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Et fase 3 åbent forlængelsesstudie, der evaluerer 6-ugers behandlingscyklusser af Rozanolixizumab i pædiatriske undersøgelsesdeltagere med generaliseret myasthenia gravis

5. november 2026 opdateret af: UCB Biopharma SRL

Et åbent udvidelsesstudie til evaluering af Rozanolixizumab i pædiatriske undersøgelsesdeltagere med generaliseret 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.

Studieoversigt

Status

Aktiv, ikke rekrutterende

Betingelser

Intervention / Behandling

Undersøgelsestype

Interventionel

Tilmelding (Faktiske)

13

Fase

  • Fase 3

Kontakter og lokationer

Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.

Studiesteder

  • Italien
      • Bologna, Italien
        • Mg0008 40290
      • Milan, Italien
        • Mg0008 40144
      • Naples, Italien
        • Mg0008 40733
  • Japan
      • Fuchu-shi, Japan
        • Mg0008 20340
      • Sagamihara, Japan
        • Mg0008 20343
      • Ōbu, Japan
        • Mg0008 20339
  • Polen
      • Lodz, Polen
        • Mg0008 40734
      • Warsaw, Polen
        • Mg0008 40155
  • Tyrkiet (Türkiye)
      • Yenimahalle, Tyrkiet (Türkiye)
        • Mg0008 40841

Deltagelseskriterier

Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.

Berettigelseskriterier

Aldre berettiget til at studere

2 år og ældre (Barn, Voksen, Ældre voksen)

Tager imod sunde frivillige

Ingen

Køn, der er berettiget til at studere

Alle

Beskrivelse

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

Studieplan

Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.

Hvordan er undersøgelsen tilrettelagt?

Design detaljer

  • Primært formål: Behandling
  • Tildeling: N/A
  • Interventionel model: Enkelt gruppeopgave
  • Maskning: Ingen (Åben etiket)

Våben og indgreb

Deltagergruppe / Arm
Intervention / Behandling
Eksperimentel: 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.
Medicin: rozanolixizumab

rozanolixizumab solution for injection

Andre navne:
  • UCB7665

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Occurrence of serious Treatment-Emergent Adverse Events (TEAEs) up to the End of Study (EOS) Visit
Tidsramme: From Baseline up to the EOS Visit (up to 52 weeks)

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:

  • Results in death
  • Is life-threatening
  • Requires in patient hospitalisation or prolongation of existing hospitalisation
  • Results in persistent disability/incapacity
  • Is a congenital anomaly or birth defect
  • Important medical events
From Baseline up to the EOS Visit (up to 52 weeks)
Occurrence of TEAEs leading to permanent withdrawal of IMP up to the EOS Visit
Tidsramme: From Baseline up to the EOS Visit (up to 52 weeks)

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)
Occurrence of Adverse Event(s) of Special Monitoring (AESM) up to the EOS Visit
Tidsramme: From Baseline up to the EOS Visit (up to 52 weeks)

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)

Sekundære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Percent change in total Immunoglobulin G (IgG) from Baseline to the end of Week 6 of each Treatment Period (TP)
Tidsramme: From Baseline to the end of Week 6 of each TP (up to 52 weeks)

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)
Absolute change in total IgG from Baseline to the end of Week 6 of each TP
Tidsramme: From Baseline to the end of Week 6 of each TP (up to 52 weeks)

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)
Change from Baseline in Myasthenia Gravis-Activities of Daily Living (MG ADL) total score at the end of Week 6 of each TP
Tidsramme: From Baseline to the end of Week 6 of each TP (up to 52 weeks)

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)
Change from Baseline in Quantitative Myasthenia Gravis (QMG) total score at the end of Week 6 of each TP
Tidsramme: From Baseline to the end of Week 6 of each TP (up to 52 weeks)

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)

Samarbejdspartnere og efterforskere

Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.

Sponsor

UCB Biopharma SRL

Efterforskere

  • Studieleder: UCB Cares, 001 844 599 2273

Datoer for undersøgelser

Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.

Studer store datoer

Studiestart (Faktiske)

8. oktober 2024

Primær færdiggørelse (Anslået)

5. oktober 2027

Studieafslutning (Anslået)

5. oktober 2027

Datoer for studieregistrering

Først indsendt

22. juli 2024

Først indsendt, der opfyldte QC-kriterier

1. august 2024

Først opslået (Faktiske)

6. august 2024

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

6. november 2026

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

5. november 2026

Sidst verificeret

1. november 2026

Mere information

Begreber relateret til denne undersøgelse

Nøgleord

Yderligere relevante MeSH-vilkår

Andre undersøgelses-id-numre

  • MG0008
  • U1111-1286-3581 (Anden identifikator: WHO universal trial number (UTN))
  • 2022-502075-34 (Registry Identifier: EU CTIS)

Plan for individuelle deltagerdata (IPD)

Planlægger du at dele individuelle deltagerdata (IPD)?

JA

IPD-planbeskrivelse

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-delingstidsramme

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-delingsadgangskriterier

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.

IPD-deling Understøttende informationstype

  • STUDY_PROTOCOL
  • SAP
  • CSR

Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter

Studerer et amerikansk FDA-reguleret lægemiddelprodukt

Ja

Studerer et amerikansk FDA-reguleret enhedsprodukt

Ingen

produkt fremstillet i og eksporteret fra U.S.A.

Ingen

Disse oplysninger blev hentet direkte fra webstedet clinicaltrials.gov uden ændringer. Hvis du har nogen anmodninger om at ændre, fjerne eller opdatere dine undersøgelsesoplysninger, bedes du kontakte register@clinicaltrials.gov. Så snart en ændring er implementeret på clinicaltrials.gov, vil denne også blive opdateret automatisk på vores hjemmeside .

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