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The European Union Clinical Trials Register   allows you to search for protocol and results information on:
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    The EU Clinical Trials Register currently displays   43974   clinical trials with a EudraCT protocol, of which   7312   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

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    EudraCT Number:2020-003265-19
    Sponsor's Protocol Code Number:PD0053
    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:2021-02-09
    Trial results
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    A. Protocol Information
    A.1Member State ConcernedSpain - AEMPS
    A.2EudraCT number2020-003265-19
    A.3Full title of the trial
    A Double-Blind, Placebo-Controlled, Randomized, 18-Month Phase 2a Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacokinetics of Oral UCB0599 in Study Participants With Early Parkinson’s Disease
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    A 18-month study to evaluate the efficacy, safety, tolerability and pharmacokinetics of oral UCB0599 in study participants with early-stage Parkinson’s Disease
    Estudio de 18 meses de duración, para evaluar la eficacia, seguridad, tolerabilidad y farmacocinética de UCB0599 oral en los participantes en el estudio, con enfermedad de parkinson en fase inicial
    A.4.1Sponsor's protocol code numberPD0053
    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 SponsorUCB Biopharma SRL
    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 supportUCB Biopharma SRL
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationUCB BIOSCIENCES GmbH
    B.5.2Functional name of contact pointClin Trial Reg & Results Disclosure
    B.5.3 Address:
    B.5.3.1Street AddressAlfred-Nobel-Strasse 10
    B.5.3.2Town/ cityMonheim
    B.5.3.3Post code40789
    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 nameUCB0599
    D.3.2Product code UCB0599
    D.3.4Pharmaceutical form Capsule
    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 INN-
    D.3.9.1CAS number 1802518-92-8
    D.3.9.2Current sponsor codeUCB0599
    D.3.9.4EV Substance CodeSUB206499
    D.3.10 Strength
    D.3.10.1Concentration unit mg milligram(s)
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number90
    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. cell therapy medicinal product No
    D. therapy medical product No
    D. Engineered Product No
    D. ATIMP (i.e. one involving a medical device) No
    D. 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
    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
    Early-stage Parkinson's disease
    E.1.1.1Medical condition in easily understood language
    Early-stage Parkinson's disease
    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 10061536
    E.1.2Term Parkinson's disease
    E.1.2System Organ Class 10029205 - Nervous system disorders
    E.1.3Condition being studied is a rare disease No
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Assess the safety and tolerability of UCB0599 and to demonstrate the superiority of UCB0599 over placebo with regard to clinical symptoms of disease progression over 12 and 18 months in participants diagnosed with early-stage Parkinson’s Disease (PD)
    Evaluar la seguridad y tolerabilidad de UCB0599 y demostrar la superioridad de UCB0599 frente a placebo respecto a los síntomas de progresión de la enfermedad a los 12 y 18 meses, en participantes diagnosticados de enfermedad de Parkinson (PD, Parkinson’s disease) en fase inicial
    E.2.2Secondary objectives of the trial
    - Demonstrate the superiority of UCB0599 over placebo with regard to neurodegeneration of dopaminergic neurons over 12 and 18 months in participants diagnosed with early-stage PD
    - Assess the effect of UCB0599 vs placebo with regard to intake of symptomatic treatment (ST) over 18 months in participants diagnosed with early-stage PD
    - Demostrar la superioridad de UCB0599 frente a placebo respecto a la neurodegeneración de las neuronas dopaminérgicas a los 12 y 18 meses, en participantes diagnosticados de PD en fase inicial
    - Evaluar el efecto de UCB0599 frente a placebo respecto a la toma de tratamiento sintomático a los 18 meses, en participantes diagnosticados de PD en fase inicial
