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    Summary
    EudraCT Number:2020-004348-27
    Sponsor's Protocol Code Number:CB8025-32048
    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 number2020-004348-27
    A.3Full title of the trial
    RESPONSE: A Placebo-controlled, Randomized, Phase 3 Study to Evaluate the Efficacy and Safety of Seladelpar in Patients With Primary Biliary Cholangitis (PBC) and an Inadequate Response to or an Intolerance to Ursodeoxycholic Acid (UDCA)
    RESPONSE: Studio di fase 3, controllato con placebo, randomizzato volto a valutare la sicurezza e l'efficacia di seladelpar in pazienti affetti da colangite biliare primitiva (Primary Biliary Cholangitis, [PBC]) e con una risposta inadeguata o un’intolleranza all’acido ursodesossicolico (Ursodeoxycholic Acid, [UDCA])
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A clinical trial to assess the safety and efficacy of seladelpar in patients with primary biliary cholangitis (PBC) and an inadequate response to or intolerance to ursodeoxycholic acid (UDCA).
    Uno studio clinico per valutare la sicurezza e l'efficacia di seladelpar nei pazienti con colangite biliare primitiva (PBC) e una risposta inadeguata a o intolleranza all'acido ursodesossicolico (UDCA).
    A.3.2Name or abbreviated title of the trial where available
    RESPONSE
    RESPONSE
    A.4.1Sponsor's protocol code numberCB8025-32048
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT04620733
    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 SponsorCymaBay Therapeutics, 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 supportCymaBay Therapeutics, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationCymaBay Therapeutics, Inc.
    B.5.2Functional name of contact pointStacy Meluskey
    B.5.3 Address:
    B.5.3.1Street Address7575 Gateway Boulevard Suite 110
    B.5.3.2Town/ cityNewark
    B.5.3.3Post code94560
    B.5.3.4CountryUnited States
    B.5.4Telephone number+15102938141
    B.5.5Fax number+15102938133
    B.5.6E-mailsmeluskey@cymabay.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/3/17/1930
    D.3 Description of the IMP
    D.3.1Product nameSeladelpar
    D.3.2Product code [MBX-8025]
    D.3.4Pharmaceutical form Capsule, hard
    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.3.8INN - Proposed INNseladelpar
    D.3.9.1CAS number 928821-40-3
    D.3.9.2Current sponsor codeMBX-8025
    D.3.9.4EV Substance CodeSUB192392
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    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.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 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/3/17/1930
    D.3 Description of the IMP
    D.3.1Product nameSeladelpar
    D.3.2Product code [MBX-8025]
    D.3.4Pharmaceutical form Capsule, hard
    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.3.8INN - Proposed INNseladelpar
    D.3.9.1CAS number 928821-40-3
    D.3.9.2Current sponsor codeMBX-8025
    D.3.9.4EV Substance CodeSUB192392
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number5
    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 placeboCapsule, hard
    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
    Primary biliary cholangitis (PBC, formerly known as primary biliary cirrhosis) is a serious and potentially life threatening autoimmune disease of the liver characterized by impaired bile flow (cholestasis) and accumulation of toxic bile acids (BA).
    Colangite biliare primitiva (PBC, precedentemente nota come biliare primaria cirrosi) è una malattia autoimmune grave e potenzialmente pericolosa per la vita, malattia del fegato caratterizzata da alterato flusso biliare (colestasi) e accumulo di acidi biliari tossici (BA).
    E.1.1.1Medical condition in easily understood language
    Disease of the liver
    Patologia del fegato
    E.1.1.2Therapeutic area Body processes [G] - Immune system processes [G12]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.0
    E.1.2Level LLT
    E.1.2Classification code 10036680
    E.1.2Term Primary biliary cirrhosis
    E.1.2System Organ Class 100000004871
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    The primary objective of this study is to evaluate the treatment effect of seladelpar on composite biochemical improvement in cholestasis markers based on ALP and total bilirubin and to evaluate the safety of seladelpar over 12 months of treatment compared to placebo.
