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The RAISE study is a multicenter, randomized, double-blind, placebo controlled study to confirm the efficacy, safety, and tolerability of zilucoplan in subjects with generalized Myasthenia Gravis. Subjects will be randomized in a 1:1 ratio to receive daily SC doses of 0.3 mg/kg zilucoplan or placebo for 12 weeks.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 0.3 mg/kg zilucoplan (RA101495) | Experimental |
| |
| Placebo | Placebo Comparator |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| zilucoplan (RA101495) | Drug | Daily subcutaneous (SC) injection |
| |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline (CFB) to Week 12 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score | The MG-ADL is an 8-item patient-reported outcome measure assessing MG symptoms and their effects on daily activities. Each item in the scale scored 0 to 3 (0=None, 3=severe disease) point scale. The total score was the sum of all individual item scores and ranged from 0 to 24. Higher scores indicated more severe disability due to MG. A decrease from Baseline score indicated improvement. | From Baseline to End of Treatment (Week 12) |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline to Week 12 in the Quantitative Myasthenia Gravis (QMG) Total Score | The QMG is a standardized and validated quantitative strength scoring system that was developed specifically for MG. The scale consisted of 13 items. Each item in the scale scored on a 0 to 3-point scale, ranging from 0 (no weakness) to 3 (severe weakness), summing up to the overall score range from 0 to 39. Higher scores indicated more severe impairment. A decrease from Baseline score indicated improvement. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| UCB Cares | 0018445992273 (UCB) | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Site 41: Diagnostic and Medical Clinic | Mobile | Alabama | 36604 | United States | ||
| Site 116: Neuromuscular Clinic and Research Center |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 37059508 | Result | Howard JF Jr, Bresch S, Genge A, Hewamadduma C, Hinton J, Hussain Y, Juntas-Morales R, Kaminski HJ, Maniaol A, Mantegazza R, Masuda M, Sivakumar K, Smilowski M, Utsugisawa K, Vu T, Weiss MD, Zajda M, Boroojerdi B, Brock M, de la Borderie G, Duda PW, Lowcock R, Vanderkelen M, Leite MI; RAISE Study Team. Safety and efficacy of zilucoplan in patients with generalised myasthenia gravis (RAISE): a randomised, double-blind, placebo-controlled, phase 3 study. Lancet Neurol. 2023 May;22(5):395-406. doi: 10.1016/S1474-4422(23)00080-7. | |
| 38400914 |
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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 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.
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.
The Participant flow refers to the Randomized Set.
The study started to enroll participants in September 2019 and concluded in December 2021.
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| ID | Title | Description |
|---|---|---|
| FG000 | Placebo | Participants self-administered zilucoplan (RA101495) matching placebo as subcutaneous (SC) injection during 12-week Treatment Period. |
| FG001 | Zilucoplan 0.3 mg/kg | Participants self-administered zilucoplan (RA101495) 0.3 milligrams/kilogram/day (mg/kg/day) SC injection during 12-week Treatment Period. |
| 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 | Dec 18, 2020 | Nov 22, 2022 |
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| Placebo |
| Drug |
Daily subcutaneous (SC) injection |
|
| From Baseline to End of Treatment (Week 12) |
