Eine offene Phase-3-Verlängerungsstudie zur Bewertung von 6-wöchigen Behandlungszyklen mit Rozanolixizumab bei pädiatrischen Studienteilnehmern mit generalisierter Myasthenia gravis
Eine offene Verlängerungsstudie zur Bewertung von Rozanolixizumab bei pädiatrischen Studienteilnehmern mit generalisierter Myasthenia gravis
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.
Studienübersicht
Status
Bedingungen
Intervention / Behandlung
Studientyp
Einschreibung (Tatsächlich)
Phase
- Phase 3
Kontakte und Standorte
Studienorte
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Bologna, Italien
- Mg0008 40290
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Milan, Italien
- Mg0008 40144
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Naples, Italien
- Mg0008 40733
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Fuchu-shi, Japan
- Mg0008 20340
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Sagamihara, Japan
- Mg0008 20343
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Ōbu, Japan
- Mg0008 20339
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Lodz, Polen
- Mg0008 40734
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Warsaw, Polen
- Mg0008 40155
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Yenimahalle, Türkei (türkiye)
- Mg0008 40841
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Teilnahmekriterien
Zulassungskriterien
Studienberechtigtes Alter
Akzeptiert gesunde Freiwillige
Studienberechtigte Geschlechter
Beschreibung
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
Studienplan
Wie ist die Studie aufgebaut?
Designdetails
- Hauptzweck: Behandlung
- Zuteilung: N / A
- Interventionsmodell: Einzelgruppenzuweisung
- Maskierung: Keine (Offenes Etikett)
Waffen und Interventionen
Teilnehmergruppe / Arm |
Intervention / Behandlung |
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Experimental: 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
Andere Namen:
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Was misst die Studie?
Primäre Ergebnismessungen
Ergebnis Maßnahme |
Maßnahmenbeschreibung |
Zeitfenster |
|---|---|---|
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Occurrence of serious Treatment-Emergent Adverse Events (TEAEs) up to the End of Study (EOS) Visit
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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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Sekundäre Ergebnismessungen
Ergebnis Maßnahme |
Maßnahmenbeschreibung |
Zeitfenster |
|---|---|---|
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Percent change in total Immunoglobulin G (IgG) from Baseline to the end of Week 6 of each Treatment Period (TP)
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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
Zeitfenster: 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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Mitarbeiter und Ermittler
Sponsor
Ermittler
- Studienleiter: UCB Cares, 001 844 599 2273
Studienaufzeichnungsdaten
Haupttermine studieren
Studienbeginn (Tatsächlich)
Primärer Abschluss (Geschätzt)
Studienabschluss (Geschätzt)
Studienanmeldedaten
Zuerst eingereicht
Zuerst eingereicht, das die QC-Kriterien erfüllt hat
Zuerst gepostet (Tatsächlich)
Studienaufzeichnungsaktualisierungen
Letztes Update gepostet (Tatsächlich)
Letztes eingereichtes Update, das die QC-Kriterien erfüllt
Zuletzt verifiziert
Mehr Informationen
Begriffe im Zusammenhang mit dieser Studie
Schlüsselwörter
Zusätzliche relevante MeSH-Bedingungen
- Erkrankungen des Nervensystems
- Neubildungen nach Standort
- Neubildungen
- Neuromuskuläre Erkrankungen
- Autoimmunerkrankungen
- Erkrankungen des Immunsystems
- Autoimmunerkrankungen des Nervensystems
- Neurodegenerative Krankheiten
- Paraneoplastische Syndrome, Nervensystem
- Neubildungen des Nervensystems
- Paraneoplastische Syndrome
- Erkrankungen der neuromuskulären Verbindungen
- Myasthenia gravis
- Rozanolixizumab
Andere Studien-ID-Nummern
- MG0008
- U1111-1286-3581 (Andere Kennung: WHO universal trial number (UTN))
- 2022-502075-34 (Registrierungskennung: EU CTIS)
Plan für individuelle Teilnehmerdaten (IPD)
Planen Sie, individuelle Teilnehmerdaten (IPD) zu teilen?
Beschreibung des IPD-Plans
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.
IPD-Sharing-Zeitrahmen
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.
IPD-Sharing-Zugriffskriterien
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.
Art der unterstützenden IPD-Freigabeinformationen
- STUDIENPROTOKOLL
- SAFT
- CSR
Arzneimittel- und Geräteinformationen, Studienunterlagen
Studiert ein von der US-amerikanischen FDA reguliertes Arzneimittelprodukt
Studiert ein von der US-amerikanischen FDA reguliertes Geräteprodukt
Produkt, das in den USA hergestellt und aus den USA exportiert wird
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