E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Myelin oligodendrocyte glycoprotein antibody-associated disease (MOG-AD) |
Enfermedad asociada a anticuerpos contra la glucoproteína de la mielina del oligodendrocito (MOG) |
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E.1.1.1 | Medical condition in easily understood language |
Myelin oligodendrocyte glycoprotein (MOG) antibody-associated disease (MOG-AD) is an inflammatory demyelinating condition of the central nervous system (CNS). |
La enfermedad asociada a anticuerpos contra la glucoproteína de la mielina del oligodendrocito (MOG) es una enfermedad desmielinizante inflamatoria del sistema nervioso central (SNC) |
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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 | HLT |
E.1.2 | Classification code | 10012302 |
E.1.2 | Term | Demyelinating disorders NEC |
E.1.2 | System Organ Class | 100000004852 |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
Evaluate the efficacy of rozanolixizumab for treatment of participants with myelin oligodendrocyte glycoprotein (MOG) antibody-associated disease (MOG-AD) |
Evaluar la eficacia de rozanolixizumab en el tratamiento de participantes con MOG-AD |
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E.2.2 | Secondary objectives of the trial |
Assess the safety and tolerability of rozanolixizumab in participants with myelin oligodendrocyte glycoprotein (MOG) antibody-associated disease (MOG-AD) |
Evaluar la seguridad y la tolerabilidad de rozanolixizumab en participantes con MOG-AD |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Participants must be ≥18 to ≤89 years of age, at the time of signing the informed consent - Participant must have a history of myelin oligodendrocyte glycoprotein antibody-associated disease (MOG-AD) with any of the following clinical presentations: a) Optic neuritis (single, recurrent, or simultaneous bilateral) b) Transverse myelitis (including Longitudinally extensive spinal cord lesion (LETM)) c) Acute disseminated encephalomyelitis or MOG antibody-associated encephalitis, brain stem encephalitis d) Combined presentations - Positivity for serum MOG-Immunglobulin G (IgG) antibodies using cell-based assay at Screening - Participant has history of relapsing MOG-AD with at least 1 documented relapse over the last 12 months prior to randomization - Participant must be clinically stable at the time of the Screening Visit and during the Screening Period |
- El participante debe tener de >/=18 a </=89 años de edad en el momento de firma del consentimiento informado - El participante debe tener historia de enfermedad asociada a anticuerpos contra la glucoproteína de la mielina del oligodendrocito (myelin oligodendrocyte glycoprotein antibody-associated disease, MOG-AD), con cualquiera de las siguientes presentaciones clínicas: a) Neuritis óptica (unilateral, recurrente o bilateral simultánea) b) Mielitis transversa (incluida lesión de médula espinal longitudinalmente extensa (longitudinally extensive spinal cord lesion, LETM)) c) Encefalomielitis aguda diseminada o encefalitis asociada a anticuerpos contra la MOG, encefalitis del tronco encefálico d) Presentaciones combinadas - En la Selección, utilizando un método de tipo celular, positividad en suero de anticuerpos de tipo inmunoglobulina G (IgG) frente a la MOG, - El participante presenta historia de MOG-AD recidivante, con como mínimo 1 recidiva documentada en el plazo de los 12 últimos meses antes de la aleatorización - El participante debe encontrarse clínicamente estable en el momento de la visita de Selección y durante el periodo de Selección |
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E.4 | Principal exclusion criteria |
- Participant has been diagnosed with a neurological autoimmune disease (including multiple sclerosis (MS) and aquaporin-4 positive neuromyelitis optica spectrum disorder (NMOSD)), or a systemic autoimmune disease that in the opinion of the investigator can interfere with the safety of the participant - Participant has a clinically relevant active infection (eg, sepsis, pneumonia, or abscess), or has had a serious infection (resulting in hospitalization or requiring parenteral antibiotic treatment) within 6 weeks prior to the first dose of investigational medicinal product (IMP) - Participant has a current or medical history of primary immunodeficiency - Participant has a current or medical history of IgA deficiency - Participant tests positive for aquaporin-4 antibodies at screening - Participant has a serum total IgG level ≤5.5g/L |
- El participante ha sido diagnosticado de enfermedad neurológica autoinmunitaria (incluida la esclerosis múltiple y el espectro clínico de la neuromielitis óptica con positividad de acuaporina 4 (aquaporin-4 positive neuromyelitis optica spectrum disorder, NMOSD)), o una enfermedad autoinmunitaria sistémica que, en opinión del investigador, pueda afectar a la seguridad del participante - El participante presenta una infección activa clínicamente importante (por ejemplo, sepsis, neumonía o absceso) o ha tenido una infección grave (resultante en la hospitalización o que precisó antibioterapia parenteral) en el plazo de las 6 semanas previas a la primera dosis del medicamento en investigación - El participante presenta o tiene antecedentes de inmunodeficiencia primaria - El participante presenta o tiene antecedentes de deficiencia de IgA - El participante presenta resultado positivo en la prueba de anticuerpos anti-acuaporina 4 en la selección - El participante tiene un nivel sérico de IgG total </=5.5g/L |
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E.5 End points |
E.5.1 | Primary end point(s) |
