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    Summary
    EudraCT Number:2016-000360-42
    Sponsor's Protocol Code Number:UX001-CL302
    National Competent Authority:Italy - Italian Medicines Agency
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2018-02-07
    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 ConcernedItaly - Italian Medicines Agency
    A.2EudraCT number2016-000360-42
    A.3Full title of the trial
    A Phase 3b Open-label Extension Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients with GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM).
    Studio di estensione di fase 3b, in aperto, per valutare la sicurezza e l’efficacia delle compresse di acido aceneuramico a rilascio prolungato (Ace-ER) in pazienti affetti da miopatia GNE (GNEM) o miopatia ereditaria da corpi inclusi (HIBM).
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to see if aceneuramic acid is safe and effective in treating people with Hereditary Inclusion Body Myopathy (HIBM), a rare muscle disease.
    Uno Studio per valutare la sicurezza ed efficacia dell'acido aceneuramico nel trattamento di pazienti affetti da Miopatia a Corpi Inclusi (HIBM), una rara malattia muscolare.
    A.3.2Name or abbreviated title of the trial where available
    A study to see if aceneuramic acid is safe and effective in treating people with Hereditary Inclusio
    Uno Studio per valutare la sicurezza ed efficacia dell'acido aceneuramico nel trattamento di pazient
    A.4.1Sponsor's protocol code numberUX001-CL302
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02736188
    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 SponsorULTRAGENYX PHARMACEUTICAL 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 supportULTRAGENYX PHARMACEUTICAL INC.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationUltragenyx Pharmaceutical Inc
    B.5.2Functional name of contact pointClinical Operations
    B.5.3 Address:
    B.5.3.1Street Address 60 Leveroni Court
    B.5.3.2Town/ cityNovato, CA
    B.5.3.3Post code94949
    B.5.3.4CountryUnited States
    B.5.4Telephone number0014154838800
    B.5.5Fax number0014154838820
    B.5.6E-mailinfo@ultragenyx.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 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 numberEU/3/12/972
    D.3 Description of the IMP
    D.3.1Product nameAceneuramic Acid; Common Name: Sialic Acid
    D.3.2Product code UX001
    D.3.4Pharmaceutical form Tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMP
    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 Information not present in EudraCT
    D.3.11.3.2Gene therapy medical product Information not present in EudraCT
    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 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
    GNE Myopathy, also known as Hereditary Inclusion Body Myopatha\y (HIBM), Distal Myopathy with Rimmed Vacuoles (DMRV), Nonaka's disease, or quadriceps sparing myopathy (QSM)
    Miopatia GNE, nota anche come Miopatia ereditaria a Corpi Inclusi(HIBM), miopatia distale con vacuoli cerchiati (DMRV), morbo di Nonaka, o miopatia con risparmio del quadricipite (QSM).
    E.1.1.1Medical condition in easily understood language
    Myopathy
    Miopatia
    E.1.1.2Therapeutic area Diseases [C] - Musculoskeletal Diseases [C05]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level PT
    E.1.2Classification code 10077945
    E.1.2Term GNE myopathy
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10075048
    E.1.2Term Hereditary inclusion body myopathy
    E.1.2System Organ Class 10010331 - Congenital, familial and genetic disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Evaluate the long-term safety of Ace-ER treatment in subjects with GNE Myopathy
    Valutare la sicurezza e l’efficacia a lungo termine del trattamento con Ace-ER in soggetti
    affetti da miopatia GNE.
    E.2.2Secondary objectives of the trial
    Overall Efficacy Endpoints: Evaluate the long-term effect of 6 g/day of Ace-ER treatment in subjects with GNEM. Efficacy will be evaluated as follows:

    Enrolling from UX001-CL301:
    • Muscle strength as measured by dynamometry
    • Mobility, strength, and function using a series of physical performance measures
    • Functional disability using an patient- and clinician-reported questionnaire
    For Subjects Enrolling from UX001-CL203:
    • Change in GNEM-FAS Expanded Version total score and mobility, upper extremity and self-care domain scores
    • Change in upper extremity strength in grip, key pinch, shoulder abductors and wrist extensors and in lower extremity muscle strength in the knee extensors as measured by dynamometry
    • Evaluate the effect on 6g/day Ace-ER on health-related quality of life (HRQoL), patient reported outcomes (PRO), and biomarkers of sialyation
    valutare l’effetto a lungo termine del trattamento con Ace-ER 6 g/die in soggetti affetti da GNEM.
