Clinical trial NCT04650854: A Study to Evaluate Rozanolixizumab in Study Participants With Generalized Myasthenia Gravis
Official title: An Open-Label Extension Study to Evaluate Rozanolixizumab in Study Participants 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: 2020-12-03
Enrollment: 165
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to assess the safety, tolerability and efficacy of additional 6-week treatment cycles with rozanolixizumab in study participants with generalized myasthenia gravis (gMG).


Eligibility criteria: Inclusion Criteria:

* Study participant must meet one of the following:

  1. completed MG0003 \[NCT03971422\]
  2. required rescue therapy during the Observation Period in MG0003 or
  3. completed at least 6 visits in MG0004 \[NCT04124965\]
* Body weight ≥35 kg at Baseline (Day 1)
* Study participants may be male or female

Exclusion Criteria:

* Study participant has a known hypersensitivity to any components of the study medication or other anti-neonatal Fc receptor (FcRn) medications
* 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)
* Study participant met any mandatory withdrawal or mandatory study drug discontinuation criteria in MG0003, or MG0004, or permanently discontinued study drug in either study
* Study participant intends to have a live vaccination during the course of the study or within 8 weeks following the final dose of rozanolixizumab
* 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

Primary outcomes:
measure: Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)
timeFrame: From Baseline (Day 1) to End of Study (up to 34 months)
measure: Percentage of Participants With TEAEs Leading to Withdrawal of Investigational Medicinal Product (IMP)
timeFrame: From Baseline (Day 1) to End of Study (up to 34 months)
Secondary outcomes:
measure: Change From Baseline (Day 1) to Day 43 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Score Within One Treatment Cycle (Cycle 1, 2, and 3)
timeFrame: From Baseline (Day 1) to Day 43 of each cycle (Cycles 1, 2, and 3)
measure: Change From Baseline (Day 1) to Day 43 in Quantitative Myasthenia Gravis (QMG) Score Within One Treatment Cycle (Cycle 1, 2, and 3)
timeFrame: From Baseline (Day 1) to Day 43 of each cycle (Cycles 1, 2, and 3)
measure: Change From Baseline (Day 1) to Day 43 in Myasthenia Gravis-Composite (MG-C) Score Within One Treatment Cycle (Cycle 1, 2, and 3)
timeFrame: From Baseline (Day 1) to Day 43 of each cycle (Cycles 1, 2, and 3)
measure: Change From Baseline (Day 1) to Day 43 in Myasthenia Gravis (MG) Symptoms Patient Reported Outcome (PRO) 'Muscle Weakness Fatigability' Score Within One Treatment Cycle (Cycle 1, 2, and 3)
timeFrame: From Baseline (Day 1) to Day 43 of each cycle (Cycles 1, 2, and 3)
measure: Change From Baseline (Day 1) to Day 43 in MG Symptoms PRO 'Physical Fatigue' Score Within One Treatment Cycle (Cycle 1, 2, and 3)
timeFrame: From Baseline (Day 1) to Day 43 of each cycle (Cycles 1, 2, and 3)
measure: Change From Baseline (Day 1) to Day 43 in MG Symptoms PRO 'Bulbar Muscle Weakness' Score Within One Treatment Cycle (Cycle 1, 2, and 3)
timeFrame: From Baseline (Day 1) to Day 43 of each cycle (Cycles 1, 2, and 3)
measure: MG-ADL Responder Rate (>=2.0-point Improvement From Baseline [Day 1]) Within One Treatment Cycle (Cycle 1, 2, and 3 [Day 43])
timeFrame: Day 43 of Cycle 1, 2, and 3
measure: Time to MG-ADL Response (>=2.0-point Improvement From Baseline [Day 1]) Within One Treatment Cycle (Cycle 1, 2, and 3)
timeFrame: From Baseline (Day 1) to Day 43 of each cycle (Cycles 1, 2, and 3)
measure: Time Between Consecutive Treatment Cycles
timeFrame: From end of the 6-week treatment cycle (Day 43) to the next 6-week treatment cycle (Day 1), assessed up to 2.5 years
Other outcomes:
Locations:
city: Orange
country: United States
facility: Mg0007 50092
state: California
city: San Francisco
country: United States
facility: Mg0007 50099
state: California
city: Miami
country: United States
facility: Mg0007 50122
