs graded as mild/moderate/severe.

Recruitment status

COMPLETED

Study phase

PHASE1

Target enrollment

10 participants

Primary outcome timeframe

Day 1 (dosing) through transfer to the long-term follow-up (LTFU) study, which was up to 15 months.

Results posted on

2026-06-02

Participant Flow

This was a multicenter study with 15 study sites in USA, Brazil, Paraguay, Spain, Germany, UK, and Israel. Ten participants met all the inclusion criteria for the trial and were enrolled, with 6 participants ultimately being dosed with FLT201 at 4 trial sites in Brazil, Spain, UK, and USA.

Participants were to undergo screening assessments for up to 16 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
FLT201
FLT201 (4.5 × 10\^11 vg/kg) was administered in the controlled environment of a trial site, which had been assessed for its ability to store, handle, and administer gene therapy products per local regulations, as well as their ability to comply with procedures in the FLT201 Pharmacy Manual. The administration of FLT201 was performed by suitably qualified and trained trial staff. Investigation has not been conducted with additional dose cohorts. Duration of treatment is 38 week/9 month.
Overall Study
STARTED
6
Overall Study
COMPLETED
6
Overall Study
NOT COMPLETED
0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

A Gene Therapy Study in Patients With Gaucher Disease Type 1

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
FLT201
n=6 Participants
FLT201 (4.5 × 10\^11 vg/kg) was administered in the controlled environment of a trial site, which had been assessed for its ability to store, handle, and administer gene therapy products per local regulations, as well as their ability to comply with procedures in the FLT201 Pharmacy Manual. The administration of FLT201 was performed by suitably qualified and trained trial staff.
Age, Categorical
<=18 years
0 Participants
n=9 Participants
Age, Categorical
Between 18 and 65 years
6 Participants
n=9 Participants
Age, Categorical
>=65 years
0 Participants
n=9 Participants
Age, Continuous
32.7 Years
STANDARD_DEVIATION 13.14 • n=9 Participants
Sex: Female, Male
Female
2 Participants
n=9 Participants
Sex: Female, Male
Male
4 Participants
n=9 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=9 Participants
Race (NIH/OMB)
Asian
0 Participants
n=9 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=9 Participants
Race (NIH/OMB)
Black or African American
1 Participants
n=9 Participants
Race (NIH/OMB)
White
5 Participants
n=9 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=9 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=9 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
3 Participants
n=9 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
3 Participants
n=9 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants
n=9 Participants
Region of Enrollment
Spain
1 Participants
n=9 Participants
Region of Enrollment
Brazil
2 Participants
n=9 Participants
Region of Enrollment
United States
2 Participants
n=9 Participants
Region of Enrollment
United Kingdom
1 Participants
n=9 Participants

PRIMARY outcome

Timeframe: Day 1 (dosing) through transfer to the long-term follow-up (LTFU) study, which was up to 15 months.

Treatment-emergent adverse events (including dose-limiting toxicities), with AEs graded as mild/moderate/severe.

Outcome measures

Outcome measures
Measure
FLT201
n=6 Participants
FLT201 (4.5 × 10\^11 vg/kg) was administered in the controlled environment of a trial site, which had been assessed for its ability to store, handle, and administer gene therapy products per local regulations, as well as their ability to comply with procedures in the FLT201 Pharmacy Manual. The administration of FLT201 was performed by suitably qualified and trained trial staff. Investigation has not been conducted with additional dose cohorts.
FLT201 (9 Month)
FLT201 (4.5 × 10\^11 vg/kg) was administered in the controlled environment of a trial site, which had been assessed for its ability to store, handle, and administer gene therapy products per local regulations, as well as their ability to comply with procedures in the FLT201 Pharmacy Manual. The administration of FLT201 was performed by suitably qualified and trained trial staff. Investigation has not been conducted with additional dose cohorts. Duration of treatment is 9 month.
Number of Participants With Treatment-emergent Adverse Events Over Time
Any IMP-related serious TEAE
2 Participants
—
Number of Participants With Treatment-emergent Adverse Events Over Time
Any TEAE leading to treatment discontinuation
0 Participants
—
Number of Participants With Treatment-emergent Adverse Events Over Time
Any IMP-related TEAE leading to treatment discontinuation
0 Participants
—
Number of Participants With Treatment-emergent Adverse Events Over Time
Any TEAE leading to death
0 Participants
—
Number of Participants With Treatment-emergent Adverse Events Over Time
Any IMP-related TEAE leading to death
0 Participants
—
Number of Participants With Treatment-emergent Adverse Events Over Time
Any AE
6 Participants
—
Number of Participants With Treatment-emergent Adverse Events Over Time
Any TEAE
6 Participants
—
Number of Participants With Treatment-emergent Adverse Events Over Time
Any IMP-related TEAE
6 Participants
—
Number of Participants With Treatment-emergent Adverse Events Over Time
Any serious TEAE
2 Participants
—
Number of Participants With Treatment-emergent Adverse Events Over Time
Any non-serious TEAE
6 Participants
—