    L’efficacia sarà valutata come indicato di seguito:
    Per i soggetti arruolati da UX001-CL202 e UX001-CL301:
    • Forza muscolare misurata mediante dinamometria
    • Mobilità, forza e funzione valutate utilizzando una serie di misure di performance fisica
    • Disabilità funzionale valutata utilizzando un questionario sugli esiti riferiti dal paziente e dal
    medico
    Per i soggetti arruolati da UX001-CL203:
    • Variazione del punteggio totale e dei punteggi nei domini mobilità, arti superiori e cura di sé
    ottenuti sulla Versione espansa della scala GNEM-FAS
    • Variazione nella forza degli arti superiori - in relazione a presa della mano, presa del pollice,
    adduttori delle spalle ed estensori del polso - e nella forza muscolare degli arti inferiori - in
    relazione agli estensori del ginocchio - misurate mediante dinamometria
    • Valutare l’effetto di Ace-ER 6 g/die sulla qualità della vita correlata alla salu
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Have completed the UX001-CL301 or UX001-CL203 study
    2. Willing and able to provide written, signed informed consent after the nature of the study has been explained, and before any research-related procedures are conducted
    3. Willing to comply with all study procedures
    4. Female participants of child‐bearing potential or male participants with female partners of child-bearing potential who have not undergone a bilateral salpingo‐oophorectomy and are sexually active must consent to use a highly effective method of contraception as determined by the site investigator (i.e., oral hormonal contraceptives, patch hormonal contraceptives, vaginal ring, intrauterine device, physical double-barrier methods, surgical hysterectomy, vasectomy, tubal ligation or true abstinence [when this is in line with the preferred and usual lifestyle of the subject], which means not having sex because the subject chooses not to), from the period following the signing of the informed consent through 30 days after last dose of study drug
    5. Females of childbearing potential must have a negative pregnancy test at Screening and be willing to have additional pregnancy tests during the study. Females considered not of childbearing potential include those who have been in menopause for at least two years, have had tubal ligation at least one year prior to Screening, or who have had a total hysterectomy or bilateral salpingo-oophorectomy
    1. Aver completato gli studi UX001-CL202, UX001-CL301 o UX001-CL203
    2. Essere disponibili e capaci di fornire per iscritto il consenso informato firmato dopo avere
    ricevuto spiegazioni sulla natura dello studio e prima che sia condotta qualsiasi procedura
    correlata alla ricerca
    3. Essere disponibili a rispettare tutte le procedure dello studio
    4. Le partecipanti in età fertile o i partecipanti con compagne in età fertile non sottoposte a
    salpingo‐ovariectomia bilaterale e che siano sessualmente attivi devono acconsentire a
    utilizzare un metodo contraccettivo altamente efficace determinato dallo sperimentatore del
    centro (ovvero, contraccettivi ormonali orali, cerotto a rilascio di contraccettivi ormonali,
    anello vaginale, dispositivo intrauterino, metodi fisici di doppia barriera, isterectomia
    chirurgica, vasectomia, legatura delle tube o astinenza totale [laddove questa sia in linea con lo
    stile di vita preferito e abituale del soggetto], ossia nessun rapporto sessuale per scelta del
    soggetto) a partire dal periodo successivo alla firma del consenso informato fino a 30 giorni
    dopo l’ultima dose di farmaco dello studio
    5. Le donne in età fertile devono presentare un test di gravidanza negativo allo Screening ed
    essere disposte a sottoporsi a test di gravidanza supplementari nel corso dello studio. Le donne
    considerate non in età fertile includono le donne che sono in menopausa da almeno due anni,
    che sono state sottoposte a legatura delle tube almeno un anno prima dello Screening o che
    hanno subito un’isterectomia totale o una salpingo-ovariectomia bilaterale
    E.4Principal exclusion criteria
    1. Ingestion of N-acetyl-D-mannosamine (ManNAc) or related metabolites; intravenous immunoglobulin (IVIG); or anything that can be metabolized to produce SA in the body within 60 days prior to the Screening Visit
    2. Has had any hypersensitivity to SA or its excipients that, in the judgment of the investigator, places the subject at increased risk for adverse effects
    3. Pregnant or breastfeeding at Screening or planning to become pregnant (self or partner) at any time during the study
    4. Use of any investigational product (except for Ace-ER/SA-ER as part of the parent study) or investigational medical device within 30 days prior to Screening, or anticipated requirement for any investigational agent prior to completion of all scheduled study assessments
    5. Has a condition of such severity and acuity, in the opinion of the investigator, that it warrants immediate surgical intervention or other treatment or may not allow safe participation in the study
    6. Has a concurrent disease, active suicidal ideation, or other condition that, in the view of the investigator, places the subject at high risk of poor treatment compliance or of not completing the study, or would interfere with study participation or would affect safety
    1. Assunzione di N-acetil-D-mannosammina (ManNAc) o metaboliti correlati; trattamento con
    immunoglobulina per via endovenosa (IVIG); o assunzione di qualsiasi sostanza che possa
    essere metabolizzata per produrre SA nell’organismo nei 60 giorni precedenti la Visita di Screening
    2. Qualsiasi ipersensibilità precedente al SA o ai suoi eccipienti che, a giudizio dello
    sperimentatore, esponga il soggetto a un aumento del rischio di effetti avversi
    3. Gravidanza o allattamento allo Screening o gravidanza (propria o della compagna) in
    programma in qualsiasi momento nel corso dello studio
    4. Utilizzo di qualsiasi prodotto sperimentale (eccetto Ace-ER/SA-ER nell’ambito dello studio
    originale) o dispositivo medico sperimentale nei 30 giorni precedenti lo Screening o necessità
    prevista di qualsiasi agente sperimentale prima del completamento di tutte le valutazioni dello
    studio in programma
    5. Una condizione di gravità e acutezza tali che, a giudizio dello sperimentatore, richieda un
    immediato intervento chirurgico o altri trattamenti o che non possa consentire la partecipazione
    sicura allo studio
    6. Malattia concomitante, ideazione suicidaria attiva o altra condizione che, a giudizio dello
    sperimentatore, esponga il soggetto a un rischio elevato di scarsa aderenza al trattamento o di
    mancato completamento dello studio o che interferirebbe con la partecipazione allo studio o
    influirebbe sulla sicurezza.
