Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register allows you to search for protocol and results information on:
  • interventional clinical trials that are conducted in the European Union (EU) and the European Economic Area (EEA);
  • clinical trials conducted outside the EU / EEA that are linked to European paediatric-medicine development.
  • Learn   more about the EU Clinical Trials Register   including the source of the information and the legal basis.


    The EU Clinical Trials Register currently displays   42891   clinical trials with a EudraCT protocol, of which   7066   are clinical trials conducted with subjects less than 18 years old.
    The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).


    Phase 1 trials conducted solely in adults and that are not part of an agreed PIP are not public in the EU CTR (refer to European Guidance 2008/C 168/02   Art. 3 par. 2 and   Commission Guideline 2012/C 302/03,   Art. 5) .

    Clinical Trials marked as "Trial now transitioned" were transitioned to the Clinical Trial Regulation 536/2014 and can be further followed in the Clinical Trial Information System  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Print Download

    Summary
    EudraCT Number:2021-000903-19
    Sponsor's Protocol Code Number:UX053-CL101
    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:2021-05-24
    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 number2021-000903-19
    A.3Full title of the trial
    A Phase 1/2 First-in-human, 2-part Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Single Ascending Doses (Part 1: Open-label) and Repeat Doses (Part 2: Randomized, Double-blind, Placebo-controlled) of UX053 in Patients with GSD III
    Studio di fase 1/2 condotto per la prima volta negli esseri umani, in 2 parti, per valutare la sicurezza, la tollerabilità e la farmacocinetica delle singole dose ascendenti (Parte 1: in aperto) e delle dosi ripetute (Parte 2: randomizzata, in doppio cieco, controllata con placebo) di UX053 in pazienti affetti da GSD III
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A first-in-human study of UX053 in Patients with Glycogen Storage Disease type III (GSD III)
    Primo studio sull'uomo di UX053 in pazienti con malattia da accumulo di glicogeno di tipo III (GSD III)
    A.3.2Name or abbreviated title of the trial where available
    UX053-CL101
    UX053-CL101
    A.4.1Sponsor's protocol code numberUX053-CL101
    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 pointUX053-CL101 Clinical Operations
    B.5.3 Address:
    B.5.3.1Street Address60 Leveroni Court
    B.5.3.2Town/ cityNovato
    B.5.3.3Post codeCA 94949
    B.5.3.4CountryUnited States
    B.5.6E-mailUX053-CL101@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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product namemRNA che codifica l’enzima umano deramificante del glicogeno formulato in una nanopaticella lipidica
    D.3.2Product code [UX053]
    D.3.4Pharmaceutical form Solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.2Current sponsor codeUX053 mRNA
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(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 No
    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) Yes
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product Yes
    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 Yes
    D.3.11.3.5.1CAT classification and reference numberEMA/CAT/615211/2020 Gene therapy medicinal product
    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.IMP: 2
    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 Yes
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product namedexamethasone (or equivalent)
    D.3.2Product code [-]
    D.3.4Pharmaceutical form Solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.9.2Current sponsor code-
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number10
    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 placeboSolution for infusion
    D.8.4Route of administration of the placeboIntravenous use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Glycogen Storage Disease Type III (GSD III)
    malattia da accumulo di glicogeno di tipo III (GSD III)
    E.1.1.1Medical condition in easily understood language
    A disease where the body cannot break down glycogen (stored form of glucose). It affects the liver, heart muscle, and skeletal muscle. It is also known as Cori disease or Forbes disease.
    Malattia in cui il corpo non è in grado di metabolizzare il glicogeno (forma depositata del glucosio). Interessa fegato, muscolo cardiaco e muscolo scheletrico. Nota come malattia di Cori o di Forbes.
