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    Summary
    EudraCT Number:2008-005667-34
    Sponsor's Protocol Code Number:G0701625
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2011-08-30
    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 ConcernedSpain - AEMPS
    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 (Zoledronate in the Prevention of Paget's).
    Ensayo aleatorio de análisis genético y terapia dirigida con ácido zoledrónico para prevenir la enfermedad de Paget en relación con el gen SQSTM1 (Zolendronato para la prevención del Paget).
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A research study to determine if a drug called Zoledronic Acid prevents a serious bone disease (Paget's Disease) in people who are known to have a change in a gene which has been linked to the disease.
    A.3.2Name or abbreviated title of the trial where available
    ZiPP study
    Estudio ZiPP
    A.4.1Sponsor's protocol code numberG0701625
    A.5.1ISRCTN (International Standard Randomised Controlled Trial) NumberISRCTN11616770
    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 Edinburgh
    B.1.3.4CountryUnited Kingdom
    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 supportMedical Research Council
    B.4.2CountryUnited Kingdom
    B.4.1Name of organisation providing supportArthritis UK
    B.4.2CountryUnited Kingdom
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationEdinburgh Clinical Trials Unit
    B.5.2Functional name of contact pointEdinburgh Clinical Trials Unit
    B.5.3 Address:
    B.5.3.1Street AddressOutpatintes Buliding, Western General Hospital, Crewe Road South
    B.5.3.2Town/ cityEdinburgh
    B.5.3.3Post codeEH4 2XU
    B.5.3.4CountryUnited Kingdom
    B.5.4Telephone number+44131537 3855
    B.5.6E-mailectu@ed.ac.uk
    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 Europharm Limited
    D.2.1.2Country which granted the Marketing AuthorisationUnited Kingdom
    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 nameÁcido zoledrónico (Aclasta) Zoledronic Acid (Aclasta)
    D.3.4Pharmaceutical form Solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous drip use (Noncurrent)
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNÁcido zoledrónico anhidro Zoledronic acid anhydrous
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number0.05
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboSolution for infusion
    D.8.4Route of administration of the placeboIntravenous drip use (Noncurrent)
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Paget's disease of the bone (PDB)
    Enfermedad ósea de Paget (PDB)
    E.1.1.1Medical condition in easily understood language
    Paget?s disease of bone (PDB) is a serious bone disease, which causes pain, arthritis and deafness and can lead to softening of the bones causing then to enlarge and become bent.
    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 LLT
    E.1.2Classification code 10033363
    E.1.2Term Paget's disease of bone
    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
    To determine if targeted intervention with Zoledronic acid can prevent
    the development of raised bone turnover and/or focal bone lesions in
    subjects who are genetically predisposed to develop PDB because they
    carry mutations in SQSTM1 that have previously been associated with
    PDB.
    Determinar si la intervención dirigida con acido zoledrónico puede evitar una elevada remodelación ósea y/o lesiones óseas localizadas en individuos que están genéticamente predispuestos a desarrollar PDB puesto que portan mutaciones que han sido previamente asociadas al PDB.
    E.2.2Secondary objectives of the trial
    1. investigate the effects of genetic testing and intervention on quality of
    life and on anxiety or depression in subjects with SQSTM1 mutations
    2. determine whether there is any difference in the biochemical makers
    which are predictive of the disease in the group who do not have the
    mutation compared to the group who have the mutation
    3. determine if specialised biomarkers of bone turnover, gene
    expression, the presence of antibodies and genetic profiling are of value
    in predicting which patients develop PDB (or complications of PDB) as
    compared with those who do not and if they are of value in predicting
    treatment response and/or the development of side effects with
    treatment.
    En este estudio se incluirá un grupo observacional que presente el mismo riesgo de desarrollar la enfermedad de Paget que la población general. El objetivo de este grupo será contestar las siguientes preguntas (dicho grupo no recibirá ningún tratamiento):
    1. ¿Someterse a un análisis genético produce un aumento de la ansiedad y la depresión, incluso si no se ha encontrado ninguna mutación en el gen SQSTM1?
    2. ¿Existen diferencias en los marcadores bioquímicos predictivos de la enfermedad en este grupo comparados con el grupo que presenta la mutación?
    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
    Criterios de inclusión en el análisis genético - Pacientes con PDB (Probandos)
    Los probandos deben presentar un diagnóstico de PDB.
    Deben tener al menos un familiar con 30 años de edad o mayor

