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    Summary
    EudraCT Number:2004-001164-41
    Sponsor's Protocol Code Number:2-55-58035-002
    National Competent Authority:Sweden - MPA
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2004-09-29
    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
    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 ConcernedSweden - MPA
    A.2EudraCT number2004-001164-41
    A.3Full title of the trial
    AN EXPLORATORY, OPEN, TWO-ARM, MATCH-CONTROL, RANDOMISED AND STRATIFIED PILOT PHASE II STUDY ASSESSING THE PREVENTION OF THE ADVERSE EFFECTS OF LONG-TERM HIGH-DOSE GLUCOCORTICOID THERAPY BY GROWTH HORMONE (NutropinAqTM) ADMINISTERED BY SUB-CUTANEOUS ROUTE IN CHILDREN WITH CHRONIC DISEASE
    A.4.1Sponsor's protocol code number2-55-58035-002
    A.7Trial is part of a Paediatric Investigation Plan Information not present in EudraCT
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorBeaufour Ipsen Pharma
    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 support
    B.4.2Country
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisation
    B.5.2Functional name of contact point
    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 Information not present in EudraCT
    D.2.1.1.1Trade name NutropinAq
    D.2.1.1.2Name of the Marketing Authorisation holderIpsen Ltd
    D.2.1.2Country which granted the Marketing AuthorisationSweden
    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 nameNutropinAq
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Information not present in EudraCT
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) Information not present in EudraCT
    D.3.11.3.1Somatic cell therapy medicinal product Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    D.3.11.3.3Tissue Engineered Product Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy Information not present in EudraCT
    D.3.11.5Radiopharmaceutical medicinal product Information not present in EudraCT
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) Information not present in EudraCT
    D.3.11.7Plasma derived medicinal product Information not present in EudraCT
    D.3.11.8Extractive medicinal product Information not present in EudraCT
    D.3.11.9Recombinant medicinal product Information not present in EudraCT
    D.3.11.10Medicinal product containing genetically modified organisms Information not present in EudraCT
    D.3.11.11Herbal medicinal product Information not present in EudraCT
    D.3.11.12Homeopathic medicinal product Information not present in EudraCT
    D.3.11.13Another type of medicinal product Information not present in EudraCT
    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
    Adverse effects of the long-term high-dose glucocorticoid administration in naïve children with chronic disease.
    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
    The primary objective of the study is to determine whether the administration of growth hormone will result in the prevention/reduction of adverse effects induced by long-term high-dose glucocorticoid therapy when administered concomitantly in children with chronic disease, evaluated by assessing the bone function, the growth and the glucidic and lipidic metabolisms.

    E.2.2Secondary objectives of the trial
    The secondary objective of the study is to assess the safety profile of patients suffering from chronic disease that requires long-term high-dose glucocorticoid therapy and receiving a daily sub-cutaneous injection of NutropinAqTM during at least 3 months and up to 12 months.
    E.2.3Trial contains a sub-study Information not present in EudraCT
    E.3Principal inclusion criteria
    Patients MUST satisfy all of the following inclusion criteria before they will be allowed to participate in the study:
    a) The patient (if applicable) and his/her parents must give written (personally signed and dated) informed consent before completing any study-related procedure, which means any assessment or evaluation that would not have formed part of the normal medical care of the patient,
    b) Male or female child aged between 4 years and bone age of 12 years for girls and bone age of 14 years for boys,
    c) Child with chronic disease condition that, based on clinical experience, requires long-term (3 months or more) and high-dose (2 mg/kg/day or more hydrocortisone-equivalent) glucocorticoid treatment: neurologic disorders (Duchene or Beckers), nephrotic syndrome, juvenile rhumatoid arthritis (JRA) or inflammatory bowel disease (IBD),
    d) Naïve patient with respect to previous glucocorticoid treatment (history of less than 2-week treatment at any time and no previous glucocorticoid treatment during the past 2 months before study entry),
    e) No investigational / commercial agents or therapies other than NutropinAqTM may be administered to the patient with the intent to treat the adverse consequences of glucocorticoid treatment in the timeframe of the study.
    E.4Principal exclusion criteria
    Patients presenting with any of the following MUST NOT be included in the study:
    a) History of hypersensitivity to any growth hormone or drugs with a similar chemical structure,
    b) History of hypersensitivity to any of the NutropinAqTM excipients (phenol, polysorbate 20, sodium citrate or citric acid)
    c) Treatment with any other investigational drug within the last 30 days before study entry,
    d) Likely to require treatment during the study with drugs that are not permitted by the study protocol (see Section 8.7),
    e) Abnormal baseline findings considered by the investigator to indicate conditions that might affect the study results, and in particular fasting glucose > 7 mmol/L, HbA1c > 5.3% or HOMA IR > 4.8,
    f) Child with a disease or syndrome affecting growth, osteoporosis, diabetes mellitus, neoplasm or multiple accidental traumas,
    g) Child with an acute critical illness or hospitalised in intensive care unit,
    h) Patient likely to be non-compliant with the protocol and the follow-up,
    i) Any mental condition rendering the subject unable to understand the nature, scope and possible consequences of the study, and/or evidence of an unco operative attitude, unless the disease under study is such that the subject presents with such symptoms and the consent of a legal guardian or carer must be sought (providing the latter is permitted by the local legislation).
    j) Any other condition that, in the opinion of the investigator, might increase the risk to the subject or decrease the chance of obtaining satisfactory data needed to achieve the objective(s) of the study.
    Under no circumstances will subjects be enrolled more than once.
    E.5 End points
    E.5.1Primary end point(s)
    As this clinical trial is an exploratory pilot Phase II study, no primaryendpoints are defined.
    The following efficacy variables will be assessed; whole body measurements, bone measurements, physical examination measurements, muscle measurements, calcium measurements, lipid metabolism measurements, quality of life measurements and hormonal functions.

    The following safety variables will be assessed; carbohydrate metabolism, Haematologyl, biochemistry, cardiographic measurements, adverse events collection and control of disease.

    Pharmacokinetic and Pharmacodynamic variables are not applicable.
    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 No
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic Information not present in EudraCT
    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) 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 Yes
    E.8.1.2Open No
    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.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    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 Information not present in EudraCT
    E.8.7Trial has a data monitoring committee Information not present in EudraCT
    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
    The end of the study for each patient is defined as the follow-up visit to be carried out 3 month after cessation of NutropinAqTM treatment.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    E.8.9.1In the Member State concerned months3
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years0
    E.8.9.2In all countries concerned by the trial months0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    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) No
    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 No
    F.2.2Male No
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients No
    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 state
    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 Decision2004-11-25
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2004-10-31
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2006-07-24
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