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    Summary
    EudraCT Number:2008-000040-13
    Sponsor's Protocol Code Number:21011075118
    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:2009-01-27
    Trial results View 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 number2008-000040-13
    A.3Full title of the trial
    Effects of recombinant human Erythropoietin on circulating and intramuscular endothelial progenitor cells, neovascularisation and oxidative metabolism of skeletal muscle in Friedreich’s Ataxia
    A.4.1Sponsor's protocol code number21011075118
    A.7Trial is part of a Paediatric Investigation Plan Information not present in EudraCT
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorMedizinische Universität Innsbruck, Univ.-Klinik für Neurologie
    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 support
    B.4.2Country
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisation
    B.5.2Functional name of contact point
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP Role
    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 Neorecormon
    D.2.1.1.2Name of the Marketing Authorisation holderRoche
    D.2.1.2Country which granted the Marketing AuthorisationAustria
    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 nameNeorecormon
    D.3.2Product code EU1/97/031-032
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation Information not present in EudraCT
    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 INNH-116PI-DE.pdf
    D.3.9.3Other descriptive nameEpoeitin beta
    D.3.10 Strength
    D.3.10.1Concentration unit IU international unit(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number16,667
    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) Information not present in EudraCT
    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 Information not present in EudraCT
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) Information not present in EudraCT
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product Information not present in EudraCT
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy Information not present in EudraCT
    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 Information not present in EudraCT
    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
    Friedreich's ataxia (FRDA) is the most common autosomal recessive neurodegenerativ disease (1:50 000) affecting the central and peripheral nervous system. Extraneural organs are also affected during the course of the disease as a significant proportian of patients develop cardiomyopathy or diabetes. FRDA is caused by a GAA triplet expansion in the FRDA gene on chromosome 9q13 resulting in a loss of function of the gene product Frataxin.
    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
    Aims of the proposed study 1) to investigate morphological and biochemical parameters of skeletal mus-cle systematically, to identify hematopoietic progenitor cells (CD34, CD133) as well as to measure baseline Frataxin expression in skeletal muscle (specimen) of FRDA patients. 2) to measure numbers of circulating CD34 and CD133 hematopoietic progenitor cells in peripheral blood of FRDA patients in response to rhuEPO treatment; 3) to study effects of rhuEPO treatment on Frataxin expression, numbers of CD34 and CD133 hematopoietic progenitor cells, satellite cells, capillary density and respiratory chain complex activi-ties of skeletal muscle tissue obtained by re-biopsy. 4) to identify changes in muscle energy metabolism (in-vivo marker) in skeletal muscle of rhuEPO treated FRDA patients applying magnetic resonance spectros-copy at baseline and study endpoint.
    E.2.2Secondary objectives of the trial
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Seven adult subjects with definite FRDA will be enroled in this study. Skeletal muscle will be ob-tained by an open biopsy from at baseline (untreated) and after 2 months of rhuEPO treatment after written informed consent. Treatment encompasses rhuEPO 3.000 IU (~100 IU/kg KG) three times weekly subcutaneously for 8 weeks. Specimen from the quadriceps muscle and MR spec-troscopy from the contralateral side will be obtained pre- and post rhuEPO treatment. Frataxin-levels will be assessed in lymphocytes and muscle specimen at baseline and study end-point. Fur-thermore, FACS analysis of circulating CD34+ and CD133+ cells in peripheral blood will be perfo-med bi-weekly. The numbers of CD34+ and CD133+ cells and capillary density per mm2 will be quantified in skeletal muscle at baseline and after 8 weeks EPO treatment. Biochemically, complex I, II, and III of the respiratory chain in the quadriceps muscle will be evaluated pre-and post treat-ment.
    E.4Principal exclusion criteria
    Exclusion criteria for participation in this study are hemoglobin-levels above 14mg/dl at the begin-ning of the study, malignancies, thrombocytosis, other diseases like chronic inflammatory disease, chronic alcohol abuse, severe diabetes mellitus type I and II (HbA1c above 8%), chronic liver in-sufficiency, epilepsy, cardiac insufficiency (NYHA above 2), history of thrombotic / thromboembolic events, anticoagulation, pregnancy and breast feeding, iron deficiency, vitamin B12 and folate de-ficiency, cardiovascular diseases, severe psychiatric disorders, known hypersensitivity to erythro-poietin, participation to another clinical trial.
    E.5 End points
    E.5.1Primary end point(s)
    1) to investigate morphological and biochemical parameters of skeletal muscle systematically, to identify hematopoietic progenitor cells (CD34, CD133) as well as to measure baseline Frataxin expression in skeletal muscle (specimen) of FRDA patients. 2) to measure numbers of circulating CD34 and CD133 hematopoietic progenitor cells in peripheral blood of FRDA patients in response to rhuEPO treatment; 3) to study effects of rhuEPO treatment on Frataxin expression, numbers of CD34 and CD133 hematopoietic progenitor cells, satellite cells, capillary density and respiratory chain complex activities of skeletal muscle tissue obtained by re-biopsy. 4) to identify changes in muscle energy metabolism (in-vivo marker) in skeletal muscle of rhuEPO treated FRDA patients applying magnetic resonance spectroscopy at baseline and study endpoint.
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis Information not present in EudraCT
    E.6.2Prophylaxis Information not present in EudraCT
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Information not present in EudraCT
    E.6.7Pharmacodynamic Information not present in EudraCT
    E.6.8Bioequivalence Information not present in EudraCT
    E.6.9Dose response Information not present in EudraCT
    E.6.10Pharmacogenetic Information not present in EudraCT
    E.6.11Pharmacogenomic Information not present in EudraCT
    E.6.12Pharmacoeconomic Information not present in EudraCT
    E.6.13Others Information not present in EudraCT
    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 No
    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) Information not present in EudraCT
    E.8.2.2Placebo Information not present in EudraCT
    E.8.2.3Other Information not present in EudraCT
    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 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 Information not present in EudraCT
    E.8.7Trial has a data monitoring committee Information not present in EudraCT
    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
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years1
    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 Information not present in EudraCT
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) Information not present in EudraCT
    F.1.1.3Newborns (0-27 days) Information not present in EudraCT
    F.1.1.4Infants and toddlers (28 days-23 months) Information not present in EudraCT
    F.1.1.5Children (2-11years) Information not present in EudraCT
    F.1.1.6Adolescents (12-17 years) Information not present in EudraCT
    F.1.2Adults (18-64 years) Information not present in EudraCT
    F.1.3Elderly (>=65 years) Information not present in EudraCT
    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 Information not present in EudraCT
    F.3.3.2Women of child-bearing potential using contraception Information not present in EudraCT
    F.3.3.3Pregnant women Information not present in EudraCT
    F.3.3.4Nursing women Information not present in EudraCT
    F.3.3.5Emergency situation Information not present in EudraCT
    F.3.3.6Subjects incapable of giving consent personally Information not present in EudraCT
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state7
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 7
    F.4.2.2In the whole clinical trial 7
    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 Decision2009-02-11
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2009-02-11
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2012-08-01
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