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    Summary
    EudraCT Number:2015-005536-17
    Sponsor's Protocol Code Number:UX007G-CL301
    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:2020-12-15
    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 number2015-005536-17
    A.3Full title of the trial
    A Phase 3, randomized, double-blind, placebo-controlled, crossover study to assess the efficacy and safety of UX007 in the treatment of movement disorders associated with Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
    Studio crossover di fase 3, randomizzato, in doppio cieco, controllato con placebo per valutare l¿efficacia e la sicurezza di UX007 nel trattamento dei disturbi del movimento associati alla sindrome da deficit del trasportatore del glucosio tipo 1 (Glut1 DS)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A trial to assess the safety and efficacy of UX007 in patients with movement disorders associated with Glucose Transporter Type 1 Deficiency Syndrome (Glut1 DS)
    Studio per valutare sicurezza ed efficacia di UX007 in pazienti con disturbi del movimento associati alla sindrome da deficit del trasportatore del glucosio tipo 1 (Glut1 DS)
    A.3.2Name or abbreviated title of the trial where available
    A trial to assess the safety and efficacy of UX007 in patients with movement disorders associated w
    Uno studio per valutare la sicurezza e l'efficacia di UX007 in pazienti con disturbi del movimento a
    A.4.1Sponsor's protocol code numberUX007G-CL301
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT02960217
    A.5.4Other Identifiers
    Name:EMA/190573Number:Unique Product Identifier (UPI)
    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 number0014154838813
    B.5.5Fax number0014154838813
    B.5.6E-mailUX007G-CL301@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/3/15/1495
    D.3 Description of the IMP
    D.3.1Product nameUX007
    D.3.2Product code UX007
    D.3.4Pharmaceutical form Oral solution
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPOral use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.1CAS number 620-67-7
    D.3.9.2Current sponsor codeUX007
    D.3.9.3Other descriptive nametriheptanoin
    D.3.9.4EV Substance CodeSUB129584
    D.3.10 Strength
    D.3.10.1Concentration unit g/ml gram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number1
    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 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
    Glucose Transporter Type 1 deficiency syndrome
    Sindrome da deficit del trasportatore del glucosio tipo 1
    E.1.1.1Medical condition in easily understood language
    Glucose transporter type 1 (Glut1) deficiency syndrome is a rare genetic metabolic disorder characterized by deficiency of a protein that is required for glucose to cross the blood-brain barrier
    Sindrome da deficit del trasportatore del glucosioTipo1(Glut1)¿ 1malattia metab genet rara caratterizzata da deficit di 1proteina richiesta per far attraversare al glucosio la membrana ematoencefalica
    E.1.1.2Therapeutic area Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 20.0
    E.1.2Level LLT
    E.1.2Classification code 10061032
    E.1.2Term Carbohydrate transport disorder
    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
    Primary Objectives:
    ¿Evaluate the efficacy and safety of UX007 in the treatment of disabling paroxysmal movement disorders associated with Glut1 DS
    Obiettivo principale: Valutare l¿efficacia e la sicurezza di UX007 nel trattamento dei disordini parossistici invalidanti del movimento associati a Glut1 DS
    E.2.2Secondary objectives of the trial
    Evaluate the efficacy of UX007 compared to placebo, as measured by:
    Duration of disabling paroxysmal movement disorder events observed during the Maintenance Period of treatment, as recorded by the subject/caregiver in an event-based daily electronic Glut1 DS symptom diary
    Walking capacity and endurance, as determined by the distance walked in 12 minutes during the 12 Minute Walk Test (12MWT)
    Patient/caregiver global impression of change in clinical status using the Clinical Global Impression ¿ Improvement (CGI-I)
    Health-related quality of life assessing physical function, mobility, upper extremity function, fatigue, pain and social health using a PROMIS-based questionnaire
    Cognitive function as measured by the Cambridge Neuropsychological Test Automated Battery (CANTAB) (assessed at select sites)
    Valutare l efficacia di UX007 rispetto al placebo, misurata come:
    Durata degli episodi di disturbi parossistici invalidanti del movimento osservati nel periodo di mantenimento del trattamento, registrati dal soggetto/caregiver in un diario elettronico giornaliero dei sintomi della Glut1 DS basato sugli eventi.
    Capacita di camminare e resistenza determinate dalla distanza percorsa in 12 minuti in base al test del cammino in 12 minuti (12MWT).