| Change From Baseline to Week 12 in the Myasthenia Gravis Composite (MGC) Scale Total Score | The total MGC score was sum of responses to 10 individual items : 1. Ptosis upward gaze (0 to 3), 2. Double vision on lateral gaze, left or right (0, to 4), 3. Eye closure (0 to 2), 4. Talking (0 to 6), 5. Chewing (0 to 6), 6. Swallowing [0 to 6], 7. Breathing (0 to 9), 8. Neck flexion or extension (0 to 4), 9. Shoulder abduction (0 to 5), 10. Hip flexion (0 to 5). The higher score for each item indicated severity. The total score ranged 0 to 50 with higher score indicative of severe disease activity). A decrease from Baseline score showed improvement. | From Baseline to End of Treatment (Week 12) |
| Change From Baseline to Week 12 in the Myasthenia Gravis - Quality of Life Revised (MG-QoL15r) Scale Total Score | The MG-QoL15r is a 15-item patient-reported outcome measure designed to assess quality of life in patients with MG. Each item in the scale scored on a 0 to 2-point scale (0=Not much at all, 1=Somewhat, 2=Very much). The total score was the sum of the 15 individual item scores, ranging from 0 to 30. Higher scores indicated more severe impact of the disease on aspects of the patient's life. A decrease from Baseline score indicated improvement. | From Baseline to End of Treatment (Week 12) |
| Time to First Receipt of Rescue Therapy Over the 12-week Treatment Period | Time to first receipt of rescue therapy over the 12-week treatment period (in days) was defined as the date of first rescue therapy use minus date of first Investigational Medicinal Product (IMP) + 1. | From Baseline to End of Treatment (Week 12) |
| Percentage of Participants Achieving Minimal Symptom Expression (MSE) at Week 12 Without Rescue Therapy | Percentage of Participants achieving MSE was defined as achieving a MG-ADL value of a 0 (No MG symptoms) or 1 (Mild MG symptoms) at Week 12 and not having taken rescue therapy. Any participant with an event of death, myasthenic crisis or rescue therapy was considered as non-responders. Any other missing data was imputed using the missing at Random (MAR) assumption. | End of Treatment (Week 12) |
| Percentage of Participants Achieving a ≥ 3-point Reduction in MG-ADL Score at Week 12 Without Rescue Therapy | Percentage of participants achieving a ≥ 3-point reduction in MG-ADL Score at Week 12 without rescue therapy were reported. The MG-ADL is an 8-item patient-reported outcome measure assessing MG symptoms and their effects on daily activities. Each item in the scale scored on a 0 to 3 (0=None, 3=severe disease) point scale. The total score was the sum of all individual item scores and ranged from 0 to 24. Higher scores indicated more severe disability due to MG. Any participant with an event of death, myasthenic crisis or rescue therapy was considered as non-responders. Any other missing data was imputed using the MAR assumption. | End of Treatment (Week 12) |
| Percentage of Participants Achieving a ≥5-point Reduction in QMG Score Without Rescue Therapy at Week 12 | Percentage of participants achieving a ≥5-point reduction in QMG Score without rescue therapy at Week 12 were reported. The QMG is a standardized and validated quantitative strength scoring system that was developed specifically for MG. The scale consisted of 13 items. Each item in the scale scored on a 0 to 3-point scale, ranging from 0 (no weakness) to 3 (severe weakness), summing up to the overall score range from 0 to 39. Higher scores indicated more severe impairment. Any participant with an event of death, myasthenic crisis or rescue therapy was considered as non-responders. Any other missing data was imputed using the MAR assumption. | End of Treatment (Week 12) |
| 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 IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs. | From Baseline (Day 1) to Safety Follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks Follow-up]) |
| Phoenix |
| Arizona |
| 85028 |
| United States |
| Site 4: University of Southern California | Los Angeles | California | 90033 | United States |
| Site 31: University of California Irvine | Orange | California | 92868 | United States |
| Site 220: Investigator Site | Pasadena | California | 91101 | United States |
| Site 160: Forbes Norris MDA/ALS Research and Treatment Center | San Francisco | California | 94115 | United States |
