Clinical trial NCT04225871: Open-Label Extension of Zilucoplan in Subjects With Generalized Myasthenia Gravis
Official title: A Phase 3, Multicenter, Open-Label Extension Study of Zilucoplan in Subjects With Generalized Myasthenia Gravis
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Generalized Myasthenia Gravis
Lead sponsor: UCB Pharma
Sponsor class: INDUSTRY
First posted: 2020-01-13
Enrollment: 200
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The RAISE-XT study is an open-label extension study to evaluate the long-term efficacy, safety, and tolerability of zilucoplan in subjects with gMG who have previously participated in a qualifying Ra Pharmaceuticals sponsored zilucoplan study.


Eligibility criteria: Inclusion Criteria:

* Completion of a qualifying zilucoplan study

Exclusion Criteria:

* With the exception of a prior zilucoplan trial, participation in another concurrent clinical trial involving an experimental therapeutic intervention (participation in observational studies and/or registry studies is permitted)

Primary outcomes:
measure: Incidence of treatment-emergent adverse events (TEAEs)
timeFrame: From Baseline (Day 1) to Safety Follow-Up Visit (up to 36 months)
Secondary outcomes:
measure: Change from Baseline to Week 12 in the Myasthenia Gravis-Activities of Daily Living (MG-ADL) Score
timeFrame: From Baseline (Day 1) to Week 12
measure: Change from Baseline to Week 12 in the the Quantitative Myasthenia Gravis (QMG) Score
timeFrame: From Baseline (Day 1) to Week 12
measure: Change from Baseline to Week 12 in the Myasthenia Gravis Composite (MGC) Score
timeFrame: From Baseline (Day 1) to Week 12
measure: Change from Baseline to Week 12 in the Myasthenia Gravis - Quality of Life revised (MG-QOL15r) Score
timeFrame: From Baseline (Day 1) to Week 12
Other outcomes:
Locations:
city: Mobile
country: United States
facility: Mg0011 41
state: Alabama
city: Phoenix
country: United States
facility: Mg0011 116
state: Arizona
city: Irvine
country: United States
facility: Mg0011 31
state: California
city: Los Angeles
country: United States
facility: MG0011 4
state: California
city: Pasadena
country: United States
facility: Mg0011 220
state: California
city: San Francisco
country: United States
facility: Mg0011 160
state: California
city: New Haven
country: United States
facility: Mg0011 24
state: Connecticut
city: Washington
country: United States
facility: Mg0011 27
state: District of Columbia
city: Tampa
country: United States
facility: Mg0011 25
state: Florida
city: Augusta
country: United States
facility: Mg0011 135
state: Georgia
city: Glenview
country: United States
facility: Mg0011 188
state: Illinois
city: Indianapolis
country: United States
facility: Mg0011 156
state: Indiana
city: Kansas City
country: United States
facility: Mg0011 32
state: Kansas
city: Detroit
country: United States
facility: Mg0011 33
state: Michigan
city: East Lansing
country: United States
facility: Mg0011 49
state: Michigan
city: Columbia
country: United States
facility: Mg0011 134
state: Missouri
city: Las Vegas
country: United States
facility: Mg0011 117
state: Nevada
city: Buffalo
country: United States
facility: Mg0011 30
state: New York
city: Great Neck
country: United States
facility: Mg0011 123
state: New York
city: New York
country: United States
facility: Mg0011 23
state: New York
city: New York
country: United States
facility: Mg0011 47
state: New York
city: Chapel Hill
country: United States
facility: Mg0011 22
state: North Carolina
