MG0014
MG0014
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
Brief summary
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).
Medical Condition
Generalized Myasthenia Gravis
Min. Age
12
Years
Max. Age
17
Years
Who Can Join?
All
Status
Recruiting
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
Study Medication Description
Study Medication:
ZILBRYSQ®
Other Descriptive Name:
zilucoplan
Placebo
No
Comparator:
No
Refer to a friend
Study Dates
April 2024
Planned Start Date of Enrollment
December 2026
Planned Study Completion Date