Fabry disease MedDRA version: 24.1 Level: PT Classification code 10016016 Term: Fabry's disease System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: FLT190
Product Code: FLT190
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: NA
Current Sponsor code: FLT190
Other descriptive name: FLT190
Sponsors
Freeline Therapeutics Ltd
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: (i) Subjects who have previously received FLT190 (including those who may have required recommencement/initiation of ERT/PCT). (ii) Subjects able to give 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. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 44 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 6
Exclusion criteria
Exclusion criteria: Non-compliance with inclusion criteria
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of this study is to assess the long-term safety of FLT190 in subjects with Fabry disease. ;Secondary Objective: • To investigate the durability of endogenous production of aGLA enzyme. • To investigate the clearance of Gb3 and LysoGb3 in plasma and urine. • To investigate the clearance of cellular Gb3 inclusions in skin and renal biopsies. • To describe the immune responses to the aGLA transgene product. • To assess viral shedding in various body fluids, where applicable.;Primary end point(s): • The primary safety endpoint will be assessed by the reporting of adverse events (AEs) • Other safety endpoints will be reviewed including: - Laboratory parameter abnormalities - Vital signs, physical examination, liver ultrasound, and electrocardiogram (ECG) abnormalities ;Timepoint(s) of evaluation of this end point: Assessments will run throughout the study | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - Production of aGLA enzyme will be summarised over time and durability investigated. Change from baseline in plasma aGLA expression will be summarised and plotted for each subject and overall. - Change from baseline in Gb3 and LysoGb3 in plasma and urine will be summarised and plotted for each subject and overall. - Change from baseline in cellular Gb3 inclusions in skin and renal biopsies will be summarised and plotted. A detailed description of the variables that will be studied is provided in a separate Biopsy Analysis Plan. - Change from baseline in aGLA antibody levels will be summarised and plotted. Where applicable, descriptive statistics (number of observations, mean, standard deviation, minimum, median, and maximum values) will be calculated for other immune response laboratory tests at applicable visits. - Clearance of vector genomes in blood (plasma), saliva, urine, stool, and semen will be assessed, and levels summarised until no further shedding is seen in a minimum of three consecutive samples. This may occur in the preceding treatment study and, in this case, will not be followed-up further within this protocol. The time to clearance (from FLT190 administration until the third consecutive clear sample) will be summarised and may be plotted. ;Timepoint(s) of evaluation of this end point: Assessments will run throughout the study. Clearance of vector genomes in blood (plasma), saliva, urine, stool, and semen will be assessed, and levels summarised until no further shedding is seen in a minimum of three consecutive samples. | — |
Countries
Austria, Canada, Denmark, France, Germany, Italy, Norway, Portugal, United Kingdom, United States
Contacts
Public ContactClinical Operations
Freeline Therapeutics Ltd
Outcome results
None listed