An extension study to find out if multiple treatment cycles of rozanolixizumab are safe and work in children with myasthenia gravis

Overview

Sponsor-declared trial summary

Key facts

Sponsor

UCB Biopharma

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Trial duration

8 Oct 2024 → ongoing

Decision date (initial)

2024-02-09

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

UCB Biopharma SRL

External identifiers

EU CT number

2022-502075-34-00

WHO UTN

U1111-1286-3581

ClinicalTrials.gov

NCT06540144

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Others, Safety

Assess the long term safety and tolerability of additional 6-week treatment cycles with rozanolixizumab in pediatric participants with generalized Myasthenia Gravis (gMG) aged ≥2 at the time of the Screening Visit of MG0006

Secondary objectives 1

1. Evaluate the activity of rozanolixizumab

Conditions and MedDRA coding

Generalized Myasthenia Gravis

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 1

1. - Study participant must meet one of the following: - −Study participant completed MG0006 according to the protocol - −Study participant completed the MG0006 Treatment Period and has a worsening of gMG symptoms in the Observation Period of MG0006

Exclusion criteria 1

1. - Study participant met any mandatory withdrawal or mandatory permanent IMP discontinuation criteria in MG0006 or permanently discontinued IMP - Study participant has a known hypersensitivity to any components of the IMP or other FcRn drugs - Study participant has any laboratory abnormality that, in the opinion of the Investigator, is clinically significant, has not resolved at Baseline, and could jeopardize or compromise the study participant’s ability to participate in this study

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. - Occurrence of serious Treatment-Emergent Adverse Events (TEAEs) up to the End of Study (EOS) Visit - Occurrence of TEAEs leading to permanent withdrawal of IMP up to the EOS Visit - Occurrence of Adverse Event(s) of Special Monitoring (AESM) up to the EOS Visit

Secondary endpoints 1

1. - Percent change in total Immunoglobulin G (IgG) from Baseline to the end of Week 6 of each Treatment Period (TP) - Absolute change in total IgG from Baseline to the end of Week 6 of each TP - Change from Baseline in Myasthenia Gravis-Activities of Daily Living (MG ADL) total score at the end of Week 6 of each TP - Change from Baseline in Quantitative Myasthenia Gravis (QMG) total score at the end of Week 6 of each TP

Investigational products

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

Test 1

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 12

Locations

2 EU/EEA countries · 5 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 39 files

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

Application history

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