Research Study Investigating How Well NDec Works in People With Sickle Cell Disease
- Conditions
- Sickle Cell Disease
- Registration Number
- NCT05405114
- Lead Sponsor
- Novo Nordisk A/S
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 84
Inclusion Criteria:<br><br> - Age above or equal to 18 years at the time of signing informed consent<br><br> - Confirmed diagnosis of SCD (including HbSS, HbSC, HbSß0 thalassaemia and HbSß+<br> thalassaemia or other Sickle Cell disease variants)<br><br> - 2-10 episodes of documented vaso-occlusive crisis (VOCs) within the last 12 months<br> prior to the screening visit<br><br> - Haemoglobin greater than or equal to 5.0 g/dL and below or equal to 10.5 g/dL at<br> visit 1<br><br> - Absolute reticulocyte count above upper limit of the normal (ULN) at visit 1<br><br> - Body weight 40 to 125 kg (inclusive).<br><br>Exclusion Criteria:<br><br> - Patient is on chronic transfusion therapy as defined by receiving scheduled<br> (pre-planned) series of blood transfusion (simple or exchange) for prophylactic<br> purposes, or the patient is likely to begin chronic transfusion therapy during the<br> course of the trial, or has received RBC or whole blood transfusion for any reason<br> within 28 days of visit 1<br><br> - Receipt of erythropoietin or other haematopoietic growth factor treatment within 28<br> days of signing ICF, or planned treatment with these agents during the trial<br><br> - Receipt of voxelotor, crizanlizumab or L-glutamine treatment within 12 weeks of<br> signing the informed consent form, or planned treatment with such agents during the<br> trial<br><br> - Platelet count greater than 800 x 10^9/L at visit 1<br><br> - Absolute neutrophil count below or equal to 1.5 x 10^9/L at visit 1<br><br> - Any condition/concurrent chronic disease involving the stomach or small intestine<br> which may affect drug absorption, as per investigator's judgement<br><br> - Female who is<br><br> - pregnant, breast-feeding or intends to become pregnant within 6 months after<br> the final trial product administration<br><br> - child-bearing potential and not using highly effective methods of contraception<br> and whose male partner is not using effective contraception, at screening and<br> until 6 months after the last dose of trial product<br><br> - Male with female partner of childbearing potential who does not agree to use condom<br> and whose female partner of childbearing potential is not using a highly effective<br> contraceptive measure from trial start to:<br><br> - Six (6) months after the last dose of trial product for patients on<br> NDec/Placebo<br><br> - Six (6) months after the last dose of trial product for patients outside US and<br> CA randomised to HU<br><br> - Twelve (12) months after the last dose of trial product for patients randomised<br> to HU in US and CA
Not provided
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Change in total haemoglobin
- Secondary Outcome Measures
Name Time Method Cmax for decitabine from pharmacokinetic assessment;Cmax for tetrahydrouridine from pharmacokinetic assessment;Change in DNA methyltransferase 1 (DNMT1) activity;Change in cytidine deaminase (CDA) activity;Change in foetal haemoglobin (g/dL);Change in foetal haemoglobin as a proportion of total haemoglobin (%HbF);Change in F-cell level as a proportion of total red blood cell (RBC) (%F-cells);Change in haemolysis measure: absolute reticulocyte count;Change in haemolysis measure: indirect bilirubin;Change in haemolysis measure: lactate dehydrogenase;Number of vaso-occlusive crises;Number of acute chest syndrome;Number of RBC units transfused;Number of adverse events of grade 3 or higher