Status:
RECRUITING
Pharmacokinetics, Efficacy and Safety of Twice Daily Dosing Regimen of Hydroxycarbamide Dispersible Tablets in Children With Sickle Cell Disease
Lead Sponsor:
Theravia
Conditions:
Sickle Cell Disease
Eligibility:
All Genders
9-11 years
Phase:
PHASE2
Brief 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 disp...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
January 21 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
February 1 2027
Estimated Enrollment :
50 Patients enrolled
Trial Details
Trial ID
NCT06578507
Start Date
January 21 2025
End Date
February 1 2027
Last Update
September 17 2025
Active Locations (6)
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1
Centre Hospitalier Intercommunal Créteil
Créteil, France
2
GHEF- Site de Marne-la-Vallée
Jossigny, France
3
Hôpital Bicêtre
Le Kremlin-Bicêtre, France
4
Institut d'Hématologie et d'oncologie pédiatrique - IHOPe
Lyon, France