Purpose

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).

Condition

Eligibility

Eligible Ages
Between 12 Years and 17 Years
Eligible Sex
All
Accepts Healthy Volunteers
No

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 Design

Phase
Phase 2/Phase 3
Study Type
Interventional
Allocation
N/A
Intervention Model
Single Group Assignment
Primary Purpose
Treatment
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Zilucoplan Arm
Study participants will receive zilucoplan in pre-defined dose based on their weight.
  • Drug: Zilucoplan
    Zilucoplan will be administered subcutaneously to pediatric study participants.
    Other names:
    • RA101495

More Details

Status
Recruiting
Sponsor
UCB Biopharma SRL

Study Contact

UCB Cares
+18445992273 (USA)
ucbcares@ucb.com

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.