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    The EU Clinical Trials Register currently displays   38870   clinical trials with a EudraCT protocol, of which   6391   are clinical trials conducted with subjects less than 18 years old.
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    Summary
    EudraCT Number:2004-001666-40
    Sponsor's Protocol Code Number:CZOL446H2202E1
    National Competent Authority:Hungary - National Institute of Pharmacy
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2004-11-25
    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 ConcernedHungary - National Institute of Pharmacy
    A.2EudraCT number2004-001666-40
    A.3Full title of the trial
    An international, multicenter, randomized, open-label, safety and
    efficacy trial of intravenous zoledronic acid administered either
    once or twice yearly in children with severe osteogenesis
    imperfecta, a 1-year extension to CZOL446H2202
    A.3.2Name or abbreviated title of the trial where available
    2202E
    A.4.1Sponsor's protocol code numberCZOL446H2202E1
    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 SponsorNovartis Pharma AG
    B.1.3.4CountrySwitzerland
    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.2.5The 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.3.2Product code ZOL446
    D.3.4Pharmaceutical form Solution for infusion
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    D.3.7Routes of administration for this IMPIntravenous use
    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) Information not present in EudraCT
    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 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 No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    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 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
    Osteogenesis Imperfecta
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Information not present in EudraCT
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    to examine the long-term safety of two different dosage regiments of zoledronic acid over an additional 12 months in pediatric patients who have completed one-year of treatment of zoledronic acid in the core CZOL446H2202 study, with focus on general safety and renal safety.
    E.2.2Secondary objectives of the trial
    to assess continued efficacy, compared to baseline (visit 1 of core) :
    • % change in lumbar spine bone mineral density at mo 18 & 24
    • change in Z score of lumbar spine at mo18 & 24
    • # clinical fractures per patient over 12-mo ext & 24-mo total
    • change in bone resorption / formation markers at mo15, 18, 21 & 24
    • change in supine length/height at mo 15, 18, 21 & 24
    • change in bone pain
    • change in bone mineral content of total body at mo 18 & 24
    • change in cortical bone thickness at mo 24
    • change in vertebral spine length at mo 24
    • change in grip strength at mo 15, 18, 21 & 24
    • % of patients need treatment before scheduled dosing visits during the extension
    E.2.3Trial contains a sub-study Information not present in EudraCT
    E.3Principal inclusion criteria
    • Children, male or female, between 1 year and 17 years of age at visit 1 of the extension, all inclusive, who have completed 1 year treatment of zoledronic acid or pamidronate in CZOL446H2202 trial.
    E.4Principal exclusion criteria
    • Any disease or planned therapy which will interfere with the procedures or data collection of this trial.
    • Any disease or abnormality that would prevent accurate bone-mineral density measurements of the lumbar spine (e.g., intra-abdominal calcification that may prohibit accurate data collection/interpretation; severe scoliosis, kyphosis, or metal implants, etc.)
    • Any surgical bone-lengthening procedure, e.g. Ilizarov procedure planned/scheduled to occur during the trial.
    • Female patients of child-bearing potential are eligible only if they are: not pregnant (serum β-hCG pregnancy-test negative)/non-lactating; are sexually abstinent or are surgically sterile (tubal ligation or hysterectomy): and if sexually active, must be practicing a medically acceptable form of birth control, defined as the use of an IUD, a barrier method with spermicide, condoms, subdermal implant or oral contraceptives. Females of child-bearing potential who are sexually active must agree to continue to practice their birth control during the trial and at least 1 year after completing the trial and must consent to a pregnancy test prior to every dose administration.
    • Renal abnormality (please see section 7.5.1): defined as a serum-creatinine value above the upper limit of normal (age- and sex-matched) or a urine dipstick greater than 2 + protein.
    • Hypocalcemia: any value (age-matched) below the normal range.
    • History or evidence of an intestinal malabsorption syndrome.
    • Patients with a significant drug-related adverse event in the original protocol.
    • Patients who discontinued treatment in the original protocol ZOL446H2202.
    E.5 End points
    E.5.1Primary end point(s)
    • Bone-mineral density (BMD) measurements (lumbar spine and total body) by dual energy absorptiometry (DEXA)
    • X-rays of the vertebral spine and the left hand: to determine vertebral spine length, bone cortical thickness and number of clinical fractures.
    • Specialized serum tests to evaluate bone resorption and formation will be performed for C-telopeptide (CTX), N-terminal propeptide of type I collagen (P1NP), parathyroid hormone (PTH) and bone specific alkaline phosphatase (SAP).
    • Bone Pain Assessment using the pediatric Wong-Baker FACES Pain Rating Scale.
    • Grip Strength using dynamometry
    • Clinical fractures
    • Height using stadiometer
    • Incidence of patients who need treatment before scheduled dosing visit.
    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 No
    E.8.4 The trial involves multiple sites in the Member State concerned Yes
    E.8.5The trial involves multiple Member States Yes
    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 Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    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
    if the trial is endangering the safety of the patients
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    E.8.9.1In the Member State concerned months2
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months2
    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) Yes
    F.1.1.5Children (2-11years) Yes
    F.1.1.6Adolescents (12-17 years) Yes
    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 Information not present in EudraCT
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Information not present in EudraCT
    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
    Pediatric patients
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state5
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 70
    F.4.2.2In the whole clinical trial 130
    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 Decision2005-02-01
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2005-01-12
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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