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    Summary
    EudraCT Number:2014-005432-33
    Sponsor's Protocol Code Number:UX001-CL301
    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:2021-01-19
    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 number2014-005432-33
    A.3Full title of the trial
    A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Sialic Acid Extended-Release Tablets in Patients with GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)
    A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Sialic Acid Extended-Release Tablets in Patients with GNE Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)

    Studio di fase 3, randomizzato, in doppio cieco, controllato verso placebo, per valutare l¿efficacia e la sicurezza di compresse a rilascio prolungato di acido sialico in pazienti affetti da miopatia da mutazione del gene GNE (glucosamina (UDP-N-acetil)-2-epimerasi/N-acetilmannosamina chinasi) (GNE Miopathy, GNEM) o miopatia ereditaria a corpi inclusi (Hereditary Inclusion Body Myopathy, HIBM)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    not available
    non disponibile
    A.3.2Name or abbreviated title of the trial where available
    not applicable
    non applicabile
    A.4.1Sponsor's protocol code numberUX001-CL301
    A.5.4Other Identifiers
    Name:non disponibileNumber:non applicabile
    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 Address60 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-mailUX001ClinOps@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 nameAcido Sialico - Sialic acid (INN: aceneuramic acid)
    D.3.2Product code UX001
    D.3.4Pharmaceutical form Prolonged-release tablet
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboProlonged-release tablet
    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
    GNE Myopathy, also known as Hereditary Inclusion Body Myopathy (HIBM), Distal Myopathy with Rimmed Vacuoles (DMRV), Nonaka's disease, or quadriceps sparing myopathy (QSM)
    miopatia da mutazione del gene GNE, miopatia ereditaria a corpi inclusi (HIBM), miopatia distale con vacuoli cerchiati (Distal Myopathy With Rimmed Vacuoles, DMRV), malattia di Nonaka o miopatia che risparmia i quadricipiti (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 LLT
    E.1.2Classification code 10075048
    E.1.2Term Hereditary inclusion body myopathy
    E.1.2System Organ Class 100000004850
    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 effect of 6 g/day SA-ER treatment of subjects with GNEM on
    upper extremity muscle strength (UEC score) as measured by
    dynamometry
    Valutare l¿effetto del trattamento con 6 g/die di SA-ER di soggetti affetti da GNEM sulla forza muscolare degli arti superiori (punteggio UEC) misurato mediante dinamometria.
    E.2.2Secondary objectives of the trial
    Key Secondary Objectives: Evaluate the effect of 6 g/day of SA-ER treatment of subjects
    with GNEM on:
    ¿ Lower extremity composite muscle strength score (LEC score) as measured by
    dynamometry
    ¿ Muscle strength in the knee extensors as measured by dynamometry
    ¿ Physical functioning as measured using the GNEM-FAS mobility domain score
    Other Secondary Objectives: Evaluate the effect of 6 g/day of SA-ER treatment of subjects with
    GNEM on:
    ¿ Physical functioning as measured using the GNEM-FAS upper extremity domain score
    ¿ Lower extremity function as measured by a timed sit-to-stand test
    ¿ Upper extremity function as measured by a timed weighted arm lift test
    ¿ Lower extremity function as measured by distance walked in the 6MWT
    Obiettivi Secondari Chiave: valutazione degli effetti di 6g/die di SA-ER in soggetti con GNEM su:
    ¿ Punteggio composito della forza muscolare degli arti inferiori (punteggio LEC) misurato con dinamometro
    ¿ Forza muscolare negli estensori del ginocchio misurato tramite dinamometro
    ¿ Funzionalit¿ fisica misurata tramite punteggio del dominio della mobilit¿ della scala GNEM-FAS
    Altri Obiettivi secondari: valutare l¿effetto di SA-ER g/die in soggetti con GNEM su:
    ¿ Funzionalit¿ fisica misurata tramite punteggio del dominio di mobilit¿ della scala GNEM-FAS degli arti superiori
    ¿ Funzionalit¿ degli arti inferiori misurata tramite test sit-to stand
    ¿ Funzionalit¿ degli arti superiori misurata tramite test del sollevamento del braccio on carico
    ¿ Funzionalit¿ degli arti inferiori misurata tramite test della distanza dei 6 minuti (6MWT)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1) Male or female, aged 18 - 50 years, inclusive
    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) Have a documented diagnosis of GNEM, HIBM, DMRV, or Nonaka
    disease due to previously demonstrated mutations in the gene encoding
    the GNE/MNK enzyme (genotyping will not be conducted in this study)
    4) Able to provide reproducible force in elbow flexors (i.e. two
    dynamometry force values with no more than 15% variability in the
    dominant arm) at Screening
    5) Able to walk a minimum of 200 meters during the 6MWT at Screening
    without the use of assistive devices, including a cane, crutch(es),
    walker, wheelchair or scooter (AFOs are permitted)
    6) Willing and able to comply with all study procedures
    7) Participants of child-bearing potential or with partners of childbearing
    potential who have not undergone a bilateral salpingooophorectomy