SECONDARY outcome

Timeframe: From baseline to week 38/9 months.

Change from baseline to week 38/month 9 of lyso-Gb1 in plasma.

Outcome measures

Outcome measures
Measure
FLT201
n=4 Participants
FLT201 (4.5 × 10\^11 vg/kg) was administered in the controlled environment of a trial site, which had been assessed for its ability to store, handle, and administer gene therapy products per local regulations, as well as their ability to comply with procedures in the FLT201 Pharmacy Manual. The administration of FLT201 was performed by suitably qualified and trained trial staff. Investigation has not been conducted with additional dose cohorts.
FLT201 (9 Month)
n=2 Participants
FLT201 (4.5 × 10\^11 vg/kg) was administered in the controlled environment of a trial site, which had been assessed for its ability to store, handle, and administer gene therapy products per local regulations, as well as their ability to comply with procedures in the FLT201 Pharmacy Manual. The administration of FLT201 was performed by suitably qualified and trained trial staff. Investigation has not been conducted with additional dose cohorts. Duration of treatment is 9 month.
Change From Baseline in Lyso-Gb1 in Plasma
-15.478 ng/ml
Standard Deviation 32.1514
-19.860 ng/ml
Standard Deviation 20.8314

SECONDARY outcome

Timeframe: From baseline to week 38/9 months.

Change from baseline to week 38/month 9 in spleen volume measured by MRI.

Outcome measures

Outcome measures
Measure
FLT201
n=4 Participants
FLT201 (4.5 × 10\^11 vg/kg) was administered in the controlled environment of a trial site, which had been assessed for its ability to store, handle, and administer gene therapy products per local regulations, as well as their ability to comply with procedures in the FLT201 Pharmacy Manual. The administration of FLT201 was performed by suitably qualified and trained trial staff. Investigation has not been conducted with additional dose cohorts.
FLT201 (9 Month)
n=2 Participants
FLT201 (4.5 × 10\^11 vg/kg) was administered in the controlled environment of a trial site, which had been assessed for its ability to store, handle, and administer gene therapy products per local regulations, as well as their ability to comply with procedures in the FLT201 Pharmacy Manual. The administration of FLT201 was performed by suitably qualified and trained trial staff. Investigation has not been conducted with additional dose cohorts. Duration of treatment is 9 month.
Change From Baseline in Spleen Volume Measured by MRI
-49.453 mL
Standard Deviation 75.3262
-44.545 mL
Standard Deviation 111.8855

SECONDARY outcome

Timeframe: From baseline to week 38/9 months.

Change from baseline to week 38/month 9 in liver volume measured by MRI.

Outcome measures

Outcome measures
Measure
FLT201
n=3 Participants
FLT201 (4.5 × 10\^11 vg/kg) was administered in the controlled environment of a trial site, which had been assessed for its ability to store, handle, and administer gene therapy products per local regulations, as well as their ability to comply with procedures in the FLT201 Pharmacy Manual. The administration of FLT201 was performed by suitably qualified and trained trial staff. Investigation has not been conducted with additional dose cohorts.
FLT201 (9 Month)
n=2 Participants
FLT201 (4.5 × 10\^11 vg/kg) was administered in the controlled environment of a trial site, which had been assessed for its ability to store, handle, and administer gene therapy products per local regulations, as well as their ability to comply with procedures in the FLT201 Pharmacy Manual. The administration of FLT201 was performed by suitably qualified and trained trial staff. Investigation has not been conducted with additional dose cohorts. Duration of treatment is 9 month.
Change From Baseline in Liver Volume Measured by MRI
-20.657 mL
Standard Deviation 47.5816
41.435 mL
Standard Deviation 87.1651

SECONDARY outcome

Timeframe: From baseline to week 38/9 months.

