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    Summary
    EudraCT Number:2019-003807-37
    Sponsor's Protocol Code Number:A4250-011
    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:2020-10-21
    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 number2019-003807-37
    A.3Full title of the trial
    A Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Odevixibat (A4250) in Children with Biliary Atresia Who Have Undergone a Kasai Hepatoportoenterostomy (BOLD)
    Studio in doppio cieco, randomizzato, controllato con placebo volto a valutare l'efficacia e la sicurezza di odevixibat (A4250) in bambini affetti da atresia delle vie biliari che sono stati sottoposti a epatoportoenterostomia secondo Kasai (BOLD)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    This Study Will Investigate the Efficacy and Safety of Odevixibat in Children with Biliary Atresia Who Have Undergone a Kasai HPE
    Questo studio esaminerà l'efficacia e la sicurezza di Odevixibat in bambini affetti da atresia delle vie biliari che sono stati sottoposti a HPE Kasai
    A.3.2Name or abbreviated title of the trial where available
    BOLD
    BOLD
    A.4.1Sponsor's protocol code numberA4250-011
    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 SponsorALBIREO AB
    B.1.3.4CountrySweden
    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 support
    B.4.2Country
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationAlbireo AB
    B.5.2Functional name of contact pointClinical Operations Department
    B.5.3 Address:
    B.5.3.1Street AddressArvid Wallgrens backe 20
    B.5.3.2Town/ cityGöteborg
    B.5.3.3Post code41346
    B.5.3.4CountrySweden
    B.5.4Telephone number0046317411480
    B.5.5Fax number004631820223
    B.5.6E-mailmedinfo@albireopharma.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 numberEMA/OD/165/18
    D.3 Description of the IMP
    D.3.1Product nameOdevixibat
    D.3.2Product code [A4250]
    D.3.4Pharmaceutical form Capsule, hard
    D.3.4.1Specific paediatric formulation Yes
    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 INNOdevixibat
    D.3.9.1CAS number 501692-44-0
    D.3.9.2Current sponsor codeA4250
    D.3.9.4EV Substance CodeSUB126128
    D.3.10 Strength
    D.3.10.1Concentration unit µg microgram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number200
    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 numberEMA/OD/165/18
    D.3 Description of the IMP
    D.3.1Product nameOdevixibat
    D.3.2Product code [A4250]
    D.3.4Pharmaceutical form Capsule, hard
    D.3.4.1Specific paediatric formulation Yes
    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 INNOdevixibat
    D.3.9.1CAS number 501692-44-0
    D.3.9.2Current sponsor codeA4250
    D.3.9.4EV Substance CodeSUB126128
    D.3.10 Strength
    D.3.10.1Concentration unit µg microgram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number600
    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: 3
    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 numberEMA/OD/165/18
    D.3 Description of the IMP
    D.3.1Product nameOdevixibat
    D.3.2Product code [A4250]
    D.3.4Pharmaceutical form Capsule, hard
    D.3.4.1Specific paediatric formulation Yes
    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 INNOdevixibat
    D.3.9.1CAS number 501692-44-0
    D.3.9.2Current sponsor codeA4250
    D.3.9.4EV Substance CodeSUB126128
    D.3.10 Strength
    D.3.10.1Concentration unit µg microgram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number200
    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
    Biliary Atresia
    Atresia biliare
    E.1.1.1Medical condition in easily understood language
    Childhood disease of the liver in which one or more bile ducts are abnormally narrow, blocked, or absent.
    Malattia infantile del fegato in cui uno o più dotti biliari sono anormalmente stretti, bloccati o assenti.
    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 10004653
    E.1.2Term Biliary atresia
    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
    The primary objective is to evaluate the efficacy of repeated once-daily doses of odevixibat versus placebo in children with biliary atresia (BA) post Kasai hepatoportoenterostomy(HPE) based on survival with native liver after 104 weeks of study treatment.
    L'obiettivo primario è valutare l'efficacia di dosi QD ripetute di odevixibat rispetto al placebo in bambini affetti da atresia delle vie biliari (AVB) successivamente a epatoportoenterostomia secondo Kasai (hepatoPortoEnterostomy, HPE) in base alla sopravvivenza con fegato nativo dopo 104 settimane di trattamento in studio.
    E.2.2Secondary objectives of the trial
    - To evaluate the effect of odevixibat compared to placebo on the time to onset of sentinel events.
    - To evaluate the effect of odevixibat compared to placebo on total bilirubin after 13, 26, 52, and 104 weeks of study treatment.
    - To evaluate the effect of odevixibat compared to placebo on serum bile acids after 13, 26, 52, and 104 weeks of study treatment.
    - To assess the long-term safety and tolerability of repeated daily doses of odevixibat compared to placebo for 104 weeks in children with BA post Kasai HPE.
