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| ID | Type | Description | Link |
|---|---|---|---|
| 2019-000968-18 | EudraCT Number |
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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).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Dosage Regimen 1 | Experimental | Study participants randomized to dosage regimen 1 will receive assigned dosage of rozanolixizumab at pre-specified time points during Treatment Period. |
|
| Dosage Regimen 2 | Experimental | Study participants randomized to dosage regimen 2 will receive assigned dosage of rozanolixizumab at pre-specified time points during Treatment Period. |
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| Placebo | Placebo Comparator | Study participants randomized to this arm will receive placebo. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Rozanolixizumab | Drug | Rozanolixizumab will be administered by subcutaneous infusion in dosage regimen 1 or 2. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline to Day 43 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Score | 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 in the score indicates worsening and a negative change indicates improvement. | Baseline and Day 43 |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants Achieving Myasthenia Gravis-Activities of Daily Living (MG-ADL) Response at Day 43 | The MG-ADL is an 8-item PRO instrument developed on the basis of the 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 in the score indicates worsening and a negative change indicates improvement. Study participants were classified as responders at Day 43 if the value was at least a 2-point improvement (decrease) from Baseline at Day 43. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| UCB Cares | +1 844 599 2273 (UCB) | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Mg0003 50081 | Phoenix | Arizona | 85013 | United States | ||
| Mg0003 50082 |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 37059507 | Result | Bril V, Druzdz A, Grosskreutz J, Habib AA, Mantegazza R, Sacconi S, Utsugisawa K, Vissing J, Vu T, Boehnlein M, Bozorg A, Gayfieva M, Greve B, Woltering F, Kaminski HJ; MG0003 study team. Safety and efficacy of rozanolixizumab in patients with generalised myasthenia gravis (MycarinG): a randomised, double-blind, placebo-controlled, adaptive phase 3 study. Lancet Neurol. 2023 May;22(5):383-394. doi: 10.1016/S1474-4422(23)00077-7. | |
| 39297052 |
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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.
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.
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.
Participant Flow refers to the Randomized Set. Two participants randomized to RLZ ~7mg/kg, were administered RLZ ~10mg/kg at baseline visit. So, these two participants were included in RLZ ~7 mg/kg group in randomized set, but in RLZ ~10 mg/kg group in safety set.
The study started to enroll study participants in Jun 2019 and concluded in Oct 2021.
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| ID | Title | Description |
|---|---|---|
| FG000 | Placebo | Participants received placebo subcutaneously on a weekly basis over a 6-week Treatment Period. After 6-week Treatment Period, participants were followed for up to 8 weeks (up to Week 14). |
| FG001 | Rozanolixizumab ~7 mg/kg |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Feb 23, 2021 | Aug 22, 2023 |
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| Placebo | Other | Subjects will receive placebo at pre-specified time points. |
|
|
| Day 43 |
| Change From Baseline to Day 43 in Myasthenia Gravis-Composite (MG-C) Total Score | MG-C scale is a validated assessment and scale tests 10 items with individual item 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. | Baseline and Day 43 |
| Change From Baseline to Day 43 in Quantitative Myasthenia Gravis (QMG) Total Score | 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 in the score indicates worsening and a negative change indicates improvement. | Baseline and Day 43 |
