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Research study investigating how well NDec works in people with sickle cell disease
EU CTIS ID: 2023-508506-22-00
What this study is testing
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)
- Therapeutic exploratory (Phase II)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 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 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)
You likely can't join if
- 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
See the full eligibility 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 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)
- 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
The study team makes the final eligibility decision.
Where it's taking place
- Turkey
- Lebanon
- United States
- Canada
- South Africa
- United Kingdom
- India
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 65+ years, 18-64 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Turkey; Lebanon; United States; Canada; South Africa; United Kingdom and 1 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.