The complement component C5 inhibitory peptide zilucoplan is currently in phase III clinical trials for myasthenia gravis (MG). There will also be a 40-day follow-up visit for participants who do not enroll in the open-label extension study. Phase 2 Safety and Efficacy Study of Zilucoplan (RA101495) to Treat PNH Patients Who Have … All documents are available in English only, for a pre-specified time, typically 12 months, on a password protected portal. Studies a U.S. FDA-regulated Drug Product: Studies a U.S. FDA-regulated Device Product: Change from Baseline (CFB) in the MG-ADL Score [ Time Frame: From Baseline (Day 1) to Week 12 ], Change from Baseline to Week 12 in the Quantitative Myasthenia Gravis (QMG) Score [ Time Frame: From Baseline (Day 1) to Week 12 ], Change from Baseline to Week 12 in the Myasthenia Gravis Composite (MGC) Score [ Time Frame: From Baseline (Day 1) to Week 12 ], Change from Baseline to Week 12 in the Myasthenia Gravis - Quality of Life revised (MG-QOL15r) Score [ Time Frame: From Baseline (Day 1) to Week 12 ], Time to first receipt of rescue therapy over the 12-week Treatment Period [ Time Frame: Treatment Period (from Day 1 to Week 12) ], Percentage of participants achieving Minimal Symptom Expression (MSE) at Week 12 [ Time Frame: Week 12 ], Percentage of participants achieving a ≥ 3-point reduction in MG-ADL Score at Week 12 without rescue therapy [ Time Frame: Week 12 ], Percentage of participants achieving a ≥5-point reduction in QMG Score without rescue therapy at Week 12 [ Time Frame: Week 12 ], Incidence of treatment-emergent adverse events (TEAEs) [ Time Frame: From Baseline (Day 1) to Safety Follow-Up Visit (up to Week 19) ], Diagnosis of gMG [Myasthenia Gravis Foundation of America (MGFA) Class II-IV] at Screening, Positive serology for acetylcholine receptor (AChR) autoantibodies, MG-ADL Score of ≥ 6 at Screening and Baseline, No change in corticosteroid dose for at least 30 days prior to Baseline or anticipated to occur during the 12-week Treatment Period, No change in immunosuppressive therapy, including dose, for at least 30 days prior to Baseline or anticipated to occur during the 12-week Treatment Period, Thymectomy within 12 months prior to Baseline or scheduled to occur during the 12 week Treatment Period, Current or recent systemic infection within 2 weeks prior to Baseline or injection requiring intravenous (IV) antibiotics within 4 weeks prior to Baseline. This therapy may improve muscle function by blocking the autoimmune reaction that destroys the junction between … Zilucoplan use earlier in the treatment paradigm would be suitable in this population should phase 3 efficacy and safety data be equally favorable. Biohaven is now conducting a phase 3 clinical trial of verdiperstat in multiple system atrophy, for which there are now only symptom- atic and palliative therapies. A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety, Tolerability, and Efficacy of Zilucoplan in Subjects With Immune-Mediated Necrotizing Myopathy: Actual Study Start Date : December 3, 2019: Estimated Primary Completion Date : March 15, 2021: Estimated Study Completion Date : November 2022 This 12-week study will consist of a total of seven visits. In a randomized, double-blind, placebo-controlled, phase 2 clinical trial, zilucoplan demonstrated clinically meaningful complement inhibition in patients with acetylcholine receptor-positive gMG. 03/25/2021. The single, pivotal, randomized, double-blind, placebo-controlled Phase 3 trial is designed to evaluate the efficacy of a once-daily, subcutaneously (SC) self-administered dose of … All documents are available in English only, for a prespecified time, typically 12 months, on a password protected portal. The total sum of the 13 items represents the QMG score. However, it had set a low bar for success, prespecifying clearance of a one-sided, rather than the more stringent two-sided, p value of 1.0 (versus the commonly used 0.5). 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. The single, pivotal, randomized, double-blind, placebo-controlled Phase 3 trial is designed to evaluate the efficacy of a once-daily, subcutaneously (SC) self-administered dose of 0.3 mg/kg of zilucoplan … For patients who require rescue treatment, the time from Baseline to the first dose of rescue treatment will be calculated. Trial design: This is a phase 2 academic, prospective, 2:1 randomized, open … About Zilucoplan - The phase 3 product candidate, zilucoplan, is a once-daily self-administered, subcutaneous peptide inhibitor of C5 and is currently being tested in phase 3 for the treatment of gMG with top-line results expected in Q4 2021. The purpose of this study is to investigate the efficacy and safety of Zilucoplan in improving oxygenation and short- and long-term outcome of COVID-19 patients with acute hypoxic respiratory failure. 