Back to resultsA Study to Investigate the Long-term Safety, Tolerability, and Efficacy of Rozanolixizumab in Adult Patients With Generalized Myasthenia Gravis
Primary Purpose
Generalized Myasthenia Gravis
Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Rozanolixizumab
Sponsored by
About this trial
This is an interventional treatment trial for Generalized Myasthenia Gravis focused on measuring UCB7665, generalized myasthenia gravis, rozanolixizumab, gMG
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)
Sites / Locations
- Mg0004 50081
- Mg0004 50072
- Mg0004 50088
- Mg0004 50120
- Mg0004 50073
- Mg0004 50114
- Mg0004 50121
- Mg0004 50077
- Mg0004 50090
- Mg0004 50096
- Mg0004 50066
- Mg0004 50070
- Mg0004 50069
- Mg0004 40125
- Mg0004 40124
- Mg0004 40128
- Mg0004 40129
- Mg0004 40132
- Mg0004 40133
- Mg0004 40131
- Mg0004 40140
- Mg0004 40078
- Mg0004 40177
- Mg0004 40144
- Mg0004 40146
- Mg0004 40148
- Mg0004 40150
- Mg0004 20078
- Mg0004 20079
- Mg0004 20077
- Mg0004 20076
- Mg0004 20032
- Mg0004 40155
- Mg0004 40151
- Mg0004 40154
- Mg0004 20027
- Mg0004 20001
- Mg0004 20028
- Mg0004 20055
- Mg0004 40159
- Mg0004 40157
- Mg0004 20080
- Mg0004 20081
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Experimental
Arm Label
Rozanolixizumab dosage regimen 1
Rozanolixizumab dosage regimen 2
Arm Description
Study participants randomized to dosage regimen 1 will receive assigned dosage of rozanolixizumab at pre-specified time points during the Treatment Period. The dose regimen may be switched based on investigator discretion.
Study participants randomized to dosage regimen 2 will receive assigned dosage of rozanolixizumab at pre-specified time points during the Treatment Period. The dose regimen may be switched based on investigator discretion.
Outcomes
Primary Outcome Measures
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)
A TEAE is defined as an AE starting on or after the time of first administration of investigational medicinal product (IMP) or any unresolved event already present before the first administration of IMP that worsened in intensity following exposure to IMP, up to 8 weeks after the last dose of IMP in study participants who discontinued the study or IMP.
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) Leading to Permanent Withdrawal of Study Medication
A TEAE is defined as an AE starting on or after the time of first administration of IMP or any unresolved event already present before the first administration of IMP that worsened in intensity following exposure to IMP, up to 8 weeks after the last dose of IMP in study participants who discontinued the study or IMP.
Secondary Outcome Measures
Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Each Scheduled Assessment During Treatment and Observation Periods
The Myasthenia Gravis Activities of Daily Living (MG-ADL) is an 8-item patient-reported outcome (PRO) instrument developed on the basis of the Quantitative Myasthenia Gravis (QMG). The MG-ADL targeted symptoms and disability across ocular, bulbar, respiratory, and axial symptoms. The total MG-ADL score was obtained by summing the responses to each individual item (8 items; Grades: 0, 1, 2, 3), where 0 represents no symptoms or impaired performance and 3 represents the most severe symptoms or impaired performance. The total score ranges from 0 to 24, with a higher score indicating more disability. A positive change indicates worsening and a negative change indicates improvement.