state: Florida
city: Miami
country: United States
facility: Mg0007 50120
state: Florida
city: Tampa
country: United States
facility: Mg0007 50073
state: Florida
city: Augusta
country: United States
facility: Mg0007 50075
state: Georgia
city: Honolulu
country: United States
facility: Mg0007 50323
state: Hawaii
city: Indianapolis
country: United States
facility: Mg0007 50114
state: Indiana
city: Lexington
country: United States
facility: Mg0007 50121
state: Kentucky
city: New York
country: United States
facility: Mg0007 50077
state: New York
city: Winston-Salem
country: United States
facility: Mg0007 50090
state: North Carolina
city: Philadelphia
country: United States
facility: Mg0007 50096
state: Pennsylvania
city: Houston
country: United States
facility: Mg0007 50113
state: Texas
city: Edmonton
country: Canada
facility: Mg0007 50071
city: Montreal
country: Canada
facility: Mg0007 50066
city: Montreal
country: Canada
facility: Mg0007 50124
city: Quebec
country: Canada
facility: Mg0007 50070
city: Toronto
country: Canada
facility: Mg0007 50069
city: Ostrava - Poruba
country: Czechia
facility: Mg0007 40125
city: Praha 2
country: Czechia
facility: Mg0007 40124
city: Aalborg
country: Denmark
facility: Mg0007 40128
city: Aarhus
country: Denmark
facility: Mg0007 40127
city: Copenhagen
country: Denmark
facility: Mg0007 40126
city: Bordeaux
country: France
facility: Mg0007 40129
city: Limoges
country: France
facility: Mg0007 40360
city: Nice Cedex 1
country: France
facility: Mg0007 40132
city: Paris
country: France
facility: Mg0007 40133
city: Strasbourg
country: France
facility: Mg0007 40131
city: Tbilisi
country: Georgia
facility: Mg0007 20160
city: Tbilisi
country: Georgia
facility: Mg0007 20161
city: Tbilisi
country: Georgia
facility: Mg0007 20163
city: Tbilisi
country: Georgia
facility: Mg0007 20164
city: Tbilisi
country: Georgia
facility: Mg0007 20165
city: Essen
country: Germany
facility: Mg0007 40134
city: Göttingen
country: Germany
facility: Mg0007 40140
city: Jena
country: Germany
facility: Mg0007 40139
city: Leipzig
country: Germany
facility: Mg0007 40078
city: Münster
country: Germany
facility: Mg0007 40177
city: Bologna
country: Italy
facility: Mg0007 40283
city: Milano
country: Italy
facility: Mg0007 40144
city: Napoli
country: Italy
facility: Mg0007 40307
city: Pavia
country: Italy
facility: Mg0007 40146
city: Roma
country: Italy
facility: Mg0007 40148
city: Roma
country: Italy
facility: Mg0007 40150
city: Bunkyo-ku
country: Japan
facility: Mg0007 20035
city: Chiba-shi
country: Japan
facility: Mg0007 20068
city: Hanamaki-shi
country: Japan
facility: Mg0007 20078
city: Hiroshima
country: Japan
facility: Mg0007 20079
city: Kobe
country: Japan
facility: Mg0007 20075
city: Nagasaki-shi
country: Japan
facility: Mg0007 20071
city: Sendai
country: Japan
facility: Mg0007 20077
city: Shinjuku-ku
country: Japan
facility: Mg0007 20070
city: Shinjuku-ku
country: Japan
facility: Mg0007 20076
city: Suita
country: Japan
facility: Mg0007 20032
city: Gdansk
country: Poland
facility: Mg0007 40155
city: Lodz
country: Poland
facility: Mg0007 40154
city: Lublin
country: Poland
facility: Mg0007 40151
city: Poznan
country: Poland
facility: Mg0007 40153
city: Novosibirsk
country: Russian Federation
facility: Mg0007 20169
city: Saint-petersburg
country: Russian Federation
facility: Mg0007 20001
city: Saint-petersburg
country: Russian Federation
facility: Mg0007 20028
city: Saint-petersburg
country: Russian Federation
facility: Mg0007 20055
city: NIS
country: Serbia
facility: Mg0007 40467
city: Barcelona
country: Spain
facility: Mg0007 40160
city: Hospitalet de Llobregat
country: Spain
facility: Mg0007 40157
city: Murcia
country: Spain
facility: Mg0007 40350
city: San Sebastián de Los Reyes
country: Spain
facility: Mg0007 40308
city: Taipei City
country: Taiwan
facility: Mg0007 20081
city: Taipei City
country: Taiwan
facility: Mg0007 20086
Study officials:
affiliation: 001 844 599 22733 (UCB)
name: UCB Cares
role: STUDY_DIRECTOR
References: