Non-transfusion Dependent Beta-thalassaemia MedDRA version: 20.0 Level: LLT Classification code 10074355 Term: Non-transfusion dependent thalassaemia System Organ Class: 100000004850
Conditions
Interventions
Product Name: -
Product Code: [VIT-2763]
Pharmaceutical Form: Capsule, hard
Current Sponsor code: VIT-2763
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 60-
Pharm
Sponsors
VIFOR (INTERNATIONAL) INC.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Documented diagnosis of NTDT, including a ß-thalassaemia intermedia-phenotype. 2. NTDT is defined as subjects having received =65 years) yes F.1.3.1 Number of subjects for this age range 1
Exclusion criteria
Exclusion criteria: 1. Documented diagnosis of TDT, including a beta-thalassaemia major phenotype (including ß0/ß0, ß+/ß+, ß0/ß+ genotype), and mixed compound heterozygous for sickling phenotype variants such as Hb S/ß-thalassaemia, or transfusion dependent non-deletional Hb H disease (i.e., Hb constant spring) or Hb C disease. 2. Subjects on concomitant ICT or subjects on prior ICT when discontinued less than 4 weeks prior randomisation. If ICT was discontinued >=4 weeks prior randomization the subject is eligible. 3. ICT naïve subjects with serum ferritin 15 mg/g liver dry weight assessed through MRI, or a documented myocardial T2* 100 kg at screening. 7. Chronic liver disease and/or ALT, AST or GGT above 3-fold the ULN range at screening. Note: A subject fulfilling this criterion will be excluded but can be rescreened at a later time point (in order to fulfil eligibility, =2 values within =1 week should be assessed and be within eligibility limits). 8. eGFR 30 mg/mmol. eGFR should be estimated according to Chronic Kidney Disease Epidemiology Collaboration formula (CKI-EPI) in adults, and Schwartz formula in adolescents. 9. Newly diagnosed folate deficiency anaemia and/or Vitamin B12 megaloblastic anaemia. Subjects with known folate deficiency anaemia and/or Vitamin B12 megaloblastic anaemia who are on =12 weeks stable replacement therapy are eligible. Note: A subject fulfilling this criterion will be excluded but can be rescreened at a later time point. 10. Any history or clinically important finding of cardiac disorders, such as clinically relevant cardiac arrhythmia, cardiomyopathy, coronary disease, valve disorder, or heart failure according to New York Heart Association classification 3-4. 11. Subjects with history of partial or total splenectomy within 6 months prior to screening.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess the safety and tolerability of VIT-2763 versus placebo in adult and adolescent NTDT subjects over a 12-week treatment period;Secondary Objective: - To assess the preliminary efficacy of VIT-2763 versus placebo on iron markers in adult and adolescent NTDT subjects over a 12-week treatment period. - To evaluate the PK of VIT-2763 in adult and adolescent NTDT subjects over a 12-week treatment period (using a population PK approach);Primary end point(s): • Reported or observed AEs: by SOC and PT (MedDRA coded term), by severity and relation to study product in each treatment group. • Reported or observed SAEs: by SOC and PT (MedDRA coded term), by severity and relation to study product in each treatment group. • Changes in vital signs (blood pressure, pulse rate, and respiratory rate), clinical laboratory safety tests (haematology, serum biochemistry, coagulation, and urinalysis), 12-lead ECG, and physical examination findings;Timepoint(s) of evaluation of this end point: For AEs: The AE reporting period ends at the last study contact Visit 9/Week 16. For SAEs: The SAE reporting period lasts until 4 weeks (28+-4 days) following the last study drug administration. For Changes in vital signs: during the study and until Visit 8/Week 12 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): • Assessment of iron parameters (total serum iron, serum ferritin, serum transferrin, unsaturated iron binding capacity, calculated TSAT, from baseline over a 12-week period (absolute and change from baseline)). • PK parameters: Individual estimates of Cmax, clearance, distribution volume, AUC will be obtained using a population PK approach in adult and adolescent subjects combined with suitable mathematical/statistical analysis, using nonlinear mixed-effects modelling. Sparse sampling for determination of VIT-2763 plasma concentration following multiple dosing will be obtained from pre-dose trough to 3 or 4 hours post-dose at selected study visits.;Timepoint(s) of evaluation of this end point: • Iron parameters: prior to first administration and over a 12-week period • PK parameters: Blood samples for the determination of VIT-2763 plasma concentrations (sparse sampling) are collected on Visit 3 and Visit 7 at pre-dose trough and at approximately 1 hour and 4 hours postdose, and on Visits 6 and 8 at pre-dose trough and approximately at 1 hour and 3 hours post-dose. | — |
Countries
Greece, Israel, Italy, Lebanon, Thailand
Contacts
Public ContactCommunications
Vifor Pharma Management Ltd.
Outcome results
None listed