Type 1 Gaucher Disease MedDRA version: 20.0 Level: SOC Classification code 10010331 Term: Congenital, familial and genetic disorders System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0 Level: PT Classification code 10075697 Term: Gaucher's disease type I System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: FLT201
Product Code: FLT201
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: Not yet available
Current Sponsor code: FLT201
Other descriptive name: FLT201
Concentration un
Sponsors
Freeline Therapeutics Limited
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Adult = 18 years of age 2. Diagnosis of Gaucher Disease Type 1 with deficient GCase enzyme activity =30% of normal in leukocytes at diagnosis Part 1 only: previously treated patients (PTP): 3. Treatment status at Screening (Screening period is 12 weeks): a. Off-treatment with enzyme replacement therapy (ERT)/substrate replacement therapy (SRT) for at least 9 months prior to Screening, or b. On treatment with SRT, with no change in regimen for at least 3 months prior to Screening, or c. On-treatment with ERT, with no change in regimen for at least 3 months prior to Screening, and ERT dose =15 U/kg and =60 U/kg every other week (or equivalent) for =24 consecutive months Part 2 only: previously untreated (naïve i.e. never received ERT/SRT) patients (PUP): 4. Patient has a haemoglobin (Hb) level =1 g/dL below the lower limit of normal adjusted for age and sex, and at least one of the following at Screening: a. Platelet count =65 years) yes F.1.3.1 Number of subjects for this age range 3
Exclusion criteria
Exclusion criteria: 1. Diagnosed or suspected Type 2 or Type 3 Gaucher disease 2. Positive for neutralising antibodies to AAVS3 at Screening 3. Evidence of significant liver dysfunction at Sceening defined as >1.5x upper limit of normal (ULN) in ALT, AST or total bilirubin 4. Evidence of any of the following at Screening: (a) Hb <8g/dL (b) Platelets <45,000/mm3 (c) Pulmonary hypertension (d) New osteonecrosis within 12 months of screening (e) Fragility fracture or bone crisis within 12 months of screening 5. History of splenectomy (partial or total)
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess the safety and tolerability of a single intravenous administration of FLT201 in adults with Gaucher disease Type 1;Secondary Objective: • To investigate the relationship of FLT201 dose to endogenous production of GCase • To investigate the clearance of LysoGb1 • To assess the impact of FLT201 on - haemoglobin - platelet count - spleen size - liver size • To assess viral shedding after systemic administration of FLT201 • To describe the immune response to FLT201 transgene product;Primary end point(s): • Incidence of treatment emergent adverse events (TEAEs) including DLTs.;Timepoint(s) of evaluation of this end point: Throughout the study | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Efficacy - Change from baseline to each assessment point in: • LysoGb1 in plasma • Spleen volume by MRI • Liver volume by MRI • Haemoglobin • Platelet count 2. Pharmacokinetic • Change from baseline to each assessment point in plasma and leukocyte GCase activity 3. Viral shedding • Clearance of vg in blood, urine, saliva, stool and semen 4. Immune response to Gcase transgene product. • Change from baseline to each assessment point in anti-GCase antibody titre and neutralising antibody titre;Timepoint(s) of evaluation of this end point: • Patients to be evaluated at baseline, throughout the study, and at the end of the study at week 38. | — |
Countries
Argentina, Brazil, Germany, Israel, Italy, Paraguay, Spain, Tunisia, United Kingdom
Contacts
Public ContactThorold Guy
Freeline Therapeutics Limited
Outcome results
None listed