drugset / Trial / NCT05405114
Research Study Investigating How Well NDec Works in People With Sickle Cell Disease
RandomizedParallel-groupQuadruple-blindTreatment
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.
Timeline
- Start
- 2022-07-07
- Primary completion
- 2025-07-24
- Completion
- 2025-07-24
Publications
- Helleberg H, Bjornsdottir I, Offenberg HK. Effects of Tetrahydrouridine Pre-dosing on Absorption, Metabolism, and Excretion of Decitabine in the Mouse. Eur J Pharm Sci. 2026 Jan 1;216:107353. doi: 10.1016/j.ejps.2025.107353. Epub 2025 Oct 27.
- Abbasi M, Srivastava A, Saraf SL. Management of Kidney Disease with Sickle Cell Disease. J Am Soc Nephrol. 2025 Oct 1;36(10):2041-2054. doi: 10.1681/ASN.0000000804. Epub 2025 Jun 26.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Decitabine | Small molecule | — | Oral |
| Comparator | Hydroxyurea | Other / unclassified | — | Oral |
| Subject | Tetrahydrouridine | Small molecule | — | Oral |