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

UCB Biopharma SRL17 sites in 5 countries8 target enrollmentOctober 16, 2024

ConditionsGeneralized Myasthenia Gravis

InterventionsZilucoplan

Overview

Phase: Phase 2
Intervention: Zilucoplan
Conditions: Generalized Myasthenia Gravis
Sponsor: UCB Biopharma SRL
Enrollment: 8
Locations: 17
Primary Endpoint: Plasma concentrations of zilucoplan (ZLP) sampled at Week 4 (Day 29)
Status: Recruiting
Last Updated: 8 days ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

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).

Registry

clinicaltrials.gov

Start Date

October 16, 2024

End Date

December 25, 2026

Last Updated

8 days ago

Study Type

Interventional

Study Design

Single Group

Sex

All

Investigators

Sponsor

UCB Biopharma SRL

Responsible Party

Sponsor

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)

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

Arms & Interventions

Zilucoplan Arm

Study participants will receive zilucoplan in pre-defined dose based on their weight.

Intervention: Zilucoplan

Outcomes

Primary Outcomes

Plasma concentrations of zilucoplan (ZLP) sampled at Week 4 (Day 29)

Time Frame: Week 4 (Day 29)

Blood samples will be collected for measurement of plasma concentrations of ZLP on Day 29 predose.

Change from Baseline in sheep red blood cell (sRBC) lysis at Week 4 (Day 29)

Time Frame: Week 4 (Day 29)

Samples for measurement of sRBC lysis will be collected on Day 29 predose.

Change from Baseline in complement component 5 (C5) levels at Week 4 (Day 29)

Time Frame: Week 4 (Day 29)

Samples for measurement of C5 will be collected on Day 29 predose.

Secondary Outcomes

Occurence of treatment-emergent adverse events (TEAEs) during the course of the study(From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15))
Occurrence of treatment-emergent serious adverse events (TESAEs)(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)(From Baseline (Day 1) to Safety-Follow-Up Visit (up to Week 15))
Occurrence of treatment-emergent infections(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)(Week 4 (Day 29))
Change in MG-activities of daily living (MG-ADL) score from Baseline to Week 4 (Day 29).(Week 4 (Day 29))
Change in Quantitative MG (QMG) score from Baseline to Week 4 (Day 29)(Week 4 (Day 29))
Myasthenia Gravis Foundation of America Post-Interventional Status (MGFA-PIS) at Week 4 (Day 29)(Week 4 (Day 29))
Change in Pediatric Quality of Life Inventory (PedsQoL), Version 4 domain scores from Baseline to Week 4 (Day 29)(Week 4 (Day 29))