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    Summary
    EudraCT Number:2019-004733-17
    Sponsor's Protocol Code Number:XAMNPEAP2019
    National Competent Authority:Spain - AEMPS
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2019-12-20
    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 number2019-004733-17
    A.3Full title of the trial
    Effect of plasma exchange by albumin replacement in Adrenomyeloneuropathy: unicentric, single arm, proof of concept trial
    Efecto del recambio plasmático con albúmina en pacientes con Adrenomieloneuropatía: estudio unicéntrico, de brazo único, en prueba de concepto
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Plasma exchange by albumin replacement in Adrenomyeloneuropathy
    Estudio del recambio plasmático de albúmina en pacientes con adrenomieloneuropatía
    A.4.1Sponsor's protocol code numberXAMNPEAP2019
    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 supportAlbus Award Grifols Scientific Awards
    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 Via de l'Hospitalet 199
    B.5.3.2Town/ cityL'Hospitalet de Llobregat
    B.5.3.3Post code08908
    B.5.3.4CountrySpain
    B.5.4Telephone number0034932607137
    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.2.1.1.1Trade name Albunorm® 5%,
    D.2.1.1.2Name of the Marketing Authorisation holderOctapharma S.A.
    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.3.1Product nameAlbumin
    D.3.4Pharmaceutical form Solution for solution for infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNALBUMIN
    D.3.9.3Other descriptive nameALBUMIN
    D.3.9.4EV Substance CodeSUB20532
    D.3.10 Strength
    D.3.10.1Concentration unit millilitre(s)/kilogram
    D.3.10.2Concentration typerange
    D.3.10.3Concentration number35 to 45
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    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 Yes
    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
    Adrenomyeloneuropathy
    Adrenomieloneuropatia (AMN)
    E.1.1.1Medical condition in easily understood language
    Adrenomyeloneuropathy
    Adrenomieloneuropatia (AMN)
    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 20.0
    E.1.2Level LLT
    E.1.2Classification code 10069075
    E.1.2Term Adrenomyeloneuropathy without cerebral involvement
    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 goal of the trial is to show that treatment with plasma exchange plus therapeutic albumin replacement in patients with AMN induces a decrease of plasma and cerebrospinal fluid VLCFA.
    El objetivo principal del ensayo es demostrar que el tratamiento con plasmaféresis más el reemplazo terapéutico de albúmina en pacientes con AMN induce una disminución de los VLCFA del plasma y del líquido cefalorraquídeo.
    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 5 patients with AMN. Efficacy will be measured in terms of motor function and quality of life. Only in case of a decrease in VLCFA other biological markers (oxidation and inflammation biomarkers) will also be studied. Safety will be evaluated by clinical and biochemical exams and recording of any adverse event.
    El objetivo secundario del ensayo es la valoración de la eficacia y seguridad del tratamiento propuesto en 5 pacientes con AMN. Mediremos la eficacia en términos de función motora y calidad de vida. Estudiaremos también otros marcadores biológicos (marcadores de oxidación e inflamación) únicamente en el caso de una disminución de los VLCFA. Evaluaremos la seguridad con exámenes clínicos y bioquímicos y registraremos los efectos secundarios
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    1. Men of 18 to 65 years old, inclusive
    2. Elevated plasma VLCFA and gene mutation identified
    3. Clinical signs of AMN with at least pyramidal signs in the lower limbs and difficulties to run
    4. Presence of motor deficit according to the EDSS scale
    5. Ability to perform the 2MWT
    6. Normal brain MRI or brain MRI showing the following abnormalities that can be observed in AMN patients without cerebral form of X‐ALD, obtained in the 6 months prior to screening:
    •abnormal hyperintensity of pyramidal tract fibers in the brain stem on FLAIR or T2 sequence
    •abnormal hyperintensity of pyramidal tract fibers in the internal capsules on FLAIR or T2 sequence
    •cerebellar atrophy
    •moderate cortical atrophy
    7. Appropriate steroid replacement if adrenal insufficiency is present
    8. Signed Informed consent
    9. Affiliated to the Spanish Public Health System
    1. Varones de 18 a 65 años, ambos incluidos
    2. VLCFA elevados en plasma e identificada la mutación en el gen.
    3. Signos clínicos de AMN con signos piramidales en las extremidades inferiores y dificultades para correr como mínimo
    4. Presencia de déficit motor según escala EDSS
    5. Aptitud para realizar el test 2MWT
    6. RMN cerebral normal o RMN cerebral con las siguientes anormalidades que pueden observarse en pacientes sin la forma cerebral de AMN, obtenida durante los 6 meses anteriores a la visita de selección:
    - hiperintensidad anormal de las fibras de los tractos piramidales en el tronco cerebral en secuencias FLAIR o T2
    - atrofia cerebelar
    - atrofia cortical moderada
    7. Reemplazo de esteroides adecuado en caso de insuficiencia adrenal
    8. Consentimiento informado firmado
    9. Afiliado al Sistema de Salud Público Español
    E.4Principal exclusion criteria
    1. Any contraindication for plasma exchange due to behavioral disorders or abnormal coagulation parameters, such for example:
    - Hypocalcemia (Ca++ < 8.7 mg/dl)
    - Thrombocytopenia (< 100.000/µl)
    - Fibrinogen < 1.5 g/l
    - Prothrombin time (Quick) p< 60% versus control (INR > 1.5)
    - Beta-blocker treatment and bradycardia < 55/min
    - Treatment with ACIs (increased risk of allergic reactions)
    2. Hemoglobin < 10 g/dl
    3. Difficult venous access precluding plasma exchange
    4. A history of frequent adverse reactions (serious or otherwise) to blood products
    5. Hipersensibility to albumin o allergies to any of the components of Albunorm® 5%
    6. Plasma creatine > 2 mg/dl
    7. Uncontrolled high blood pressure (systolic blood pressure of 160 mmHg or higher and/or diastolic blood pressure of 100 mmHg or higher despite regular treatment during the last 3 months)
    8. Liver cirrhosis or any liver problem with GPT > 2.5 x ULN, or bilirubin > 2 mg/dl
    9. Heart diseases as evidenced by myocardial infarction, severe or unstable angina, or heart failure in the past 12 months
    10. Gadolinium enhancement on T1 sequence of any abnormal hypersignal of white matter, including myelinated pyramidal tracts, visible at brain MRI on FLAIR sequences
    11. Significant peripheral edema (2+ or more on the Assessment Chart for Pitting Edema) of the extremities of any etiology
    12. Any evolutive malignancy during the last five years or any condition complicating adherence to the study protocol
    13. Smokers (one pack/ day or more for at least 20 years), current or former
    14. Any psychiatric disease
    15. Present participation to another therapeutic clinical trial for X-ALD, or the receipt of any other investigational drug in the three months prior to the start of the study
    16. Patients being treated with anticoagulants or antiplatelet therapy
    17. Not easily contactable by the investigator in case of emergency or not capable to call the investigator
    1. Cualquier contraindicación para el recambio plasmático debido a trastornos del comportamiento o parámetros de coagulación anormales, tales como:
    - Hipocalcemia (Ca++ < 8.7 mg/dl)
    - Trombocitopenia (< 100.000/μl)
    - Fibrinógeno < 1.5 g/l
    - Tiempo de protrombina (Quick) p<60% versus control (INR) > 1.5)
    - Tratamiento con beta-bloqueantes y bradicardia < 55/min
    - Tratamiento con ACIs (riesgo aumentado de reacciones alérgicas)
    2. Hemoglobina < 10 g/dl
    3. Dificultad de acceso a las venas que impida el recambio plasmático
    4. Historial de reacciones adversas frecuentes (serias o de otro tipo)
    5. Hipersensibilidad a la albúmina o alergias a alguno de los componentes de Albunorm® 5%
    6. Creatina plasmática > 2 mg/dl
    7. Presión sanguínea alta no controlada (presión sistólica de 160 mmHg o superior y/o presión diastólica de 100 mmHg o superior, a pesar de tratamiento regular durante los últimos 3 meses)
    8. Cirrosis hepática o algun problema hepático con GPT > 2.5 x ULN o bilirrubina > 2 mg/dl
    9. Enfermedades cardíacas tales como infarto de miocardio severo o angina de pecho inestable, o insuficiencia cardíaca en los últimos 12 meses
    10. Captación de gadolinio en secuencia T1 de cualquier hiperseñal anormal de sustancia blanca, incluyendo tractos piramidales mielinizados, visible en RMN cerebral en secuencias FLAIR
    11. Edema periférico significativo (2+ o más en el modelo de evaluación para el edema con fóvea) de las extremidades de cualquier etiología
    12. Alguna neoplasia evolutiva durante los últimos 5 años o cualquier otro trastorno que complique el cumplimiento del protocolo del estudio
    13. Fumadores (un paquete/día o más durante los últimos 20 años), actual o anterior
    14. Alguna enfermedad psiquiátrica
    15. Estar participando en otro ensayo clínico para pacientes X-ALD, o haber recibido cualquier otro fármaco en investigación durante los tres meses anteriores al inicio del estudio
    16. Pacientes que estén siendo tratados con anticoagulantes o antiplaquetarios
    17. Sujeto no fácilmente localizable por el investigador en caso de emergencia o incapaz de llamar al investigador.
    E.5 End points
    E.5.1Primary end point(s)
    The main efficacy evaluation criterion is changes in plasma VLCFA between M0 and M6. C26:0, C24:0 and C26:0/C22:0 will be measured in plasma.
    El principal criterio de evaluación de la eficacia son cambios en los VLCFA plasmáticos entre M0 y M6. Mediremos C26:0, C24:0 y 26:0/C22:0 en plasma.
    E.5.1.1Timepoint(s) of evaluation of this end point
    The assessment of the main variable is in month 6.
    La valoración de la variable principal es al mes 6.
    E.5.2Secondary end point(s)
    Efficacy endpoints:

    1) Tests evaluating the motor functions of treated AMN patients. They include: the Timed Up and Go (TUG) test, the Time to walk 25 feet (TW25), the 2 minute walk test (2MWT) and the 6 minute walk test (6MWT) on M0, M6 and M12.
    2) Quality of life: score of SF-Qualiveen on M0, M6 and M12.
    3) VLCFA in CSF (optional): C26:0, C24:0 and C26:0/C22:0 will be measured in CSF in those patients with AMN who voluntarily accept to undergo a lumbar puncture on M0, M6 and M12.
    4) Biological measures (only in case of a decrease in VLCFA):
    - Marker of oxidative stress: 8oxo-dG in urine. 8oxo-dG will be measured on M0, M6 and M12.
    - 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 will be measured on M0, M6 and M12.

    Safety endpoints:

    Safety will be evaluated by clinical and biochemical exams and recording of any adverse event.
    Variables de eficacia:

    1) Tests de evaluación de las funciones motoras de los pacientes AMN tratados. Incluyen: prueba cronometrada de "levántate y anda" (TUG), tiempo empleado para caminar 25 pies (TW25), prueba de caminata de 2 minutos (2MWT) y prueba de caminata de 6 minutos (6MWT) a M0, M6 y M12
    2) Calidad de vida: puntuación SF-Qualiveen a M0, M6 y M12
    3) VLCFA en LCR (opcional): mediremos C26:0, C24:0 and C26:0/C22:0 en LCR en aquellos pacientesAMN que acepten voluntariamente someterse a una punción lumbar en M0, M6 y M12
    4) Medidas biológicas: (sólo en el caso de disminución de VLCFA):
    - Marcadores de estrés oxidativo: 8oxo-dG en orina. Mediremos 8oxo-dG a M0, M6 y M12
    - Marcadores de inflamación: CCR3, CXCL5, CXCL9, IL9R, PPARd, GPX4 y STAT1 en RNA obtenido a partir de MNC y HGF, IL6, IL8, MCP-1, NGF, TNF y adiponectina en plasma a M0, M6 y M12

    Variables de seguridad:

    Evaluaremos la seguridad mediante exámenes clínicos y bioquímicos y registraremos los acontecimientos adversos
    E.5.2.1Timepoint(s) of evaluation of this end point
    The assessment of the secondary endpoints are in month 0, 6 i 12 (M0, M6, M12)
    La valoración de las variables secundarias son al mes 0, 6 i 12 (M0, M6, M12)
    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 No
    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
    Last visit last subject
    Último paciente última visita
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years
    E.8.9.1In the Member State concerned months13
    E.8.9.1In the Member State concerned days
    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: 5
    F.1.3Elderly (>=65 years) No
    F.2 Gender
    F.2.1Female No
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations No
    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 No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state5
    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)
    Patients at the end of the trial treatment will continue treatment according to usual clinical practice.
    Los pacientes al finalizar el tratamiento del ensayo continuaran tratamiento según práctica clínica habitual.
    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 Decision2020-02-25
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2020-01-30
    P. End of Trial
    P.End of Trial StatusCompleted
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