Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register allows you to search for protocol and results information on:
  • interventional clinical trials that are conducted in the European Union (EU) and the European Economic Area (EEA);
  • clinical trials conducted outside the EU / EEA that are linked to European paediatric-medicine development.
  • Learn   more about the EU Clinical Trials Register   including the source of the information and the legal basis.


    The EU Clinical Trials Register currently displays   42567   clinical trials with a EudraCT protocol, of which   7008   are clinical trials conducted with subjects less than 18 years old.
    The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).


    Phase 1 trials conducted solely in adults and that are not part of an agreed PIP are not public in the EU CTR (refer to European Guidance 2008/C 168/02   Art. 3 par. 2 and   Commission Guideline 2012/C 302/03,   Art. 5) .

    Clinical Trials marked as "Trial now transitioned" were transitioned to the Clinical Trial Regulation 536/2014 and can be further followed in the Clinical Trial Information System  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Print Download

    Summary
    EudraCT Number:2011-006156-37
    Sponsor's Protocol Code Number:FA_BBK_8
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2012-07-26
    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 number2011-006156-37
    A.3Full title of the trial
    A double-blind, randomized, placebo-controlled, clinical trial to test the efficacy of Epoetin alfa on physical performance of Friedreich Ataxia patients.
    Studio in doppio-cieco, randomizzato, controllato con placebo, per testare efficacia, sicurezza e tollerabilita' dell'Epoetina alfa su parametri funzionali nell'Atassia di Friedreich.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Erythropoieitn in Friedreich Ataxia
    Eritropoietina nell'Atassia di Friedreich
    A.3.2Name or abbreviated title of the trial where available
    FRIEMAX
    FRIEMAX
    A.4.1Sponsor's protocol code numberFA_BBK_8
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT01493973
    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 SponsorUNIVERSITA' DEGLI STUDI DI NAPOLI FEDERICO II
    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 supportFARA
    B.4.2CountryUnited States
    B.4.1Name of organisation providing supportAISA ONLUS
    B.4.2CountryItaly
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationUniversita' degli Studi di Napoli ''Federico II''
    B.5.2Functional name of contact pointDipartimento di Scienze Neurologich
    B.5.3 Address:
    B.5.3.1Street AddressVia Pansini, 5
    B.5.3.2Town/ cityNapoli
    B.5.3.3Post code80131
    B.5.3.4CountryItaly
    B.5.4Telephone number0817432671
    B.5.5Fax number0815463663
    B.5.6E-mailfrancesco.sacca@unina.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 EPREX*1SIR 40000UI/ML 1ML
    D.2.1.1.2Name of the Marketing Authorisation holderJANSSEN CILAG 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 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 INNEPOETIN ALFA
    D.3.10 Strength
    D.3.10.1Concentration unit IU international unit(s)
    D.3.10.2Concentration typeup to
    D.3.10.3Concentration number80000
    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 Yes
    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.IMP: 2
    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 EPREX*1SIR 10000UI 1ML
    D.2.1.1.2Name of the Marketing Authorisation holderJANSSEN CILAG 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 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 INNEPOETIN ALFA
    D.3.10 Strength
    D.3.10.1Concentration unit IU international unit(s)
    D.3.10.2Concentration typeup to
    D.3.10.3Concentration number80000
    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 Yes
    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.IMP: 3
    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 BINOCRIT*1SIR 40000UI/1ML
    D.2.1.1.2Name of the Marketing Authorisation holderSANDOZ 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 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 INNEPOETIN ALFA
    D.3.10 Strength
    D.3.10.1Concentration unit IU international unit(s)
    D.3.10.2Concentration typeup to
    D.3.10.3Concentration number80000
    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 Yes
    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 injection
    D.8.4Route of administration of the placeboSubcutaneous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    FRIEDREICH ATAXIA
    ATASSIA DI FRIEDREICH
    E.1.1.1Medical condition in easily understood language
    FRIEDREICH ATAXIA
    ATASSIA DI FREIDREICH
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level PT
    E.1.2Classification code 10008025
    E.1.2Term Cerebellar ataxia
    E.1.2System Organ Class 10029205 - Nervous system disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    efficacy of Epoetin alfa on exercise capacity in patients with Friedreich Ataxia
    efficacia dell’Epoetina alfa sulla capacità fisica nei pazienti con Atassia di Friedreich
    E.2.2Secondary objectives of the trial
    • Assess the efficacy of the study drug on frataxin levels • Assess the efficacy of the study drug on cardiomyopathy • Assess the efficacy of the study drug on vascular reactivity* • Assess the efficacy of the study drug on clinical progression • Assess the safety and tolerability of the study drug • Quality of life
    •Determinare l’efficacia dell’EPO sui livelli di fratassina•Determinare l’efficacia dell’EPO sulla cardiopatia•Determinare l’efficacia dell’EPO sulla reattività vascolare*•Determinare l’efficacia dell’EPO sulla progressione di malattia•Determinare la sicurezza e tollerabilità dell’EPO•Effetto dell'EPO sulla qualità di vita
    E.2.3Trial contains a sub-study Yes
    E.2.3.1Full title, date and version of each sub-study and their related objectives
    OTHER SUBSTUDIES:
    Vascular reactivity