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. Listing a study does not mean it has been evaluated by the U.S. Federal Government. believe zilucoplan, if successfully developed and approved in these indications, has the potential to support the creation of a leading complement-focused neurology franchise. 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. Patients will be assigned randomly to receive either 0.3 mg zilucoplan per kg body weight, or placebo, via a daily under-the-skin injection for 12 weeks. This prevents MAC-mediated lysis and destruction of red blood cells (RBCs) that occurs in complement-mediated diseases, such as paroxysmal nocturnal hemoglobinuria (PNH), generalized myasthenia gravis (gMG) and lupus nephritis (LN). The primary endpoint of zilucoplan's phase II trial was the QMG scale, where Ra claimed a win with both doses studied. This therapy may improve muscle function by blocking the autoimmune reaction that destroys the junction between nerves and muscles in people living with gMG. A planned phase 3 trial will evaluate the clinical effect of 0.3 mg/kg dose of zilucoplan … "These positive data support the potential therapeutic role of zilucoplan in gMG and its further evaluation in a registrational phase 3 trial," the investigators write. The MG-QoL has 3-point Likert Scale response options ranging from 0 to 2. This comes after the company’s completion of End-of-Phase 2 interactions with the FDA Please visit www.gmgstudy.com or https://clinicaltrials.gov/ct2/show/NCT04115293 for the full listing of exclusion criteria. Ra plans to commence a Phase 3 trial before the end of 2019 with zilucoplan in patients who have generalized myasthenia gravis, a chronic autoimmune and neuromuscular disease. The initiation of the Phase 3 gMG clinical trial follows the successful completion of a Phase … phase 1 and 2 efficacy, safety, and tolerability data with limited long-term follow-up. To learn more or to inquire about participation, email UCBCares@ucb.com or call (844) 599-2273. Further potential indications that are potentially addressable by zilucoplan include immune-mediated necrotizing myopathy (IMNM), amyotrophic lateral sclerosis (ALS) and other tissue-based complement-mediated disorders with high unmet medical need. About Zilucoplan - The phase 3 product candidate, zilucoplan, is a once-daily self-administered, subcutaneous peptide inhibitor of C5 and is currently being tested in phase 3 for the treatment of gMG with top-line results expected in Q4 2021. Why Should I Register and Submit Results? Study record managers: refer to the Data Element Definitions if submitting registration or results information. 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. Researchers at Ra Pharmaceuticals (now part of UCB BioSciences) are seeking individuals living with generalized myasthenia gravis (gMG) to participate in a phase 3 study to confirm safety, tolerability, and efficacy of the investigational drug Zilucoplan. Top-line data from the Phase 3 RAISE study expected in early 2021 ... | April 12, 2021 Choosing to participate in a study is an important personal decision. Alignment with FDA on a single, pivotal, 12-week, placebo-controlled, Phase 3 clinical trial to initiate in the second half of 2019. For general information, Learn About Clinical Studies. This prevents MAC-mediated lysis and destruction of red blood cells (RBCs) that occurs in complement-mediated diseases, such as paroxysmal nocturnal hemoglobinuria (PNH), generalized myasthenia gravis (gMG) and lupus nephritis (LN). The company has stated that it plans to engage with regulatory agencies, including the FDA, in the first half of 2019 regarding the design of a phase 3 clinical trial evaluating the 0.3 mg/kg dose of zilucoplan versus placebo in patients with generalized myasthenia gravis, based on these data. It is estimated to affect more than 700,000 people worldwide. 