An Extension Study Evaluating 6-week Treatment Cycles of Rozanolixizumab in Pediatric Study Participants With Generalized Myasthenia Gravis

NCT06540144 | Enrolling By Invitation Phase 3 DMC FDA Drug

• 3 other identifiers: MG00082022-502075-34U1111-1286-3581
• Study Type: interventional
• Target: 12
• Locations: 4 countries
• Sites: 10

Brief Summary

The purpose of the study is to 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.

Conditions

Generalized Myasthenia Gravis

Keywords

Generalized Myasthenia Gravis; gMG; rozanolixizumab; pediatric

Outcome Measures

Primary Outcomes (3)

• Occurrence of serious Treatment-Emergent Adverse Events (TEAEs) up to the End of Study (EOS) Visit

  Serious TEAEs are any untoward medical incidence in a subject during administered study treatment, whether or not these events are related to study treatment and additionally are emergent untoward medical occurrence that at any dose: * Results in death * Is life-threatening * Requires in patient hospitalisation or prolongation of existing hospitalisation * Results in persistent disability/incapacity * Is a congenital anomaly or birth defect * Important medical events

  From Baseline up to the EOS Visit (up to 52 weeks)

• Occurrence of TEAEs leading to permanent withdrawal of IMP up to the EOS Visit

  An Adverse Event (AE) is any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product.

  From Baseline up to the EOS Visit (up to 52 weeks)

• Occurrence of Adverse Event(s) of Special Monitoring (AESM) up to the EOS Visit

  AESMs are: Severe and/or serious headache, suspected aseptic meningitis, severe Gastrointestinal (GI) disorders, and opportunistic infection.

  From Baseline up to the EOS Visit (up to 52 weeks)

Secondary Outcomes (4)

• Percent change in total Immunoglobulin G (IgG) from Baseline to the end of Week 6 of each Treatment Period (TP)

  From Baseline to the end of Week 6 of each TP (up to 52 weeks)

• Absolute change in total IgG from Baseline to the end of Week 6 of each TP

  From Baseline to the end of Week 6 of each TP (up to 52 weeks)

• Change from Baseline in Myasthenia Gravis-Activities of Daily Living (MG ADL) total score at the end of Week 6 of each TP

  From Baseline to the end of Week 6 of each TP (up to 52 weeks)

• Change from Baseline in Quantitative Myasthenia Gravis (QMG) total score at the end of Week 6 of each TP

  From Baseline to the end of Week 6 of each TP (up to 52 weeks)

Study Arms (1)

rozanolixizumab

EXPERIMENTAL

Study participants will receive pre-defined doses of rozanolixizumab. Each treatment cycle in each Treatment Period (TP) consists of 6 subcutaneous (sc) administrations of rozanolixizumab at 1-week intervals. Each Treatment Period will be initiated upon the discretion of the Investigator based on the medical needs of the study-participant.

Drug: rozanolixizumab

Interventions

rozanolixizumab DRUG

rozanolixizumab solution for injection

Also known as: UCB7665

rozanolixizumab

Eligibility Criteria

• Age: 2 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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 generalized myasthenia gravis (gMG) symptoms in the Observation Period of MG0006

You may not qualify if:

• Study participant met any mandatory withdrawal or mandatory permanent investigational medicinal product (IMP) discontinuation criteria in MG0006 or permanently discontinued IMP
• Study participant has a known hypersensitivity to any components of the IMP or other neonatal Fc receptor (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

Sponsors & Collaborators

• UCB Biopharma SRL: lead

Study Sites (10)

Mg0008 40290

Bologna, Italy

Location

Mg0008 40144

Milan, Italy

Location

Mg0008 40733

Naples, Italy

Location

Mg0008 20340

Fuchu-shi, Japan

Location

Mg0008 20339

Ōbu, Japan

Location

Mg0008 20343

Sagamihara, Japan

Location

Mg0008 40155

Warsaw, Poland

Location

Mg0008 40734

Warsaw, Poland

Location

Mg0008 20081

Taipei, Taiwan

Location

Mg0008 20095

Taipei, Taiwan

Location

MeSH Terms

Conditions

Myasthenia Gravis

Interventions

rozanolixizumab

Condition Hierarchy (Ancestors)

Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases

Study Officials

• UCB Cares

  001 844 599 2273

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 22, 2024
• First Posted: August 6, 2024
• Study Start: October 8, 2024
• Primary Completion (Estimated): August 17, 2027
• Study Completion (Estimated): August 17, 2027
• Last Updated: April 13, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, CSR
• Time Frame: 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.
• Access Criteria: Qualified researchers may request access to anonymized IPD and redacted study documents which may include: raw datasets, analysis-ready datasets, study protocol, blank case report form, 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 pre-specified time, typically 12 months, on a password protected portal.

Locations

PL (2); IT (3); TW (2); JP (3)