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    Summary
    EudraCT Number:2014-001586-27
    Sponsor's Protocol Code Number:SOMCT01-C
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Prematurely Ended
    Date on which this record was first entered in the EudraCT database:2014-06-02
    Trial results View 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 number2014-001586-27
    A.3Full title of the trial
    Study of SOM0226 in familial amyloid polyneuropathy (FAP) patients and asymptomatic carriers to evaluate protein stabilization activity
    Estudio de SOM0226 en pacientes con polineuropatía amiloide familiar (FAP) y portadores asintomáticos para evaluar la estabilización de la proteina TTR.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Study of SOM0226 in familial amyloid polyneuropathy (FAP) patients and asymptomatic carriers to evaluate protein stabilization activity
    Estudio de SOM0226 en pacientes con polineuropatía amiloide familiar (FAP) y portadores asintomáticos para evaluar la estabilización de la proteina TTR.
    A.4.1Sponsor's protocol code numberSOMCT01-C
    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 SponsorSOM Innovation Biotech SL (SOM Biotech)
    B.1.3.4CountrySpain
    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 supportFundació Vall d'Hebron-Institut de Recerca
    B.4.2CountrySpain
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationARO-VHIR
    B.5.2Functional name of contact pointMiriam García Diez
    B.5.3 Address:
    B.5.3.1Street AddressPasseig de la Vall d?Hebron, 119-129
    B.5.3.2Town/ cityBarcelona
    B.5.3.3Post code08035
    B.5.3.4CountrySpain
    B.5.4Telephone number+349348930002703
    B.5.6E-mailmiriam.garcia@vhir.org
    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 Yes
    D.2.1.1.1Trade name Tasmar
    D.2.1.1.2Name of the Marketing Authorisation holderMeda AB
    D.2.1.2Country which granted the Marketing AuthorisationSpain
    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.3.4Pharmaceutical form Coated tablet
    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 INNTOLCAPONE
    D.3.9.1CAS number 134308-13-7
    D.3.9.2Current sponsor codeSOM0226
    D.3.9.4EV Substance CodeSUB11151MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number100
    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
    Patients with familial amyloid polyneuropathy (FAP), asymptomatic carriers and healthy volunteers
    Pacientes con polineuropatía amiloide familiar (FAP), portadores asintomáticos y voluntarios sanos
    E.1.1.1Medical condition in easily understood language
    Patients with familial amyloid polyneuropathy (FAP), asymptomatic carriers and healthy volunteers
    Pacientes con polineuropatía amiloide familiar (FAP), portadores asintomáticos y voluntarios sanos
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 17.0
    E.1.2Level LLT
    E.1.2Classification code 10057949
    E.1.2Term Familial amyloid polyneuropathy
    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
    Determine whether SOM0226 induces TTR stabilization by evaluating whether treatment with SOM0226 prevents urea-induced denaturation of tetrameric TTR.
    Determinar si SOM0226 (tolcapona) induce la estabilización de TTR en voluntarios sanos, en pacientes con polineuropatía amiloide familiar (FAP) y en pacientes portadores asintomáticos, impidiendo la desnaturalización de TTR inducida por urea.
    E.2.2Secondary objectives of the trial
    - The secondary objective is to determine the plasma concentration of SOM0226 needed to induce a maximal stabilization of TTR in healthy volunteers, patients and asymptomatic carriers.
    - A pharmacodynamics assessment will be used to determine an effective dose of SOM0226 to confer TTR stabilization, by determining the molar ratio SOM0226:TTR that must be reached in plasma to induce maximal stabilization of TTR in healthy individuals, patients and asymptomatic carriers.
    Como objetivos secundarios se ha establecido determinar la concentración de fármaco en plasma necesaria para inducir la máxima estabilización de TTR en voluntarios sanos, pacientes con FAP y en portadores asintomáticos, así como la dosis efectiva de SOM0226 para la estabilización de TTR determinada por el ratio molar SOM0226:TTR.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Participant is willing and able to give informed consent for participation in the study.
