Studie k vyhodnocení subkutánního zilucoplanu u pediatrických účastníků s generalizovanou myasthenia gravis (ziMyG)
Multicentrická otevřená, nekontrolovaná studie k hodnocení farmakokinetiky, farmakodynamiky, bezpečnosti, snášenlivosti a aktivity zilukoplanu u účastníků pediatrické studie ve věku od 2 do méně než 18 let s pozitivními protilátkami proti acetylcholinovému receptoru s generalizovanou 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).
Přehled studie
Typ studie
Zápis (Odhadovaný)
Fáze
- Fáze 2
- Fáze 3
Kontakty a umístění
Studijní kontakt
- Jméno: UCB Cares
- Telefonní číslo: 001 844 599 2273
Studijní záloha kontaktů
- Jméno: UCB Cares
- Telefonní číslo: +18445992273 (USA)
- E-mail: ucbcares@ucb.com
Studijní místa
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Milan, Itálie
- Nábor
- Mg0014 40144
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Seoul, Jižní Korea
- Nábor
- Mg0014 20220
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Seoul, Jižní Korea
- Staženo
- Mg0014 20104
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Katowice, Polsko
- Nábor
- Mg0014 40774
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Warsaw, Polsko
- Aktivní, ne nábor
- Mg0014 40218
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Glasgow, Spojené království
- Nábor
- Mg0014 40735
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London, Spojené království
- Nábor
- Mg0014 40736
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Illinois
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Chicago, Illinois, Spojené státy, 60611
- Staženo
- Mg0014 50168
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Texas
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Denton, Texas, Spojené státy, 76208
- Staženo
- Mg0014 50574
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Kritéria účasti
Kritéria způsobilosti
Věk způsobilý ke studiu
Přijímá zdravé dobrovolníky
Pohlaví způsobilá ke studiu
Popis
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
Studijní plán
Jak je studie koncipována?
Detaily designu
- Primární účel: Léčba
- Přidělení: N/A
- Intervenční model: Přiřazení jedné skupiny
- Maskování: Žádné (otevřený štítek)
Zbraně a zásahy
Skupina účastníků / Arm |
Intervence / Léčba |
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Experimentální: Zilucoplan Arm
Study participants will receive zilucoplan in pre-defined dose based on their weight. |
Zilucoplan will be administered subcutaneously to pediatric study participants.
Ostatní jména:
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Co je měření studie?
Primární výstupní opatření
Měření výsledku |
Popis opatření |
Časové okno |
|---|---|---|
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Plasma concentrations of zilucoplan (ZLP) sampled at Week 4 (Day 29)
Časové okno: 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)
Časové okno: 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)
Časové okno: 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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Sekundární výstupní opatření
Měření výsledku |
Popis opatření |
Časové okno |
|---|---|---|
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Occurence of treatment-emergent adverse events (TEAEs) during the course of the study
Časové okno: 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)
Časové okno: 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)
Časové okno: 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
Časové okno: 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)
Časové okno: 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).
Časové okno: 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)
Časové okno: 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)
Časové okno: 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)
Časové okno: 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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Spolupracovníci a vyšetřovatelé
Sponzor
Vyšetřovatelé
- Ředitel studie: UCB Cares, 001 844 599 2273
Termíny studijních záznamů
Hlavní termíny studia
Začátek studia (Aktuální)
Primární dokončení (Odhadovaný)
Dokončení studie (Odhadovaný)
Termíny zápisu do studia
První předloženo
První předloženo, které splnilo kritéria kontroly kvality
První zveřejněno (Aktuální)
Aktualizace studijních záznamů
Poslední zveřejněná aktualizace (Aktuální)
Odeslaná poslední aktualizace, která splnila kritéria kontroly kvality
Naposledy ověřeno
Více informací
Termíny související s touto studií
Klíčová slova
Další relevantní podmínky MeSH
- Nemoci nervového systému
- Novotvary podle místa
- Novotvary
- Neuromuskulární onemocnění
- Autoimunitní onemocnění
- Onemocnění imunitního systému
- Autoimunitní onemocnění nervového systému
- Neurodegenerativní onemocnění
- Paraneoplastické syndromy, nervový systém
- Novotvary nervového systému
- Paraneoplastické syndromy
- Nemoci neuromuskulárního spojení
- Myasthenia Gravis
- Zilucoplan
Další identifikační čísla studie
- MG0014
- U1111-1290-3349 (Jiný identifikátor: World Health Organization (WHO))
- 2022-502072-23 (Identifikátor registru: EU Clinical Trials)
Plán pro data jednotlivých účastníků (IPD)
Plánujete sdílet data jednotlivých účastníků (IPD)?
Popis plánu IPD
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.
Časový rámec sdílení IPD
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.
Kritéria přístupu pro sdílení IPD
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.
Typ podpůrných informací pro sdílení IPD
- PROTOKOL STUDY
- MÍZA
- CSR
Informace o lécích a zařízeních, studijní dokumenty
Studuje lékový produkt regulovaný americkým FDA
Studuje produkt zařízení regulovaný americkým úřadem FDA
produkt vyrobený a vyvážený z USA
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