    L'obiettivo principale di questo studio è valutare l'effetto del trattamento di seladelpar sul miglioramento biochimico composito dei marcatori di colestasi basati su ALP e bilirubina totale e valutare la sicurezza di seladelpar per 12 mesi di trattamento rispetto al placebo.
    E.2.2Secondary objectives of the trial
    The key secondary objectives of this study are to evaluate the effect of seladelpar on the normalization of ALP values at 12 months of treatment compared to placebo and to evaluate the effect of seladelpar on pruritus at 6 months of treatment compared to placebo in subjects with baseline moderate to severe pruritus. Other secondary objectives of the study are to evaluate the effect of seladelpar on other measures of cholestasis, metabolic markers, and PBC prognostic criteria; to evaluate the effect of seladelpar on quality of life (QoL); and to evaluate the effect of seladelpar on PBC-associated clinical outcomes.
    Gli obiettivi secondari chiave sono valutare l’effetto di seladelpar sulla normalizzazione dei valori di ALP a 12 mesi di trattamento rispetto al placebo e valutare l’effetto di seladelpar sul prurito a 6 mesi di trattamento rispetto al placebo in soggetti con prurito moderato e grave al basale
    Altri obiettivi secondari sono Valutare l’effetto di seladelpar su altre misure di colestasi, marcatori metabolici e criteri prognostici della colangite biliare primitiva (Primary biliary cholangitis [PBC]) ; valutare l’effetto di seladelpar sulla qualità della vita (Quality of life, [QoL]) valutare l’effetto di seladelpar sugli esiti clinici associati alla PBC
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Subjects must meet all of the following criteria to be eligible for study participation:
    1. Must have given written informed consent (signed and dated) and any authorizations required by local law
    2. 18 to 75 years old (inclusive)
    3. Male or female with a diagnosis of PBC based on any two of the following criteria:
    a. History of ALP above 1.0× ULN for at least 6 months
    b. Positive AMA titer (>1:40 on immunofluorescence or M2 positive by enzyme linked immunosorbent assay [ELISA]) or positive PBC-specific antinuclear antibodies (ANAs)
    c. Documented liver biopsy results consistent with PBC
    4. UDCA for the past 12 months (stable dose for >3 months prior to screening) OR intolerant to UDCA (last dose of UDCA >3 months prior to screening)
    5. Laboratory parameters measured by the Central Laboratory at screening:
    a. ALP >=1.67× ULN
    b. Aspartate aminotransferase (AST) <=3× ULN
    c. ALT <=3× ULN
    d. Total bilirubin <=2× ULN
    e. Estimated glomerular filtration rate (eGFR) >60 mL/min/1.73 m2 (calculated by the Modification of Diet in Renal Disease study equation)
    f. International normalized ratio (INR) below 1.1× ULN
    For subjects on anticoagulation therapy, INR must be maintained in the range required for prophylaxis for their specific disease.
    g. Platelet count >=100×103/µL
    6. Females of reproductive potential (Refer to section 8.1.1) must use at least 1 barrier contraceptive and a second effective birth control method during the study and for at least 90 days after the last dose. Male subjects who are sexually active with female partners of reproductive potential must use barrier contraception, and their female partners must use a second effective birth control method during the study and for at least 90 days after the last dose
    L’idoneità alla partecipazione allo studio è subordinata alla soddisfazione dei seguenti criteri da parte dei soggetti:
    1. Aver fornito il consenso informato scritto (firmato e datato) e tutte le autorizzazioni previste dalla legge locale
    2. Età tra 18 e 75 anni (compresi)
    3. Sesso maschile o femminile e diagnosi di PBC basata su due tra i seguenti criteri:
    a. Anamnesi di ALP al di sopra di 1,0× limite superiore della norma (ULN) da almeno 6 mesi
    b. Titolo AMA positivo (>1:40 su immunofluorescenza o M2 positivo mediante ELISA) o ANA specifico per PBC positivo
    c. Esiti della biopsia epatica documentata coerenti con PBC
    4. UDCA per gli ultimi 12 mesi (dose stabile per >3 mesi prima dello screening) OPPURE intolleranti a UDCA (ultima dose di UDCA >3 mesi prima dello screening)
    5. Parametri di laboratorio misurati dal laboratorio centrale allo screening:
    a. ALP >=1,67× ULN
    b. Aspartato aminotransferasi (AST) <=3× ULN
    c. Alanina aminotransferasi (ALT) <=3× ULN
    d. Bilirubina totale <=2× ULN
    e. Velocità di filtrazione glomerulare stimata >60 ml/min/1,73 m2 (calcolata mediante equazione dello studio di Modifica della dieta nella malattia renale)
    f. Rapporto normalizzato internazionale (International Normalized Ratio, [INR]) inferiore a 1,1× ULN
    Per i soggetti in terapia anticoagulante, l’INR deve essere mantenuto nell’intervallo previsto per la profilassi della loro specifica malattia.
