A Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia Gravis

NCT ID: NCT06055959

Last Updated: 2026-01-06

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE2/PHASE3

Total Enrollment

8 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-10-16

Study Completion Date

2026-12-25

Brief Summary

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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).

Detailed Description

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Conditions

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Generalized Myasthenia Gravis

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Zilucoplan Arm

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

Group Type EXPERIMENTAL

Zilucoplan

Intervention Type DRUG

Zilucoplan will be administered subcutaneously to pediatric study participants.

Interventions

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Zilucoplan

Zilucoplan will be administered subcutaneously to pediatric study participants.

Intervention Type DRUG

Other Intervention Names

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RA101495

Eligibility Criteria

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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


* 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
Minimum Eligible Age

12 Years

Maximum Eligible Age

17 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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UCB Biopharma SRL

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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UCB Cares

Role: STUDY_DIRECTOR

001 844 599 2273

Locations

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Mg0014 50168

Chicago, Illinois, United States

Site Status RECRUITING

Mg0014 50574

Denton, Texas, United States

Site Status RECRUITING

Mg0014 40144

Milan, , Italy

Site Status RECRUITING

Mg0014 40774

Katowice, , Poland

Site Status RECRUITING

Mg0014 40218

Warsaw, , Poland

Site Status RECRUITING

Mg0014 20104

Seoul, , South Korea

Site Status RECRUITING

Mg0014 20220

Seoul, , South Korea

Site Status RECRUITING

Mg0014 40735

Glasgow, , United Kingdom

Site Status RECRUITING

Mg0014 40736

London, , United Kingdom

Site Status RECRUITING

Countries

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United States Italy Poland South Korea United Kingdom

Central Contacts

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UCB Cares

Role: CONTACT

+18445992273 (USA)

UCB Cares

Role: CONTACT

001 844 599 2273

Other Identifiers

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U1111-1290-3349

Identifier Type: OTHER

Identifier Source: secondary_id

2022-502072-23

Identifier Type: REGISTRY

Identifier Source: secondary_id

MG0014

Identifier Type: -

Identifier Source: org_study_id

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