    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 20.1
    E.1.2Level PT
    E.1.2Classification code 10053250
    E.1.2Term Glycogen storage disease type III
    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 safety of UX053 in adults with GSD III
    Valutare la sicurezza di UX053 in adulti con GSD III
    E.2.2Secondary objectives of the trial
    Characterize the PK of UX053 in adults with GSD III
    Caratterizzare la PK di UX053 in adulti con GSD III
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Confirmed diagnosis of Glycogen Storage Disease type III (GSD III) (all subtypes) based on pathogenic mutations in the amylo-a-1,6-glucosidase 4-alpha-glucanotransferase (AGL) gene on both alleles or glycogen debranching enzyme (GDE) deficiency based on biopsy of liver, muscle, or fibroblasts
    2. History of any of the following:
    a. Severe hypoglycemia, defined as neuroglycopenia (eg, altered mental status, seizure, dizziness, slurred speech, blurry vision, abnormal behavior, perioral paresthesia, requiring intervention by a caregiver) or blood glucose < 54 mg/dL (3 mmol/L) within the last year
    b. = 2 incidents of symptomatic hypoglycemia (defined as blood glucose < 70 mg/dL [3.9 mmol/L] if measured at the time of symptoms) within the last year, despite nutrition management
    c. Ongoing liver injury, defined as alanine aminotransferase (ALT) > 2.5x the upper limit of normal (ULN) within the last year
    3. ALT = 5x ULN during the 3 months prior to the Baseline Visit
    4. Males or females = 18 years of age
    5. After nutrition optimization (if necessary) for subjects in the repeat dose (RD) Period, during the beginning of the Screening Period, willing and able to maintain nutritional intake consistent with the nutrition guidelines based on expert recommendations for the remainder of the study
    6. Willing and able to provide access to medical records surrounding medical treatment that occurred prior to enrollment
    7. Willing and able to provide written informed consent, or in the case of adult subjects with cognitive limitation, provide written assent (if required) and written informed consent by a legally authorized representative after the nature of the study has been explained and prior to any test procedures or assessments
    8. Females of childbearing potential must have a negative pregnancy test at Screening (ISV) and be willing to have additional pregnancy tests during the study. Subjects of childbearing potential or fertile males who are sexually active with partners of child-bearing potential must consent to use a highly effective contraceptive method, as described in Appendix 4 of the Protocol, from the Period following the signing of the informed consent through 30 days after last dose of study drug
    1. diagnosi confermata di GSD III (tutti i sottotipi) sulla base di mutazioni patogeniche del gene amilo-a-1,6-glucosidasi 4-alfa-glucanotransferasi (AGL) su entrambi gli alleli o deficit dell'enzima deramificante il glicogeno (GDE) sulla base di biopsia del fegato, del muscolo o dei fibroblasti;
    2. Anamnesi di uno qualsiasi dei seguenti eventi:
    a. Grave ipoglicemia, definita come neuroglicopenia (es. stato mentale alterato, crisi convulsive, capogiri, disartria, visione offuscata, comportamento anormale, parestesia periorale, con necessità di intervento di un caregiver) o glucosio nel sangue <54 mg/dl (3 mmol/l) nell’ultimo anno;
    b. =2 eventi di ipoglicemia sintomatica (definita come glucosio nel sangue <70 mg/dl [3,9 mmol/l] se misurato al momento dell’insorgenza dei sintomi) nell’ultimo anno, nonostante la gestione del regime nutrizionale;
    c. lesione epatica in corso, definita come alanina aminotransferasi (ALT) >2,5 volte il limite superiore della norma (ULN) nell’ultimo anno;
    3. ALT =5 volte ULN nei 3 mesi precedenti la Visita basale;
    4. pazienti di sesso maschile o femminile di età =18 anni;
    5. a seguito dell’ottimizzazione del regime nutrizionale (se necessaria) per i soggetti nel periodo RD, durante l’inizio del periodo di screening (Sezione 7.1), essere disposti e in grado di seguire un apporto nutrizionale secondo le linee guida nutrizionali basate sulle raccomandazioni di un esperto (Tabella 2) per la parte rimanente dello studio;
    6. essere disposti e in grado di fornire accesso alle cartelle cliniche relative al trattamento medico ricevuto prima dell’arruolamento;
    7. essere disposti e in grado di fornire consenso informato scritto oppure, in caso di soggetti adulti con limitazione cognitiva, fornire assenso scritto (se richiesto) e consenso informato scritto da parte di un rappresentante legale in seguito alla spiegazione della natura dello studio e prima di qualsiasi procedura o valutazione;
    8. le donne in età fertile devono avere un test di gravidanza negativo allo Screening (VSI) ed essere disposte a sottoporsi ad altri test di gravidanza durante lo studio. I soggetti di sesso femminile in età fertile o di sesso maschile in grado di procreare che sono sessualmente attivi con partner in età fertile devono acconsentire all’utilizzo di un metodo contraccettivo altamente efficace, come descritto nell’Allegato 4, dal periodo successivo alla firma del consenso informato fino a 30 giorni dopo l’ultima dose del farmaco in studio.
    E.4Principal exclusion criteria
    1. History of liver transplant, including hepatocyte cell therapy/transplant, or active listing for liver transplant
    2. History of cirrhosis, or presence of any of the following:
    a. Total bilirubin = 1.3 mg/dL and international normalized ratio (INR) = 1.3
    b. Evidence of portal hypertension, including, but not limited to the following symptoms splenomegaly, ascites, thrombocytopenia, esophageal varices, or history of hepatic encephalopathy
    c. Model for End Stage Liver Disease (MELD) score > 12
    3. Current Hepatitis B or C infection or history of chronic Hepatitis B or C infection
    4. Severe renal impairment defined as a glomerular filtration rate (GFR) = 29 mL/min
    5. Any prior history of hepatocellular carcinoma or presence of liver adenoma > 5 cm at the longest diameter or > 3 cm and = 5 cm in size that has an annual growth rate of = 0.5 cm per year
    6. Current or history of malignancies in the 3 years prior to the Screening Visit (ISV for RD)
    7. Hospitalizations related to GSD III disease between the Screening (ISV for RD) and Baseline Visit
    8. Known history of human immunodeficiency virus infection
    9. Presence or history of any hypersensitivity reactions requiring medical evaluation and management (including injection/infusion associated reactions, such as lymphadenopathy) to UX053, its excipients, or any drug products that contain polysorbate or PEG. This may include mRNA-based vaccines that contain PEG or polysorbate
    10. Significant cardiac disease, including heart failure with New York Heart Association (NYHA) Function Capacity III or IV or Objective Assessment C or D, unstable angina, or ejection fraction (EF) < 35%, or uncontrolled arrhythmia or resistant hypertension. Asymptomatic cardiomyopathy and left ventricular hypertrophy (LVH) are allowed
    11. Presence or history of any co-morbid condition or abnormal labs that, in the view of the Investigator, places the subject’s safety at risk; places the subject at high risk of poor treatment compliance or not completing the study; or would significantly affect the interpretation of study results
    12. Poorly controlled diabetes, defined as the presence of any of the following:
    a. Hemoglobin A1C > 8%
    b. History of diabetic nephropathy, neuropathy, or retinopathy
    c. History of diabetic ketoacidosis during the past year
    13. Poorly controlled hypothyroidism, based on the judgement of the Investigator or Ultragenyx, whichever is most conservative
    14. History of chronic coagulopathy, thrombophilia, or disorder of complement activation
    15. Use of concomitant medications that alter PT/INR, including warfarin and direct oral anticoagulants (eg, rivaroxaban, apixaban, and edoxaban). Patients who receive medications that affect platelet function, such as aspirin or clopidogrel, are allowed, unless they have comorbidities that in the judgment of the Investigator place them at undue risk to participate in the study.
    16. Current treatment with long-term immunosuppressive medications. This includes subjects with autoimmune conditions managed with immunosuppressive medications and solid organ transplant recipients.
    17. Active tuberculosis requiring treatment in the past 3 years
    18. Symptomatic COVID-19 infection
    19. History of active alcohol and/or drug abuse that in the Investigator’s assessment would impair the subject’s ability to comply with the protocol

    Insufficient space, please refer to the Protocol for other criteria.
