Evaluating Hydroxycarbamide for Children with Sickle Cell Disease
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)
This study is testing if a new form of Hydroxycarbamide can safely help children with Sickle Cell Disease feel better over a year of treatment.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 50 (estimated) |
| Ages | 9 Months to 11 Years |
| Sex | All |
| Sponsor | Theravia Industry-sponsored |
| Locations | 6 sites (Créteil and 5 other locations) |
| Trial ID | NCT06578507 on ClinicalTrials.gov |
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.
Who should consider this trial
Good fit: Ideal candidates are children aged 9 months to 11 years with HbSS or HbSβ0 Sickle Cell Disease who have not previously received Hydroxycarbamide.
Not a fit: Patients currently participating in other clinical trials or those with contraindications to Hydroxycarbamide will not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide a new effective treatment option for managing Sickle Cell Disease in children.
How similar studies have performed: Previous studies have shown promising results with Hydroxycarbamide in treating Sickle Cell Disease, indicating potential for success in this approach.
Eligibility criteria
Show full inclusion / exclusion criteria
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 this trial is running
Créteil and 5 other locations
- Centre Hospitalier Intercommunal Créteil — Créteil, France (Recruiting)
- GHEF- Site de Marne-la-Vallée — Jossigny, France (Recruiting)
- Hôpital Bicêtre — Le Kremlin-Bicêtre, France (Recruiting)
- Institut d'Hématologie et d'oncologie pédiatrique - IHOPe — Lyon, France (Active_not_recruiting)
- Hôpital Necker-Enfants malades — Paris, 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
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.