[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"conditionNormalized\":\"generalized-myasthenia-gravis\",\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:generalized-myasthenia-gravis":28},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,25,0,[8,47,74,107,133,156,178,209,232,247,270,295,317,341,372,401,423,445,475,505,533,552,576,597,624],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":26,"conditions":27,"keywords":29,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":35,"lastUpdatePostDateStruct":36,"startDateStruct":39,"completionDateStruct":41,"leadSponsor":43,"locationsCount":46},"100570209","phase-1-study-to-assess-safety-efficacy-and-cellular-kinetics-of-ytb323-in-generalized-myasthenia-gravis-100570209",false,"NCT06704269","Study to Assess Safety, Efficacy, and Cellular Kinetics of YTB323 in Generalized Myasthenia Gravis","An Open-label, Multi-center, Phase I\u002FII Study to Assess Safety, Efficacy, and Cellular Kinetics of YTB323 in Participants With Treatment-resistant Generalized Myasthenia Gravis","Inclusion Criteria:\n\n1. Confirmed gMG diagnosis supported by the following:\n\n   * Documented report of positive serology testing for either AChR antibodies or MuSK antibodies at screening AND at least one of the following:\n   * History of abnormal neuromuscular transmission test demonstrated by repetitive nerve stimulation or single-fiber electromyography\n   * History of positive acetylcholinesterase inhibitor test\n   * Improvement in MG signs on an oral acetylcholinesterase inhibitor as assessed by the treating physician\n2. MGFA Class III-IVa (gMG) at screening\n3. Treatment-resistant gMG as defined by: MG-ADL score ≥ 6 (≥50% non-ocular) at screening despite adequate treatment trials with at least two different non-steroidal immunosuppressive drugs given at adequate doses and duration of therapy.\n4. If on chronic corticosteroids, must be on a stable dose of corticosteroids for ≥1 month prior to screening and have the ability and willingness to taper to a maximum dose of 10 mg prednisolone daily or equivalent at least one week before leukapheresis\n5. If treated with cholinesterase inhibitors, patients must be on a stable dose for at least two weeks prior to screening\n\nExclusion Criteria:\n\n1. Exclusively ocular myasthenia gravis (MGFA I), mild symptoms (MGFA II), or severe bulbar disease or MG crisis, MGFA Class IVb or V at screening\n2. History of bone marrow\u002Fhematopoietic stem cell or solid organ transplantation.\n3. Clinically significant active, opportunistic, chronic or recurrent infection (including positive for hepatitis B or hepatitis C) confirmed by clinical evidence, imaging, or positive laboratory tests one month prior to leukapheresis\n4. Other uncontrolled disease states, such as asthma, or inflammatory bowel disease, where flares are commonly treated with oral or parenteral corticosteroids, at screening\n5. Participants with a known immunodeficiency syndrome (AIDS, hereditary immune deficiency, drug induced immune deficiency), or tested positive for HIV antibody, at screening\n6. Prior treatment with anti-CD19 therapy, adoptive T cell therapy or any prior gene therapy product (e.g. CAR-T cell therapy).\n\nOther protocol-defined inclusion\u002Fexclusion criteria may apply","ALL","18 Years","65 Years",{"count":20,"type":21},15,"ESTIMATED","INTERVENTIONAL",[24,25],"PHASE1","PHASE2","This is a phase I\u002FII study to assess safety, efficacy, and cellular kinetics of YTB323 in participants with treatment-resistant generalized myasthenia gravis. YTB323 is a Biological CAR-T cell therapy.",[28],"Generalized Myasthenia Gravis",[30,31,32,33],"myasthenia gravis","generalized myasthenia gravis","YTB323","CAR-T","RECRUITING","2026-06-25",{"date":37,"type":38},"2026-06-26","ACTUAL",{"date":40,"type":38},"2025-04-22",{"date":42,"type":21},"2029-10-26",{"name":44,"class":45},"Novartis Pharmaceuticals","INDUSTRY",11,{"id":48,"slug":49,"hasResults":11,"nctId":50,"briefTitle":51,"officialTitle":52,"acronym":4,"eligibilityCriteria":53,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":54,"targetDuration":4,"studyType":22,"phases":56,"briefSummary":58,"conditions":59,"keywords":60,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":35,"lastUpdatePostDateStruct":65,"startDateStruct":67,"completionDateStruct":69,"leadSponsor":71,"locationsCount":73},"100551707","phase-3-efficacy-and-safety-of-a-new-formulation-of-oral-cladribine-compared-with-placebo-in-participants-with-generalized-myasthenia-gravis-myclad-100551707","NCT06463587","Efficacy and Safety of a New Formulation of Oral Cladribine Compared With Placebo in Participants With Generalized Myasthenia Gravis (MyClad)","A Phase 3, Randomized, Double-Blind, Placebo-Controlled, 3-Arm, 3-Period Study to Assess the Efficacy and Safety of a New Formulation of Oral Cladribine Compared With Placebo in Participants With Generalized Myasthenia Gravis (MyClad)","Inclusion Criteria:\n\n* Adults of ≥ 18 years of age at the time of signing the informed consent.\n* Diagnosis of Myasthenia Gravis with generalized muscle weakness, meeting clinical criteria for Myasthenia Gravis Foundation of America Class II to IVa classification.\n\n  * In participants positive for Acetylcholine receptor antibody (anti-AChR) or muscle-specific kinase antibody(anti-MuSK)\n  * In participants that are autoantibody seronegative i.e. not positive for anti-AChR and anti-MuSK antibodies and participants who are positive for anti-low-density lipoprotein receptor-related protein 4 antibodies (anti-LRP4)\n* Has a Screening and Baseline MG-ADL score more than or equal to (\\>=) 6 with \\>= 50 percentage (%) of the total score due to non-ocular symptoms. Screening and Baseline MG-ADL scores must be stable. The difference between the Screening and Baseline scores should not be more than 2 and there should be no reported MG exacerbation during the Screening period\n* If treated with oral corticosteroids: should be on a stable daily dose for at least 3 months prior to and during screening. In such case, the daily dose of oral steroids should not exceed 20 milligrams(mg)\u002Fday for prednisone\u002F prednisolone, 16 mg\u002Fday for methylprednisolone, 3 mg\u002Fday for dexamethasone, or 80 mg for hydrocortisone or equivalent doses for other corticosteroids.\n* If treated with acetylcholinesterase inhibitor should be on a stable daily dose (pyridostigmine dose ≤ 480 mg\u002Fday or neostigmine ≤ 300 mg\u002Fday) for at least 3 months prior to and during screening\n* Have a body weight \\>= 40 kilograms\n* Other protocol defined inclusion criteria could apply\n\nExclusion Criteria:\n\n* Immunologic disorder other than MG or any other condition requiring chronic oral, intravenous, intramuscular, or intraarticular corticosteroid therapy. Well-controlled thyroid disease, as per the Treating Investigator or the participants regular treating physician recorded in the source documents, is not exclusionary\n* Molecularly characterized or suspected congenital myasthenic syndrome, Lambert-Eaton myasthenic syndrome, inherited myopathy, muscular dystrophy, acquired myopathy or any other neurologic or systematic disease that mimics MG muscular weakness\n* Active, clinically significant viral, bacterial, or fungal infection, including brain MRI or chest X-ray findings consistent with signs of infection such as PML or TB, or any major episode of infection requiring hospitalization or treatment with parenteral anti-infectives within 8 weeks prior or during Screening, or completion of oral anti-infectives within 8 weeks prior or during Screening. Vaginal candidiasis, onychomycosis, and genital or oral herpes simplex virus considered by the Investigator to be sufficiently controlled would not be exclusionary\n* Has a history of or current diagnosis of active tuberculosis (TB) or is currently undergoing treatment for latent TB infection or has an untreated latent TB infection as determined by documented results within 3 months of the Screening Visit of a positive TB skin test .