Evaluating Hydroxycarbamide for Children with Sickle Cell Disease

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This phase II interventional study aims to assess the pharmacokinetics, efficacy, and safety of Hydroxycarbamide dispersible tablets administered twice daily to children aged 9 months to 11 years with Sickle Cell Disease. Participants will take the medication daily for 12 months and attend clinic visits at specified intervals for monitoring. The study is non-randomized and open-label, focusing on children who are naïve to Hydroxycarbamide treatment.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* 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 Criteria:

* 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.

Where it's running

• Centre Hospitalier Intercommunal Créteil
  Créteil, France Recruiting
• GHEF- Site de Marne-la-Vallée
  Jossigny, France Recruiting
• Hôpital Bicêtre
  Le Kremlin-Bicêtre, France Recruiting
• Hôpital Necker-Enfants malades
  Paris, France Recruiting
• CH Saint-Denis
  Saint-Denis, France Recruiting
• Centre hospitalier de Cayenne
  Cayenne, French Guiana Recruiting

Study contacts

• Principal investigator: Josephine Brice, MD — Hôpital Necker-Enfants Malades
• Study coordinator: Laura Thomas-bourgneuf
• Email: laura.thomas-bourgneuf@theravia.com
• Phone: + 33 1 49 70 95 83

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Other studies for the same condition or treatment. Recruiting studies are listed first.

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.