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    Summary
    EudraCT Number:2012-001294-84
    Sponsor's Protocol Code Number:OPBGC&RS2011-004_CA2012
    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:2012-08-07
    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 number2012-001294-84
    A.3Full title of the trial
    Prospective open label trial to evaluate the maintenance of the efficacy and the long term safety of EPI-743 in children with Leigh Disease
    Studio prospettico in aperto per valutare il mantenimento della efficacia e la sicurezza a lungo termine di EPI-743 nei bambini con malattia di Leigh
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Efficacy and safety of EPI-743 in children with Leigh Disease
    Efficacia e sicurezza di EPI-743 nei bambini con malattia di Leigh
    A.4.1Sponsor's protocol code numberOPBGC&RS2011-004_CA2012
    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 SponsorEDISON PHARMACEUTICALS INC.
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportEdison Pharmaceuticals Inc
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationOPBG C&RS Services
    B.5.2Functional name of contact pointProject Management
    B.5.3 Address:
    B.5.3.1Street Addressvia Ferdinando Baldelli, 41
    B.5.3.2Town/ cityroma
    B.5.3.3Post code00165
    B.5.3.4CountryItaly
    B.5.4Telephone number06-454037922
    B.5.5Fax number06-454037925
    B.5.6E-mailcarmelo.pantaleo@opbgcrs.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 No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community Yes
    D.2.5.1Orphan drug designation number# 10-3163
    D.3 Description of the IMP
    D.3.1Product nameEPI-743
    D.3.2Product code NA
    D.3.4Pharmaceutical form Oral solution
    D.3.4.1Specific paediatric formulation Yes
    D.3.7Routes of administration for this IMPOral use
    Gastric use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.3Other descriptive namealpha-tocotrienol quinone
    D.3.10 Strength
    D.3.10.1Concentration unit g gram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number20
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Leigh Disease
    Sindrome di Leigh
    E.1.1.1Medical condition in easily understood language
    inherited disease of the mitochondrial respiratory chain
    malattia ereditaria della catena respiratoria mitocondriale
    E.1.1.2Therapeutic area Diseases [C] - Nutritional and Metabolic Diseases [C18]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 14.1
    E.1.2Level SOC
    E.1.2Classification code 10027433
    E.1.2Term Metabolism and nutrition disorders
    E.1.2System Organ Class 10027433 - Metabolism and nutrition disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the maintenance of the efficacy of EPI-743 in patients with Leigh syndrome, who previously received EPI-743 and who continue to benefit from the use of EPI-743, on disease severity as assessed by combined scores of the Newcastle Pediatric Mitochondrial Disease Scale (NPMDS) sections 1-3.
    Valutare il mantenimento dell’efficacia dell’EPI-743 in pazienti affetti dalla sindrome di Leigh, che sono già stati in trattamento con il prodotto sperimentale e che continuano a beneficiare dal trattamento con EPI-743, attraverso la valutazione dei punteggi combinati delle sezioni 1 – 3 del questionario Newcastle Mitochondrial Disease Scale (NPMDS).
    E.2.2Secondary objectives of the trial
    To evaluate the maintenance of the efficacy of EPI-743 in patients with Leigh syndrome, who previously received EPI-743 and who continue to benefit from the use of EPI-743, on: 1.Neuromuscular function 2.Respiratory function 3.Disease morbidity and mortality To evaluate the long-term safety of EPI-743 in patients with Leigh syndrome who previously received EPI-743.
    Valutare il mantenimento dell’efficacia dell’EPI-743 in pazienti affetti dalla sindrome di Leigh, che sono già stati in trattamento con il prodotto sperimentale e che continuano a beneficiare dal trattamento con EPI-743, per quanto riguarda: 1.Funzione neuromuscolare 2.Morbilità e Mortalità della malattia 3.Funzione respiratoria 4. Valutare la sicurezza a lungo termine dell’ EPI-743 in pazienti affetti dalla sindrome di Leigh e che sono già stati in trattamento con il prodott
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Subjects with Diagnosis of Leigh syndrome who has previously received EPI-743 and continues to receive benefit from the use of EPI-743 2. Patient or parent’s or legal representative(s) able to consent and comply with protocol requirements 3. Subject (where appropriate, depending on age) and their parent(s) or legal representative(s) who have voluntarily signed the Informed Consent/Assent Form (ICF). Children will be informed about the program and asked to give assent (where appropriate, depending on age).
    1. Pazienti con diagnosi di sindrome di Leigh che sono già stati in trattamento con EPI-743 e che continuano a beneficiare dal trattamento con EPI-743 2. Espressione di un consenso informato da parte del genitore o tutore legale 3. Presenza di una persona che garantisca una compliance con lo studio
    E.4Principal exclusion criteria
    1. Any condition, which in the opinion of the investigator could compromise the subject’s safety or adherence to treatment with EPI-743. 2. Previously demonstrated clinically significant allergy or hypersensitivity to with EPI-743 or to any of the excipients of with EPI-743 (eg., sesame oil).
    1. Qualsiasi condizione che secondo lo sperimentatore possa compromettere la sicurezza del paziente e la sua aderenza al trattamento 2. Allergia o significativa ipersensibilità dimostrata precedentemente al prodotto EPI-743 o all’olio di sesamo .
    E.5 End points
    E.5.1Primary end point(s)
    Change from baseline to end of treatment (Week 144, V18) in combined score of sections 1-3 of the Newcastle Pediatric Mitochondrial Disease Scale
    Variazione rispetto all baseline della Qualità della vita misurata attraverso l’uso combinato del Newcastle Pediatric Mitochondrial Disease Scale sezione 1-3
    E.5.1.1Timepoint(s) of evaluation of this end point
    Every year for three years
    Una volta all'anno per tre anni
    E.5.2Secondary end point(s)
    -Change from baseline to end of treatment (Week 144, V18) in: 1. Scores of Movement Disorders-Childhood rating Scale and Gross Motor Function Measure for the neuromuscular function 2. Scores of Sleep Questionnaire for respiratory function 3. Mortality for disease morbidity and mortality 4. HRQOL score from section 4 of the Newcastle Pediatric Mitochondrial Disease Scale for disease morbidity and mortality 5. Glutathione cycle biomarkers -Safety will be evaluated by physical examinations, vital signs assessments, 12-lead electrocardiograms, routine clinical laboratory tests and adverse event assessments over the 144 weeks of study treatment.
    -Eventi avversi e effetti indesiderati del farmaco,parametri vitali, elettrocardiogramma, esami laboratoristici di routine. - Mantenimento dell’efficacia dell’EPI-743 sulla progressione delle malattia attraverso la valutazione dei punteggi dei questionari MD-CRS, GMFM e NPMDS sezione 4 sulla qualità della vita, dei punteggi del questionario sul sonno e, del numero degli eventi di morte e dei biomarker del glutatione.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Every two month
    Ogni due mesi
    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 No
    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) 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.8.2.4Number of treatment arms in the trial1
    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 No
    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
    LVLS
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years3
    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) Yes
    F.1.1.5.1Number of subjects for this age range: 5
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 5
    F.1.2Adults (18-64 years) No
    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 No
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    minors
    minori
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state10
    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)
    Treatment will continue until the market with follow-up visits every 2 months
    Continueranno il Trattamento fino alla commercializzazione con visite di follow up ogni 2 mesi
    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 Decision2012-09-03
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-03-14
    P. End of Trial
    P.End of Trial StatusOngoing
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