    E.2.3Trial contains a sub-study No
    E.3Principal inclusion criteria
    - Study participant must be 40 to 70 years of age inclusive, at the time of signing the informed consent
    - Study participant has Parkinson’s Disease (PD), with a diagnosis made by a neurologist according to the 2015 Movement Disorder Society criteria within 2 years of Baseline Visit
    - The following diagnostic criteria must be met: bradykinesia AND at least ONE of the following: muscular rigidity, or resting tremor
    - A Screening Dopamine Transporter Imaging with Single Photon Emission Computed Tomography (DaT-SPECT) is consistent with PD as determined by a central reader
    - Study participant has no clear family history of autosomal-dominant forms of PD or confirmation of such mutations by genetic testing. Historical genetic testing information will be considered but is not required and will not be conducted for Screening purposes
    - Study participant is in the </= 2 modified Hoehn and Yahr stage at Screening
    - Study participant has never taken medications for the treatment of motor symptoms of PD and is not expected to require starting symptomatic treatment (ST) with a high likelihood in the next 6 months as far as clinical judgement allows
    - Study participant has never taken part in disease-modifying treatment studies directed at neurodegenerative disease (NDD)
    - Study participant is willing, competent, and able to comply with all aspects of the protocol, including follow-up schedule and biospecimen collection
    - A male participant must agree to use contraception during the Treatment Period and for at least 90 days after the last dose of study medication and refrain from donating sperm during this period
    - A female participant is eligible to participate if she is not pregnant, not breastfeeding, and at least one of the following conditions applies:
    (i)Not a woman of childbearing potential (WOCBP)
    A WOCBP who agrees to follow the contraceptive guidance during the Treatment Period and for at least 1 month after the last dose of study medication. The study participant must have a negative serum pregnancy test at Screening (Visit 1), which is to confirmed negative by urine testing prior to the first dose of study medication at Baseline (Visit 3). If oral contraception is used, an additional barrier method will be required during the study
    - Participantes en el estudio deben tener 40 a 70 años, inclusive, en el momento de firmar el consentimiento informado
    - El participante en el estudio presenta enfermedad de Parkinson (PD), diagnosticada por un neurólogo en los 2 años previos a la visita basal de acuerdo con los criterios de 2015 de la Movement Disorder Society (MDS)
    - Se deberán cumplir los siguientes criterios de diagnóstico: bradicinesia Y al menos UNO de los siguientes: rigidez muscular o temblor en reposo
    - La DaT-SPECT practicada en la Selección es compatible con PD, a juicio de un evaluador central
    - El participante en el estudio no presenta antecedentes familiares claros de formas de PD autosómicas dominantes ni el análisis genético confirma este tipo de mutaciones. Se tendrá en cuenta la información genética histórica, pero no será obligatoria ni se llevará a cabo a efectos de la Selección
    - El estadio de Hoehn y Yahr modificado del participante en el estudio es </= 2 en la Selección.
    - El participante en el estudio no ha recibido nunca medicación para el tratamiento de los síntomas motores de la PD y no se prevé que la probabilidad de que necesite iniciar tratamiento sintomático en los próximos 6 meses sea elevada, hasta donde alcanza el juicio clínico
    - El participante en el estudio nunca ha participado en estudios terapéuticos con modificadores de la enfermedad dirigidos a una enfermedad neurodegenerativa (NDD, neurodegenerative disease)
    - El participante en el estudio está dispuesto y es competente y capaz de cumplir con todos los aspectos del protocolo, incluidos el calendario de seguimiento y la recogida de muestras biológicas.
    - Un varón participante debe estar de acuerdo en usar medidas anticonceptivas durante el periodo de Tratamiento y durante al menos 90 días después de la última dosis del medicamento del estudio y se abstendrá de donar semen durante este periodo.
    - Una mujer es elegible para participar en el estudio si no está embarazada ni en periodo de lactancia y si cumple al menos una de las siguientes condiciones:
    . La mujer no es potencialmente fértil (WOCBP, woman of childbearing potential)
    O BIEN,
    Si la mujer es potencialmente fértil, está de acuerdo en usar medidas anticonceptivas durante el periodo de Tratamiento y durante al menos 1 mes después de la última dosis del medicamento del estudio. La participante en el estudio debe tener una prueba de embarazo en suero negativa en la Selección (Visita 1) y la negatividad se confirmará con una prueba en orina antes de la primera dosis del medicamento del estudio en el momento Basal (Visita 3). Si durante el estudio se utilizan anticonceptivos orales, se deberá usar también un método de barrera
    E.4Principal exclusion criteria
    - Study participant has a known hypersensitivity to any components (and/or its excipients) of the study medication or comparative drugs as stated in the protocol
    -Study participant has a brain magnetic resonance imaging (MRI) scan performed during Screening indicative of a clinically significant abnormality or a historical MRI scan during the 6 months before Screening Visit 1 of sufficient quality to show such abnormalities. In case of doubt, the significance is determined on a case-by-case basis in close collaboration with the Medical Monitor and should not include abnormalities like age-appropriate brain atrophy, minor white matter signals, or mild vasculopathy
    - Study participant has any contraindication for the brain MRI or Dopamine Transporter Imaging with Single Photon Emission Computed Tomography (DaT-SPECT) imaging
    - Study participant has a Montreal Cognitive Assessment (MoCA) score less than 23, indicating mild cognitive impairment or other significant cognitive impairment or clinical dementia at Screening that, in the opinion of the Investigator, would interfere with study evaluation
    - Study participant has abnormalities in lumbar spine previously known or determined by a Screening lumbar x-ray (if conducted) that could preclude lumbar puncture, in the opinion of the Investigator
    - Study participant has clinically significant electrocardiogram (ECG) abnormality at Screening, in the opinion of the Investigator
    - Study participant has past history of use of medications for the treatment of motor symptoms of PD (with the exception of short pharmacological testing for confirmation of diagnosis) or drugs with a potential PD disease modifying effect
    - El participante en el estudio presenta hipersensibilidad conocida a cualquiera de los componentes (y/o sus excipientes) de la medicación del estudio o de los medicamentos de comparación descritos en el protocolo
    - El participante en el estudio posee un estudio de resonancia magnética (MRI) cerebral practicado durante la Selección indicativo de anomalía clínicamente importante o en su historia consta una resonancia magnética de los 6 meses anteriores a la Visita 1 de Selección de calidad suficiente para mostrar dicha anomalía. En caso de duda, la importancia del hallazgo se determinará, tras consideración individual del caso, en estrecha colaboración con el Monitor Médico; el hallazgo no deberá consistir en anomalías propias de la edad, como atrofia cerebral, señales de grado menor en sustancia blanca o vasculopatía leve
    - El participante en el estudio presenta cualquier contraindicación a la resonancia magnética cerebral o al diagnóstico por imagen del transportador de dopamina mediante tomografía computerizada de emisión monofotónica (DaT-SPECT)
    - El participante en el estudio presenta una puntuación de la Montreal Cognitive Assessment (MoCA) menor de 23, indicativa de afectación cognitiva leve, u otra afectación cognitiva o demencia clínica importantes en la Selección que, en opinión del Investigador, podrían interferir con las evaluaciones del estudio
    - El participante en el estudio presenta anomalías en columna lumbar, ya conocidas u observadas en el estudio radiológico lumbar de la Selección (si se practica), que podrían impedir, en opinión del Investigador, la punción lumbar
    - El participante en el estudio presenta una anomalía en el electrocardiograma (ECG) de la Selección que es clínicamente importante en opinión del Investigador
    - El participante en el estudio tiene antecedentes de uso de medicamentos para el tratamiento de síntomas motores de la enfermedad de Parkinson (con la excepción de su empleo en pruebas farmacológicas breves para confirmación del diagnóstico) o de fármacos con posible efecto modificador de la enfermedad de Parkinson
    E.5 End points
    E.5.1Primary end point(s)
    1. Movement Disorder Society Unified Parkinson's Disease Rating Scale (MDS-UPDRS) Parts I-IIIsum score
    1.Suma de las puntuaciones de las Partes I-III de la escala unificada de evaluación de la enfermedad de Parkinson de la Movement Disorder Society (MDS-UPDRS, Movement Disorder Society-Unified Parkinson’s Disease Rating Scale
    E.5.1.1Timepoint(s) of evaluation of this end point
    1: From Baseline up to 18 Months
    1: Desde momento basal hasta 18 meses
    E.5.2Secondary end point(s)
    1. MDS-UPDRS Part III subscale
    2. MDS-UPDRS Part II subscale
    3. MDS-UPDRS Part I subscale
    4. Time to worsening of the disease on MDS-UPDRS Part III scale
    5. Change in Montreal Cognitive Assessment (MoCA)
    6. Change in Dopamine Transporter Imaging with Single Photon Emission Computed Tomography (DaT-SPECT) mean striatum specific binding ratios (SBR)
    7. Time to start of symptomatic treatment (ST)
    8. Symptomatic treatment intake
    9. Incidence of treatment-emergent adverse events (TEAEs)
    10. Incidence of serious adverse events (SAEs)
    11. Incidence of TEAEs leading to participant withdrawal
    1. Subescala de la Parte III de la MDS-UPDRS
    2. Subescala de la Parte II de la MDS-UPDRS
    3. Subescala de la Parte I de la MDS-UPDRS
    4. Tiempo hasta el empeoramiento de la enfermedad
    5. Modificación de la evaluación cognitiva de Montreal (MoCA, Montreal Cognitive Assessment)
    6. Modificación de la tasa media de unión específica (SBR, specific binding ratio) en el cuerpo estriado, en la imagen de transportadores de dopamina con tomografía computadorizada de emisión de fotón único (DaT-SPECT, Dopamine Transporter Imaging with Single Photon Emission Computed Tomography)
    7. Tiempo hasta el inicio del tratamiento sintomático (ST, symptomatic treatment)
    8. Toma de ST
    9. Incidencia de acontecimientos adversos surgidos durante el tratamiento (TEAE, treatment-emergent adverse event)
    10. Incidencia de acontecimientos adversos graves (SAE, serious adverse event)
    11. Incidencia de TEAE que ocasionan la retirada del participante
    E.5.2.1Timepoint(s) of evaluation of this end point
    1-4+7+8: From Baseline up to 18 Months
    5+6: From Screening up to 18 Months
    10: From Screening up to 19 Months
    9+11: From Baseline up to 19 Months
    1-4+7+8: Desde momento basal hasta 18 meses
    5+6: Desde selección hasta 18 meses
    10: Desde selección hasta 19 meses
    9+11: Desde momento basal hasta 19 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 Yes
    E.6.7Pharmacodynamic No
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic Yes
    E.6.11Pharmacogenomic Yes
    E.6.12Pharmacoeconomic No
    E.6.13Others Yes
    E.6.13.1Other scope of the trial description
    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. 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 concerned6
    E.8.5The trial involves multiple Member States Yes
    E.8.5.1Number of sites anticipated in the EEA35
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    United States
    United Kingdom
    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
    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 months7
    E.8.9.1In the Member State concerned days0
    E.8.9.2In all countries concerned by the trial years2
    E.8.9.2In all countries concerned by the trial months9
    E.8.9.2In all countries concerned by the trial days22
    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: 150
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 150
    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 169
    F.4.2.2In the whole clinical trial 300
    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)
    Study participants who complete the Treatment Period will have the option to transition into an open label extension (OLE) study (PD0055).
    Los participantes en el estudio que completen el periodo de tratamiento tendrán la opción de pasar a un estudio abierto de extensión (OLE; PD0055).
    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 Decision2021-05-26
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-02-24
    P. End of Trial
    P.End of Trial StatusOngoing
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