Change From Baseline in Myasthenia Gravis-Composite (MG-C) Total Score at Each Scheduled Assessment During Treatment and Observation Periods
MG-C scale is a validated assessment and scale tests 10 items with individual items being weighted differently. The items included ptosis/upward gaze (range: 0 [>45 second] - 3 [Immediate]), double vision on lateral gaze (range: 0 [>45 second] - 4 [Immediate]), eye closure (range: 0 [Normal] - 2 [severe weakness]), talking (range: 0 [Normal] - 6 [difficult to understand speech]), chewing (range: 0 [Normal] - 6 [gastric tube]), swallowing (range: 0 [Normal] - 6 [gastric tube]), breathing (range: 0 [Normal] - 9 [ventilator dependence]), neck flexion (range: 0 [Normal] - 4 [severe weakness]), shoulder abduction (range: 0 [Normal] - 5 [severe weakness]) and hip flexion (range: 0 [Normal] - 5 [severe weakness]), lower scores= lower disease activity. Total MG-C score was obtained by summing responses to each individual item and score ranges from 0 to 50, with lower scores indicating lower disease activity. A positive change indicates worsening and a negative change indicates improvement.
Change From Baseline in Quantitative Myasthenia Gravis (QMG) Total Score at Each Scheduled Assessment During Treatment and Observation Periods
The QMG is a validated assessment and the scale tested 13 items, including ocular and facial involvement, swallowing, speech, limb strength, and forced vital capacity. The total QMG score was obtained by summing the responses to each individual item (13 items; Responses: None=0, Mild=1, Moderate=2, Severe=3) and the score ranges from 0 to 39, with lower scores indicating lower disease activity. A positive change indicates worsening and a negative change indicates improvement.
Percentage of Participants Using Rescue Medication (Intravenous Infusion of Immunoglobulin G (IVIg) or Plasma Exchange (PEX))
Rescue therapy consisted of IVIg or PEX. Study participants who experienced disease worsening (eg, an increase of 2 points on the MG-ADL or 3 points on the QMG scale between 2 consecutive visits) may be considered for rescue therapy at the discretion of the Investigator.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT04124965
Brief Title
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
2. Study Status
Record Verification Date
August 2023
Overall Recruitment Status
Completed
Study Start Date
October 29, 2019 (Actual)
Primary Completion Date
September 1, 2021 (Actual)
Study Completion Date
September 1, 2021 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
UCB Biopharma SRL
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
No
Data Monitoring Committee
Yes
5. Study Description
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).
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Generalized Myasthenia Gravis
Keywords
UCB7665, generalized myasthenia gravis, rozanolixizumab, gMG
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
71 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Rozanolixizumab dosage regimen 1
Arm Type
Experimental
Arm Description
Study participants randomized to dosage regimen 1 will receive assigned dosage of rozanolixizumab at pre-specified time points during the Treatment Period. The dose regimen may be switched based on investigator discretion.
Arm Title
Rozanolixizumab dosage regimen 2
Arm Type
Experimental
Arm Description
Study participants randomized to dosage regimen 2 will receive assigned dosage of rozanolixizumab at pre-specified time points during the Treatment Period. The dose regimen may be switched based on investigator discretion.
Intervention Type
Drug
Intervention Name(s)
Rozanolixizumab
Other Intervention Name(s)
UCB7665
Intervention Description
Rozanolixizumab will be administered by subcutaneous infusion in dosage regimen 1 or 2.
Primary Outcome Measure Information:
Title
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)
Description
A TEAE is defined as an AE starting on or after the time of first administration of investigational medicinal product (IMP) or any unresolved event already present before the first administration of IMP that worsened in intensity following exposure to IMP, up to 8 weeks after the last dose of IMP in study participants who discontinued the study or IMP.
Time Frame
From Baseline until End of Study (up to Week 60)
Title
Percentage of Participants With Treatment-emergent Adverse Events (TEAEs) Leading to Permanent Withdrawal of Study Medication
Description
A TEAE is defined as an AE starting on or after the time of first administration of IMP or any unresolved event already present before the first administration of IMP that worsened in intensity following exposure to IMP, up to 8 weeks after the last dose of IMP in study participants who discontinued the study or IMP.
