Pharmacokinetics, Efficacy and Safety of Twice Daily Dosing Regimen of Hydroxycarbamide Dispersible Tablets in Children With Sickle Cell Disease
Recruiting · NCT06578507 · Interventional (participants receive a specific treatment) · Lead sponsor: Theravia
View the official record on ClinicalTrials.gov →Interventions studied
Hydroxycarbamid
What this trial is about
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
Who can take part
Age range
9 Months to 11 Years
Sex
All (male and female)
Healthy volunteers
No - a diagnosis or condition is required
Phase
Phase 2
Study type
Interventional (participants receive a specific treatment)
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.
Where it is running
6 locations listed.
- Centre hospitalier de Cayenne - Cayenne, French Guiana
- 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