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    Summary
    EudraCT Number:2017-004972-74
    Sponsor's Protocol Code Number:20160227
    National Competent Authority:Hungary - National Institute of Pharmacy
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2020-07-10
    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 ConcernedHungary - National Institute of Pharmacy
    A.2EudraCT number2017-004972-74
    A.3Full title of the trial
    An Open-label, Ascending Multiple dose Study to Evaluate Safety,
    Tolerability, Pharmacokinetics, and Pharmacodynamics of Romosozumab in Children and Adolescents With Osteogenesis Imperfecta
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    An Open-label, Ascending Multiple dose Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Romosozumab in Children and Adolescents with Osteogenesis Imperfecta
    A.4.1Sponsor's protocol code number20160227
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/066/2016
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorAmgen Inc.
    B.1.3.4CountryUnited States
    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 supportAmgen Inc
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAmgen Kft.
    B.5.2Functional name of contact point
    B.5.3 Address:
    B.5.3.1Street AddressSzabadság tér 7.
    B.5.3.2Town/ cityBudapest
    B.5.3.3Post code1054
    B.5.3.4CountryHungary
    B.5.4Telephone number+361354 4700
    B.5.6E-maileu-hu-medinfo@amgen.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 nameRomosozumab
    D.3.2Product code AMG 785
    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 INNROMOSOZUMAB
    D.3.9.1CAS number 909395-70-6
    D.3.9.2Current sponsor codeAMG 785
    D.3.9.3Other descriptive nameAMG 785
    D.3.9.4EV Substance CodeSUB187492
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number90
    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 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
    Osteogenesis imperfecta (OI) is a group of genetic skeletal disorders
    characterized by increased bone fragility, low bone mass , and increased
    bone turnover contributing to osteoporosis, fractures, and other
    conditions. OI is the most common form of primary osteoporosis in
    children with an estimated incidence of 1 per 25,000 live births.
    E.1.1.1Medical condition in easily understood language
    Osteogenesis Imperfecta (Brittle bone disease)
    E.1.1.2Therapeutic area Body processes [G] - Bones and nerves physological processes [G11]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    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
    To evaluate the pharmacokinetics (PK) profile following multiple SC doses of romosozumab in children and adolescents with OI
    E.2.2Secondary objectives of the trial
    To evaluate the safety, tolerability, and immunogenicity profile
    following multiple SC doses of romosozumab in children and adolescents
    with OI

    To evaluate the pharmacodynamic (PD) profile following multiple SC doses of romosozumab in children and adolescents with OI
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Subject's legally acceptable representative has provided informed
    consent and the subject has provided written assent based on local
    regulations and/or guidelines prior to initiation of any study-specific
    activities/procedures
    Ambulatory male or female children 5 to less than 12 years of age
    (cohorts 2,4, and 6) or adolescents 12 to less than 18 years of age
    (cohorts 1, 3 and 5) upon entry into screening
    Clinical diagnosis of OI defined as a clinical history consistent with type
    I-IV OI as determined by presence of expected phenotype (eg, facial
    shape, voice, blue sclera, dentinogenesis imperfecta, typical radiographic
    features, fracture pattern) and lack of additional features unrelated to
    type I-IV OI (eg, blindness, mental retardation, neuropathy,
    craniosynostosis, premature exfoliation of deciduous teeth)
    • If familial, also must be autosomal dominant
    E.4Principal exclusion criteria
    History of an electrophoresis pattern inconsistent with type I to type IV
    OI
    History of known mutation in a gene other than collagen type I
    alpha/collagen type I alpha 2 (COL1AI/COL1A2) causing OI or other
    metabolic bone disease
    History of congenital dislocation of the radial head, interosseous
    membrane calcification, or exuberant callus formation
    History of osteomalacia or rickets
    Body weight less than 10 kg or greater than 90 kg
    History of other bone diseases that affect bone metabolism (eg,
    osteoporosis pseudoglioma syndrome, idiopathic juvenile osteoporosis,
    osteopetrosis, hypophosphatasia)
    History of Kawasaki disease, rheumatic myocarditis, ischemic
    cardiomyopathy, inherited cardiomyopathies, valvular heart disease,
    nephrotic syndrome, familial hypercholesterolemia, stroke, or any
    thromboembolic disorder
    Evidence of untreated or unhealed oral cavities or oral infections
    Unhealed or planned invasive dental or tooth procedure; removal of baby
    teeth is acceptable and not considered an invasive dental procedure
    Unhealed fracture as defined by orthopedic opinion
    Osteotomy, rodding surgery or spinal fusion surgery within 5 months
    prior to screening, or not yet healed per orthopedic surgeon
    Any planned major surgery, including skeletal surgery (eg, rodding
    surgery, spinal surgery) within the next 6 months from Day 1 that would
    interfere with study procedures or would require missing of any IP
    Symptoms associated with skull abnormalities such as basilar
    invagination, basilar impression or Chiari malformation (headache
    induced by coughing or straining for stool, or parasthesia or weakness)
    History of malabsorption (in children with serum albumin < lower limit
    normal [LLN]), malabsorption should be clinically ruled out by the
    investigator to confirm eligibility)
    History of long QT syndrome
    History of malignancy
    History of any solid organ or bone marrow transplant
    Positive blood screen for hepatitis B surface antigen (HbsAg), hepatitis B
    core antibody (HBcAb), or hepatitis C antibody (HepCAb), human
    immunodeficiency virus (HIV) -1 or -2 antibody
    History of hyper- or hypothyroidism, unless subject is on stable therapy
    > 6 months and has supporting laboratory documentation within 6
    months prior to or at screening indicating normal serum thyroidstimulating
    hormone [TSH] value
    Evidence of any of the following:
    Current hyper- or hypoparathyroidism (parathyroid hormone outside the
    normal range)
    Renal disease: Estimated glomerular filtration rate (eGFR) < 60
    mL/min/1.73 m2 (calculated by the bedside Schwartz equation at
    screening)
    eGFR (mL/min/1.73 m2)= 0.413 X (height/serum creatinine)
    (Height is in centimeters, and serum creatinine is in mg/dL)
    Current hypocalcemia (albumin-adjusted serum calcium < LLN of the
    laboratory's reference range) or hypercalcemia (albumin-adjusted serum
    calcium > ULN of the laboratory's reference range) at the time of
    screening. Serum calcium levels may be retested once in case of an
    elevated serum calcium level within 1.1 x ULN of the laboratory's
    reference range.
