A study to learn how zilucoplan moves throughout the body over time and how safe it is in children with generalized myasthenia gravis

Overview

Sponsor-declared trial summary

Key facts

Sponsor

UCB Biopharma

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Trial duration

11 Jul 2025 → ongoing

Decision date (initial)

2023-11-29

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

External identifiers

EU CT number

2022-502072-23-00

WHO UTN

U1111-1290-3349

ClinicalTrials.gov

NCT06055959

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Pharmacodynamic, Others, Pharmacokinetic

Assess the pharmacokinetics (PK) of zilucoplan (ZLP) administered by subcutaneous (sc) injection once daily for pediatric study participants from 2 to <18 years of age with generalized Myasthenia Gravis (gMG). Assess the pharmacodynamics (PD) of ZLP administered by sc injection once daily in pediatric study participants from 2 to <18 years of age with gMG.

Secondary objectives 1

1. Assess the safety and tolerability of zilucoplan (ZLP) administered by subcutaneous (sc) injection once daily in pediatric study participants from 2 to <18 years of age with gMG. Assess the immunogenicity of ZLP administered by sc injection once daily in pediatric study participants from 2 to <18 years of age with gMG. Evaluate the activity of ZLP administered by sc injection once daily in pediatric study participants from 2 to <18 years of age with gMG.

Conditions and MedDRA coding

Generalized Myasthenia Gravis

Regulatory references

EMA paediatric investigation plan (PIP)

EMEA-002747-PIP01-20

Plan to share IPD

Yes

IPD plan description

Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe, or global development is discontinued, and 18 months after trial completion. Investigators may request access to anonymized individual patient-level data and redacted trial documents which may include: analysis-ready datasets, study protocol, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a prespecified time, typically 12 months, on a password protected portal. This plan may change if the risk of re-identifying trial participants is determined to be too high after the trial is completed; in this case and to protect participants, individual patient-level data would not be made available.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 1

1. 1. Participant must be 2 to <18 years of age at the time of signing the Informed consent/assent according to local regulation. 2. Participant has a diagnosis of generalized myasthenia gravis (gMG) confirmed by a prior positive serologic test result to acetylcholine receptor (AChR) prior to Screening. 3. Participant meets the criteria as defined by the Myasthenia Gravis Foundation of America (MGFA) Clinical Classification II to IV at Screening 4. 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. 5. 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 1

1. 1. Participant has known positive serology for muscle-specific kinase 2. 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. 3. Participant has had a thymectomy within 6 months prior to Baseline 4. Participant has minimal Manifestation Status of MG based on the clinical judgement of the Investigator 5. Participant has a prior history of meningococcal disease. 6. Current or recent systemic infection within 2 weeks prior to Baseline or infection requiring intravenous antibiotics within 4 weeks prior to Baseline.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. • Plasma concentrations of zilucoplan (ZLP) sampled at Week 4 (Day 29) • Change from Baseline in sheep red blood cell (sRBC) lysis at Week 4 (Day 29) • Change from Baseline in complement component 5 (C5) levels at Week 4 (Day 29)

Secondary endpoints 9

1. • Occurrence of treatment-emergent adverse events (TEAEs) during the course of the study
2. • Occurrence of treatment-emergent serious adverse events (TESAE)s
3. • Occurrence of TEAEs leading to permanent withdrawal of investigational medicinal product (IMP)
4. • Occurrence of treatment-emergent infections
5. • Occurrence of antidrug antibody (ADA) and anti- polyethylene glycol (PEG) antibodies at Week 4 (Day 29).
6. • Change in MG-activities of daily living (MG-ADL) score from Baseline to Week 4 (Day 29).
7. • Change in Quantitative MG (QMG) score from Baseline to Week 4 (Day 29).
8. • Myasthenia Gravis Foundation of America Post-Interventional Status (MGFA PIS) at Week 4 (Day 29).
9. • Change in Pediatric Quality of Life Inventory (PedsQoL), Version 4 domain scores from Baseline to Week 4 (Day 29).

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 4

Auxiliary 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

UCB Biopharma

Sponsor organisation

UCB Biopharma

Address

Researchdreef 60

City

Anderlecht

Postcode

1070

Country

Belgium

Scientific contact point

Organisation

UCB Biopharma

Contact name

UCB Cares

Public contact point

Organisation

UCB Biopharma

Contact name

UCB Cares

Third parties 14

Locations

2 EU/EEA countries · 3 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 45 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

11 submissions — initial application plus substantial / non-substantial modifications