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    Summary
    EudraCT Number:2014-000698-38
    Sponsor's Protocol Code Number:MD1003CT2014-01AMN
    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:2014-06-13
    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 number2014-000698-38
    A.3Full title of the trial
    MD1003 IN ADRENOMYELONEUROPATHY: A RANDOMIZED DOUBLE BLIND PLACEBO CONTROLLED STUDY
    MD1003 en la adrenomieloneuropatía: un estudio aleatorizado con doble enmascaramiento controlado por placebo
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    MD1003 IN ADRENOMYELONEUROPATHY
    A.3.2Name or abbreviated title of the trial where available
    MD1003-AMN
    A.4.1Sponsor's protocol code numberMD1003CT2014-01AMN
    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 SponsorMEDDAY SAS
    B.1.3.4CountryFrance
    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 supportMEDDAY SAS
    B.4.2CountryFrance
    B.4.1Name of organisation providing supportELA L'Association Européenne contre les Leucodystrophies
    B.4.2CountryFrance
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationDra Aurora Pujol
    B.5.2Functional name of contact pointProfesor de Investigación ICREA
    B.5.3 Address:
    B.5.3.1Street AddressHospital Duran y Reinals, IDIBELL
    B.5.3.2Town/ cityL'Hospitalet de Llobregat, Barcelona
    B.5.3.3Post code08098
    B.5.3.4CountrySpain
    B.5.4Telephone number0034932607137
    B.5.5Fax number0034932607414
    B.5.6E-mailcontact@medday-pharma.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 No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product namebiotin
    D.3.2Product code MD1003
    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 INND-BIOTIN
    D.3.9.2Current sponsor codeMD1003
    D.3.9.4EV Substance CodeSUB32335
    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
    D.8 Placebo: 1
    D.8.1Is a Placebo used in this Trial?Yes
    D.8.3Pharmaceutical form of the placeboCapsule, hard
    D.8.4Route of administration of the placeboOral use
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Adrenomyeloneuropathy
    Adrenomieloneuropatía
    E.1.1.1Medical condition in easily understood language
    Adrenomyeloneuropathy
    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 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
    To demonstrate the superiority of MD1003 (biotin) at 300 mg/day over placebo in the clinical improvement of patients with Adrenomyeloneuropathy
    Demostrar la mayor eficacia de MD1003 respecto del placebo para la mejora clínica de pacientes con AMN.
    E.2.2Secondary objectives of the trial
    To evaluate safety of MD1003 in patients with Adrenomyeloneuropathy
    Evaluar la seguridad de MD1003 en el tratamiento de la AMN.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Men of 18 to 60 years old, inclusive
    - ABCD1 gene mutation identified
    - Elevated plasma VLCFA
    - Clinical signs of AMN with at least pyramidal signs in the lower limbs and difficulties to walk
    - EDSS score ? 3.5 and ? 6.5
    - Normal brain MRI or brain MRI showing :
    - abnormalities that can be observed in AMN patients without cerebral demyelination with a maximum Loes score of 4
    - and/or stable (?6 months) cerebral demyelination without gadolinium enhancement with a Loes score ?12.
    - Appropriate steroid replacement if adrenal insufficiency is present
    - Likely to be able to participate in all scheduled evaluation visits and complete all required study procedures
    - Signed and dated written informed consent to participate in the study in accordance with local regulations
    - Affiliated to a Health Insurance
    ° Hombres de 18 a 60 años con AMN:
    - mutación de ABCD1 identificada;
    - elevadas concentraciones plasmáticas de AGCML (ácidos grasos de cadena muy larga).
    ° Signos clínicos de AMN con al menos un síndrome del piramidal en las extremidades inferiores y dificultad para caminar.
    ° Puntuación de entre 3,5 y 6,5 en la escala EDSS (Escala Expandida del Estado de Discapacidad).
    ° IRM cerebral normal o IRM cerebral que muestre:
    (a) anomalías que pudiera observarse en pacientes con AMN sin desmielinización cerebral y con puntuación máxima de 4 en la escala de Loes; y/o
    (b) desmielinización estable (≥ 6 meses) sin captación de gadolinio con puntuación ≤ 12 en la escala de Loes.
    ° Sustitución adaptada con esteroides en caso de insuficiencia surrenal.
    ° Capacidad de realizar todas las evaluaciones previstas.
    ° Firma del consentimiento informado.
    E.4Principal exclusion criteria
    - Brain MRI abnormalities with a Loes score > 12 or with gadolinium enhacement
    - Any progressive neurological disease other than AMN
    - Impossibility to perform the walk tests and the TUG test
    - Patients with uncontrolled hepatic disorder, renal or cardiovascular disease, or any evolutive malignancy
    - Any new medication for AMN including Fampridine initiated less than 1 month prior to inclusion
    - Contraindications for MRI procedure such as subjects with paramagnetic materials in the body, such as aneurysm clips, pacemakers, intraocular metal or cochlear implants.
    - Inclusion in another therapeutic clinical trial for ALD
    - Patient not easily reachable by the investigator in case of emergency or not capable to call the investigator
    ° IRM cerebral anómala con puntuación en la escala de Loes > 12 o con captación de gadolinio.
    ° Cualquier neuropatía distinta de la AMN.
    ° Incapacidad de realizar las pruebas de marcha y la prueba funcional de TUG.
    ° Enfermedad hepática, renal o cardiovascular, o cualquier patología maligna evolutiva.
    ° Cualquier nuevo tratamiento para la AMN en que se haya iniciado la administración de fampridina menos de un mes antes de la inclusión.
    ° Pacientes en que la IRM esté contraindicada.
    ° Inclusión en otro ensayo para el tratamiento de la AMN.
    ° Difícil acceso al investigador en caso de urgencia o imposibilidad de acudir al investigador durante el estudio en general.
    E.5 End points
    E.5.1Primary end point(s)
    Mean change of 2 minutes walking test (2MWT) between M12 and baseline
    variación de 2 minutos en la prueba de M12 respecto del valor inicial.
    E.5.1.1Timepoint(s) of evaluation of this end point
    Baseline, 12 months, 24 months
    Valor inicial, 12 meses y 24 meses
    E.5.2Secondary end point(s)
    - Proportion of patients with improved 2-Minutes-Walk-Tests (2MWT) of at least 20% at M9 and M12 compared to the best value among screening and baseline?
    - Proportion of patients with improved TW25 (time to walk 25 feet) of at least 20% at M9 and M12 compared to the best value among screening and baseline
    - Timed up and Go test (TUG)
    - Euroqol EQ-5D questionnaire
    - Qualiveen to evaluate urinary function
    - To evaluate the safety of high dose of biotin assessed by: adverse events, clinical examination (vital signs, ECG), standard laboratory tests (hematology, biochemistry)
    Exploratory studies (optional):
    1) Brain MRI with conventional and non conventional sequences:
    a) diffusion tensor imaging (DTI) parameters in the upper cervical spinal cord and brain; b) magnetic resonance spectroscopy (MRS) in the cerebral white matter.
    2) Evaluation of muscle strength by the use of a dynamometer
    3) Nerve conduction velocities in lower limbs (NCV)
    - Proporción de pacientes que presente una mejora del 20 % en la 2MWT (prueba de marcha 2 min) de M12 respecto de la de M9
    - Proporción de pacientes que presente una mejora del 20 % en la TW25 (prueba cronometrada de marcha de 25 pies) de M12 respecto de la de M9
    - Prueba TUG cronometrada
    - Cuestionario Euroqol EQ-5D
    - Cuestionario Qualiveen (función urinaria)
    - Evaluación de la tolerancia a dosis elevadas de biotina: acontecimientos adversos, examen clínico (constantes vitales, ECG), análisis biológicos estándar (hematología y bioquímica)
    Criterios de exploración (optativos):
    1) IRM cerebral:
    a) IRM convencional; b) Imagen por tensor de difusión (ITD) cerebral y medular; c) Espectroscopia por resonancia magnética (ERM) de la sustancia blanca del cerebral.
    2) Evaluación de la fuerza muscular mediante dinamómetro.
    3) Velocidad de conducción nerviosa (VCN) de las extremidades inferiores.
    E.5.2.1Timepoint(s) of evaluation of this end point
    Baseline, 9 months, 12 months, 18 months, 24 months
    Valor inicial, 9 meses, 12 meses, 18 meses y 24 meses
    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) Yes
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled Yes
    E.8.1.1Randomised Yes
    E.8.1.2Open No
    E.8.1.3Single blind No
    E.8.1.4Double blind Yes
    E.8.1.5Parallel group Yes
    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 Yes
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    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 EEA4
    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
    Last visit of the last patient
    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 months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years2
    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: 60
    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 state20
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 60
    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
    G. Investigator Networks to be involved in the Trial
    G.4 Investigator Network to be involved in the Trial: 1
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2014-08-13
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2014-06-06
    P. End of Trial
    P.End of Trial StatusCompleted
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