Uno studio per valutare lo Zilucoplan sottocutaneo in partecipanti pediatrici con miastenia gravis generalizzata (ziMyG)
Uno studio multicentrico in aperto, non controllato, per valutare la farmacocinetica, la farmacodinamica, la sicurezza, la tollerabilità e l'attività di Zilucoplan in partecipanti pediatrici allo studio di età compresa tra 2 e meno di 18 anni con miastenia gravis generalizzata positiva per il recettore dell'acetilcolina
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).
Panoramica dello studio
Stato
Condizioni
Intervento / Trattamento
Tipo di studio
Iscrizione (Stimato)
Fase
- Fase 2
- Fase 3
Contatti e Sedi
Contatto studio
- Nome: UCB Cares
- Numero di telefono: 001 844 599 2273
Backup dei contatti dello studio
- Nome: UCB Cares
- Numero di telefono: +18445992273 (USA)
- Email: ucbcares@ucb.com
Luoghi di studio
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Seoul, Corea del Sud
- Reclutamento
- Mg0014 20220
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Seoul, Corea del Sud
- Ritirato
- Mg0014 20104
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Milan, Italia
- Reclutamento
- Mg0014 40144
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Katowice, Polonia
- Reclutamento
- Mg0014 40774
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Warsaw, Polonia
- Attivo, non reclutante
- Mg0014 40218
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Glasgow, Regno Unito
- Reclutamento
- Mg0014 40735
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London, Regno Unito
- Reclutamento
- Mg0014 40736
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Illinois
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Chicago, Illinois, Stati Uniti, 60611
- Ritirato
- Mg0014 50168
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Texas
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Denton, Texas, Stati Uniti, 76208
- Ritirato
- Mg0014 50574
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Criteri di partecipazione
Criteri di ammissibilità
Età idonea allo studio
Accetta volontari sani
Sessi ammissibili allo studio
Descrizione
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
Piano di studio
Come è strutturato lo studio?
Dettagli di progettazione
- Scopo principale: Trattamento
- Assegnazione: N / A
- Modello interventistico: Assegnazione di gruppo singolo
- Mascheramento: Nessuno (etichetta aperta)
Armi e interventi
Gruppo di partecipanti / Arm |
Intervento / Trattamento |
|---|---|
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Sperimentale: Zilucoplan Arm
Study participants will receive zilucoplan in pre-defined dose based on their weight. |
Zilucoplan will be administered subcutaneously to pediatric study participants.
Altri nomi:
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Cosa sta misurando lo studio?
Misure di risultato primarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
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Plasma concentrations of zilucoplan (ZLP) sampled at Week 4 (Day 29)
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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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Misure di risultato secondarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
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Occurence of treatment-emergent adverse events (TEAEs) during the course of the study
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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)
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Occurrence of treatment-emergent infections
Lasso di tempo: 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)
Lasso di tempo: 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).
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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)
Lasso di tempo: 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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Collaboratori e investigatori
Sponsor
Investigatori
- Direttore dello studio: UCB Cares, 001 844 599 2273
Studiare le date dei record
Studia le date principali
Inizio studio (Effettivo)
Completamento primario (Stimato)
Completamento dello studio (Stimato)
Date di iscrizione allo studio
Primo inviato
Primo inviato che soddisfa i criteri di controllo qualità
Primo Inserito (Effettivo)
Aggiornamenti dei record di studio
Ultimo aggiornamento pubblicato (Effettivo)
Ultimo aggiornamento inviato che soddisfa i criteri QC
Ultimo verificato
Maggiori informazioni
Termini relativi a questo studio
Parole chiave
Termini MeSH pertinenti aggiuntivi
- Malattie del sistema nervoso
- Neoplasie per sede
- Neoplasie
- Malattie neuromuscolari
- Malattie autoimmuni
- Malattie del sistema immunitario
- Malattie autoimmuni del sistema nervoso
- Malattie Neurodegenerative
- Sindromi Paraneoplastiche, Sistema Nervoso
- Neoplasie del sistema nervoso
- Sindromi paraneoplastiche
- Malattie della giunzione neuromuscolare
- Miastenia grave
- Zilucoplan
Altri numeri di identificazione dello studio
- MG0014
- U1111-1290-3349 (Altro identificatore: World Health Organization (WHO))
- 2022-502072-23 (Identificatore di registro: EU Clinical Trials)
Piano per i dati dei singoli partecipanti (IPD)
Hai intenzione di condividere i dati dei singoli partecipanti (IPD)?
Descrizione del piano 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.
Periodo di condivisione 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.
Criteri di accesso alla condivisione 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.
Tipo di informazioni di supporto alla condivisione IPD
- STUDIO_PROTOCOLLO
- LINFA
- RSI
Informazioni su farmaci e dispositivi, documenti di studio
Studia un prodotto farmaceutico regolamentato dalla FDA degli Stati Uniti
Studia un dispositivo regolamentato dalla FDA degli Stati Uniti
prodotto fabbricato ed esportato dagli Stati Uniti
Queste informazioni sono state recuperate direttamente dal sito web clinicaltrials.gov senza alcuna modifica. In caso di richieste di modifica, rimozione o aggiornamento dei dettagli dello studio, contattare register@clinicaltrials.gov. Non appena verrà implementata una modifica su clinicaltrials.gov, questa verrà aggiornata automaticamente anche sul nostro sito web .