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Acceptability of a New Paediatric Formulation of Hydroxycarbamide in Children With Sickle Cell Disease.

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05470270
Recruitment Status : Completed
First Posted : July 22, 2022
Last Update Posted : November 23, 2022
Sponsor:
Information provided by (Responsible Party):
ADDMEDICA SASA

Brief Summary:
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).

Condition or disease Intervention/treatment Phase
Sickle Cell Disease Drug: Hydroxycarbamide Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 33 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-label, Non-comparative, Multicentre Study to Evaluate the Acceptability of a New Paediatric Formulation of Hydroxycarbamide in Children With Sickle Cell Disease.
Actual Study Start Date : July 8, 2022
Actual Primary Completion Date : October 28, 2022
Actual Study Completion Date : October 28, 2022

Resource links provided by the National Library of Medicine

Drug Information available for: Hydroxyurea

Arm Intervention/treatment
Experimental: New formulation of hydroxycarbamide
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.




Primary Outcome Measures :
  1. Acceptability score [ Time Frame: At Day 1 (inclusion visit at study drug administration) ]
    Acceptability score evaluated by the parent(s) of the child (2-6 years old) and by the child (4-6 years old)


Secondary Outcome Measures :
  1. Percentage of children with acceptable acceptability score (neutral to positive scores) [ Time Frame: At Day 1 (inclusion visit at study drug administration) ]
    Neutral to positive scores reported by the parent(s) of the child (2-6 years old), and by the child (4-6 years old)

  2. Distribution of the scores related to the ease of administration [ Time Frame: At Day 1 (inclusion visit at study drug administration) ]
    Score reported by parent(s), based on a 5-point Likert scale,

  3. 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 [ Time Frame: At Day 1 (inclusion visit at study drug administration) ]
    Score reported by the parent(s) based on a 5-point Likert scale,

  4. Score related to the usefulness of the dispersible form, compared with the tablets currently used [ Time Frame: At Day 1 (inclusion visit at study drug administration) ]
    Score reported by the parent(s), based on a 5-point Likert scale

  5. Free comments collected by the investigator [ Time Frame: At Day 1 (inclusion visit at study drug administration) ]
    Questions from child/parent, reactions before/after drug intake

  6. Number of adverse events [ Time Frame: At Day 1 (inclusion visit at study drug administration) ]
    Number of adverse events and percentage of patients reporting at least one adverse event



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Ages Eligible for Study:   2 Years to 6 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Written informed consent, signed and dated by both parents or by the legally acceptable representative(s) of the children,
  • Child with sickle cell disease, treated with 100 mg and/or 1000 mg Siklos® film-coated tablets at the same daily dose for more than 4 weeks,
  • Child aged between 2 and 6 years old,
  • Parents capable of communicating with the investigator and understanding the requirements and constraints of the study protocol and willing to comply with the study requirements,
  • Children affiliated to a social security plan (including universal health coverage) or beneficiary of a similar insurance plan.

Exclusion Criteria:

  • Participation in any other clinical study for any other pharmaceutical product within 4 weeks preceding study inclusion,
  • Known hypersensitivity or allergy to the excipients,
  • Any surgical or medical condition or any significant illness (of which severe hepatic impairment (Child-Pugh classification C), severe renal impairment, toxic ranges of myelosuppression) 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05470270


Locations
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France
InterCommunal Hospital Centre of Creteil
Créteil, France, 94000
Necker University Hospital
Paris, France, 75015
Robert Debré Hospital
Paris, France, 75019
Sponsors and Collaborators
ADDMEDICA SASA
Investigators
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Principal Investigator: Bérengère Koel, MD Hopital Universitaire Robert-Debre
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Responsible Party: ADDMEDICA SASA
ClinicalTrials.gov Identifier: NCT05470270    
Other Study ID Numbers: SIK-FR-22-1
First Posted: July 22, 2022    Key Record Dates
Last Update Posted: November 23, 2022
Last Verified: November 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by ADDMEDICA SASA:
hydroxycarbamide
pediatric
acceptability
Additional relevant MeSH terms:
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Anemia, Sickle Cell
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
Hydroxyurea
Antineoplastic Agents
Antisickling Agents
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Nucleic Acid Synthesis Inhibitors