E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Generalized myasthenia gravis
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Myasthénie grave généralisée |
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E.1.1.1 | Medical condition in easily understood language |
Myasthenia gravis is an autoimmune disease that causes weakness in your muscles; it is caused by a communication problem between nerves and muscles.
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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 | 21.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10028417 |
E.1.2 | Term | Myasthenia gravis |
E.1.2 | System Organ Class | 10029205 - Nervous system disorders |
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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 |
Assess the safety and tolerability of additional 6-week treatment cycles with rozanolixizumab in study participants with generalized myasthenia gravis (gMG)
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Évaluer la tolérance et la sécurité d'emploi des cycles supplémentaires de 6 semaines de traitement par rozanolixizumab chez des participants à l’étude atteints de MGg |
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E.2.2 | Secondary objectives of the trial |
Assess the efficacy of 6-week treatment cycles with rozanolixizumab in study participants with generalized myasthenia gravis (gMG)
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Évaluer l'efficacité des cycles de 6 semaines de traitement par rozanolixizumab chez des participants à l’étude atteints de MGg |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Study participant must meet one of the following: a) completed MG0003 [NCT03971422] or MGC003, b) required rescue therapy during the Observation Period in MG0003 or c) completed at least 6 visits in MG0004 [NCT04124965] - Body weight ≥35 kg at Baseline (Day 1) - Study participants may be male or female
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-Présence d’un des critères suivants pour le participant : a) participant à l’étude ayant terminé l’étude MG0003 ou MGC003, b) participant à l’étude ayant nécessité un traitement de secours (sauf IgIV ou PEX) pendant la période d’observation de l’étude MG0003 ou c) participant à l’étude ayant effectué au moins 6 visites dans l’étude MG0004 - Poids corporel ≥ 35 kg à l’inclusion (Jour 1) - Hommes ou femmes |
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E.4 | Principal exclusion criteria |
- Study participant has a known hypersensitivity to any components of the study medication - Evidence of active or latent tuberculosis (TB) as documented by medical history and examination, if applicable, chest x-rays (posterior anterior and lateral), and TB testing by a positive (not indeterminate) QuantiFERON®-TB Gold Plus - Study participant met any mandatory withdrawal or mandatory study drug discontinuation criteria in MG0003, MGC003, or MG0004, or permanently discontinued study drug in either study - Study participant intends to have a live vaccination during the course of the study or within 8 weeks following the final dose of rozanolixizumab - Study participant with severe (defined as Grade 3 on the Myasthenia Gravis-Activities of Daily Living (MG-ADL) scale) weakness affecting oropharyngeal or respiratory muscles, or who has myasthenic crisis or impending crisis |
- Hypersensibilité connue à l’un des composants du médicament à l’étude - Signes de tuberculose (TB) active ou latente, documentés par les antécédents médicaux et l’examen, le cas échéant, la radiographie thoracique (face et profil) et résultat du dépistage de TB positif (non indéterminé) par le test QuantiFERON®-TB Gold Plus - Présence d’un critère de retrait obligatoire ou d’arrêt obligatoire du médicament à l’étude dans l’étude MG0003, MGC003 ou MG0004, ou arrêt définitif du médicament à l’étude dans une de ces études. - Tentative d’administration d’un vaccin vivant pendant l’étude ou dans les 8 semaines suivant la dose finale de rozanolixizumab - Faiblesse sévère (définie comme de Grade 3 sur l’échelle MG-AVQ) affectant les muscles oropharyngés ou respiratoires ou présentant une crise myasthénique ou une crise imminente |
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E.5 End points |
E.5.1 | Primary end point(s) |
1. Percentage of participants with treatment-emergent adverse events (TEAEs) 2. Percentage of participants with (TEAEs) leading to permanent withdrawal of investigational medicinal product (IMP)
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1. Pourcentage de participants avec survenue d'événements indésirables apparaissant sous traitement (EIAT) 2. Pourcentage de participants avec survenue d'EIAT conduisant à un retrait définitif du médicament à l'étude |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
1.; 2. From Baseline (Day 1) to End of Study (average of 20 months)
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1.; 2. De l'inclusion jusqu'à la fin d'étude (moyenne de 20 mois) |
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E.5.2 | Secondary end point(s) |
1. Change from Baseline (Day 1) to Day 43 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) score within one treatment cycle 2. Change from Baseline (Day 1) to Day 43 in Quantitative Myasthenia Gravis (QMG) score within one treatment cycle 3. Change from Baseline (Day 1) to Day 43 in Myasthenia Gravis-Composite (MG-C) score within one treatment cycle 4. Change from Baseline (Day 1) to Day 43 in Myasthenia Gravis (MG) Symptoms Patient Reported Outcome (PRO) ‘Muscle Weakness Fatigability’ score within one treatment cycle 5. Change from Baseline (Day 1) to Day 43 in MG Symptoms PRO ‘Physical Fatigue’ score within one treatment cycle 6. Change from Baseline (Day 1) to Day 43 in MG Symptoms PRO ‘Bulbar symptoms’ score within one treatment cycle 7. MG-ADL responder (≥2.0-point improvement from Baseline [Day 1] to end of Day 43) within one treatment cycle 8. Time to MG-ADL response (≥2.0-point improvement from Baseline [Day 1]) within one treatment cycle 9. Time between consecutive treatment cycles |
1. Variation entre l’inclusion (Jour 1) et le Jour 43 du score au Questionnaire Myasthénie grave activités de la vie quotidienne [MG-AVQ] (MG-ADL, Myasthenia Gravis-Activities of Daily Living) au cours d’un cycle de traitement 2. Variation entre l’inclusion (Jour 1) et le Jour 43 du score à l’échelle quantitative de myasthénie grave (QMG, Quantitative Myasthenia Gravis) au cours d’un cycle de traitement 3. Variation entre l’inclusion (Jour 1) et le Jour 43 du score à l’échelle composite de myasthénie grave (MG-C, Myasthenia Gravis Composite) au cours d’un cycle de traitement 4. Variation entre l’inclusion (Jour 1) et le Jour 43 du score de résultat rapporté par le patient (PRO, patient reported outcome) concernant les symptômes de MG « Fatigabilité faiblesse musculaires » au cours d’un cycle de traitement 5. Variation entre l’inclusion (Jour 1) et le Jour 43 du score PRO des symptômes de MG « Fatigue physique », au cours d’un cycle de traitement 6. Variation entre l’inclusion (Jour 1) et le Jour 43 du score PRO des symptômes de MG « Symptômes bulbaires », au cours d’un cycle de traitement |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
1. - 8. From Baseline (Day 1) to end of treatment cycle (up to 6 weeks) 9. From end of the previous treatment cycle (Day 43) to the next treatment cycle (Day 1)
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1. - 8. À partir de l'inclusion (Jour 1) jusqu’à la fin du cycle de traitement (jusqu’à 6 semaines) 9. De la fin du cycle de traitement précédent (Jour 43) au cycle de traitement suivant (Jour 1) |
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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 | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Yes |
E.6.13.1 | Other scope of the trial description |
Tolerability, Immunogenicity
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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 | No |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
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 | No |
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 | 8 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 61 |
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 |
Belgium |
Canada |
China |
Czech Republic |
Denmark |
France |
Georgia |
Germany |
Hungary |
Italy |
Japan |
Poland |
Russian Federation |
Serbia |
Spain |
United Kingdom |
United States |
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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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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | 3 |
E.8.9.1 | In the Member State concerned days | 13 |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 4 |
E.8.9.2 | In all countries concerned by the trial days | 13 |