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    Summary
    EudraCT Number:2021-001389-39
    Sponsor's Protocol Code Number:VLX-601
    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:2022-09-06
    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 number2021-001389-39
    A.3Full title of the trial
    A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Volixibat in the Treatment of Cholestatic Pruritus in Patients with Primary Biliary Cholangitis (VANTAGE)
    Studio randomizzato, in doppio cieco, controllato con placebo per valutare l’efficacia e la sicurezza di Volixibat nel trattamento del prurito colestatico in pazienti con colangite biliare primitiva (VANTAGE)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to evaluate efficacy and safety of an investigational drug named volixibat in patients with itching caused by primary biliary cholangitis
    Studio per valutare l'efficacia e sicurezza di un farmaco sperimentale denominato volixibat in pazienti con prurito dovuto a colangite biliare primitiva
    A.3.2Name or abbreviated title of the trial where available
    VANTAGE
    VANTAGE
    A.4.1Sponsor's protocol code numberVLX-601
    A.5.2US NCT (ClinicalTrials.gov registry) numberNCT05050136
    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 SponsorMirum Pharmaceuticals 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 supportMirum Pharmaceuticals, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationMirum Pharmaceuticals, Inc.
    B.5.2Functional name of contact pointMirum Clinical Lead
    B.5.3 Address:
    B.5.3.1Street Address950 Tower Lane, Suite 1050
    B.5.3.2Town/ cityFoster City California
    B.5.3.3Post codeCA 94404
    B.5.3.4CountryUnited States
    B.5.4Telephone number16506674085
    B.5.5Fax number16504122500
    B.5.6E-mailclinicaltrials@mirumpharma.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 namevolixibat
    D.3.2Product code [volixibat]
    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 INNvolixibat
    D.3.9.1CAS number 1431935-92-0
    D.3.9.2Current sponsor codeVLX, V-0027
    D.3.9.3Other descriptive namevolixibat
    D.3.9.4EV Substance CodeSUB190237
    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.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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product namevolixibat
    D.3.2Product code [volixibat]
    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 INNvolixibat
    D.3.9.1CAS number 1431935-92-0
    D.3.9.2Current sponsor codeVLX, V-0027
    D.3.9.3Other descriptive namevolixibat
    D.3.9.4EV Substance CodeSUB190237
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number20
    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
    Cholestatic Pruritus in Patients with Primary Biliary Cholangitis (PBC)
    Prurito colestatico in pazienti con colangite biliare primitiva (CBP)
    E.1.1.1Medical condition in easily understood language
    Itching in patients with Primary Biliary Cholangitis (PBC)
    Prurito in pazienti con colangite biliare primitiva (CBP)
    E.1.1.2Therapeutic area Diseases [C] - Digestive System Diseases [C06]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.0
    E.1.2Level PT
    E.1.2Classification code 10080429
    E.1.2Term Primary biliary cholangitis
    E.1.2System Organ Class 10019805 - Hepatobiliary disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the efficacy of volixibat versus placebo for the treatment of pruritus in participants with PBC as measured by the change in the Adult ItchRO tool in participants with PBC
    Valutare l’efficacia di volixibat rispetto a placebo per il trattamento del prurito nei partecipanti con CBP sulla base della variazione dell' Adult ItchRO nei partecipanti con CBP
    E.2.2Secondary objectives of the trial
    - To evaluate the efficacy of volixibat versus placebo for the treatment of pruritus in participants with PBC using additional measures of pruritus efficacy.
    - To evaluate safety and tolerability of volixibat versus placebo in participants with PBC-associated pruritus.
    - To assess volixibat pharmacodynamics.
    - To evaluate Quality of Life in participants with PBC-associated pruritus treated with volixibat versus placebo.
    -Valutare l’efficacia di volixibat rispetto a placebo per il trattamento del prurito nei partecipanti con CBP utilizzando misure aggiuntive dell'efficacia sul prurito
    -Valutare la sicurezza e la tollerabilità di volixibat rispetto a placebo in partecipanti con prurito associato a CBP
    -Valutare la farmacodinamica di volixibat
    -Valutare la qualità della vita nei partecipanti con prurito associato alla CBP trattati con volixibat rispetto a placebo
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Provide signed informed consent at the screening visit as well as comply with all study visits and requirements through the end of the study.
    2. Male or female, age greater than or equal to 18 years at the screening visit.
    3. Confirmed diagnosis of PBC in line with the AASLD guidelines.
    4. UDCA and anti-pruritic medication use will be allowed if meeting additional criteria.
    5. Qualified pruritus associated with PBC as assessed by Adult ItchRO.
    1. Fornire consenso informato firmato alla visita di screening e rispettare tutti gli appuntamenti per le visite e i requisiti dello studio fino alla fine dello studio
    2. Soggetti di sesso maschile o femminile di età maggiore o uguale 18 anni alla visita di screening
    3 Diagnosi confermata di CBP conforme alle linee guida AASLD
    4. Uso di terapie UDCA e anti-prurito sarà consentito se soddisfa criteri aggiuntivi
    5. Prurito qualificato associato a CBP come valutato da Adult ItchRO
    E.4Principal exclusion criteria
    1. Pruritus associated with an etiology other than PBC.
    2. Evidence or clinical suspicion of decompensated cirrhosis or a history of decompensation events.
    3. Current symptomatic cholelithiasis or inflammatory gallbladder disease.
    4. History of small bowel surgery/resection impacting the terminal ileum that may disrupt the enterohepatic circulation.
    5. Evidence, history, or suspicion of other liver diseases.
    1. Prurito associato a un'eziologia diversa dalla CBP
    2. Evidenza o sospetto clinico di cirrosi scompensata o anamnesi di eventi di scompenso
    3. Colelitiasi sintomatica o colecistite acuta in corso
    4. Anamnesi di chirurgia/resezione dell’intestino tenue con impatto sull’ileo terminale che può comportare un’interruzione della circolazione enteroepatica
    5. Evidenza, anamnesi o sospetto di altre malattie epatiche
    E.5 End points
    E.5.1Primary end point(s)
    Mean change in Adult ItchRO score comparing baseline with the average of the weekly averaged daily itch scores from Week 16 to end of double-blind study treatment
    Variazione media del punteggio Adult ItchRO ottenuta confrontando il basale alla media della media settimanale dei punteggi giornalieri del questionario del prurito dalla Settimana 16 alla fine del periodo di trattamento in doppio cieco
    E.5.1.1Timepoint(s) of evaluation of this end point
    Baseline and Week 16 to end of double-blind study treatment
    Basale e settimana 16 fino alla fine del periodo di trattamento in doppio cieco
    E.5.2Secondary end point(s)
    - Proportion of participants achieving a reduction of at least 2 points in the weekly averaged daily Adult ItchRO score from baseline to the end of double-blind study treatment
    - Proportion of participants with relative reductions from baseline in the weekly averaged daily Adult ItchRO score at end of double-blind study treatment of:
    greater than or equal to 30%
    greater than or equal to 50%
    - Mean number of days for each participant with daily Adult ItchRO score at least 2 points lower than the baseline score from baseline to end of double-blind study treatment
    - Incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), events of clinical interest (ECIs), and AEs that lead to discontinuation of study drug
    - Incidence of clinically relevant laboratory abnormalities
    - Changes in liver enzymes (ALT, AST, ALP, total bilirubin, and direct bilirubin)
    - Changes in fasting sBA
    - Change in Primary Biliary Cholangitis Quality of Life Measure (PBC-40) from baseline to end of double-blind study treatment
    - Change in Patient-Reported Outcomes Measurement Information System (PROMIS®) fatigue score from baseline to end of double-blind study treatment
    - Change in PROMIS sleep score from baseline to end of double-blind study treatment
    -Percentuale di partecipanti che ottengono una riduzione di almeno 2 punti della media settimanale dei punteggi giornalieri del questionario Adult ItchRO dal basale alla fine del periodo di trattamento in doppio cieco
    -Percentuale di partecipanti che ottengono le seguenti riduzioni relative rispetto al basale della media settimanale dei punteggi giornalieri del questionario Adult ItchRO alla fine del periodo di trattamento in doppio cieco:
    maggiore o uguale 30%
    maggiore o uguale 50%
    -Numero medio di giorni per ciascun partecipante con punteggio Adult ItchRO giornaliero inferiore di almeno 2 punti rispetto al punteggio basale dal basale alla fine del periodo di trattamento in doppio cieco
    -Incidenza di eventi avversi emergenti dal trattamento (TEAE), eventi avversi gravi (SAE), eventi di interesse clinico (ECI) e AE che portano all’interruzione del trattamento con il farmaco in studio
    -Incidenza di anomalie di laboratorio clinicamente rilevanti
    -Variazioni degli enzimi epatici (ALT, AST, ALP, bilirubina totale e bilirubina diretta)
    -Variazioni degli acidi biliari sierici a digiuno
    -Variazione del punteggio del questionario sulla qualità della vita nella colangite biliare primitiva (Primary Biliary Cholangitis Quality of Life Measure, PBC-40) dal basale alla fine del periodo di trattamento in doppio cieco
    -Variazione della valutazione eseguita con lo strumento PROMIS®(Patient-Reported Outcomes Measurement Information System) relativa all’astenia dal basale alla fine del periodo di trattamento in doppio cieco
    -Variazione della valutazione eseguita con lo strumento PROMIS relativa al sonno dal basale alla fine del periodo di trattamento in doppio cieco
    E.5.2.1Timepoint(s) of evaluation of this end point
    Baseline and end of double-blind study treatment
    Basale e fine del periodo di trattamento in doppio cieco
    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 Yes
    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) 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) 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 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 trial3
    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 concerned7
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA25
    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
    Israel
    United States
    France
    Germany
    Italy
    United Kingdom
    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 years3
    E.8.9.1In the Member State concerned months8
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years5
    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: 180
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 80
    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 state15
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 60
    F.4.2.2In the whole clinical trial 260
    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)
    standard of care
    standard di cura
    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 Decision2022-09-28
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2022-09-21
    P. End of Trial
    P.End of Trial StatusOngoing
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