\n* Active malignancy, or history of cancer or signs of malignancy in any Screening assessment\n* Treatment with nonsteroidal immunosuppressants, used in gMG, such as azathioprine, mycophenolate mofetil, methotrexate, cyclosporine, cyclophosphamide, tacrolimus within 4 weeks prior to randomization\n* Treatment with FcRn or complement inhibitors (such as eculizumab, rozanolixizumab efgartigimod, ravulizumab, zilucoplan or nipocalimab) within 8 weeks prior to randomization\n* History of thymectomy within 6 months prior to Screening.\n* History of generalized seizures (except for history of febrile seizures during the participant's childhood).\n* Negative or indeterminate for Varicella Zoster Virus antibodies at screening\n* History of myasthenic crisis in the last 12 months prior to and during screening\n* History of recurrent infections (that is 3 or more infections per year documented in available source data) within the last 2 years\n* Discontinuation of treatment with any non-steroidal immunosuppressants used in gMG, such as azathioprine, mycophenolate mofetil, methotrexate, cyclosporine, cyclophosphamide, tacrolimus within the last 6 months prior to Screening\n* If treated with non-steroidal immunosuppressants for gMG, the dose at Screening higher than 50 mg\u002Fday for azathioprine, 500 mg\u002Fday for mycophenolate mofetil, 1 mg\u002Fday for tacrolimus, 50 mg\u002Fday for cyclosporine, 25 mg\u002Fday for cyclophosphamide, or 7.5 mg\u002Fweek for methotrexate\n* Participation in clinical study of any investigational drug within 6 months, or 5 half-lives of the investigational drug used in the previous clinical study prior to randomization, whichever is longer. However, participants with any prior exposure to cladribine may not enter the study regardless of timing of exposure\n* Other protocol defined exclusion criteria could apply",{"count":55,"type":21},264,[57],"PHASE3","The purpose of this clinical study is to determine the efficacy and safety of a new oral cladribine formulation in participants with Generalized Myasthenia Gravis (gMG) in comparison to placebo. It will also investigate the sustained efficacy, the need for retreatment, and the long-term safety of oral cladribine in gMG. An additional component is included to characterize the Pharmacokinetics (PK) of the new cladribine formulation in gMG participants. This study is divided into 3 periods: the double-blind placebo control (DBPC) pivotal period, and 2 extensions, the blinded extension (BE) and the retreatment (RT) period. Furthermore, in trial interviews will be conducted as a sub-study to MyClad with a sub-set of participants to gain an in depth understanding of the participant cladribine treatment and study experience.",[28],[61,62,63,64],"Anti-AChR antibody positive","anti-MuSK antibody positive","anti-LRP4 antibody positive","seronegative gMG",{"date":66,"type":38},"2026-06-29",{"date":68,"type":38},"2024-06-25",{"date":70,"type":21},"2030-11-12",{"name":72,"class":45},"Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, Germany",144,{"id":75,"slug":76,"hasResults":11,"nctId":77,"briefTitle":78,"officialTitle":79,"acronym":4,"eligibilityCriteria":80,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":81,"enrollmentInfo":82,"targetDuration":4,"studyType":22,"phases":84,"briefSummary":85,"conditions":86,"keywords":87,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":98,"lastUpdatePostDateStruct":99,"startDateStruct":101,"completionDateStruct":103,"leadSponsor":105,"locationsCount":106},"100555874","phase-3-a-phase-iii-study-to-investigate-efficacy-safety-and-tolerability-of-iptacopan-compared-with-placebo-in-participants-aged-18-to-85-years-with-gmg-100555874","NCT06517758","A Phase III Study to Investigate Efficacy, Safety and Tolerability of Iptacopan Compared With Placebo in Participants Aged 18 to 85 Years With gMG.","A Randomized, Double-blind, Placebo-controlled Phase III Study to Evaluate the Efficacy, Safety, and Tolerability of Iptacopan in Patients With Generalized Myasthenia Gravis, Followed by an Open-label Extension Phase","Inclusion Criteria:\n\n* Adult patients with generalized Myasthenia Gravis (age 18-85 years) at screening\n* Positive serology testing for AChR+ antibody at screening\n* Myasthenia Gravis Foundation of America (MGFA) Class II-IV gMG at screening and likely not in need of a respirator for the duration of the study, as judged by the Investigator.\n* The confirmation of the diagnosis of gMG should be documented and supported by ≥1 of the following 3 tests:\n* History of abnormal neuromuscular transmission demonstrated by single-fiber electromyography or repetitive nerve stimulation.\n* History of positive test with short-acting acetylcholinesterase inhibitors (e.g. neostigmine or edrophonium chloride)\n* Patient has demonstrated improvement in MG signs on oral acetylcholinesterase inhibitors as assessed by the treating physician.\n* Baseline MG-ADL score ≥6, with ≥50% of the total score due to non-ocular symptoms\n* Participants receiving at least one of the following treatments for gMG for ≥ 6 months prior to baseline;\n* One or more NSISTs or\n* plasmapheresis, plasma exchange, or intravenous immunoglobulin (at least quarterly) to control symptoms despite treatment with steroids and NSISTs; or\n* an approved FcRN antagonist approved for gMG; or\n* rituximab or\n* other approved gMG disease modifying therapies excluding complement inhibitors.\n* Vaccination against Neisseria meningitidis and Streptococcus pneumoniae infection is required prior to the start of study treatment. If the participant has not been previously vaccinated, or if a booster was required, the vaccine should be given according to local guidelines at least 2 weeks prior to first study drug administration. If study treatment has to start earlier than 2 weeks post-vaccination, prophylactic antibiotic treatment should be initiated at the start of study treatment and continued until at least 2 weeks after vaccination or booster was completed.\n\nNote: For US sites participating in Study CLNP023Q12301, the completion of the meningococcal vaccination or booster is required for patients with gMG prior to initiating study treatment, irrespective of prophylactic antibiotic use.\n\nExclusion Criteria:\n\n* Have been treated with intravenous immunoglobulin (IVIG)\u002Fplasma exchange (PLEX) in the past month, with rituximab in the past 6 months, eculizumab in the past 2 months, ravulizumab or other complement inhibitors in the past 3 months, efgartigimod or other anti- FcRn therapies in the past 3 months, or had a thymectomy in the past 6 months or a planned thymectomy during the trial period.\n* Participants with clinically significant active or chronic uncontrolled bacterial, viral, or fungal infection at screening, including patients who test positive for an active viral infection at screening with: Active Hepatitis B Virus (HBV); Active Hepatitis C Virus (HCV);\n\n  * Human Immunodeficiency Virus (HIV) positive serology associated with an Acquired Immune Deficiency Syndrome (AIDS)-defining condition or with a cluster of differentiation 4 (CD4) count\n\n    * 200 cells\u002Fmm3\n* Female participants who are pregnant or lactating, or are intending to become pregnant.