Change from baseline to week 38/month 9 in hemoglobin.

Outcome measures

Outcome measures
Measure
FLT201
n=4 Participants
FLT201 (4.5 × 10\^11 vg/kg) was administered in the controlled environment of a trial site, which had been assessed for its ability to store, handle, and administer gene therapy products per local regulations, as well as their ability to comply with procedures in the FLT201 Pharmacy Manual. The administration of FLT201 was performed by suitably qualified and trained trial staff. Investigation has not been conducted with additional dose cohorts.
FLT201 (9 Month)
n=2 Participants
FLT201 (4.5 × 10\^11 vg/kg) was administered in the controlled environment of a trial site, which had been assessed for its ability to store, handle, and administer gene therapy products per local regulations, as well as their ability to comply with procedures in the FLT201 Pharmacy Manual. The administration of FLT201 was performed by suitably qualified and trained trial staff. Investigation has not been conducted with additional dose cohorts. Duration of treatment is 9 month.
Change From Baseline in Hemoglobin
0.03 g/dL
Standard Deviation 1.384
0.30 g/dL
Standard Deviation 1.697

SECONDARY outcome

Timeframe: From baseline to week 38/9 months.

Change from baseline to week 38/month 9 in platelet count.

Outcome measures

Outcome measures
Measure
FLT201
n=4 Participants
FLT201 (4.5 × 10\^11 vg/kg) was administered in the controlled environment of a trial site, which had been assessed for its ability to store, handle, and administer gene therapy products per local regulations, as well as their ability to comply with procedures in the FLT201 Pharmacy Manual. The administration of FLT201 was performed by suitably qualified and trained trial staff. Investigation has not been conducted with additional dose cohorts.
FLT201 (9 Month)
n=2 Participants
FLT201 (4.5 × 10\^11 vg/kg) was administered in the controlled environment of a trial site, which had been assessed for its ability to store, handle, and administer gene therapy products per local regulations, as well as their ability to comply with procedures in the FLT201 Pharmacy Manual. The administration of FLT201 was performed by suitably qualified and trained trial staff. Investigation has not been conducted with additional dose cohorts. Duration of treatment is 9 month.
Change From Baseline in Platelet Count
38.8 10^9 platelets/L
Standard Deviation 75.61
-4.0 10^9 platelets/L
Standard Deviation 28.28

Adverse Events

FLT201

Serious events: 2 serious events
Other events: 6 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
FLT201
n=6 participants at risk
FLT201 (4.5 × 10\^11 vg/kg) was administered in the controlled environment of a trial site, which had been assessed for its ability to store, handle, and administer gene therapy products per local regulations, as well as their ability to comply with procedures in the FLT201 Pharmacy Manual. The administration of FLT201 was performed by suitably qualified and trained trial staff.
Investigations
Neutralising antibodies
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Investigations
Neutralising antibodies positive
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.