    and are sexually active must consent to use an 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) from the period following the signing of the
    informed consent through 3 months after last dose of study drug
    8) 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 salpingooophorectomy
    1. Soggetti di sesso maschile e femminile, età 18 ¿ 50 anni (compresi)
    2. Disponibilità e capacità a fornire per iscritto il consenso informato firmato dopo avere ricevuto spiegazioni sulla natura dello studio e prima che siano espletate le procedure riguardanti lo studio
    3. Avere una diagnosi documentata di GNEM, HIBM, miopatia distale con vacuoli cerchiati (Distal Myopathy With Rimmed Vacuoles, DMRV) o malattia di Nonaka dovuta a mutazioni precedentemente dimostrate nel gene di codifica dell’enzima GNE/MNK (ManNAc chinasi) (la genotipizzazione non sarà condotta in questo studio)
    4. Capacità di applicare una forza riproducibile nei flessori del gomito (ossia, due valori di forza dinamometrica con variabilità non superiore al 15% nel braccio dominante) allo Screening
    5. Capacità di camminare almeno 200 metri durante il test 6MWT allo Screening senza l’ausilio di dispositivi di assistenza, tra cui bastone, stampella/e, deambulatore, sedia a rotelle o scooter (le ortesi di piede e caviglia [Ankle-Foot Orthosis, AFO] sono consentite)
    6. Disponibilità e capacità di rispettare tutte le procedure dello studio
    7. I partecipanti in età fertile o con partner in età fertile che non abbiano subito una salpingo-ovariectomia e che siano sessualmente attivi devono acconsentire ad utilizzare un metodo contraccettivo altamente efficace come determinato dallo sperimentatore del centro (ossia, contraccettivi ormonali orali, cerotto a rilascio transdermico di contraccettivi ormonali, anello vaginale, dispositivo intrauterino, metodi di doppia barriera fisica, isterectomia chirurgica, vasectomia, legatura delle tube o astinenza totale[quando questo è
    in linea con lo stile di vita preferito e abituale del soggetto], il che significa non fare sesso perché
    il soggetto sceglie di non farlo) a partire dal periodo successivo alla firma del consenso informato fino a 3 mesi dopo l’ultima dose di farmaco in studio
    8. I soggetti di sesso femminile in età fertile devono presentare un test di gravidanza negativo allo Screening ed essere disposti a sottoporsi a test di gravidanza supplementari nel corso dello studio. I soggetti di sesso femminile considerati non in età fertile includono coloro che sono in menopausa da almeno due anni, che sono stati sottoposti a intervento di 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), SA, 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) History of more than 30 days treatment with SA-ER and/or SA-IR in
    prior clinical trials in the past year
    3) 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
    4) Has serum transaminase (i.e. aspartate aminotransferase [AST] or
    gamma-glutamyl transpeptidase [GGT]) levels greater than 3X the upper
    limit of normal (ULN) for age/gender, or serum creatinine of greater
    than 2X ULN at Screening
    5) Pregnant or breastfeeding at Screening or planning to become
    pregnant (self or partner) at any time during the study
    6) Use of any investigational product 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
    7) 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
    8) 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), SA o metaboliti correlati, immunoglobulina per via endovenosa (Intravenous Immunoglobulin, IVIG) o tutto ciò che possa essere metabolizzato per produrre SA nell’organismo nei 60 giorni precedenti la Visita di screening
    2. Anamnesi di trattamento superiore a 30 giorni con SA-ER e/o SA-IR in precedenti sperimentazioni cliniche nel corso dell’ultimo anno
    3. Pregressa ipersensibilità all’SA o ai suoi eccipienti che, a giudizio dello sperimentatore, esponga il soggetto a un rischio maggiore di effetti avversi
    4. Livelli di transaminasi sieriche (ossia, aspartato aminotransferasi [AST] o gamma-glutammiltransferasi [GGT]) 3 volte superiori al limite normale superiore (Upper Limit of Normal, ULN) per età/sesso o creatinina sierica 2 volte superiore l’ULN allo Screening
    5. Soggetti di sesso femminile in gravidanza o allattamento al momento dello Screening o che stanno pianificando una gravidanza (il soggetto stesso o il partner) in qualsiasi momento nel corso dello studio
    6. Utilizzo di qualsiasi prodotto sperimentale o dispositivo medico sperimentale nei 30 giorni precedenti lo Screening o prevista richiesta di qualsiasi farmaco sperimentale prima del completamento di tutte le valutazioni di studio in programma
    7. 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
    8. Una malattia concomitante, idee di suicidio attive 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 ne altererebbe la sicurezza
    E.5 End points
    E.5.1Primary end point(s)
    The primary clinical efficacy analysis will be the change from baseline in UEC score for the
    SA-ER group compared with placebo based on bilateral strength recorded in the following
    muscle groups using a dynamometer: gross grip, shoulder abductors, elbow flexors, and
    elbow extensors. The UEC is derived from the sum of the muscle groups. Each muscle
    group value will represent the average of the right and left total values (measured in kg).