| Site 24: Yale University | New Haven | Connecticut | 06510 | United States |
| Site 27: George Washington University | Washington D.C. | District of Columbia | 20037 | United States |
| Site 182: Gelasio Baras Neurology | Miami | Florida | 33175 | United States |
| Site 25: University of South Florida | Tampa | Florida | 33612 | United States |
| Site 135: Augusta University Medical Center | Augusta | Georgia | 30912 | United States |
| Site 176: Hawaii Pacific Neuroscience | Honolulu | Hawaii | 96817 | United States |
| Site 188: North Shore Medical Group - Glenview | Glenview | Illinois | 60026-1339 | United States |
| Site 156: Indiana University Health Neuroscience Center | Indianapolis | Indiana | 46202 | United States |
| Site 32: Kansas University Medical Center Research Institute | Kansas City | Kansas | 66160 | United States |
| Site 221: Neurology Center of New England | Foxborough | Massachusetts | 02035 | United States |
| Site 33: Detroit medical Center - University Health Center | Detroit | Michigan | 48202 | United States |
| Site 49: Michigan State University | East Lansing | Michigan | 48824 | United States |
| Site 127: University of Minnesota | Minneapolis | Minnesota | 55455 | United States |
| Site 134: Neurology and Sleep Disorders Clinic | Columbia | Missouri | 65212 | United States |
| Site 117: Las Vegas Clinic | Las Vegas | Nevada | 89145 | United States |
| Site 123: Northwell Health Neuroscience Institute | Great Neck | New York | 11021 | United States |
| Site 23: Hospital for Special Surgery | New York | New York | 10021 | United States |
| Site 47: Mount Sinai Hospital | New York | New York | 10029 | United States |
| Site 22: University of North Carolina | Chapel Hill | North Carolina | 27599 | United States |
| Site 15: Duke University | Durham | North Carolina | 27710 | United States |
| Site 122: Cleveland Clinic | Cleveland | Ohio | 44195 | United States |
| Site 38: Ohio State University | Columbus | Ohio | 43210 | United States |
| Site 40: Allegheny Neurological Associates | Pittsburgh | Pennsylvania | 15212 | United States |
| Site 128: Medical University of South Carolina | Charleston | South Carolina | 29425 | United States |
| Site 185: Neurology Clinic Cordova | Cordova | Tennessee | 38018 | United States |
| Site 131: Austin Neuromuscular Center | Austin | Texas | 78756 | United States |
| Site 19: University of Texas Southwestern | Dallas | Texas | 75390 | United States |
| Site 39: University of Utah | Salt Lake City | Utah | 84132 | United States |
| Site 164: University of Virginia Health System | Charlottesville | Virginia | 22908 | United States |
| Site 154: University of Washington | Seattle | Washington | 98195 | United States |
| Site 45: Center for Neurological Disorders | Milwaukee | Wisconsin | 53215 | United States |
| Site 44: London Health Sciences Centre University Hospital | London | Ontario | N6A 5A5 | Canada |
| Site 11: Montreal Neurological Institute and Hospital (McGill University) | Montreal | Quebec | H3A 2B4 | Canada |
| Site 191: Centre Hosptitalier Universitaire d'Angers | Angers | France |
| Site 204: Centre Hospitalier Régional Universitaire de Lille | Lille | France |
| Site 118: Hôpital Pasteur | Nice | 06000 | France |
| Site 105: Pitié-Salpêtrière University Hospital | Paris | 75013 | France |
| Site 137: Les Hôpitaux Universitaires de Strasbourg | Strasbourg | 67091 | France |
| Site 150: Universitätsmedizin Göttingen | Göttingen | 37075 | Germany |
| Site 129: Universitätsklinikum Tübingen | Tübingen | 72076 | Germany |
| Site 126: Fondazione IRCCS Istituto Neurologico Carlo Besta | Milan | 20133 | Italy |
| Site 132: Università Cattolica del Sacro Cuore - Campus di Milano | Roma | 20123 | Italy |
| Site 169: International University of Health and Welfare Narita Hospital | Narita | Chiba | 286-8520 | Japan |
| Site 151: Chiba University Hospital | Chiba | 260-8677 | Japan |
| Site 136: General Hanamaki Hospital | Iwata | 025-0075 | Japan |