\n* Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant from menarche until becoming post-menopausal, unless they are using effective methods of contraception during dosing of study treatment and an additional one week following cessation of study treatment. Women are considered post-menopausal if they have had 12 months of natural (spontaneous) amenorrhea with an appropriate clinical profile (e.g., hormonal profile confirming menopause and\u002For age-appropriate history of vasomotor symptoms).\n* Active systemic bacterial, viral (including COVID-19) or fungal infection or any major episode of infection that required hospitalization or injectable antimicrobial therapy within 14 days prior to study drug administration.\n* History of recurrent invasive infections caused by encapsulated organisms, e.g., N. meningitidis and S. pneumoniae.\n* Presence of fever ≥ 38 °C (100.4 °F) within 7 days prior to study drug administration","85 Years",{"count":83,"type":21},146,[57],"The study is a randomized, double-blind, placebo-controlled, multicenter, Phase III study, to evaluate efficacy, safety and tolerability of iptacopan in patients with AChR+ gMG who are on stable SOC treatment. Participants who meet the eligibility criteria will be randomized in a ratio of 1:1, to receive either iptacopan or matching placebo, for 6 months (180 days) while continuing on a stable SOC treatment. The randomization will be stratified based on region.",[28],[88,89,90,91,92,93,94,95,96,97],"Myasthenia Gravis","gMG","randomized","placebo-controlled","double-blind","phase III","AChR+","SOC treatment","iptacopan","LNP023","2026-06-19",{"date":100,"type":38},"2026-06-23",{"date":102,"type":38},"2024-07-31",{"date":104,"type":21},"2032-05-27",{"name":44,"class":45},125,{"id":108,"slug":109,"hasResults":11,"nctId":110,"briefTitle":111,"officialTitle":112,"acronym":4,"eligibilityCriteria":113,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":114,"enrollmentInfo":115,"targetDuration":4,"studyType":22,"phases":117,"briefSummary":118,"conditions":119,"keywords":120,"overallStatus":122,"whyStopped":4,"lastUpdateSubmitDate":123,"lastUpdatePostDateStruct":124,"startDateStruct":126,"completionDateStruct":128,"leadSponsor":130,"locationsCount":132},"100641863","phase-3-efficacy-and-safety-of-ravulizumab-in-chinese-adults-participants-with-generalized-myasthenia-gravis-gmg-100641863","NCT07596784","Efficacy and Safety of Ravulizumab in Chinese Adults Participants With Generalized Myasthenia Gravis (gMG)","An Open-label, Single-arm, Multi-center, Interventional Study to Evaluate the Efficacy, Safety, Pharmacokinetics, Pharmacodynamics, and Immunogenicity of Ravulizumab in Chinese Adult Patients With Generalized Myasthenia Gravis (gMG)","Inclusion (key)\n\n* Confirmed generalized MG: Diagnosis ≥6 months before screening, anti-AChR antibody positive, and supportive diagnostic evidence (e.g., abnormal SFEMG\u002FRNS or response to anticholinesterase therapy).\n* Disease severity: MGFA Class II-IV at screening.\n* Symptoms threshold: MG-ADL ≥6 at screening and on Day 1.\n* Meningococcal vaccination: Up to date within 3 years or vaccinated before first dose to mitigate risk with complement inhibition.\n* Body weight: ≥40 kg.\n* Vaccinated against meningococcal infections within the 3 years prior to, or at the time of, initiating study drug.\n\nExclusion (key)\n\n* Thymic disease:\n\n  * Untreated thymic malignancy\u002Fcarcinoma\u002Fthymoma excluded.\n  * Prior thymic malignancy allowed only if treatment completed \\>5 years, no recurrence in last 5 years, and clear CT\u002FMRI within 6 months.\n  * Prior benign thymoma allowed if confirmed benign, treatment \\>12 months ago, no recurrence in last 12 months, and clear CT\u002FMRI within 6 months; otherwise follow malignancy rules.\n  * Thymectomy within the last 12 months\n* Infection risk:\n\n  * History of meningococcal disease or unresolved infection, or active systemic infection within 14 days of Day 1 excluded.\n  * Persistent\u002Frecurrent infections in past 12 months that add risk\n  * HIV, active HBV (HBsAg+ or anti-HBc+ with anti-HBs-), or active HCV (unless documented successful treatment\u002FSVR)\n* Safety\u002Fmedical status:\n\n  * Hypersensitivity to study drug components (including murine proteins)\n  * Recent hospitalization ≥24 hours within 28 days of screening\n  * Substance use disorder per DSM within 12 months.\n  * Recent\u002Fother malignancy within 5 years (except as above for thymic).\n* Prior\u002FConcomitant Therapy\n\n  * Complement inhibitor within \\\u003C 5 half-lives before Day 1.\n  * Human neonatal Fc receptor (FcRn) inhibitor within \\\u003C 5 half-lives before Day 1.\n  * Rituximab, ocrelizumab or other B cell-depleting therapy within ≤ 6 months (180 days) before Day 1.\n  * Periodic (chronic) administration of PP\u002FPE, or IVIg as maintenance therapy received or scheduled within ≤ 6 months before Day 1\n* Key labs:\n\n  * ALT \\>2× ULN, direct bilirubin \\>2× ULN.\n  * eGFR \\\u003C30 mL\u002Fmin\u002F1.73 m² or on dialysis.\n  * Any other clinically significant lab abnormality making participation unsafe.\n\nNote: Other protocol-defined criteria may apply and should be verified during full eligibility review.","130 Years",{"count":116,"type":21},20,[57],"The primary purpose of this study is to evaluate the efficacy, safety, pharmacokinetics, pharmacodynamics, and immunogenicity of ravulizumab in Chinese adult participants with Acetylcholine receptor (AChR) + Generalized Myasthenia Gravis (gMG).",[28,89],[31,89,121],"ravulizumab","NOT_YET_RECRUITING","2026-06-17",{"date":125,"type":38},"2026-06-22",{"date":127,"type":21},"2026-07-30",{"date":129,"type":21},"2027-08-27",{"name":131,"class":45},"Alexion Pharmaceuticals, Inc.",10,{"id":134,"slug":135,"hasResults":11,"nctId":136,"briefTitle":137,"officialTitle":138,"acronym":4,"eligibilityCriteria":139,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":140,"targetDuration":4,"studyType":22,"phases":142,"briefSummary":143,"conditions":144,"keywords":145,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":146,"lastUpdatePostDateStruct":147,"startDateStruct":149,"completionDateStruct":151,"leadSponsor":153,"locationsCount":155},"100551168","phase-3-a-study-of-telitacicept-for-the-treatment-of-generalized-myasthenia-gravis-upstream-mg-100551168","NCT06456580","A Study of Telitacicept for the Treatment of Generalized Myasthenia Gravis (UPSTREAM MG)","A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study With an Open-label Extension Period to Evaluate the Efficacy and Safety of Telitacicept in Patients With Generalized Myasthenia Gravis","Key Inclusion Criteria:\n\n1. Male or female patient aged ≥18 years at screening.\n2. Patients have prior confirmed diagnosis of gMG with generalized muscle weakness (typical pattern of weakness meeting the clinical criteria for diagnosis of MG as defined by the Myasthenia Gravis Foundation of America (MGFA) clinical classification II-IV.\n3. Patients have positive antibodies against AChR or MuSK at screening.\n4. MG-ADL score ≥6 points at screening and baseline with ocular-related score \\\u003C50% of the total score.\n5. QMG score ≥8 points, and ≥ 4 items score at least 2 points at screening and baseline.\n\nKey Exclusion Criteria:\n\n1. Patients have been diagnosed with any other autoimmune disease which can potentially pose a safety or efficacy confounding risk.