Time Frame
From Baseline until End of Study (up to Week 60)
Secondary Outcome Measure Information:
Title
Change From Baseline in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score at Each Scheduled Assessment During Treatment and Observation Periods
Description
The Myasthenia Gravis Activities of Daily Living (MG-ADL) is an 8-item patient-reported outcome (PRO) instrument developed on the basis of the Quantitative Myasthenia Gravis (QMG). The MG-ADL targeted symptoms and disability across ocular, bulbar, respiratory, and axial symptoms. The total MG-ADL score was obtained by summing the responses to each individual item (8 items; Grades: 0, 1, 2, 3), where 0 represents no symptoms or impaired performance and 3 represents the most severe symptoms or impaired performance. The total score ranges from 0 to 24, with a higher score indicating more disability. A positive change indicates worsening and a negative change indicates improvement.
Time Frame
Baseline, Weeks 5, 7, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 52 and 60
Title
Change From Baseline in Myasthenia Gravis-Composite (MG-C) Total Score at Each Scheduled Assessment During Treatment and Observation Periods
Description
MG-C scale is a validated assessment and scale tests 10 items with individual items being weighted differently. The items included ptosis/upward gaze (range: 0 [>45 second] - 3 [Immediate]), double vision on lateral gaze (range: 0 [>45 second] - 4 [Immediate]), eye closure (range: 0 [Normal] - 2 [severe weakness]), talking (range: 0 [Normal] - 6 [difficult to understand speech]), chewing (range: 0 [Normal] - 6 [gastric tube]), swallowing (range: 0 [Normal] - 6 [gastric tube]), breathing (range: 0 [Normal] - 9 [ventilator dependence]), neck flexion (range: 0 [Normal] - 4 [severe weakness]), shoulder abduction (range: 0 [Normal] - 5 [severe weakness]) and hip flexion (range: 0 [Normal] - 5 [severe weakness]), lower scores= lower disease activity. Total MG-C score was obtained by summing responses to each individual item and score ranges from 0 to 50, with lower scores indicating lower disease activity. A positive change indicates worsening and a negative change indicates improvement.
Time Frame
Baseline, Weeks 5, 7, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 52 and 60
Title
Change From Baseline in Quantitative Myasthenia Gravis (QMG) Total Score at Each Scheduled Assessment During Treatment and Observation Periods
Description
The QMG is a validated assessment and the scale tested 13 items, including ocular and facial involvement, swallowing, speech, limb strength, and forced vital capacity. The total QMG score was obtained by summing the responses to each individual item (13 items; Responses: None=0, Mild=1, Moderate=2, Severe=3) and the score ranges from 0 to 39, with lower scores indicating lower disease activity. A positive change indicates worsening and a negative change indicates improvement.
Time Frame
Baseline, Weeks 5, 7, 9, 13, 17, 21, 25, 29, 33, 37, 41, 45, 49, 52 and 60
Title
Percentage of Participants Using Rescue Medication (Intravenous Infusion of Immunoglobulin G (IVIg) or Plasma Exchange (PEX))
Description
Rescue therapy consisted of IVIg or PEX. Study participants who experienced disease worsening (eg, an increase of 2 points on the MG-ADL or 3 points on the QMG scale between 2 consecutive visits) may be considered for rescue therapy at the discretion of the Investigator.
