CTRI/2023/01/049275进行中（未招募）2 期
A multicentre trial evaluating the efficacy and safety of oral decitabine-tetrahydrouridine (NDec) in patients with sickle cell disease
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 84
- 试验地点
- 13
- 主要终点
- Change in total haemoglobin
研究概览
简要总结
This study examines how well a new, potential medicine called NDec works and is tolerated in people with sickle cell disease. NDec is a combination of two medicines (decitabine-tetrahydrouridine). Both medicines are new for the treatment of sickle cell disease. Participants who are not taking Hydroxyurea (HU) will get NDec, NDec and placebo, or placebo.
Participants who are on HU treatment before joining the study will get NDec, NDec and placebo, or continue on HU. Which treatment participants get is decided by chance. Participants getting NDec and/or Placebo will get capsules to take twice weekly.
The study will last for about a year.
研究设计
- 研究类型
- Interventional
- 分配方式
- Computer generated randomization
- 盲法
- Participant, Investigator, Outcome Assessor and Date-entry Operator Blinded
入排标准
- 年龄范围
- 18.00 Year(s) 至 99.00 Year(s)（—）
- 性别
- All
入选标准
- •Age above or equal to 18 years at the time of signing informed consent
- •Confirmed diagnosis of SCD (including HbSS, HbSC, HbS beta zero thalassaemia and HbS beta plus thalassaemia or other Sickle Cell disease variants)
- •2-10 episodes of documented vaso-occlusive crisis (VOCs) within the last 12 months prior to the screening visit
- •Haemoglobin greater than or equal to 5.0 g/dL and below or equal to 10.5 g/dL at visit 1
- •Absolute reticulocyte count above upper limit of the normal (ULN) at visit 1
- •Body weight 40 to 125 kg (inclusive).
排除标准
- •Patient is on chronic transfusion therapy as defined by receiving scheduled (pre-planned) series of blood transfusion (simple or exchange) for prophylactic purposes, or the patient is likely to begin chronic transfusion therapy during the course of the trial, or has received RBC or whole blood transfusion for any reason within 28 days of visit 1
- •Receipt of erythropoietin or other haematopoietic growth factor treatment within 28 days of signing ICF, or planned treatment with these agents during the trial
- •Receipt of voxelotor, crizanlizumab or L-glutamine treatment within 12 weeks of signing the informed consent form, or planned treatment with such agents during the trial
- •Platelet count greater than 800 x 10 to the power of 9 per L at visit 1
- •Absolute neutrophil count below or equal to 1.5 x 10 to the power of 9 per L at visit 1
- •Any condition per concurrent chronic disease involving the stomach or small intestine which may affect drug absorption, as per investigators judgement
- •Female who is a.
- •pregnant, breast-feeding or intends to become pregnant within 6 months after the final trial product administration b.
- •child-bearing potential and not using highly effective methods of contraception and whose male partner is not using effective contraception, at screening and until 6 months after the last dose of trial product
- •Male with female partner of childbearing potential who does not agree to use condom and whose female partner of childbearing potential is not using a highly effective contraceptive measure from trial start to: a.
- •Six (6) months after the last dose of trial product for patients on NDec or Placebo b.
- •Six (6) months after the last dose of trial product for patients outside US and CA randomised to HU c.
- •Twelve (12) months after the last dose of trial product for patients randomised to HU in US and CA.
结局指标
主要结局
Change in total haemoglobin
时间窗: [Time Frame: From baseline (week 0) to week 24] measured in g/dL
次要结局
- Cmax for decitabine from pharmacokinetic assessment([Time Frame: At week 24])
- Cmax for tetrahydrouridine from pharmacokinetic assessment([Time Frame: At week 24])
- Change in DNA methyltransferase 1 (DNMT1) activity([Time Frame: From baseline (week 0) to week 24])
- Change in cytidine deaminase (CDA) activity([Time Frame: From baseline (week 0) to week 24])
- Change in foetal haemoglobin (g/dL)([Time Frame: From baseline (week 0) to week 24])
- Change in foetal haemoglobin as a proportion of total haemoglobin (%HbF)([Time Frame: From baseline (week 0) to week 24])
- Change in F-cell level as a proportion of total red blood cell (RBC) (%F-cells)([Time Frame: From baseline (week 0) to week 24])
- Change in haemolysis measure: absolute reticulocyte count([Time Frame: From baseline (week 0) to week 24])
- Change in haemolysis measure: indirect bilirubin([Time Frame: From baseline (week 0) to week 24])
- Change in haemolysis measure: lactate dehydrogenase([Time Frame: From baseline (week 0) to week 24])
- Number of vaso-occlusive crises([Time Frame: From baseline (week 0) to week 48])
- Number of acute chest syndrome([Time Frame: From baseline (week 0) to week 48])
- Number of RBC units transfused([Time Frame: From baseline (week 0) to week 48])
- Number of adverse events of grade 3 or higher([Time Frame: From baseline (week 0) to week 52])
研究者
研究点 (13)
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