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    Summary
    EudraCT Number:2021-000996-36
    Sponsor's Protocol Code Number:A4250-015
    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:2022-01-27
    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 number2021-000996-36
    A.3Full title of the trial
    An Open Label Study to Evaluate the Long-term Safety and Efficacy of Odevixibat (A4250) in Patients with Alagille Syndrome (ASSERT-EXT)
    Studio in aperto teso a valutare la sicurezza e l’efficacia a lungo termine di odevixibat (A4250) in pazienti affetti da sindrome di Alagille (ASSERT-EXT)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A long term Investigation of the Safety and Efficacy of Odevixibat in Patients with Alagille syndrome
    Un'indagine a lungo termine sulla sicurezza e l' efficacia di Odevixibat (A4250) nei pazienti con sindrome di Alagille (ASSERT-EXT)
    A.3.2Name or abbreviated title of the trial where available
    NA
    NA
    A.4.1Sponsor's protocol code numberA4250-015
    A.5.4Other Identifiers
    Name:INDNumber:145988
    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 supportAlbireo AB
    B.4.2CountrySweden
    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.5.6E-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/023/12
    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.3.9.3Other descriptive nameA4250
    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/023/12
    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 codeA4250-015
    D.3.9.3Other descriptive nameA4250-015
    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 number400
    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/023/12
    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.3.9.3Other descriptive nameA4250
    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: 4
    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/023/12
    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.3.9.3Other descriptive nameA4250
    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 number1200
    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
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Alagille Syndrome
    Sindrome di Alagille
    E.1.1.1Medical condition in easily understood language
    Alagille syndrome is a childhood disease that affects the liver and different parts of the body. These children have buildup of a fluid made by the liver called bile which can cause severe itch.
    La sindrome di Alagille è una malattia infantile che colpisce il fegato e varie parti del corpo.Questi bambini hanno un accumulo di un fluido prodotto dal fegato(bile)che può causare un forte prurito.
    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 PT
    E.1.2Classification code 10053870
    E.1.2Term Alagille syndrome
    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
    To demonstrate a sustained effect of odevixibat on pruritus in patients with ALGS who have completed study A4250-012 (ASSERT).
    Dimostrare un effetto prolungato di odevixibat sul prurito in pazienti affetti da ALGS che hanno completato lo studio A4250-012 (ASSERT).
    E.2.2Secondary objectives of the trial
    - To demonstrate a sustained effect of odevixibat on serum bile acids in patients with ALGS who have completed study A4250-012;
    - To evaluate an effect of odevixibat on parameters related to quality of life;
    - To evaluate the long-term safety and tolerability of repeated daily doses of odevixibat in patients with ALGS.
    - Dimostrare un effetto prolungato di odevixibat sui livelli sierici di acidi biliari in pazienti affetti da ALGS che hanno completato lo studio A4250-012;
    - Valutare l’effetto di odevixibat sui parametri relativi alla qualità della vita;
    - Valutare la sicurezza e la tollerabilità a lungo termine di dosi giornaliere ripetute di odevixibat in pazienti affetti da ALGS.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Completion of the 24-week Treatment Period of Study A4250-012;
    2. Signed informed consent and assent as appropriate. Patients who turn 18 years of age (or legal age per country) during the study will be required to re-consent to remain on the study;
    3. Caregivers (and age-appropriate patients) must be willing and able to use an electronic diary (eDiary) device as required by the study;
    4. Sexually active males and females must agree to use a reliable contraceptive method with = 1% failure rate (such as hormonal contraception, intra-uterine device, or complete abstinence) from signed informed consent through 90 days after last dose of study drug.
    1.Completamento del periodo di trattamento di 24 settimane dello studio A4250-012;
    2.Firma del consenso informato e dell'assenso, ove appropriato. I pazienti che compiono 18 anni (o l'età legale secondo il Paese) durante lo studio dovranno rinnovare il consenso per rimanere nello studio;
    3.I caregiver (e i pazienti di età appropriata) devono essere disposti a, e in grado di utilizzare un diario elettronico (eDiary) come richiesto dallo studio;
    4.I soggetti di sesso maschile e femminile sessualmente attivi devono accettare di utilizzare un metodo contraccettivo affidabile con un tasso di insuccesso = 1% (come contraccezione ormonale, dispositivo intrauterino o astinenza completa) a partire dalla firma del consenso informato e per 90 giorni dopo l’ultima dose del farmaco in studio.
    E.4Principal exclusion criteria
    1. Decompensated liver disease, history or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy;
    2. Patients who were not compliant with study drug treatment or procedures in Study A4250-012;
    3. Any other conditions or abnormalities which, in the opinion of the investigator, may compromise the safety of the patient, or interfere with the patient participating in or completing the study;
    4. Known hypersensitivity to any components of odevixibat.
    1.Malattia epatica scompensata, anamnesi o presenza di ascite clinicamente significativa, emorragia da varici e/o encefalopatia;
    2.Pazienti che non hanno seguito il trattamento o le procedure con il farmaco in studio durante lo studio A4250-012;
    3.Altra condizione o anomalia che, a giudizio dello sperimentatore, possa compromettere la sicurezza del/la paziente o interferire con la partecipazione o il completamento dello studio da parte del/la paziente;
    4.Ipersensibilità nota a uno dei componenti di odevixibat.
    E.5 End points
    E.5.1Primary end point(s)
    Change from baseline in scratching through Week 72 as measured by the Albireo ObsRO caregiver instrument.
    Variazione del grattarsi dal basale alla settimana 72 misurata mediante lo strumento Albireo ObsRO (esiti riferiti dall’osservatore).
    E.5.1.1Timepoint(s) of evaluation of this end point
    Change from baseline in scratching through Week 72.
    Variazione del grattarsi dal basale alla settimana 72.
    E.5.2Secondary end point(s)
    • Change in serum bile acid levels from baseline to Week 72;
    • Change from baseline through Week 72 in patient reported and observer reported itching and scratching severity scores, respectively, for the morning and evening assessment;
    • Percentage of patients achieving a clinically meaningful decrease in pruritus (pruritus responders) as measured by the Albireo ObsRO/patient reported outcomes (PRO) instruments;
    • Change from baseline to Week 72 in sleep parameters as measured with the Albireo ObsRO/PRO instruments (e.g. tiredness and number of awakenings);
    • Change from baseline to Week 72 in Pediatric Quality of Life Inventory (PedsQL) scores;
    • Assessment of Global Symptom Relief from baseline to Weeks 4, 12, 24, 48, and 72 as measured by patient, caregiver, and clinician Global impression of Symptoms (PGIS, CaGIS, CGIS) items;
    • Assessment of Global Symptom Relief as measured by patient, caregiver, and clinician Global Impression of Change (PGIC, CaGIC, CGIC) items at Weeks 4, 12, 24, 48, and 72;
    • Change in serum bile acid levels from baseline through Week 72.
    • Variazione dei livelli sierici di acidi biliari dal basale alla settimana 72;
    • Varazione dal basale alla settimana 72 dei punteggi di gravità del prurito e del grattarsi riferiti dal/la paziente e dall’osservatore, rispettivamente, per la valutazione mattutina e serale;
    • Percentuale di pazienti che raggiungono una riduzione clinicamente significativa del prurito (responder per prurito) misurata dagli strumenti Albireo ObsRO/PRO (esiti riferiti dal paziente);
    • Variazione dal basale alla settimana 72 dei parametri del sonno misurati con gli strumenti Albireo ObsRO/PRO (ad es. stanchezza e numero di risvegli);
    • Variazione dal basale alla settimana 72 dei punteggi del PedsQL (Pediatric Quality of Life Inventory);
    • Valutazione del sollievo globale dei sintomi dal basale alle settimane 4, 12, 24, 48 e 72 secondo la misurazione effettuata dal paziente, dal caregiver e dal medico in base all’impressione globale dei sintomi (PGIS, CaGIS, CGIS);
    • Valutazione del sollievo globale dei sintomi effettuata dal paziente, dal caregiver e dal medico in base all’impressione globale del cambiamento (PGIC, CaGIC, CGIC) alle settimane 4, 12, 24, 48 e 72;
    • Variazione dei livelli sierici di acidi biliari dal basale alla settimana 72.
    E.5.2.1Timepoint(s) of evaluation of this end point
    • Change in serum bile acid levels from baseline through Week 72;
    • Change from baseline through Week 72;
    • Assessment of Global Symptom Relief from baseline to Weeks 4, 12, 24, 48, and 72.
    • Variazione dei livelli sierici di acidi biliari dal basale alla settimana 72;
    • Varazione dal basale alla settimana 72;
    • Valutazione del sollievo globale dei sintomi dal basale alle settimane 4, 12, 24, 48 e 72.
    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 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 No
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    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 No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial1
    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 concerned4
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA16
    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
    Belgium
    Canada
    France
    Germany
    Israel
    Italy
    Malaysia
    Netherlands
    New Zealand
    Poland
    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 for a patient: last study visit by the patient, either at the clinic or via telephone, whichever comes latest;
    • 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 per un paziente: ultima visita di studio del paziente, in ambulatorio o telefonicamente, a seconda di quale si verifica per ultima;
    • Fine dello studio in un paese: l'ultima visita dell'ultimo paziente (LPLV) nel paese e i centri nel paese sono chiusi;
    • Fine dello studio a livello globale: LPLV a livello globale e tutti i centri studio chiusi.
    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 months3
    E.8.9.1In the Member State concerned days15
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months3
    E.8.9.2In all countries concerned by the trial days15
    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: 2
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 22
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 21
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 15
    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 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
    Subjects under age incapable of giving consent personally.
    Soggetti minorenni incapaci di prestare personalmente il consenso.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state10
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 30
    F.4.2.2In the whole clinical trial 60
    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 may have the option to enroll in another monitored program (e.g. managed access program) of odevixibat provided they meet eligibility criteria for that study.
    Dopo il completamento di questo studio, i pazienti potrebbero la possibilità di iscriversi a un altro programma monitorato (ad es. programma di accesso gestito) di odevixibat a condizione che soddisfino i criteri di idoneità per tale studio.
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2021-11-09
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-11-08
    P. End of Trial
    P.End of Trial StatusOngoing
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