A Phase 2 interventional study of Hydroxycarbamide in Sickle Cell Disease, sponsored by Theravia. Completed at 3 sites in France. Open to participants aged 2 Years to 6 Years. Per ClinicalTrials.gov, last updated 2022-11-23.
Sponsored by Theravia (part of Norgine) · Phase 2, Interventional, and Treatment
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).
1,134 studies on the registry are indexed under Anemia, Sickle Cell; 238 are open to participants now.
This study's enrollment of 33 is below the median of 40 across 769 interventional studies indexed under Anemia, Sickle Cell.
Browse Anemia, Sickle Cell studies →Theravia is the lead sponsor of 8 studies on the registry; 1 is open to participants now.
First submitted before Theravia became part of Norgine.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
A score of how well the liver works in people with liver disease such as cirrhosis: class A is mild, B moderate and C severe.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Excipients
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Child-Pugh class
A score of how well the liver works in people with liver disease such as cirrhosis: class A is mild, B moderate and C severe.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Single arm study with a single administration of hydroxycarbamide
Drug: Hydroxycarbamide
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.
Acceptability score
Acceptability score evaluated by the parent(s) of the child (2-6 years old) and by the child (4-6 years old)
Time frame: At Day 1 (inclusion visit at study drug administration)
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)
Time frame: At Day 1 (inclusion visit at study drug administration)
Distribution of the scores related to the ease of administration
Score reported by parent(s), based on a 5-point Likert scale,
Time frame: 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,
Time frame: 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
Time frame: At Day 1 (inclusion visit at study drug administration)
Free comments collected by the investigator
Questions from child/parent, reactions before/after drug intake
Time frame: 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
Time frame: At Day 1 (inclusion visit at study drug administration)
Plan to share: No
No publications or documents are linked to this record.
This study is completed, as verified in Nov 2022. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Hydroxyurea