city: Cleveland
country: United States
facility: Mg0011 122
state: Ohio
city: Columbus
country: United States
facility: Mg0011 38
state: Ohio
city: Pittsburgh
country: United States
facility: Mg0011 40
state: Pennsylvania
city: Charleston
country: United States
facility: Mg0011 128
state: South Carolina
city: Cordova
country: United States
facility: Mg0011 28
state: Tennessee
city: Austin
country: United States
facility: Mg0011 131
state: Texas
city: Dallas
country: United States
facility: Mg0011 19
state: Texas
city: Salt Lake City
country: United States
facility: Mg0011 39
state: Utah
city: Charlottesville
country: United States
facility: Mg0011 164
state: Virginia
city: Seattle
country: United States
facility: Mg0011 154
state: Washington
city: Milwaukee
country: United States
facility: Mg0011 45
state: Wisconsin
city: Montreal
country: Canada
facility: Mg0011 11
city: Lille
country: France
facility: Mg0011 204
city: Nice
country: France
facility: Mg0011 118
city: Paris
country: France
facility: Mg0011 105
city: Strasbourg
country: France
facility: Mg0011 137
city: Göttingen
country: Germany
facility: Mg0011 150
city: Tübingen
country: Germany
facility: Mg0011 129
city: Milano
country: Italy
facility: Mg0011 132
city: Roma
country: Italy
facility: Mg0011 126
city: Chiba
country: Japan
facility: Mg0011 151
city: Hanamaki-shi
country: Japan
facility: Mg0011 136
city: Kita-gun
country: Japan
facility: Mg0011 179
city: Meguro-ku
country: Japan
facility: Mg0011 153
city: Nagasaki-shi
country: Japan
facility: Mg0011 146
city: Narita
country: Japan
facility: Mg0011 169
city: Sapporo
country: Japan
facility: Mg0011 152
city: Sendai
country: Japan
facility: Mg0011 144
city: Shinjuku-ku
country: Japan
facility: Mg0011 163
city: Tokyo
country: Japan
facility: Mg0011 141
city: Bergen
country: Norway
facility: Mg0011 140
city: Oslo
country: Norway
facility: Mg0011 143
city: Katowice
country: Poland
facility: Mg0011 195
city: Katowice
country: Poland
facility: Mg0011 213
city: Krakow
country: Poland
facility: Mg0011 192
city: Krakow
country: Poland
facility: Mg0011 193
city: Kraków
country: Poland
facility: Mg0011 211
city: Lublin
country: Poland
facility: Mg0011 205
city: Nowa Sól
country: Poland
facility: Mg0011 194
city: Poznań
country: Poland
facility: Mg0011 209
city: Warszawa
country: Poland
facility: Mg0011 201
city: Zabrze
country: Poland
facility: Mg0011 210
city: Łódź
country: Poland
facility: Mg0011 214
city: Barcelona
country: Spain
facility: Mg0011 133
city: Barcelona
country: Spain
facility: Mg0011 168
city: Bilbao
country: Spain
facility: Mg0011 138
city: Oxford
country: United Kingdom
facility: Mg0011 119
city: Sheffield
country: United Kingdom
facility: Mg0011 130
Study officials:
affiliation: 001 844 599 2273
name: UCB Cares
role: STUDY_DIRECTOR
References:
citation: Weiss MD, Freimer M, Leite MI, Maniaol A, Utsugisawa K, Bloemers J, Boroojerdi B, Howard E, Savic N, Howard JF Jr. Improvement of fatigue in generalised myasthenia gravis with zilucoplan. J Neurol. 2024 May;271(5):2758-2767. doi: 10.1007/s00415-024-12209-3. Epub 2024 Feb 24.
citation: de la Borderie G, Chimits D, Boroojerdi B, Brock M, Duda PW, Grimson F, Mahoney P, Strimenopoulou F, Cutter G, Aban I, Brauner S, Petersson M, Howard JF Jr, Bennett N. Maintenance of zilucoplan efficacy in patients with generalised myasthenia gravis up to 24 weeks: a model-informed analysis. Ther Adv Neurol Disord. 2024 Sep 21;17:17562864241279125. doi: 10.1177/17562864241279125. eCollection 2024.