ROZANOLIXIZUMAB for Generalized Myasthenia Gravis

Inclusion criteria

• {"criterion_text":"-\tStudy participant must be ≥2 to <18 years of age inclusive, at the time of signing the informed consent/assent according to local regulation\n-\tStudy participant must have a documented diagnosis of generalized Myasthenia Gravis (gMG) at Screening that includes a record confirming the presence of MG specific autoantibodies to acetylcholine receptor (AChR) or muscle-specific kinase (MuSK) prior to Screening\n-\tStudy participant has Myasthenia Gravis Foundation of America (MGFA) Clinical Classification II to IVa at Screening\n-\tStudy participant has received existing conventional treatment(s) for gMG (eg, pyridostigmine, corticosteroids, and/or immune suppressants) prior to Screening\n-\tStudy participant has had an unsatisfactory clinical response or worsening of gMG symptoms and is in need of additional therapy (for example, plasma exchange (PEX) or treatment with intravenous immunoglobulin g (IVIg))"}

Exclusion criteria

• {"criterion_text":"-\tStudy participant with severe weakness affecting oropharyngeal or respiratory muscles, or who has myasthenic crisis or impending crisis at Screening or Baseline\n-\tStudy participant has a known hypersensitivity to any components of the Investigational Medicinal Product (IMP) or other anti-FcRn medications\n-\tStudy participant with any active or untreated thymoma\n-\tStudy participant has a history of thymectomy within 6 months prior to Screening\n-\tStudy participant has a clinically relevant active infection (eg, sepsis, pneumonia, or abscess) in the opinion of the Investigator, or had a serious infection (resulting in hospitalization or requiring parenteral antibiotic treatment) within 6 weeks prior to the first dose of IMP\n-\tStudy participant has received a live vaccination within 4 weeks prior to Baseline or intends to have a live vaccination during the course of the study"}

Primary endpoints

• {"endpoint_text":"-\tOccurrence of serious Treatment-Emergent Adverse Events (TEAEs) up to the End of Study (EOS) Visit (up to 18 weeks)\n-\tOccurrence of TEAEs leading to permanent withdrawal of Investigational Medicinal Product (IMP) up to the EOS Visit (up to 18 weeks)\n-\tOccurrence of Adverse Event(s) of Special Monitoring (AESM) up to the EOS Visit (up to 18 weeks)","definition_or_measurement_approach":"-Measured as occurrence of events during study treatment and follow-up up to the End of Study visit (timeframe: up to 18 weeks)"}

Secondary endpoints

• {"endpoint_text":"- Percent change in total Immunoglobulin G (IgG) from Baseline at the end of Week 6","definition_or_measurement_approach":"Percent change from baseline in total IgG measured at Week 6"}
• {"endpoint_text":"- Absolute change in total IgG from Baseline at the end of Week 6","definition_or_measurement_approach":"Absolute difference in total IgG from baseline measured at Week 6"}
• {"endpoint_text":"- Percent change from Baseline in MG-specific autoantibodies (anti-AChR or anti-MuSK) levels at the end of Week 6","definition_or_measurement_approach":"Percent change from baseline in anti-AChR or anti-MuSK levels measured at Week 6"}
• {"endpoint_text":"- Absolute change from Baseline in MG-specific autoantibodies (anti-AChR or anti-MuSK) levels at the end of Week 6","definition_or_measurement_approach":"Absolute change from baseline in anti-AChR or anti-MuSK levels measured at Week 6"}
• {"endpoint_text":"- Change from Baseline in myasthenia gravis-activities of daily living (MG-ADL) total score at the end of Week 6","definition_or_measurement_approach":"Change from baseline in MG-ADL total score assessed at Week 6"}
• {"endpoint_text":"- Change from Baseline in Quantitative Myasthenia Gravis (QMG) total score at the end of Week 6","definition_or_measurement_approach":"Change from baseline in QMG total score assessed at Week 6"}
• {"endpoint_text":"- Occurrence of other TEAEs (including headache, nausea, and infusion site reactions) during Treatment Period 1 (TP1) and Observation Period 1(OP1)","definition_or_measurement_approach":"Occurrence and reporting of specified TEAEs during TP1 and OP1"}
• {"endpoint_text":"- Evaluation of local tolerability at each scheduled assessment during TP1","definition_or_measurement_approach":"Assessment of local tolerability at scheduled visits during Treatment Period 1"}
• {"endpoint_text":"- Plasma concentration of rozanolixizumab at the 6-week treatment cycle","definition_or_measurement_approach":"Pharmacokinetic measurement: plasma concentration of rozanolixizumab during 6-week cycle"}
• {"endpoint_text":"- Incidence of antidrug antibodies (ADAs) at the end of Week 6","definition_or_measurement_approach":"Incidence (presence/absence) of ADAs measured at Week 6"}

