Clinical trial NCT06055959: A Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia Gravis
Official title: 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
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
PHASE3
Conditions: Generalized Myasthenia Gravis
Keywords: RA101495
gMG
generalized myasthenia gravis
zilucoplan
pediatric
MG0014
Lead sponsor: UCB Pharma
Sponsor class: INDUSTRY
First posted: 2023-09-28
Enrollment: 8
Sex: ALL
Minimum age (years): 12
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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).


Eligibility criteria: 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

Primary outcomes:
measure: Plasma concentrations of zilucoplan (ZLP) sampled at Week 4 (Day 29)
timeFrame: Week 4 (Day 29)
measure: Change from Baseline in sheep red blood cell (sRBC) lysis at Week 4 (Day 29)
timeFrame: Week 4 (Day 29)
measure: Change from Baseline in complement component 5 (C5) levels at Week 4 (Day 29)
timeFrame: Week 4 (Day 29)
Secondary outcomes:
measure: Occurence of treatment-emergent adverse events (TEAEs) during the course of the study
timeFrame: From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)
measure: Occurrence of treatment-emergent serious adverse events (TESAEs)
timeFrame: From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)
measure: Occurrence of TEAEs leading to permanent withdrawal of investigational medicinal product (IMP)
timeFrame: From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)
measure: Occurrence of treatment-emergent infections
timeFrame: From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15)
measure: Occurrence of antidrug antibody (ADA) and anti- polyethylene glycol (PEG) antibodies at Week 4 (Day 29)
timeFrame: Week 4 (Day 29)
measure: Change in MG-activities of daily living (MG-ADL) score from Baseline to Week 4 (Day 29).
timeFrame: Week 4 (Day 29)
measure: Change in Quantitative MG (QMG) score from Baseline to Week 4 (Day 29)
timeFrame: Week 4 (Day 29)
measure: Myasthenia Gravis Foundation of America Post-Interventional Status (MGFA-PIS) at Week 4 (Day 29)
timeFrame: Week 4 (Day 29)
measure: Change in Pediatric Quality of Life Inventory (PedsQoL), Version 4 domain scores from Baseline to Week 4 (Day 29)
timeFrame: Week 4 (Day 29)
Other outcomes:
Locations:
city: Chicago
country: United States
facility: Mg0014 50168
state: Illinois
city: Denton
country: United States
facility: Mg0014 50574
state: Texas
city: Milano
country: Italy
facility: Mg0014 40144
city: Seoul
country: Korea, Republic of
facility: Mg0014 20104
city: Seoul
country: Korea, Republic of
facility: Mg0014 20220
city: Katowice
country: Poland
facility: Mg0014 40774
city: Warszawa
country: Poland
facility: Mg0014 40218
city: Glasgow
country: United Kingdom
facility: Mg0014 40735
city: London
country: United Kingdom
facility: Mg0014 40736
Study officials:
affiliation: 001 844 599 2273
name: UCB Cares
role: STUDY_DIRECTOR
References: