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    Summary
    EudraCT Number:2012-005021-76
    Sponsor's Protocol Code Number:OPBGC&RS_12_003
    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:2012-12-11
    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 number2012-005021-76
    A.3Full title of the trial
    A Phase 2A Randomized, Placebo Controlled Trial of EPI-743 in Children with Rett Syndrome
    Studio di Fase 2A, Randomizzato, Controllato con Placebo, di EPI-743 in Bambini con Sindrome di Rett
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Phase 2A clinical trial conducted on children affected by Rett syndrome and who are casually administered with the experimental treatment, EPI-743, compared versus the se of placebo.
    Sperimentazione clinica di fase 2A in bambini affetti dalla malattia di Rett con assegnazione casuale del trattamento sperimentale, EPI-743 in confronto con l'utilizzo del placebo.
    A.4.1Sponsor's protocol code numberOPBGC&RS_12_003
    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 Clinical & Research Services srl
    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 number+39-06-45403-7922
    B.5.5Fax number+39-06-45403-7925
    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 number10-3163
    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.3.9.1CAS number 1213269-98-7
    D.3.9.2Current sponsor codeEPI-743
    D.3.9.3Other descriptive nameAlpha-tocotrienol quinone
    D.3.10 Strength
    D.3.10.1Concentration unit mg/kg milligram(s)/kilogram
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1.5
    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 Yes
    D.3.11.13.1Other medicinal product typeagente antiossidante
    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 placeboOral solution
    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
    Rett Syndrome
    Sindrome di Rett
    E.1.1.1Medical condition in easily understood language
    Postnatal progressive neurodevelopmental disorder due to inherited disfunction of the mitochondrial respiratory chain
    Disordine postnatale dello sviluppo neurologico dovuto ad una disfunzione 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 HLGT
    E.1.2Classification code 10021605
    E.1.2Term Inborn errors of metabolism
    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 efficacy of EPI-743 in patients with Rett syndrome, on disease progression as assessed by the Rett Syndrome clinical Severity Score (RSSS)
    Valutare l'efficacia di EPI-743, in pazienti affetti da sindrome di Rett, sulla progressione della malattia stimata attraverso il punteggio del questionario 'Rett Syndrome clinical Severity'
    E.2.2Secondary objectives of the trial
    Efficacy.To evaluate the efficacy of EPI-743 in patients with Rett syndrome, on: 1.Head circumference 2.Behavior using Rett syndrome behavioral questionnaire 3.Health related quality of life as measured by the PedsQL 4.Disease biomarkers 5.EEG 6.Polysomnography Safety. To evaluate the safety of EPI-743 in patients with Rett syndrome by: absence of dose-limiting AE’s through 6 months, defined as any adverse event ≥ grade 3, per CTCAE version 4.0, that is deemed related to treatment and not related to the underlying disease or the occurrence of any exclusion criteria including the following: 1.Allergy to EPI-743 or sesame oil 2.Liver Function tests greater than 3 times ULN 3.Renal insufficiency requiring dialysis
    Efficacia. Valutare l'efficacia di EPI-743 su: 1.Circonferenza della Testa 2.Comportamento, attraverso l'uso del questionario 'Rett Syndrome Behavioral Questionnaire' 3.Qualità della vita relativa stato di salute misurata dal questionario PedsQL 4.Biomarcatori della malattia 5.EEG 6.Polisonnografia Sicurezza. Valutare la sicurezza di EPI-743 attraverso l'assenza di eventi aversi limitanti la dose nei 6 mesi di trattamento, definiti come qualsiasi evento avverso di grado uguale o superiore a 3, in accordo ai CTCAE V 4.0, che sia considerato correlato al trattamento and non correlato alla malattia studiata o il verificarsi di uno qualsiasi dei criteri di esclusione inclusi i seguenti: 1.Allergia a EPI-743 o all'olio di sesamo; 2.Valori dei parametri di laboratorio sulla funzione epatica superiori di 3 volte ai limiti superiori di normalità. 3.Insufficienza renale che richieda dial
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1.Diagnosis of Rett syndrome Stage 1-2 2.Abnormal disease biomarkers 3.Confirmed MeCP2 mutation 4.Patient or patient’s guardian able to consent and comply with protocol requirements 5.Abstention from use of Coenzyne Q10, vitamin E and Idebenone two weeks prior enrollment of treatment with EPI-743
    1.Diagnosi di sindrome di Rett di stadio 1-2 2.Livelli anormali dei biomarker della malattia 3.Conferma di mutazione del gene MeCP2 4.Paziente o Rappresentanti del paziente in grado di dare il consenso e di soddisfare i requisiti del protocollo 5.Astensione dall'uso di Coenzima Q10, Vitamina E e Idebenone nelle due settimane precedenti l'arruolamento al trattamento con EPI-743
