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    Summary
    EudraCT Number:2022-000168-22
    Sponsor's Protocol Code Number:SIK-FR-22-1
    National Competent Authority:France - ANSM
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2022-03-11
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedFrance - ANSM
    A.2EudraCT number2022-000168-22
    A.3Full title of the trial
    An open-label, non-comparative, multicentre study to evaluate the acceptability of a new paediatric formulation of hydroxycarbamide in children with sickle cell disease
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Evaluation of a new paediatric formulation of hydroxycarbamide in children with sickle cell disease
    A.4.1Sponsor's protocol code numberSIK-FR-22-1
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorAddmedica
    B.1.3.4CountryFrance
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportAddmedica
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationADDMEDICA
    B.5.2Functional name of contact pointLaura Fabre Thomas-Bourgneuf
    B.5.3 Address:
    B.5.3.1Street Address37 rue de Caumartin
    B.5.3.2Town/ cityParis
    B.5.3.3Post code75009
    B.5.3.4CountryFrance
    B.5.4Telephone number33172 69 01 86
    B.5.6E-maillaura.thomas-bourgneuf@addmedica.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameHydroxycarbamide
    D.3.4Pharmaceutical form Dispersible tablet
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNHYDROXYCARBAMIDE
    D.3.9.1CAS number 127-07-01
    D.3.9.4EV Substance CodeSUB08076MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number50
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Drepanocytosis
    E.1.1.1Medical condition in easily understood language
    Sickle cell disease
    E.1.1.2Therapeutic area Body processes [G] - Genetic Phenomena [G05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.0
    E.1.2Level LLT
    E.1.2Classification code 10051835
    E.1.2Term Drepanocytosis
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective is to evaluate the acceptability of the
    hydroxycarbamide 50 mg film-coated dispersible tablets in 2- to 6-
    year old children with sickle cell disease.
    E.2.2Secondary objectives of the trial
    • To calculate the proportion of children with an acceptable
    acceptability score (neutral to positive scores),
    • To evaluate the ease of administration of the
    hydroxycarbamide dispersible tablets, reported by the
    parent(s),
    • To evaluate the ease of preparation of the solution reported
    by the parent(s),
    • To assess the usefulness of the new form (hydroxycarbamide
    dispersible tablets) compared with the form currently used
    (Siklos® 100 mg and 1000 mg film-coated tablets),
    • To collect free comments (collection by the investigator)
    about the study product and administration made by the
    child/parent(s),
    • To assess the safety profile of the hydroxycarbamide
    dispersible tablets.
    E.2.3Trial contains a sub-study No
    E.3Principal 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® filmcoated 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.
    E.4Principal 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 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.
    E.5 End points
    E.5.1Primary end point(s)
    The acceptability score reported by the parent(s) of the child (2-6 years old) on a 5-point Likert scale and reported by the child (4-6 years old) on a 5-point hedonic scale will be summarised using descriptive statistics.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Visit inclusion
    E.5.2Secondary end point(s)
    • A neutral to positive acceptability score (scores 3-5) will be considered as
    acceptable. The percentages of children with acceptable acceptability scores,
    reported by the child (based on a 5-point hedonic scale) and reported by the parent
    (based on a 5-point Likert scale), will be calculated. A value of 80% of the sample
    population in agreement that the product is acceptable is generally considered to be
    the current standard requirement (8).
    • Distribution of the scores related to the ease of administration, reported by
    parent(s), based on a 5-point Likert scale, will be analysed in the FAS and in the
    PPS.
    • 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 (calculation
    of the number of tablets), reported by the parent(s) based on a 5-point Likert scale,
    will be analysed in the FAS and in the PPS.
    • Score related to the usefulness of the hydroxycarbamide dispersible form, reported
    by the parent(s), based on a 5-point Likert scale (when asked to compare it with the
    usual Siklos® tablets) will be analysed in the FAS and in the PPS.
    • Free comments, reactions before/after drug intake and questions by the child and
    the parents will be displayed in a listing of comments and not further analysed
    statistically.
    • Safety analysis: the frequency and severity of AEs will be analysed in the SS
    using descriptive statistics and displayed by system organ class (SOC) and
    preferred term (PT) according to the Medical Dictionary for Regulatory Activities
    (MedDRA) terminology.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Visit inclusion
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy No
    E.6.4Safety No
    E.6.5Efficacy No
    E.6.6Pharmacokinetic No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic No
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    Evaluation of the acceptability of of a new paediatric formulation of hydroxycarbamide.
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group No
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    E.8.3 The trial involves single site in the Member State concerned No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.4.1Number of sites anticipated in Member State concerned3
    E.8.5The trial involves multiple Member States No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years0
    E.8.9.1In the Member State concerned months4
    E.8.9.1In the Member State concerned days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    F.1.1Number of subjects for this age range: 45
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 45
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations No
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state45
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    None
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2022-04-25
    N.Ethics Committee Opinion of the trial application
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2022-10-28
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