    g. Conta piastrinica >=100×103/µl
    6. I soggetti di sesso femminile potenzialmente fertili (riferirsi alla sezione 8.1 .1) devono utilizzare almeno 1 metodo contraccettivo barriera e un secondo metodo contraccettivo efficace durante lo studio e per almeno 90 giorni dopo l’ultima dose. I soggetti di sesso maschile sessualmente attivi con partner di sesso femminile potenzialmente fertili devono utilizzare metodi contraccettivi barriera, e le loro partner devono utilizzare un secondo metodo contraccettivo efficace durante lo studio e per almeno 90 giorni dopo l’ultima dose.
    E.4Principal exclusion criteria
    1. Previous exposure to seladelpar (MBX-8025)
    2. A medical condition other than PBC that, in the investigator’s opinion, would preclude full participation in the study (eg, cancer) or confound its results (eg, Paget’s disease)
    3. Advanced PBC as defined by the Rotterdam criteria (albumin below the lower limit of normal AND total bilirubin above 1.0× ULN)
    4. Presence of clinically important hepatic decompensation, including the following:
    a. History of liver transplantation, current placement on liver transplantation list, or current Model for End-Stage Liver Disease (MELD) score >=12
    b. Complications of portal hypertension, including known esophageal varices, history of variceal bleeds or related interventions (eg, transjugular intrahepatic portosystemic shunt placement), ascites, and hepatic encephalopathy
    c. Cirrhosis with complications, including history or presence of spontaneous bacterial peritonitis, hepatocellular carcinoma, or hepatorenal syndrome
    5. Other chronic liver diseases:
    a. Current features of autoimmune hepatitis as determined by the investigator based on immunoserology, liver biochemistry, or historic confirmed liver histology
    b. Primary sclerosing cholangitis determined by the presence of diagnostic cholangiographic findings
    c. History or clinical evidence of alcoholic liver disease
    d. History or clinical evidence of alpha-1-antitrypsin deficiency
    e. History of biopsy confirmed NASH
    f. History or evidence of Gilbert’s syndrome with elevated total bilirubin
    g. History or evidence of hemochromatosis
    h. Hepatitis B, defined as the presence of hepatitis B surface antigen (HBsAg)
    i. Hepatitis C, defined as the presence of hepatitis C virus (HCV) ribonucleic acid (RNA)
    j. History, evidence, or high suspicion of hepatobiliary malignancy based on imaging, screening laboratory values, and/or clinical symptoms
    6. Known history of human immunodeficiency virus (HIV) or positive antibody test at screening
    7. Clinically important alcohol consumption, defined as more than 2 drink units per day (equivalent to 20 g) in women and 3 drink units per day (equivalent to 30 g) in men, or inability to quantify alcohol intake reliably
    8. History of malignancy diagnosed or treated, actively or within 2 years, or ongoing evaluation for malignancy; localized treatment of squamous or noninvasive basal cell skin cancers and cervical carcinoma in situ is allowed if appropriately treated prior to screening.
