Mg0003 50081 |
Phoenix, Arizona, United States, 85013 |
Mg0003 50082 |
Scottsdale, Arizona, United States, 85251 |
Mg0003 50072 |
Los Angeles, California, United States, 90033 |
Mg0003 50092 |
Orange, California, United States, 92868 |
Mg0003 50097 |
San Francisco, California, United States, 94109 |
Mg0003 50099 |
San Francisco, California, United States, 94117 |
Mg0003 50101 |
Aurora, Colorado, United States, 80045 |
Mg0003 50078 |
New Britain, Connecticut, United States, 06053 |
Mg0003 50088 |
Washington, District of Columbia, United States, 20037 |
Mg0003 50122 |
Miami, Florida, United States, 33136 |
Mg0003 50120 |
Miami, Florida, United States, 33144 |
Mg0003 50073 |
Tampa, Florida, United States, 33612 |
Mg0003 50075 |
Augusta, Georgia, United States, 30912 |
Mg0003 50323 |
Honolulu, Hawaii, United States, 96817 |
Mg0003 50109 |
Chicago, Illinois, United States, 60637 |
Mg0003 50114 |
Indianapolis, Indiana, United States, 46202 |
Mg0003 50074 |
Fairway, Kansas, United States, 66205 |
Mg0003 50121 |
Lexington, Kentucky, United States, 40536 |
Mg0003 50110 |
Ann Arbor, Michigan, United States, 48109 |
Mg0003 50102 |
Detroit, Michigan, United States, 48202 |
Mg0003 50104 |
Rochester, Minnesota, United States, 55905 |
Mg0003 50105 |
Saint Louis, Missouri, United States, 63110 |
Mg0003 50077 |
New York, New York, United States, 10021 |
Mg0003 50117 |
Charlotte, North Carolina, United States, 28204 |
Mg0003 50086 |
Charlotte, North Carolina, United States, 28207 |
Mg0003 50090 |
Winston-Salem, North Carolina, United States, 27157 |
Mg0003 50087 |
Centerville, Ohio, United States, 45459 |
Mg0003 50076 |
Columbus, Ohio, United States, 43210 |
Mg0003 50055 |
Portland, Oregon, United States, 97239 |
Mg0003 50096 |
Philadelphia, Pennsylvania, United States, 19104 |
Mg0003 50089 |
Philadelphia, Pennsylvania, United States, 19140 |
Mg0003 50084 |
Charleston, South Carolina, United States, 29425 |
Mg0003 50113 |
Houston, Texas, United States, 77030 |
Mg0003 40121 |
Bruxelles, Belgium |
Mg0003 40123 |
Bruxelles, Belgium |
Mg0003 40122 |
Edegem, Belgium |
Mg0003 40120 |
Liège, Belgium |
Mg0003 50067 |
Calgary, Canada |
Mg0003 50071 |
Edmonton, Canada |
Mg0003 50065 |
London, Canada |
Mg0003 50066 |
Montréal, Canada |
Mg0003 50124 |
Montréal, Canada |
Mg0003 50070 |
Québec, Canada |
Mg0003 50069 |
Toronto, Canada |
Mg0003 40125 |
Ostrava, Czechia |
Mg0003 40124 |
Praha 2, Czechia |
Mg0003 40128 |
Aalborg, Denmark |
Mg0003 40127 |
Aarhus n, Denmark |
Mg0003 40126 |
Copenhagen, Denmark |
Mg0003 40489 |
Odense, Denmark |
Mg0003 40129 |
Bordeaux, France |
Mg0003 40070 |
Clermont-Ferrand, France |
Mg0003 40512 |
Garches, France |
Mg0003 40510 |
Le Kremlin-Bicêtre, France |
Mg0003 40360 |