Methods

• Recruitment brochures and procedures exist for Italy and Poland (documents: 'IT Recruitment Brochure it IT MG0006 Public' and 'POL-Recruitment-Brochure-MG0006-Public-pl-PL' and related recruitment procedure documents) indicating site-level recruitment materials
• Site staff augmentation and patient travel cost reimbursement services provided by Curandus sp. z o.o. (listed for Poland)
• Engagement with patient organisation/advocacy (Center For Information And Study On Clinical Research Participation Inc.) indicated in third parties

Italy

Earliest CTIS Part Ii Submission Date

08-01-2024

Latest Decision Or Authorization Date

16-03-2026

Processing Time Days

798

Number Of Sites

3

Number Of Participants

3

Poland

Earliest CTIS Part Ii Submission Date

19-12-2023

Latest Decision Or Authorization Date

24-02-2026

Processing Time Days

798

Number Of Sites

2

Number Of Participants

4

Primary sponsor

Full Name

UCB Biopharma

Organisation Type

Pharmaceutical company

Country Of Registered Address

Belgium

Contract research organisations

Name

Parexel International (IRL) Limited

Responsibilities

Operational support roles (codes listed: 1,11,12,13,2,5,6,8)

Name

IQVIA Laboratories LLC

Responsibilities

Laboratory/testing services (code: 4)

Name

4G Clinical B.V.

Responsibilities

Operational support (code: 3)

Name

Drug Development Solutions Limited

Responsibilities

Operational support (code: 4)

Name

BioAgilytix Europe GmbH

Responsibilities

Bioanalytical services (code: 4)

Name

CluePoints

Responsibilities

Data quality assessment services

Name

Advarra Inc.

Responsibilities

Site document repository / ethical review support

Name

Medidata Solutions International Limited

Responsibilities

Data platform / eClinical services (code: 7)

Name

Certara USA Inc.

Responsibilities

Modelling/statistics support (code: 7)

Name

RWS Life Sciences Inc.

Responsibilities

Scales & linguistic validation services

Third parties

• {"country":"United States","full_name":"Advarra Inc.","duties_or_roles":"Site document repository","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"RWS Life Sciences Inc.","duties_or_roles":"Scales & Linguistic validation services","organisation_type":"Pharmaceutical company"}
• {"country":"Ireland","full_name":"Parexel International (IRL) Limited","duties_or_roles":"Codes: 1,11,12,13,2,5,6,8","organisation_type":"Pharmaceutical company"}
• {"country":"Poland","full_name":"Curandus sp. z o.o.","duties_or_roles":"Site staff augmentation services, patients travel costs reimbursement services","organisation_type":"Health care"}
• {"country":"United Kingdom","full_name":"Medidata Solutions International Limited","duties_or_roles":"Code: 7","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Center For Information And Study On Clinical Research Participation Inc.","duties_or_roles":"Code: 11","organisation_type":"Patient organisation/association"}
• {"country":"United States","full_name":"Certara USA Inc.","duties_or_roles":"Code: 7","organisation_type":"Pharmaceutical company"}
• {"country":"Belgium","full_name":"CluePoints","duties_or_roles":"Data quality assessment services","organisation_type":"Pharmaceutical company"}
• {"country":"Netherlands","full_name":"4G Clinical B.V.","duties_or_roles":"Code: 3","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United Kingdom","full_name":"Drug Development Solutions Limited","duties_or_roles":"Code: 4","organisation_type":"Pharmaceutical company"}
• {"country":"Germany","full_name":"BioAgilytix Europe GmbH","duties_or_roles":"Code: 4","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"IQVIA Laboratories LLC","duties_or_roles":"Code: 4","organisation_type":"Laboratory/Research/Testing facility"}