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A MULTICENTER OPEN-LABEL, UNCONTROLLED STUDY TO EVALUATE THE PHARMACOKINETICS, PHARMACODYNAMICS, SAFETY, TOLERABILITY, AND ACTIVITY OF ZILUCOPLAN IN PEDIATRIC STUDY PARTICIPANTS FROM 2 TO LESS THAN 18 YEARS OF AGE WITH ACETYLCHOLINE RECEPTOR ANTIBODY POSITIVE GENERALIZED MYASTHENIA GRAVIS

Status
Recruiting
Phases
Phase 2Phase 3
Study type
Interventional
Source
EU CTIS
Registry ID
CTIS2022-502072-23-00
Acronym
MG0014
Enrollment
4
Registered
2023-11-29
Start date
2025-07-11
Completion date
Unknown
Last updated
2025-12-17

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Generalized Myasthenia Gravis

Brief summary

• Plasma concentrations of zilucoplan (ZLP) sampled at Week 4 (Day 29) • Change from Baseline in sheep red blood cell (sRBC) lysis at Week 4 (Day 29) • Change from Baseline in complement component 5 (C5) levels at Week 4 (Day 29)

Detailed description

• Occurrence of treatment-emergent adverse events (TEAEs) during the course of the study, • Occurrence of treatment-emergent serious adverse events (TESAE)s, • Occurrence of TEAEs leading to permanent withdrawal of investigational medicinal product (IMP), • Occurrence of treatment-emergent infections, • Occurrence of antidrug antibody (ADA) and anti- polyethylene glycol (PEG) antibodies at Week 4 (Day 29)., • Change in MG-activities of daily living (MG-ADL) score from Baseline to Week 4 (Day 29)., • Change in Quantitative MG (QMG) score from Baseline to Week 4 (Day 29)., • Myasthenia Gravis Foundation of America Post-Interventional Status (MGFA PIS) at Week 4 (Day 29)., • Change in Pediatric Quality of Life Inventory (PedsQoL), Version 4 domain scores from Baseline to Week 4 (Day 29).

Interventions

Sponsors

UCB Biopharma
Lead SponsorINDUSTRY

Eligibility

Sex/Gender
All
Age
0 Years to 17 Years

Design outcomes

Primary

MeasureTime frame
• Plasma concentrations of zilucoplan (ZLP) sampled at Week 4 (Day 29) • Change from Baseline in sheep red blood cell (sRBC) lysis at Week 4 (Day 29) • Change from Baseline in complement component 5 (C5) levels at Week 4 (Day 29)

Secondary

MeasureTime frame
• Occurrence of treatment-emergent adverse events (TEAEs) during the course of the study, • Occurrence of treatment-emergent serious adverse events (TESAE)s, • Occurrence of TEAEs leading to permanent withdrawal of investigational medicinal product (IMP), • Occurrence of treatment-emergent infections, • Occurrence of antidrug antibody (ADA) and anti- polyethylene glycol (PEG) antibodies at Week 4 (Day 29)., • Change in MG-activities of daily living (MG-ADL) score from Baseline to Week 4 (Day 29)., • Change in Quantitative MG (QMG) score from Baseline to Week 4 (Day 29)., • Myasthenia Gravis Foundation of America Post-Interventional Status (MGFA PIS) at Week 4 (Day 29)., • Change in Pediatric Quality of Life Inventory (PedsQoL), Version 4 domain scores from Baseline to Week 4 (Day 29).

Countries

Italy, Poland

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026