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| ID | Type | Description | Link |
|---|---|---|---|
| 2024-512399-37 | Registry Identifier | EU Clinical Trials | |
| U1111-1306-6625 | Other Identifier | World Health Organization (WHO) |
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The RAISE-XT study is an open-label extension study to evaluate the long-term efficacy, safety, and tolerability of zilucoplan in subjects with gMG who have previously participated in a qualifying Ra Pharmaceuticals sponsored zilucoplan study.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 0.3 mg/kg zilucoplan (RA101495) | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| zilucoplan (RA101495) | Drug | Daily subcutaneous (SC) injection |
|
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of treatment-emergent adverse events (TEAEs) | A treatment-emergent adverse event is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study medication, whether or not considered related to the study medication. | From Baseline (Day 1) to Safety Follow-Up Visit (up to 36 months) |
| Measure | Description | Time Frame |
|---|---|---|
| Change from Baseline to Week 12 in the Myasthenia Gravis-Activities of Daily Living (MG-ADL) Score | The MG-ADL profile provides an assessment of MG symptom severity and measures 8 items on a 0-3 scale, with 0 being the least severe. The total sum of the 8 items represents the ADL score. The ADL score can range from 0 (least severe) to 24 (most severe). | From Baseline (Day 1) to Week 12 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| UCB Cares | 001 844 599 2273 | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Mg0011 41 | Mobile | Alabama | 36604 | United States | ||
| Mg0011 116 |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 38400914 | 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. |
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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.
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| Change from Baseline to Week 12 in the the Quantitative Myasthenia Gravis (QMG) Score | The QMG test is a standardized quantitative strength scoring system and measures 13 items on a 0-3 scale, with 0 being the least severe. The total sum of the 13 items represents the QMG score. The QMG score can range from 0 (least severe) to 39 (most severe). | From Baseline (Day 1) to Week 12 |
| Change from Baseline to Week 12 in the Myasthenia Gravis Composite (MGC) Score | The MGC is a 10-item scale that has been used to measure the clinical status of patients with Myasthenia Gravis (MG) in order to evaluate treatment response. The MGC has 4-point Likerttype Scale response options ranging from 0 to 2, 3, 4, 5, 6 or 9 according to the item (weighted response options). The total score is the sum of all items (range 0-50) where higher scores indicate more severe impairment due to the disease. | From Baseline (Day 1) to Week 12 |
| Change from Baseline to Week 12 in the Myasthenia Gravis - Quality of Life revised (MG-QOL15r) Score | The MG-QOL15r is a 15-item survey that was designed to assess quality of life in patients with MG. The MG-QoL has 3-point Likert Scale response options ranging from 0 to 2. The MGQoL15r score can range from 0 to 30, where higher scores indicate more severe impact of the disease on aspects of the patient's life. | From Baseline (Day 1) to Week 12 |
| Phoenix |
| Arizona |
| 85028 |
| United States |
| Mg0011 31 | Irvine | California | 92697 | United States |
| MG0011 4 | Los Angeles | California | 90033 | United States |
| Mg0011 220 | Pasadena | California | 91101 | United States |
| Mg0011 160 | San Francisco | California | 94115 | United States |
| Mg0011 24 | New Haven | Connecticut | 06511 | United States |
| Mg0011 27 | Washington D.C. | District of Columbia | 20052 | United States |
