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    Summary
    EudraCT Number:2011-000745-21
    Sponsor's Protocol Code Number:P1200_12
    National Competent Authority:Belgium - FPS Health-DGM
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2012-09-18
    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 ConcernedBelgium - FPS Health-DGM
    A.2EudraCT number2011-000745-21
    A.3Full title of the trial
    An international, multicenter, open-label, efficacy and safety trial of intravenous zoledronic acid in infants less than one year of age, with severe osteogenesis imperfecta
    Une étude clinique multicentrique en ouvert sur d'efficacité et la sécurité de l'acide zoledronique intraveineux chez les nourrissons de moins d'un an souffrant d'une ostéogénèse imparfaite sévère.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An international, multicenter, efficacy and safety trial of intravenous zoledronic acid in infants less than one year of age, with severe osteogenesis imperfecta
    Une étude clinique multicentrique sur d'efficacité et la sécurité de l'acide zoledronique intraveineux chez les nourrissons de moins d'un an atteints d'une ostéogénèse imparfaite sévère.
    A.3.2Name or abbreviated title of the trial where available
    SHC - INFOI
    A.4.1Sponsor's protocol code numberP1200_12
    A.5.1ISRCTN (International Standard Randomised Controlled Trial) NumberISRCTN95879580
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT00982124
    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 SponsorSchriners Hospitals for Children
    B.1.3.4CountryCanada
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportShriners Hospital for Children McGill Montreal
    B.4.2CountryCanada
    B.4.1Name of organisation providing supportNovartis Pharma Canana Inc.
    B.4.2CountryCanada
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCliniques Saint Luc Université catholique de Louvain
    B.5.2Functional name of contact pointService de Rhumatologie
    B.5.3 Address:
    B.5.3.1Street AddressAvenue Hippocrate 10
    B.5.3.2Town/ cityBruxelles
    B.5.3.3Post codeB-1200
    B.5.3.4CountryBelgium
    B.5.4Telephone number+3227645395
    B.5.5Fax number+3227645394
    B.5.6E-mailgenevieve.depresseux@uclouvain.be
    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 Yes
    D.2.1.1.1Trade name ACLASTA
    D.2.1.1.2Name of the Marketing Authorisation holderNovartis
    D.2.1.2Country which granted the Marketing AuthorisationBelgium
    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 namezoledronic acid
    D.3.4Pharmaceutical form Solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNRITUXIMAB
    D.3.9.1CAS number 174722-31-7
    D.3.9.4EV Substance CodeSUB12570MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number500
    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
    Children suffering from severe Osteogenesis Imperfecta
    E.1.1.1Medical condition in easily understood language
    Children suffering from severe Osteogenesis Imperfecta
    E.1.1.2Therapeutic area Diseases [C] - Musculoskeletal Diseases [C05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level PT
    E.1.2Classification code 10031243
    E.1.2Term Osteogenesis imperfecta
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    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 of this trial is to assess the change in lumbar spine bone mineral density Z-score at month 24 relative to baseline in zoledronic acid treated infants with severe osteogenesis imperfecta aged between 2 weeks to 1 year of age at entry, compared to historical controls. The efficacy of zoledronic acid will be demonstrated if it is shown to be a gain in z-score of at least 1.
    E.2.2Secondary objectives of the trial
    To assess the effect of zoledronic acid
    • on the change in whole body bone mineral content after 12 and 24 months of treatment relative to baseline compared to historical controls in infants 2 weeks to 1 year of age.
    • on the number of clinical fractures that occur over a two year period compared to untreated historical controls in infants 2 weeks to 1 year of age.
    • on the number of vertebral compression fractures that occur over a two-year period compared to untreated historical controls in infants 2 weeks to 1 year of age as assessed by vertebral morphometry.
    •on the change in bone resorption markers after 12 and 24 months of treatment relative to baseline compared to untreated historical controls by measuring urine N-terminal telopeptide of type I collagen (u-NTX) in infants 2 weeks to 1 year of age.
    •Change in bone pain, assessed monthly, using a pain rating scale relative to baseline in infants 2 weeks to  1 year of age.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Children, male or female 2 weeks to < 12 months of age, at least at 38 weeks gestational age.
    • Any child with phenotypic OI type II, III or IV.
    • No previous treatment with bisphosphonates.
    • Negative urine protein as measured by dipstick. One repeat assessment of the urine protein will be allowed. The assessment will be made 2 weeks after the first assessment and the sample must be a urine collection after a 4-hour fast.
    E.4Principal exclusion criteria
    • Blood oxygen saturation of less than 90% in room air.
    • Serum creatinine level greater than 56 µmol/L.
    • Any clinically significant clinical laboratory abnormalities at screening.
    • Treatment with any investigational drug within the past 30 days.
    • Patients who are unlikely to be able to complete the study or comply with the visit schedule.
    • Any disease or planned therapy which will interfere with the procedures or data collection of this trial.
    E.5 End points
    E.5.1Primary end point(s)
    The efficacy of zoledronic acid will be demonstrated if it is shown to be a gain in z-score of at least 1.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Week 104
    E.5.2Secondary end point(s)
    To assess the effect of zoledronic acid
    • change in whole body bone mineral content
    • number of clinical fractures
    • number of vertebral compression fractures
    •change in bone resorption markers
    •Change in bone pain using a pain rating scale
    E.5.2.1Timepoint(s) of evaluation of this end point
    • change in whole body bone mineral content after 12 and 24 months
    • number of clinical fractures that occur over a two year period
    • number of vertebral compression fractures that occur over a two-year period
    •change in bone resorption markers after 12 and 24 months
    •Change in bone pain, assessed monthly, using a pain rating scale
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis Yes
    E.6.3Therapy Yes
    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 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 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.8.3 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 Yes
    E.8.5.1Number of sites anticipated in the EEA1
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Canada
    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
    Reasons that a patient may discontinue participation in a clinical study are considered to constitute one of the following:
    1. adverse event(s)
    2. abnormal laboratory value(s)
    3. abnormal test procedure result(s)
    4. unsatisfactory therapeutic effect
    5. subject's condition no longer requires study treatment
    6. protocol violation
    7. subject withdrew consent
    8. lost to follow-up
    9. administrative problems
    10. death
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    E.8.9.1In the Member State concerned months1
    E.8.9.1In the Member State concerned days1
    E.8.9.2In all countries concerned by the trial years5
    E.8.9.2In all countries concerned by the trial months1
    E.8.9.2In all countries concerned by the trial days1
    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: 2
    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) Yes
    F.1.1.3.1Number of subjects for this age range: 2
    F.1.1.4Infants and toddlers (28 days-23 months) Yes
    F.1.1.4.1Number of subjects for this age range: 2
    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 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 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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Children 2 weeks to <12 months
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 2
    F.4.2.2In the whole clinical trial 15
    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)
    Standard of care
    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 Decision2012-11-13
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-12-10
    P. End of Trial
    P.End of Trial StatusOngoing
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