Research study investigating how well NDec works in people with sickle cell disease

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 87

Inclusion Criteria

Age above or equal to 18 years at the time of signing informed consent, Confirmed diagnosis of SCD (including HbSS, HbSC, HbSß0 thalassaemia and HbSß+ 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)

Exclusion Criteria

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

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: 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);Secondary Objective: To investigate the safety and tolerability of NDec in HU-non-eligible patients with SCD compared to placebo, To evaluate clinical efficacy measures of NDec in HU-non-eligible patients with SCD compared to placebo, To further describe the pharmacokinetics and pharmacodynamics of NDec in HU-non-eligible patients with SCD;Primary end point(s): Change from baseline (week 0) to week 24 in total haemoglobin in g/d

Secondary Outcome Measures

Name	Time	Method

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Updated 8/19/2024