A study to evaluate if VIT-2763 may be beneficial in the treatment of Nontransfusion Dependent Beta-thalassaemia.
- Conditions
- Chronic anemia due to ineffective erythropoiesis (IE) in subjects with ß-thalassaemiathalassemia
- Registration Number
- LBCTR2020021295
- Lead Sponsor
- Vifor (International) Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Complete
- Sex
- All
- Target Recruitment
- 25
1.Documented diagnosis of NTDT, including a ß-thalassaemia intermedia-phenotype.
2.NTDT is defined as subjects having received <5 units of red blood cells (RBCs) during the 24-week period prior to randomisation/first drug administration of VIT-2763 or placebo (Day 1; 1 unit is defined as 200 to 350 ml of transfused packed RBCs and last RBC transfusion must have been received =14 days prior to randomisation).
Note: Subjects who are supposed to receive RBC transfusions after randomisation in the Investigator’s opinion, and according to local practise, and having received at least 1 dose of VIT-2763, may be considered to stay on study treatment for safety reasons, and in case there are no tolerability concerns. Subjects will be censored for secondary efficacy.
3.Male and female adult NTDT subjects, 18-65 years of age inclusive (Cohort I only) at time of screening.
4.Male and female adolescent NTDT subjects, 12-17 years of age inclusive (Cohort II only) at time of screening.
5.Subjects must have a mean baseline Hb =11 g/dl, based on 2 consecutive measurements =1 week apart within 6 weeks prior to randomisation/baseline, and obtained Hb values show less than 10% relative difference (and equal or less than 1.0 g/dl absolute change between the highest and lowest value) between at least 2 measurements.
Note: If there is 1 retrospective Hb value available for the subject at maximum of 2 weeks prior to screening (Day -28), the Hb value can be taken into consideration. A subject not meeting this criterion would be excluded but can be rescreened at maximum 2 times at a later time point.
1.Documented diagnosis of transfusion dependent thalassaemia (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 iron chelation therapy (ICT) or subjects on prior ICT when discontinued less than 4 weeks prior randomisation. Note: If ICT was discontinued =4 weeks prior randomisation the subject is eligible.
3.ICT naïve subjects with serum ferritin <150 ng/ml and documented LIC =1 mg/g liver dry weight assessed through MRI, or subjects on prior ICT with serum ferritin <300 ng/ml and documented LIC <3 mg/g liver dry weight assessed through MRI.
4.Subjects with TSAT <30%.
5.Subjects with documented LIC >15 mg/g liver dry weight assessed through MRI, or a documented myocardial T2-star (T2*) <20 ms.
6.Adult or adolescent subjects with body weight <40.0 kg or >100 kg at screening and/or randomisation.
7.Chronic liver disease and/or alanine transaminase (ALT), aspartate transaminase (AST) or gamma-glutamyl transpeptidase (GGT) above 3-fold the upper limit of normal (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.Estimated glomerular filtration rate (eGFR) <30 ml/min/1.73 m2 (according to chronic kidney disease classification Stage 4 or higher), and/or significant albuminuria >30 mg/mmol. eGFR should be estimated according to Cockcroft-Gault.
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 partial or total splenectomy.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method