    - Male or Female, aged 18 years or above at time of consent.
    - Two cohorts defined by TTR variant:
    Wild type TTR: healthy patients
    V30M TTR mutant: Asymptomatic carriers with documented
    mutation V30M TTR or diagnosed TTR-FAP patients (stage 1 or
    2) not undergoing pharmacological treatment with Vyndaqel®.
    - Body Mass Index (BMI) > 17.5 kg/m2
    - Female participants of child bearing potential and male participants whose partner is of child bearing potential must be willing to ensure that they or their partner use effective contraception during the study and for 2 month thereafter.
    - Able (in the Investigators opinion) and willing to comply with all study requirements.
    - Pacientes que estén dispuestos y sean capaces de dar su consentimiento para participar en el estudio
    - Pacientes de ambos sexos, que en el momento del consentimiento sean mayores o iguales a 18 años
    - 2 cohortes definidas por la variante de TTR:
    TTR tipo salvaje: voluntarios sanos
    Pacientes con la mutación V30M en la proteína TTR:
    portadores asintomáticos de la mutación de TTR V30M o
    pacientes diagnosticados de FAP-TTR (estadios 1 o 2) que no
    estén siendo tratados con Vyndaqel®.
    - Índice de masa corporal (IMC) > 17.5 kg/m2
    - Mujeres en edad fértil u hombres cuya pareja sexual femenina esté en edad fértil, deben asegurar que usarán un método anticonceptivo eficaz durante el estudio y en los 2 meses después de su finalización.
    - Pacientes que en opinión del investigador puedan completar los requerimientos del protocolo.
    E.4Principal exclusion criteria
    The participant may not enter the study if ANY of the following apply:
    - Female participants who is pregnant, lactating or planning pregnancy during the course of the study.
    - Evidence of history of clinically significant hepatic disease.
    - An ALT or AST measurement > 2 times the ULN (Upper Limit of Normal).
    - Scheduled elective surgery or other procedures requiring general anaesthesia during the study.
    - Any other significant disease or disorder which, in the opinion of the Investigator, may either put the participants at risk because of participation in the study, or may influence the result of the study, or the participant?s ability to participate in the study.
    - Donation of blood during the study or within the past 4 weeks.
    - Treatment (during the study or within the past 4 weeks) with a prescription or investigational drug for the treatment of TTR amyloidosis.
    - Treatment with NSAIDs during the study or within the past 4 weeks. The following NSAID are allowed: acetylsalicylic acid, etodolac, ibuprofen, indomethacin, ketoprofen, nabumetone, naproxen, nimesulide, piroxicam and sulindac
    - Mujeres que estén embarazadas o amamantando o planeen quedarse embarazadas durante el estudio.
    - Evidencia en la historia clínica de enfermedades hepáticas relevantes.
    - Valores de ALT o AST por encima de 2 veces el valor máximo normal.
    - Cirugía electiva programada o cualquier otro procedimiento que necesite anestesia general durante el transcurso del estudio.
    - Cualquier otra enfermedad o trastorno significativo que, en opinión del investigador, podrían poner a los participantes en situación de riesgo debido a la participación en el estudio, o pueden influir en el resultado del estudio, o la capacidad del participante para participar en el estudio
    - Donar sangre durante el estudio o en las últimas 4 semanas.
    - Durante el estudio o en las 4 semanas anteriores usar un tratamiento por prescripción o con un producto en investigación para la amiloidosis TTR.
    - Tratamiento con AINES durante el estudio o en las últimas 4 semanas. Los siguientes AINES sí están permitidos: ácido acetilsalicílico, etodolaco, ibuprofeno, indometacina, ketoprofeno, nabumetona, naproxeno, nimesulida, piroxicam y sulindaco.
    E.5 End points
    E.5.1Primary end point(s)
    TTR stabilization will be measured by determining the concentration of tetrameric TTR after denaturation. The ratio of TTR tetramer after denaturation / TTR tetramer before denaturation of plasma samples from treated subjects will be compared to their baseline ratio. TTR stabilization will be determined as percentage of the baseline ratio.

