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En undersøgelse til evaluering af subkutan Zilucoplan hos pædiatriske deltagere med generaliseret myasthenia gravis (ziMyG)

4. november 2026 opdateret af: UCB Biopharma SRL

En multicenter open-label, ukontrolleret undersøgelse til evaluering af farmakokinetik, farmakodynamik, sikkerhed, tolerabilitet og aktivitet af Zilucoplan i pædiatriske undersøgelsesdeltagere fra 2 til under 18 år med acetylcholinreceptorantistof Positiv generaliseret myasthenia gravis

The purpose of this study is to assess the pharmacokinetics, pharmacodynamics, safety, tolerability, immunogenicity and activity of zilucoplan (ZLP) in pediatric study participants with generalized myasthenia gravis (gMG).

Studieoversigt

Status

Rekruttering

Betingelser

Intervention / Behandling

Undersøgelsestype

Interventionel

Tilmelding (Anslået)

8

Fase

  • Fase 2
  • Fase 3

Kontakter og lokationer

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

Studiekontakt

  • Navn: UCB Cares
  • Telefonnummer: 001 844 599 2273

Undersøgelse Kontakt Backup

Studiesteder

  • Det Forenede Kongerige
      • Glasgow, Det Forenede Kongerige
        • Rekruttering
        • Mg0014 40735
      • London, Det Forenede Kongerige
        • Rekruttering
        • Mg0014 40736
  • Forenede Stater
    • Illinois
      • Chicago, Illinois, Forenede Stater, 60611
        • Trukket tilbage
        • Mg0014 50168
    • Texas
      • Denton, Texas, Forenede Stater, 76208
        • Trukket tilbage
        • Mg0014 50574
  • Italien
      • Milan, Italien
        • Rekruttering
        • Mg0014 40144
  • Polen
      • Katowice, Polen
        • Rekruttering
        • Mg0014 40774
      • Warsaw, Polen
        • Aktiv, ikke rekrutterende
        • Mg0014 40218
  • Sydkorea
      • Seoul, Sydkorea
        • Rekruttering
        • Mg0014 20220
      • Seoul, Sydkorea
        • Trukket tilbage
        • Mg0014 20104

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

12 år til 17 år (Barn)

Tager imod sunde frivillige

Ingen

Køn, der er berettiget til at studere

Alle

Beskrivelse

Inclusion Criteria:

United States of America (USA) specific inclusion criterion:

- Participant must be 12 to <18 years of age at the time of signing the Informed consent/assent according to local regulation

Rest of world (ROW) specific inclusion criterion:

- Participant must be 2 to <18 years of age at the time of signing the Informed consent/assent according to local regulation

Global inclusion criteria:

  • Participant has a diagnosis of generalized myasthenia gravis (gMG) confirmed by a prior positive serologic test result to acetylcholine receptor (AChR) prior to Screening
  • Participant meets the criteria as defined by the Myasthenia Gravis Foundation of America (MGFA) Clinical Classification II to IV at Screening
  • Participants with gMG, including:
  • An MG-activities of daily living (MG-ADL) total score of 6 or more in adolescents from 12 years to <18 years of age at Screening
  • Documented weakness in at least 1 limb, neck, or bulbar muscle in children from 2 years to <12 years of age at Screening (does not apply to US)
  • Documented vaccination against meningococcal infections within 3 years prior to study start. If not fully vaccinated, participants must receive appropriate prophylactic antibiotic treatment until at least 2 weeks after the initial dose of vaccine(s)

Exclusion Criteria:

  • Participant has known positive serology for muscle-specific kinase
  • Participant has any medical or psychiatric condition that, in the opinion of the Investigator, could jeopardize or would compromise the participant's ability to participate in this study
  • Participant has had a thymectomy within 6 months prior to Baseline
  • Participant has minimal Manifestation Status of MG based on the clinical judgement of the Investigator
  • Current or recent systemic infection within 2 weeks prior to Baseline or infection requiring intravenous antibiotics within 4 weeks prior to Baseline

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: Zilucoplan Arm

Study participants will receive zilucoplan in pre-defined dose based on their weight.
Medicin: Zilucoplan

Zilucoplan will be administered subcutaneously to pediatric study participants.

Andre navne:
  • RA101495

Hvad måler undersøgelsen?

Primære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Plasma concentrations of zilucoplan (ZLP) sampled at Week 4 (Day 29)
Tidsramme: Week 4 (Day 29)

Blood samples will be collected for measurement of plasma concentrations of ZLP on Day 29 predose.
Week 4 (Day 29)
Change from Baseline in sheep red blood cell (sRBC) lysis at Week 4 (Day 29)
Tidsramme: Week 4 (Day 29)

Samples for measurement of sRBC lysis will be collected on Day 29 predose.
Week 4 (Day 29)
Change from Baseline in complement component 5 (C5) levels at Week 4 (Day 29)
Tidsramme: Week 4 (Day 29)

Samples for measurement of C5 will be collected on Day 29 predose.
Week 4 (Day 29)

Sekundære resultatmål

Resultatmål
Foranstaltningsbeskrivelse
Tidsramme
Occurence of treatment-emergent adverse events (TEAEs) during the course of the study
Tidsramme: From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)

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 (Day 1) to Safety-Follow-Up Visit (up to Week 15)
Occurrence of treatment-emergent serious adverse events (TESAEs)
Tidsramme: From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)

A serious adverse event (SAE) is defined as any untoward medical occurrence that, at any dose:

Results in death Is life-threatening Requires inpatient hospitalization or prolongation of existing hospitalization Results in persistent disability/incapacity Is a congenital anomaly/birth defect Important medical events
From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)
Occurrence of TEAEs leading to permanent withdrawal of investigational medicinal product (IMP)
Tidsramme: From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)

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. AEs leading to permanent withdrawal of study medication.
From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)
Occurrence of treatment-emergent infections
Tidsramme: From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)

Percentage of participants who experienced treatment-emergent infections as adverse events.

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.
From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)
Occurrence of antidrug antibody (ADA) and anti- polyethylene glycol (PEG) antibodies at Week 4 (Day 29)
Tidsramme: Week 4 (Day 29)

ADA and anti-PEG antibodies will be evaluated in serum samples.
Week 4 (Day 29)
Change in MG-activities of daily living (MG-ADL) score from Baseline to Week 4 (Day 29).
Tidsramme: Week 4 (Day 29)

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.
Week 4 (Day 29)
Change in Quantitative MG (QMG) score from Baseline to Week 4 (Day 29)
Tidsramme: Week 4 (Day 29)

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.
Week 4 (Day 29)
Myasthenia Gravis Foundation of America Post-Interventional Status (MGFA-PIS) at Week 4 (Day 29)
Tidsramme: Week 4 (Day 29)

The MGFA-PIS is a physician-determined assessment of clinical symptoms of MG after initiation of MG specific therapy. For the purpose of the current study, Minimal Manifestation will be determined at each scheduled time point after treatment initiation (rather than after 1 year). Change in status (improved, unchanged, worse, exacerbation, or died of MG) will also be determined.
Week 4 (Day 29)
Change in Pediatric Quality of Life Inventory (PedsQoL), Version 4 domain scores from Baseline to Week 4 (Day 29)
Tidsramme: Week 4 (Day 29)

The PedsQoL generic core scale (Version 4) is a validated instrument that is suitable for use with pediatric populations. PedsQoL generic core scales assess Physical Functioning, Emotional Functioning, Social Functioning, and School Functioning. The scale has 23 items with a score range of 0 to 4. Following transformation, the score range of each domain as well as the total score is 0-100 with higher scores indicating higher HRQoL.
Week 4 (Day 29)

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)

16. oktober 2024

Primær færdiggørelse (Anslået)

12. maj 2027

Studieafslutning (Anslået)

22. juni 2027

Datoer for studieregistrering

Først indsendt

20. september 2023

Først indsendt, der opfyldte QC-kriterier

20. september 2023

Først opslået (Faktiske)

28. september 2023

Opdateringer af undersøgelsesjournaler

Sidste opdatering sendt (Faktiske)

5. november 2026

Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier

4. november 2026

Sidst verificeret

1. november 2026

Mere information

Begreber relateret til denne undersøgelse

Nøgleord

Yderligere relevante MeSH-vilkår

Andre undersøgelses-id-numre

  • MG0014
  • U1111-1290-3349 (Anden identifikator: World Health Organization (WHO))
  • 2022-502072-23 (Registry Identifier: EU Clinical Trials)

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 study 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 .

Abonner