Clinical trial NCT03971422: A Study to Test Efficacy and Safety of Rozanolixizumab in Adult Patients With Generalized Myasthenia Gravis
Official title: A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study Evaluating Efficacy and Safety of Rozanolixizumab in Adult Patients With Generalized Myasthenia Gravis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Generalized Myasthenia Gravis
Keywords: UCB7665
generalized myasthenia gravis
rozanolixizumab
gMG
Lead sponsor: UCB Pharma
Sponsor class: INDUSTRY
First posted: 2019-06-03
Enrollment: 200
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of the MycarinGstudy is to demonstrate the clinical efficacy and to assess safety and tolerability of rozanolixizumab in patients with generalized myasthenia gravis (MG).


Eligibility 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

Primary outcomes:
measure: Change From Baseline to Day 43 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Score
timeFrame: Baseline and Day 43
Secondary outcomes:
measure: Percentage of Participants Achieving Myasthenia Gravis-Activities of Daily Living (MG-ADL) Response at Day 43
timeFrame: Day 43
measure: Change From Baseline to Day 43 in Myasthenia Gravis-Composite (MG-C) Total Score
timeFrame: Baseline and Day 43
measure: Change From Baseline to Day 43 in Quantitative Myasthenia Gravis (QMG) Total Score
timeFrame: Baseline and Day 43
measure: Change From Baseline to Day 43 in the Myasthenia Gravis (MG) Symptoms Patient Reported Outcome (PRO) 'Muscle Weakness Fatigability' Score
timeFrame: Baseline and Day 43
measure: Change From Baseline to Day 43 in the Myasthenia Gravis (MG) Symptoms Patient Reported Outcome (PRO) 'Physical Fatigue' Score
timeFrame: Baseline and Day 43
measure: Change From Baseline to Day 43 in the Myasthenia Gravis (MG) Symptoms Patient Reported Outcome (PRO) 'Bulbar Symptoms' Score
timeFrame: Baseline and Day 43
measure: Number of Participants With Treatment-emergent Adverse Events (TEAEs)
timeFrame: From Baseline until End of Study Visit (up to Week 14)
measure: Number of Participants With Treatment-emergent Adverse Events (TEAEs) Leading to Withdrawal of Investigational Medicinal Product (IMP)
timeFrame: From Baseline until End of Study Visit (up to Week 14)
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Mg0003 50081
state: Arizona
city: Scottsdale
country: United States
facility: Mg0003 50082
state: Arizona
city: Los Angeles
country: United States
facility: Mg0003 50072
state: California
city: Orange
country: United States
facility: Mg0003 50092
state: California
city: San Francisco
country: United States
facility: Mg0003 50097
state: California
city: San Francisco
country: United States
facility: Mg0003 50099
state: California
city: Aurora
country: United States
facility: Mg0003 50101
state: Colorado
city: Washington
country: United States
facility: Mg0003 50088
state: District of Columbia
city: Miami
country: United States
facility: Mg0003 50122
state: Florida
city: Miami
country: United States
facility: Mg0003 50120
state: Florida
city: Tampa
country: United States
facility: Mg0003 50073
state: Florida
city: Augusta
country: United States
facility: Mg0003 50075
state: Georgia
city: Honolulu
country: United States
facility: Mg0003 50323
state: Hawaii
city: Chicago
country: United States
facility: Mg0003 50109
state: Illinois
city: Indianapolis
country: United States
facility: Mg0003 50114
state: Indiana
city: Fairway
country: United States
facility: Mg0003 50074
state: Kansas
city: Lexington
country: United States