\n2. Patients having acute or chronic infection.\n3. Patients having thymoma within 5 years or received thymectomy ≤6 months prior to screening. Patients with thymoma diagnosed 3-5 years prior to screening may be eligible if thymoma was at a localized stage and definitively treated with complete surgical resection.\n4. Patients having current or history of primary immunodeficiency.\n5. Patients having history of malignancy within the last 5 years.\n6. Patient having prior or continuing diagnosis of serious cardiovascular, liver, kidney, respiratory system, endocrine or hematologic disease.",{"count":141,"type":21},180,[57],"The purpose of this study is to evaluate the efficacy and safety of telitacicept in the treatment of generalized myasthenia gravis.",[28],[88,89],"2026-06-16",{"date":148,"type":38},"2026-06-18",{"date":150,"type":38},"2024-07-17",{"date":152,"type":21},"2029-01",{"name":154,"class":45},"Vor Biopharma",111,{"id":157,"slug":158,"hasResults":11,"nctId":159,"briefTitle":160,"officialTitle":161,"acronym":162,"eligibilityCriteria":163,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":164,"targetDuration":4,"studyType":166,"phases":4,"briefSummary":167,"conditions":168,"keywords":4,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":169,"lastUpdatePostDateStruct":170,"startDateStruct":171,"completionDateStruct":172,"leadSponsor":174,"locationsCount":177},"100636815","monitor-the-evolution-of-myasthenia-gravis-symptoms-in-real-life-in-patients-with-anti-achr-and-anti-musk-generalised-myasthenia-gravis-in-therapy-with-rystiggo-rozanolixizumab-100636815","NCT07570589","Monitor the Evolution of Myasthenia Gravis Symptoms in Real-life in Patients With Anti-AChR and Anti-MUSK Generalised Myasthenia Gravis in Therapy With RYSTIGGO® (Rozanolixizumab)","An Observational, Prospective, Multicenter, Open-label Study to Monitor the Evolution of Myasthenia Gravis Symptoms in Real-life in Patients With Anti-AChR and Anti-MUSK Generalised Myasthenia Gravis in Therapy With RYSTIGGO® (Rozanolixizumab)","REAL MG PRO","Inclusion Criteria:\n\n* Male or female subjects aged ≥18 years;\n* Diagnosed with MG with confirmed documentation and supported by a physical exam and confirmed seropositivity for anti AChR or anti MUSK antibodies;\n* Meets the clinical criteria as defined by the Myasthenia Gravis Foundation of America (MFGA) for generalized MG class IIa, IIb, IIIa, and IIIb;\n* Patient suitable for ROZ treatment (IgG ≥ 5,5 g\u002Fl);\n* Patient naïve for ROZ treatment, or receiving the last dose more than 3 months prior inclusion;\n* MG-ADL score ≥ 3 (with ≥ 3 points of non-ocular symptoms);\n* Patient must have received all mandatory vaccinations according to local regulations prior to study enrollment;\n* Capable of understanding the written informed consent, and providing signed, dated, and witnessed written informed consent;\n* Willing and able to comply with scheduled visits, treatment plan, study restrictions, laboratory tests, contraceptive guidelines, and other study procedures;\n* Patient affiliated to a European social security system.\n\nExclusion Criteria:\n\n* Any current mental condition (psychiatric disorder, senility, or dementia) that, in the opinion of the investigator, may affect study compliance or prevent understanding of the aims, investigational procedures, or possible consequences of the study; and\u002For patient with a Mini-mental State evaluation (MMSE) of \\\u003C25;\n* History of any illness or any clinical condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject;\n* History of ongoing malignancy (except basal cell and squamous cell carcinomas of the skin, or in situ carcinoma of the cervix uteri that have been completely excised and cured);\n* Clinically significant uncontrolled active or chronic bacterial, viral, or fungal infection at inclusion;\n* Patient receiving concomitant treatment with rituximab, anti-FcRn, complement inhibitors and\u002For PLEX;\n* Patients discontinued from rituximab without respecting the administration rules of ROZ;\n* Patient with end-stage diseases or with a disease that will enable him to be evaluated and\u002For treated;\n* Patients with non-treated active infections;\n* Patients with hypersensitivity to the active substance(s) or to any of the excipients, specifically: histidine, histidine hydrochloride monohydrate, proline, polysorbate 80, or water for injections.\n* Subject, or close relative of the subject, is the investigator or a sub-investigator, research assistant, pharmacist, study coordinator, or other staff directly involved with the conduct of the study at that site;\n* Patient protected by law, under guardianship or curator ship, or not able to participate in a clinical study according to the article L.1121-16 of the French Public Health Code",{"count":165,"type":21},50,"OBSERVATIONAL","Generalized Myasthenia Gravis (gMG) is a rare autoimmune disease (a disease in which the body attacks its own tissues) that causes muscle weakness and significant fatigue.\n\nCurrent treatments (corticosteroids, plasma exchange, intravenous immunoglobulin infusions) improve symptoms in many patients. However, many continue to suffer from fatigue and fatigability that are not well measured by standard tools. Moreover, these treatments can cause significant long-term side effects, reducing quality of life.\n\nNew treatments such as Rozanolixizumab (ROZ) are now available. They act rapidly and are well tolerated, allowing better symptom control while reducing the risks associated with conventional treatments.\n\nTo properly evaluate these new treatments, it is essential to understand patients' perspectives on their effectiveness. The scales used by physicians do not always capture all the symptoms experienced by patients, particularly fatigability. This is why a new tool has been developed: the MG symptoms PRO. This questionnaire allows patients to assess their own symptoms (fatigue, weakness of the eyes, mouth, breathing, muscle fatigability) in detail.\n\nThis research aims to better understand the effectiveness of treatments from the patients' perspective in order to improve their care.\n\nThe goal of the study is to evaluate the impact of Rozanolixizumab administration in real-world practice through the MG symptoms PRO questionnaire. This is an observational study, meaning that the medication is prescribed by the physician according to current regulations, and the study simply collects routine medical data during your follow-up, over a period of approximately 9 months.",[28],"2026-06-15",{"date":146,"type":38},{"date":169,"type":38},{"date":173,"type":21},"2028-03",{"name":175,"class":176},"Centre Hospitalier Universitaire de Nice","OTHER",1,{"id":179,"slug":180,"hasResults":11,"nctId":181,"briefTitle":182,"officialTitle":183,"acronym":4,"eligibilityCriteria":184,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":185,"enrollmentInfo":186,"targetDuration":4,"studyType":22,"phases":188,"briefSummary":189,"conditions":190,"keywords":191,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":200,"lastUpdatePostDateStruct":201,"startDateStruct":202,"completionDateStruct":204,"leadSponsor":206,"locationsCount":208},"100596012","phase-3-study-to-assess-the-efficacy-and-safety-of-imvt-1402-in-participants-with-mild-to-severe-generalized-myasthenia-gravis-100596012","NCT07039916","Study to Assess the Efficacy and Safety of IMVT-1402 in Participants With Mild to Severe Generalized Myasthenia Gravis","A Phase 3, Multicenter, Randomized, Placebo-Controlled, Double-Blind Study to Assess the Efficacy and Safety of IMVT-1402 in Patients With Mild to Severe Generalized Myasthenia Gravis","Inclusion Criteria:\n\n* Participants with the ability to understand the requirements of the trial, provide written informed consent, and comply with the trial protocol procedures.\n* Have mild to severe gMG by Myasthenia Gravis Foundation of America (MGFA) classification of Class II, III, or IVa at the Screening Visit\n* Have an MG activities of daily living (MG-ADL) score of ≥ 6 at the Screening Visit and Baseline Visit (Day 1)\n\nAdditional inclusion criteria are defined in the protocol.\n\nExclusion Criteria:\n\n* Have experienced myasthenic crisis within 12 weeks prior to the Screening Visit.\n* Have had a thymectomy performed \\\u003C 6 months prior to the Screening Visit or have a planned thymectomy during the study\n* Have any active or untreated malignant thymoma\n\nAdditional exclusion criteria are defined in the protocol.","80 Years",{"count":187,"type":21},231,[57],"The purpose of the study is to assess the efficacy, safety and tolerability of IMVT-1402 in adult participants with mild to severe generalized myasthenia gravis.",[28],[192,28,193,194,195,196,197,198,199],"IMVT-1402","Acetylcholine receptor","Muscle-specific kinase","Autoantibody","Lipoprotein receptor-related protein 4","Autoimmune disease","Neonatal fragment crystallizable receptor (FcRn)","Imeroprubart","2026-06-12",{"date":169,"type":38},{"date":203,"type":38},"2025-05-27",{"date":205,"type":21},"2028-12",{"name":207,"class":45},"Immunovant Sciences GmbH",84,{"id":210,"slug":211,"hasResults":11,"nctId":212,"briefTitle":213,"officialTitle":214,"acronym":4,"eligibilityCriteria":215,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":216,"targetDuration":4,"studyType":166,"phases":4,"briefSummary":218,"conditions":219,"keywords":4,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":223,"lastUpdatePostDateStruct":224,"startDateStruct":225,"completionDateStruct":227,"leadSponsor":229,"locationsCount":231},"100615558","adapt-forward---master-protocol-of-a-platform-study-to-evaluate-the-safety-and-efficacy-of-multiple-regimens-in-participants-with-myasthenia-gravis-100615558","NCT07294170","ADAPT Forward - Master Protocol of a Platform Study to Evaluate the Safety and Efficacy of Multiple Regimens in Participants With Myasthenia Gravis","A Master Protocol for an Exploratory, Phase 2a, Proof-of-Concept Platform Study to Evaluate the Safety, Tolerability, and Efficacy of Multiple Regimens in Participants With Myasthenia Gravis","Inclusion Criteria:\n\n* Is at least 18 years of age and the local legal age of consent for clinical studies\n* Has been diagnosed with MG with consistent clinical features per the investigator's clinical judgment\n* If receiving MG therapy, including nonsteroidal immunosuppressive drugs (NSIDs), corticosteroids, or acetylcholinesterase (AChE) inhibitors either in combination or alone, the participant should receive a stable dosage before master protocol screening\n\nExclusion Criteria:\n\n* Known autoimmune disease or any medical condition other than the indication under study that would interfere with an accurate assessment of clinical symptoms of MG or puts the participant at undue risk\n* Is MGFA (Myasthenia Gravis Foundation of America) Class V",{"count":217,"type":21},70,"ADAPT Forward is a platform study with the aim to look at how safe different drugs are and how well they work for people with myasthenia gravis. The goal is to find the best therapeutic approach to reduce patients' side effects and improve their quality of life.",[88,220,89,28,221,222],"MG","Generalized Myasthenia Gravis (gMG)","AChR-Ab Seropositive Generalized Myasthenia Gravis","2026-06-11",{"date":200,"type":38},{"date":226,"type":38},"2025-12-19",{"date":228,"type":21},"2028-03-07",{"name":230,"class":45},"argenx",18,{"id":233,"slug":234,"hasResults":11,"nctId":235,"briefTitle":236,"officialTitle":237,"acronym":4,"eligibilityCriteria":238,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":239,"targetDuration":4,"studyType":22,"phases":240,"briefSummary":241,"conditions":242,"keywords":4,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":223,"lastUpdatePostDateStruct":243,"startDateStruct":244,"completionDateStruct":245,"leadSponsor":246,"locationsCount":20},"100614809","phase-2-adapt-forward-1---isa1---a-study-to-evaluate-empasiprubart-iv-as-add-on-therapy-to-efgartigimod-iv-in-participants-with-achr-ab-seropositive-generalized-myasthenia-gravis-with-a-partial-clinical-response-to-efgartigimod-100614809","NCT07284420","ADAPT Forward 1 - ISA1 - a Study to Evaluate Empasiprubart IV as add-on Therapy to Efgartigimod IV in Participants With AChR-Ab Seropositive Generalized Myasthenia Gravis With a Partial Clinical Response to Efgartigimod","An ISA to Master Protocol ARGX-999-2-MG-2000 for an Exploratory, Phase 2a, Proof-of-Concept Study to Evaluate the Safety, Tolerability, and Efficacy of Empasiprubart IV as Add-On Therapy to Efgartigimod IV in Participants With AChR-Ab Seropositive Generalized Myasthenia Gravis With a Partial Clinical Response to Efgartigimod","Inclusion Criteria:\n\n* Is seropositive for anti-acetylcholine receptor antibodies (AChR-Ab)\n* Has confirmed diagnosis of gMG and is Myasthenia Gravis Foundation of America (MGFA) Class II, III, IVa, or IVb\n* Has documented immunization against encapsulated bacterial pathogens (Neisseria meningitidis and Streptococcus pneumoniae) within 5 years of ISA screening or is willing to receive immunization at least 14 days before the first study drug administration\n\nExclusion Criteria:\n\n* Clinical diagnosis of systemic lupus erythematosus (SLE)\n* Any known complement deficiency\n* Current administration of a complement inhibitor or received zilucoplan or eculizumab \\\u003C2 months or ravulizumab \\\u003C6 months before the first study drug administration\n* Patients proven to be refractory to efgartigimod (ie, not achieving a clinically meaningful improvement in total Myasthenia Gravis Activities of Daily Living (MG-ADL) score defined as an improvement of ≥2 points)",{"count":217,"type":21},[25],"This study is part of the ADAPT Forward platform study (NCT07294170). ADAPT Forward is a platform study with the aim to look at how safe different drugs are and how well they work for people with myasthenia gravis. The goal is to find the best therapeutic approach to reduce patients' side effects and improve their quality of life.\n\nThe aim of this ISA1 is to evaluate the safety and therapeutic relevance of empasiprubart as add-on therapy to efgartigimod in participants with AChR-Ab seropositive generalized myasthenia gravis.\n\nThe ADAPT Forward master protocol is registered on https:\u002F\u002Fclinicaltrials.gov\u002Fstudy\u002FNCT07294170",[222,88,220,89,28,221],{"date":200,"type":38},{"date":226,"type":38},{"date":228,"type":21},{"name":230,"class":45},{"id":248,"slug":249,"hasResults":11,"nctId":250,"briefTitle":251,"officialTitle":252,"acronym":253,"eligibilityCriteria":254,"healthyVolunteers":11,"sex":16,"minAge":255,"maxAge":256,"enrollmentInfo":257,"targetDuration":4,"studyType":22,"phases":259,"briefSummary":260,"conditions":261,"keywords":4,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":262,"lastUpdatePostDateStruct":263,"startDateStruct":265,"completionDateStruct":267,"leadSponsor":269,"locationsCount":116},"100546242","phase-2-a-study-of-efgartigimod-ph20-sc-in-children-between-2-and-less-than-18-years-of-age-with-generalized-myasthenia-gravis-100546242","NCT06392386","A Study of Efgartigimod PH20 SC in Children Between 2 and Less Than 18 Years of Age With Generalized Myasthenia Gravis","An Open-label, Uncontrolled Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, and Activity of Efgartigimod PH20 SC in Participants From 2 to Less Than 18 Years of Age With Generalized Myasthenia Gravis","ADAPT Jr SC","Inclusion Criteria:\n\n* The participant (and\u002For their legally authorized representative) understands the requirements of the study and is capable of providing written informed consent\u002Fassent and complying with protocol requirements\n* The participant is aged 2 to \\\u003C18 years at the time of informed consent\u002Fassent\n* The participant has been diagnosed with generalised Myasthenia Gravis that is supported by a physical examination and confirmed seropositivity for anti-acetylcholine receptor antibodies\n* The participant has had an unsatisfactory response to immunosuppressants, corticosteroids, or acetylcholinesterase inhibitors but is on stable concomitant MG therapy. If receiving corticosteroids and\u002For immunosuppressants, must be on a stable dose for ≥1 month before screening\n* The participant agrees to use birth control consistent with local regulations and people of child-bearing potential must have a negative blood pregnancy test at screening and a negative urine pregnancy test before receiving the study drug\n\nExclusion Criteria:\n\n* Is a female adolescent of child-bearing potential who is pregnant and\u002For lactating or intends to become pregnant during their participation in the study\n* Has worsening muscle weakness secondary to a concurrent infection or as a result of a medication\n* Has a documented lack of clinical response to plasma exchange (PLEX)\n* Received a live or live-attenuated vaccine within \\\u003C4 weeks before screening\n* Received a thymectomy within 3 months before screening or is planning to get a thymectomy during their participation in the study\n* Has a known autoimmune disease or any medical condition that would interfere with an accurate assessment of clinical symptoms of generalised Myasthenia Gravis or puts the participant at undue risk\n* History of malignancy, cancer, unless considered cured by adequate treatment with no evidence of recurrence for ≥3 years. Adequately treated participants with the following cancers can be included at any time: Basal cell or squamous cell skin cancer, Carcinoma in situ of the cervix, Carcinoma in situ of the breast, Incidental histological findings of prostate cancer\n* Clinically significant active infection that is not sufficiently resolved in the investigator's opinion or positive serum test at screening for active infection with any of the following: Hepatitis B virus (HBV), Hepatitis C virus (HCV), HIV\n* Has a positive PCR test for SARS-CoV-2 at screening\n* Has\u002Fhad a clinically significant disease, had recent major surgery (within 3 months of screening) or intends to have major surgery during the study, or has\u002Fhad any other medical condition that, in the investigator's opinion, would confound the results of the study or put the participant at undue risk\n* Has received a different study drug in another clinical study within \\\u003C12 before screening\n* Is currently participating in another interventional clinical study\n* Has previously participated in an efgartigimod clinical study and received at least one dose of study drug\n* Has a known hypersensitivity to study drug or any of its excipients\n* Has a history of or current episode of alcohol, drug, or medication abuse as assessed by the investigator\n* Use of some medications before screening (more information is found in the protocol)\n\nThe complete list of exclusion criteria can be found in the protocol.","2 Years","17 Years",{"count":258,"type":21},12,[25,57],"The purpose of this study is to measure the pharmacokinetics (PK), pharmacodynamics (PD), safety, tolerability, and immunogenicity of efgartigimod PH20 SC in pediatric participants with gMG aged 2 to \\\u003C18 years. The primary goal is to confirm an appropriate dose of efgartigimod PH20 SC for pediatric patients using PK and PD results from this study. Participants will receive injections of efgartigimod PH20 SC and will be monitored for safety until the end of the study. At the end of the follow-up period, eligible participants may roll over to an open-label extension (OLE) study.\n\nThe participants will be in the study for up to 14 weeks.",[28],"2026-06-02",{"date":264,"type":38},"2026-06-04",{"date":266,"type":38},"2024-06-28",{"date":268,"type":21},"2026-09-30",{"name":230,"class":45},{"id":271,"slug":272,"hasResults":11,"nctId":273,"briefTitle":274,"officialTitle":275,"acronym":276,"eligibilityCriteria":277,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":278,"enrollmentInfo":279,"targetDuration":4,"studyType":22,"phases":280,"briefSummary":281,"conditions":282,"keywords":283,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":286,"lastUpdatePostDateStruct":287,"startDateStruct":289,"completionDateStruct":291,"leadSponsor":293,"locationsCount":294},"100573333","phase-3-a-study-to-investigate-the-efficacy-safety-and-tolerability-of-remibrutinib-versus-placebo-in-adult-patients-with-generalized-myasthenia-gravis-100573333","NCT06744920","A Study to Investigate the Efficacy, Safety and Tolerability of Remibrutinib Versus Placebo in Adult Patients With Generalized Myasthenia Gravis","A Randomized, Double-blind, Placebo-controlled Phase III Study to Evaluate the Efficacy, Safety, and Tolerability of Remibrutinib in Patients With Generalized Myasthenia Gravis, Followed by an Open-label Extension Phase","RELIEVE","Inclusion Criteria:\n\n* Adult patients with gMG (age 18-75 years)\n* Confirmed diagnosis of Myasthenia Gravis Foundation of America (MGFA) Class II-IV gMG at screening and likely not in need of a respirator for the duration of the study, as judged by the Investigator\n* Documented evidence of positive serologic testing for AChR+ antibody or MuSK+ antibody at screening, OR seronegative for both AChR and MuSK antibodies at screening\n* Baseline MG-ADL score ≥ 6 with ≥ 50% of the total score due to non ocular symptoms\n* Participants who have been on a stable dose of standard-of-care treatment as specified in the protocol\n* Able to safely swallow the study medication according to investigator clinical judgement based on a bedside swallowing test or another formal swallowing test in line with local practice, both at Screening and Baseline\n\nExclusion Criteria:\n\n* Prior to baseline have been treated with intravenous immunoglobulins or plasma exchange (IVIg\u002FPLEX) in the past month, with rituximab in the past 6 months, eculizumab in the past 2 months, ravulizumab or other complement inhibitors in the past 3 months, efgartigimod or other anti-FcRn therapies in the past 3 months, or had a thymectomy in the past 6 months or a planned thymectomy during the trial period\n* Women of child bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception during dosing and for 1 week after stopping of study treatment\n\nOther protocol-defined inclusion\u002Fexclusion criteria may apply.","75 Years",{"count":141,"type":21},[57],"A study to evaluate the efficacy, safety and tolerability of Remibrutinib versus placebo in adult patients with Generalized Myasthenia Gravis who are on stable, standard-of-care (SOC) treatment.",[28],[88,89,28,284,285,91,92,93],"LOU064","Remibrutinib","2026-05-29",{"date":288,"type":38},"2026-06-01",{"date":290,"type":38},"2025-02-07",{"date":292,"type":21},"2033-02-26",{"name":44,"class":45},129,{"id":296,"slug":297,"hasResults":11,"nctId":298,"briefTitle":299,"officialTitle":300,"acronym":4,"eligibilityCriteria":301,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":278,"enrollmentInfo":302,"targetDuration":4,"studyType":22,"phases":304,"briefSummary":305,"conditions":306,"keywords":4,"overallStatus":122,"whyStopped":4,"lastUpdateSubmitDate":307,"lastUpdatePostDateStruct":308,"startDateStruct":310,"completionDateStruct":312,"leadSponsor":314,"locationsCount":316},"100638778","phase-2-a-phase-ii-study-of-shr-2173-injection-in-patients-with-myasthenia-gravis-100638778","NCT07622342","A Phase II Study of SHR-2173 Injection in Patients With Myasthenia Gravis","A Phase II, Randomized, Double-Blind, Placebo-Controlled Clinical Trial to Evaluate the Efficacy and Safety of SHR-2173 in Patients With Generalized Myasthenia Gravis","Inclusion Criteria:\n\n1. Male and female participants aged 18-75 years, confirmed diagnosis of generalized myasthenia gravis (gMG) (Myasthenia Gravis Foundation of America \\[MGFA\\] class II-IV).\n2. Positive for anti-AChR antibody or anti-MuSK antibody.\n3. MG-ADL total score ≥5 at screening and baseline, with \\>50% of the score attributable to non-ocular items.\n4. QMG score ≥11 at screening and baseline.\n5. Maintenance on stable standard of care (SOC) therapy.\n6. No contraindication to at least one rescue therapy: IVIg or PLEX.\n7. Provided written informed consent (ICF) after full understanding of the study content, procedures, and potential adverse reactions.\n8. Female subjects with fertility or male participants whose partners are women of childbearing age must avoid donating sperm\u002Feggs from the date of signing the ICF until 12 weeks after the last study medication, and agree to take contraceptive measures as specified in the protocol\n\nExclusion Criteria:\n\n1. Presence of any of the following medical histories or comorbidities:\n\n   1. Any untreated thymic epithelial tumor, mediastinal germ cell tumor, or other malignant mediastinal mass; or any thymic cyst or other mass requiring immediate intervention per investigator judgment;\n   2. Previous history of thymic tumor not meeting protocol requirements;\n   3. Myasthenic crisis (MGFA Class V) within 3 months prior to randomization;\n   4. Any known disease other than gMG that may interfere with study procedures and assessments;\n   5. A history of progressive multifocal leukoencephalopathy (PML);\n   6. A history of body irradiation or organ transplantation.\n2. Use of any of the following drugs\u002Ftreatments or participation in a clinical study:\n\n   1. Prior treatment with CAR-T or other cellular therapy, or T-cell engager (TCE) therapy;\n   2. Anti-CD20 monoclonal antibody within 6 months prior to randomization; other B-cell or plasma cell-depleting therapy within 6-12 months prior to randomization;\n   3. Alkylating agent within 12 weeks prior to randomization;\n   4. Any biologic for MG treatment within 12 weeks prior to randomization;\n   5. Neonatal Fc receptor antagonist therapy within 8 weeks prior to randomization;\n   6. Janus kinase (JAK), Bruton tyrosine kinase (BTK), or tyrosine kinase 2 (TYK2) inhibitor within 12 weeks prior to randomization;\n   7. IVIg, subcutaneous immunoglobulin, or PLEX therapy within 4 weeks prior to randomization;\n   8. Live\u002Fattenuated live vaccine within 4 weeks prior to randomization, or planned vaccination during the study.\n3. A history of malignancy within 5 years prior to screening;\n4. Infection-related medical history and examinations:\n\n   1. A history of herpes zoster meeting any of the following: 1) A history of disseminated herpes zoster, herpes zoster encephalitis, or ocular herpes zoster involving the retina; 2) Recurrent herpes zoster with 2 or more episodes within 2 years; 3) Herpes zoster infection not fully resolved within 12 weeks prior to screening;\n   2. A history of tuberculosis (TB) or latent TB infection;\n   3. A known history of primary immunodeficiency, splenectomy, or any underlying condition predisposing to infection;\n   4. A history of recurrent infections requiring hospitalization and intravenous antibiotics;\n   5. Any infection requiring hospitalization and\u002For intravenous antimicrobial therapy within 8 weeks prior to randomization, or any infection requiring oral antimicrobial therapy within 2 weeks prior to randomization;\n   6. Positive test result for hepatitis B surface antigen (HBsAg), hepatitis C virus antibody, treponemal pallidum antibody, or human immunodeficiency virus (HIV) antibody; for patients with HBsAg-negative but hepatitis B core antibody (HBcAb)-positive, regardless of the status of hepatitis B surface antibody (HBsAb), HBV-DNA testing is required to confirm their condition, with HBV-DNA-positive patients excluded and HBV-DNA-negative patients eligible to participate in the study.\n5. General situation:\n\n   1. Pregnant or lactating females;\n   2. A history of alcohol abuse or illicit drug abuse within 1 year prior to screening;\n   3. A history of allergic diathesis, or known hypersensitivity\u002Fintolerance to any component of the investigational product;\n   4. Major surgery within 3 months prior to the screening, or planned major surgery during the study;\n   5. Any condition that, in the investigator's judgment, may affect evaluation of study drug safety and efficacy, or any other condition that renders the subject ineligible.",{"count":303,"type":21},60,[25],"This study is a multicenter, randomized, double-blind, placebo-controlled Phase II clinical trial designed to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of SHR-2173 compared to placebo as an add-on therapy to standard of care (SOC) for the treatment of generalized myasthenia gravis (gMG). The study consists of a 4-week screening period, a 24-week treatment period, and a 12-week safety follow-up period.",[28],"2026-05-27",{"date":309,"type":38},"2026-06-03",{"date":311,"type":21},"2026-06",{"date":313,"type":21},"2027-12",{"name":315,"class":45},"Guangdong Hengrui Pharmaceutical Co., Ltd",2,{"id":318,"slug":319,"hasResults":11,"nctId":320,"briefTitle":321,"officialTitle":322,"acronym":323,"eligibilityCriteria":324,"healthyVolunteers":11,"sex":16,"minAge":255,"maxAge":256,"enrollmentInfo":325,"targetDuration":4,"studyType":22,"phases":326,"briefSummary":327,"conditions":328,"keywords":329,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":307,"lastUpdatePostDateStruct":333,"startDateStruct":334,"completionDateStruct":336,"leadSponsor":338,"locationsCount":340},"100591985","phase-2-a-study-to-evaluate-the-pharmacokinetics-pharmacodynamics-safety-and-tolerability-of-inebilizumab-in-children-with-generalized-myasthenia-gravis-gmg-100591985","NCT06987539","A Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety and Tolerability of Inebilizumab in Children With Generalized Myasthenia Gravis (gMG)","A Phase 2 Open-label Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety and Tolerability of Inebilizumab in Children From 2 Years to Less Than 18 Years of Age With Generalized Myasthenia Gravis (gMG)","THYME","Inclusion Criteria\n\n* Participant's legally authorized representative has provided informed consent when the participant is legally too young to provide informed consent and the participant has provided written assent based on local regulations and\u002For guidelines before any study-specific activities\u002Fprocedures being initiated.\n* Age ≥ 2 to \\\u003C 18 years of age on the day of enrollment.\n* Diagnosis of gMG defined as:\n\n  * Positive serologic test for anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody (Ab) titers as confirmed at screening (1 retest allowed), and\n  * At least 1 of the following:\n\n    * History of abnormal neuromuscular transmission test results demonstrated by single-fiber electromyography or repetitive nerve stimulation; or\n    * History of positive anticholinesterase test (eg, edrophonium chloride test); or\n    * Participant demonstrated improvement in gMG signs on oral cholinesterase inhibitors, as assessed by the treating physician; or\n    * Clinical syndrome consistent with a diagnosis of gMG, and not otherwise explained by another condition.\n* Myasthenia Gravis Foundation of America Clinical Classification Class II, III, or IV at the time of screening.\n* Participants must be on:\n\n  * Corticosteroids only, with no dose increase within 4 weeks prior to screening, or\n  * One allowed non-steroidal immunosuppressive therapies (IST) (azathioprine, mycophenolate mofetil, or mycophenolic acid) with continuous use for at least 6 months prior to screening and no dose increase within 4 months prior to screening, or\n  * Combination of (1) corticosteroids with no dose increase within 4 weeks prior to screening and (2) one allowed non-steroidal IST with continuous use for at least 6 months prior to screening and no dose increase within 4 months prior to screening.\n\nTacrolimus is allowed in Japan only, with continued use for ≥ 6 months prior to screening and no dose increase within 4 months prior to screening.\n\n* Participants may enter the study on a stable dose of acetylcholinesterase inhibitors (pyridostigmine dose). The acetylcholinesterase inhibitor dose must have been stable for at least 2 weeks prior to enrollment.\n* Vital signs and laboratory parameters within the normal ranges at screening, or, if outside normal ranges, deemed not clinically significant by the investigator.\n\nExclusion Criteria\n\n* Employees of the Sponsor, contract research organization (CRO), site staff, and their family members.\n* Thymectomy within 12 months prior to baseline (Day 1) visit or planned thymectomy during the duration of the treatment period.\n* Unresected thymoma- Participants with benign thymoma resected \\> 12 months prior to screening may enroll.\n* History of recurrent significant infections.\n* Known immunodeficiency disorder, including current infection or positive test for human immunodeficiency virus (HIV).\n* Positive test for chronic hepatitis B infection at screening.\n* History of untreated hepatitis C infection, or positive antibody test for hepatitis C virus (HCV).\n* History of active or latent tuberculosis (TB), or a positive QuantiFERON®-TB Gold test at screening, unless treatment for TB was completed per local guidelines.\n* History of progressive multifocal leukoencephalopathy.\n* Participants diagnosed with congenital myasthenic syndromes.\n* Receipt of any biologic B-cell-depleting therapy (eg, rituximab, ocrelizumab, obinutuzumab, ofatumumab, inebilizumab) or any experimental B-cell-depleting agent in the 6 months prior to screening.\n* Receipt of any other monoclonal antibody (mAb) or large molecule biologic, including but not limited to FcRn inhibitors, anti-TNF mAbs, anti-janus kinase (JAK) Stat mAbs, and complement inhibitors within 6 months prior to screening.\n* Receipt of the following medications or treatments at any time prior to randomization: alemtuzumab, total lymphoid irradiation, bone marrow transplant, T-cell vaccination therapy, natalizumab.\n* Participants who are pregnant or breastfeeding or planning to get pregnant.",{"count":20,"type":21},[25],"The primary objectives of this study are to characterize the pharmacokinetics (PK) and pharmacodynamics (PD) of inebilizumab administered in pediatric participants with gMG, and to assess the safety and tolerability of inebilizumab administered in pediatric participants with gMG.",[28],[28,330,331,332],"Inebilizumab","AMG 335","Uplizna",{"date":286,"type":38},{"date":335,"type":21},"2026-08-31",{"date":337,"type":21},"2030-03-13",{"name":339,"class":45},"Amgen",3,{"id":342,"slug":343,"hasResults":11,"nctId":344,"briefTitle":345,"officialTitle":346,"acronym":4,"eligibilityCriteria":347,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":278,"enrollmentInfo":348,"targetDuration":4,"studyType":22,"phases":350,"briefSummary":351,"conditions":352,"keywords":353,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":362,"lastUpdatePostDateStruct":363,"startDateStruct":365,"completionDateStruct":367,"leadSponsor":369,"locationsCount":371},"100530984","phase-2-kysa-6-a-study-of-anti-cd19-chimeric-antigen-receptor-t-cell-therapy-in-patients-with-generalized-myasthenia-gravis-100530984","NCT06193889","KYSA-6: A Study of Anti-CD19 Chimeric Antigen Receptor T-Cell Therapy, in Patients With Generalized Myasthenia Gravis","KYSA-6: A Phase 2\u002F3, Open-Label, Randomized, Controlled, Multicenter Study of KYV-101, an Autologous Fully Human Anti-CD19 Chimeric Antigen Receptor T-cell (CD19 CAR T) Therapy, Versus Ongoing Standard-Of-Care Immunosuppressive Therapy in Patients With Generalized Myasthenia Gravis","Key Inclusion Criteria\n\n1. Presence of autoantibodies to AChR or MuSK\n2. Myasthenia Gravis Foundation of America (MGFA) Class II-IV\n3. MG-Activities of Daily Living (MG-ADL) total score of ≥6 at screening and confirmed at baseline visit\n4. QMG total score of ≥11 at screening an confirmed at baseline visit\n5. Failed treatment with 2 or more immunosuppressive\u002Fimmunomodulatory therapies, or failed at least 1 immunosuppressive therapy and required chronic plasmapheresis, or IVIG (or subcutaneous or intramuscular Ig) to control symptoms\n6. On a stable dose of glucocorticoids and\u002For other immunotherapies for ≥1 month prior to screening. For patients treated with azathioprine, a stable dose for ≥2 months prior to screening is required\n7. No change in dose of acetylcholinesterase inhibitors for ≥2 weeks prior to screening\n8. No use of intravenous immune globulin (IVIG) or plasmapheresis (PLEX) within 4 weeks of screening or pre-dose baseline (unless this is part of their SOC treatment regimen)\n9. No use of rituximab (or any other anti-CD20 or CD19 monoclonal antibody) within 12 weeks prior to screening\n10. Able and willing to attend the necessary visits to the study site\n\nKey Exclusion Criteria\n\n1. Unable to washout or interrupt autoimmune disease therapy prior to apheresis and\u002For baseline if required\n2. Co-occurring neurological autoimmune disease (ie, Lambert-Eaton Myasthenic Syndrome) or any disease affecting the neuromuscular junction or muscle causing weakness (eg, myositis, myopathy, motor neuropathy)\n3. History of stroke (with residual sequalae and\u002For risk for recurrence), seizure (even if well controlled on antiepileptics), neurodegenerative disease, altered mental status (unexplained and\u002For recent\u002Fcurrent), or uncontrolled\u002Fsevere psychiatric disease\n4. Any serious and\u002For uncontrolled medical condition that, in the investigator's judgment, would cause unacceptable safety risk, interfere with study procedures or results, or compromise compliance with the protocol, including but not limited to, clinically significant cardiac or pulmonary disease\n5. History of primary immunodeficiency, organ or allogeneic bone marrow transplant, or splenectomy\n6. Active, uncontrolled, viral, bacterial, or systemic fungal infection or recent history of repeated infections\n7. