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En studie for å evaluere subkutan Zilucoplan hos pediatriske deltakere med generalisert myasthenia gravis (ziMyG)

4. november 2026 oppdatert av: UCB Biopharma SRL

En multisenter åpen, ukontrollert studie for å evaluere farmakokinetikken, farmakodynamikken, sikkerheten, toleransen og aktiviteten til Zilucoplan i pediatriske studiedeltakere fra 2 til under 18 år med acetylkolinreseptorantistoff Positiv generalisert 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).

Studieoversikt

Status

Rekruttering

Forhold

Intervensjon / Behandling

Studietype

Intervensjonell

Registrering (Antatt)

8

Fase

  • Fase 2
  • Fase 3

Kontakter og plasseringer

Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.

Studiekontakt

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

Studer Kontakt Backup

Studiesteder

  • Forente stater
    • Illinois
      • Chicago, Illinois, Forente stater, 60611
        • Tilbaketrukket
        • Mg0014 50168
    • Texas
      • Denton, Texas, Forente stater, 76208
        • Tilbaketrukket
        • Mg0014 50574
  • Italia
      • Milan, Italia
        • Rekruttering
        • Mg0014 40144
  • Polen
      • Katowice, Polen
        • Rekruttering
        • Mg0014 40774
      • Warsaw, Polen
        • Aktiv, ikke rekrutterende
        • Mg0014 40218
  • Storbritannia
      • Glasgow, Storbritannia
        • Rekruttering
        • Mg0014 40735
      • London, Storbritannia
        • Rekruttering
        • Mg0014 40736
  • Sør -Korea
      • Seoul, Sør -Korea
        • Rekruttering
        • Mg0014 20220
      • Seoul, Sør -Korea
        • Tilbaketrukket
        • Mg0014 20104

Deltakelseskriterier

Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.

Kvalifikasjonskriterier

Alder som er kvalifisert for studier

12 år til 17 år (Barn)

Tar imot friske frivillige

Nei

Kjønn som er kvalifisert for studier

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

Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.

Hvordan er studiet utformet?

Designdetaljer

  • Primært formål: Behandling
  • Tildeling: N/A
  • Intervensjonsmodell: Enkeltgruppeoppdrag
  • Masking: Ingen (Open Label)

Våpen og intervensjoner

Deltakergruppe / Arm
Intervensjon / Behandling
Eksperimentell: Zilucoplan Arm

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

Zilucoplan will be administered subcutaneously to pediatric study participants.

Andre navn:
  • RA101495

Hva måler studien?

Primære resultatmål

Resultatmål
Tiltaksbeskrivelse
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
Tiltaksbeskrivelse
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)

Samarbeidspartnere og etterforskere

Det er her du vil finne personer og organisasjoner som er involvert i denne studien.

Sponsor

UCB Biopharma SRL

Etterforskere

  • Studieleder: UCB Cares, 001 844 599 2273

Studierekorddatoer

Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.

Studer hoveddatoer

Studiestart (Faktiske)

16. oktober 2024

Primær fullføring (Antatt)

12. mai 2027

Studiet fullført (Antatt)

22. juni 2027

Datoer for studieregistrering

Først innsendt

20. september 2023

Først innsendt som oppfylte QC-kriteriene

20. september 2023

Først lagt ut (Faktiske)

28. september 2023

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

5. november 2026

Siste oppdatering sendt inn som oppfylte QC-kriteriene

4. november 2026

Sist bekreftet

1. november 2026

Mer informasjon

Begreper knyttet til denne studien

Nøkkelord

Ytterligere relevante MeSH-vilkår

Andre studie-ID-numre

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

Plan for individuelle deltakerdata (IPD)

Planlegger du å dele individuelle deltakerdata (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.

Tilgangskriterier for IPD-deling

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 Støtteinformasjonstype

  • STUDY_PROTOCOL
  • SEVJE
  • CSR

Legemiddel- og utstyrsinformasjon, studiedokumenter

Studerer et amerikansk FDA-regulert medikamentprodukt

Ja

Studerer et amerikansk FDA-regulert enhetsprodukt

Nei

produkt produsert i og eksportert fra USA

Nei

Denne informasjonen ble hentet direkte fra nettstedet clinicaltrials.gov uten noen endringer. Hvis du har noen forespørsler om å endre, fjerne eller oppdatere studiedetaljene dine, vennligst kontakt register@clinicaltrials.gov. Så snart en endring er implementert på clinicaltrials.gov, vil denne også bli oppdatert automatisk på nettstedet vårt. .

Abonnere