    Impressione globale del paziente/caregiver dei cambiamenti dello stato clinico mediante la scala di Impressione clinica globale - Miglioramento (CGI-I).
    Qualita della vita correlata alla salute quale criterio di valutazione di funzionalita fisica, mobilita, funzionalita delle estremita superiori, astenia, dolore e salute sociale mediante un questionario basato su PROMIS.
    Funzione cognitiva misurata mediante la batteria automatizzata del test neuropsicologico di Cambridge (CANTAB) (valutata presso centri selezionati)
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1)Diagnosis of Glut1 DS confirmed by SLC2A1 mutation
    2)Males and females, aged =6 years old at the time of informed consent
    3)At least 8 disabling paroxysmal movement disorder events in the 12 weeks prior to the Screening, by subject or caregiver report
    OR
    At least 6 disabling paroxysmal movement disorder events in any 6 consecutive week period, over the last 12 week period prior to the Screening, by subject or caregiver report

    4)At least 4 disabling paroxysmal movement disorder events in 6 week Run-in Period, reported in the daily electronic Glut1 DS symptom diary
    5)=80% compliance with daily electronic Glut1 DS symptom diary completion during the Run-in Period
    6)Not on KD, modified KD, or ketosis-inducing modified-fat diet for at least 3 months prior to Screening
    7)Plasma level of beta-hydroxybutyrate (BHB) = 1 mmol/L (non-fasting) at Screening
    8)Provide written or verbal assent (if possible) and written informed consent by the patient (if an adult), or by a legally authorized representative after the nature of the study has been explained, and prior to any research-related procedures
    9)Must, in the opinion of the Investigator, be willing and able to complete key aspects of the study and be likely to complete the 22-week, placebo-controlled, treatment period
    10)Patient (or caregiver) must, in the opinion of the Investigator, be able to comply with accurate completion of the study daily electronic Glut1 DS symptom diary
    11)Females of child-bearing potential must have a negative urine pregnancy test at Screening and Baseline and be willing to have additional pregnancy tests during the study. Females considered not to be of childbearing potential include those who have not experienced menarche, are post-menopausal (defined as having no menses for at least 12 months without an alternative medical cause) or are permanently sterile due to total hysterectomy, bilateral salpingectomy, or bilateral oophorectomy.
    12)Participants of child-bearing potential or fertile males with partners of child-bearing potential who are sexually active must consent to use a highly effective method of contraception as determined by the site Investigator from the period following the signing of the informed consent through 30 days after last dose of study drug
    1)Diagnosi di Glut1 DS confermata da mutazione del SLC2A1.
    2)Soggetti di sesso maschile e femminile di età =6 anni al momento del consenso informato.
    3)Almeno 8 episodi di disturbi parossistici invalidanti del movimento nelle 12 settimane precedenti lo screening, riferiti dal soggetto o dal caregiver.
    OPPURE
    Almeno 6 episodi di disturbi parossistici invalidanti del movimento in un periodo di 6 settimane consecutive, nelle ultime 12 settimane precedenti lo screening, riferiti dal soggetto o dal caregiver.
    4)Almeno 4 episodi di disturbi parossistici invalidanti del movimento nelle 6 settimane del periodo di run-in, riportati nel diario elettronico giornaliero dei sintomi della Glut1 DS.
    5)=80% di compliance nella compilazione del diario elettronico giornaliero dei sintomi della Glut1 DS nel periodo di run-in.
    6)Non a dieta KD, KD modificata, o dieta a base di grassi modificati per indurre chetosi, per almeno 3 mesi prima dello screening.
    7)Livelli plasmatici di beta-idrossibutirrato (BHB) = 1 mmol/l (a stomaco pieno) allo screening.
    8)Assenso scritto o verbale (se possibile) e consenso informato scritto da parte del paziente (se adulto) o di un rappresentante legalmente autorizzato, dopo aver spiegato la natura dello studio e prima di iniziare qualsiasi procedura correlata alla ricerca.
    9)Il paziente deve, a giudizio dello sperimentatore, essere disposto a e in grado di ottemperare a tutti gli aspetti essenziali dello studio e portare a termine il periodo di trattamento, controllato verso placebo della durata di 22 settimane.
    10)Il paziente (o il caregiver) deve, a giudizio dello sperimentatore, essere in grado di compilare con accuratezza il diario elettronico giornaliero dei sintomi della Glut1 DS.
    11)Le donne in età fertile devono presentare un test di gravidanza sulle urine negativo allo screening e al basale ed essere disposte a sottoporsi ad ulteriori test di gravidanza durante lo studio. Non sono considerate fertili le donne che non hanno avuto il menarca, sono in post-menopausa (non hanno le mestruazioni da almeno 12 mesi senza una causa medica diversa) o sono sterili definitivamente a causa di isterectomia totale, salpingectomia bilaterale o ovariectomia bilaterale.
    12)Le/I partecipanti in età fertile o con partner in età fertile sessualmente attive devono acconsentire ad utilizzare un metodo contraccettivo altamente efficace secondo le indicazioni dello sperimentatore del centro dal periodo successivo alla firma del consenso informato e per 30 giorni dopo l’ultima dose di farmaco dello studio.
    E.4Principal exclusion criteria
    1)Any known hypersensitivity to triheptanoin or safflower oil that, in the judgment of the Investigator, places the subject at increased risk for adverse effects
    2)Prior use of triheptanoin within 30 days prior to Screening
    3)History of, or current suicidal ideation, behavior and/or attempts per C-SSRS at Screening or Baseline
    4)Pregnant and/or breastfeeding an infant at Screening or Baseline
    5)Participants unwilling or unable to discontinue use of a prohibited medication or other substance that may confound study objectives (Section 7.4.6.1 [MCT oil, barbiturates, pancreatic lipase inhibitors, KetoCal or other KD supplements, and/or KD])
    6)Glut1 DS treatment regimen, including AEDs, should be stable for at least 30 days prior to Screening
    7)Use of any investigational product (drug, medical food, or supplement, including medium chain triglyceride [MCT] oil, including coconut oil) within 30 days prior to Screening
    8)Has a concurrent disease or condition, or laboratory abnormality 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 introduces additional safety concerns
    9)Feeding or nutrition that, in the opinion of the dietitian, potentially affects consistent administration of study drug
    1)Qualsiasi reazione di ipersensibilità nota alla trieptanoina o all'olio di cartamo che, a giudizio dello sperimentatore, aumenti il rischio per il soggetto di andare incontro ad effetti avversi.
    2)Uso pregresso di trieptanoina nei 30 giorni che precedono la visita di screening.
    3)Ideazione, comportamento e/o tentativi suicidari, attuali o pregressi, valutati secondo la C-SSRS allo screening o al basale.
    4)Gravidanza e/o allattamento allo screening o al basale.
    5)Partecipante non disposto o impossibilitato a interrompere l'uso di un medicinale o di altra sostanza non consentita che potrebbe confondere gli obiettivi dello studio (Sezione 7.4.6.1 [olio MCT, barbiturici, inibitori della lipasi pancreatica, KetoCal o altri integratori KD, e/o KD]).
    6)Regime di trattamento per la Glut1 DS, compresi i farmaci antiepilettici (AED), stabile per almeno 30 giorni prima dello screening.
    7)Uso di qualsiasi prodotto sperimentale (farmaco, alimenti a fini medici speciali o integratori, compreso l'olio contenente trigliceridi a catena media [MCT], come l'olio di cocco) entro 30 giorni prima dello screening.
    8)Presenza di una malattia o di una condizione concomitante oppure valori anomali di laboratorio che, a giudizio dello sperimentatore, espongono fortemente il soggetto al rischio di aderire al trattamento in modo non soddisfacente o di non completare lo studio, oppure che potrebbero interferire con la partecipazione allo studio o suscitare ulteriori preoccupazioni per la sicurezza.
    9)Alimentazione o nutrizione che, a giudizio del dietologo, possono compromettere la capacità di assumere il farmaco dello studio in modo regolare.
    E.5 End points
    E.5.1Primary end point(s)
    The primary endpoint is the frequency of disabling paroxysmal movement disorders captured as movement disorder events observed during the Maintenance Period of treatment, as recorded by the subject/caregiver in an event-based daily Glut1 DS symptom diary.
