Research Study Investigating How Well NDec Works in People With Sickle Cell Disease

• Status: Not yet recruiting
• Trial end date: 2024-03-29
• Target enrollment: 0
• Participant gender: All

Summary

This study will look at how well a potentially new 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 will either get NDec, placebo or continue on Hydroxyurea (HU) - which treatment participants get is decided by chance. If participants get NDec or the placebo, they will get capsules to take twice weekly.

The study will last for about a year.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Novo Nordisk A/S
• Treatments: Decitabine; Hydroxyurea; Tetrahydrouridine

Criteria

Inclusion Criteria:

• 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)

• 2-10 episodes of documented vaso-occlusive crisis (VOCs) within the last 12 months prior to the screening visit

• Haemoglobin greather than or equal to 5.0 g/dL and below or equal to 10.5 g/dL at visit 1

• Reticulocyte count greater than1.5 x upper limit of the normal (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

• 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 10^9/L at visit 1

• Absolute neutrophil count below1.5 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 is of child-bearing potential and not using highly effective methods of contraception (or adequate contraceptive measures as required by local regulation or practice) starting at screening and throughout the trial period and for 6 months after the last dose of trial product

• Male of reproductive age 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 (or adequate contraceptive measure as required by local regulation or practice) from trial start to:

• Six (6) months after the last dose of trial product for patients on NDec/Placebo

• Six (6) months after the last dose of trial product for patients outside US and CA randomised to HU

• Twelve (12) months after the last dose of trial product for patients randomised to HU in US and CA