facility: Mg0003 50121
state: Kentucky
city: Ann Arbor
country: United States
facility: Mg0003 50110
state: Michigan
city: Detroit
country: United States
facility: Mg0003 50102
state: Michigan
city: Rochester
country: United States
facility: Mg0003 50104
state: Minnesota
city: Saint Louis
country: United States
facility: Mg0003 50105
state: Missouri
city: New York
country: United States
facility: Mg0003 50077
state: New York
city: Charlotte
country: United States
facility: Mg0003 50117
state: North Carolina
city: Charlotte
country: United States
facility: Mg0003 50086
state: North Carolina
city: Winston-Salem
country: United States
facility: Mg0003 50090
state: North Carolina
city: Columbus
country: United States
facility: Mg0003 50076
state: Ohio
city: Philadelphia
country: United States
facility: Mg0003 50096
state: Pennsylvania
city: Philadelphia
country: United States
facility: Mg0003 50089
state: Pennsylvania
city: Charleston
country: United States
facility: Mg0003 50084
state: South Carolina
city: Houston
country: United States
facility: Mg0003 50113
state: Texas
city: Bruxelles
country: Belgium
facility: Mg0003 40121
city: Calgary
country: Canada
facility: Mg0003 50067
city: Montréal
country: Canada
facility: Mg0003 50066
city: Québec
country: Canada
facility: Mg0003 50070
city: Toronto
country: Canada
facility: Mg0003 50069
city: Ostrava
country: Czechia
facility: Mg0003 40125
city: Praha 2
country: Czechia
facility: Mg0003 40124
city: Aalborg
country: Denmark
facility: Mg0003 40128
city: Aarhus n
country: Denmark
facility: Mg0003 40127
city: Copenhagen
country: Denmark
facility: Mg0003 40126
city: Odense
country: Denmark
facility: Mg0003 40489
city: Bordeaux
country: France
facility: Mg0003 40129
city: Clermont-Ferrand
country: France
facility: Mg0003 40070
city: Garches
country: France
facility: Mg0003 40512
city: Le Kremlin-Bicêtre
country: France
facility: Mg0003 40510
city: Limoges
country: France
facility: Mg0003 40360
city: Lyon
country: France
facility: Mg0003 40426
city: Marseille
country: France
facility: Mg0003 40130
city: Nantes
country: France
facility: Mg0003 40017
city: Nice Cedex 1
country: France
facility: Mg0003 40132
city: Paris
country: France
facility: Mg0003 40133
city: Strasbourg
country: France
facility: Mg0003 40131
city: Tbilisi
country: Georgia
facility: Mg0003 20160
city: Tbilisi
country: Georgia
facility: Mg0003 20161
city: Tbilisi
country: Georgia
facility: Mg0003 20163
city: Tbilisi
country: Georgia
facility: Mg0003 20165
city: Essen
country: Germany
facility: Mg0003 40134
city: Gummersbach
country: Germany
facility: Mg0003 40135
city: Göttingen
country: Germany
facility: Mg0003 40140
city: Jena
country: Germany
facility: Mg0003 40139
city: Leipzig
country: Germany
facility: Mg0003 40078
city: Münster
country: Germany
facility: Mg0003 40177
city: Kistarcsa
country: Hungary
facility: Mg0003 40082
city: Nyiregyhaza
country: Hungary
facility: Mg0003 40178
city: Bologna
country: Italy
facility: Mg0003 40283
city: Lazio
country: Italy
facility: Mg0003 40149
city: Milano
country: Italy
facility: Mg0003 40144
city: Napoli
country: Italy
facility: Mg0003 40307
city: Pavia
country: Italy
facility: Mg0003 40146
city: Roma
country: Italy
facility: Mg0003 40148
city: Roma
country: Italy
facility: Mg0003 40150
city: Bunkyō-Ku
country: Japan
facility: Mg0003 20035
city: Chiba-Shi
country: Japan
facility: Mg0003 20068
city: Hanamaki-Shi
country: Japan
facility: Mg0003 20078
city: Hiroshima
country: Japan
facility: Mg0003 20079
city: Kobe
country: Japan
facility: Mg0003 20075
city: Nagasaki
country: Japan
facility: Mg0003 20071
city: Sapporo
country: Japan
facility: Mg0003 20067
city: Sendai
country: Japan
facility: Mg0003 20077
city: Shinjuku-Ku
country: Japan
facility: Mg0003 20070
city: Shinjuku-Ku
country: Japan
facility: Mg0003 20076
city: Suita
country: Japan
facility: Mg0003 20032
city: Ōsaka-sayama
country: Japan
facility: Mg0003 20074
city: Gdańsk
country: Poland
facility: Mg0003 40155
city: Lublin
country: Poland
facility: Mg0003 40151
city: Poznań
country: Poland
facility: Mg0003 40153
city: Łódź
country: Poland
facility: Mg0003 40154
city: Krasnoyarsk
country: Russian Federation
facility: Mg0003 20168
city: Moscow
country: Russian Federation
facility: Mg0003 20027
city: Novosibirsk
country: Russian Federation
facility: Mg0003 20169
city: Saint Petersburg
country: Russian Federation
facility: Mg0003 20001
city: Saint Petersburg
country: Russian Federation
facility: Mg0003 20028
city: Saint Petersburg
country: Russian Federation
facility: Mg0003 20029
city: Saint Petersburg
country: Russian Federation
facility: Mg0003 20055
city: Samara
country: Russian Federation
facility: Mg0003 20197
city: Belgrade
country: Serbia
facility: Mg0003 40468
city: Niš
country: Serbia
facility: Mg0003 40467
city: Barcelona
country: Spain
facility: Mg0003 40159
city: Barcelona
country: Spain
facility: Mg0003 40160
city: Barcelona
country: Spain
facility: Mg0003 40267
city: Hospitalet de Llobregat
country: Spain
facility: Mg0003 40157
city: Madrid
country: Spain
facility: Mg0003 40161
city: Madrid
country: Spain
facility: Mg0003 40162
city: Murcia
country: Spain
facility: Mg0003 40350
city: Málaga
country: Spain
facility: Mg0003 40341
city: San Sebastián De Los Reyes
country: Spain
facility: Mg0003 40308
city: Taichung
country: Taiwan
facility: Mg0003 20080
city: Taipei
country: Taiwan
facility: Mg0003 20081
city: Taipei
country: Taiwan
facility: Mg0003 20086
city: Taoyuan
country: Taiwan
facility: Mg0003 20082
city: London
country: United Kingdom
facility: Mg0003 40175
city: Nottingham
country: United Kingdom
facility: Mg0003 40168
Study officials:
affiliation: +1 844 599 2273 (UCB)
name: UCB Cares
role: STUDY_DIRECTOR
References:
citation: Bril V, Druzdz A, Grosskreutz J, Habib AA, Mantegazza R, Sacconi S, Utsugisawa K, Vissing J, Vu T, Boehnlein M, Bozorg A, Gayfieva M, Greve B, Woltering F, Kaminski HJ; MG0003 study team. Safety and efficacy of rozanolixizumab in patients with generalised myasthenia gravis (MycarinG): a randomised, double-blind, placebo-controlled, adaptive phase 3 study. Lancet Neurol. 2023 May;22(5):383-394. doi: 10.1016/S1474-4422(23)00077-7.
citation: Habib AA, Sacconi S, Antonini G, Cortes-Vicente E, Grosskreutz J, Mahuwala ZK, Mantegazza R, Pascuzzi RM, Utsugisawa K, Vissing J, Vu T, Wiendl H, Boehnlein M, Greve B, Woltering F, Bril V. Efficacy and safety of rozanolixizumab in patients with muscle-specific tyrosine kinase autoantibody-positive generalised myasthenia gravis: a subgroup analysis of the randomised, double-blind, placebo-controlled, adaptive phase III MycarinG study. Ther Adv Neurol Disord. 2024 Sep 12;17:17562864241273036. doi: 10.1177/17562864241273036. eCollection 2024.
citation: Bril V, Benatar M, Andersen H, Vissing J, Brock M, Greve B, Kiessling P, Woltering F, Griffin L, Van den Bergh P; MG0002 Investigators. Efficacy and Safety of Rozanolixizumab in Moderate to Severe Generalized Myasthenia Gravis: A Phase 2 Randomized Control Trial. Neurology. 2021 Feb 9;96(6):e853-e865. doi: 10.1212/WNL.0000000000011108. Epub 2020 Nov 20.