Clinical trial NCT04124965: A Study to Investigate the Long-term Safety, Tolerability, and Efficacy of Rozanolixizumab in Adult Patients With Generalized Myasthenia Gravis
Official title: A Randomized, Open-Label Extension Study to Investigate the Long-Term Safety, Tolerability, and Efficacy 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: 2019-10-14
Enrollment: 71
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 the MycarinGstudy is to evaluate the long-term safety, tolerability and long-term efficacy of rozanolixizumab in study participants with generalized myasthenia gravis (MG).


Eligibility criteria: Inclusion Criteria:

* Participant was eligible for MG0003 \[NCT03971422\] or MGC003 at the time of enrollment into either study and the participant either completed the observation Period of MG0003 or MGC003 or required rescue therapy during the Observation Period of the lead-in studies
* Body weight ≥35 kg at Visit 1
* Study participants may be male or female

Exclusion Criteria:

* Evidence of active or latent tuberculosis (TB) as documented by medical history and examination, if applicable, chest X-rays (posterior anterior and lateral), and TB testing by a positive (not indeterminate) QuantiFERON®-TB Gold Plus
* Participant has received a live vaccination within 8 weeks prior to the Baseline visit; or intends to have a live vaccination during the course of the study or within 8 weeks following the final dose of study medication
* 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-activates of daily living (MG-ADL) scale) weakness affecting oropharyngeal or respiratory muscles, or who has myasthenic crisis or impending crisis
* Participant has any laboratory abnormality that, in the opinion of the Investigator, is clinically significant, has not resolved at randomization, and could jeopardize or compromise the study participant's ability to participate in this study
* Study participant met any mandatory withdrawal or mandatory study drug discontinuation criteria MG0003 \[NCT03971422\] or MGC003, or discontinued study medication in either study, with the exception of discontinuation due to a need for rescue treatment
* Study participant is not considered capable of adhering to the protocol visit schedule, or medication intake according to the judgment of the Investigator
* Study participant has a lifetime history of suicide attempt (including an active attempt, interrupted attempt, or aborted attempt), or had suicidal ideation since the last visit in MG0003 as indicated by a positive response (Yes) to either Question 4 or Question 5 of the Columbia-Suicide Severity Rating Scale (C-SSRS)

Primary outcomes:
measure: Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)
timeFrame: From Baseline until End of Study (up to Week 60)
measure: Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) Leading to Permanent Withdrawal of Study Medication
timeFrame: From Baseline until End of Study (up to Week 60)
Secondary outcomes:
measure: Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Each Scheduled Assessment During Treatment and Observation Periods
timeFrame: Baseline, Weeks 5, 7, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 52 and 60
measure: Change From Baseline in Myasthenia Gravis-Composite (MG-C) Total Score at Each Scheduled Assessment During Treatment and Observation Periods
timeFrame: Baseline, Weeks 5, 7, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 52 and 60
measure: Change From Baseline in Quantitative Myasthenia Gravis (QMG) Total Score at Each Scheduled Assessment During Treatment and Observation Periods
timeFrame: Baseline, Weeks 5, 7, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 52 and 60
measure: Percentage of Participants Using Rescue Medication (Intravenous Infusion of Immunoglobulin G (IVIg) or Plasma Exchange (PEX))
timeFrame: From Baseline until End of Study (up to Week 60)
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Mg0004 50081
state: Arizona
city: Los Angeles
country: United States
facility: Mg0004 50072
state: California
city: Washington
country: United States
facility: Mg0004 50088
state: District of Columbia
city: Miami
country: United States
facility: Mg0004 50120
state: Florida
city: Tampa
country: United States
facility: Mg0004 50073
state: Florida
city: Indianapolis
country: United States
facility: Mg0004 50114
state: Indiana
city: Lexington
country: United States
facility: Mg0004 50121
state: Kentucky
city: New York
country: United States
facility: Mg0004 50077
state: New York
city: Winston-Salem
country: United States
facility: Mg0004 50090
state: North Carolina
city: Philadelphia
country: United States
facility: Mg0004 50096
state: Pennsylvania
city: Montréal
country: Canada
facility: Mg0004 50066
city: Québec
country: Canada
facility: Mg0004 50070
city: Toronto
country: Canada
facility: Mg0004 50069
city: Ostrava-Poruba
country: Czechia
facility: Mg0004 40125
city: Praha 2
country: Czechia
facility: Mg0004 40124
city: Aalborg
country: Denmark
facility: Mg0004 40128
city: Bordeaux
country: France
facility: Mg0004 40129
city: Nice
country: France
facility: Mg0004 40132
city: Paris
country: France
facility: Mg0004 40133
city: Strasbourg
country: France
facility: Mg0004 40131
city: Göttingen
country: Germany
facility: Mg0004 40140
city: Leipzig
country: Germany
facility: Mg0004 40078
city: Münster
country: Germany
facility: Mg0004 40177
city: Milano
country: Italy
facility: Mg0004 40144
city: Pavia
country: Italy
facility: Mg0004 40146
city: Roma
country: Italy
facility: Mg0004 40148
city: Roma
country: Italy
facility: Mg0004 40150
city: Hanamaki shi
country: Japan
facility: Mg0004 20078
city: Hiroshima
country: Japan
facility: Mg0004 20079
city: Miyagi
country: Japan
facility: Mg0004 20077
city: Shinjuku-Ku
country: Japan
facility: Mg0004 20076
city: Suita
country: Japan
facility: Mg0004 20032
city: Gdańsk
country: Poland
facility: Mg0004 40155
city: Lublin
country: Poland
facility: Mg0004 40151
city: Łódź
country: Poland
facility: Mg0004 40154
city: Moscow
country: Russian Federation
facility: Mg0004 20027
city: Saint Petersburg
country: Russian Federation
facility: Mg0004 20001
city: Saint Petersburg
country: Russian Federation
facility: Mg0004 20028
city: Saint Petersburg
country: Russian Federation
facility: Mg0004 20055
city: Barcelona
country: Spain
facility: Mg0004 40159
city: Hospitalet de Llobregat
country: Spain
facility: Mg0004 40157
city: Taichung
country: Taiwan
facility: Mg0004 20080
city: Taipei
country: Taiwan
facility: Mg0004 20081
Study officials:
affiliation: 001 844 599 22733 (UCB)
name: UCB Cares
role: STUDY_DIRECTOR
References: