Une étude d'extension ouverte de phase 3 évaluant les cycles de traitement de 6 semaines par le rozanolixizumab chez les participants à l'étude pédiatrique atteints de myasthénie grave généralisée
Une étude d'extension ouverte pour évaluer le rozanolixizumab chez les participants à l'étude pédiatrique atteints de myasthénie grave généralisée
The purpose of the study is to assess the long term safety and tolerability of additional 6-week treatment cycles with rozanolixizumab in pediatric participants with generalized Myasthenia Gravis (gMG) aged ≥2 at the time of the Screening Visit of MG0006.
Aperçu de l'étude
Statut
Les conditions
Intervention / Traitement
Type d'étude
Inscription (Réel)
Phase
- Phase 3
Contacts et emplacements
Lieux d'étude
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Bologna, Italie
- Mg0008 40290
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Milan, Italie
- Mg0008 40144
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Naples, Italie
- Mg0008 40733
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Fuchu-shi, Japon
- Mg0008 20340
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Sagamihara, Japon
- Mg0008 20343
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Ōbu, Japon
- Mg0008 20339
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Lodz, Pologne
- Mg0008 40734
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Warsaw, Pologne
- Mg0008 40155
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Yenimahalle, Turquie (Türkiye)
- Mg0008 40841
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Critères de participation
Critère d'éligibilité
Âges éligibles pour étudier
Accepte les volontaires sains
Sexes éligibles pour l'étude
La description
Inclusion Criteria:
Study participant must meet one of the following:
- Study participant completed MG0006 according to the protocol
- Study participant completed the MG0006 Treatment Period and has a worsening of generalized myasthenia gravis (gMG) symptoms in the Observation Period of MG0006
Exclusion Criteria:
- Study participant met any mandatory withdrawal or mandatory permanent investigational medicinal product (IMP) discontinuation criteria in MG0006 or permanently discontinued IMP
- Study participant has a known hypersensitivity to any components of the IMP or other neonatal Fc receptor (FcRn) drugs
- Study participant has any laboratory abnormality that, in the opinion of the Investigator, is clinically significant, has not resolved at Baseline, and could jeopardize or compromise the study participant's ability to participate in this study
Plan d'étude
Comment l'étude est-elle conçue ?
Détails de conception
- Objectif principal: Traitement
- Répartition: N / A
- Modèle interventionnel: Affectation à un seul groupe
- Masquage: Aucun (étiquette ouverte)
Armes et Interventions
Groupe de participants / Bras |
Intervention / Traitement |
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Expérimental: rozanolixizumab
Study participants will receive pre-defined doses of rozanolixizumab. Each treatment cycle in each Treatment Period (TP) consists of 6 subcutaneous (sc) administrations of rozanolixizumab at 1-week intervals. Each Treatment Period will be initiated upon the discretion of the Investigator based on the medical needs of the study-participant. |
rozanolixizumab solution for injection
Autres noms:
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Que mesure l'étude ?
Principaux critères de jugement
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
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Occurrence of serious Treatment-Emergent Adverse Events (TEAEs) up to the End of Study (EOS) Visit
Délai: From Baseline up to the EOS Visit (up to 52 weeks)
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Serious TEAEs are any untoward medical incidence in a subject during administered study treatment, whether or not these events are related to study treatment and additionally are emergent untoward medical occurrence that at any dose:
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From Baseline up to the EOS Visit (up to 52 weeks)
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Occurrence of TEAEs leading to permanent withdrawal of IMP up to the EOS Visit
Délai: From Baseline up to the EOS Visit (up to 52 weeks)
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An Adverse Event (AE) is any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product. |
From Baseline up to the EOS Visit (up to 52 weeks)
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Occurrence of Adverse Event(s) of Special Monitoring (AESM) up to the EOS Visit
Délai: From Baseline up to the EOS Visit (up to 52 weeks)
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AESMs are: Severe and/or serious headache, suspected aseptic meningitis, severe Gastrointestinal (GI) disorders, and opportunistic infection. |
From Baseline up to the EOS Visit (up to 52 weeks)
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Mesures de résultats secondaires
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
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Percent change in total Immunoglobulin G (IgG) from Baseline to the end of Week 6 of each Treatment Period (TP)
Délai: From Baseline to the end of Week 6 of each TP (up to 52 weeks)
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Plasma concentration analyses of total IgG will be done for all study participants on an ongoing basis to the end of Week 6 of each TP. |
From Baseline to the end of Week 6 of each TP (up to 52 weeks)
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Absolute change in total IgG from Baseline to the end of Week 6 of each TP
Délai: From Baseline to the end of Week 6 of each TP (up to 52 weeks)
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Plasma concentration analyses of total IgG will be done for all study participants on an ongoing basis to the end of Week 6 of each TP. |
From Baseline to the end of Week 6 of each TP (up to 52 weeks)
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Change from Baseline in Myasthenia Gravis-Activities of Daily Living (MG ADL) total score at the end of Week 6 of each TP
Délai: From Baseline to the end of Week 6 of each TP (up to 52 weeks)
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The MG-ADL score is an 8-item patient-reported outcome (PRO) instrument. The MG-ADL targets symptoms and disability across ocular, bulbar, respiratory, and axial symptoms. The item responses are scored from 0 to 3, and the total score of MG-ADL is the sum of the 8 items and ranges from 0 to 24, with a higher score indicating more disability. |
From Baseline to the end of Week 6 of each TP (up to 52 weeks)
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Change from Baseline in Quantitative Myasthenia Gravis (QMG) total score at the end of Week 6 of each TP
Délai: From Baseline to the end of Week 6 of each TP (up to 52 weeks)
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QMG score is a standardized and validated quantitative strength scoring system that was developed specifically for MG. The QMG total score is obtained by summing the responses to each individual item (13 items; Responses: None=0, Mild=1, Moderate=2, Severe=3). The score ranges from 0 to 39, with lower scores indicating lower disease activity. |
From Baseline to the end of Week 6 of each TP (up to 52 weeks)
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Collaborateurs et enquêteurs
Parrainer
Les enquêteurs
- Directeur d'études: UCB Cares, 001 844 599 2273
Dates d'enregistrement des études
Dates principales de l'étude
Début de l'étude (Réel)
Achèvement primaire (Estimé)
Achèvement de l'étude (Estimé)
Dates d'inscription aux études
Première soumission
Première soumission répondant aux critères de contrôle qualité
Première publication (Réel)
Mises à jour des dossiers d'étude
Dernière mise à jour publiée (Réel)
Dernière mise à jour soumise répondant aux critères de contrôle qualité
Dernière vérification
Plus d'information
Termes liés à cette étude
Termes MeSH pertinents supplémentaires
- Maladies du système nerveux
- Tumeurs par site
- Tumeurs
- Maladies neuromusculaires
- Maladies auto-immunes
- Maladies du système immunitaire
- Maladies auto-immunes du système nerveux
- Maladies neurodégénératives
- Syndromes paranéoplasiques, système nerveux
- Tumeurs du système nerveux
- Syndromes paranéoplasiques
- Maladies de la jonction neuromusculaire
- Myasthénie grave
- rozanolixizumab
Autres numéros d'identification d'étude
- MG0008
- U1111-1286-3581 (Autre identifiant: WHO universal trial number (UTN))
- 2022-502075-34 (Identificateur de registre: EU CTIS)
Plan pour les données individuelles des participants (IPD)
Prévoyez-vous de partager les données individuelles des participants (DPI) ?
Description du régime IPD
Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe, or global development is discontinued, and 18 months after trial completion. Investigators may request access to anonymized individual patient-level data and redacted trial documents which may include: analysis-ready datasets, study protocol, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a prespecified time, typically 12 months, on a password protected portal. This plan may change if the risk of re-identifying trial participants is determined to be too high after the trial is completed; in this case and to protect participants, individual patient-level data would not be made available.
Délai de partage IPD
Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe or global development is discontinued, and 18 months after trial completion.
Critères d'accès au partage IPD
Qualified researchers may request access to anonymized IPD and redacted study documents which may include: raw datasets, analysis-ready datasets, study protocol, blank case report form, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed.All documents are available in English only, for a pre-specified time, typically 12 months, on a password protected portal.
Type d'informations de prise en charge du partage d'IPD
- PROTOCOLE D'ÉTUDE
- SÈVE
- RSE
Informations sur les médicaments et les dispositifs, documents d'étude
Étudie un produit pharmaceutique réglementé par la FDA américaine
Étudie un produit d'appareil réglementé par la FDA américaine
produit fabriqué et exporté des États-Unis.
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