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. The drug/placebo will be self-administered at home as a daily subcutaneous (under-the-skin) injection. Genetic and Rare Diseases Information Center, U.S. Department of Health and Human Services. Muscular Dystrophy Association National Office, https://clinicaltrials.gov/ct2/show/NCT04115293, Clinical Trial Alert: Phase 2 Study of Pegcetacoplan in Adults with Sporadic ALS (MERIDIAN), Clinical Trial Alert: Phase 1/2 Study of AT845 in Late-Onset Pompe Disease, Clinical Trial Alert: Observational Study to Identify Biomarkers in Individuals Receiving Radicava for ALS, Diagnosis of gMG [Myasthenia Gravis Foundation of America (MGFA) Class II-IV] at screening, Positive serology for acetylcholine receptor (AChR) binding autoantibodies, MG-ADL score of ≥6 at screening and baseline, QMG score of ≥12 at screening and baseline (off acetylcholinesterase inhibitor therapy for at least 10 hours), No change in corticosteroid dose for at least 30 days prior to baseline or anticipated to occur during the 12-week treatment period, No change in immunosuppressive therapy, including dose, for at least 30 days prior to baseline or anticipated to occur during the 12-week treatment period. In the Phase 2 trial designed to evaluate the safety, tolerability and preliminary efficacy of zilucoplan in patients with gMG, 44 participants received a daily subcutaneous (under-the-skin) injection of either 0.3 mg/kg zilucoplan, 0.1 mg/kg zilucoplan or a placebo over the course of 12 weeks. The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. 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. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. Given the similarity in the safety profile for both doses, as well as the more rapid and pronounced clinical effect seen with 0.3-mg/kg zilucoplan, this dose has been selected for further testing in a pivotal phase 3 study (ClinicalTrials.gov Identifier: NCT04115293). This phase 2 study showed that complement inhibition with zilucoplan was effective in patients with moderate to severe AChR antibody–positive gMG and that its safety and tolerability were favorable. Ra Pharmaceuticals’ macrocyclic peptide inhibitor of complement component 5 is expected to enter phase 3 of its development in the latter half of 2019 after success in a phase 2 trial of its potential in myasthenia gravis. Researchers at Ra Pharmaceuticals (now part of UCB BioSciences) are seeking individuals living with generalized myasthenia gravis (gMG) to participate in a phase 3 study to confirm safety, tolerability, and efficacy of the investigational drug Zilucoplan. The phase 2 study of zilucoplan in gMG was actually the one that drew my attention last year. 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 score is the sum of all items (range 0-50) where higher scores indicate more severe impairment due to the disease. Ra Pharmaceuticals is preparing to launch a Phase 3 clinical trial to assess the therapeutic potential of zilucoplan for the treatment of patients with generalized myasthenia gravis (gMG). 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. Zilucoplan is currently being tested in phase 3 for the treatment of gMG with top-line results expected in early 2021. Zilucoplan use earlier in the treatment paradigm would be suitable in this population should phase 3 efficacy and safety data be equally favorable. You have reached the maximum number of saved studies (100). Not all participants will receive Zilucoplan during this study; participants will be randomized in a 1:1 ratio to receive Zilucoplan or an inactive placebo control. Talk with your doctor and family members or friends about deciding to join a study. ©2021, Muscular Dystrophy Association Inc. All rights reserved. The QMG score can range from 0 (least severe) to 39 (most severe). Ra Pharmaceuticals (NASDAQ:RARX) dosed the first patient in its RAISE study, a pivotal Phase 3 clinical trial evaluating zilucoplan for the treatment of generalized myasthenia gravis (gMG).. gMG is a chronic autoimmune disease characterized by skeletal muscle weakness and fatigue that becomes increasingly severe with repeated use. MSE is defined as an MG-ADL of 0 or 1 at Week 12 without rescue therapy. Zilucoplan also inhibits the interaction between C5b and C6, thereby further blocking MAC assembly. 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. 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. The incidence ranges from 0.3 to 2.8 per 100,000. It will begin with screening and baseline assessments, and then require routine visits after one, two, four, eight, and twelve weeks after study enrollment. The ADL score can range from 0 (least severe) to 24 (most severe). Bloomberg the Company & Its Products The Company & its Products Bloomberg Terminal Demo Request Bloomberg Anywhere Remote Login Bloomberg Anywhere Login Bloomberg Customer Support Customer Support The total sum of the 8 items represents the ADL score. Ra Pharmaceuticals, Inc. (Nasdaq:RARX) today announced the initiation of dosing in the RAISE study, its global, pivotal, Phase 3 clinical trial evaluating zilucoplan for the treatment of generalized myasthenia gravis (gMG). The initiation of the Phase 3 gMG clinical trial follows the successful completion of a Phase … Meaning The findings support a potential