| Site 179: Kagawa University Hospital - Collagen disease/Rheumatic int | Kita-gun | Japan |
| Site 146: Nagasaki University Hospital | Nagasaki | 852-8501 | Japan |
| Site 152: Hokkaido Medical Center | Sapporo | 063-0005 | Japan |
| Site 144: Sendai Medical Center | Sendai | 983-8520 | Japan |
| Site 153: Toho University Ohashi Medical Center | Tokyo | 153-8515 | Japan |
| Site 163: Tokyo Medical University Hospital | Tokyo | 160-0023 | Japan |
| Site 141: Keio University Hospital | Tokyo | 160-8582 | Japan |
| Site 165: Osaka University Hospital | Tokyo | 565-0871 | Japan |
| Site 140: Haukeland University Hospital / Health Bergen | Bergen | 5021 | Norway |
| Site 143: Oslo Universitetssykehus | Oslo | 0450 | Norway |
| Site 209: Niepubliczny Zakład Opieki Zdrowotnej NEURO - KARD | Poznan | Greater Poland Voivodeship | 61-853 | Poland |
| Site 192: Krakowski Szpital Specjalistyczny im. Jana Pawła II | Krakow | Lesser Poland Voivodeship | 31-202 | Poland |
| Site 205: Prywatny Gabinet Lekarski Urszula Chyrchel-Paszkiewicz | Lublin | Lublin Voivodeship | 20-093 | Poland |
| Site 194: Twoja Przychodnia - Centrum Medyczne Nowa Sól | Nowa Sól | Lubusz Voivodeship | 67-100 | Poland |
| Site 214: AmiCare Centrum Medyczne | Lodz | Lódzkie | 90-644 | Poland |
| Site 201: Centrum Medyczne Pratia - Warszawa | Warsaw | Masovian Voivodeship | 01-868 | Poland |
| Site 195: Wielospecjalistyczna Poradnia Lekarska Synapsis | Katowice | Silesian Voivodeship | 40-123 | Poland |
| Site 213: Niepubliczny Zakład Opieki Zdrowotnej NOVO-MED | Katowice | Silesian Voivodeship | 40-650 | Poland |
| Site 193: Krakowska Akademia Neurologii - Centrum Neurologii Kliniczne | Krakow | Poland |
| Site 211: Specjalistyczne Gabinety Sp. z o.o. | Krakow | Poland |
| Site 210: Clinhouse Centrum Medyczne | Lublin | 20-093 | Poland |
| Site 133: Hospital Universitari Vall d'Hebrón | Barcelona | 08035 | Spain |
| Site 168: Hospital de la Santa Creu i Sant Pau | Barcelona | Spain |
| Site 138: Hospital Universitario de Basurto | Bilbao | 48013 | Spain |
| Site 119: Oxford University Hospitals NHS Foundation Trust | Oxford | OX3 9DU | United Kingdom |
| Site 130: Royal Hallamshire Hospital | Sheffield | S10 2JF | United Kingdom |
| Result |
| Weiss MD, Freimer M, Leite MI, Maniaol A, Utsugisawa K, Bloemers J, Boroojerdi B, Howard E, Savic N, Howard JF Jr. Improvement of fatigue in generalised myasthenia gravis with zilucoplan. J Neurol. 2024 May;271(5):2758-2767. doi: 10.1007/s00415-024-12209-3. Epub 2024 Feb 24. |
| 39314260 | Result | de la Borderie G, Chimits D, Boroojerdi B, Brock M, Duda PW, Grimson F, Mahoney P, Strimenopoulou F, Cutter G, Aban I, Brauner S, Petersson M, Howard JF Jr, Bennett N. Maintenance of zilucoplan efficacy in patients with generalised myasthenia gravis up to 24 weeks: a model-informed analysis. Ther Adv Neurol Disord. 2024 Sep 21;17:17562864241279125. doi: 10.1177/17562864241279125. eCollection 2024. |
| 40504283 | Result | Hewamadduma C, Freimer M, Genge A, Leite MI, Utsugisawa K, Vu T, Boroojerdi B, Grimson F, Savic N, Vanderkelen M, Howard JF Jr; RAISE-XT study team. Changes in corticosteroid and non-steroidal immunosuppressive therapy with long-term zilucoplan treatment in generalized myasthenia gravis. J Neurol. 2025 Jun 12;272(7):457. doi: 10.1007/s00415-025-13113-0. |
| 40450840 | Derived | Utsugisawa K, Masuda M, Boroojerdi B, Grimson F, Howard JF Jr; RAISE Study Team. Efficacy of zilucoplan in patients with generalised myasthenia gravis who have not previously received immunoglobulin or plasma exchange: A subgroup analysis from the Phase 3 RAISE study. J Neurol Sci. 2025 Jul 15;474:123550. doi: 10.1016/j.jns.2025.123550. Epub 2025 May 16. |
| COMPLETED |
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| NOT COMPLETED |
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The modified Intention-to-Treat (mITT) population included all randomized participants who received at least 1 dose of study drug and had at least 1 post-dosing MG-ADL score.
