Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
This is a prospective, interventional, phase II, open-label, multicentre, national, non-comparative study of a single administration of the new dispersible form of hydroxycarbamide at the usual dose in children with sickle cell disease who are already treated with the current form of hydroxycarbamide (Siklos® 100 mg and/or 1000 mg film-coated tablets).
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| New formulation of hydroxycarbamide | Experimental | Single arm study with a single administration of hydroxycarbamide |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Hydroxycarbamide | Drug | Single administration of the new dispersible form of hydroxycarbamide at the usual dose in children with sickle cell disease who are already treated with the current form of hydroxycarbamide. |
| Measure | Description | Time Frame |
|---|---|---|
| Acceptability score | Acceptability score evaluated by the parent(s) of the child (2-6 years old) and by the child (4-6 years old) | At Day 1 (inclusion visit at study drug administration) |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of children with acceptable acceptability score (neutral to positive scores) | Neutral to positive scores reported by the parent(s) of the child (2-6 years old), and by the child (4-6 years old) | At Day 1 (inclusion visit at study drug administration) |
| Distribution of the scores related to the ease of administration |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Bérengère Koel, MD | Hopital Universitaire Robert-Debre | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| InterCommunal Hospital Centre of Creteil | Créteil | 94000 | France | |||
| Necker University Hospital |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| ID | Term |
|---|---|
| D000755 | Anemia, Sickle Cell |
| ID | Term |
|---|---|
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| D006918 | Hydroxyurea |
| ID | Term |
|---|---|
| D014508 | Urea |
| D000577 | Amides |
| D009930 | Organic Chemicals |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Score reported by parent(s), based on a 5-point Likert scale, |
| At Day 1 (inclusion visit at study drug administration) |
| Distribution of the scores related to the ease of preparation including the ease of constitution of the liquid form and the ease to follow the prescription | Score reported by the parent(s) based on a 5-point Likert scale, | At Day 1 (inclusion visit at study drug administration) |
| Score related to the usefulness of the dispersible form, compared with the tablets currently used | Score reported by the parent(s), based on a 5-point Likert scale | At Day 1 (inclusion visit at study drug administration) |
| Free comments collected by the investigator | Questions from child/parent, reactions before/after drug intake | At Day 1 (inclusion visit at study drug administration) |
| Number of adverse events | Number of adverse events and percentage of patients reporting at least one adverse event | At Day 1 (inclusion visit at study drug administration) |
| Paris |
| 75015 |
| France |
| Robert Debré Hospital | Paris | 75019 | France |
| D006425 |
| Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |