Clinical trial · Interventional
Zilucoplan for Severe gMG Exacerbations
Zilucoplan Treatment of Severe MG Exacerbations Leading to Hospitalization of Participants With Acetylcholine Receptor Antibody Positive gMG
NCT07215949CI-TRIAL-00124295recruitingPhase 3ClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
This is an open-label, multicenter, interventional phase 3b study in participants with AChR+ gMG and severe exacerbation that require hospitalization. Patients will receive subcutaneous zilucoplan injections daily for 12 weeks. Participation in the study will last for approximately 18 weeks.
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Generalized Myasthenia Gravis (gMG) | — | UNRESOLVED | — |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Zilucoplan® | Drug | — | UNRESOLVED |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- Zilucoplan
- description
- Subcutaneous zilucoplan
- interventionNames
- Drug: Zilucoplan®
Primary outcomes (1)
- measure
- Change from baseline to Week 2 in the Myasthenia Gravis - Activities of Daily Living (MG-ADL) score
- timeFrame
- From baseline to Week 2
- description
- The MG-ADL is an assessment that measures MG symptom severity. There are 8 items measured on a scale of 0-3 with 0 being the least severe. The total of the 8 items represents the MG-ADL score. The score can range from 0 (least severe) to 24 (most severe).
Secondary outcomes (1)
- measure
- Change from baseline to week 12 in Myasthenia Gravis - Activities of Daily Living (MG-ADL) score
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
Show eligibility criteria text
Inclusion Criteria: * Patient determined to have severe MG exacerbation (e.g. bulbar and/or respiratory symptoms requiring hospitalization, neck extension weakness) * MGFA class II - IVb * Male or female aged ≥18 * MG-ADL ≥6 in non-ocular domains * Serology - AChR antibody positive (or historically available data) * If female of child-bearing potential (i.e., not surgically sterile or post-menopausal defined as age \> 51 years without menses for ≥ 2 years), negative serum pregnancy test at screening * Women of child-bearing potential or men with sexual partners of childbearing potential must be willing to use an acceptable method of birth control for the duration of the study and for 40 days after the last dose of study drug therapy. Acceptable methods of birth control include abstinence, oral contraceptives, the contraceptive patch, intra-uterine device, the contraceptive ring, and or barrier contraception such as condoms with spermicide. * Completed or updated meningococcal vaccination or initiated meningococcal vaccination with appropriate antibiotic prophylaxis according to current USPI and ACIP guidelines Exclusion Criteria: * History of meningococcal disease * Participants requiring intubation prior to study start. * Recent significant infections which could have caused exacerbation e.g. sepsis and wound infections * Pregnancy or lactating * Recent surgery (\<4 weeks). Minor procedures/surgeries allowed at the discretion of the site principal investigator * Current use or known failure of C5 inhibitors in the previous 3 months * Initiation of plasma exchange or IVIG in the past 4 weeks * Participation in concurrent clinical trial with a therapeutic medication * Rituximab use in the previous 9 months * Any clinically significant condition or illness, which, in the opinion of the PI, would pose a risk to the subject or might confound the study
References
Publications (12)
- BACKGROUNDVu T, Meisel A, Mantegazza R, Annane D, Katsuno M, Aguzzi R, Enayetallah A, Beasley KN, Rampal N, Howard JF. Terminal Complement Inhibitor Ravulizumab in Generalized Myasthenia Gravis. NEJM Evid. 2022 May;1(5):EVIDoa2100066. doi: 10.1056/EVIDoa2100066. Epub 2022 Apr 26. PMID 38319212
- BACKGROUNDHoward 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. PMID 37059508
- BACKGROUNDKusner LL, Kaminski HJ. The role of complement in experimental autoimmune myasthenia gravis. Ann N Y Acad Sci. 2012 Dec;1274(1):127-32. doi: 10.1111/j.1749-6632.2012.06783.x. PMID 23252907
- BACKGROUNDKapoor M, Spillane J, Englezou C, Sarri-Gonzalez S, Bell R, Rossor A, Manji H, Reilly MM, Lunn MP, Carr A. Thromboembolic risk with IVIg: Incidence and risk factors in patients with inflammatory neuropathy. Neurology. 2020 Feb 11;94(6):e635-e638. doi: 10.1212/WNL.0000000000008742. Epub 2019 Dec 18. PMID 31852814
- BACKGROUNDMandawat A, Kaminski HJ, Cutter G, Katirji B, Alshekhlee A. Comparative analysis of therapeutic options used for myasthenia gravis. Ann Neurol. 2010 Dec;68(6):797-805. doi: 10.1002/ana.22139. PMID 21061395
- BACKGROUNDChang CC, Yeh JH, Chiu HC, Liu TC, Chen YM, Jhou MJ, Lu CJ. Assessing the length of hospital stay for patients with myasthenia gravis based on the data mining MARS approach. Front Neurol. 2023 Dec 14;14:1283214. doi: 10.3389/fneur.2023.1283214. eCollection 2023. PMID 38156090