Research Study Investigating How Well NDec Works in People With Sickle Cell Disease (NCT05405114) | Clinical Trial Compass
CompletedPhase 2
Research Study Investigating How Well NDec Works in People With Sickle Cell Disease
United States, Canada, France96 participantsStarted 2022-07-07
Plain-language 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). Both medicines are new for the treatment of sickle cell disease. Participants who are not taking Hydroxyurea (HU) will get NDec, NDec and placebo, or placebo. Participants who are on HU treatment before joining the study will get NDec, NDec and placebo, or continue on HU. Which treatment participants get is decided by chance. Participants getting NDec and/or Placebo will get capsules to take twice weekly. The study will last for about a year.
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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 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 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 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…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1This trial is Phase 2 and has already completed — what does that mean for how much we know about whether NDec is actually safe and effective for sickle cell disease so far?
2Since the trial measured changes in total haemoglobin as its main outcome, what would a meaningful improvement in haemoglobin levels actually look like for my specific situation, and how does that compare to what my haemoglobin levels are doing now?
3Because this study is completed, is there any published data or results I could review, and would those results change how you think about my current treatment plan?
4How does NDec compare to the treatments I'm already on or might consider, like hydroxyurea or voxelotor, in terms of what we know about safety and benefit at this stage?
5Given that this was a Phase 2 trial, are there any follow-up or Phase 3 studies of NDec I might be eligible for, or would you recommend sticking with established standard-of-care options for now?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.