Limoges, France |
Mg0003 40426 |
Lyon, France |
Mg0003 40130 |
Marseille, France |
Mg0003 40017 |
Nantes, France |
Mg0003 40132 |
Nice Cedex 1, France |
Mg0003 40133 |
Paris, France |
Mg0003 40131 |
Strasbourg, France |
Mg0003 20160 |
Tbilisi, Georgia |
Mg0003 20161 |
Tbilisi, Georgia |
Mg0003 20163 |
Tbilisi, Georgia |
Mg0003 20165 |
Tbilisi, Georgia |
Mg0003 40136 |
Duesseldorf, Germany |
Mg0003 40134 |
Essen, Germany |
Mg0003 40135 |
Gummersbach, Germany |
Mg0003 40140 |
Göttingen, Germany |
Mg0003 40141 |
Halle, Germany |
Mg0003 40024 |
Hannover, Germany |
Mg0003 40139 |
Jena, Germany |
Mg0003 40078 |
Leipzig, Germany |
Mg0003 40177 |
Münster, Germany |
Mg0003 40310 |
Wermsdorf, Germany |
Mg0003 40082 |
Kistarcsa, Hungary |
Mg0003 40178 |
Nyiregyhaza, Hungary |
Mg0003 40283 |
Bologna, Italy |
Mg0003 40144 |
Milano, Italy |
Mg0003 40307 |
Napoli, Italy |
Mg0003 40146 |
Pavia, Italy |
Mg0003 40148 |
Roma, Italy |
Mg0003 40150 |
Roma, Italy |
Mg0003 20035 |
Bunkyō-Ku, Japan |
Mg0003 20068 |
Chiba-Shi, Japan |
Mg0003 20078 |
Hanamaki-Shi, Japan |
Mg0003 20079 |
Hiroshima, Japan |
Mg0003 20075 |
Kobe, Japan |
Mg0003 20071 |
Nagasaki, Japan |
Mg0003 20067 |
Sapporo, Japan |
Mg0003 20077 |
Sendai, Japan |
Mg0003 20070 |
Shinjuku-Ku, Japan |
Mg0003 20076 |
Shinjuku-Ku, Japan |
Mg0003 20032 |
Suita, Japan |
Mg0003 20074 |
Ōsaka-sayama, Japan |
Mg0003 40155 |
Gdańsk, Poland |
Mg0003 40151 |
Lublin, Poland |
Mg0003 40153 |
Poznań, Poland |
Mg0003 40154 |
Łódź, Poland |
Mg0003 20168 |
Krasnoyarsk, Russian Federation |
Mg0003 20027 |
Moscow, Russian Federation |
Mg0003 20169 |
Novosibirsk, Russian Federation |
Mg0003 20001 |
Saint Petersburg, Russian Federation |
Mg0003 20028 |
Saint Petersburg, Russian Federation |
Mg0003 20029 |
Saint Petersburg, Russian Federation |
Mg0003 20055 |
Saint Petersburg, Russian Federation |
Mg0003 20197 |
Samara, Russian Federation |
Mg0003 40468 |
Belgrade, Serbia |
Mg0003 40467 |
Niš, Serbia |
Mg0003 40159 |
Barcelona, Spain |
Mg0003 40160 |
Barcelona, Spain |
Mg0003 40267 |
Barcelona, Spain |
Mg0003 40157 |
Hospitalet de Llobregat, Spain |
Mg0003 40161 |
Madrid, Spain |
Mg0003 40162 |
Madrid, Spain |
Mg0003 40350 |
Murcia, Spain |
Mg0003 40341 |
Málaga, Spain |
Mg0003 40308 |
May 29, 2019
|
June 3, 2019
|
June 4, 2021
|
June 3, 2019
|
October 2021 (Final data collection date for primary outcome measure)
|
Change from Baseline to Day 43 (Visit 10) in Myasthenia Gravis-Activities of Daily Living (MG-ADL) score [ Time Frame: Baseline and Visit 10 (Day 43) ] The total MG-ADL score is obtained by summing the responses to each individual item (8 items; Grades: 0, 1, 2, 3). The score ranges from 0 to 24, with a higher score indicating more disability.
|
Change from Baseline to Visit 10 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) score [ Time Frame: Baseline and Visit 10 (Day 43) ] The total MG-ADL score is obtained by summing the responses to each individual item (8 items; Grades: 0, 1, 2, 3). The score ranges from 0 to 24, with a higher score indicating more disability.
|
|
- Percentage of participants achieving Myasthenia Gravis-Activities of Daily Living (MG-ADL) response at Visit 10 [ Time Frame: Visit 10 (Day 43) ]
The total MG-ADL score is obtained by summing the responses to each individual item (8 items; Grades: 0, 1, 2, 3). The score ranges from 0 to 24, with a higher score indicating more disability.
- Change from Baseline to Day 43 (Visit 10) in Myasthenia Gravis-Composite (MG-C) score [ Time Frame: Baseline and Visit 10 (Day 43) ]
The total Myasthenia Gravis-Composite (MG-C) score is obtained by summing the responses to each individual item (10 items; Grade:0-9 depending on item). The score ranges from 0 to 50, with lower scores indicating lower disease activity.
- Change from Baseline to Day 43 (Visit 10) in Quantitative Myasthenia Gravis (QMG) score [ Time Frame: Baseline and Visit 10 (Day 43) ]
The total QMG score is obtained by summing the responses to each individual item (13 items; Responses: None=0, Mild=1, Moderate=2, Severe=3). The score ranges from 0 to 39, with lower scores indicating lower disease activity.
- Change from Baseline to Day 43 (Visit 10) in the Myasthenia Gravis (MG) Symptoms Patient Reported Outcome (PRO) 'Muscle Weakness Fatigability' score [ Time Frame: Baseline and Visit 10 (Day 43) ]
The MG symptoms PRO instrument consists of 42 items across 5 scales: ocular symptoms (items 1-5); bulbar symptoms (items 6-15); respiratory symptoms (items 16-18); physical fatigue (items 19-33) and muscle weakness fatigability (items 34-42).
The study participant will be asked to choose the response option that best describes the severity of ocular, bulbar, and respiratory symptoms over the past 7 days using a 4-point Likert scale ("none" to "severe") and how frequently they experience physical fatigue and muscle weakness fatigability over the past 7 days using a 5-point Likert scale ("none of the time" to "all of the time"), respectively.
- Change from Baseline to Day 43 (Visit 10) in the Myasthenia Gravis (MG) Symptoms Patient Reported Outcome (PRO) 'Physical Fatigue' score [ Time Frame: Baseline and Visit 10 (Day 43) ]
The MG symptoms PRO instrument consists of 42 items across 5 scales: ocular symptoms (items 1-5); bulbar symptoms (items 6-15); respiratory symptoms (items 16-18); physical fatigue (items 19-33) and muscle weakness fatigability (items 34-42).
The study participant will be asked to choose the response option that best describes the severity of ocular, bulbar, and respiratory symptoms over the past 7 days using a 4-point Likert scale ("none" to "severe") and how frequently they experience physical fatigue and muscle weakness fatigability over the past 7 days using a 5-point Likert scale ("none of the time" to "all of the time"), respectively.
- Change from Baseline to Day 43 (Visit 10) in the Myasthenia Gravis (MG) Symptoms Patient Reported Outcome (PRO) 'Bulbar Symptoms' score [ Time Frame: Baseline and Visit 10 (Day 43) ]
The MG symptoms PRO instrument consists of 42 items across 5 scales: ocular symptoms (items 1-5); bulbar symptoms (items 6-15); respiratory symptoms (items 16-18); physical fatigue (items 19-33) and muscle weakness fatigability (items 34-42).
The study participant will be asked to choose the response option that best describes the severity of ocular, bulbar, and respiratory symptoms over the past 7 days using a 4-point Likert scale ("none" to "severe") and how frequently they experience physical fatigue and muscle weakness fatigability over the past 7 days using a 5-point Likert scale ("none of the time" to "all of the time"), respectively.
- Occurrence of treatment-emergent adverse events (TEAEs) [ Time Frame: From Baseline until End of Study Visit (up to Week 14) ]
An adverse event (AE) is any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product.
A treatment-emergent adverse event (TEAE) is defined as any event that was not present prior to the first administration of IMP or any unresolved event already present before the first administration of IMP that worsens in intensity following exposure to treatment.
- Treatment-emergent adverse events (TEAEs) leading to withdrawal of investigational medicinal product (IMP) [ Time Frame: From Baseline until End of Study Visit (up to Week 14) ]
One of the secondary outcome measures is to assess safety and tolerability of the IMP in the MG patients. This can be measured by treatment-emergent adverse events (TEAEs) leading to withdrawal of IMP.
A TEAE is defined as any event that was not present prior to the first administration of IMP or any unresolved event already present before the first administration of IMP that worsens in intensity following exposure to treatment.
|
- Percentage of participants achieving Myasthenia Gravis-Activities of Daily Living (MG-ADL) response at Visit 10 [ Time Frame: Visit 10 (Day 43) ]
- Change from Baseline to Visit 10 in Myasthenia Gravis-Composite score [ Time Frame: Baseline and Visit 10 (Day 43) ]
The total Myasthenia Gravis(MG)-composite score is obtained by summing the responses to each individual item (10 items; Grade:0-9 depending on item). The score ranges from 0 to 50, with lower scores indicating lower disease activity.
- Change from Baseline to Visit 10 in Quantitative Myasthenia Gravis (QMG) score [ Time Frame: Baseline and Visit 10 (Day 43) ]
The total QMG score is obtained by summing the responses to each individual item (13 items; Responses: None=0, Mild=1, Moderate=2, Severe=3). The score ranges from 0 to 39, with lower scores indicating lower disease activity.
- Change from Baseline to Visit 10 in the Myasthenia Gravis (MG) Symptoms Patient Reported Outcome (PRO) 'Fatigability' score [ Time Frame: Baseline and Visit 10 (Day 43) ]
The MG symptoms PRO instrument consists of 42 items across 5 scales: ocular symptoms (items 1-5); bulbar symptoms (items 6-15); respiratory symptoms (items 16-18); physical fatigue (items 19-33) and muscle weakness fatigability (items 34-42).
The study participant will be asked to choose the response option that best describes the severity of ocular, bulbar, and respiratory symptoms over the past 7 days using a 4-point Likert scale ("none" to "severe") and how frequently they experience physical fatigue and muscle weakness fatigability over the past 7 days using a 5-point Likert scale ("none of the time" to "all of the time"), respectively.
- Change from Baseline to Visit 10 in the Myasthenia Gravis (MG) Symptoms Patient Reported Outcome (PRO) 'Physical Fatigue, Limb and Axial Weakness' score [ Time Frame: Baseline and Visit 10 (Day 43) ]
The MG symptoms PRO instrument consists of 42 items across 5 scales: ocular symptoms (items 1-5); bulbar symptoms (items 6-15); respiratory symptoms (items 16-18); physical fatigue (items 19-33) and muscle weakness fatigability (items 34-42).
The study participant will be asked to choose the response option that best describes the severity of ocular, bulbar, and respiratory symptoms over the past 7 days using a 4-point Likert scale ("none" to "severe") and how frequently they experience physical fatigue and muscle weakness fatigability over the past 7 days using a 5-point Likert scale ("none of the time" to "all of the time"), respectively.
- Change from Baseline to Visit 10 in the Myasthenia Gravis (MG) Symptoms Patient Reported Outcome (PRO) 'Bulbar' score [ Time Frame: Baseline and Visit 10 (Day 43) ]
The MG symptoms PRO instrument consists of 42 items across 5 scales: ocular symptoms (items 1-5); bulbar symptoms (items 6-15); respiratory symptoms (items 16-18); physical fatigue (items 19-33) and muscle weakness fatigability (items 34-42).
