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    Summary
    EudraCT Number:2018-004145-16
    Sponsor's Protocol Code Number:Z7219K01
    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:2019-06-11
    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 number2018-004145-16
    A.3Full title of the trial
    A 12-weeks, multicentre, randomized, double-blind, placebo-controlled, exploratory, pilot study to evaluate the safety and efficacy of safinamide 200 mg once daily, as add-on therapy, in patients with possible or probable parkinsonian variant of multiple system atrophy.
    Estudio piloto, exploratorio, multicéntrico, aleatorizado, doble ciego y controlado por placebo de 12 semanas de duración para evaluar la seguridad y la eficacia de Safinamida 200 mg una vez al día, como tratamiento complementario, en pacientes con posible o probable variante parkinsoniana de Atrofia Multi-Sistémica.
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A study evaluating the safety and efficacy of safinamide for patients with Multiple System Atrophy
    Estudio para evaluar la seguridad y eficacia de Safinamida en pacientes con Atrofia Multi-Sistémica
    A.3.2Name or abbreviated title of the trial where available
    Safinamide for Multiple System Atrophy
    A.4.1Sponsor's protocol code numberZ7219K01
    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 SponsorZambon SpA
    B.1.3.4CountryItaly
    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 supportZambon SpA
    B.4.2CountryItaly
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationZambon SpA
    B.5.2Functional name of contact pointClinical Development
    B.5.3 Address:
    B.5.3.1Street AddressVia Lillo del Duca 10
    B.5.3.2Town/ cityBresso, Milan
    B.5.3.3Post code20091
    B.5.3.4CountryItaly
    B.5.4Telephone number+3902665241
    B.5.6E-mailclinicaltrials@zambongroup.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 Yes
    D.2.1.1.1Trade name Xadago
    D.2.1.1.2Name of the Marketing Authorisation holderZambon SpA
    D.2.1.2Country which granted the Marketing AuthorisationEuropean Union
    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 Film-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 INNsafinamide methanesulfonate
    D.3.9.1CAS number 133865-89-1
    D.3.9.3Other descriptive nameSAFINAMIDE
    D.3.9.4EV Substance CodeSUB32946
    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 placeboFilm-coated tablet
    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
    Parkinsonian variant of Multiple System Atrophy
    variante parkinsoniana de atrofia multisistémica
    E.1.1.1Medical condition in easily understood language
    Multiple System Atrophy
    atrofia multisistémica
    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 PT
    E.1.2Classification code 10064060
    E.1.2Term Multiple system atrophy
    E.1.2System Organ Class 10029205 - Nervous system disorders
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    To evaluate the safety and tolerability of safinamide, 200 mg od, compared with placebo
    Evaluar la seguridad y la tolerabilidad de Safinamida, 200 mg una vez al día, en comparación con un placebo.
    E.2.2Secondary objectives of the trial
    To evaluate the potential efficacy of safinamide 200 mg od, as add-on therapy, on motor function and/or quality of life
    Evaluar la posible eficacia de Safinamida 200 mg, una vez al día como tratamiento complementario, en la función motora y/o la calidad de vida.
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    Participants are eligible to be included in the study only if all of the following criteria apply:
    Age
    1. Participant must be 30 to 80 years of age inclusive, at the time of signing the informed consent.
    Type of Participant and Disease Characteristics
    2. Participants who are diagnosed (with MRI confirmation) with possible or probable parkinsonian variant of Multiple System Atrophy less than 2 years ago.
    3. Participants with an anticipated survival of at least 3 years in the opinion of the investigator.
    Sex
    4. Male or female
    • A female participant is eligible to participate if she is not pregnant, not breastfeeding, and at least one of the following conditions applies (see Appendix 4):
    i. Not a woman of childbearing potential (WOCBP)
    OR
    ii. A WOCBP who agrees to follow the contraceptive guidance during the treatment period and for at least 30 days after the last dose of study intervention.
    Informed Consent
    5. Capable of giving signed informed consent as described in Appendix 1 which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
    Los participantes solo podrán ser incluidos en el estudio si cumplen todos los criterios siguientes:
    Edad
    1.El participante deberá tener entre 30 y 80 años de edad, ambos inclusive, en el momento de firmar el consentimiento informado.
    Tipo de participante y características de la enfermedad
    2.Participantes diagnosticados (con confirmación mediante RM) de posible o probable variante parkinsoniana de atrofia multisistémica hace menos de 2 años.
    3.Participantes con una supervivencia esperada de al menos 3 años en opinión del investigador.
    Sexo
    4.Varones o mujeres
    • Las mujeres podrán participar si no están embarazadas ni en período de lactancia y se cumple al menos una de las condiciones siguientes (véase el Apéndice 4):
    i.No es una mujer en edad fértil (MEF)
    O
    ii.Es una MEF que se compromete a seguir las normas anticonceptivas durante el período de tratamiento y durante al menos 30 días después de la última dosis de la intervención del estudio.