    TTR stabilization will be measured in plasma samples obtained from each participant before first dose and at different times during each treatment period. In terms of assessing efficacy, a positive result will be a TTR stabilization value superior to 20%.
    La estabilización de TTR se mide mediante la determinación de la concentración de TTR tetramérica después de la desnaturalización. La relación de TTR tetrámero después de desnaturalización versus TTR antes de la desnaturalización de las muestras de plasma de los sujetos tratados, se comparará con su relación de línea de base. La estabilización de TTR se determina como porcentaje de la relación de la línea de base.

    La estabilización de TTR se mide en muestras de plasma obtenidas de cada participante antes de la primera dosis y en diferentes momentos durante cada período de tratamiento. En términos de evaluación de la eficacia, un resultado positivo será un valor de estabilización TTR superior a 20%.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Phase A (24 hours) and Phase B (32 hours). Between these two phases there is a washout period of 6 weeks (+-2 weeks)
    Fase A (24 horas) y Fase B (32 horas). Entre estas dos fases hay un periodo de lavado de 6 semanas (+- 2 semanas)
    E.5.2Secondary end point(s)
    - The pharmacodynamics characteristics of SOM0226 will be assessed to determine which plasma SOM0226:TTR molar ratio is needed to reach a plateau of TTR stabilization.
    - The concentration of SOM0226 will be determined in all
    plasma samples obtained at different times during each
    treatment period.
    - The concentration of TTR will be determined in all
    plasma samples, and the molar ratio SOM0226:TTR in
    each sample will be calculated.
    - Safety will be assessed by ALT and AST liver function measures in samples obtained at the end of each treatment phase (24 hours in Phase A and 32 hours in Phase B).
    - Las características farmacodinámicas de SOM0226 serán evaluadas para determinar qué relación molar plasma SOM0226:TTR en plasma es necesaria para obtener la máxima estabilización de TTR.
                         - Se determinará la concentración de SOM0226 en todas
    las muestras de plasma obtenidas en diferentes
    tiempos durante cada período de tratamiento.
                         - Se determinará la concentración de TTR en todas las
    muestras de plasma, y la relación molar SOM0226:TTR se
    calculará en cada muestra.
    - La seguridad se evaluará por medidas de la función hepática (ALT y AST) en muestras obtenidas al final de cada fase de tratamiento (24 horas en la fase A y 32 horas en la fase B).
    E.5.2.1Timepoint(s) of evaluation of this end point
    Phase A (24 hours) and Phase B (32 hours). Between these two phases there is a washout period of 6 weeks (+-2 weeks)
    Fase A (24 horas) y Fase B (32 horas). Entre estas dos fases hay un periodo de lavado de 6 semanas (+- 2 semanas)
    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 Yes
    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) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    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.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.5The trial involves multiple Member States No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA No
    E.8.6.2Trial being conducted completely outside of the EEA No
    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 included
    - There is evidence of an unacceptable risk for trial subjects
    - There is reason to conclude that it will not be possible to collect the data necessary to reach the study objectives and it is therefore not ethical to continue enrollment of more patients
    - If during part A, some fatal safety events related to the investigational product are detected, part B will be cancelled.
    - NVLS incluido
    - Hay evidencia de un riesgo inaceptable para los sujetos del ensayo
    - Hay razones para concluir que no será posible recoger los datos necesarios para alcanzar los objetivos del estudio y por lo tanto no es ético continuar la inclusión de más pacientes
    - Si durante la parte A, se detectan algunos eventos de seguridad fatales relacionadas con el producto en investigación, se cancelará la parte B.
    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 months2
    E.8.9.1In the Member State concerned days7
    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: 20
    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 Yes
    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 state20
    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 Decision2014-07-25
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-06-27
    P. End of Trial
    P.End of Trial StatusPrematurely Ended
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