Pharmacokinetics, Efficacy and Safety of Twice Daily Dosing Regimen of Hydroxycarbamide Dispersible Tablets in Children With Sickle Cell Disease: a Single-group, Non-randomised, Open-label Study (KID-BID)

NCT06578507 Phase 2 RECRUITING

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

Details

Lead sponsor	Theravia
Phase	Phase 2
Status	RECRUITING
Enrolment	50
Start date	2025-01-21
Completion	2027-02

Conditions

• Sickle Cell Disease

Interventions

• Hydroxycarbamid

Primary outcomes

• 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

Countries

France, French Guiana