EUCTR2019-002221-29-GR进行中（未招募）1 期
A Phase 2a, Double-blind, Randomised, Placebo-controlled, Parallel Group, Multicentre Study on Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of Multiple Doses of VIT-2763 in Subjects with Non-transfusion Dependent Beta-thalassaemia.
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 36
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •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 packedRBCs and last RBC transfusion must have been received =14 days prior to randomisation).
- •Note: Subjects who are supposed to receive RBC transfusions after randomization 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.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 12
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 23
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 1
排除标准
- •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 <150 ng/ml and/or documented LIC =1 mg/g liver dry weight assessed through MRI, or subjects on prior ICT with serum ferritin <300 ng/ml and/or documented LIC <3 mg/g liver dry weight assessed through MRI.
- •Note: If documented LIC MRI scans retrieved within 24 months prior to randomization are not available per local practice, serum ferritin will be used only to document iron overload status.
- •4. Subjects with TSAT <30%.
- •5. Subjects with documented LIC >15 mg/g liver dry weight assessed through MRI, or a documented myocardial T2* <20 ms, if available per local practice and retrieved within 24 months prior to randomization.
- •6. Adult or adolescent subjects with body weight <40.0 kg or >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 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 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.
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