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| ID | Type | Description | Link |
|---|---|---|---|
| U1111-1290-3349 | Other Identifier | World Health Organization (WHO) | |
| 2022-502072-23 | Registry Identifier | EU Clinical Trials |
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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).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Zilucoplan Arm | Experimental | Study participants will receive zilucoplan in pre-defined dose based on their weight. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Zilucoplan | Drug | Zilucoplan will be administered subcutaneously to pediatric study participants. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Plasma concentrations of zilucoplan (ZLP) sampled at 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) | 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) | Samples for measurement of C5 will be collected on Day 29 predose. | Week 4 (Day 29) |
| Measure | Description | Time Frame |
|---|---|---|
| Occurence of treatment-emergent adverse events (TEAEs) during the course of the study | 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. |
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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:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| UCB Cares | Contact | +18445992273 (USA) | ucbcares@ucb.com | |
| UCB Cares | Contact | 001 844 599 2273 |
| Name | Affiliation | Role |
|---|---|---|
| UCB Cares | 001 844 599 2273 | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Mg0014 50168 | Withdrawn | Chicago | Illinois | 60611 | United States | |
| Mg0014 50574 |
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.
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.
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.
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| From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15) |
| Occurrence of treatment-emergent serious adverse events (TESAEs) | 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) | 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 | 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) | 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). | 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) | 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) | 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) | 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) |
| Withdrawn |
| Denton |
| Texas |
| 76208 |
| United States |
| Mg0014 40144 | Recruiting | Milan | Italy |
| Mg0014 40774 | Recruiting | Katowice | Poland |
| Mg0014 40218 | Active, not recruiting | Warsaw | Poland |
| Mg0014 20104 | Withdrawn | Seoul | South Korea |
| Mg0014 20220 | Recruiting | Seoul | South Korea |
| Mg0014 40735 | Recruiting | Glasgow | United Kingdom |
| Mg0014 40736 | Recruiting | London | United Kingdom |
| ID | Term |
|---|---|
| D009157 | Myasthenia Gravis |
| ID | Term |
|---|---|
| D020361 | Paraneoplastic Syndromes, Nervous System |
| D009423 | Nervous System Neoplasms |
| D009371 | Neoplasms by Site |
| D009369 | Neoplasms |
| D010257 | Paraneoplastic Syndromes |
| D020274 | Autoimmune Diseases of the Nervous System |
| D009422 | Nervous System Diseases |
| D019636 | Neurodegenerative Diseases |
| D020511 | Neuromuscular Junction Diseases |
| D009468 | Neuromuscular Diseases |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
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| ID | Term |
|---|---|
| C000719268 | zilucoplan |
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