| Change From Baseline to Day 43 in the Myasthenia Gravis (MG) Symptoms Patient Reported Outcome (PRO) 'Muscle Weakness Fatigability' Score | MG symptoms PRO instrument consisted of 42 items across 5 scales: ocular muscle weakness (items 1-5); bulbar muscle weakness (items 6-15); respiratory muscle weakness (items 16-18); physical fatigue (items 19-33) and muscle weakness fatigability (items 34-42). Study participants were asked to choose response option that how frequently they experienced muscle weakness fatigability (items 34-42) over the past 7 days using a 5-point Likert scale (1="none of the time" to 5="all of the time") for each item. Sum of each item score is linearly transformed to have all domain scores ranging from 0 to 100. Total score is calculated as: (sum of item scores within the scale)/(raw score range) x (total number of items in the scale)/(number of non-missing items in the scale) x100 and ranged from 0 to 100, where higher scores indicated severe symptoms. | Baseline and Day 43 |
| Change From Baseline to Day 43 in the Myasthenia Gravis (MG) Symptoms Patient Reported Outcome (PRO) 'Physical Fatigue' Score | The MG symptoms PRO instrument consisted of 42 items across 5 scales: ocular muscle weakness (items 1-5); bulbar muscle weakness (items 6-15); respiratory muscle weakness (items 16-18); physical fatigue (items 19-33) and muscle weakness fatigability (items 34-42). Study participants were asked to choose the response option that how frequently they experienced physical fatigue (items 19-33) over the past 7 days using a 5-point Likert scale (1="none of the time" to 5="all of the time") for each item. Sum of each item score is linearly transformed to have all domain scores ranging from 0 to 100. Total score is calculated as: (sum of item scores within the scale)/(raw score range) x (total number of items in the scale)/(number of non-missing items in the scale) x100 and ranged from 0 to 100, where higher scores indicated severe symptoms. | Baseline and Day 43 |
| Change From Baseline to Day 43 in the Myasthenia Gravis (MG) Symptoms Patient Reported Outcome (PRO) 'Bulbar Symptoms' Score | The MG symptoms PRO instrument consisted of 42 items across 5 scales: ocular muscle weakness (items 1-5); bulbar muscle weakness (items 6-15); respiratory muscle weakness (items 16-18); physical fatigue (items 19-33) and muscle weakness fatigability (items 34-42). Study participants were asked to choose response option that best described severity of bulbar muscle weakness (items 6-15) symptoms over past 7 days using a 4-point Likert scale (1="none" to 4="severe") for each item. Sum of each item score is linearly transformed to have all domain scores ranging from 0 to 100. Total score is calculated as: (sum of item scores within the scale)/(raw score range) x (total number of items in the scale)/(number of non-missing items in the scale) x100 and ranged from 0 to 100, where higher scores indicated severe symptoms. | Baseline and Day 43 |
| Number 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) up to and including 8 weeks after the last dose. | From Baseline until End of Study Visit (up to Week 14) |
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) Leading to Withdrawal of Investigational Medicinal Product (IMP) | A TEAE is defined as an AE starting on or after the time of first administration of IMP up to and including 8 weeks after the last dose. | From Baseline until End of Study Visit (up to Week 14) |
| Scottsdale |
| Arizona |
| 85251 |
| United States |
| Mg0003 50072 | Los Angeles | California | 90033 | United States |
| Mg0003 50092 | Orange | California | 92868 | United States |
| Mg0003 50097 | San Francisco | California | 94109 | United States |
| Mg0003 50099 | San Francisco | California | 94117 | United States |
| Mg0003 50101 | Aurora | Colorado | 80045 | United States |
| Mg0003 50088 | Washington D.C. | District of Columbia | 20037 | United States |
| Mg0003 50122 | Miami | Florida | 33136 | United States |
| Mg0003 50120 | Miami | Florida | 33144 | United States |
| Mg0003 50073 | Tampa | Florida | 33612 | United States |
| Mg0003 50075 | Augusta | Georgia | 30912 | United States |
| Mg0003 50323 | Honolulu | Hawaii | 96817 | United States |
| Mg0003 50109 | Chicago | Illinois | 60637 | United States |
| Mg0003 50114 | Indianapolis | Indiana | 46202 | United States |
| Mg0003 50074 | Fairway | Kansas | 66205 | United States |
| Mg0003 50121 | Lexington | Kentucky | 40536 | United States |
| Mg0003 50110 | Ann Arbor | Michigan | 48109 | United States |
| Mg0003 50102 | Detroit | Michigan | 48202 | United States |
| Mg0003 50104 | Rochester | Minnesota | 55905 | United States |
| Mg0003 50105 | St Louis | Missouri | 63110 | United States |
| Mg0003 50077 | New York | New York | 10021 | United States |
| Mg0003 50117 | Charlotte | North Carolina | 28204 | United States |
| Mg0003 50086 | Charlotte | North Carolina | 28207 | United States |
| Mg0003 50090 | Winston-Salem | North Carolina | 27157 | United States |