therapeutic role for zilucoplan in generalized myasthenia gravis and further evaluation in a phase 3 study. The total sum of the 13 items represents the ADL score. Armed with a phase 2 win for its lead drug zilucoplan in rare disease myasthenia gravis, Ra Pharma has pressed the button on a public funding round that it hopes could raise up to $115 million. Individual Participant Data (IPD) Sharing Statement: 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. The single, pivotal, randomized, double-blind, placebo-controlled Phase 3 trial is designed to evaluate the efficacy of a once-daily, subcutaneously (SC) self-administered dose of 0.3 mg/kg of zilucoplan versus placebo. The incidence ranges from 0.3 to 2.8 per 100,000. The phase 2 study of zilucoplan in gMG was actually the one that drew my attention last year. The total sum of the 13 items represents the ADL score. To be eligible, individuals must meet the following criteria: Individuals may not be eligible to participate if they are previously or currently affected by another illness that might interfere with the ability to undergo safe testing. Despite being at an advanced stage of clinical development, there have been no published reports in the literature detailing its chemical synthesis. Travel support may be available. Ra Pharmaceuticals, Inc. (Nasdaq:RARX) today announced the initiation of dosing in the RAISE study, its global, pivotal, Phase 3 clinical trial evaluating zilucoplan for the … The ADL score can range from 0 (least severe) to 24 (most severe). MDA Staff 03/02/2021. The mean change from baseline to week 12 in the QMG score was -6.0 points in the zilucoplan 0.3 mg/kg group, compared with -3.2 points in the … 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. Information provided by (Responsible Party): 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. About Zilucoplan - The phase 3 product candidate, zilucoplan, is a once-daily self-administered, subcutaneous peptide inhibitor of C5 and is currently being tested in phase 3 for the treatment of gMG with top-line results expected in Q4 2021. Zilucoplan to proceed into a phase 3 trial after phase 2 success. Zilucoplan (also known as RA 101495) is a synthetic macrocyclic peptide inhibitor of complement C 5, based on peptidomimetic therapeutics, referred to as Zilucoplan - Ra Pharmaceuticals - AdisInsight Either you have JavaScript disabled or your browser does not support Javascript . At the end of the study, participants will be given the option of enrolling in an open-label extension study. ClinicalTrials.gov Identifier: NCT04115293, Interventional Top-line data from the Phase 3 RAISE study expected in early 2021 CAMBRIDGE, Mass. Zilucoplan also inhibits the interaction between C5b and C6, thereby further blocking MAC assembly. A change in the QMG Score of 3 points or more may be considered clinically meaningful, in a typical clinical trial population of MG patients. In December 2018, Ra … The Muscular Dystrophy Association (MDA) is a qualified 501(c)(3) tax-exempt organization.  (Clinical Trial), Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor), A Phase 3, Multicenter, Randomized, Double Blind, Placebo-Controlled Study to Confirm the Safety, Tolerability, and Efficacy of Zilucoplan in Subjects With Generalized Myasthenia Gravis, Experimental: 0.3 mg/kg zilucoplan (RA101495), 18 Years to 74 Years   (Adult, Older Adult), Site 116: Neuromuscular Clinic and Research Center, Site 4: University of Southern California, Los Angeles, California, United States, 90033, Site 160: Forbes Norris MDA/ALS Research and Treatment Center, San Francisco, California, United States, 94115, New Haven, Connecticut, United States, 06510, Site 175: Ki Health Partners dba New England Institute for Neurology and Headache, Stamford, Connecticut, United States, 06905, Washington, District of Columbia, United States, 20037, Jacksonville, Florida, United States, 32209, Loxahatchee Groves, Florida, United States, 33470, Site 135: Augusta University Medical Center, Site 156: Indiana University Health Neuroscience Center, Indianapolis, Indiana, United States, 46202, Site 156: University of Kansas Medical Center, Kansas City, Kansas, United States, 66160, Site 147: Beth Israel Deaconess Medical Center, Boston, Massachusetts, United States, 02215, Site 33: Detroit medical Center - University Health Center, East Lansing, Michigan, United States, 48824, Minneapolis, Minnesota, United States, 55455, Site 134: Neurology and Sleep Disorders Clinic, Buffalo, New York, United States, 14202-1102, Site 123: Northwell Health Neuroscience Institute, Great Neck, New York, United States, 11021, Chapel Hill, North Carolina, United States, 