    Serum phosphorous < LLN
    Vitamin D insufficiency, defined as 25OHD levels < 20 ng/mL; vitamin D repletion will be allowed and subjects may be retested.
    Aspartate aminotransferase (AST), alanine aminotransferase (ALT) > 1.5
    x upper limit of normal (ULN)
    Total bilirubin (TBL) > 1.5 x ULN (subjects with Gilbert syndrome are
    eligible)
    Prior treatment with:
    romosozumab or other anti-sclerostin antibody
    fluoride or strontium for bone disease
    parathyroid hormone (PTH) or PTH derivatives within 12 months prior to
    screening
    Denosumab within 12 months prior to first dose of romosozumab
    zoledronic acid within 6 months prior to first dose of romosozumab
    oral bisphosphonates or intravenous bisphosphonates other than
    zoledronic acid if the first dose of romosozumab would be before their
    next scheduled bisphosphonate dose would have been given
    Subject unwilling to stop IV or oral bisphosphonate prior to the first
    dose of IP
    Administration of any of the following treatment within 3 months prior
    to screening:
    Systemic glucocorticoids (≥ 5.0 mg prednisone equivalent/day for more
    than 10 days) within 3 months prior to screening. Topical and inhaled
    glucocorticoids will be allowed
    Growth hormone (subjects on stable dose of growth hormone for at least
    3 months prior to screening will be allowed)
    Calcitonin
    Other bone active drugs including anticonvulsants (except gabapentin
    and benzodiazepines) and heparin
    Chronic systemic ketoconazole, androgens (except subjects who have
    received testosterone therapy for physiologic replacement in the setting
    of documented hormonal deficiency), adrenocorticotropic hormone
    (ACTH), cinacalcet, aluminum, lithium, protease inhibitors,
    gonadotropin-releasing hormone agonists
    E.5 End points
    E.5.1Primary end point(s)
    Romosozumab serum PK parameters: maximum-observed concentration
    (Cmax), time to Cmax (tmax), area under the curve (AUC) and terminal
    half life (t1/2)
    E.5.1.1Timepoint(s) of evaluation of this end point
    Investigational product will be dosed on study days 1, 29 and 57 after completion of all pre-dose procedures. Safety assessments, including blood samples for anti-romosozumab antibodies, PK
    and PD measurements and time points are defined in the Schedule of Assessments as per the Protocol.
    E.5.2Secondary end point(s)
    • Treatment-emergent adverse events, including events of injection
    site reactions and changes in cranial nerve function.Vital signs,
    electrocardiograms, physical examinations, and safety laboratory tests,
    including serum calcium
    • Incidence of anti-romosozumab antibodies
    • Bone turnover markers including serum P1NP and serum CTX
    measurements.
    • Lumbar spine BMD, bone mineral content (BMC), bone area, and BMD
    Z-score as assessed by dual-energy X-ray absorptiometry (DXA)
    E.5.2.1Timepoint(s) of evaluation of this end point
    Investigational product will be dosed on study days 1, 29 and 57 after completion of all pre-dose procedures. Safety assessments, including blood samples for anti-romosozumab antibodies, PK
    and PD measurements and time points are defined in the Schedule of Assessments as per the Protocol.
    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 No
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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) Yes
    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) 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.8.3 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.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA13
    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
    France
    Germany
    Greece
    Hungary
    Italy
    Spain
    Turkey
    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
    Defined as the date when the last subject is assessed or receives an intervention for evaluation in the study (ie, last subject last visit), following any additional parts in the study (eg, long-term follow-up), as applicable
    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
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months6
    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: 12
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 12
    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 No
    F.3.3.1Women of childbearing potential not using contraception No
    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 No
    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 16
    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 Decision2020-09-15
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-08-28
    P. End of Trial
    P.End of Trial StatusOngoing
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