Status:
COMPLETED
Research Study Investigating How Well NDec Works in People With Sickle Cell Disease
Lead Sponsor:
Novo Nordisk A/S
Conditions:
Sickle Cell Disease
Eligibility:
All Genders
18+ years
Phase:
PHASE2
Brief Summary
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). Bot...
Eligibility Criteria
Inclusion
- 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 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).
Exclusion
- 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 below or equal to 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
- 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
- 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
Key Trial Info
Start Date :
July 7 2022
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
July 24 2025
Estimated Enrollment :
96 Patients enrolled
Trial Details
Trial ID
NCT05405114
Start Date
July 7 2022
End Date
July 24 2025
Last Update
December 30 2025
Active Locations (69)
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1
University Of South Alabama
Mobile, Alabama, United States, 36617
2
UCSF Oakland Benioff Children's Hospital
Oakland, California, United States, 94609
3
Center for Inherited Blood Dis
Orange, California, United States, 92868
4
Harbor-UCLA Medical Center
Torrance, California, United States, 90502-2004