Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT05405114 | Research Study Investigating How Well NDec Works in People With Sickle Cell Disease | PHASE2 | COMPLETED | 96 | — | — | Jul 7, 2022 | Jul 24, 2025 | Dec 30, 2025 | 69 | United States, Canada +10 |
measured in g/dL
| Arm | Type | Description |
|---|---|---|
| HU-non-eligible - NDec plus placebo | EXPERIMENTAL | HU-non eligible patients randomised to treatment with NDec on one day and placebo on the other day |
| HU-non-eligible - NDec plus NDec | EXPERIMENTAL | HU-non eligible patients randomised to treatment with NDec on both days |
| HU-non-eligible - Placebo plus placebo | PLACEBO_COMPARATOR | HU-non eligible patients randomised to treatment with placebo on both days |
| HU-active - NDec plus placebo | EXPERIMENTAL | HU-active patients randomised to treatment with NDec on one day and placebo on the other day |
| HU-active - NDec plus NDec | EXPERIMENTAL | HU-active patients randomised to treatment with NDec on both days |
| HU-active - HU | ACTIVE_COMPARATOR | HU-active patients randomised to continue on open-label HU treatment |
| Name | Type | Description |
|---|---|---|
| NDec - oral decitabine-tetrahydrouridine | DRUG | Participants will get capsules (oral administration) to take once or twice weekly. The number of capsules will be based on their body weight |
| HU - Hydroxyurea | DRUG | Participants will get capsules daily (oral administration) according to local labelling |
| Placebo | DRUG | Participants will get capsules (oral administration) to take once or twice weekly. The number of capsules will be based on their body weight |
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 las...