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| ID | Title | Description |
|---|---|---|
| BG000 | Placebo | Participants self-administered zilucoplan (RA101495) matching placebo as subcutaneous (SC) injection during 12-week Treatment Period. |
| BG001 | Zilucoplan 0.3 mg/kg | Participants self-administered zilucoplan (RA101495) 0.3 milligrams/kilogram/day (mg/kg/day) SC injection during 12-week Treatment Period. |
| BG002 | 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 | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 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 | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Change From Baseline (CFB) to Week 12 in Myasthenia Gravis-Activities of Daily Living (MG-ADL) Total Score | The MG-ADL is an 8-item patient-reported outcome measure assessing MG symptoms and their effects on daily activities. Each item in the scale scored 0 to 3 (0=None, 3=severe disease) point scale. The total score was the sum of all individual item scores and ranged from 0 to 24. Higher scores indicated more severe disability due to MG. A decrease from Baseline score indicated improvement. | The modified Intention-to-Treat (mITT) population included all randomized participants who received at least 1 dose of study drug and had at least 1 post-dosing MG-ADL score. | Posted | Least Squares Mean | 95% Confidence Interval | score on a scale | From Baseline to End of Treatment (Week 12) |
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| Secondary | Change From Baseline to Week 12 in the Quantitative Myasthenia Gravis (QMG) Total Score | The QMG is a standardized and validated quantitative strength scoring system that was developed specifically for MG. The scale consisted of 13 items. Each item in the scale scored on a 0 to 3-point scale, ranging from 0 (no weakness) to 3 (severe weakness), summing up to the overall score range from 0 to 39. Higher scores indicated more severe impairment. A decrease from Baseline score indicated improvement. | The modified Intention-to-Treat (mITT) population included all randomized participants who received at least 1 dose of study drug and had at least 1 post-dosing MG-ADL score. | Posted | Least Squares Mean | 95% Confidence Interval | score on a scale | From Baseline to End of Treatment (Week 12) |
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| Secondary | Change From Baseline to Week 12 in the Myasthenia Gravis Composite (MGC) Scale Total Score | The total MGC score was sum of responses to 10 individual items : 1. Ptosis upward gaze (0 to 3), 2. Double vision on lateral gaze, left or right (0, to 4), 3. Eye closure (0 to 2), 4. Talking (0 to 6), 5. Chewing (0 to 6), 6. Swallowing [0 to 6], 7. Breathing (0 to 9), 8. Neck flexion or extension (0 to 4), 9. Shoulder abduction (0 to 5), 10. Hip flexion (0 to 5). The higher score for each item indicated severity. The total score ranged 0 to 50 with higher score indicative of severe disease activity). A decrease from Baseline score showed improvement. | The modified Intention-to-Treat (mITT) population included all randomized participants who received at least 1 dose of study drug and had at least 1 post-dosing MG-ADL score. | Posted | Least Squares Mean | 95% Confidence Interval | score on a scale | From Baseline to End of Treatment (Week 12) |
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| Secondary | Change From Baseline to Week 12 in the Myasthenia Gravis - Quality of Life Revised (MG-QoL15r) Scale Total Score | The MG-QoL15r is a 15-item patient-reported outcome measure designed to assess quality of life in patients with MG. Each item in the scale scored on a 0 to 2-point scale (0=Not much at all, 1=Somewhat, 2=Very much). The total score was the sum of the 15 individual item scores, ranging from 0 to 30. Higher scores indicated more severe impact of the disease on aspects of the patient's life. A decrease from Baseline score indicated improvement. | The modified Intention-to-Treat (mITT) population included all randomized participants who received at least 1 dose of study drug and had at least 1 post-dosing MG-ADL score. | Posted | Least Squares Mean | 95% Confidence Interval | score on a scale | From Baseline to End of Treatment (Week 12) |
