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Tests treatment safety and results for Sickle Cell Disease
Official title Pharmacokinetics, Efficacy and Safety of Twice Daily Dosing Regimen of Hydroxycarbamide Dispersible Tablets in Children With Sickle Cell Disease
ClinicalTrials.gov ID: NCT06578507
What this study is testing
What is Hydroxycarbamid?
Hydroxycarbamid is an investigational medicine, given as an once-daily pill taken by mouth, being studied as a potential treatment for sickle cell disease.
Also referred to as Hydroxyurea.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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
- Phase 2: a mid-size study of how well it works
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 9 to 11
You may be able to join if
- Written informed consent, signed and dated by both parents or by the legally acceptable representative(s) of the children, and, if possible, assent...
- 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...
You likely can't join if
- 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,
See the full eligibility 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.
- 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...
- 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.
The study team makes the final eligibility decision.
Where it's taking place
- Créteil, France
- Jossigny, France
- Le Kremlin-Bicêtre, France
- Lyon, France
- Paris, France
- Cayenne, French Guiana
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 9 months to 11 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Créteil, France; Jossigny, France; Le Kremlin-Bicêtre, France; Lyon, France; Paris, France; Cayenne, French Guiana. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.