An Open-label Extension Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia Gravis

Generalized Myasthenia Gravis Clinical Trial

— ziMyG+  

Official title:

An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Activity of Zilucoplan in Pediatric Study Participants With Acetylcholine Receptor Antibody Positive Generalized Myasthenia Gravis

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06435312
• Other study ID #: MG0015
• Secondary ID: 2022-502073-42-0
• Status: Not yet recruiting
• Phase: Phase 3
• Start date: August 13, 2024
• Est. completion date: December 7, 2027

Study information

• Verified date: May 2024
• Source: UCB Pharma
• Contact: UCB Cares
• Phone: +18445992273
• Email: ucbcares[@]ucb.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The purpose of this study is to assess the long-term safety and tolerability of an additional 52 weeks of Zilucoplan treatment administered by subcutaneous injection once daily in pediatric study participants

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 8
• Est. completion date: December 7, 2027
• Est. primary completion date: October 26, 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years and older

Eligibility

Inclusion Criteria:

United States of America (USA) specific inclusion criterion:

• Participant must be = 12 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 years of age at the time of signing the Informed Consent/Assent according to local regulation.

Global specific inclusion criteria:

• Participant has completed the MG0014 according to the protocol, and further treatment with zilucoplan is in the interest of the participant in the investigator´s opinion

• Participant agrees to receive booster vaccinations against meningococcal infections during the study, if clinically indicated according to the local standard of care

Exclusion Criteria:

• Study participant met any mandatory investigational medicinal product (IMP) withdrawal or mandatory permanent discontinuation criteria in MG0014 or permanently discontinued IMP

• Participant has known positive serology for muscle-specific kinase

• Participant has known hypersensitivity to any components of the IMP

• Participant has a prior history of meningococcal disease

Related Conditions & MeSH terms

• Generalized Myasthenia Gravis
• Muscle Weakness
• Myasthenia Gravis

Intervention

Drug:
• Zilucoplan
Zilucoplan will be administered subcutaneously to pediatric study participants

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
UCB Biopharma SRL

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Occurence of treatment emergent adverse events during the course of the study	An adverse event (AE) is any untoward medical occurence in a patient or clinical investigation where the study participant 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.	Baseline (Day 1) to Safety Follow-up (up to Week 60)
Primary	Occurence 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 Results in persistent disability/incapacity Is a congenital anomaly/birth defect Important medical event.	Baseline (Day 1) to Safety Follow-up (up to Week 60)
Primary	Occurence of treatment-emergent advserse events leading to permanent withdrawal of investigational medicinal product	An adverse event (AE) is any untoward medical occurence in a participant or clinical investigation administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. AEs leading to permanent withdrawal of study medication.	Baseline (Day 1) to Safety Follow-up (up to Week 60)
Primary	Occurence 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.	Baseline (Day 1) to Safety Follow-up (up to Week 60)
Secondary	Plasma concentration of Zilucoplan at Week 52	Blood samples for the measurement of plasma concentrations of Zilucoplan will be collected at Week 52.	Week 52
Secondary	Sheep red blood cell (sRBC) lysis activity at Week 52	Blood samples for measurement of sRBC lysis will be collected at Week 52.	Week 52
Secondary	Blood complement component 5 (C5) levels at Week 52	Blood samples for measurement of C5 will be collected at Week 52.	Week 52
Secondary	Myasthenia Gravis Activity of Daily Living (MG-ADL) score at Week 52	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 52
Secondary	Quantitative Myasthenia Gravis (QMG) score at Week 52	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 52