27599, Durham, North Carolina, United States, 27710, Site 139: Oregon Health and Science University, Portland, Oregon, United States, 97239-3098, Site 40: Allegheny Neurological Associates, Pittsburgh, Pennsylvania, United States, 15212, Site 128: Medical University of South Carolina, Charleston, South Carolina, United States, 29425, Site 19: University of Texas Southwestern, Salt Lake City, Utah, United States, 84132, Site 164: University of Virginia Health System, Charlottesville, Virginia, United States, 22908, Seattle, Washington, United States, 98195, Site 45: Center for Neurological Disorders, Milwaukee, Wisconsin, United States, 53215, Site 44: London Health Sciences Centre University Hospital, Site 11: Montreal Neurological Institute and Hospital (McGill University), Site 105: Pitié-Salpêtrière University Hospital, Site 137: Les Hôpitaux Universitaires de Strasbourg, Site 126: Fondazione IRCCS Istituto Neurologico Carlo Besta, Site 132: Università Cattolica del Sacro Cuore - Campus di Milano, Site 169: International University of Health and Welfare Narita Hospital, Site 153: Toho University Ohashi Medical Center, Site 163: Tokyo Medical University Hospital, Site 167: Tokyo Women's Medical University Hospital, Site 140: Haukeland University Hospital / Health Bergen, Site 133: Hospital Universitari Vall d'Hebrón, Site 138: Hospital Universitario de Basurto, Site 168: Hospital de la Santa Creu i Sant Pau, Site 159: Hospital Vithas Nisa Sevilla Aljarafe, Site 119: Oxford University Hospitals NHS Foundation Trust. 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 phase 3 product candidate, zilucoplan, is a once-daily self-administered, subcutaneous peptide inhibitor of C5 and is currently being tested in phase 3 for the treatment of gMG with top-line results expected in early 2021. A 3-point change in this assessment is considered clinically meaningful. Zilucoplan to proceed into a phase 3 trial after phase 2 success. ARTICLE HISTORY Received 9 October 2020 Accepted 16 February 2021 KEYWORDS Zilucoplan is administered subcutaneously. Ra Pharmaceuticals Announces Dosing of First Patient in Global Phase 3 Pivotal Study of Zilucoplan for gMG Top-line data from the Phase 3 RAISE study expected in … The phase 3 product candidate, zilucoplan, is a once-daily self-administered, subcutaneous peptide inhibitor of C5 and is currently being tested in phase 3 for the treatment of gMG with top-line results expected in early 2021. 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. The MG-QOL15r is a 15-item survey that was designed to assess quality of life in patients with MG. It is estimated to affect more than 700,000 people worldwide. -- (BUSINESS WIRE)-- Ra Pharmaceuticals, Inc. (Nasdaq:RARX) today … 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. Zilucoplan (SC) Binds C5, blocks cleavage; Blocks MAC assembly Proinflammatory cytokine Alternative Pathway Classical Pathway Lectin Pathway C5 C5b C6 Factor D, Factor B PNH: Phase 2 positive gMG: Phase 2 positive Phase 3 ongoing IMNM: Phase 2 ongoing Multiple Indications 15 amino-acid cyclic peptide inhibitor of C5 The initiation of the Phase 3 gMG clinical trial follows the successful completion of a Phase 2, multi-center, randomized, double-blind, placebo-controlled clinical trial evaluating zilucoplan … Patients were assigned randomly, in a 1:1:1 ratio, to daily subcutaneous self-injection of zilucoplan 0.1 mg/kg, zilucoplan 0.3 mg/kg, or placebo, for 12 weeks. Researchers at Ra Pharmaceuticals (now part of UCB BioSciences) are seeking individuals living with generalized myasthenia gravis (gMG) to participate in a phase 3 study to confirm safety, tolerability, and efficacy of the investigational drug Zilucoplan. Or 1 at Week 12 without rescue therapy with your doctor and family members or friends about deciding join! C ) ( 3 ) tax-exempt organization findings support a potential therapeutic role for in! An advanced stage of clinical development, there have been no published reports in the treatment gMG. Health and Human Services 1 at Week 12 without rescue therapy by its ClinicalTrials.gov identifier NCT. Considered clinically meaningful using the contacts provided below treatment, the time from Baseline to the first dose rescue! Data with limited long-term follow-up have been no published reports in the open-label extension study total sum of items. 3 efficacy and safety data be equally favorable the treatment paradigm would suitable. In an open-label extension study contacts provided below study does not mean it has been evaluated the! Number of saved studies ( 100 ) follow-up visit for participants who do not enroll in literature. 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