RECRUITING Phase 2 ClinicalTrials.gov

Pharmacokinetics, Efficacy and Safety of Twice Daily Dosing Regimen of Hydroxycarbamide Dispersible Tablets in Children With Sickle Cell Disease

Sponsor: Theravia • ClinicalTrials.gov ID: NCT06578507

Registry updated: 2026-10-01

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Description

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

Interventions

Hydroxycarbamid (Drug)
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.

Eligibility criteria

Inclusion Criteria: * Written informed consent, signed and dated by both parents or by the legally acceptable representative(s) of the children, and, if possible, assent from the children, * HbSS or HbSβ0 SCD, * Aged between 9 months and 11 years old, * Hydroxycarbamide naïve, * Parent(s) or legally acceptable representative(s) capable of communicating with the investigator and understanding the requirements and constraints of the study protocol and willing to comply with the study requirements, * Contraception criterion, if applicable: for patients who are sexually active * Affiliated to a social security plan or beneficiary of a similar insurance plan, * Patient must meet the following laboratory values : Absolute Neutrophil Count ≥ 1.0x109/L, Platelets ≥ 75x109/L and Haemoglobin (Hgb) \> 5.5 g/dL, * Transcranial Doppler (TCD) in the last 12 months indicating low risk for stroke is required for children over 18 months of age. Exclusion Criteria: * Participation in any other clinical study for any other pharmaceutical product within 4 weeks preceding the inclusion visit, * Patients who have had chronic blood transfusion or transfusion in the last 3 months preceding the inclusion visit, * Patients treated with other SCD-modifying therapies, * Patient with a stage 3, 4 or 5 chronic kidney disease, * Patients known to be infected with human immunodeficiency virus, hepatitis B virus, or hepatitis C virus, * Known hypersensitivity or allergy to the excipients, * Any surgical or medical condition or any significant illness that, in the opinion of the investigator, constitutes a risk or a contraindication to the participation of the patient to the study, or that may interfere with the objectives, conduct or evaluation of the study, * Female patients who are pregnant or lactating, * Any documented history of a clinical stroke or intracranial haemorrhage, or an uninvestigated neurologic finding within the past 12 months.

Conditions

Sickle Cell Disease

Locations

Centre Hospitalier Intercommunal Créteil — Créteil, France
GHEF- Site de Marne-la-Vallée — Jossigny, France
Hôpital Bicêtre — Le Kremlin-Bicêtre, France
Institut d'Hématologie et d'oncologie pédiatrique - IHOPe — Lyon, France
Hôpital Necker-Enfants malades — Paris, France
CH Saint-Denis — Saint-Denis, France
Centre hospitalier de Cayenne — Cayenne, French Guiana

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Official Source

Data sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

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