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| Secondary | Time to First Receipt of Rescue Therapy Over the 12-week Treatment Period | Time to first receipt of rescue therapy over the 12-week treatment period (in days) was defined as the date of first rescue therapy use minus date of first Investigational Medicinal Product (IMP) + 1. | The modified Intention-to-Treat (mITT) population included all randomized participants who received at least 1 dose of study drug and had at least 1 post-dosing MG-ADL score. | Posted | Median | Full Range | Days | From Baseline to End of Treatment (Week 12) |
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| Secondary | Percentage of Participants Achieving Minimal Symptom Expression (MSE) at Week 12 Without Rescue Therapy | Percentage of Participants achieving MSE was defined as achieving a MG-ADL value of a 0 (No MG symptoms) or 1 (Mild MG symptoms) at Week 12 and not having taken rescue therapy. Any participant with an event of death, myasthenic crisis or rescue therapy was considered as non-responders. Any other missing data was imputed using the missing at Random (MAR) assumption. | The mITT population included randomized participants who received at least 1 dose of study drug and had at least 1 post-dosing MG-ADL score. | Posted | Number | percentage of participants | End of Treatment (Week 12) |
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| Secondary | Percentage of Participants Achieving a ≥ 3-point Reduction in MG-ADL Score at Week 12 Without Rescue Therapy | Percentage of participants achieving a ≥ 3-point reduction in MG-ADL Score at Week 12 without rescue therapy were reported. The MG-ADL is an 8-item patient-reported outcome measure assessing MG symptoms and their effects on daily activities. Each item in the scale scored on a 0 to 3 (0=None, 3=severe disease) point scale. The total score was the sum of all individual item scores and ranged from 0 to 24. Higher scores indicated more severe disability due to MG. Any participant with an event of death, myasthenic crisis or rescue therapy was considered as non-responders. Any other missing data was imputed using the MAR assumption. | The mITT population included randomized participants who received at least 1 dose of study drug and had at least 1 post-dosing MG-ADL score. | Posted | Number | percentage of participants | End of Treatment (Week 12) |
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| Secondary | Percentage of Participants Achieving a ≥5-point Reduction in QMG Score Without Rescue Therapy at Week 12 | Percentage of participants achieving a ≥5-point reduction in QMG Score without rescue therapy at Week 12 were reported. The QMG is a standardized and validated quantitative strength scoring system that was developed specifically for MG. The scale consisted of 13 items. Each item in the scale scored on a 0 to 3-point scale, ranging from 0 (no weakness) to 3 (severe weakness), summing up to the overall score range from 0 to 39. Higher scores indicated more severe impairment. Any participant with an event of death, myasthenic crisis or rescue therapy was considered as non-responders. Any other missing data was imputed using the MAR assumption. | The mITT population included randomized participants who received at least 1 dose of study drug and had at least 1 post-dosing MG-ADL score. | Posted | Number | percentage of participants | End of Treatment (Week 12) |
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| Secondary | 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 IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs. | The Safety Set (SS) included all participants who received at least 1 dose of study drug based on the actual study treatment received. | Posted | Number | percentage of participants | From Baseline (Day 1) to Safety Follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks Follow-up]) |
|
|
From Baseline (Day 1) to Safety follow-up visit (19 Weeks [12 weeks Treatment Period plus up to 7 weeks follow-up])
A TEAE is defined as an AE starting on or after the time of first administration of IMP and up to and including 40 days after the final dose (or last contact depending on which occurs first). Adverse events starting before the date of the first administration of IMP were not considered TEAEs.
