Tutkimus ihonalaisen Zilucoplanin arvioimiseksi lapsipotilailla, joilla on yleistynyt myasthenia gravis (ziMyG)
Monikeskus, avoin, kontrolloimaton tutkimus Zilucoplanin farmakokinetiikkaa, farmakodynamiikkaa, turvallisuutta, siedettävyyttä ja aktiivisuutta arvioimiseksi 2–18-vuotiailla lapsitutkimuksen osallistujilla, joilla on asetyylikoliinireseptorivasta-ainepositiivinen yleistynyt 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).
Tutkimuksen yleiskatsaus
Opintotyyppi
Ilmoittautuminen (Arvioitu)
Vaihe
- Vaihe 2
- Vaihe 3
Yhteystiedot ja paikat
Opiskeluyhteys
- Nimi: UCB Cares
- Puhelinnumero: 001 844 599 2273
Tutki yhteystietojen varmuuskopiointi
- Nimi: UCB Cares
- Puhelinnumero: +18445992273 (USA)
- Sähköposti: ucbcares@ucb.com
Opiskelupaikat
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Seoul, Etelä -Korea
- Rekrytointi
- Mg0014 20220
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Seoul, Etelä -Korea
- Peruutettu
- Mg0014 20104
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Milan, Italia
- Rekrytointi
- Mg0014 40144
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Katowice, Puola
- Rekrytointi
- Mg0014 40774
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Warsaw, Puola
- Aktiivinen, ei rekrytointi
- Mg0014 40218
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Glasgow, Yhdistynyt kuningaskunta
- Rekrytointi
- Mg0014 40735
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London, Yhdistynyt kuningaskunta
- Rekrytointi
- Mg0014 40736
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Illinois
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Chicago, Illinois, Yhdysvallat, 60611
- Peruutettu
- Mg0014 50168
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Texas
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Denton, Texas, Yhdysvallat, 76208
- Peruutettu
- Mg0014 50574
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Osallistumiskriteerit
Kelpoisuusvaatimukset
Opintokelpoiset iät
Hyväksyy terveitä vapaaehtoisia
Sukupuolet, jotka voivat opiskella
Kuvaus
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
Opintosuunnitelma
Miten tutkimus on suunniteltu?
Suunnittelun yksityiskohdat
- Ensisijainen käyttötarkoitus: Hoito
- Jako: Ei käytössä
- Inventiomalli: Yksittäinen ryhmätehtävä
- Naamiointi: Ei mitään (avoin tarra)
Aseet ja interventiot
Osallistujaryhmä / Arm |
Interventio / Hoito |
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Kokeellinen: Zilucoplan Arm
Study participants will receive zilucoplan in pre-defined dose based on their weight. |
Zilucoplan will be administered subcutaneously to pediatric study participants.
Muut nimet:
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Mitä tutkimuksessa mitataan?
Ensisijaiset tulostoimenpiteet
Tulosmittaus |
Toimenpiteen kuvaus |
Aikaikkuna |
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Plasma concentrations of zilucoplan (ZLP) sampled at Week 4 (Day 29)
Aikaikkuna: Week 4 (Day 29)
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Blood samples will be collected for measurement of plasma concentrations of ZLP on Day 29 predose. |
Week 4 (Day 29)
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Change from Baseline in sheep red blood cell (sRBC) lysis at Week 4 (Day 29)
Aikaikkuna: Week 4 (Day 29)
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Samples for measurement of sRBC lysis will be collected on Day 29 predose. |
Week 4 (Day 29)
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Change from Baseline in complement component 5 (C5) levels at Week 4 (Day 29)
Aikaikkuna: Week 4 (Day 29)
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Samples for measurement of C5 will be collected on Day 29 predose. |
Week 4 (Day 29)
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Toissijaiset tulostoimenpiteet
Tulosmittaus |
Toimenpiteen kuvaus |
Aikaikkuna |
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Occurence of treatment-emergent adverse events (TEAEs) during the course of the study
Aikaikkuna: From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)
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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 (Day 1) to Safety-Follow-Up Visit (up to Week 15)
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Occurrence of treatment-emergent serious adverse events (TESAEs)
Aikaikkuna: From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)
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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)
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Occurrence of TEAEs leading to permanent withdrawal of investigational medicinal product (IMP)
Aikaikkuna: From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)
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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. AEs leading to permanent withdrawal of study medication. |
From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)
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Occurrence of treatment-emergent infections
Aikaikkuna: From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)
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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)
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Occurrence of antidrug antibody (ADA) and anti- polyethylene glycol (PEG) antibodies at Week 4 (Day 29)
Aikaikkuna: Week 4 (Day 29)
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ADA and anti-PEG antibodies will be evaluated in serum samples. |
Week 4 (Day 29)
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Change in MG-activities of daily living (MG-ADL) score from Baseline to Week 4 (Day 29).
Aikaikkuna: Week 4 (Day 29)
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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. |
Week 4 (Day 29)
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Change in Quantitative MG (QMG) score from Baseline to Week 4 (Day 29)
Aikaikkuna: Week 4 (Day 29)
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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. |
Week 4 (Day 29)
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Myasthenia Gravis Foundation of America Post-Interventional Status (MGFA-PIS) at Week 4 (Day 29)
Aikaikkuna: Week 4 (Day 29)
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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)
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Change in Pediatric Quality of Life Inventory (PedsQoL), Version 4 domain scores from Baseline to Week 4 (Day 29)
Aikaikkuna: Week 4 (Day 29)
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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)
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Yhteistyökumppanit ja tutkijat
Sponsori
Tutkijat
- Opintojohtaja: UCB Cares, 001 844 599 2273
Opintojen ennätyspäivät
Opi tärkeimmät päivämäärät
Opiskelun aloitus (Todellinen)
Ensisijainen valmistuminen (Arvioitu)
Opintojen valmistuminen (Arvioitu)
Opintoihin ilmoittautumispäivät
Ensimmäinen lähetetty
Ensimmäinen toimitettu, joka täytti QC-kriteerit
Ensimmäinen Lähetetty (Todellinen)
Tutkimustietojen päivitykset
Viimeisin päivitys julkaistu (Todellinen)
Viimeisin lähetetty päivitys, joka täytti QC-kriteerit
Viimeksi vahvistettu
Lisää tietoa
Tähän tutkimukseen liittyvät termit
Muita asiaankuuluvia MeSH-ehtoja
- Hermoston sairaudet
- Neoplasmat sivustoittain
- Neoplasmat
- Neuromuskulaariset sairaudet
- Autoimmuunisairaudet
- Immuunijärjestelmän sairaudet
- Hermoston autoimmuunisairaudet
- Neurodegeneratiiviset sairaudet
- Paraneoplastiset oireyhtymät, hermosto
- Hermoston kasvaimet
- Paraneoplastiset oireyhtymät
- Neuromuskulaariset liitossairaudet
- Myasthenia Gravis
- zilukoplan
Muut tutkimustunnusnumerot
- MG0014
- U1111-1290-3349 (Muu tunniste: World Health Organization (WHO))
- 2022-502072-23 (Rekisterin tunniste: EU Clinical Trials)
Yksittäisten osallistujien tietojen suunnitelma (IPD)
Aiotko jakaa yksittäisten osallistujien tietoja (IPD)?
IPD-suunnitelman kuvaus
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-jaon aikakehys
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-jaon käyttöoikeuskriteerit
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-jakamista tukeva tietotyyppi
- STUDY_PROTOCOL
- MAHLA
- CSR
Lääke- ja laitetiedot, tutkimusasiakirjat
Tutkii yhdysvaltalaista FDA sääntelemää lääkevalmistetta
Tutkii yhdysvaltalaista FDA sääntelemää laitetuotetta
Yhdysvalloissa valmistettu ja sieltä viety tuote
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