2023-508506-22-00已完成2 期
A multicentre trial evaluating the efficacy and safety of oral decitabine-tetrahydrouridine (NDec) in patients with sickle cell disease
适应症
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 15
- 试验地点
- 10
- 主要终点
- Change from baseline (week 0) to week 24 in total haemoglobin in g/dL
研究概览
简要总结
To investigate the efficacy of two dosing regimens of oral decitabine-tetrahydrouridine (NDec) combination as measured by improvement in haemoglobin compared to placebo in hydroxyurea (HU)-non-eligible patients with sickle cell disease (SCD)
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 18 years 至 65+ years（65+ Years, 18-64 Years）
- 接受健康志愿者
- 否
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, HbSβ0 thalassaemia and HbSβ+ thalassaemia or other Sickle Cell disease variants)
- •2–10 episodes of documented VOCs within the last 12 months prior to the screening visit
- •Haemoglobin ≥5.0 g/dL and ≤10.5 g/dL at visit 1
- •Absolute reticulocyte (absolute) count above 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
- •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 Six (6) months after the last dose of trial product for patients outside US and CA randomised to HU
- •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 Twelve (12) months after the last dose of trial product for patients randomised to HU in US and CA
- •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 >800 x 10^9/L at visit 1
- •Absolute neutrophil count ≤1.5 x 10^9/L at visit 1
- •Any condition/concurrent chronic disease involving the stomach or small intestine which may affect drug absorption, as per investigator's judgement
- •Female who is: pregnant, breast-feeding or intends to become pregnant within 6 months after the final trial product administration or
- •Female who is: 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 Six (6) months after the last dose of trial product for patients on NDec/Placebo
结局指标
主要结局
Change from baseline (week 0) to week 24 in total haemoglobin in g/dL
Change from baseline (week 0) to week 24 in total haemoglobin in g/dL
次要结局
- Change in foetal haemoglobin (g/dL) From baseline (week 0) to week 24 in g/dL
- Cmax (maximum concentration) for decitabine from pharmacokinetic assessment At week 24 in ng/mL
- Cmax (maximum concentration) for tetrahydrouridine from pharmacokinetic assessment at week 24 in ng/mL
- Change in DNMT1 activity From baseline (week 0) to week 24 in MFI
- Change in CDA activity From baseline (week 0) to week 24 in µmol/L/min
- Change in foetal haemoglobin as a proportion of total haemoglobin (%HbF) From baseline (week 0) to week 24 in %
- Change in F-cell level as a proportion of total red blood cells (%F-cells) From baseline (week 0) to week 24 in %
- Change in haemolysis measure: absolute reticulocyte count From baseline (week 0) to week 24 in cells × 10^9/L
- Change in haemolysis measure: indirect bilirubin From baseline (week 0) to week 24 in mg/dL
- Change in haemolysis measure: lactate dehydrogenase From baseline (week 0) to week 24 in U/L
- Number of vaso-occlusive crises From baseline (week 0) to week 48 in Number of events
- Number of acute chest syndrome From baseline (week 0) to week 48 in Number of events
- Number of RBC units transfused From baseline (week 0) to week 48 in units
- Number of adverse events of grade 3 or higher From baseline (week 0) to week 52 in Number of events
研究者
EU Submission Hub
Scientific
Novo Nordisk A/S
研究点 (10)
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