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    Summary
    EudraCT Number:2017-002325-38
    Sponsor's Protocol Code Number:A4250-008
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Ongoing
    Date on which this record was first entered in the EudraCT database:2018-09-17
    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 ConcernedSpain - AEMPS
    A.2EudraCT number2017-002325-38
    A.3Full title of the trial
    An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of A4250 in Children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2 (PEDFIC 2)
    Estudio abierto de extensión para evaluar la eficacia y la seguridad a largo plazo de A4250 en niños con colestasis intrahepática familiar progresiva de tipo 1 y 2 (PEDFIC 2)
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study to determine the long term effect and safety of A4250 in treatment of children with Progressive Familial Intrahepatic Cholestasis Types 1 and 2.
    Estudio para determinar los efectos y la seguridad a largo plazo del A4250 en el tratamiento de niños con colestasis intrahepática familiar progresiva de tipo 1 y 2.
    A.4.1Sponsor's protocol code numberA4250-008
    A.7Trial is part of a Paediatric Investigation Plan Yes
    A.8EMA Decision number of Paediatric Investigation PlanP/306/2017
    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.5.4Telephone number+34675781547
    B.5.5Fax number+4631820223
    B.5.6E-mailjquintero@vhebron.net
    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/3/12/1028
    D.3 Description of the IMP
    D.3.2Product code A4250
    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 INNNot applicable
    D.3.9.3Other descriptive nameA4250 (1200 μg)
    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 No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    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/3/12/1028
    D.3 Description of the IMP
    D.3.2Product code A4250
    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 INNNot applicable
    D.3.9.3Other descriptive nameA4250 (600 μg)
    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 No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    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 numberEU/3/12/1028
    D.3 Description of the IMP
    D.3.2Product code A4250
    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 INNNot applicable
    D.3.9.3Other descriptive nameA4250 (400 μg)
    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 No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    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 numberEU/3/12/1028
    D.3 Description of the IMP
    D.3.2Product code A4250
    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 INNNot applicable
    D.3.9.3Other descriptive nameA4250 (200 μg)
    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 No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    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
    Progressive Familial Intrahepatic Cholestasis Types 1 and 2
    Colestasis intrahepática familiar progresiva de tipo 1 y 2
    E.1.1.1Medical condition in easily understood language
    An inherited condition causing reduced bile acid flow and progressive
    liver disease in children and young people.
    Se trata de una enfermedad heredada que reduce el flujo de ácidos biliares y provoca una enfermedad progresiva del hígado en niños y jóvenes.
    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 10076033
    E.1.2Term Progressive familial intrahepatic cholestasis
    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
    The primary objective of this study is to demonstrate a sustained effect of A4250 on serum bile acid (s-BAs) and pruritus in children with PFIC Types 1 and 2.
    El objetivo principal de este estudio es demostrar el efecto continuado de A4250 sobre los ácidos biliares séricos (ABs) y el prurito en niños con colestasis intrahepática familiar progresiva (CIFP) de tipo 1 o 2.
    E.2.2Secondary objectives of the trial
    • To evaluate the long-term safety and tolerability of repeated daily doses of A4250
    • To evaluate the effect of A4250 on growth
    • To evaluate the effect of A4250 on biliary diversion and/or listing for liver transplantation
    • To evaluate the effect of A4250 on biochemical markers of cholestasis and liver disease
    • Evaluar la seguridad y tolerabilidad a largo plazo de dosis diarias repetidas de A4250
    • Evaluar el efecto de A4250 sobre el crecimiento
    • Evaluar el efecto de A4250 en la derivación biliar o en la lista de espera para un trasplante de hígado
    • Evaluar el efecto de A4250 en los marcadores bioquímicos de colestasis y hepatopatía
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Completion of the 24-week Treatment Period of Study A4250-005 or withdrawn from Study A4250-005 due to patient/caregiver judgment of lack of improvement/intolerable symptoms after completing at least 12 weeks of treatment
    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. Patients expected to have a consistent caregiver for the duration of the study
    4. Caregivers (and age appropriate patients) must be willing and able to use an eDiary device as required by the study
    1. Haber completado el período de tratamiento de 24 semanas del estudio A4250-005 o haberse retirado del estudio A4250-005 debido a la ausencia de mejoría/síntomas intolerables, a juicio del paciente o su cuidador, tras completar al menos una tanda de 12 semanas.
    2. Haber firmado el consentimiento informado y el asentimiento, según corresponda. Los pacientes que cumplan 18 años de edad (o la mayoría de edad que corresponda a cada país) durante el estudio deberán volver a firmar un consentimiento para permanecer en el estudio.
    3. Se espera que los pacientes tengan un mismo cuidador durante todo el transcurso del estudio.
    4. De acuerdo con los requisitos del estudio, los cuidadores (y los pacientes de la edad apropiada) deben estar dispuestos a usar un diario electrónico (eDiary) y ser capaces de hacerlo.
    E.4Principal exclusion criteria
    Patients meeting any of the following criteria at Visit 1 will not be eligible for study participation:
    1. Decompensated liver disease: coagulopathy, history, or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy
    2. Sexually active males and females who are not using a reliable contraceptive method with ≤1% failure rate (such as barrier protection, hormonal contraception, intra-uterine device, or complete abstinence) throughout the duration of the study and 90 days thereafter (from signed informed consent through 90 days after last dose of study drug).
    3. Patients not compliant with treatment in study A4250-005
    4. Any other conditions or abnormalities which, in the opinion of the investigator or Medical Monitor, may compromise the safety of the patient, or interfere with the patient participating in or completing the study
    Los pacientes que cumplan cualquiera de los criterios siguientes en la visita 1 no serán aptos para participar en el estudio:
    1. Hepatopatía descompensada: coagulopatía, antecedentes o presencia de ascitis clínicamente relevante, hemorragia por varices o encefalopatía
    2. Varones y mujeres sexualmente activos que no estén utilizando un método anticonceptivo fiable con ≤ 1 % de fallos (como los métodos de barrera, anticoncepción hormonal, dispositivo intrauterino o abstinencia total) durante toda la duración del estudio y durante los 90 días posteriores (desde la firma del consentimiento informado hasta 90 días después de la última administración del fármaco en estudio)
    3. Pacientes que no hayan cumplido con el tratamiento en el estudio A4250-005
    4. Cualquier otra patología o alteración que, en opinión del investigador o el monitor médico, pueda poner en peligro la seguridad del paciente o interferir con la participación del paciente en el estudio o impedir que lo finalice
    E.5 End points
    E.5.1Primary end point(s)
    The primary efficacy endpoint is:
    Change from baseline in s-BA after 72 weeks of treatment.
    El criterio principal de valoración de la eficacia es: cambio en los niveles de ABs con respecto a los valores basales tras 72 semanas de tratamiento.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Screening/ Inclusion Visit and week 72
    Selección/ Visita de inclusión y semana 72
    E.5.2Secondary end point(s)
    Secondary efficacy endpoints include:
    • All-cause mortality, number of patients undergoing biliary diversion
    surgery, or being listed for liver transplantation.
    • Change in growth after initiation of A4250 treatment
    • Change in AST to platelet ratio index (APRI) score and Fib-4 score
    from baseline to Week 72
    • Change in pediatric end-stage liver disease (PELD)/model for endstage
    liver disease (MELD) score from baseline to Week 72
    • Change in use of antipruritic medication
    Los Criterios secundarios de valoración de la eficacia incluyen:
    • Mortalidad por cualquier causa, número de pacientes sometidos a cirugía de derivación biliar o que se añaden a la lista de espera para trasplante de hígado.
    • Cambio en el crecimiento tras el inicio del tratamiento con A4250
    • Cambio en la puntuación del índice APRI (aspartato-aminotransferasa/plaquetas) y la puntuación FIB-4 entre el valor basal y la semana 72
    • Cambio en la puntuación para la hepatopatía pediátrica en etapa terminal (PELD —Pediatric End-stage Liver Disease)/modelo para la hepatopatía en fase terminal (MELD —Model for End-stage Liver Disease) entre el valor basal y la semana 72
    • Cambio en el uso de medicación antiprurítica
    E.5.2.1Timepoint(s) of evaluation of this end point
    • Weeks 24, 48, and 72
    Semanas 24, 48 y 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 Yes
    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 Yes
    E.6.13.1Other scope of the trial description
    Quality of Life Assessment (using PedsQL)
    Evaluación de calidad de vida (usando PedsQL)
    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 No
    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) No
    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 concerned2
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA20
    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 No
    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
    France
    Germany
    Israel
    Italy
    Netherlands
    Poland
    Saudi Arabia
    Spain
    Sweden
    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
    End of study in one country: last patient last visit (LPLV) and sites are closed.
    End of study globally: LPLV globally and all sites closed
    Final del estudio en un país: última visita del último paciente (UVUP), habiéndose cerrado los centros.
    Final del estudio global: UVUP en términos globales, habiéndose cerrado todos los centros
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months4
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months4
    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.1.1Number of subjects for this age range: 60
    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: 20
    F.1.1.5Children (2-11years) Yes
    F.1.1.5.1Number of subjects for this age range: 30
    F.1.1.6Adolescents (12-17 years) Yes
    F.1.1.6.1Number of subjects for this age range: 8
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 2
    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
    Minors unable to speak and read will be enrolled onto this study.
    Parents' consent will be sought.
    Los menores que no puedan hablar y leer serán inscritos en este
    estudio. Se solicitará el consentimiento de los padres.
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state2
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 29
    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)
    None
    Ninguno
    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 Decision2018-12-12
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2018-11-13
    P. End of Trial
    P.End of Trial StatusOngoing
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