NCT05405114

Research Study Investigating How Well NDec Works in People With Sickle Cell Disease

A Multicentre Trial Evaluating the Efficacy and Safety of Oral Decitabine Tetrahydrouridine (NDec) in Patients With Sickle Cell Disease

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 96 (actual)
• Sponsor: Novo Nordisk A/S · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Sickle Cell Disease

Interventions

Type	Name	Description
DRUG	NDec - oral decitabine-tetrahydrouridine	Participants will get capsules (oral administration) to take once or twice weekly. The number of capsules will be based on their body weight
DRUG	HU - Hydroxyurea	Participants will get capsules daily (oral administration) according to local labelling
DRUG	Placebo	Participants will get capsules (oral administration) to take once or twice weekly. The number of capsules will be based on their body weight

Timeline

• Start date: 2022-07-07
• Primary completion: 2025-07-24
• Completion: 2025-07-24
• First posted: 2022-06-06
• Last updated: 2025-12-30

Locations

69 sites across 12 countries: United States, Canada, France, Greece, India, Italy, Lebanon, Oman, South Africa, Spain, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study