Pharmacokinetics, Efficacy and Safety of Twice Daily Dosing Regimen of Hydroxycarbamide Dispersible Tablets in Children With Sickle Cell Disease
Condition(s) studied
Investigational drug(s) / intervention(s)
Hydroxycarbamid: Hydroxycarbamide Paediatric dispersible tablets will be provided in the form of film-coated dispersible tablets containing 50 mg of hydroxycarbamide. The IMP will be administered as half-strength twice daily, based on the body weight of the patient.
Study summary
The purpose of this interventional, phase II, national, multicentric, non-randomised, open-label study is to evaluate the pharmacokinetics (PK), efficacy and safety of Hydroxycarbamide Paediatric dispersible tablets with a twice daily dosing regimen in children with Sickle Celle Disease between 9 months to 11 years of age.
Participants will:
* Take Hydroxycarbamide twice a day every day for 12 months
* Visit the clinic at screening, baseline, 1, 3, 6, 9 and 12 months
Eligibility
Primary outcome measure(s)
- Evaluate the PK exposure for Hydroxycarbamide Paediatric dispersible tablets administered BID through area under the curve (AUC) — 1, 3, 6, 9 and 12 months after treatment initiation
- Evaluate the PK exposure for Hydroxycarbamide Paediatric dispersible tablets administered BID through time to obtain the maximum concentration (Tmax) — 1, 3, 6, 9 and 12 months after treatment initiation
- Evaluate the PK exposure for Hydroxycarbamide Paediatric dispersible tablets administered BID through maximum plasma concentration (Cmax) — 1, 3, 6, 9 and 12 months after treatment initiation
Trial sites (7)
| Facility | City | Region | Status |
|---|---|---|---|
| Centre Hospitalier Intercommunal Créteil | Créteil | France | Recruiting |
| GHEF- Site de Marne-la-Vallée | Jossigny | France | Recruiting |
| Hôpital Bicêtre | Le Kremlin-Bicêtre | France | Recruiting |
| Institut d'Hématologie et d'oncologie pédiatrique - IHOPe | Lyon | France | Suspended |
| Hôpital Necker-Enfants malades | Paris | France | Recruiting |
| CH Saint-Denis | Saint-Denis | France | Recruiting |
| Centre hospitalier de Cayenne | Cayenne | French Guiana | Recruiting |
Other trials for the same condition
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT06578507 on ClinicalTrials.gov ↗ ← All trials in France