    Criterios de inclusión en el análisis genético - Familiares
    Los familiares deben tener 30 años de edad o mayores.
    Sin diagnóstico de PDB.

    Criterios de inclusión en el estudio intervencional
    Familiares de pacientes con mutaciones en SQSTM1 con 30 años de edad o mayores que presenten mutaciones en SQSTM1.
    Todavía no diagnosticados de PDB al comienzo del estudio.

    Criterios de inclusión en el estudio observacional
    Familiares con 30 años de edad o mayores.
    Familiares a los que no se les ha detectado mutaciones en SQSTM1 en el análisis.
    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
    Severe liver or renal disease (bilirubin or transaminases elevated more than twice the upper limit of normal)
    Osteonecrosis of the jaw (ONJ)
    Estimated GFR (eGFR) < 35ml/min
    Hypocalcaemia (serum adjusted caclium <2.2mmol/l)
    Metastatic cancer or cancer diagnosed less than 2 years ago where treatment is still ongoing
    Active history of uveitis, iritis, or episcleritis
    Already taking part in another randomised controlled clinical trial

    Female patients of child bearing potential are eligible only if they
    are:
    a. not pregnant - negative serum b-hCG pregnancy test done on the baseline screening visit and a negative urine pregnancy test on the day of infusion day, with results available, prior to infusion
    b. consent to a pregnancy test prior to every dose administration
    b. non-lactating
    c. are sexually abstinent or are surgically sterile (tubal ligation or hysterectomy)
    d. if sexually active:
    must receive specific advice from their consultant about possible risks associated with getting pregnant whilst on the trial and
    must agree to practice a medically acceptable form of birth control for at least 12 months post infusion (acceptable birth control defined as the use of an IUD, a barrier method with spermicide, condoms, subdermal implant or oral contraceptives)

    Exclusion Criteria for the Observational Study
    Subjects diagnosed with PDB
    Criterios de exclusión del análisis genético (Pacientes con PDB y familiares)
    Los sujetos no desean someterse a un análisis de sangre.
    Los sujetos no desean o no pueden firmar el consentimiento informado.

    Criterios de exclusión del estudio intervencional
    Ya diagnosticados de PDB
    No desear o no poder firmar el consentimiento informado
    Los bifosfonatos están contraindicados en ese paciente.
    Recibir terapia con bifosfonatos por otra razón
    Enfermedad de hígado o riñón grave (nivel de bilirrubina o transaminasas mayor del doble del límite superior normal)
    Osteonecrosis mandibular (ONJ)
    GFR (eGFR) estimada < 35ml/min
    Hipocalcemia (niveles de calcio ajustados en suero <2.2 mmol/l)
    Cáncer metastático o cáncer diagnosticado hace menos de 2 años con tratamiento todavía en curso
    Uveitis, iritis, o episcleritis activas.
    Ya participa en otro ensayo clínico aleatorio controlado.

