E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Generalized Myasthenia Gravis |
Miastenia gravis generalizzata |
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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. |
La miastenia gravis è una malattia autoimmune che causa debolezza nei tuoi muscoli; è causata da un problema di comunicazione tra i nervi e i muscoli. |
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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 | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To evaluate the ability of study participants with generalized Myasthenia Gravis (gMG) to successfully self-administer rozanolixizumab after training in the self-administration technique using the syringe driver and manual push methods |
Valutare la capacità dei partecipanti allo studio con miastenia gravis generalizzata (gMG) di auto- somministrarsi con successo rozanolixizumab dopo essere stati addestrati nella tecnica di auto-somministrazione con i metodi della pompa a siringa e della spinta manuale. |
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E.2.2 | Secondary objectives of the trial |
To evaluate the safety of subcutaneous (SC) self-administration |
Valutare la sicurezza dell’auto-somministrazione sottocutanea (SC) di rozanolixizumab |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Study participant must have a documented diagnosis of generalized Myasthenia Gravis (gMG) - Study participant is willing to perform and capable of performing home self-administration - Study participant is considered for additional rozanolixizumab treatment with the posology proposed in this study - Body weight >=35 kg - Study participants may be male or female |
- Il partecipante allo studio deve avere una diagnosi documentata di miastenia gravis generalizzata (gMG) - Il partecipante allo studio è disposto ad eseguire ed è in grado di eseguire l'auto-somministrazione domiciliare - Il partecipante allo studio è considerato per un ulteriore trattamento con rozanolixizumab con la posologia proposta in questo studio - Peso corporeo >=35 kg - I partecipanti allo studio possono essere maschi o femmine |
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E.4 | Principal exclusion criteria |
- Study participant has a known hypersensitivity to any components of the study medication or other anti-neonatal Fc receptor (FcRn) medications - Study participant with a known tuberculosis (TB) infection, at high risk of acquiring TB infection, or latent tuberculosis infection (LTBI), or current or history of nontuberculous mycobacterial infection (NTMBI) - Study participant has a clinically relevant active infection or a history of serious infection (resulting in hospitalization or requiring IV antibiotic treatment) within 6 weeks before the Baseline Visit - The study participant previously participated in any rozanolixizumab MG study and met any mandatory withdrawal or mandatory study drug discontinuation criteria - Study participant has received a live vaccination within 4 weeks before starting treatment, or a Bacillus Calmette Guérin (BCG) vaccine within 1 year before starting treatment; or intends to have a live vaccination during the course of the study or within 8 weeks following the last 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 |
- Il partecipante allo studio ha una nota ipersensibilità a qualsiasi componente del farmaco in studio o ad altri farmaci anti-FcRn - Partecipante allo studio con infezione nota da tubercolosi (TB), ad alto rischio di contrarre infezione da tubercolosi, o infezione tubercolare latente (LTBI), o attuale o storia di infezione micobatterica non tubercolare (NTMBI) - Il partecipante allo studio ha un'infezione attiva clinicamente rilevante o una storia di infezione grave (con conseguente ospedalizzazione o che richiede un trattamento antibiotico per via endovenosa) entro 6 settimane prima della visita di base - Il partecipante allo studio ha precedentemente partecipato a qualsiasi studio MG con rozanolixizumab e ha soddisfatto qualsiasi criterio per il ritiro obbligatorio o sospensione obbligatoria del farmaco in studio - Il partecipante allo studio ha ricevuto una vaccinazione viva entro 4 settimane prima di iniziare il trattamento o un vaccino contro il Bacillus Calmette Guérin (BCG) entro 1 anno prima di iniziare il trattamento; o intende sottoporsi a una vaccinazione viva nel corso dello studio o entro 8 settimane dall'ultima dose di rozanolixizumab - Il partecipante allo studio ha grave debolezza (definita come Grado 3 sulla scala delle attività di vita quotidiana della miastenia gravis (MG-ADL)) che colpisce i muscoli orofaringei o respiratori, o che ha crisi miasteniche o crisi imminenti |
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E.5 End points |
E.5.1 | Primary end point(s) |
1. Successful self-administration of rozanolixizumab (with correct use of syringe driver and manual push, respectively) during the Self-administration Period at Visit 13 2. Successful self-administration of rozanolixizumab (with correct use of syringe driver and manual push, respecively) during the Self-administration Period at Visit 19 |
1. Efficacia dell’auto-somministrazione di rozanolixizumab (con uso corretto rispettivamente della pompa a siringa e della spinta manuale) durante il periodo di auto-somministrazione alla Visita 13 2. Efficacia dell’auto-somministrazione di rozanolixizumab (con uso corretto rispettivamente della pompa a siringa e della spinta manuale) durante il periodo di auto-somministrazione alla Visita 19 |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
1. Visit 13 (Week 12; last dose of Self-administration Period 1) 2. Visit 19 (Week 18; last dose of Self-administration Period 2) |
1. Visita 13 (Settimana 12; ultima dose del periodo di auto-somministrazione 1) 2. Visita 19 (Settimana 18; ultima dose del periodo di auto- somministrazione 2) |
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E.5.2 | Secondary end point(s) |
1. Occurrence of Treatment-Emergent Adverse Events (TEAEs) after syringe driver or manual push self-administration from Visit 2 up to the End of Study Visit 2. Occurrence of local site reactions up to 24 hours after each administration during the Training Period and Self-administration Periods 3. Occurrence of medication errors associated with adverse reactions during the 2 Self-administration Periods of the study |
1. Frequenza di eventi avversi emergenti dal trattamento (TEAE) dopo l’auto-somministrazione con pompa a siringa o mediante spinta manuale dalla Visita 2 fino alla Visita di fine studio 2. Frequenza di reazioni locali nel sito di iniezione fino a 24 ore dopo ciascuna somministrazione durante il Periodo di formazione e i periodi di auto-somministrazione. 3. Frequenza di errori terapeutici associati a reazioni avverse durante i 2 periodi di auto-somministrazione dello studio. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
1. From Visit 2 (Week 1) up to the End of Study Visit (Visit 21 (Week 26)) 2. Up to 24 hours after each administration during the Training Period (Baseline to Visit 7 (Week 6) and Self-administration Periods (Visit 8 (Week 7) to Visit 19 (Week 18)) 3. During the Self-administration Periods (Visit 8 (Week 7) to Visit 19 (Week 18)) |
1. Dalla Visita 2 (Settimana 1) fino alla Visita di fine studio (Visita 21 [Settimana 26]) 2. Fino a 24 ore dopo ciascuna somministrazione durante il Periodo di formazione (dalla visita base alla Visita 7 (Settimana 6) e i periodi di auto-somministrazione (dalla Visita 8 (Settimana 7) alla Visita 19 (Settimana 18)). 3. Durante i periodi di auto-somministrazione (dalla Visita 8 (Settimana 7) alla Visita 19 (Settimana 18)). |
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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 | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
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 | 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) | 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 | 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 | Yes |
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 | Yes |
E.8.2.3.1 | Comparator description |
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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 | 12 |
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 | Information not present in EudraCT |
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 |
Canada |
Japan |
United States |
Poland |
Spain |
Germany |
Italy |
Georgia |
United Kingdom |
Serbia |
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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 | 1 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 1 |
E.8.9.2 | In all countries concerned by the trial days | 20 |