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    Summary
    EudraCT Number:2008-005667-34
    Sponsor's Protocol Code Number:ZIPP-STUDY-IT
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2011-02-08
    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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2008-005667-34
    A.3Full title of the trial
    Randomised Trial of Genetic Testing and Targeted Zoledronic acid Therapy to Prevent SQSTM1 Mediated Paget’s Disease
    Studio clinico randomizzato di analisi genetica e terapia mirata con acido zoledronico per la prevenzione della malattia ossea di Paget nei portatori della mutazione del gene SQSTM1 codice protocollo
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Randomised Trial of Genetic Testing and Targeted Zoledronic acid Therapy to Prevent SQSTM1 Mediated Paget’s Disease
    Studio clinico randomizzato di analisi genetica e terapia mirata con acido zoledronico per la prevenzione della malattia ossea di Paget nei portatori della mutazione del gene SQSTM1 codice protocollo
    A.3.2Name or abbreviated title of the trial where available
    ZIPP-STUDY-IT - CZOL446HGB16T
    ZIPP-STUDY-IT - CZOL446HGB16T
    A.4.1Sponsor's protocol code numberZIPP-STUDY-IT
    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 SponsorAZIENDA OSPEDALIERA SENESE
    B.1.3.4CountryItaly
    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 supportUniversita` degli Studi di Edimburgo, Dpt. of Rheumatology, Molacular Medicine Centre, Prof. Stuard Ralston
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationUniversita` degli Studi di Siena
    B.5.2Functional name of contact pointServizio Informazione sulla Sperime
    B.5.3 Address:
    B.5.3.1Street AddressViale Bracci 14
    B.5.3.2Town/ citySiena
    B.5.3.3Post code53100
    B.5.3.4CountryItaly
    B.5.4Telephone number0577-585468
    B.5.6E-mailgennari@unisi.it
    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*IV 1FL 100ML 0,05MG/ML
    D.2.1.1.2Name of the Marketing Authorisation holderNOVARTIS FARMA SpA
    D.2.1.2Country which granted the Marketing AuthorisationItaly
    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.3.4Pharmaceutical form Concentrate for 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 INNZoledronic acid
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.3Concentration number5
    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 Yes
    D.3.11.13.1Other medicinal product typebisfosfonati
    D.8 Information on Placebo
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboConcentrate for solution for infusion
    D.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Relatives of patients with Paget Disease of Bone that are positive for SQSTM1 mutation screening
    Familiari dei pazienti con malattia ossea di Paget risultati positivi al test genetico per le mutazioni del gene SQSTM1
    E.1.1.1Medical condition in easily understood language
    Relatives of patients with Paget Disease of Bone that are positive for SQSTM1 mutation screening
    Familiari dei pazienti con malattia ossea di Paget risultati positivi al test genetico per le mutazioni del gene SQSTM1
    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 SOC
    E.1.2Classification code 10028395
    E.1.2Term Musculoskeletal and connective tissue disorders
    E.1.2System Organ Class 10028395 - Musculoskeletal and connective tissue disorders
    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 Intervention study will be to determine whether targeted intervention with Zoledronic acid can prevent the development of new focal bone lesions in carriers of SQSTM1 gene mutations.
    Lo studio prevede la conduzione di indagini genetiche per l’identificazione delle mutazioni del gene SQSTM1 nei familiari dei sogg affetti da malattia di Paget. E’previsto anche un monitoraggio nel tempo dei sogg che sono risultati negativi per la mutazione nel gene SQSTM1 al test genetico(studio osservazionale),per verif se ci sono differenze nella qualità della vita e nei test ematici tra coloro che hanno la mutazione genetica e coloro che non ce l’hanno. L’ob. prim sarà quello di determinare se il trattamento previene lo sviluppo di lesioni ossee pagetiche nei portatori delle mutazioni
    E.2.2Secondary objectives of the trial
    we will also investigate the effects of genetic testing and intervention on bone markers and quality of life as assessed by the SF36 and the Brief Pain Inventory (BPI) questionnaire and on anxiety or depression (assessed by the HADS questionnaire) in study participants. For the observational study, the secondary objective is to determine if bone turnover is affected in non-gene carriers.
    Sara` inoltre valutato l’effetto del trattamento sull`incremento dei marker di turnover osseo, sul dolore osseo e sulla qualita` della vita,mediante l’utilizzo di appositi questionari precedentemente valicati.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Inclusion Criteria for Genetic Test - Patients with PDB (Probands) Probands must be diagnosed with PDB. Have at least one relative aged 30 years old or greater. Inclusion criteria for a Genetic Test - Relatives Relatives are aged 30 years old or greater. Relatives not yet been diagnosed with PDB. Inclusion Criteria for the Intervention Study Relatives of patients with SQSTM1 mutations aged 30 years old or greater who carry SQSTM1 mutations. Not already diagnosed with PDB at study entry. Inclusion Criteria for the Observational Study Relatives aged between 30 years old or greater. Relatives who on screening are found NOT to have SQSTM1 mutations.
