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    Summary
    EudraCT Number:2012-000550-60
    Sponsor's Protocol Code Number:V7/22032018
    National Competent Authority:Austria - BASG
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2013-11-27
    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 ConcernedAustria - BASG
    A.2EudraCT number2012-000550-60
    A.3Full title of the trial
    Pulsed oral sirolimus in autosomal dominant polycystic kidney disease
    Gepulstes orales Sirolimus bei autosomal-dominanter polyzystischer Nierenerkrankung
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Pulsed oral sirolimus in autosomal dominant polycystic kidney disease
    Gepulstes orales Sirolimus bei autosomal-dominanter polyzystischer Nierenerkrankung
    A.3.2Name or abbreviated title of the trial where available
    The Vienna RAP Study
    Die Wiener RAP Studie
    A.4.1Sponsor's protocol code numberV7/22032018
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02055079
    A.5.4Other Identifiers
    Name:Ethic Commission Medical University of ViennaNumber:1060/2012
    Name:AGES PharmedNumber:7251768
    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 SponsorMedizinische Universität Wien, Klinische Abteilung für Nephrologie und Dialyse, Universitätsklinik für Innere Medizin 3
    B.1.3.4CountryAustria
    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 supportOesterreichische Nationalbank
    B.4.2CountryAustria
    B.4.1Name of organisation providing supportMedizinische Universität Wien
    B.4.2CountryAustria
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationMedizinische Universität Wien
    B.5.2Functional name of contact pointClinical Trials Information
    B.5.3 Address:
    B.5.3.1Street AddressWähringer Gürtel 18-20
    B.5.3.2Town/ cityWien
    B.5.3.3Post code1090
    B.5.3.4CountryAustria
    B.5.4Telephone number004314040043910
    B.5.5Fax number004314040043920
    B.5.6E-mailmarkus.riegersperger@gmail.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 Yes
    D.2.1.1.1Trade name Rapamune 1mg tablets
    D.2.1.1.2Name of the Marketing Authorisation holderPfizer Pharmaceuticals
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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 nameRapamune 1mg tablets
    D.3.4Pharmaceutical form Coated tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNSIROLIMUS
    D.3.9.1CAS number 53123-88-9
    D.3.9.4EV Substance CodeSUB10537MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1
    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 placeboCapsule, hard
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Autosomal dominant polycystic kidney disease (ADPKD) is a genetic disorder characterized by the development and uncontrolled proliferation of innumerable epithelial-lined cysts that stem from renal tubular cells, which compress and/or destroy vital renal tissue with a gradual decline in renal function, and terminal kidney failure with the need for renal reaplacement therapy. As yet, other than supportive care there is no viable therapy.
    E.1.1.1Medical condition in easily understood language
    Autosomal dominant polycystic kidney disease (ADPKD) is an incurable hereditary disease that leads to renal insufficiency and need for renal replacement therapy.
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10036046
    E.1.2Term Polycystic kidney, autosomal dominant
    E.1.2System Organ Class 100000004850
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Tubular cells, the target of mTOR-I in ADPKD, develop a resistance towards sirolimus in vitro as well as in vivo with continuous exposure to the drug. Pulsed administration of the mTOR-inhibitor sirolimus in a fixed weekly oral dose of 3 mg compared to placebo significantly reduces cyst growth and preserves excretory renal function in patients with autosomal-dominant polycystic kidney disease and an estimated glomerular filtration rate below 60 mL/min per 1.73m2.

    Null hypothesis:
    Pulsed administration of the mTOR-inhibitor sirolimus in a fixed weekly oral dose of 3 mg
    compared to placebo does not reduce cyst growth and preserve excretory renal function in
    patients with autosomal-dominant polycystic kidney disease and an estimated glomerular
    filtration rate below 60 mL/min per 1.73m2.
    E.2.2Secondary objectives of the trial
    Safety, change in proteinuria, as indicated by albumin/creatinine- and protein/creatinine ratio, respectively.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    • ADPKD, as confirmed by history, ultrasound, computed- or magnetic resonance tomography
    • Eighteen years of age, or older
    • Baseline estimated glomerular filtration rate (eGFR; 4 variable MDRD equation) below 60 mL/min per 1.73m2
    • Negative serum pregnancy test prior to administration of sirolimus and agreement to use contraception throughout the study and three months after
    • Written informed consent
    E.4Principal exclusion criteria
    • Need for renal replacement therapy
    • Pregnancy/lactation
    • Plans to become pregnant in the near future
    • Refusal to use sufficient contraception
    • Proteinuria as defined as proteine:creatinine ratio >1000 or >1g/d, respectively
    • History of life threatening complications of ADPKD
    • Evidence of active systemic- or localized major infection
    • Evidence of infiltrate or consolidation on chest X-ray
    • Use of any investigational drug or -treatment up to 4 weeks prior to enrolment and during the study
    • Known allergy/hypersensitivity to sirolimus and its derivatives
    • Medication that will interfere with the CYP3A4/CYP3A5 system
    • Total white blood cell count below or equal to 3000/mm3
    • Platelet count below or equal to 100.000/mm3
    • Fasting triglycerides above or equal to 400 mg/dL
    • Fasting total cholesterol above or equal to 300 mg/dL
    • Concomitant glomerular diseases
    • Psychiatric disorders and any condition that might prevent full comprehension of the purposes and risks of the study
    • History of malignancy, with the exception of adequately treated basal cell- and squamous cell carcinoma of the skin
    • HIV positivity
    E.5 End points
    E.5.1Primary end point(s)
    Fifty percent reduction in doubling of serum creatinine, or initiation of dialysis over a period of two years. Less or equal than 1.5 fold increase in serum creatinine without initiation of dialysis over two years is considered a beneficial outcome, increases in serum creatinine greater than 1.5 over two years or initiation of dialysis are considered a nonbeneficial outcome.
    E.5.1.1Timepoint(s) of evaluation of this end point
    After 24 months of therapy.
    E.5.2Secondary end point(s)
    Safety, change in proteinuria, as indicated by albumin/creatinine- and protein/creatinine ratio, respectively.
    E.5.2.1Timepoint(s) of evaluation of this end point
    After 24 months of therapy.
    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 No
    E.6.5Efficacy No
    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) 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 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.8.5The 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 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
    Last visit of the last subject undergoing the trial after 24 months of trial particpiation.
    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 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: 68
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 68
    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 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 state68
    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)
    Every individual who enters the trial will partake for 24 months. The expected overall trial period is 36 to 60 months. After per protocol trial termination or withdrawal each individual will have regular follow-up according to the centers practice pattern at the outpatient clinic of the Division of Nephrology and Dialysis, Department of Medicine III, Medical University of Vienna. In dependency of the individuals’ kidney function these follow-up usually range from one to four times per year.
    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 Decision2014-01-17
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-11-07
    P. End of Trial
    P.End of Trial StatusCompleted
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