Pharmacokinetics, Efficacy and Safety of Twice Daily Dosing Regimen of Hydroxycarbamide Dispersible Tablets in Children With Sickle Cell Disease
NCT Number: NCT06578507
Acronym: KID-BID
Status: NOT_YET_RECRUITING
Study Type: INTERVENTIONAL
Phases: PHASE2
Conditions: Sickle Cell Disease
Interventions: DRUG: Hydroxycarbamid
Sponsor: Theravia
Sex: ALL
Age: CHILD
Enrollment: 50
Study Design: Allocation: NA|Intervention Model: SINGLE_GROUP|Masking: NONE|Primary Purpose: TREATMENT
Start Date: 2024-09
Completion Date: 2026-04
Locations: Centre Hospitalier Intercommunal Créteil, Créteil, France|Hôpital Bicêtre, Le Kremlin-Bicêtre, France|Hôpital Necker-Enfants malades, Paris, France|Centre hospitalier de Cayenne, Cayenne, French Guiana

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

Primary Outcome Measures: 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
Secondary Outcome Measures: Absolute mean change from baseline in HbF levels, Baseline, 3, 6, 9 and 12 months after treatment initiation|HC plasma concentrations and HbF levels, Baseline, 3, 6, 9 and 12 months after treatment initiation|Daily AUC (AUC0-24h) at maintenance dose derived from the final PPK model, Baseline, 3, 6, 9 and 12 months after treatment initiation|Proportion of patients with a relative difference in Cmax ≥ 30% from BID maintenance dose relative to the one simulated on a once daily regimen giving an equivalent AUC0-24h., Baseline, 3, 6, 9 and 12 months after treatment initiation|Absolute mean change from baseline in haematological parameters, Baseline,1, 3, 6, 9 and 12 months after treatment initiation|Acceptability score based on a hedonic face scale evaluated by the child from 3 years old, 3 months after treatment initiation|Acceptability score based on a 5-point Likert scale evaluated by the parent(s) or legally acceptable representative(s), 3 months after treatment initiation|Distribution of the scores related to the ease of using the administration kit for treatment administration to the child based on a 5-point Likert scale evaluated by the parent(s) or legally acceptable representative(s), Score 1 : very difficult to score 5 : very easy, 3 months after treatment initiation|Compliance with Hydroxycarbamide Paediatric dispersible tablets administered BID by treatment unit accountability calculated by the pharmacy (patient will bring the kits with used and unused bottles to the pharmacy at each visit), Baseline, 1, 3, 6, 9, 12 months after treatment initiation|Number of SCD events occurring during the study, Baseline, 1, 3, 6, 9, 12 months after treatment initiation|Number of adverse events (AEs) and percentage of patients reporting at least one AE, Baseline, 1, 3, 6, 9, 12 months after treatment initiation