    ALTRI SOTTOSTUDI:
    Reattività vascolare

    E.3Principal inclusion criteria
    • Molecular diagnosis of Friedreich Ataxia • Age ≥12 years • Body weight ≥30 Kg • SARA score ≤30 • Patient able to read and sign the informed consent
    • Diagnosi molecolare di Atassia di Friedreich, con espansione GAA omozigote • Età ≥12 anni • Peso corporeo ≥30 Kg • SARA ≤30 • Pazienti in grado di comprendere il consenso informato
    E.4Principal exclusion criteria
    • Treatment with Erythropoietin in the previous 12 months • Treatment with Idebenone • Contraindications to cardiopulmonary test: valvulopathies, ischemic cardiomyopathy, atrial fibrillation, asthma, chronic obstructive pulmonary disease, other arrhythmias judged as not compatible with exercise. • Any Cardiac and/or Hepatic and/or Renal disease judged as clinically significant by the investigator (any abnormal and clinically non significant cardiac disease associated with Friedreich Ataxia is not an exclusion criteria) • Any clinically significant ECG abnormalities that may interfere with the study • Any abnormal and clinically significant laboratory exams at screening visit that may interfere with the trial • Anemia with Hemoglobin <10 g/dL • Positive history for venous and/or arterial thrombosis • Drug-resistant arterial hypertension • Positive history for drug-resistant epilepsy • Patients in treatment with not allowed study drugs (starting from 1 month prior to screening) • Any acute/chronic disease that might interfere with the clinical trial, as judged by the investigator • Hypersensitivity to Epoetin alfa or any other component of the study drug • Patients not able to comply to the study • For female patients (Sexually not active, hysterectomized, sterilized, menopause patients are excluded from the following criteria): - Pregnancy, or - Breastfeeding, or - Inadequate contraception
    • Trattamento con Eritropoietina nei 12 mesi precedenti • Trattamento con Idebenone • Controindicazioni al test cardiopolmonare: valvulopatie, cardiopatia ischemica, fibrillazione atriale, asma, BPCO, altre condizioni mediche giudicate incompatibili con il test. • Malattia cardiaca e/o epatica e/o renale giudicata come clinicamente significativa da parte dell’investigatore (qualunque anomalia cardiaca non clinicamente significativa associata all’atassia di Friedreich non rappresenta un criterio di esclusione) • Anomalie all’ECG che potrebbero interferire con lo studio • Alterazione nei parametri di laboratorio alla visita di screening che potrebbero interferire con lo studio • Anemia con emoglobina &lt;10 g/dl • Storio clinica di trombosi arteriosa e/o venosa • Ipertensione farmaco resistente • Storia clinica di epilessia farmaco resistente • Pazienti in trattamento con farmaci non permessi (con effetto retrospettivo nei 30 giorni precedenti allo screening) • Malattia acuta o cronica che, a giudizio dello sperimentatore, potrebbe interferire con lo studio • Ipersensibilità all’eritropoietina o ai suoi eccipienti • Pazienti con compliance insufficiente • Per pazienti di sesso femminile (donne sessualmente non attive, isterectomizzate, sterilizzate, in menopausa, sono escluse dai successivi criteri): - gravidanza, o - allattamento, o - contraccezione inadeguata
    E.5 End points
    E.5.1Primary end point(s)
    Primary Endpoint is the peak oxygen consumption (VO2 max) at the cardiopulmonary exercise test (CPET)
    Endpoint primario è il picco nel consumo di ossigeno (VO2 max) al test cadiopolmonare (CPET)
    E.5.1.1Timepoint(s) of evaluation of this end point
    0, 24, and 48 weeks
    0, 24, 48 settimane
    E.5.2Secondary end point(s)
    • Secondary outcome variables at the CPET • Difference in frataxin levels in peripheral blood mononuclear cells (PBMCs), at all time points • Difference in the main Echocardiographic parameters at 6, and 12 months • Difference in vascular reactivity measured with FMD at 6, and 12 months* • Difference in the clinical scale SARA at 6, and 12 months • Analysis of serious and non-serious adverse events recorded during the clinical trial • EQ-5D/ADL/IADL • 9HPT
    • Variabili secondarie alla CPET • Differenza nei liveli di fratassina nelle cellule mononucleate del sangue (PBMCs) • Differenza nei principali parametri ecocardiografici • Differenza nella reattività vascolare misurata con la FMD* • Differenza nella scala clinica SARA • Eventi seri e non-seri emersi durante la sperimentazione • EQ-5D/ADL/IADL • 9HPT
    E.5.2.1Timepoint(s) of evaluation of this end point
    0, 12, 24, 36, and 48 weeks
    0, 12, 24, 36, 48 settimane
    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 Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    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 Yes
    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 concerned5
    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
    final data analysis
    Analisi finale dei dati
    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 Yes
    F.1.1Number of subjects for this age range: 10
    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) Yes
    F.1.1.6.1Number of subjects for this age range: 10
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 50
    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 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 state56
    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)
    no program
    Nessun programma
    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 Decision2012-07-10
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-05-11
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2014-12-16
    For support, visit the EMA Service Desk , log in using your EMA account and open a ticket specifying "EU CTR" in your request.
    If you do not have an account, please visit the EMA Account management page page click on "Create an EMA account" and follow the instructions.
    The status of studies in GB is no longer updated from 1.1.2021
    For the UK, as from 1.1.2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI
    EU Clinical Trials Register Service Desk: https://servicedesk.ema.europa.eu
    European Medicines Agency © 1995-2022 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    Legal notice
    EMA HMA