| Mg0003 50076 | Columbus | Ohio | 43210 | United States |
| Mg0003 50096 | Philadelphia | Pennsylvania | 19104 | United States |
| Mg0003 50089 | Philadelphia | Pennsylvania | 19140 | United States |
| Mg0003 50084 | Charleston | South Carolina | 29425 | United States |
| Mg0003 50113 | Houston | Texas | 77030 | United States |
| Mg0003 40121 | Brussels | Belgium |
| Mg0003 50067 | Calgary | Canada |
| Mg0003 50066 | Montreal | Canada |
| Mg0003 50070 | Québec | Canada |
| Mg0003 50069 | Toronto | Canada |
| Mg0003 40125 | Ostrava | Czechia |
| Mg0003 40124 | Prague | 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 | 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 40134 | Essen | Germany |
| Mg0003 40140 | Göttingen | Germany |
| Mg0003 40135 | Gummersbach | Germany |
| Mg0003 40139 | Jena | Germany |
| Mg0003 40078 | Leipzig | Germany |
| Mg0003 40177 | Münster | Germany |
| Mg0003 40082 | Kistarcsa | Hungary |
| Mg0003 40178 | Nyíregyháza | Hungary |
| Mg0003 40283 | Bologna | Italy |
| Mg0003 40149 | Lazio | Italy |
| Mg0003 40144 | Milan | Italy |
| Mg0003 40307 | Naples | Italy |
| Mg0003 40146 | Pavia | Italy |
| Mg0003 40148 | Roma | Italy |
| Mg0003 40150 | Roma | Italy |
| Mg0003 20035 | Bunkyō City | Japan |
| Mg0003 20068 | Chiba | Japan |
| Mg0003 20078 | Hanamaki-Shi | Japan |
| Mg0003 20079 | Hiroshima | Japan |
| Mg0003 20075 | Kobe | Japan |
| Mg0003 20071 | Nagasaki | Japan |
| Mg0003 20074 | Ōsaka-sayama | Japan |
| Mg0003 20067 | Sapporo | Japan |
| Mg0003 20077 | Sendai | Japan |
| Mg0003 20070 | Shinjuku-Ku | Japan |
| Mg0003 20076 | Shinjuku-Ku | Japan |
| Mg0003 20032 | Suita | Japan |
| Mg0003 40155 | Gdansk | Poland |
| Mg0003 40154 | Lodz | Poland |
| Mg0003 40151 | Lublin | Poland |
| Mg0003 40153 | Poznan | Poland |
| Mg0003 20168 | Krasnoyarsk | Russia |
| Mg0003 20027 | Moscow | Russia |
| Mg0003 20169 | Novosibirsk | Russia |
| Mg0003 20001 | Saint Petersburg | Russia |
| Mg0003 20028 | Saint Petersburg | Russia |
| Mg0003 20029 | Saint Petersburg | Russia |
| Mg0003 20055 | Saint Petersburg | Russia |
| Mg0003 20197 | Samara | Russia |
| Mg0003 40468 | Belgrade | Serbia |
| Mg0003 40467 | Niš | Serbia |
| Mg0003 40159 | Barcelona | Spain |
| Mg0003 40160 | Barcelona | Spain |
| Mg0003 40267 | Barcelona | Spain |
| Mg0003 40157 | L'Hospitalet de Llobregat | Spain |
| Mg0003 40161 | Madrid | Spain |
| Mg0003 40162 | Madrid | Spain |
| Mg0003 40341 | Málaga | Spain |
| Mg0003 40350 | Murcia | Spain |
| Mg0003 40308 | San Sebastián de los Reyes | Spain |
| Mg0003 20080 | Taichung | Taiwan |
| Mg0003 20081 | Taipei | Taiwan |
| Mg0003 20086 | Taipei | Taiwan |
| Mg0003 20082 | Taoyuan | Taiwan |
| Mg0003 40175 | London | United Kingdom |
| Mg0003 40168 | Nottingham | United Kingdom |
| Result |
| Habib AA, Sacconi S, Antonini G, Cortes-Vicente E, Grosskreutz J, Mahuwala ZK, Mantegazza R, Pascuzzi RM, Utsugisawa K, Vissing J, Vu T, Wiendl H, Boehnlein M, Greve B, Woltering F, Bril V. Efficacy and safety of rozanolixizumab in patients with muscle-specific tyrosine kinase autoantibody-positive generalised myasthenia gravis: a subgroup analysis of the randomised, double-blind, placebo-controlled, adaptive phase III MycarinG study. Ther Adv Neurol Disord. 2024 Sep 12;17:17562864241273036. doi: 10.1177/17562864241273036. eCollection 2024. |
| 40755069 | Result | Kaminski HJ, Antozzi C, Habib AA, Pascuzzi RM, Sacconi S, Utsugisawa K, Vissing J, Regnault A, Hareendran A, Grimson F, Tarancon T, Bril V; MycarinG study investigators. Improvement in Patient-Reported Symptoms of Generalised Myasthenia Gravis With Rozanolixizumab in the Randomised Phase 3 MycarinG Study Using the MG Symptoms PRO. Eur J Neurol. 2025 Aug;32(8):e70231. doi: 10.1111/ene.70231. |
| 41205003 | Result | Barnett-Tapia C, Cortes Vicente E, Pascuzzi RM, Utsugisawa K, Bloemers J, Grimson F, Tarancon T, Bril V; MycarinG study investigators. Measuring the effect of rozanolixizumab using the Myasthenia Gravis Impairment Index: analyses from the randomized phase 3 MycarinG study. J Neurol. 2025 Nov 8;272(12):752. doi: 10.1007/s00415-025-13480-8. |
| 42388397 | Derived | Bril V, Druzdz A, Grosskreutz J, Habib AA, Mantegazza R, Sacconi S, Utsugisawa K, Vu T, Boehnlein M, Grimson F, Houston N, Kerbusch V, Pulido-Valdeolivas I, Tarancon T, Vissing J. Long-term use of rozanolixizumab in generalised myasthenia gravis: final pooled analysis of the phase III MycarinG study and two open-label extensions. Ther Adv Neurol Disord. 2026 Jun 29;19:17562864261458532. doi: 10.1177/17562864261458532. eCollection 2026. |
| 38978809 | Derived | Regnault A, Habib AA, Creel K, Kaminski HJ, Morel T. Clinical meaningfulness and psychometric robustness of the MG Symptoms PRO scales in clinical trials in adults with myasthenia gravis. Front Neurol. 2024 Jun 24;15:1368525. doi: 10.3389/fneur.2024.1368525. eCollection 2024. |
| 38099334 | Derived | Matic A, Alfaidi N, Bril V. An evaluation of rozanolixizumab-noli for the treatment of anti-AChR and anti-MuSK antibody-positive generalized myasthenia gravis. Expert Opin Biol Ther. 2023 Jul-Dec;23(12):1163-1171. doi: 10.1080/14712598.2023.2296126. Epub 2023 Dec 28. |
| 33219142 | Derived | 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. |
Participants received rozanolixizumab (RLZ) equivalent to approximately 7 milligrams/kilogram (mg/kg), subcutaneously on a weekly basis over a 6-week Treatment Period. After 6-week Treatment Period, participants were followed for up to 8 weeks (up to Week 14).
| FG002 | Rozanolixizumab ~10 mg/kg | Participants received rozanolixizumab equivalent to approximately 10 mg/kg, subcutaneously on a weekly basis over a 6-week Treatment Period. After 6-week Treatment Period, participants were followed for up to 8 weeks (up to Week 14). |
| COMPLETED |
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| NOT COMPLETED |
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Baseline Characteristics refers to the Randomized Set (RS) which consisted of all study participants who were randomized and analyzed according to the treatment assigned instead of the actual treatment received.
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Placebo | Participants received placebo subcutaneously on a weekly basis over a 6-week Treatment Period. After 6-week Treatment Period, participants were followed for up to 8 weeks (up to Week 14). |
| BG001 | Rozanolixizumab ~7 mg/kg | Participants received rozanolixizumab equivalent to approximately 7 mg/kg, subcutaneously on a weekly basis over a 6-week Treatment Period. After 6-week Treatment Period, participants were followed for up to 8 weeks (up to Week 14). |
| BG002 | Rozanolixizumab ~10 mg/kg | Participants received rozanolixizumab equivalent to approximately 10 mg/kg, subcutaneously on a weekly basis over a 6-week Treatment Period. After 6-week Treatment Period, participants were followed for up to 8 weeks (up to Week 14). |
| BG003 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
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| Age, Categorical | Count of Participants | Participants |
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| Age, Continuous | Mean | Standard Deviation | years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Race/Ethnicity, Customized | Count of Participants | Participants |
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| Race/Ethnicity, Customized | Count of Participants | Participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| Primary | Change From Baseline to Day 43 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Score | 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 in the score indicates worsening and a negative change indicates improvement. | The Randomized Set consisted of all study participants who were randomized and analyzed according to the treatment assigned instead of the actual treatment received. | Posted | Least Squares Mean | Standard Error | units on a scale | Baseline and Day 43 |
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| Secondary | Percentage of Participants Achieving Myasthenia Gravis-Activities of Daily Living (MG-ADL) Response at Day 43 | The MG-ADL is an 8-item PRO instrument developed on the basis of the 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 in the score indicates worsening and a negative change indicates improvement. Study participants were classified as responders at Day 43 if the value was at least a 2-point improvement (decrease) from Baseline at Day 43. | The Randomized Set consisted of all study participants who were randomized and analyzed according to the treatment assigned instead of the actual treatment received. | Posted | Number | percentage of participants | Day 43 |
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| Secondary | Change From Baseline to Day 43 in Myasthenia Gravis-Composite (MG-C) Total Score | MG-C scale is a validated assessment and scale tests 10 items with individual item 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. | The Randomized Set consisted of all study participants who were randomized and analyzed according to the treatment assigned instead of the actual treatment received. | Posted | Least Squares Mean | Standard Error | units on a scale | Baseline and Day 43 |
