Phase 2 Sickle Cell Disease Trial, Recruiting
Who may be able to join
AI generated eligibility summary. Written by an AI model from the official source data and checked on a sample basis. It can contain mistakes, so confirm anything important against the original source. How we use AI
Who might be able to join this trial:
- Parents or legal guardians must have signed and dated a written consent form, and children should give their own agreement to participate if they are able to
- Children diagnosed with a specific form of sickle cell disease — either HbSS or HbSβ0 type
- Children aged between 9 months and 11 years old
- Children who have never previously been treated with a medicine called hydroxycarbamide (also known as hydroxyurea)
- Parents or legal guardians who are able to communicate with the research team and are willing to follow the study requirements throughout the trial
- Children who are sexually active must meet certain contraception requirements, if applicable (confirm with trial site)
- Children who are covered by a social security or equivalent health insurance plan
- Children whose recent blood test results show: a neutrophil count of at least 1.0×10⁹/L, a platelet count of at least 75×10⁹/L, and a haemoglobin level above 5.5 g/dL
- Children over 18 months of age must have had a specific brain blood flow scan (called a Transcranial Doppler) within the last 12 months showing a low risk of stroke
Who may not be able to join:
- Children who have taken part in another clinical trial for a pharmaceutical product within the 4 weeks before joining this study
- Children who have received regular blood transfusions, or any blood transfusion in the 3 months before joining this study
- Children currently being treated with other medicines that modify sickle cell disease
- Children with moderate to severe chronic kidney disease (classified as stage 3, 4, or 5)
- Children known to be infected with HIV, hepatitis B, or hepatitis C
- Children with a known allergy or sensitivity to any ingredients in the study medicine
- Children with any serious medical or surgical condition that, in the opinion of the trial doctor, could put them at risk or affect how the study runs
- Girls who are currently pregnant or breastfeeding
- Children with a known history of a stroke, bleeding in the brain, or any unexplained neurological symptoms within the past 12 months
Important: Always verify eligibility with the trial site directly before applying.
Based on publicly available eligibility criteria from ClinicalTrials.gov. Verify directly with the trial site before acting. This is not medical advice.
Contact this trial
Principal Investigator: Josephine Brice, MD, Hôpital Necker-Enfants Malades
Phone: + 33 1 49 70 95 83
Contact details sourced from ClinicalTrials.gov. Verify directly with the trial site before attending.
GP referral letter
Print a one-page summary to share with your doctor.
Trial details
Where this trial is recruiting
Primary endpoints
Evaluate the PK exposure for Hydroxycarbamide Paediatric dispersible tablets administered BID through area under the curve (AUC); Evaluate the PK exposure for Hydroxycarbamide Paediatric dispersible tablets administered BID through time to obtain the maximum concentration (Tmax); Evaluate the PK exposure for Hydroxycarbamide Paediatric dispersible tablets administered BID through maximum plasma concentration (Cmax)
Can't join this trial?
Data last synced from ClinicalTrials.gov: 26 July 2026. Trial status can change. Always verify current status directly with the trial site before making any decision.