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    Summary
    EudraCT Number:2011-006113-34
    Sponsor's Protocol Code Number:XAMNPIOAP2011
    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:2013-09-12
    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 number2011-006113-34
    A.3Full title of the trial
    Effect of pioglitazone administred to patients with Adrenomyeloneuropathy: A phase II, Singlearm, Monocentric Trial.
    Efecto de la pioglitazona administrada a pacientes con Adrenomieloneuropatia: ensayo unicéntrico, de brazo único, en fase II
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Effect of pioglitazone in patienst with Adrenomyeloneuropathy.
    Efecto de la pioglitazona en pacientes con Adrenomieloneuropatia.
    A.3.2Name or abbreviated title of the trial where available
    Pioglitazone in Adrenomyeloneuropathy
    Pioglitazona en Adrenomieloneuropatia
    A.4.1Sponsor's protocol code numberXAMNPIOAP2011
    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 SponsorAURORA PUJOL ONOFRE
    B.1.3.4CountrySpain
    B.3.1 and B.3.2Status of the sponsorNon-Commercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportFundación Hesperia
    B.4.2CountrySpain
    B.4.1Name of organisation providing supportELA Research Foundation
    B.4.2CountrySpain
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationIdibell (Institut d'Investigació Biomèdica de Bellvitge)
    B.5.2Functional name of contact pointAURORA PUJOL ONOFRE
    B.5.3 Address:
    B.5.3.1Street AddressGran Vía de l'Hospitalet, 199 3ª Planta
    B.5.3.2Town/ cityL'Hospitalet de Ll. (Barcelona)
    B.5.3.3Post code08908
    B.5.3.4CountrySpain
    B.5.4Telephone number349326075003332
    B.5.5Fax number34932607414
    B.5.6E-mailapujol@idibell.cat
    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.2.5The 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 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 INNPIOGLITAZONE
    D.3.9.1CAS number 111025-46-8
    D.3.9.4EV Substance CodeSUB09857MIG
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number30
    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
    X-linked adrenoleukodystrophy
    Adrenoleucodistrofia ligada al cromosoma X
    E.1.1.1Medical condition in easily understood language
    Adrenoleukodystrophy
    Adrenoleucodistrofia
    E.1.1.2Therapeutic area Diseases [C] - Nervous System Diseases [C10]
    MedDRA Classification
    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 goal of the trial is to show that pioglitazone treatment will provide clinical benefits in terms of functional capacity in the 20 enrolled AMN patients. Clinical benefits will be primarily measured by changes in the 6 Minutes Walking test, the primary efficacy endpoint.
    El objetivo principal del estudio es demostrar la eficacia clínica de la pioglitazona estimada en términos de
    capacidad funcional, en 20 pacientes con adrenoleucodistrofia, fenotipo AMN, sin afectación cerebral. Los beneficios clínicos se medirán primariamente por cambios en el test de la marcha de 6 minutos (6MWT), que es la variable principal de eficacia.
    E.2.2Secondary objectives of the trial
    The secondary goal of the trial is the assessment of efficacy and safety of the proposed treatment in 20 patients with AMN. Efficacy will be measured in terms of motor function, quality of life, neuroimagery, evoked potentials, ENG/EMG and biological markers. Safety will be evaluated by clinical, biological and brain imaging exams and recording of any adverse event.
    El objetivo secundario del ensayo es la valoración de la eficacia y la seguridad del tratamiento propuesto en los 20 pacientes AMN.
    La eficacia se medirá en términos de:
    - función motora: TUG test, SPRS y dinamómetro
    - calidad de vida: MSIS-29
    - neuroimagen: MRI con puntuación Loes, DTI y MRS
    - potenciales evocados: visuales, auditivos, somato-sensoriales, motores y laser
    - ENG/EMG: NCV, SNAP y CMAP
    - marcadores biológicos de oxidación: GSA, CEL, CML y MDAL
    - marcadores biológicos de inflamación ( ).
    La seguridad se evaluará con exámenes clínicos, biológicos e imágenes cerebrales y registro de efectos adversos.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Men and women of 18 to 65 years old, inclusive.Clinical signs of AMN with at least pyramidal signs in the lower limbs and difficulties to run.Presence of motor deficit according to the EDSS scale. Ability to perform the 6MWT
    Este estudio ambulatorio está dedicado a 20 pacientes varones AMN o mujeres heterocigotas, de entre 18 y 65 años de edad, sin la forma cerebral desmielinizante de la enfermedad. Deben presentar déficits motores según escala de discapacidad EDSS. En la práctica, los pacientes deben necesitar, en el peor de los casos, soporte unilateral permanente (pe. bastón) y ser capaces de realizar, como mínimo, la prueba de la marcha de 6 minutos.
    E.4Principal exclusion criteria
    Gadolinium enhancement on T1 sequence of any abnormal hypersignal of white matter, including myelinated pyramidal tracts, visible at brain MRI on FLAIR sequences.History of heart failure or cardiac disease.Prior or current bladder cancer. Women with history of osteoporosis. Gross hematuria of unknown origin.
    Enfermedad inflamatoria cerebral avanzada con trastorno cognitivo, y/o necesitar la ayuda de 2 bastones para caminar. Historia de enfermedad coronaria que requiera colocación de stens, bypass o infarto de miocardio previo. Haber padecido o padecer en la actualidad cáncer de vejiga o hematuria macroscopia no filiada. Mujeres con antecedentes de osteoporosis. La hematuria macroscópica de origen desconocido.
    E.5 End points
    E.5.1Primary end point(s)
    The main efficacy evaluation criterion is changes in the 6 Minute Walk Test (6MWT) between M0 and M24. The score at this test corresponds to the distance traveled by the patient during 6 minutes, on a flat surface. Details about the 6MWT are given in 9.1.
    El principal criterio para la evaluación de la eficacia será el Test de la marcha de 6 minutos (6 minutes walk test,
    6MWT) entre el inicio (M0) y el final del tratamiento (M24). La puntuación del test se corresponde con la distancia recorrida por el paciente durante 6 minutos, en una superficie plana.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Evaluation will be done at the beginning (M0) and at the end (M24) of the trial.
    La evaluación se realizará al inicio (M0) y al final (M24) del Ensayo.
    E.5.2Secondary end point(s)
    Secondary efficacy endpoints consist in:
    1) Tests evaluating the motor functions of treated AMN patients. They include: the Timed Up and Go (TUG) test, and evaluation of muscle strength by the use of a dynamoter on M0, M6, M12, M18 and M24 and the Spastic Paraplegia Rating Scale (SPRS) on M0, M12 and M24.
    2) Quality of life: score of the Multiple Sclerosis Impact Scale (MSIS?29) questionnaire on M0 and M24.
    3) Neuroimagery: a) brain abnormalities (hypersignal of pyramidal tracts, cerebellar and cerebral atrophy) on conventional MRI that will be assessed by the Loes score; b) diffusion tensor imaging (DTI) parameters in the cervical spinal cord and brain; c) and magnetic resonance spectroscopy (MRS) parameters in the cerebral white matter on M0 and M24. Tests performed in the Hospital Universitari de Bellvitge during the period of one year before the start of the trial will be considered valid.
    4) Evoked potentials: visual, auditory, somatosensory, motor and laser evoked potentials will be studied at the beginning (M0) and at the end of the trial (M24). Tests performed in the Hospital Universitari de Bellvitge during the period of one year before the start of the trial will be considered valid.
    5) ENG/EMG: nerve conduction velocity (NCV), amplitude of sensory potential (SNAP) in sensory nerves and motor evoked potential amplitude (CMAP) in motor nerves of the upper and lower extremities will be recorded on M0 and M24. Tests performed in the Hospital Universitari de Bellvitge during the period of one year before the start of the trial will be considered valid.
    6) Biological measures:
    - Markers of oxidative stress: GSA, CEL, MDAL, and CML in plasma and/or peripheral blood mononuclear cells and cerebrospinal fluid (only in patients who voluntarily agree) and 8oxo-dG in urine. Markers in plasma/PBMC/urine will be measured on M0, M6 and M24. Markers in CSF will be measured on M0 and M24.
    - Markers of inflammation: CCR3, CXCL5, CXCL9, IL9R, PPARd, GPX4 and STAT1 in RNA obtained from MNC and HGF, IL6, IL8, MCP-1, NGF, TNF and adiponectin in plasma.