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| Secondary | Change From Baseline to Day 43 in Quantitative Myasthenia Gravis (QMG) Total Score | 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 in the score indicates worsening and a negative change indicates improvement. | The Randomized Set consisted of all study participants who were randomized and analyzed according to the treatment assigned instead of the actual treatment received. | Posted | Least Squares Mean | Standard Error | units on a scale | Baseline and Day 43 |
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| Secondary | Change From Baseline to Day 43 in the Myasthenia Gravis (MG) Symptoms Patient Reported Outcome (PRO) 'Muscle Weakness Fatigability' Score | MG symptoms PRO instrument consisted of 42 items across 5 scales: ocular muscle weakness (items 1-5); bulbar muscle weakness (items 6-15); respiratory muscle weakness (items 16-18); physical fatigue (items 19-33) and muscle weakness fatigability (items 34-42). Study participants were asked to choose response option that how frequently they experienced muscle weakness fatigability (items 34-42) over the past 7 days using a 5-point Likert scale (1="none of the time" to 5="all of the time") for each item. Sum of each item score is linearly transformed to have all domain scores ranging from 0 to 100. Total score is calculated as: (sum of item scores within the scale)/(raw score range) x (total number of items in the scale)/(number of non-missing items in the scale) x100 and ranged from 0 to 100, where higher scores indicated severe symptoms. | The Randomized Set consisted of all study participants who were randomized and analyzed according to the treatment assigned instead of the actual treatment received. | Posted | Least Squares Mean | Standard Error | units on a scale | Baseline and Day 43 |
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| Secondary | Change From Baseline to Day 43 in the Myasthenia Gravis (MG) Symptoms Patient Reported Outcome (PRO) 'Physical Fatigue' Score | The MG symptoms PRO instrument consisted of 42 items across 5 scales: ocular muscle weakness (items 1-5); bulbar muscle weakness (items 6-15); respiratory muscle weakness (items 16-18); physical fatigue (items 19-33) and muscle weakness fatigability (items 34-42). Study participants were asked to choose the response option that how frequently they experienced physical fatigue (items 19-33) over the past 7 days using a 5-point Likert scale (1="none of the time" to 5="all of the time") for each item. Sum of each item score is linearly transformed to have all domain scores ranging from 0 to 100. Total score is calculated as: (sum of item scores within the scale)/(raw score range) x (total number of items in the scale)/(number of non-missing items in the scale) x100 and ranged from 0 to 100, where higher scores indicated severe symptoms. | The Randomized Set consisted of all study participants who were randomized and analyzed according to the treatment assigned instead of the actual treatment received. | Posted | Least Squares Mean | Standard Error | units on a scale | Baseline and Day 43 |
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| Secondary | Change From Baseline to Day 43 in the Myasthenia Gravis (MG) Symptoms Patient Reported Outcome (PRO) 'Bulbar Symptoms' Score | The MG symptoms PRO instrument consisted of 42 items across 5 scales: ocular muscle weakness (items 1-5); bulbar muscle weakness (items 6-15); respiratory muscle weakness (items 16-18); physical fatigue (items 19-33) and muscle weakness fatigability (items 34-42). Study participants were asked to choose response option that best described severity of bulbar muscle weakness (items 6-15) symptoms over past 7 days using a 4-point Likert scale (1="none" to 4="severe") for each item. Sum of each item score is linearly transformed to have all domain scores ranging from 0 to 100. Total score is calculated as: (sum of item scores within the scale)/(raw score range) x (total number of items in the scale)/(number of non-missing items in the scale) x100 and ranged from 0 to 100, where higher scores indicated severe symptoms. | The Randomized Set consisted of all study participants who were randomized and analyzed according to the treatment assigned instead of the actual treatment received. | Posted | Least Squares Mean | Standard Error | units on a scale | Baseline and Day 43 |