    - Valutare l'effetto di odevixibat rispetto al placebo sul tempo all'insorgenza di eventi sentinella.
    - Valutare l'effetto di odevixibat rispetto al placebo sulla bilirubina totale dopo 13, 26, 52 e 104 settimane di trattamento in studio.
    - Valutare l'effetto di odevixibat rispetto al placebo sugli acidi biliari nel siero dopo 13, 26, 52 e 104 settimane di trattamento in studio.
    - Valutare la sicurezza e la tollerabilità a lungo termine di dosi giornaliere ripetute di odevixibat rispetto al placebo per 104 settimane in bambini affetti da AVB, che sono stati sottoposti a HPE secondo Kasai.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. A male or female patient with a clinical diagnosis of BA
    2. Age at Kasai HPE =90 days
    3. Eligible to start study treatment within 3 weeks post-Kasai HPE
    1. Paziente maschio o femmina con diagnosi clinica di AVB
    2. Età all'HPE secondo Kasai =90 giorni
    3. Eleggibilità a iniziare il trattamento in studio entro 3 settimane dall'HPE secondo Kasai
    E.4Principal exclusion criteria
    1. Patients with intractable ascites
    2. Ileal resection surgery
    3. ALT =10× upper limit of normal (ULN) at screening
    4. Patient on total parenteral nutrition at randomization
    5. Acute ascending cholangitis (patients may be randomized after resolution of acute ascending cholangitis)
    6. Choledochal cystic disease
    1. Pazienti con ascite intrattabile
    2. Chirurgia di resezione ileale
    3. ALT =10 × limite superiore del normale (ULN) allo screening
    4. Paziente su nutrizione parenterale totale alla randomizzazione
    5. Colangite acuta ascendente (i pazienti possono essere randomizzati dopo la risoluzione della colangite acuta ascendente)
    6. Malattia cistica coledocica
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy endpoint is the proportion of patients who are alive and have not undergone a liver transplant after 104 weeks of study treatment.
    L'endpoint primario di efficacia consiste nella percentuale di pazienti vivi e che non hanno subito un trapianto di fegato dopo 104 settimane di trattamento in studio.
    E.5.1.1Timepoint(s) of evaluation of this end point
    104 weeks
    104 settimane
    E.5.2Secondary end point(s)
    1. Time to onset of any sentinel events.
    2. Total bilirubin level after 13, 26, 52, and 104 weeks of study
    treatment.
    3. Serum bile acid level after 13, 26, 52, and 104 weeks of study
    treatment.
    4. Safety parameters including AEs, SAEs, findings on physical
    examination, laboratory assessments and abdominal ultrasound, and
    stool frequency.
    1. Tempo all'insorgenza di qualsiasi evento sentinella.
    2. Livello di bilirubina totale dopo 13, 26, 52 e 104 settimane di trattamento in studio.
    3. Livello degli acidi biliari nel siero dopo 13, 26, 52 e 104 settimane di trattamento in studio.
    4. Parametri di sicurezza, tra cui AE, eventi avversi seri (SAE), reperti all'esame obiettivo, valutazioni di laboratorio, ecografia addominale e frequenza di evacuazione.
    E.5.2.1Timepoint(s) of evaluation of this end point
    13, 26, 52 and 104 weeks.
    13, 26, 52 e 104 settimane.
    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 No
    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) 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) Yes
    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 concerned5
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA21
    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
    Belgium
    Canada
    China
    Denmark
    France
    Germany
    Hungary
    Israel
    Italy
    Korea, Republic of
    Malaysia
    Netherlands
    New Zealand
    Poland
    Spain
    Taiwan
    Turkey
    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 follows:
    • End of study in one country: last patient last visit (LPLV) in the country and sites in the country are closed
    • End of study globally: LPLV globally and all study sites closed
    La fine dello studio è definita come segue:
    • Fine dello studio in un paese: ultima visita dell'ultimo paziente (LPLV) nel
    paese e centri nel paese chiusi
    • Fine dello studio a livello globale: LPLV a livello globale e tutti i centri di studio chiusi
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years4
    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 years4
    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 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) Yes
    F.1.1.4.1Number of subjects for this age range: 200
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) No
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception No
    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
    Subjects under age incapable of giving consent personally.
    Soggetti minorenni incapaci di dare il consenso personalmente.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state9
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 93
    F.4.2.2In the whole clinical trial 200
    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)
    Following completion of this study, patients will be eligible to receive open-label odevixibat 120 µg/kg/day through an open-label extension study.
    Dopo il completamento di questo studio, i pazienti avranno diritto a ricevere odevixibat in aperto 120 µg / kg / giorno attraverso uno studio di estensione in aperto.
    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 Decision2020-09-10
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-05-25
    P. End of Trial
    P.End of Trial StatusOngoing
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