    Criteri di inclusione per il test genetico I soggetti di eta` superiore ai 30 anni, che hanno una storia familiare positiva di malattia ossea di Paget ma che non hanno ancora sviluppato segni clinici. Criteri di inclusione per la randomizzazione (gruppo 1 e 2) - I soggetti di eta` superiore ai 30 anni, che hanno una storia familiare positiva di PDB che sono risultati portatori della mutazione, in assenza di segni clinici di malattia Criteri di inclusione per lo studio osservazionale (gruppo 3) - I soggetti di eta` superiore ai 30 anni, che hanno una storia familiare positiva di PDB che sono risultati non portatori della mutazione
    E.4Principal exclusion criteria
    Exclusion Criteria for Genetic Test (Patient with PDB and Relatives) Subjects not willing to have a blood sample taken. Subjects who are unwilling or unable to consent. Exclusion Criteria for the Intervention Study Already diagnosed with PDB. Unwilling or unable to consent. Bisphosphonates contraindicated. Receiving bisphosphonate therapy for another reason. Osteonecrosis of the jaw (ONJ). Estimated GFR (eGFR) < 35ml/min. Hypocalcaemia . Metastatic cancer or cancer diagnosed less than 2 years ago where treatment is still ongoing. Active uveitis, iritis, or episcleritis. Already taking part in another randomised controlled clinical trial. Pregnancy or unwillingness to practice a medically acceptable form of birth control for at least 12 months post infusion. Lactation. Exclusion waivers will not be permitted. Exclusion Criteria for the Observational Study Subjects diagnosed with PDB or SQSTM1 mutation positive
    Criteri di esclusione per il test genetico Soggetti non disponibili a sottoscrivere il modello di consenso informato al prelievo. Criteri di esclusione per la randomizzazione (gruppo 1 e 2) - Precedente diagnosi di malattia di Paget - Soggetti non disponibili a sottoscrivere il modello di consenso informato - Soggetti con controindicazione al trattamento con bisfosfonati - Soggetti gia` in trattamento, per altre ragioni, con bisfosfonati - Osteonecrosi della mandibola - Soggetti con insufficienza renale (GFR &lt; 35ml/min) - Ipocalcemia - Patologia tumorale e/o metastatica nei 2 anni precedenti lo studio - Uveite, irite o episclerite in fase attiva - Soggetti che partecipano ad altro studio clinico - Soggetti di sesso femminile in gravidanza (valutato con specifico test il giorno dell’infusione o il giorno precedente l’infusione) - Soggetti di sesso femminile, in eta` fertile, che non siano disposte ad adottare misure contraccettive adeguate per almeno 12 mesi successivi all’infusione. - Soggetti di sesso femminile in allattamento. Criteri di esclusione per lo studio osservazionale (gruppo 3) - Soggetti non disponibili a sottoscrivere il modello di consenso informato - Soggetti con malattia ostea di Paget gia` diagnosticata.
    E.5 End points
    E.5.1Primary end point(s)
    Prevention of Paget disease of bone in genetically predisposed subjects.
    Prevenzione della malattia ossea di Paget nei soggetti geneticamente predisposti
    E.5.1.1Timepoint(s) of evaluation of this end point
    5-8 years
    da 5 a 8 anni
    E.5.2Secondary end point(s)
    -the development of elevated bone turnover, as measured by ALP and other biochemical markers of bone turnover. - quality of life, and anxiety and depression assessed by the SF-36, BPI and HADS questionnaires.
    prevenire l`incremento dei parametri di turnover osseo e valutare gli effetti sulla qualità della vita
    E.5.2.1Timepoint(s) of evaluation of this end point
    5-8 years
    da 5 a 8 anni
    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 Yes
    E.6.11Pharmacogenomic Yes
    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 Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    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 Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA9
    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
    Australia
    New Zealand
    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
    LVLS 2019
    LVLS 2019
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years7
    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 years7
    E.8.9.2In all countries concerned by the trial months0
    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 No
    F.1.1Number of subjects for this age range: 0
    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) Yes
    F.1.2.1Number of subjects for this age range: 150
    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 Yes
    F.3.2Patients No
    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 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 state150
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 200
    F.4.2.2In the whole clinical trial 260
    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)
    N.A.
    non applicabile
    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 Decision2010-10-20
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2010-09-15
    P. End of Trial
    P.End of Trial StatusOngoing
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