| Mg0011 25 | Tampa | Florida | 33612 | United States |
| Mg0011 135 | Augusta | Georgia | 30912 | United States |
| Mg0011 188 | Glenview | Illinois | 60026-1339 | United States |
| Mg0011 156 | Indianapolis | Indiana | 46202 | United States |
| Mg0011 32 | Kansas City | Kansas | 66160 | United States |
| Mg0011 33 | Detroit | Michigan | 48201 | United States |
| Mg0011 49 | East Lansing | Michigan | 48824 | United States |
| Mg0011 134 | Columbia | Missouri | 65201 | United States |
| Mg0011 117 | Las Vegas | Nevada | 89145 | United States |
| Mg0011 30 | Buffalo | New York | 14202-1102 | United States |
| Mg0011 123 | Great Neck | New York | 11021 | United States |
| Mg0011 23 | New York | New York | 10021 | United States |
| Mg0011 47 | New York | New York | 10029 | United States |
| Mg0011 22 | Chapel Hill | North Carolina | 27599 | United States |
| Mg0011 122 | Cleveland | Ohio | 44195 | United States |
| Mg0011 38 | Columbus | Ohio | 43210 | United States |
| Mg0011 40 | Pittsburgh | Pennsylvania | 15212 | United States |
| Mg0011 128 | Charleston | South Carolina | 29425-8908 | United States |
| Mg0011 28 | Cordova | Tennessee | 38018 | United States |
| Mg0011 131 | Austin | Texas | 78759 | United States |
| Mg0011 19 | Dallas | Texas | 75390 | United States |
| Mg0011 39 | Salt Lake City | Utah | 84105 | United States |
| Mg0011 164 | Charlottesville | Virginia | 22903 | United States |
| Mg0011 154 | Seattle | Washington | 98195 | United States |
| Mg0011 45 | Milwaukee | Wisconsin | 53215 | United States |
| Mg0011 11 | Montreal | Canada |
| Mg0011 204 | Lille | France |
| Mg0011 118 | Nice | France |
| Mg0011 105 | Paris | France |
| Mg0011 137 | Strasbourg | France |
| Mg0011 150 | Göttingen | Germany |
| Mg0011 129 | Tübingen | Germany |
| Mg0011 132 | Milan | Italy |
| Mg0011 126 | Roma | Italy |
| Mg0011 151 | Chiba | Japan |
| Mg0011 136 | Hanamaki-shi | Japan |
| Mg0011 179 | Kita-gun | Japan |
| Mg0011 153 | Meguro-ku | Japan |
| Mg0011 146 | Nagasaki | Japan |
| Mg0011 169 | Narita | Japan |
| Mg0011 152 | Sapporo | Japan |
| Mg0011 144 | Sendai | Japan |
| Mg0011 163 | Shinjuku-ku | Japan |
| Mg0011 141 | Tokyo | Japan |
| Mg0011 140 | Bergen | Norway |
| Mg0011 143 | Oslo | Norway |
| Mg0011 195 | Katowice | Poland |
| Mg0011 213 | Katowice | Poland |
| Mg0011 192 | Krakow | Poland |
| Mg0011 193 | Krakow | Poland |
| Mg0011 211 | Krakow | Poland |
| Mg0011 214 | Lodz | Poland |
| Mg0011 205 | Lublin | Poland |
| Mg0011 194 | Nowa Sól | Poland |
| Mg0011 209 | Poznan | Poland |
| Mg0011 201 | Warsaw | Poland |
| Mg0011 210 | Zabrze | Poland |
| Mg0011 133 | Barcelona | Spain |
| Mg0011 168 | Barcelona | Spain |
| Mg0011 138 | Bilbao | Spain |
| Mg0011 119 | Oxford | United Kingdom |
| Mg0011 130 | Sheffield | United Kingdom |
| 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. |
| 38638673 | Derived | Howard JF Jr, Bresch S, Farmakidis C, Freimer M, Genge A, Hewamadduma C, Hinton J, Hussain Y, Juntas-Morales R, Kaminski HJ, Maniaol A, Mantegazza R, Masuda M, Nowak RJ, Sivakumar K, Smilowski M, Utsugisawa K, Vu T, Weiss MD, Zajda M, Bloemers J, Boroojerdi B, Brock M, de la Borderie G, Duda PW, Vanderkelen M, Leite MI; RAISE-XT Study Team*. Long-term safety and efficacy of zilucoplan in patients with generalized myasthenia gravis: interim analysis of the RAISE-XT open-label extension study. Ther Adv Neurol Disord. 2024 Apr 17;17:17562864241243186. doi: 10.1177/17562864241243186. eCollection 2024. |
| 37059508 | Derived | 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. |
| 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 |
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| ID | Term |
|---|---|
| C000719268 | zilucoplan |
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