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| Secondary | Number 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) up to and including 8 weeks after the last dose. | The Safety Set consisted of all randomized study participants who received at least one dose of IMP and were analyzed according to the actual treatment the participants received. Two participants randomized to RLZ ~7mg/kg, were administered RLZ ~10mg/kg at baseline visit. So, these two participants were included in RLZ ~7 mg/kg group in randomized set, but in RLZ ~10 mg/kg group in safety set. | Posted | Count of Participants | Participants | From Baseline until End of Study Visit (up to Week 14) |
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| Secondary | Number of Participants With Treatment-emergent Adverse Events (TEAEs) Leading to Withdrawal of Investigational Medicinal Product (IMP) | A TEAE is defined as an AE starting on or after the time of first administration of IMP up to and including 8 weeks after the last dose. | The Safety Set consisted of all randomized study participants who received at least one dose of IMP and were analyzed according to the actual treatment the participants received. Two participants randomized to RLZ ~7mg/kg, were administered RLZ ~10mg/kg at baseline visit. So, these two participants were included in RLZ ~7 mg/kg group in randomized set, but in RLZ ~10 mg/kg group in safety set. | Posted | Count of Participants | Participants | From Baseline until End of Study Visit (up to Week 14) |
|
From Baseline until End of Study Visit (up to Week 14)
Safety Set was analyzed for TEAEs. Two participants randomized to RLZ ~7mg/kg, were administered RLZ ~10mg/kg at baseline visit. So, these two participants were included in RLZ ~7 mg/kg group in randomized set, but in RLZ ~10 mg/kg group in safety set.
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Placebo | Participants received placebo subcutaneously on a weekly basis over a 6-week Treatment Period. After 6-week Treatment Period, participants were followed for up to 8 weeks (up to Week 14). | 0 | 67 | 6 | 67 | 26 | 67 |
| EG001 | Rozanolixizumab ~7 mg/kg | Participants received rozanolixizumab equivalent to approximately 7 mg/kg, subcutaneously on a weekly basis over a 6-week Treatment Period. After 6-week Treatment Period, participants were followed for up to 8 weeks (up to Week 14). | 0 | 64 | 5 | 64 | 40 | 64 |
| EG002 | Rozanolixizumab ~10 mg/kg | Participants received rozanolixizumab equivalent to approximately 10 mg/kg, subcutaneously on a weekly basis over a 6-week Treatment Period. After 6-week Treatment Period, participants were followed for up to 8 weeks (up to Week 14). | 0 | 69 | 7 | 69 | 42 | 69 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Gastritis | Gastrointestinal disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Chest pain | General disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| COVID-19 pneumonia | Infections and infestations | MedDRA v24.0 | Non-systematic Assessment |
| |
| Thoracic vertebral fracture | Injury, poisoning and procedural complications | MedDRA v24.0 | Non-systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Muscular weakness | Musculoskeletal and connective tissue disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Metastatic squamous cell carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA v24.0 | Non-systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Myasthenia gravis | Nervous system disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Myasthenia gravis crisis | Nervous system disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Seizure | Nervous system disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Device dislocation | Product Issues | MedDRA v24.0 | Non-systematic Assessment |
| |
| Nephrolithiasis | Renal and urinary disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Cervical dysplasia | Reproductive system and breast disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Acute respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA v24.0 | Non-systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Diarrhoea | Gastrointestinal disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA v24.0 | Non-systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA v24.0 | Non-systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA v24.0 | Non-systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA v24.0 | Non-systematic Assessment |
|
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| UCB | Cares | 001 844 599 2273 | UCBCares@ucb.com |
| Prot_001.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Nov 10, 2021 | Aug 22, 2023 | SAP_002.pdf |