Part A: 1. Time from randomization to first independently centrally adjudicated relapse (TTFR) during the DB Treatment Period Part B: 2. Incidence of treatment-emergent adverse events (TEAEs) during OLE Treatment Period 3. Incidence of treatment-emergent adverse events (TEAEs) leading to permanent withdrawal of investigational medicinal product (IMP) during OLE Treatment Period |
Parte A: 1. Tiempo desde la aleatorización hasta la primera recaída confirmada por un comité central e independiente (time to first relapse, TTFR) durante el periodo de tratamiento en DB. Parte B: 2. Incidencia de acontecimientos adversos surgidos durante el tratamiento (treatment-emergent adverse events, TEAE) durante el periodo de tratamiento de OLE 3. Incidencia de acontecimientos adversos surgidos durante el tratamiento (TEAE) que conducen a la retirada definitiva del medicamento en investigación (investigational medicinal product, IMP) durante el periodo de tratamiento de OLE |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
1. Baseline (Week 1) to EDB/EWD Visit (until a confirmed relapse or up to 132 weeks) 2, 3: OLE Treatment Period (OLE Week 1) to EOS/EWD Visit (up to OLE Week 52) |
1. Basal (semana 1) hasta la visita de EDB/EWD (hasta una recaíada confirmada o hasta 132 semanas) 2, 3: Periodo de tratamiento de OLE (OLE semana 1) hasta la visita de EOS/EWD (hasta la semana 52 de OLE) |
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E.5.2 | Secondary end point(s) |
Part A 1. Change from Baseline in Low-Contrast Monocular Visual Acuity (Worst Affected Eye) measured by low-contrast Landolt C Broken Rings Chart at the EDB/EWD Visit 2. Disability as assessed by Expanded Disability Status Scale (EDSS) scores at the end of the EDB/EWD Visit (with confirmation at 3 months) 3. Number of MOG-AD related inpatient hospitalizations during the DB Treatment Period 4. Independently centrally adjudicated annualized relapse rate (ARR) during the DB Treatment Period 5. Change from Baseline in Low-Contrast Monocular Visual Acuity (Least Affected Eye) measured by low-contrast Landolt C Broken Rings Chart at the EDB/EWD Visit (with confirmation at 3 months) 6. Incidence of treatment-emergent adverse events (TEAEs) during DB Treatment Period Part B 7. Independently centrally adjudicated annualized relape rate (ARR) during the DB and OLE Treatment Period |
Parte A: 1. Cambio con respecto al valor basal en la agudeza visual monocular de bajo contraste (ojo más afectado) medida mediante la tabla de anillos rotos o de C de Landolt de bajo contraste en la visita de EDB/EWD 2. Discapacidad evaluada mediante las puntuaciones de la Escala ampliada del estado de discapacidad (Expanded Disability Status Scale, EDSS) en la visita de EDB/EWD (con confirmación a los 3 meses) 3. Número de ingresos hospitalarios relacionados con la MOG-AD durante el periodo de tratamiento en DB 4. Tasa de recaída anualizada (annualized relapse rate, ARR) confirmada por un comité central e independiente durante el periodo de tratamiento en DB 5. Cambio con respecto al valor basal en la agudeza visual monocular de bajo contraste (ojo menos afectado) medida mediante la tabla de anillos rotos o de C de Landolt de bajo contraste en la visita de EDB/EWD (con confirmación a los 3 meses) 6. Incidencia de acontecimientos adversos surgidos durante el tratamiento (treatment-emergent adverse events, TEAE) durante el periodo de tratamiento en DB Parte B 7. ARR evaluada por un comité central e independiente durante el período de tratamiento en doble ciego y el período de tratamiento de OLE |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
1. From Baseline (Week 1) to EDB/EWD Visit (up to 132 weeks) 2. Baseline (Week 1), EDB/EWD Visit (up to 132 weeks) 3, 4, 5, 6: Baseline (Week 1) to EDB/EWD Visit (up to 132 weeks) 7: Baseline (Week 1) to EOS/EWD Visit´(up to OLE Week 52) |
1. Des de la basal (semana 1) hasta la visita de EDB/EWD (hasta 132 semanas) 2. Basal (semana 1), visita de EDB/EWD (hasta 132 semanas) 3, 4, 5, 6: Basal (semana 1), hasta la visita de EDB/EWD (hasta 132 semanas) 7. Basal (semana 1) hasta la visita de EOS/EWD (hasta la semana 52 de OLE) |
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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 | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | Yes |
E.6.11 | Pharmacogenomic | Yes |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Yes |
E.6.13.1 | Other scope of the trial description |
Tolerability and immunogenicity |
tolerabilidad e inmunogenicidad |
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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) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
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 | Yes |
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 | 2 |
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 | 3 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 34 |
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 |
Australia |
Brazil |
China |
Japan |
Korea, Republic of |
Mexico |
Turkey |
Ukraine |
United States |
Belgium |
France |
Germany |
Italy |
Portugal |
Spain |
Sweden |
Switzerland |
United Kingdom |
Czechia |
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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
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LVLS |
Último paciente última visita |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 3 |
E.8.9.1 | In the Member State concerned months | 6 |
E.8.9.1 | In the Member State concerned days | 8 |
E.8.9.2 | In all countries concerned by the trial years | 3 |
E.8.9.2 | In all countries concerned by the trial months | 9 |
E.8.9.2 | In all countries concerned by the trial days | 27 |