    The comparison of the two treatment groups will be based on the change from baseline in
    mean UEC score between SA-ER and placebo using GEE repeated measures analysis with
    baseline, gender, and geographic region as covariates.
    L’endpoint di efficacia primario è la variazione rispetto al basale del punteggio della forza muscolare composita degli arti superiori (UEC) tra il gruppo trattato con SA-ER verso placebo, basato sulla forza bilaterale misurata con un dinamometro nei seguenti gruppi di muscoli: forza di presa abduttori, delle spalle, dei flessori del gomito e degli estensori del gomito. Il punteggio UEC è derivato dalla somma dei gruppi di muscoli. Il valore di ciascun gruppo di muscoli è dato dalla media tra i valori totali a destra e a sinistra (misurati in kg). Il confronto tra i due gruppi di trattamento è, utilizzando l’equazione generalizzata di stima sulle misure ripetute, con basale, genere e regione geografica come covariate.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Baseline, Week 8, 16, 24, 32, 40, 48
    Basale, Settimane 8, 16, 24, 32, 40, 48
    E.5.2Secondary end point(s)
    Key Secondary Endpoints: the key secondary clinical efficacy analyses will assess the change from baseline for the SA-ER group compared with the placebo group using GEE for the following variables: ¿ LEC score based on a sum of the mean bilateral strength recorded in the following muscle groups: knee flexors, hip flexors, hip extensors, hip abductors and hip adductors
    ¿ Muscle strength in the knee extensors: bilateral total force (measured in kg)
    ¿ GNEM-FAS mobility domain score

    Other Secondary Endpoints: the analysis of other secondary endpoints will assess the change from baseline for the SA-ER group compared to the placebo group using GEE for the following variables:
    ¿ GNEM-FAS upper extremity domain score
    ¿ Sit-to-stand score calculated as the number of times a subject can rise from a sitting to a standing position in a 30-second period
    ¿ Weighted arm lift score calculated as the number of times a subject can raise a 1 kg weight overhead in a 30-second period
    ¿ Walking ability as measured by the 6MWT, which will be reported as distance in meters and percent predicted based on normative data for age and gender.
    Endpoint secondari chiave: gli endpoint secondari chiave dell¿analisi di efficacia clinica valutano le variazioni rispetto al basale tra il gruppi di trattamento con SA-ER e con placebo usando l¿equazione generalizzata di stima GEE sulle seguenti variabili:
    ¿ Punteggio compositio delle estremit¿ inferiori LEC basato sulla somma della forza media bilaterale misurata nei seguenti gruppi di muscoli: flessori del ginocchio, flessori dell¿anca, estensori dell¿anca, adduttori ed abduttori dell¿anca.
    ¿ Forza muscolare degli estensori del ginocchio: forza totale bilaterale (misurata in kg)
    ¿ Punteggio del dominio della mobilit¿ della scala GNEM- FAS

    Altri endpoint secondari: l¿analisi degli altri obiettivi secondari valuta la variazione rispetto al basale dei gruppi di trattamento con SA-ER e placebo, usando l¿equazione generalizzata di stima GEE sulle seguenti variabili:
    ¿ Punteggio del dominio della scala GNEM-Fas degli arti superiori
    ¿ Punteggio del test sit-to-stand, calcolato come numero di volte in cui un soggetto passa da una posizione seduta ad una posizione eretta in 30 secondi
    ¿ Punteggio del test del tempo di sollevamento del braccio sotto carico, calcolato come numero di volte in cui un soggetto pu¿ sollevare 1 kg di peso in 30 secondi
    ¿ Capacit¿ di deambulazione misurata tramite test del cammino dei 6 minuti (6MWT), riportato come distanza in metri e percentuale dei valori predetti per et¿ e genere
    E.5.2.1Timepoint(s) of evaluation of this end point
    Baseline, Week 8, 16, 24, 32, 40, 48
    Basale, Settimane 8, 16, 24, 32, 40, 48
    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 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 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 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 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
    United States
    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
    LVLS
    LVLS
    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 months7
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years1
    E.8.9.2In all countries concerned by the trial months7
    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.1.1.1In 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: 89
    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 state11
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 50
    F.4.2.2In the whole clinical trial 89
    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)
    An open-label study, UX001-CL302, will be available to subjects that complete the 48-week treatment period in this study.
    Uno studio in aperto, UX001-CL302, sar¿ disponibile per pazienti che completino il trattamento di 48 settimane nello studio
    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 Decision2015-12-16
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2015-06-30
    P. End of Trial
    P.End of Trial StatusCompleted
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