| ID | Term |
|---|---|
| D009157 | Myasthenia Gravis |
| ID | Term |
|---|---|
| D020361 | Paraneoplastic Syndromes, Nervous System |
| D009423 | Nervous System Neoplasms |
| D009371 | Neoplasms by Site |
| D009369 | Neoplasms |
| D010257 | Paraneoplastic Syndromes |
| D020274 | Autoimmune Diseases of the Nervous System |
| D009422 | Nervous System Diseases |
| D019636 | Neurodegenerative Diseases |
| D020511 | Neuromuscular Junction Diseases |
| D009468 | Neuromuscular Diseases |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C000627812 | rozanolixizumab |
Not provided
Not provided
Not provided
| Between 18 and 65 years |
|
| >=65 years |
|
| Male |
|
| Black |
|
| Native Hawaiian or other Pacific Islander |
|
| White |
|
| Missing |
|
| Not Hispanic or Latino |
|
| Missing |
|
| Superiority |
| The parallel gatekeeping testing procedure with a truncated Hochberg test was used to control the familywise type I error rate at a 2-sided alpha level of 0.05. | Lehmacher and Wassmer method | <0.001 | Confidence intervals and p-values are based on stage-wise inverse normal combination using the Lehmacher and Wassmer method. | LS Mean Difference | -2.619 | 2-Sided | 95 | -3.994 | -1.163 | MMRM analysis of covariance (ANCOVA) model included treatment group, Baseline score, region, stratification factor (MuSK+ or AChR+) and treatment group by day (interaction term) as fixed factors and participant as a random effect. | Superiority |
| OG002 | Rozanolixizumab ~10 mg/kg | Participants received rozanolixizumab equivalent to approximately 10 mg/kg, subcutaneously on a weekly basis over a 6-week Treatment Period. After 6-week Treatment Period, participants were followed for up to 8 weeks (up to Week 14). |
|
|
|
| Rozanolixizumab ~7 mg/kg |
Participants received rozanolixizumab equivalent to approximately 7 mg/kg, subcutaneously on a weekly basis over a 6-week Treatment Period. After 6-week Treatment Period, participants were followed for up to 8 weeks (up to Week 14). |
| OG002 | Rozanolixizumab ~10 mg/kg | Participants received rozanolixizumab equivalent to approximately 10 mg/kg, subcutaneously on a weekly basis over a 6-week Treatment Period. After 6-week Treatment Period, participants were followed for up to 8 weeks (up to Week 14). |
|
|
|
| Rozanolixizumab ~10 mg/kg |
Participants received rozanolixizumab equivalent to approximately 10 mg/kg, subcutaneously on a weekly basis over a 6-week Treatment Period. After 6-week Treatment Period, participants were followed for up to 8 weeks (up to Week 14). |
|
|
|
Participants received rozanolixizumab equivalent to approximately 7 mg/kg, subcutaneously on a weekly basis over a 6-week Treatment Period. After 6-week Treatment Period, participants were followed for up to 8 weeks (up to Week 14).
| OG002 | Rozanolixizumab ~10 mg/kg | Participants received rozanolixizumab equivalent to approximately 10 mg/kg, subcutaneously on a weekly basis over a 6-week Treatment Period. After 6-week Treatment Period, participants were followed for up to 8 weeks (up to Week 14). |
|
|
|
Participants received rozanolixizumab equivalent to approximately 7 mg/kg, subcutaneously on a weekly basis over a 6-week Treatment Period. After 6-week Treatment Period, participants were followed for up to 8 weeks (up to Week 14).
| OG002 | Rozanolixizumab ~10 mg/kg | Participants received rozanolixizumab equivalent to approximately 10 mg/kg, subcutaneously on a weekly basis over a 6-week Treatment Period. After 6-week Treatment Period, participants were followed for up to 8 weeks (up to Week 14). |
|
|
|
Participants received rozanolixizumab equivalent to approximately 7 mg/kg, subcutaneously on a weekly basis over a 6-week Treatment Period. After 6-week Treatment Period, participants were followed for up to 8 weeks (up to Week 14).
| OG002 | Rozanolixizumab ~10 mg/kg | Participants received rozanolixizumab equivalent to approximately 10 mg/kg, subcutaneously on a weekly basis over a 6-week Treatment Period. After 6-week Treatment Period, participants were followed for up to 8 weeks (up to Week 14). |
|
|
|
Participants received rozanolixizumab equivalent to approximately 10 mg/kg, subcutaneously on a weekly basis over a 6-week Treatment Period. After 6-week Treatment Period, participants were followed for up to 8 weeks (up to Week 14). |
|
|
Participants received rozanolixizumab equivalent to approximately 10 mg/kg, subcutaneously on a weekly basis over a 6-week Treatment Period. After 6-week Treatment Period, participants were followed for up to 8 weeks (up to Week 14). |
|
|