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Recruiting Phase 2

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

NCT06578507 · tracked via the Priya Life Science France tracker
Sponsor
Theravia
Phase
Phase 2
Started
2025-01-21
Last updated
2026-10-01

Condition(s) studied

Sickle Cell Disease

Investigational drug(s) / intervention(s)

Hydroxycarbamid →

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

Sex
ALL
Min age
9 Months
Max age
11 Years
Healthy volunteers
No
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.

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)

FacilityCityRegionStatus
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
Official registry record

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