Time Frame
From Baseline until End of Study (up to Week 60)
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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)
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
UCB Cares
Organizational Affiliation
001 844 599 22733 (UCB)
Official's Role
Study Director
Facility Information:
Facility Name
Mg0004 50081
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85013-4409
Country
United States
Facility Name
Mg0004 50072
City
Los Angeles
State/Province
California
ZIP/Postal Code
90033
Country
United States
Facility Name
Mg0004 50088
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20037
Country
United States
Facility Name
Mg0004 50120
City
Miami
State/Province
Florida
ZIP/Postal Code
33144
Country
United States
Facility Name
Mg0004 50073
City
Tampa
State/Province
Florida
ZIP/Postal Code
33612
Country
United States
Facility Name
Mg0004 50114
City
Indianapolis
State/Province
Indiana
ZIP/Postal Code
46202
Country
United States
Facility Name
Mg0004 50121
City
Lexington
State/Province
Kentucky
ZIP/Postal Code
40536
Country
United States
Facility Name
Mg0004 50077
City
New York
State/Province
New York
ZIP/Postal Code
10021
Country
United States
Facility Name
Mg0004 50090
City
Winston-Salem
State/Province
North Carolina
ZIP/Postal Code
27157
Country
United States
Facility Name
Mg0004 50096
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
Mg0004 50066
City
Montréal
Country
Canada
Facility Name
Mg0004 50070
City
Québec
Country
Canada
Facility Name
Mg0004 50069
City
Toronto
Country
Canada
Facility Name
Mg0004 40125
City
Ostrava-Poruba
Country
Czechia
Facility Name
Mg0004 40124
City
Praha 2
Country
Czechia
Facility Name
Mg0004 40128
City
Aalborg
Country
Denmark
Facility Name
Mg0004 40129
City
Bordeaux
Country
France
Facility Name
Mg0004 40132
City
Nice
Country
France
Facility Name
Mg0004 40133
City
Paris
Country
France
Facility Name
Mg0004 40131
City
Strasbourg
Country
France
Facility Name
Mg0004 40140
City
Göttingen
Country
Germany
Facility Name
Mg0004 40078
City
Leipzig
Country
Germany
Facility Name
Mg0004 40177
City
Münster
Country
Germany
Facility Name
Mg0004 40144
City
Milano
Country
Italy
Facility Name
Mg0004 40146
City
Pavia
Country
Italy
Facility Name
Mg0004 40148
City
Roma
Country
Italy
Facility Name
Mg0004 40150
City
Roma
Country
Italy
Facility Name
Mg0004 20078
City
Hanamaki shi
Country
Japan
Facility Name
Mg0004 20079
City
Hiroshima
Country
Japan
Facility Name
Mg0004 20077
City
Miyagi
Country
Japan
Facility Name
Mg0004 20076
City
Shinjuku-Ku
Country
Japan
Facility Name
Mg0004 20032
City
Suita
Country
Japan
Facility Name
Mg0004 40155
City
Gdańsk
Country
Poland
Facility Name
Mg0004 40151
City
Lublin
Country
Poland
Facility Name
Mg0004 40154
City
Łódź
Country
Poland
Facility Name
Mg0004 20027
City
Moscow
Country
Russian Federation
Facility Name
Mg0004 20001
City
Saint Petersburg
Country
Russian Federation
Facility Name
Mg0004 20028
City
Saint Petersburg
Country
Russian Federation
Facility Name
Mg0004 20055
City
Saint Petersburg
Country
Russian Federation
Facility Name
Mg0004 40159
City
Barcelona
Country
Spain
Facility Name
Mg0004 40157
City
Hospitalet de Llobregat
Country
Spain
Facility Name
Mg0004 20080
City
Taichung
Country
Taiwan
Facility Name
Mg0004 20081
City
Taipei
Country
Taiwan
12. IPD Sharing Statement
Plan to Share IPD
Yes
IPD Sharing Plan Description
Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe, or global development is discontinued, and 18 months after trial completion. Investigators may request access to anonymized individual patient-level data and redacted trial documents which may include: analysis-ready datasets, study protocol, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a prespecified time, typically 12 months, on a password protected portal. This plan may change if the risk of re-identifying trial participants is determined to be too high after the trial is completed; in this case and to protect participants, individual patient-level data would not be made available.
IPD Sharing Time Frame
Data from this study may be requested by qualified researchers six months after product approval in the US and/or Europe or global development is discontinued, and 18 months after trial completion.
IPD Sharing Access Criteria
Qualified researchers may request access to anonymized IPD and redacted study documents which may include: raw datasets, analysis-ready datasets, study protocol, blank case report form, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a pre-specified time, typically 12 months, on a password protected portal.
IPD Sharing URL
http://www.Vivli.org
Learn more about this trial
A Study to Investigate the Long-term Safety, Tolerability, and Efficacy of Rozanolixizumab in Adult Patients With Generalized Myasthenia Gravis
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