    Consentimiento informado
    5.Ser capaz de otorgar el consentimiento informado firmado según se describe en el Apéndice 1, lo que incluye el cumplimiento de los requisitos y restricciones que se enumeran en el documento de consentimiento informado (DCI) y en este protocolo.
    E.4Principal exclusion criteria
    Participants are excluded from the study if any of the following criteria apply:
    Medical Conditions
    1. History of neurosurgical procedure, including stereotactic surgery.
    2. History of Deep Brain Stimulation (DBS)
    3. History of bipolar disorder, severe depression, schizophrenia or other psychotic disorder.
    4. History of drug and/or alcohol abuse within 12 months prior to screening as defined by the current edition of the Diagnostic and Statistical Manual of Mental Disorders
    5. History of dementia (DSM-V criteria)
    6. Ophthalmologic history including any of the following conditions: albinism, uveitis, retinitis pigmentosa, retinal degeneration, active retinopathy, severe progressive diabetic retinopathy, inherited retinopathy or family history of hereditary retinal disease.
    7. Active hepatitis B or C
    8. History of human immunodeficiency virus (HIV) infection
    9. Subjects not able to swallow oral medications
    10. Subjects with severe orthostatic symptoms
    11. Impaired ambulation, i.e. falling more than once per week, bedridden patients or confined to a wheelchair during the whole day.
    12. Subjects with active malignant neoplasms.
    13. Movement disorders other than MSA (e.g. Parkinson Disease, dementia with Lewy bodies, essential tremor, progressive supranuclear palsy, pharmacological or post-encephalic parkinsonism).
    14. Any clinically significant or unstable medical or surgical condition that, in the opinion of the investigator, might preclude safe completion of the study or might affect the results of the study.
    Prior/Concomitant Therapy
    15. Not on a stable regime, for at least 4 weeks prior to the randomization (baseline visit), of
    a. oral levodopa (including controlled release [CR], immediate release [IR] or a combination of CR/IR), with or without benserazide/carbidopa, with or without addition of a catechol O-methyltransferase (COMT) inhibitor
    or
    b. dopamine agonist, anticholinergic and/or amantadine.
    Prior/Concurrent Clinical Study Experience
    16. Patients should not have received treatment with monoamine oxidase inhibitors in the 2 weeks prior to the randomization visit, nor treatment with levodopa infusion, pethidine, opiates, opioids, fluoxetine, fluvoxamine in the 4 weeks prior to the randomization visit.
    17. Patients should not have received treatment with an oral or depot neuroleptic within 12 weeks prior to the randomization visit.
    18. Use of any investigational drug within 30 days prior to screening or 5 half-lives, whichever is the longest.
    Diagnostic assessments
    19. Montreal Cognitive Assessment (MoCA) ≤ 20
    20. Laboratory assessments showing moderate or severe hepatic impairment (2x ULN)
    Other Exclusions
    21. Allergy/sensitivity or contraindications to the investigational medicinal products (IMPs) or their excipients, anticonvulsants, levodopa or other anti-parkinsonian drugs.
    22. Any clinically significant condition which, in the opinion of the Investigator, would not be compatible with study participation or represent a risk for patients while in the study.
    Se excluirá del estudio a los participantes que cumplan alguno de los criterios siguientes:
    Enfermedades
    1.Antecedentes de intervención neuroquirúrgica, incluida la cirugía estereotáctica.
    2.Antecedentes de estimulación cerebral profunda (ECP)
    3.Antecedentes de trastorno bipolar, depresión grave, esquizofrenia u otro trastorno psicótico.
    4.Antecedentes de alcoholismo o toxicomanía en los 12 meses previos a la selección, según se define en la edición vigente del Manual diagnóstico y estadístico de los trastornos mentales
    5.Antecedentes de demencia (criterios DSM-V)
    6.Antecedentes oftalmológicos, incluidos cualquiera de los siguientes problemas: albinismo, uveítis, retinitis pigmentaria, degeneración retiniana, retinopatía activa, retinopatía diabética progresiva grave, retinopatía hereditaria o antecedentes familiares de retinopatía hereditaria.
    7.Hepatitis B o C activa
    8.Antecedentes de infección por el virus de la inmunodeficiencia humana (VIH).
    9.Sujetos incapaces de tragar medicamentos orales
    10.Sujetos con síntomas ortostáticos intensos
    11.Deterioro de la deambulación, por ejemplo, caída más de una vez a la semana, postrado en cama o confinado a una silla de ruedas durante todo el día.
    12.Sujetos con neoplasias malignas activas.
    13.Trastornos del movimiento distintos de la AMS (p. ej., enfermedad de Parkinson, demencia con cuerpos de Lewy, temblor esencial, parálisis supranuclear progresiva, parkinsonismo farmacológico o posencefalítico).