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 self-administered zilucoplan (RA101495) matching placebo as subcutaneous (SC) injection during 12-week Treatment Period. | 1 | 88 | 13 | 88 | 34 | 88 |
| EG001 | Zilucoplan 0.3 mg/kg | Participants self-administered zilucoplan (RA101495) 0.3 milligrams/kilogram/day (mg/kg/day) SC injection during 12-week Treatment Period. | 1 | 86 | 11 | 86 | 41 | 86 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA 24 | Non-systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 24 | Non-systematic Assessment |
| |
| Aphthous ulcer | Gastrointestinal disorders | MedDRA 24 | Non-systematic Assessment |
| |
| Oesophageal candidiasis | Infections and infestations | MedDRA 24 | Non-systematic Assessment |
| |
| Oral candidiasis | Infections and infestations | MedDRA 24 | Non-systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA 24 | Non-systematic Assessment |
| |
| COVID-19 pneumonia | Infections and infestations | MedDRA 24 | Non-systematic Assessment |
| |
| Herpes simplex meningoencephalitis | Infections and infestations | MedDRA 24 | Non-systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 24 | Non-systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA 24 | Non-systematic Assessment |
| |
| Lipase increased | Investigations | MedDRA 24 | Non-systematic Assessment |
| |
| Metastases to meninges | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 24 | Non-systematic Assessment |
| |
| Basal cell carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 24 | Non-systematic Assessment |
| |
| Cerebral haemorrhage | Nervous system disorders | MedDRA 24 | Non-systematic Assessment |
| |
| Cerebrovascular accident | Nervous system disorders | MedDRA 24 | Non-systematic Assessment |
| |
| Myasthenia gravis | Nervous system disorders | MedDRA 24 | Non-systematic Assessment |
| |
| Hyperemesis gravidarum | Pregnancy, puerperium and perinatal conditions | MedDRA 24 | Non-systematic Assessment |
| |
| Chronic obstructive pulmonary disease | Respiratory, thoracic and mediastinal disorders | MedDRA 24 | Non-systematic Assessment |
| |
| Pulmonary embolism | Respiratory, thoracic and mediastinal disorders | MedDRA 24 | Non-systematic Assessment |
| |
| Angioedema | Skin and subcutaneous tissue disorders | MedDRA 24 | Non-systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Diarrhoea | Gastrointestinal disorders | MedDRA 24 | Non-systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 24 | Non-systematic Assessment |
| |
| Injection site bruising | General disorders | MedDRA 24 | Non-systematic Assessment |
| |
| Injection site pain | General disorders | MedDRA 24 | Non-systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 24 | Non-systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 24 | Non-systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA 24 | Non-systematic Assessment |
| |
| Lipase increased | Investigations | MedDRA 24 | Non-systematic Assessment |
| |
| Amylase increased | Investigations | MedDRA 24 | Non-systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 24 | Non-systematic Assessment |
| |
| Myasthenia gravis | Nervous system disorders | MedDRA 24 | Non-systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 24 | Non-systematic Assessment |
|
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| UCB | Cares | 001 844 599 2273 | UCBCares@ucb.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Dec 6, 2021 | Nov 22, 2022 | SAP_001.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 |
|---|---|
| C000719268 | zilucoplan |
Not provided
Not provided
Not provided
| >=65 years |
|
| Male |
|
| Asian |
|
| Black |
|
| Native Hawaiian or other Pacific Islander |
|
| White |
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| Other/Mixed |
|
| Missing |
|
| Not Hispanic or Latino |
|
| Missing |
|
| Participants |
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| Participants |
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| Counts |
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| Participants |
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| Units | Counts |
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| Participants |
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| Units | Counts |
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| Participants |
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