A Multicenter, Long-term, Follow-up Study to Investigate the Safety and Durability of Response Following Dosing of an Adeno-associated Viral Vector (FLT201) in Subjects with Gaucher Disease (GALILEO-2)

Conditions

Gaucher Disease Type 1

Brief summary

Incidence of treatment-emergent adverse events (TEAEs) (including DLTs)

Detailed description

1. Incidence of AEs, SAEs, and changes from baseline in vital signs, 12-lead ECG, physical examination, and laboratory assessments., 2. Change from baseline in GCase activity (Dried Blood Spot [DBS]), plasma GCase activity and relative concentration, and leukocyte GCase activity over time., 3. Change from baseline in lyso-Gb1 concentration., 4. Change from baseline in hemoglobin, platelet count, spleen volume as measured by MRI, and liver volume as measured by MRI., 5. Change from baseline in GCase antibody levels., 6. Time to clearance of vector genomes in plasma and semen until three consecutive negative samples, where applicable (this may be completed in the preceding treatment trial and, if so, will not be followed-up within this trial.)

Related papers

No related papers found yet.

Interventions

DRUGFLT201

Eligibility

Sex/Gender

All

Age

18 Years to No maximum

Design outcomes

Primary

Measure	Time frame
Incidence of treatment-emergent adverse events (TEAEs) (including DLTs)	—

Secondary

Measure	Time frame
1. Incidence of AEs, SAEs, and changes from baseline in vital signs, 12-lead ECG, physical examination, and laboratory assessments., 2. Change from baseline in GCase activity (Dried Blood Spot [DBS]), plasma GCase activity and relative concentration, and leukocyte GCase activity over time., 3. Change from baseline in lyso-Gb1 concentration., 4. Change from baseline in hemoglobin, platelet count, spleen volume as measured by MRI, and liver volume as measured by MRI., 5. Change from baseline in GCase antibody levels., 6. Time to clearance of vector genomes in plasma and semen until three consecutive negative samples, where applicable (this may be completed in the preceding treatment trial and, if so, will not be followed-up within this trial.)	—

Measure

Time frame

1. Incidence of AEs, SAEs, and changes from baseline in vital signs, 12-lead ECG, physical examination, and laboratory assessments., 2. Change from baseline in GCase activity (Dried Blood Spot [DBS]), plasma GCase activity and relative concentration, and leukocyte GCase activity over time., 3. Change from baseline in lyso-Gb1 concentration., 4. Change from baseline in hemoglobin, platelet count, spleen volume as measured by MRI, and liver volume as measured by MRI., 5. Change from baseline in GCase antibody levels., 6. Time to clearance of vector genomes in plasma and semen until three consecutive negative samples, where applicable (this may be completed in the preceding treatment trial and, if so, will not be followed-up within this trial.)

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Countries

Spain

Outcome results

None listed