E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Parkinson's Disease |
Enfermedad de Parkinson |
|
E.1.1.1 | Medical condition in easily understood language |
Parkinson's Disease |
Enfermedad de Parkinson |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Nervous System Diseases [C10] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10061536 |
E.1.2 | Term | Parkinson's disease |
E.1.2 | System Organ Class | 10029205 - Nervous system disorders |
|
E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To evaluate the dose-related safety of BIIB054. |
evaluar la seguridad relacionada con la dosis de BIIB054 |
|
E.2.2 | Secondary objectives of the trial |
- To evaluate the pharmacodynamic effects of BIIB054 on the integrity of nigrostriatal dopaminergic nerve terminals in Year 1 of the study. - To assess the PK profile of BIIB054. - To evaluate the immunogenicity of BIIB054. |
- Evaluar los efectos farmacodinámicos de BIIB054 en la integridad de los terminales nerviosos dopaminérgicos nigroestriatales en el año 1 del estudio - Evaluar el perfil farmacocinético (FC) de BIIB054 - Evaluar la inmunogenicidad de BIIB054 |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Diagnosed with Parkinson's disease (PD) within a maximum of 3 years prior to Screening. - Score of ≤2.5 on the Modified Hoehn and Yahr Scale. - Has not received levodopa or any other treatment for PD (dopamine agonists, amantadine, anticholinergics, MAO-B inhibitors, or safinamide) for at least 12 weeks prior to Day 1 and, in the opinion of the Investigator, is not expected to require PD treatment for at least 6 months following Day 1. Maximum total duration of prior PD regimens should not exceed 30 days. - Screening dopamine transporter (DaT)/ single-photon emission computed tomography (SPECT) results consistent with neurodegenerative Parkinsonism (central reading). - All women of childbearing potential and all men must practice highly effective contraception during the study and for 6 months after their last dose of study treatment. |
- diagnosticado con Enfermedad de Parkinson (EP) establecida clínicamente durante un máximo de 3 años antes de la selección, - puntuación de ≤2,5 en la Escala de Hoehn y Yahr modificada - Los sujetos no deben recibir levodopa o ningún otro tratamiento para la EP durante las 12 semanas previas al día 1, y la duración total de cualquier pauta de tratamiento anterior para la EP no debe exceder los 30 días. - resultados de detección de DaT/SPECT en la visita de selección que demuestren una actividad consistente con parkinsonismo neurodegenerativo |
|
E.4 | Principal exclusion criteria |
- Presence of freezing of gait. - MOCA score <23 or other significant cognitive impairment or clinical dementia that, in the opinion of the Investigator, would interfere with study evaluation. - History of or screening brain magnetic resonance imaging (MRI) scan indicative of clinically significant abnormality, as read by central reader. - History of severe allergic or anaphylactic reactions, or history of hypersensitivity to BIIB054 or any of the inactive ingredients in the drug product or to radioligands or iodine used in the study. - Participation in any active immunotherapy study targeting alpha-synuclein. - Use of allowed medications not previously specified at doses that have not been stable for at least 8 weeks before Day 1, and/or that are not expected to remain stable for the duration of the study. - Clinically significant abnormal laboratory test values at Screening, as determined by the Investigator. - Blood donation (1 unit or more) within 8 weeks before Day 1 (must also refrain from donating blood for the duration of the study). |
- Presencia de congelamiento al andar - Puntuación MOCA <23 u otros deterioros cognitivos significantes o demencia clínica que, en opinión del investigador podría interferir con la evolución del estudio. - Historia o a la visita de inicio tras la resonancia magnética (MRI) indicando anormalidades, tras la interpretación del radiólogo central. - Historia de alergias severas o reacciones anafilácticas, o historia de hipersensibilidad a BIIB054 u otro ingrediente inactivo en el fármaco en investigación o a radioligandos o yodo usados en el estudio. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
- Percentage of Participants With Adverse Event (AEs) and Serious Adverse Event (SAEs) - Change from Baseline in Clinical Laboratory Test Data, Vital Sign Measurements, Neurological and Physical Examination Findings, ECGs, and Brain MRIs. |
- Incidencia evento adversos (AEs) y eventos adversos graves (SAEs) - Cambios con respecto a la línea basal en los datos de análisis clínicos, mediciones de constantes vitales, resultados de las exploraciones neurológicas y físicas, electrocardiogramas (ECG) y hallazgos de seguridad en resonancias magnéticas (RM) cerebrales. |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
- Up to Week 52 - Baseline, Week 52 |
- Hasta semana 52 - Basal, semana 52 |
|
E.5.2 | Secondary end point(s) |
- Change in Striatal Binding Ratio (SBR) in Putamen,Striatum, and Caudate as Measured by Striatal-Photon Emission Computed Tomography (SPECT) Imaging of the Dopamine Transporter With Ioflupane I123 (DaTscan™) - Concentration of BIIB054 in the Serum - Percentage of Participants With Anti-BIIB054 Antibodies in the Serum |
- Cambio en el índice de unión estriatal en el putamen, el cuerpo estriado y el caudado, medido por tomografía computarizada por emisión de fotón único (SPECT) del transportador de dopamina (DaT) con ioflupano I123 (DaTscan™) - concentración de BIIB054 en el suero - Incidencia y título de anticuerpos anti-BIIB054 en suero |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
- Baseline, Week 52 - Baseline and at multiple time points (up to 2 years) - Baseline and at multiple time points (up to 2 years) |
- Basal, semana 52 - Basal y en múltiples periodos de tiempo (hasta los 2 años) - Basal y en múltiples periodos de tiempo (hasta los 2 años) |
|
E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | No |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | Yes |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 4 |
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.8.4.1 | Number of sites anticipated in Member State concerned | 10 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 49 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Austria |
Canada |
France |
Germany |
Israel |
Italy |
Spain |
Sweden |
United Kingdom |
United States |
|
E.8.7 | Trial 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
|
The end of study is last subject, last visit |
El fin del ensayo es la última visita del último paciente |
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 4 |
E.8.9.1 | In the Member State concerned months | 9 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 4 |
E.8.9.2 | In all countries concerned by the trial months | 9 |