    14.Cualquier trastorno médico o quirúrgico clínicamente importante o inestable que, en opinión del investigador, podría impedir la finalización segura del estudio o afectar a los resultados del estudio.
    Tratamiento previo/concomitante
    15.Sin una pauta estable, durante al menos 4 semanas antes de la aleatorización (visita basal), de
    a.levodopa oral (incluidos levodopa de liberación controlada [LC], liberación inmediata [LI] o una combinación de LC/LI), con o sin benserazida/carbidopa, con o sin adición de un inhibidor de la catecol O-metiltransferasa (COMT) o
    b.agonista dopaminérgico, anticolinérgico y/o amantadina.
    Experiencia previa/concurrente en estudios clínicos
    16.Los pacientes no deberán haber recibido tratamiento con inhibidores de la monoaminooxidasa en las 2 semanas previas a la visita de aleatorización, ni tratamiento con infusión de levodopa, ni petidina, opiáceos, fluoxetina, fluvoxamina en las 4 semanas previas a la visita de aleatorización.
    17.Los pacientes no podrán haber recibido tratamiento con un neuroléptico oral o de liberación retardada en las 12 semanas previas a la visita de aleatorización.
    18.Uso de cualquier fármaco en investigación en los 30 días previos a la selección o 5 semividas, lo que suponga más tiempo.
    Evaluaciones diagnósticas
    19.Evaluación cognitiva de Montreal (MoCA) < 20
    20.Evaluaciones analíticas que indiquen insuficiencia hepática moderada o grave (2 x LSN)
    Otras exclusiones
    21.Alergia/sensibilidad o contraindicaciones a los medicamentos en investigación (MEI) o sus excipientes, antiepilépticos, levodopa u otros antiparkinsonianos.
    22.Cualquier trastorno clínicamente significativo que, en opinión del investigador, no sería compatible con la participación en el estudio o representaría un riesgo para los pacientes durante su participación en el estudio.
    E.5 End points
    E.5.1Primary end point(s)
    • The nature, frequency, severity, relationship (to study drug), actions taken, and outcome of TEAEs and SAEs.
    • Changes in physical and neurological examination findings.
    • Changes in vital sign (heart rate, systolic and diastolic blood pressure) values, including occurrence of abnormalities.
    • Changes in 12-lead ECG parameter measures, including occurrence of abnormalities.
    • Changes in clinical chemistry and hematology values, including shifts from Baseline and occurrence of abnormalities.
    • Number of withdrawals (and reason if given).
    •Naturaleza, frecuencia, intensidad, relación (con el fármaco del estudio), medidas adoptadas y desenlace de los AAAT y AAG.
    •Variaciones en los hallazgos de exploración física y neurológica.
    •Variaciones de los valores de las constantes vitales (frecuencia cardíaca, presión arterial sistólica y diastólica), incluida la aparición de anomalías.
    •Variaciones de los parámetros del ECG de 12 derivaciones, incluida la aparición de anomalías.
    •Variaciones de los valores de bioquímica clínica y hematología, incluidas las desviaciones con respecto a los valores basales y la aparición de anomalías.
    •Número de retiradas (y motivo si se indica).
    E.5.1.1Timepoint(s) of evaluation of this end point
    Outcome will be assessed after 12 weeks of treatment, when progression is unlikely to be a confounder of change.
    El resultado se evaluará después de 12 semanas de tratamiento, cuando es improbable que la progresión sea un factor de confusión.
    E.5.2Secondary end point(s)
    • The change from baseline to week 12 in the goniometric measurement for “lateral” displacement
    • Change from baseline at week 12 in Unified MSA Rating Scale, MSA Health-Related Quality of Life (MSA-QoL) scale, Montreal Cognitive Assessment (MoCA) scale, Unified Dystonia Rating Scale (UDRS).
    •Variación entre la visita basal y la semana 12 de la medida goniométrica del desplazamiento lateral.
    •Variación entre la visita basal y la semana 12 en la Escala unificada de valoración de la AMS, la Escala de calidad de vida relacionada con la salud en la AMS (MSA-QoL), la Escala de evaluación cognitiva de Montreal (MoCA) y la Escala unificada de valoración de la distonía (UDRS).
    E.5.2.1Timepoint(s) of evaluation of this end point
    Outcome will be assessed after 12 weeks of treatment, when progression is unlikely to be a confounder of change.
    El resultado se evaluará después de 12 semanas de tratamiento, cuando es improbable que la progresión sea un factor de confusión.
    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 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 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 concerned8
    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 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
    LVLS
    Ultima vistia del ultimo paciente
    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 months
    E.8.9.1In the Member State concerned days
    E.8.9.2In all countries concerned by the trial years1
    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: 24
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 24
    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 state40
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 48
    F.4.2.2In the whole clinical trial 48
    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)
    standard of care
    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 Decision2019-07-24
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2019-07-23
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2021-01-05
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