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    Summary
    EudraCT Number:2009-016492-29
    Sponsor's Protocol Code Number:D3561C00002
    Clinical Trial Type:Outside EU/EEA
    Date on which this record was first entered in the EudraCT database:2011-11-17
    Trial results View 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
    H.4 THIRD COUNTRY IN WHICH THE TRIAL WAS FIRST AUTHORISED
    Expand All   Collapse All
    A. Protocol Information
    A.2EudraCT number2009-016492-29
    A.3Full title of the trial
    An Efficacy and 2-Year Safety Study of Open-label Rosuvastatin in Children and Adolescents (aged from 6 to less than 18 years) with Familial Hypercholesterolaemia
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An Efficacy and 2-year Safety Study of Rosuvastatin in Children and Adolescents (aged from 6 to less than 18 years) with Familial (inherited) Hypercholesterolaemia (high level of cholesterol in blood)
    A.3.2Name or abbreviated title of the trial where available
    CHARON
    A.4.1Sponsor's protocol code numberD3561C00002
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/229/2010
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorAstraZeneca AB
    B.1.3.4CountrySweden
    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 supportAstraZeneca AB
    B.4.2CountrySweden
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAstraZeneca
    B.5.2Functional name of contact pointInformation center
    B.5.6E-mailInformation.center@astrazeneca.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 nameCRESTOR
    D.3.2Product code AZD4522
    D.3.4Pharmaceutical form Film-coated tablet
    D.3.4.1Specific paediatric formulation No
    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 INNROSUVASTATIN CALCIUM
    D.3.9.1CAS number 147098-20-2
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number5
    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.IMP: 2
    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 nameCRESTOR
    D.3.2Product code AZD4522
    D.3.4Pharmaceutical form Film-coated tablet
    D.3.4.1Specific paediatric formulation No
    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 INNROSUVASTATIN CALCIUM
    D.3.9.1CAS number 147098-20-2
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    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.IMP: 3
    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 nameCRESTOR
    D.3.2Product code AZD4522
    D.3.4Pharmaceutical form Film-coated tablet
    D.3.4.1Specific paediatric formulation No
    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 INNROSUVASTATIN CALCIUM
    D.3.9.1CAS number 147098-20-2
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number20
    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
    Male and female children and adolescents (aged 6 to < 18 years) with FH* and at least 1 of the following criteria: 1. Fasting LDL-C>190 mg/dL (4.92 mmol/L) at baseline OR 2. Fasting LDL-C >160 mg/dL (4.10 mmol/L) at baseline in combination with evidence of other risk factors. *Familial hypercholesterolaemia is defined by a documented genetic defect in LDL receptor or Apo B or documented evidence of familial hypercholesterolaemia in a first-degree relative.
    E.1.1.1Medical condition in easily understood language
    The constant high level of cholesterol in blood
    E.1.1.2Therapeutic area Body processes [G] - Metabolic Phenomena [G03]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To assess the efficacy of rosuvastatin in paediatric patients with FH.

    To establish long-term safety, tolerability and efficacy of rosuvastatin in paediatric patients with FH.

    To characterise the PK profile of rosuvastatin in paediatric patients with FH, aged from 6 to less tha Tanner Stage II, with familial hypercholesterolaemia.
    E.2.2Secondary objectives of the trial
    To assess cIMT by sonography at baseline and every year in patients and in healthy siblings.

    To assess growth and maturation in children or adolescents with FH who are receiving long-term rosuvastatin treatment.

    Assessment of adherence to rosuvastatin during a 2-year period of treatment.