    1. anamnesi di trapianti di fegato, inclusi trapianto/terapia cellulare con epatociti o essere in lista di attesa con stato “attivo” per trapianto di fegato;
    2. anamnesi di cirrosi o presenza di una qualsiasi delle seguenti condizioni:
    a. bilirubina totale =1,3 mg/dl e rapporto normalizzato internazionale (INR) =1,3;
    b. evidenza di ipertensione portale, inclusi a titolo esemplificativo i seguenti sintomi: splenomegalia, ascite, trombocitopenia, varici esofagee o anamnesi di encefalopatia epatica;
    c. punteggio Model for End Stage Liver Disease (MELD) >12;
    3. infezione da epatite B o C in corso o anamnesi di infezione da epatite B o C cronica;
    4. grave compromissione renale definita come velocità di filtrazione glomerulare (GFR) =29 ml/min (Levey et al., 2005);
    5. qualsiasi precedente anamnesi di carcinoma epatocellulare o presenza di adenoma epatico >5 cm a livello del diametro massimo oppure di dimensione >3 cm e =5 cm con un tasso di crescita annuale di = 0,5 cm all’anno;
    6. presenza o anamnesi di neoplasie nei 3 anni precedenti la Visita di screening (VSI per RD);
    7. ricoveri ospedalieri correlati alla malattia GSD III tra la Visita di screening (VSI per RD) e la Visita basale;
    8. anamnesi nota di infezione da virus dell’immunodeficienza umana;
    9. presenza o anamnesi di qualsiasi reazione di ipersensibilità che richiede valutazione e gestione medica (incluse reazioni associate all’infusione/iniezione, come linfadenopatia) a UX053, ai suoi eccipienti o a qualsiasi prodotto farmaceutico che contiene polisorbato o PEG. Ciò potrebbe includere i vaccini a base di mRNA che contengono PEG o polisorbato;
    10. cardiopatia significativa, inclusa insufficienza cardiaca con capacità funzionale III o IV o valutazione obiettiva C o D secondo la New York Heart Association (NYHA), angina instabile o frazione di eiezione (EF) <35% oppure aritmia non controllata o ipertensione resistente (Carey et al., 2018). Cardiomiopatia asintomatica e ipertrofia ventricolare sinistra sono consentite;
    11. presenza o anamnesi di condizione concomitante o anomalia di laboratorio che, a parere dello sperimentatore, pone la sicurezza del soggetto a rischio, pone il soggetto ad alto rischio di scarsa aderenza al trattamento o di mancato completamento dello studio o che interferirebbe in modo significativo con l’interpretazione dei risultati dello studio;
    12. diabete scarsamente controllato, definito dalla presenza di uno qualsiasi dei seguenti valori:
    a. emoglobina A1C >8% (Qaseem et al., 2018);
    b. anamnesi di nefropatia, neuropatia o retinopatia diabetica;
    b. anamnesi di chetoacidosi diabetica durante l’anno precedente;
    13. ipotiroidismo scarsamente controllato, in base al giudizio dello Sperimentatore o di Ultragenyx, a seconda di quale sia il più prudente;
    14. anamnesi di coagulopatia cronica, trombofilia o disturbo dell’attivazione del complemento;
    15. utilizzo di farmaci concomitanti che alterano PT/INR, inclusi warfarin e anticoagulanti orali diretti (es. rivaroxaban, apixaban ed edoxaban). I pazienti che ricevono farmaci che influiscono sulla funzione piastrinica, come aspirina o clopidogrel, possono partecipare a meno che non siano affetti da comorbidità che, secondo il giudizio dello Sperimentatore, pongano un rischio non necessario sul paziente in caso di partecipazione allo studio;
    16. trattamento in corso con farmaci immunosoppressivi a lungo termine. Ciò include soggetti con condizioni autoimmuni gestite con farmaci immunosoppressivi e i riceventi di trapianto di organi solidi;

    Spazio insuffiente, fare riferimento al Protocollo.
    E.5 End points
    E.5.1Primary end point(s)
    The incidence and severity of TEAEs, serious TEAE and related TEAEs in the SAD and RD Periods
    L’incidenza e la gravità dei TEAE, TEAE seri e TEAE correlati nei periodi SAD e RD.
    E.5.1.1Timepoint(s) of evaluation of this end point
    SAD Period: Adverse Events monitored from Screening through to D90. In the event of an ET, all efforts will be made to monitor the subject through the end of the study. At minimum, a safety follow-up phone call will occur within the 4 weeks following the subject’s last treatment.

