What is Hydroxycarbamide studying for Sickle Cell Disease?

Hydroxycarbamide is being studied to understand how it works in the body and its safety when given twice daily to children with Sickle Cell Disease. Researchers want to learn about the drug's effects and how well children tolerate it during the study led by Theravia. Participants will help provide important information on dosing and outcomes.

Who may be eligible for this Sickle Cell Disease clinical trial?

Children aged between 9 months and 11 years with a diagnosis of HbSS or HbSβ0 Sickle Cell Disease may be eligible. They must not have taken Hydroxycarbamide before and should have a recent low-risk stroke assessment if over 18 months old. Both boys and girls can participate. Please review eligibility criteria for more details.

Is there any cost to participate in this Sickle Cell Disease clinical trial?

Study-related care, including the study drug and all trial visits, is provided at no cost. However, personal travel expenses may vary and are not guaranteed to be covered. Participants and families can expect visits at several intervals over the 12 months.

Actively Recruiting

Phase 2

Age: 9Months - 11Years

All Genders

ID06578507

Study of Twice Daily Hydroxycarbamide Dispersible Tablets for Children with Sickle Cell Disease Aged 9 Months to 11 Years

Led by Theravia · Updated on 2025-09-17

Participants Needed

Research Sites

N/A

Total Duration

AI-Summary

What this Trial Is About

Researchers are evaluating the pharmacokinetics, efficacy, and safety of Hydroxycarbamide pediatric dispersible tablets given twice daily in children aged 9 months to 11 years with Sickle Cell Disease. This phase II, national, multicenter, non-randomized, open-label study aims to understand how the drug is processed in the body and its effects over a 12-month period. Participants will take Hydroxycarbamide twice daily for 12 months. They will attend clinic visits at screening, baseline, and at 1, 3, 6, 9, and 12 months during the study. The drug dosage is adjusted based on body weight and administered as half-strength dispersible tablets. Childrens involvement includes regular clinic visits for blood tests, monitoring of drug levels, and assessments of blood parameters and Sickle Cell Disease events. Acceptability of the treatment will be evaluated by both children and their parents using scales. Compliance will be tracked by pharmacy accountability of the medication units. Safety and adverse events will be closely monitored throughout the study duration.

CONDITIONS

Brief Title

Pharmacokinetics, Efficacy and Safety of Twice Daily Dosing Regimen of Hydroxycarbamide Dispersible Tablets in Children With Sickle Cell Disease

Who Can Participate

Age: 9Months - 11Years

All Genders

Eligibility Criteria

You may qualify if you...

Written informed consent signed by both parents or legal representatives, and assent from children if possible
Diagnosis of HbSS or HbSβ0 Sickle Cell Disease
Age between 9 months and 11 years
Never treated with Hydroxycarbamide before
Parent or legal representative can communicate with the investigator and comply with study requirements
Contraception use if patient is sexually active
Affiliated with a social security or similar insurance plan
Laboratory values: Absolute Neutrophil Count ≥ 1.0 x 10^9/L, Platelets ≥ 75 x 10^9/L, Hemoglobin > 5.5 g/dL
Transcranial Doppler in last 12 months showing low stroke risk for children over 18 months old

You will not qualify if you...

Participation in any other drug clinical study within 4 weeks before inclusion
Chronic blood transfusion or transfusion in last 3 months before inclusion
Treatment with other Sickle Cell Disease modifying therapies
Chronic kidney disease stage 3, 4, or 5
Known infection with HIV, hepatitis B, or hepatitis C virus
Known allergy to any excipients
Any medical condition posing risk or interfering with study participation as judged by investigator
Female patients who are pregnant or breastfeeding
History of clinical stroke, intracranial hemorrhage, or unexplained neurological findings within past 12 months

Research Team

Laura Thomas-bourgneuf

Nesrine Flissi

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Study of Twice Daily Hydroxycarbamide Dispersible Tablets for Children with Sickle Cell Disease Aged 9 Months to 11 Years

On this page

AI-Summary

What this Trial Is About

Brief Title

Who Can Participate

Eligibility Criteria

Research Team

Not the Right Trial for You?

On this page

Who Can Participate

Eligibility Criteria