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    The EU Clinical Trials Register currently displays   38003   clinical trials with a EudraCT protocol, of which   6235   are clinical trials conducted with subjects less than 18 years old.
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    Summary
    EudraCT Number:2012-002887-29
    Sponsor's Protocol Code Number:Uni-Koeln-1574
    National Competent Authority:Germany - PEI
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2012-11-07
    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 ConcernedGermany - PEI
    A.2EudraCT number2012-002887-29
    A.3Full title of the trial
    Translational therapy in patients with Osteogenesis imperfecta - a pilot trial on treatment with the RANKL-antibody Denosumab
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    New therapeutic approach in OI with the antibody Denosumab
    Neuer therapeutischer Ansatz bei OI mit dem Antikörper Denosumab
    A.3.2Name or abbreviated title of the trial where available
    OI-AK
    A.4.1Sponsor's protocol code numberUni-Koeln-1574
    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 SponsorUniversity of Cologne
    B.1.3.4CountryGermany
    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 supportUniversity of Cologne, Medical Faculty
    B.4.2CountryGermany
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationChildren's Hospital of the University of Cologne
    B.5.2Functional name of contact pointKlinisches Studienzentrum Pädiatrie
    B.5.3 Address:
    B.5.3.1Street AddressKerpenerstr 62
    B.5.3.2Town/ cityCologne
    B.5.3.3Post code50924
    B.5.3.4CountryGermany
    B.5.4Telephone number+492214784361
    B.5.5Fax number+492214783479
    B.5.6E-mailjoerg.semler@uk-koeln.de
    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 Prolia
    D.2.1.1.2Name of the Marketing Authorisation holderAmgen
    D.2.1.2Country which granted the Marketing AuthorisationGermany
    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 nameDenusomab
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNDENOSUMAB
    D.3.9.1CAS number 615258-40-7
    D.3.9.3Other descriptive nameDENOSUMAB
    D.3.9.4EV Substance CodeSUB29173
    D.3.10 Strength
    D.3.10.1Concentration unit g/l gram(s)/litre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number60
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    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) 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 Yes
    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
    In this study we will evaluate the efficacy of Denosumab in children with Osteogenesis imperfecta. Subjects will be treated every 12 weeks over 36 weeks with Denosumab 1mg/kg body weight s.c.. Efficacy will be evaluated by DXA measurements of the spine for bone mineral density.
    E.1.1.1Medical condition in easily understood language
    Efficacy of Denosumab in children with Osteogenesis imperfecta should be evaluated in this trial.
    Bei Kindern mit Glasknochen soll die Wirksamkeit von Denosumab (keine Veschlechertung der Knochendichte) bei Therapie über 36 Wochen nach 48 Wochen untersucht werden (Dosis 1mg/kgKG alle 3 Monate).
    E.1.1.2Therapeutic area Diseases [C] - Musculoskeletal Diseases [C05]
    MedDRA Classification
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Pilot study to assess the safety and efficacy of a therapy with the RANKL-antibody Denosumab in children 5-10 years of age with mutation in COL1A1 or COL1A2 leading to a defect in collagen production (Osteogenesis imperfecta). Efficacy will be assessed by DXA measurements at the lumbar spine (BMD).
    E.2.2Secondary objectives of the trial
    • Decrease of osteoclastic activity measured by
    Deoxypyridinolin (DPD) and changes of bone metabolism
    (Parathormone, N-Telopeptides, Osteocalcin).
    • Mobility of patients (Gross motor function measurement
    score)
    • Skeletal pain (visual pain scale)
    • Changes of bone mineral density of the whole body
    • Morphometry of spine (Severity Score of the spine)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • Male or female subjects between 5 years and 10 years of age with molecular proven Osteogenesis imperfecta type III/IV (COL1A1/1A2 mutation)
    • Subjects must have been treated for a minimum of 2 years with Neridronate prior to study entry
    E.4Principal exclusion criteria
    • Hypocalcemia (<1.03 mmol/l ionisized Calcium)
    • Subjects with reduced renal function (estimated GFR (Schwartz formula) <30ml/min/1.73m2)
    • Any other abnormal finding such as physical examination or laboratory evaluation, in the opinion of the investigator that is indicative of a disease that would compromise the safety of the patient when getting Denosumab s.c.
    E.5 End points
    E.5.1Primary end point(s)
    Primary efficacy endpoint:
    Changes of bone mineral density (BMD [g/cm2]) between study week 0 and 48 of the lumbar spine after 36 weeks of treatment with Denosumab
    E.5.1.1Timepoint(s) of evaluation of this end point
    Baseline Study week 0 and Study week 48
    E.5.2Secondary end point(s)
    Key secondary endpoint(s):
    • Decrease of osteoclastic activity measured by Deoxypyridinolin (DPD) and changes of bone metabolism (Parathormone, N-Telopeptides, Osteocalcin).
    • Mobility of patients (Gross motor function measurement score)
    • Skeletal pain (visual pain scale)
    • Changes of bone mineral density of the whole body
    • Morphometry of spine (Severity Score of the spine)
    E.5.2.1Timepoint(s) of evaluation of this end point
    Key secondary endpoint(s):
    • Decrease of osteoclastic activity measured by Deoxypyridinolin (DPD) and changes of bone metabolism (Parathormone, N-Telopeptides, Osteocalcin).
    Study week -12, 0, 12, 24, 36, 48.
    • Mobility of patients (Gross motor function measurement score)
    Study week 0, 24 and 48
    • Skeletal pain (visual pain scale)
    Study week -12, 0, 12, 24, 36, 48.
    • Changes of bone mineral density of the whole body study week 0 and 48
    • Morphometry of spine (Severity Score of the spine)
    Study week 0 and 48
    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 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) 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 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 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
    Last visit of the last subject
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months0
    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: 10
    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: 10
    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 age 5-10 years
    Kinder im Alter 5-10 Jahre
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state10
    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)
    Subjects will be treated individually after the end of the trial. After analyzing the DXA measurements subjects will get the opportunity to be treated with bisphosphonates or Denosumab depending on their response to the new treatment after absolving the complete trial course.
    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 Decision2013-02-08
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-02-13
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2015-01-26
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