    9. Treatment with OCA, fibrates (eg, bezafibrate, fenofibrate, elafibranor, lanifibranor, pemafibrate, saroglitizar) 3 months prior to screening
    10. Treatment with colchicine, methotrexate, azathioprine, or long-term systemic corticosteroids (>2 weeks) during 2 months prior to screening
    11. Treatment with anti-pruritic drugs (eg, cholestyramine, naltrexone, rifampicin, sertraline, or any experimental approach) must be on a stable dose within 1 month prior to screening
    12. Treatment with any other investigational therapy or device within 30 days or within 5 half-lives, whichever is longer, prior to screening
    13. For females, pregnancy or breastfeeding
    14. Any other condition(s) that would compromise the safety of the subject or compromise the quality of the clinical study, as judged by the investigator
    Per essere idonei a partecipare allo studio, i soggetti non devono presentare alcuno dei seguenti criteri:
    1. Precedente esposizione a seladelpar (MBX-8025)
    2. Una condizione medica diversa da PBC che, a giudizio dello sperimentatore, precluderebbe la partecipazione allo studio (ad es. un tumore) o ne confonderebbe i risultati (ad es. la malattia di Paget)
    3. PBC in stadio avanzato, come definita dai criteri di Rotterdam (albumina sotto il limite inferiore alla norma E bilirubina totale superiore a 1,0× ULN)
    4. Presenza di scompenso epatico clinicamente importante, incluso quanto segue:
    a. anamnesi di trapianto di fegato, attuale iscrizione alla lista d’attesa per trapianti di fegato o punteggio attuale >=12 nel Modello per la malattia epatica allo stadio terminale (Model for End-Stage Liver Disease, [MELD])
    b. Complicazioni di ipertensione portale, comprese varici esofagee note, anamnesi di emorragia da varici o interventi correlati (ad es. posizionamento di shunt portosistemico intraepatico transgiugulare), ascite ed encefalopatia epatica
    c. Cirrosi con complicazioni, inclusa anamnesi o presenza di peritonite batterica spontanea, carcinoma epatocellulare, o sindrome epato-renale
    5. Altre malattie epatiche croniche:
    a. Attuali caratteristiche di AIH come stabilito dallo sperimentatore sulla base di immunosierologia, biochimica epatica o istologia epatica storicamente confermata
    b. Colangite sclerosante primitiva (CSP) determinata dalla presenza di riscontri colangiografici diagnostici
    c. Anamnesi o evidenza clinica di epatopatia alcolica
    d. Anamnesi o evidenza clinica di deficit di alfa-1-antitripsina
    e. Anamnesi di NASH confermata da biopsia
    f. Anamnesi o evidenza di sindrome di Gilbert, con livelli elevati di bilirubina totale
    g. Anamnesi o evidenza di emocromatosi
    h. Epatite B, definita dalla presenza dell’antigene di superficie dell’epatite B
    i. Epatite C, definita dalla presenza dell’acido ribonucleico del virus dell’epatite C
    j. Anamnesi, evidenza, o forte sospetto di neoplasia maligna epatobiliare sulla base delle immagini diagnostiche, dei valori di laboratorio allo screening e/o dei sintomi clinici
    6. Anamnesi nota di virus dell’immunodeficienza umana o positività al test degli anticorpi allo screening
    7. Consumo di alcol clinicamente importante, definito come più di 2 unità di bevande alcoliche al giorno (equivalente a 20 g) nelle donne e 3 unità di bevande alcoliche al giorno (equivalenti a 30 g) negli uomini, o incapacità di quantificare l’assunzione di alcol in modo affidabile
    8. Anamnesi di neoplasia maligna diagnosticata o trattata, attiva o entro 2 anni, o valutazione in corso per neoplasia maligna; trattamento localizzato di tumori della pelle a cellule basali o squamose non invasivi, e di carcinoma cervicale in situ, sono ammessi se trattati adeguatamente prima dello screening.
    9. Trattamento con acido obeticolico (obeticholic acid, [OCA]), fibrati (ad es. bezafibrato, fenofibrato, elafibranor, lanifibranor, pemafibrate, saroglitizar) 3 mesi prima dello screening
    10. Trattamento con colchicina, metotrexato, azatioprina, o corticosteroidi sistemici (>2 settimane) nel corso di 2 mesi prima dello screening
    11. Il trattamento con farmaci anti-prurito (ad es. colestiramina, naltrexone, rifampicina, sertralina, o qualsiasi approccio sperimentale) deve essere ad una dose stabile entro
    1 mese prima dello screening
    12. Trattamento con qualsiasi altra terapia o dispositivo sperimentale entro 30 giorni o entro 5 emivite, a seconda di quale periodo è più lungo, precedenti allo screening
    13. Per i soggetti di sesso femminile, gravidanza o allattamento al seno
    14. Qualsiasi altra condizione che potrebbe compromettere la sicurezza del soggetto o la qualità dello studio clinico, secondo il giudizio dello sperimentatore
    E.5 End points
    E.5.1Primary end point(s)
    1. Proportion of subjects who are considered responders at 12 months based on the following composite endpoint of ALP and total bilirubin at 12 months requiring
    a. ALP <1.67× ULN
    b. >=15% decrease in ALP
    c. Total bilirubin <=1.0× ULN
    2. Assessment of TEAEs (National Cancer Institute [NCI] Common Terminology Criteria for Adverse Events [CTCAE] Version 5.0), biochemistry, and hematology
    1. Proporzione di soggetti che sono considerati responder a 12 mesi in base al seguente endpoint composito di ALP e bilirubina totale a 12 mesi che richiedono
    a. ALP <1,67 × ULN
    b. Diminuzione di ALP >=15%
    c. Bilirubina totale <= 1,0 × ULN
    2. Valutazione dei TEAE (criteri comuni di terminologia per gli eventi avversi [CTCAE] versione 5.0 del National Cancer Institute [NCI]), biochimica ed ematologia
    E.5.1.1Timepoint(s) of evaluation of this end point
    1. 12 months
    2. Throughout the study
    1. 12 mesi
    2. Durante lo studio
    E.5.2Secondary end point(s)
    1. Proportion of subjects with ALP<=1.0× ULN at 12 months (ie, normalization)
    2. Change from baseline in weekly averaged pruritus NRS in subjects with baseline NRS >=4 at 6 months
    1. Percentuale di soggetti con ALP =1,0× ULN a 12 mesi (ossia, normalizzazione)
    2. Variazione rispetto al basale nella media del prurito settimanale in base alla scala NRS, in soggetti con punteggio NRS = 4 al basale a 6 mesi
    E.5.2.1Timepoint(s) of evaluation of this end point
    1. 12 months
    2. 6 months
    1. 12 mesi
    2. 6 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 Yes
    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 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 concerned8
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA70
    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
    Argentina
    Australia
    Belgium
    Canada
    Chile
    France
    Germany
    Greece
    Hungary
    Israel
    Italy
    Korea, Republic of
    Mexico
    Netherlands
    New Zealand
    Poland
    Romania
    Russian Federation
    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
    LVLS is the Post-Treatment Safety Follow-up visit that will occur 1 month (4 weeks±7 days) after the last dose of the study drug.
    At the end of the visit, subjects will be invited to participate in the long-term study (Study CB8025-31731_RE).
    L’ultima visita dell’ultimo soggetto (Last Visit Last Subject, [LVLS]), è la visita di follow-up di sicurezza post-trattamento che si terrà 1 mese (4 settimane ±7 giorni) dopo l'ultima dose del farmaco dello studio. Alla fine della visita, i soggetti saranno invitati a partecipare allo studio a lungo termine (studio CB8025-31731_RE).
    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 months0
    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 months2
    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: 125
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 25
    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 state4
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 70
    F.4.2.2In the whole clinical trial 180
    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)
    Subjects who will successfully complete this study will be offered open-label treatment in a long-term study (Study CB8025-31731_RE); subjects receiving placebo will be switched to the active treatment with seladelpar.
    Ai soggetti che completeranno con successo questo studio verrà offerto un trattamento openlabel in uno studio a lungo termine (Studio CB8025-31731_RE); soggetti che ricevono il placebo passeranno al trattamento attivo con seladelpar.
    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 Decision2021-01-29
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-02-16
    P. End of Trial
    P.End of Trial StatusOngoing
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