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)
Key facts
- Where
- Cayenne, Creteil Cedex, Le Kremlin-Bicêtre, Meaux, Paris Cedex 15 és St Denis Cedex, FranciaországSee the 6 sites
- Phase
- 2. fázis
Where in Franciaország
- Centre Hospitalier De CayenneCayenne, FranciaországTaking people
- Centre Hospitalier Intercommunal CreteilCreteil Cedex, FranciaországTaking people
- Hôpital Bicêtre APHPLe Kremlin-Bicêtre, FranciaországTaking people
- Grand Hopital de l'Est FrancilienMeaux, FranciaországTaking people
- CEREDIH Groupe Hospitalier Necker-Enfants MaladesParis Cedex 15, FranciaországTaking people
- Centre Hospitalier General De St DenisSt Denis Cedex, FranciaországTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Theravia
- Trial code
- EU 2024-513750-30-00
- Record updated
- 2026. július 24.
- Original description
The primary objective is to develop a population pharmacokinetic (PPK) model of Siklos® Paediatric dispersible tablets administered BID in children aged 9 months to 11 years.Read full original descriptionShow less
The primary objective is to develop a population pharmacokinetic (PPK) model of Siklos® Paediatric dispersible tablets administered BID in children aged 9 months to 11 years.
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; HC-naïve (absence of previous or current treatment with HC); 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 Female patients of childbearing potential (post menarche) at screening agreeing to use a highly effective form of contraception (oral, injected or implanted hormonal contraception, intrauterine device, o abstinence) during the trial and for 3 months after HC discontinuation. Male patients with partners of childbearing potential agreeing to use a highly effective contraception during the trial and for 3 months after HC discontinuation. Male patients with pregnant or lactating partners should be advised to use a barrier method of contraception (condom) to prevent the foetus or breastfed infant from exposure to HC; Affiliated to a social security plan (including universal health coverage) 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: Patients treated with other SCD-modifying therapies; Patient with a stage 3, 4 or 5 chronic kidney disease (estimated glomerular filtration rate < 60 mL/min per 1.73 m2; 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 (of which severe hepatic impairment [Child-Pugh classification C], severe renal impairment, toxic ranges of myelosuppression) 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; Any documented history of a clinical stroke or intracranial haemorrhage, or an uninvestigated neurologic finding within the past 12 months; 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; Female patients who are pregnant or lactating.