Haemophilia B MedDRA version: 20.0 Level: LLT Classification code 10018939 Term: Haemophilia B (Factor IX) System Organ Class: 100000004850
Conditions
Interventions
Product Name: FLT180a
Product Code: FLT180a
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: NA
Current Sponsor code: FLT180A
Other descriptive name: FLT180A
Concentrati
Sponsors
University College London (UCL)
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: 1. Adults males, = 18 years of age; 2. Confirmed diagnosis of HB defined as one of the following: Documented severe FIX deficiency with plasma FIX activity of =65 years) yes F.1.3.1 Number of subjects for this age range 8
Exclusion criteria
Exclusion criteria: 1. Presence of neutralising anti-human FIX antibodies (inhibitor, determined by the Bethesda inhibitor assay) at the time of enrolment or a previous history of FIX inhibitor; 2. Patients at high risk of thromboembolic events (high risk patients would include those with a history of arterial or venous thromboembolism (e.g. deep vein thrombosis, pulmonary embolism, non-haemorrhagic stroke, arterial embolus) and those with acquired thrombophilia including conditions such as atrial fibrilation). 3. Use of investigational therapy for haemophilia within 30 days before enrolment; 4. Patients with active hepatitis B or C, and HBsAg or HCV RNA viral load positivity, respectively, or currently on antiviral therapy for hepatitis B or C. (Negative viral assays in 2 samples, collected at least 5 months apart, will be required to be considered negative. Both natural clearers and those who have cleared HCV on antiviral therapy are eligible.); 5. Serological evidence of HIV-1; 6. Evidence of liver dysfunction (persistently elevated alanine aminotransferase, aspartate aminotransferase, bilirubin >1.5 x upper limit of normal). 7. Platelet count 1); 12. Prior treatment with any gene transfer medicinal product; 13. Known or suspected intolerance, hypersensitivity or contrainidication to the investigational product and non-investigational medicinal products or their excipients; 14.Planned major elective surgery prior to the end of trial. 15. Current or relevant history of a physical or psychiatric illness or any medical condition that in the opinion of the investigator could affect the patients safety or interfere with the study assessments. 16. CMV IgG positive patients who are CMV PCR positive at screening.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Primary end point(s): Safety Safety as assessed by the reporting of AEs according to Common Terminology Criteria for Adverse Events (CTCAE) v5.0. Efficacy The following primary endpoints will be analysed: 1. The proportion of patients achieving clinical FIX response at Week 26, at the terminal dose level. A clinical FIX response is defined as achieving FIX activity of 5% to 150%. 2. The proportion of patients also achieving normalized FIX response at Week 26, at the terminal dose level. A normalized FIX response is defined as achieving FIX activity in the normal range (50-150%);Timepoint(s) of evaluation of this end point: Safety Throughout the study Efficacy Week 26;Main Objective: Safety To assess the safety of systemic administration of FLT180a in adults with Haemophilia B at up to 3 different dose cohorts. Efficacy To assess Factor IX (FIX) levels following systemic administration of FLT180a, at the terminal dose level.;Secondary Objective: To investigate the endogenous production of FIX following systemic administration of FLT 180a at up to 3 different dose cohorts. To investigate the effectiveness of a single administration of FLT180a on annualized bleeding rate and exogenous FIX consumption. To assess the immune response to the FIX transgene product following systemic administration of FLT180a. To assess viral shedding in various body fluids after systemic administration of FLT180a. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Safety Safety as assessed by reporting of abnormal or change from baseline findings from safety assessments including laboratory assessments, vital signs, ECG, physical exam and liver ultrasound. 1. Endogenous FIX (hFIX) production 2. Haemostatic effectiveness 3. Immune response 4. Shedding ;Timepoint(s) of evaluation of this end point: 1. Throughout study 2. Throughout study 3. Throughout study 4. Throughout study or until 3 consecutive samples test negative for vector sequences. | — |
Countries
United Kingdom, United States
Contacts
Public ContactRegulatory Manager
University College London
Outcome results
None listed