A Study to Test Efficacy and Safety of Rozanolixizumab in Adult Patients With Generalized Myasthenia Gravis

• Register: ClinicalTrials.gov
• Last refreshed on: 15 May 2023
• Main ID: NCT03971422
• Date of registration: 29/05/2019
• Prospective Registration: Yes
• Primary sponsor: UCB Biopharma SRL
• Public title: A Study to Test Efficacy and Safety of Rozanolixizumab in Adult Patients With Generalized Myasthenia Gravis
• Scientific title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study Evaluating Efficacy and Safety of Rozanolixizumab in Adult Patients With Generalized Myasthenia Gravis
• Date of first enrolment: June 3, 2019
• Target sample size: 200
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03971422
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 3

Countries of recruitment

Belgium, Canada, Czechia, Denmark, France, Georgia, Germany, Hungary, Italy, Japan, Poland, Russian Federation, Serbia, Spain, Taiwan, United Kingdom, United States

Contacts

• Name: UCB Cares
• Affiliation: 001 844 599 2273 (UCB)

Key inclusion & exclusion criteria

Inclusion Criteria:

- Study participant must be =18 years of age, at the time of signing the informed
consent

- Study participant has documented diagnosis of generalized myasthenia gravis (gMG) at
Visit 1, based on study participant's history and supported by previous evaluations

- Study participant has a confirmed positive record of autoantibodies against
acetylcholine receptor (AChR) or muscle-specific kinase (MuSK) at Screening (Visit
1).The presence of autoantibodies may be confirmed with repeat testing at Visit 1

- Study participant has Myasthenia Gravis Foundation of America (MGFA) Class II to IVa
at Visit 1

- Study participant with a Myasthenia Gravis-Activities of Daily Living (MG-ADL) score
of at least 3 (with =3 points from non-ocular symptom) AND a quantitative myasthenia
gravis (QMG) score of at least 11 at Visit 1 and at Baseline (Visit 2)

- Study participant is considered for additional treatment such as intravenous
immunoglobulin g (IVIg) or plasma exchange (PEX) by the Investigator

Exclusion Criteria:

- Study participant has a known history of hyperprolinemia

- Study 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 investigational medicinal product (IMP)

- Study participant with a known tuberculosis (TB) infection, at high risk of acquiring
TB infection, or latent tuberculosis infection (LTBI), or current/history of
nontuberculous mycobacterial infection (NTMBI) will be excluded

- Study participant has experienced hypersensitivity reaction after exposure to other
anti-neonatal Fc receptor (FcRn) drugs

- Study participant with severe (defined as Grade 3 on the Myasthenia Gravis-Activities
of Daily Living (MG-ADL) scale) weakness affecting oropharyngeal or respiratory
muscles, or who has myasthenic crisis or impending crisis at Visit 1 or Visit 2

- Study participant has a history of a solid organ transplant or hematopoietic stem
cell/marrow transplant



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Generalized Myasthenia Gravis

Intervention(s)

Other: Placebo

Drug: Rozanolixizumab

Primary Outcome(s)

Change from Baseline to Day 43 (Visit 10) in Myasthenia Gravis-Activities of Daily Living (MG-ADL) score [Time Frame: Baseline and Visit 10 (Day 43)]

Secondary Outcome(s)

Percentage of participants achieving Myasthenia Gravis-Activities of Daily Living (MG-ADL) response at Visit 10 [Time Frame: Visit 10 (Day 43)]

Change from Baseline to Day 43 (Visit 10) in the Myasthenia Gravis (MG) Symptoms Patient Reported Outcome (PRO) 'Muscle Weakness Fatigability' score [Time Frame: Baseline and Visit 10 (Day 43)]

Occurrence of treatment-emergent adverse events (TEAEs) [Time Frame: From Baseline until End of Study Visit (up to Week 14)]

Change from Baseline to Day 43 (Visit 10) in Quantitative Myasthenia Gravis (QMG) score [Time Frame: Baseline and Visit 10 (Day 43)]

Change from Baseline to Day 43 (Visit 10) in Myasthenia Gravis-Composite (MG-C) score [Time Frame: Baseline and Visit 10 (Day 43)]

Change from Baseline to Day 43 (Visit 10) in the Myasthenia Gravis (MG) Symptoms Patient Reported Outcome (PRO) 'Physical Fatigue' score [Time Frame: Baseline and Visit 10 (Day 43)]

Change from Baseline to Day 43 (Visit 10) in the Myasthenia Gravis (MG) Symptoms Patient Reported Outcome (PRO) 'Bulbar Symptoms' score [Time Frame: Baseline and Visit 10 (Day 43)]

Treatment-emergent adverse events (TEAEs) leading to withdrawal of investigational medicinal product (IMP) [Time Frame: From Baseline until End of Study Visit (up to Week 14)]

Secondary ID(s)

MG0003

2019-000968-18

Source(s) of Monetary Support

Please refer to primary and secondary sponsors