The study participant will be asked to choose the response option that best describes the severity of ocular, bulbar, and respiratory symptoms over the past 7 days using a 4-point Likert scale ("none" to "severe") and how frequently they experience physical fatigue and muscle weakness fatigability over the past 7 days using a 5-point Likert scale ("none of the time" to "all of the time"), respectively.
- Occurrence of treatment-emergent adverse events (TEAEs) [ Time Frame: From Baseline until End of Study Visit (up to Week 14) ]
An adverse event (AE) is any untoward medical occurrence in a patient or clinical investigation subject administered a pharmaceutical product, which does not necessarily have a causal relationship with this treatment. An AE could therefore be any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product.
A treatment-emergent adverse event (TEAE) is defined as any event that was not present prior to the first administration of IMP or any unresolved event already present before the first administration of IMP that worsens in intensity following exposure to treatment.
- Treatment-emergent adverse events (TEAEs) leading to withdrawal of investigational medicinal product (IMP) [ Time Frame: From Baseline until End of Study Visit (up to Week 14) ]
One of the secondary outcome measures is to assess safety and tolerability of the IMP in the MG patients. This can be measured by treatment-emergent adverse events (TEAEs) leading to withdrawal of IMP.
A TEAE is defined as any event that was not present prior to the first administration of IMP or any unresolved event already present before the first administration of IMP that worsens in intensity following exposure to treatment.
|
Not Provided
|
Not Provided
|
|
A Study to Test Efficacy and Safety of Rozanolixizumab in Adult Patients With Generalized Myasthenia Gravis
|
A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study Evaluating Efficacy and Safety of Rozanolixizumab in Adult Patients With Generalized Myasthenia Gravis
|
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).
|
Not Provided
|
Interventional
|
Phase 3
|
Allocation: Randomized Intervention Model: Parallel Assignment Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) Primary Purpose: Treatment
|
Generalized Myasthenia Gravis
|
|
- Experimental: Dosage Regimen 1
Study participants randomized to dosage regimen 1 will receive assigned dosage of rozanolixizumab at pre-specified time points during Treatment Period.
Intervention: Drug: Rozanolixizumab
- Experimental: Dosage Regimen 2
Study participants randomized to dosage regimen 2 will receive assigned dosage of rozanolixizumab at pre-specified time points during Treatment Period.
Intervention: Drug: Rozanolixizumab
- Placebo Comparator: Placebo
Study participants randomized to this arm will receive placebo.
Intervention: Other: Placebo
|
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.
|
|
Recruiting
|
240
|
Same as current
|
December 2021
|
October 2021 (Final data collection date for primary outcome measure)
|
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
|
Sexes Eligible for Study: |
All |
|
18 Years and older (Adult, Older Adult)
|
No
|
Contact: UCB Cares |
+1844599 ext 2273 |
UCBCares@ucb.com |
|
|
Belgium, Canada, Czechia, Denmark, France, Georgia, Germany, Hungary, Italy, Japan, Poland, Russian Federation, Serbia, Spain, Taiwan, United Kingdom, United States
|
|
|
NCT03971422
|
MG0003 2019-000968-18 ( EudraCT Number )
|
Yes
|
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 |
|
Plan to Share IPD: |
Yes |
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. |
Supporting Materials: |
Study Protocol |
Supporting Materials: |
Statistical Analysis Plan (SAP) |
Supporting Materials: |
Clinical Study Report (CSR) |
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. |
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. |
URL: |
https://www.Vivli.org |
|
UCB Pharma ( UCB Biopharma SRL )
|
UCB Biopharma SRL
|
Not Provided
|
Study Director: |
UCB Cares |
+1 844 599 2273 (UCB) |
|
UCB Pharma
|
June 2021
|