    To assess the feasibility and acceptability of the current marketed tablet formulation of rosuvastatin for use in children.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Prior to any study related procedures being performed, provision of written informed consent from a parent/both parents or guardian and statement of assent from the child or adolescent (if required by institutional review board [IRB] or independent ethics committee [IEC] according to local regulations and guidelines). Communication between the investigator, patient/guardian and child/adolescent to confirm understanding and required compliance with the requirements of the study.
    2. Male and female children and adolescents (aged 6 to less than 18 years) with FH* and at least 1 of the following criteria:
    -Fasting LDL-C >4.92 mmol/L (190 mg/dL) prior to Visit 3, per Visit 1 laboratory results (statin-naïve only) or Visit 2 laboratory results (previously treated).
    or
    -Fasting LDL-C >4.10 mmol/L (158 mg/dL) prior to Visit 3, per Visit 1 laboratory results (statin-naïve only) or Visit 2 laboratory results (previously treated) in combination with evidence of other risk factors, such as family history in first or second degree relatives of premature cardiovascular disease (CVD), defined as onset of clinical atherosclerotic disease before age 55 in males or age 65 in females at Visit 1.
    *FH is defined by a documented genetic defect in LDL-R or ApoB (by DNA analysis) or documented evidence of FH in a first-degree relative (LDL C >4.90 mmol/L [189 mg/dL] in an adult; >4.10 mmol/L [158 mg/dL] in a child <18 years of age).
    3. Patients aged between 6 and less than 10 years of age must be statin treatment naïve.
    4. Negative serum pregnancy test (b-human chorionic gonadotropin analysis [b-HCG]) prior to baseline in females of child-bearing potential.
    - Female patients of child-bearing potential must adhere to a pregnancy prevention method (abstinence, chemical or mechanical) during the study.
    - Male patients should refrain from fathering a child (including sperm donation) during the study and up to 3 months following the last dose.
    5. Willing to follow all study procedures including adherence to dietary guidelines, study visits, fasting blood draws and compliance with study treatment regimens.
    For inclusion of Healthy siblings in the study,
    1. Documented absence of the genetic defect in LDL receptor or Apo B (by DNA analysis)
    2. Historical documented LDL-C of <3.00 mmol/L without lipid lowering medication
    Healthy siblings of non-participating patients must have a sibling with FH*.
    *FH is defined by a documented genetic defect in LDL-R or ApoB (by DNA analysis) or documented evidence of FH in a first-degree relative (LDL�C >4.90 mmol/L [189 mg/dL] in an adult; >4.10 mmol/L [158 mg/dL] in a child <18 years of age).

    E.4Principal exclusion criteria
    1. History of statin-inducted myopathy or serious hypersensitivity reaction to other HMG-CoA reductase inhibitors (statins), including rosuvastatin, at Visit 1.
    2. Fasting TG ≥ 2.87 mmol/L (254 mg/dL) at Visit 1 for statin-naïve patients and at Visit 2 for patients who were on prior statin treatment.
    3. Fasting serum glucose of >9.99 mmol/L (180 mg/dL) or glycosylated haemoglobin (HbA1c) >9% at Visit 1 or patients with a history of diabetic ketoacidosis within the past year.
    4. Uncontrolled hypothyroidism defined as thyroid stimulating hormone (TSH) >1.5 times the upper limit of normal (ULN) at Visit 1 (Week –4) or patients whose thyroid replacement therapy was initiated or modified within the last 3 months prior to Visit 3 (Week 0).
    5. Current active liver disease or hepatic dysfunction (except a confirmed diagnosis of Gilbert’s disease) as defined as elevations of 1.5 times the ULN for any age in any of the following liver functions test at Visit 1 or Visit 2: ALT, AST, or bilirubin.
    6. Serum CK ≥ 3 times ULN (unless explained by exercise) at Visit 1.
    7. Estimated glomerular filtration rate (GFR) by Schwartz formula <50 mL/min at Visit 1.
    8. A ≥ 2+ proteinuria on urine dipstick at any of the screening visits: Visit 1 or Visit 2, will exclude the patient from participation.
    9. Stage 2 hypertension (systolic and/or diastolic blood pressure [BP] greater than 5 mmHg above the 99th percentile for age, gender and height) at Visit 1 and Visit 2 (where applicable).
    10. History of solid organ transplantation at Visit 1.
    11. Involvement in the planning and/or conduct of the study (applies to both AstraZeneca staff and/or staff at the study site).
    12. Previous enrolment in the present study.
    13. Participation in a clinical study where an investigational product was ingested during the last 30 days before Visit 3 (Week 0) of the current study.
    14. Any acute illness within 2 weeks before the start of the study (Visit 1).
    15. Any clinically significant abnormalities in clinical chemistry and haematology or urinalysis results at the discretion of the investigator.
    16. Any contraindication from the following: a detailed medical and drug history, a complete physical examination including vital signs, blood chemistry, haematology, coagulation factors and urinalysis.
    17. Definite or suspected personal history or family history of adverse drug reactions (ADRs), or hypersensitivity to drugs with a similar chemical structure to rosuvastatin as well as other statins.
    18. History or presence of gastrointestinal, hepatic or renal disease or other condition known to interfere with absorption, distribution, metabolism or excretion of drugs.
    19. Treatment in the previous 3 months with any drug known to have a well-defined potential for hepatotoxicity (eg, halothane).
    20. Treatment with any lipid lowering medications within 4 weeks or less of initial dosing.
    21. Clinical judgement by the investigator that the volunteer should not participate in the study.
    22. Patients weighing <20 kg (44 lbs).
    Healthy siblings should not enter the study if the following exclusion criteria are fulfilled:
    1. Participation in a study requiring ingestion of a lipid lowering therapy.
    2. Under the care of a specialist if deemed exclusionary per investigator discretion.
    Procedures for withdrawal of incorrectly enrolled patients are presented in Section 5.3.
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy parameter is the percentage reduction in LDL-C in patients from baseline to 3 months, 12 months and 24 months of treatment with 5 mg, 10 mg or 20 mg rosuvastatin.

    To establish the long-term safety, tolerability and efficacy of rosuvastatin in psediatric patients with FH.
    The assessment of growth by assessment of height (including linear growth [cm and standard deviationscore]) and secondary characteristics of sexual maturation by Tanner staging at baseline, 12 months and 24 months.

    Single dose pharmacokinetics of rosuvastatin: Cmax, tmax, and AUC(0-24).
    Population pharmacokinetics of rosuvastatin: CL/F and AUC(0-24) at steady state.
    E.5.1.1Timepoint(s) of evaluation of this end point
    baseline (week 0), 12 months and 24 months
    E.5.2Secondary end point(s)
    to assess cIMT by sonography at baseline and every year in patients and healthy siblings;
    to assess growth and maturation in children or adolescents with FH who are receiving long-term rosuvastatin treatment;
    to assess adherence to rosuvastatine during a 2-year period of treatment;
    to assess the feasibility and acceptability of the current marketed tablet formulation of rosuvastatine for use in children
    E.5.2.1Timepoint(s) of evaluation of this end point
    week 0 (baseline) and every year
    2-year period of treatment
    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 Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    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 No
    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) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised Information not present in EudraCT
    E.8.1.2Open Information not present in EudraCT
    E.8.1.3Single blind Information not present in EudraCT
    E.8.1.4Double blind Information not present in EudraCT
    E.8.1.5Parallel group Information not present in EudraCT
    E.8.1.6Cross over Information not present in EudraCT
    E.8.1.7Other Information not present in EudraCT
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    E.8.3 Will this trial be conducted at a single site globally? No
    E.8.4 Will this trial be conducted at multiple sites globally? Yes
    E.8.6 Trial involving sites outside the EEA
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3Specify the countries outside of the EEA in which trial sites are planned
    Belgium
    Canada
    Netherlands
    Norway
    United States
    E.8.7Trial has a data monitoring committee Yes
    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
    End of trial will be defined as data base lock.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months10
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 Yes
    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: 90
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 90
    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 Yes
    F.3.3.1Women of childbearing potential not using contraception For clinical trials recorded in the database before the 10th March 2011 this question read: "Women of childbearing potential" and did not include the words "not using contraception". An answer of yes could have included women of child bearing potential whether or not they would be using contraception. The answer should therefore be understood in that context. This trial was recorded in the database on 2011-11-17. Yes
    F.3.3.2Women of child-bearing potential using contraception Yes
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Ensure each patient’s parents/legal guardian provides signed and dated informed consent before conducting any procedure specifically for the study
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.2 For a multinational trial
    F.4.2.2In the whole clinical trial 200
    G. Investigator Networks to be involved in the Trial
    H.4 Third Country in which the Trial was first authorised
    H.4.1Third Country in which the trial was first authorised: Canada
    The status of studies in GB is no longer updated from 1.1.2021
    For the UK, as from 1.1.2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI
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