Informations générales (source: ClinicalTrials.gov)
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) (KID-BID)
Interventional
Phase 2
Theravia (Voir sur ClinicalTrials)
janvier 2025
août 2026
05 avril 2025
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
Etablissements
| Les établissements d'Île-de-France ayant mis à jour leurs données Origine et niveau de fiabilité des données | |||||
|---|---|---|---|---|---|
| CHI DE CRETEIL | Cécile ARNAUD | 10/04/2025 14:50:10 | Contacter | ||
| Les établissements d'Île-de-France dont les données sont issues de ClinicalTrials.gov Origine et niveau de fiabilité des données | |||||
| AP-HP - Hôpital Necker-Enfants Malades | Contact (sur clinicalTrials) | ||||
| Les établissements sans correspondance certaine dans le répertoire FINESS dont les données sont issues de ClinicalTrials.gov Origine et niveau de fiabilité des données | |||||
| Centre Hospitalier Intercommunal Créteil - Créteil - France | Cecile Arnaud, MD | Contact (sur clinicalTrials) | |||
| GHEF- Site de Marne-la-Vallée - Jossigny - France | Contact (sur clinicalTrials) | ||||
| Hôpital Bicêtre - Le Kremlin-Bicêtre - France | Corinne Guitton, MD | Contact (sur clinicalTrials) | |||
| Institut d'Hématologie et d'oncologie pédiatrique - IHOPe - Lyon - France | Contact (sur clinicalTrials) | ||||
Critères
Tous
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.
- 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.
- 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.