Other adverse events

Other adverse events
Measure
FLT201
n=6 participants at risk
FLT201 (4.5 × 10\^11 vg/kg) was administered in the controlled environment of a trial site, which had been assessed for its ability to store, handle, and administer gene therapy products per local regulations, as well as their ability to comply with procedures in the FLT201 Pharmacy Manual. The administration of FLT201 was performed by suitably qualified and trained trial staff.
General disorders
Peripheral swelling
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
General disorders
Temperature intolerance
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Skin papilloma
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Vascular disorders
Blood pressure fluctuation
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Vascular disorders
Hypertension
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Vascular disorders
Hypotension
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Vascular disorders
Peripheral coldness
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
General disorders
Adverse drug reaction
33.3%
2/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
General disorders
Discomfort
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
General disorders
Fatigue
50.0%
3/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
General disorders
Malaise
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Respiratory, thoracic and mediastinal disorders
Cough
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Respiratory, thoracic and mediastinal disorders
Nasal congestion
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Respiratory, thoracic and mediastinal disorders
Rhinitis allergic
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Psychiatric disorders
Anxiety
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Psychiatric disorders
Mood altered
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Psychiatric disorders
Panic attack
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Investigations
Activated partial thromboplastin
33.3%
2/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Investigations
Alanine aminotransferase increased
100.0%
6/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Investigations
Aspartate aminotransferase increased
33.3%
2/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Investigations
Cardiac murmur
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Investigations
Electrocardiogram ST segment elevation
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Investigations
Fibrin D dimer increased
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Investigations
Glomerular filtration rate decreased
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Investigations
Immature granulocyte count increased
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Investigations
Mean cell volume increased
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Investigations
Neutrophil count increased
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Investigations
Prothrombin time prolonged
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Investigations
Scan bone marrow abnormal
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Investigations
Weight increased
33.3%
2/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Injury, poisoning and procedural complications
Contusion
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Injury, poisoning and procedural complications
Cardiomegaly
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Injury, poisoning and procedural complications
Palpitations
33.3%
2/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Nervous system disorders
Action tremor
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Nervous system disorders
Burning sensation
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Nervous system disorders
Headache
50.0%
3/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Nervous system disorders
Migraine
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Nervous system disorders
Tremor
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Blood and lymphatic system disorders
Leukocytosis
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Blood and lymphatic system disorders
Leukopenia
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Blood and lymphatic system disorders
Lymphopenia
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Blood and lymphatic system disorders
Neutropenia
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Blood and lymphatic system disorders
Neutrophilia
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Blood and lymphatic system disorders
Splenomegaly
33.3%
2/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Blood and lymphatic system disorders
Thrombocytopenia
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Ear and labyrinth disorders
Ear pain
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Eye disorders
Visual field defect
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Eye disorders
Vitreous floaters
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Gastrointestinal disorders
Abdominal distension
33.3%
2/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Gastrointestinal disorders
Abdominal pain
33.3%
2/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Gastrointestinal disorders
Abdominal pain upper
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Gastrointestinal disorders
Constipation
33.3%
2/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Gastrointestinal disorders
Diarrhoea
83.3%
5/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Gastrointestinal disorders
Dyspepsia
33.3%
2/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Gastrointestinal disorders
Flatulence
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Gastrointestinal disorders
Frequent bowel movements
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Gastrointestinal disorders
Vomiting
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Skin and subcutaneous tissue disorders
Dermatitis
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Skin and subcutaneous tissue disorders
Hyperhidrosis
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Skin and subcutaneous tissue disorders
Macule
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Skin and subcutaneous tissue disorders
Pruritus
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Skin and subcutaneous tissue disorders
Skin atrophy
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Renal and urinary disorders
Micturition urgency
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Renal and urinary disorders
Oliguria
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Renal and urinary disorders
Pollakiuria
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Renal and urinary disorders
Proteinuria
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Renal and urinary disorders
Urinary incontinence
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Musculoskeletal and connective tissue disorders
Arthralgia
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Musculoskeletal and connective tissue disorders
Muscle spasms
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Musculoskeletal and connective tissue disorders
Musculoskeletal chest pain
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Musculoskeletal and connective tissue disorders
Pain in extremity
50.0%
3/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Infections and infestations
Epstein-Barr virus infection
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Infections and infestations
Folliculitis
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Infections and infestations
Gastroenteritis
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Infections and infestations
Gastrointestinal infection
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Infections and infestations
Hordeolum
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Infections and infestations
Nasopharyngitis
33.3%
2/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Infections and infestations
Pharyngitis
33.3%
2/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Infections and infestations
Sinusitis
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Infections and infestations
Urinary tract infection
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Metabolism and nutrition disorders
Decreased appetite
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Metabolism and nutrition disorders
Dehydration
33.3%
2/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Metabolism and nutrition disorders
Hyperglycaemia
50.0%
3/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.
Metabolism and nutrition disorders
Vitamin D deficiency
16.7%
1/6 • The time-period for reporting AEs was from baseline until transfer to the long-term follow-up (LTFU) study, which was up to 15 months.
Incidence of treatment-emergent adverse events (TEAEs; including dose limiting toxicities) from Day 1 to the last follow-up visit. Two Important Medical Events were reported in line with SAE procedures per protocol. No AEs met the criteria for SAEs.

Additional Information

Spur Clinical Trials Contact

Spur Therapeutics (formerly known as Freeline Therapeutics Ltd)

Phone: +44 (0)1438 906870

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place