    E.5 End points
    E.5.1Primary end point(s)
    Overall Safety Endpoint (Primary Endpoint): Evaluate the long-term safety of 6 g/day Ace-ER treatment in subjects with GNE Myopathy
    E.5.1.1Timepoint(s) of evaluation of this end point
    Evaluation for all subjects at 2, 4, 6, 12, 18, and 24 Months. A safety follow-up at visit to be conducted by phone 30 days (+/- 5 days) after last dose.
    E.5.2Secondary end point(s)
    verall Efficacy Endpoint: Evaluate the long-term effect of 6 g/day of Ace-ER treatment in subjects with GNEM. Efficacy will be evaluated as follows: For Subjects Enrolling from UX001-CL301: • Muscle strength as measured by dynamometry • Mobility, strength, and function using a series of physical performance measures • Functional disability using an patient- and clinician-reported questionnaire For Subjects Enrolling from UX001-CL203: • Change in GNEM-FAS Expanded Version total score and mobility, upper extremity and self-care domain scores • Change in upper extremity strength in grip, key pinch, shoulder abductors and wrist extensors and in lower extremity muscle strength in the knee extensors as measured by dynamometry • Evaluate the effect on 6g/day Ace-ER on health-related quality of life, patient reported outcomes (PRO), and biomarkers of sialyation.
    Endpoint di efficacia globale: valutare l’effetto a lungo termine del trattamento con Ace-ER 6 g/die in soggetti affetti da GNEM. L’efficacia sarà valutata come indicato di seguito: Per i soggetti arruolati da UX001-CL202 e UX001-CL301: • Forza muscolare misurata mediante dinamometria • Mobilità, forza e funzione valutate utilizzando una serie di misure di performance fisica • Disabilità funzionale valutata utilizzando un questionario sugli esiti riferiti dal paziente e dal medico Per i soggetti arruolati da UX001-CL203: • Variazione del punteggio totale e dei punteggi nei domini mobilità, arti superiori e cura di sé ottenuti sulla Versione espansa della scala GNEM-FAS • Variazione nella forza degli arti superiori - in relazione a presa della mano, presa del pollice, adduttori delle spalle ed estensori del polso - e nella forza muscolare degli arti inferiori - in relazione agli estensori del ginocchio - misurate mediante dinamometria • Valutare l’effetto di Ace-ER 6 g/die sulla qualità della vita correlata alla salute (health-related quality of life, HRQoL), sugli esiti riferiti dal paziente (patient reported outcomes, PRO) e sui biomarcatori di sialilazione.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Subjects Rolling Over from CL301 timepoints are 2, 4, 6, 12, 18, and 24 Months Subjects Rolling Over from CL203 timepoints are 6, 12, 18, and 24 Months
    Per i soggetti arruolati dal CL301 le visite saranno a 2, 4, 6, 12, 18, e 24 mesi. Per i soggetti arruolati dal CL203 le visite sarannoa 6, 12, 18, e 24 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) No
    E.7.3Therapeutic confirmatory (Phase III) Yes
    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 Yes
    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) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    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 concerned3
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA7
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA Information not present in EudraCT
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Bulgaria
    Canada
    France
    Israel
    Italy
    United Kingdom
    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 years2
    E.8.9.1In the Member State concerned months0
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1Number of subjects for this age range: 1
    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: 110
    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 state20
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 50
    F.4.2.2In the whole clinical trial 110
    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)
    The total treatment duration will be 24 months or until commercial availability of study drug in a subject’s region. A Safety Follow-up visit will be conducted by phone 30 days (±5 days) after last dose of study drug.
    La durata totale del trattamento sarà di 24 mesi. Trenta giorni (+ 5 giorni) dopo l’ultima dose di
    farmaco dello studio verrà condotta per telefono una Visita di Follow-up di Sicurezza.
    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 Decision2017-02-06
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2016-12-15
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
    P.Date of the global end of the trial2018-01-10
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