    Variables secundarias:
    1) Evaluación de la función motora en pacientes con AMN. Incluyen: Prueba cronometrada de ?levántate y anda? (Timed Up and Go test, TUG), Evaluación de la fuerza muscular utilizando un dinamómetro (M0,M6,M12, M18 y M24) y Valoración de la Escala de puntuación de paraparesia espástica (Spastic Paraplegia Rating Scale, SPRS) (M0,M12 y M24).
    2) Calidad de vida. Cuestionario de calificación con la escala de impacto de la Esclerosis Múltiple (Multiple
    Sclerosis Impact Scale, MSIS-29) en M0 y M24.
    3) Estudio neurológico por la imagen:a) Anomalías cerebrales (hiperseñal de tractos piramidales, atrofia cerebral y cerebelar) por resonancia magnética
    convencional (MRI) que serán valoradas con la puntuación Loes a M0 y M24.Imágenes con tensor de difusión (DTI) en la médula espinal cervical y cerebro. Resonancia magnética espectroscópica (MRS) en materia blanca cerebral.
    4)Estudios de conducción nerviosa en extremidades superiores e inferiores:Velocidad de
    conducción nerviosa (NCV), amplitud del potencial sensorial (SNAP) en nervios sensitivos y amplitud de potencial evocado motor (CMAP) en nervios motores.
    5) Potenciales evocados:Potenciales evocados visuales (VEP), auditivos (BEP), somatosensoriales (SEP), motores (MEP) y laser (LEP).
    6) Determinaciones biológicas:
    -Marcadores de estrés oxidativo en proteínas: GSA, CEL, MDAL y CML en plasma y/o células mononucleares de sangre periférica. Estos marcadores varían independientemente el uno del otro. La cuantificación se realizará por espectroscopia de masas y la concentración se expresará como ?mol por mol de lisina.
    -Marcadores de estrés oxidativo en DNA: 8oxoDG en orina. La cuantificación se realizará por HPLC-ECD y la
    concentración se expresará como ng de 8-oxoDG/mg creatinina. El análisis se realizará en el laboratorio de
    enfermedades neurometabólicas del IDIBELL.
    -Marcadores de inflamación: Se cuantificará, en MNC por Q-PCR, las siguientes citoquinas: CCR3, CXCL5, CXCL9, IL9R, PPARd, GPX4, STAT1 (IDIBELL). En el Departamento de Bioquímica de la UB se cuantificará las siguientes adipoquinas por tecnología Milliplex en plasma: HGF, IL6, IL8, MCP-1, NGF, TNF, adiponectina.
    E.5.2.1Timepoint(s) of evaluation of this end point
    1) Timed Up and Go (TUG) test, and evaluation of muscle strength by the use of a dynamoter on M0, M6, M12, M18 and M24 and the Spastic Paraplegia Rating Scale (SPRS) on M0, M12 and M24.
    2) Quality of life: MSIS?29 on M0 and M24.
    3) Neuroimagery: MRI, DTI, MRS on M0 and M24.
    4) Evoked potentials: M0 and M24
    5) ENG/EMG: M0 and M24
    6) Biological measures:
    - Markers of oxidative stress: on M0, M6 and M24 (markers in CSF will be measured on M0 and M24)
    - Markers of inflammation: on M0, M6 and M24
    1) Prueba cronometrada de ?levántate y anda? y evaluación de la fuerza muscular con dinamómetro en M0, M6, M12, M18 y M24; escala de puntuación de paraparesia espástica (Spastic Paraplegia Rating Scale, SPRS) en M0, M12 y M24.
    2) Calidad de vida. MSIS-29 en M0 y M24.
    3) Estudio neurológico por la imagen:MRI, DTI, MRS en M0 y M24
    4)Estudios de conducción nerviosa en extremidades superiores e inferiores: M0 y M24
    5) Potenciales evocados: M0 y M24
    6) Determinaciones biológicas:
    -Marcadores de estrés oxidativo en proteínas: en M0, M6 y M24. (Marcadores CSF en M0 y M24)
    -Marcadores de inflamación: en M0, M6 y M24.
    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 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
    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 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: 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 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 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 Decision2013-10-24
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2013-09-09
    P. End of Trial
    P.End of Trial StatusOngoing
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