    L'endpoint primario consiste nella frequenza di disturbi parossistici invalidanti del movimento definita come episodi di disturbi del movimento osservati nel periodo di mantenimento del trattamento, registrati dal soggetto/caregiver in un diario giornaliero dei sintomi della Glut1 DS basato sugli eventi.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Daily Glut1 Symptom Diary review: Weeks 0,2,4,6,10,12,14,16,18,22,26,30,34,46,58,70,82,94,106,118,130,142,154,178
    Revisione del Diario Giornaliero dei sintomi da Glut1: Settimane 0,2,4,6,10,12,14,16,18,22,26,30,34,46,58,70,82,94,106,118,130,142,154,178
    E.5.2Secondary end point(s)
    Duration of disabling paroxysmal movement disorder events observed during the Maintenance Period of treatment, as recorded by the subject/caregiver in an event-based daily electronic Glut1 DS symptom diary
    Walking capacity and endurance, as determined by the distance walked in 12 minutes during the 12 Minute Walk Test (12MWT)
    Patient/caregiver global impression of change in clinical status using the Clinical Global Impression Improvement (CGI-I)
    Health-related quality of life assessing physical function, mobility, upper extremity function, fatigue, pain and social health using a PROMIS-based questionnaire
    Cognitive function as measured by the Cambridge Neuropsychological Test Automated Battery (CANTAB) (assessed at select sites)
    Durata degli eventi degli episodi di disturbi parossistici invalidanti del movimento osservati nel periodo di mantenimento del trattamento, registrati dal soggetto/caregiver in un diario giornaliero dei sintomi della Glut1 DS basato sugli eventi.
    Capacita di camminare e resistenza determinate dalla distanza percorsa in 12 minuti in base al test del cammino in 12 minuti (12MWT).
    Impressione globale del paziente/caregiver dei cambiamenti dello stato clinico mediante la scala di Impressione clinica globale - Miglioramento (CGI-I).
    Qualita della vita correlata alla salute quale criterio di valutazione di funzionalita fisica, mobilita, funzionalita delle estremita superiori, astenia, dolore e salute sociale mediante un questionario basato su PROMIS.
    Funzione cognitiva misurata mediante la batteria automatizzata del test neuropsicologico di Cambridge (CANTAB) (valutata presso centri selezionati)
    E.5.2.1Timepoint(s) of evaluation of this end point
    12MWT: Weeks 0,10,12,22,34,58,82,106,130,154
    PROMIS: Weeks 0,10,12,22,26,30,34,46,58,70,82,94,106,118,142,154,178
    Patient/Caregiver CGI-I: Weeks 10,12,22,34,58,82,106,130,154
    Duration of disabling paroxysmal movement disorders:Weeks 0,2,4,6,10,12,14,16,18,22,26,30,34,46,58,70,82,94,106,118,130,142,154,178
    CANTAB: Weeks 0,10,12,22,34,58,82,106,130,154
    12MWT: settimane 0,10,12,22,34,,58,82,106,130,154
    PROMIS: Settimane 0,10,12,22,26,30,34,46,58,70,82,94,106,118,142,154,178
    CGI-I del paziente/caregiver: Settimane 10,12,22,34,58,82,106,130,154
    Durata degli episodi di disturbi parossistici invalidanti del movimento: Settimane 0,2,4,6,10,12,14,16,18,22,26,30,34,46,58,70,82,94,106,118,130,142,154,178
    CANTAB: settimane 0,10,12,22,34,58,82,106,130,154
    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 Yes
    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 No
    E.8.1.6Cross over Yes
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    Periodo di Estensione in aperto dopo il periodo iniziale di 22 settimane in doppio cieco
    open-label Extension Period after the initial 22-week double-blind period
    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 Yes
    E.8.5.1Number of sites anticipated in the EEA11
    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
    Australia
    France
    Germany
    Israel
    Italy
    Spain
    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
    Last patient last visit
    Ultima Visita Ultimo paziente
    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 months6
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months6
    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 Yes
    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: 6
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 25
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 4
    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 Yes
    F.3.3.6.1Details of subjects incapable of giving consent
    Paediatric subjects
    soggetti pediatrici
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state5
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 20
    F.4.2.2In the whole clinical trial 40
    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)
    After the initial 22-week double-blind Treatment Period, the open-label Extension Period will begin, wherein all subjects will be treated with UX007 through Week 178 of the study.
    Dopo l¿iniziale periodo di trattamento di 22 settimane in doppio cieco, inizier¿ il periodo di estensione in aperto, in cui tutti i soggetti verranno trattati con UX007 fino alla settimana 178 dello 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 Decision2017-05-09
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2017-05-08
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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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
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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