    RD Period: Adverse Events monitored from Screening through to W48. In the event of an ET, all efforts will be made to monitor the subject through the end of the study. At minimum, a safety follow-up phone call will occur within the 4 weeks following the subject’s last treatment.
    Periodo SAD: Eventi avversi monitorati dallo Screening fino al G90. In caso di ET, devono essere compiuti tutti gli sforzi per monitorare il soggetto fino alla fine dello studio. Sarà eseguita almeno una telefonata di follow-up di sicurezza entro le 4 settimane successive all’ultimo trattamento del soggetto.
    Periodo RD: Eventi avversi monitorati dallo Screening fino alla S48. In caso di ET, devono essere compiuti tutti gli sforzi per monitorare il soggetto fino alla fine dello studio. Sarà eseguita almeno una telefonata di follow-up di sicurezza entro le 4 settimane successive all’ultimo trattamento del soggetto.
    E.5.2Secondary end point(s)
    PK parameters of AGL (amylo-a-1,6-glucosidase 4-alpha-glucanotransferase) mRNA and ATX95, including Tmax, Cmax, AUClast, AUCinf, AUCtau (RD Period only), RAUC (RD Period only), Tlast, T1/2, CL, Vss (SAD Period only)
    Parametri PK per mRNA AGL (amilo-a-1,6-glucosidasi 4-alfa- glucanotransferasi) e ATX95, compresi Tmax, Cmax, AUClast, AUCinf, AUCtau (solo Periodo RD), RAUC (solo Periodo RD), Tlast, T1/2, CL, Vss (solo Periodo SAD)
    E.5.2.1Timepoint(s) of evaluation of this end point
    SAD Period: AGL mRNA and ATX95 D0, D1, D4, D7, D14, D21 and D28
    RD Period: AGL mRNA and ATX95 W0, W1, W2, W8, W9, W10 and W12
    Periodo SAD: mRNA AGL e ATX95 G0, G1, G4, G7, G14, G21 e G28
    Periodo RD: mRNA AGL e ATX95 S0, S1, S2, S8, S9, S10 e S12
    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 No
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response Yes
    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) Yes
    E.7.1.1First administration to humans Yes
    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 No
    E.8.1.6Cross over No
    E.8.1.7Other Yes
    E.8.1.7.1Other trial design description
    Parte 1 in aperto. Parte 2 randomizzata, in doppio cieco, controllata con placebo
    Part 1 Open-label. Part 2 Randomised, Double-blind, Placebo-controlled
    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 EEA6
    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
    Canada
    France
    Germany
    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
    The end of the study is defined as the date of the last protocol-specified visit (including safety follow-up telephone calls in the event of early termination [ET]) for the last subject in the study.
    La fine dello studio è definita come data dell’ultima visita specificata nel protocollo (comprese le telefonate di follow-up di sicurezza in caso di interruzione anticipata [ET]) per l’ultimo soggetto nello studio.
    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.1.1.1In 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: 27
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 3
    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
    In the case of adult subjects with cognitive limitation, provide written assent (if required) and written informed consent by a legally authorized representative
    Nel caso di soggetti adulti con limitazione cognitiva, fornire un assenso scritto (se richiesto) e un consenso informato scritto da parte di un rappresentante legalmente autorizzato
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state3
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 19
    F.4.2.2In the whole clinical trial 30
    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)
    Once chronic toxicity data are available, all subjects, including those previously treated with placebo, will be allowed to receive long-term open-label treatment with UX053.
    Quando saranno disponibili di dati di tossicità cronica, tutti i soggetti, compresi quelli precedentemente trattati con placebo, potranno ricevere il trattamento a lungo termine in aperto con UX053.
    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 Decision2021-12-14
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-06-08
    P. End of Trial
    P.End of Trial StatusOngoing
    For support, visit the EMA Service Desk , log in using your EMA account and open a ticket specifying "EU CTR" in your request.
    If you do not have an account, please visit the EMA Account management page page click on "Create an EMA account" and follow the instructions.
    The status of studies in GB is no longer updated from 1.1.2021
    For the UK, as from 1.1.2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI
    EU Clinical Trials Register Service Desk: https://servicedesk.ema.europa.eu
    European Medicines Agency © 1995-2022 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    Legal notice
    EMA HMA