Actively Recruiting
Pharmacokinetics, Efficacy and Safety of Twice Daily Dosing Regimen of Hydroxycarbamide Dispersible Tablets in Children With Sickle Cell Disease
Led by Theravia · Updated on 2025-09-17
50
Participants Needed
6
Research Sites
105 weeks
Total Duration
On this page
AI-Summary
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
CONDITIONS
Official Title
Pharmacokinetics, Efficacy and Safety of Twice Daily Dosing Regimen of Hydroxycarbamide Dispersible Tablets in Children With Sickle Cell Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Written informed consent signed and dated by both parents or legal representatives, plus assent from children if possible
- Diagnosed with HbSS or HbSβ0 type Sickle Cell Disease
- Aged between 9 months and 11 years old
- Has never been treated with Hydroxycarbamide before
- Parent(s) or legal representatives able to communicate with investigators and willing to follow study requirements
- Meets contraception requirements if sexually active
- Covered by social security or similar insurance
- Laboratory values: Absolute Neutrophil Count ≥ 1.0x10^9/L, Platelets ≥ 75x10^9/L, Hemoglobin > 5.5 g/dL
- For children older than 18 months, a Transcranial Doppler (TCD) within the last 12 months showing low stroke risk
You will not qualify if you...
- Participating in another clinical study involving pharmaceutical products within 4 weeks before inclusion
- Received chronic blood transfusion or any transfusion within 3 months before inclusion
- Currently treated with other Sickle Cell Disease modifying therapies
- Has stage 3, 4, or 5 chronic kidney disease
- Known infection with HIV, hepatitis B, or hepatitis C viruses
- Known allergy or hypersensitivity to study tablet ingredients
- Any medical or surgical condition that poses risk or interferes with study participation or evaluation
- Female patients who are pregnant or breastfeeding
- History of clinical stroke, intracranial hemorrhage, or unexplained neurological findings within the past 12 months
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 6 locations
1
Centre Hospitalier Intercommunal Créteil
Créteil, France
Actively Recruiting
2
GHEF- Site de Marne-la-Vallée
Jossigny, France
Actively Recruiting
3
Hôpital Bicêtre
Le Kremlin-Bicêtre, France
Actively Recruiting
4
Institut d'Hématologie et d'oncologie pédiatrique - IHOPe
Lyon, France
Active, Not Recruiting
5
Hôpital Necker-Enfants malades
Paris, France
Actively Recruiting
6
Centre hospitalier de Cayenne
Cayenne, French Guiana
Actively Recruiting
Research Team
L
Laura Thomas-bourgneuf
CONTACT
N
Nesrine Flissi
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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