    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.Clinically significant allergy or hypersensitivity to EPI-743 or to any of the excipients of with EPI-743 (eg., sesame oil) 3.Clinically significant allergy or hypersensitivity to VitaminE 4.Lack of confirmation of MeCP2 mutation 5.Clinical history of bleeding or abnormal baseline PT/PTT 6.Diagnosis of any other concurrent inborn error of metabolism 7.Hepatic insufficiency with LFTs greater than 3 times upper limit of normal 8.Renal insufficiency requiring dialysis 9.End stage cardiac failure 10.Fat malabsorption syndromes precluding drug absorption
    1.Qualsiasi condizione clinica, che nell'opinione dello Sperimentatore potrebbe compromettere la sicurezza del soggetto o l'aderenza al trattamento con EPI-743 2.Allergia clinicamente significativa o ipersensibilità a EPI-743 o a qualsiasi eccipiente presente nella formulazione di EPI-743 (es. olio di sesamo) 3.Allergia clinicamente significativa o ipersensibilità alla VitaminaE 4.Mancanza di conferma della mutazione del gene MeCP2 5.Storia clinica di sanguinamento o valori di base anormali per i parametri di laboratorio PT/PTT 6.Diagnosi di qualsiasi disfunzione congenita del metabolismo 7.Insufficienza epatica, con i parametri di laboratorio della funzionalità epatica superiori di 3 volte ai valori superiori di normalità 8.Insufficienza renale che richieda dialisi 9.Grave insufficienza cardiaca 10.Sindrome da mal assorbimento dei grassi che sia in grado di interferire con l'assorbimento dei farmaci
    E.5 End points
    E.5.1Primary end point(s)
    Change from baseline to end of treatment (Week 24, V6) in score of Rett Syndrome clinical Severity Scale (RSSS)
    Cambio del punteggio del questionario 'Rett Syndrome clinical Severity' nel periodo che va dal basale alla fine del trattamento (settimana 24, V6)
    E.5.1.1Timepoint(s) of evaluation of this end point
    At 12 weeks and at 24 weeks from baseline
    A 12 settimane e a 24 dal basale
    E.5.2Secondary end point(s)
    Efficacy. Change from baseline to end of treatment (Week 24, V6) in: 1.Head circumference 2.Level of disease biomarkers 3.Score of Rett syndrome behavioral questionnaire 4.Score of PedsQL questionnaire 5.EEG parameters 6.Polysomnography parameters Safety. Safety will be evaluated by abnormal liver function and hepatic function laboratory tests and serious and non serious adverse event assessments over the 24 weeks of study treatment.
    Efficacia. Cambio, nel periodo che va dal basale alla fine del trattamento (settimana 24, V6), de: 1.Circonferenza della testa 2.Livelli dei biomarcatori della malattia 3.Punteggio del questionario Rett syndrome behavioral 4.Punteggio del questionario PedsQL 5.Parametri del EEG 6.Parametri della polisonnografia Sicurezza. La sicurezza sarà valutata attraverso i parametri di anormalità della funzionalità del rene, della funzionalità del fegato e della coagulazione e attraverso gli eventi avversi seri e non seri nel periodo di trattamento di 24 settimane.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Every 4 weeks
    Ogni 4 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 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 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 Yes
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    Monocentrico
    Monocentre
    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 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
    LSLV
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years0
    E.8.9.1In the Member State concerned months12
    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: 20
    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: 10
    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) No
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male No
    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 Yes
    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 affected by neurodevelopmental disorder
    minori affetti da disordini dello sviluppo neurologico
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state20
    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)
    Following evaluation and approval from EC, treatment will continue until the market with follow-up visits every month
    Continueranno, in seguito a valutazione ed approvazione del CE, il trattamento fino alla commercializzazione con visite di follow up ogni mese
    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 Decision2013-01-31
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2012-11-23
    P. End of Trial
    P.End of Trial StatusCompleted
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
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