    Las pacientes con posibilidad de tener hijos se elegirán solo si:
    a. no están embarazadas- prueba de embarazo en suero b-hCG negativa llevada a cabo en la visita de exploración inicial y prueba de embarazo negativa en orina el día de aplicación de la dosis, con resultados disponibles antes de la administración de la dosis
    b. consentimiento de una prueba de embarazo antes de la administración de cada dosis
    c. no encontrarse amamantando
    d. presentar abstinencia sexual o ser estéril quirúrgicamente (ligación de trompas o histerectomía)
    e. si es sexualmente activa:
    debe recibir una advertencia específica de su médico sobre los posibles riesgos asociados al embarazo durante el ensayo y
    debe estar de acuerdo en llevar a cabo un control de natalidad médicamente aceptable al menos durante los 12 meses posteriores a la administración de la dosis (se incluye en control de natalidad aceptable el uso de DIU, espermicidas, condones, implantes subdérmicos o contraceptivos orales)

    Criterios de exclusión del estudio observacional
    Sujetos diagnosticados de PDB
    E.5 End points
    E.5.1Primary end point(s)
    The total number of subjects who develop new bone lesions after 5 years.
    The development of elevated bone turnover over 3 years, as measured by ALP.
    In the observational group, the primary endpoint will be anxiety / depression over 3 years, measured using the HAD scale.
    Número total de sujetos que desarrollan nuevas lesiones óseas al cabo de 5 años.
    Desarrollo de una remodelación ósea elevada después de los 3 años, medida por la ALP.
    En el grupo observacional, el punto final primordial será la ansiedad/depresión al cabo de 3 años, medida empleando la escala HDA.
    E.5.1.1Timepoint(s) of evaluation of this end point
    The total number of subjects who develop new bone lesions after 5 years.
    The development of elevated bone turnover over 3 years, as measured by ALP.
    In the observational group, the primary endpoint will be anxiety / depression over 3 years, measured using the HAD scale.
    E.5.2Secondary end point(s)
    In the biochemical marker sub-study, patients will be followed up for 5 years and investigated for the development of bone lesions. At the end of the study, we will perform a pooled analysis of data from the bone lesion sub-study and biochemical marker sub-study to determine if there is an overall effect of treatment on bone lesions.

    Quality of life, and anxiety and depression assessed by the SF-36, BPI and HADS questionnaires will be evaluated in the biochemical marker and bone-lesion sub-studies.

    The development of elevated bone turnover, as measured by ALP and quality of life, assessed by the SF-36 questionnaire will be evaluated in the observational sub-study.

    Specialised markers will be used to determine if it is possible to predict which patients develop PDB (or complications of PDB) as compared with those who do not and to determine if they are of value in predicting treatment response and/or the development of side effects with treatment.
    E.5.2.1Timepoint(s) of evaluation of this end point
    In the biochemical marker sub-study, patients will be followed up for 5 years and investigated for the development of bone lesions.
    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) No
    E.7.4Therapeutic use (Phase IV) Yes
    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 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 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 Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    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 EEA10
    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
    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
    The end of the study in terms of the participant's involvement is the last participant's final clinical follow up. The end of study is defined as the end of the funding period.
    El final del estudio en términos de la relación de los participantes es la última revisión clínica de los mismos. El final del estudio se define con el final del periodo de la subvención.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years6
    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 years6
    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.1.1.1In 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: 870
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 870
    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 Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception For clinical trials recorded in the database before the 10th March 2011 this question read: "Women of childbearing potential" and did not include the words "not using contraception". An answer of yes could have included women of child bearing potential whether or not they would be using contraception. The answer should therefore be understood in that context. This trial was recorded in the database on 2011-08-30. Yes
    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 state50
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 600
    F.4.2.2In the whole clinical trial 870
    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)
    Due to the nature of this study, longer term follow up of these subjects
    may well be an option. This will depend on end of
    trial analysis or results and continuing funding. If this occurs,
    intervention will continue.
    If this does not occur, participants will need to speak to their local
    doctor/clinician to determine if this is something they would consider
    doing.
    Debido a la naturaleza del estudio, se considera la opción de un seguimiento a largo plazo de los sujetos, lo que dependerá del final del análisis del ensayo o los resultados y de la continuidad de la subvención. Si esto sucede, entonces continuará la intervención. En caso contrario, los participantes necesitarán contactar con su médico de familia para determinar si éste considera llevarlo a cabo.
    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 Decision2012-02-02
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-01-04
    P. End of Trial
    P.End of Trial StatusOngoing
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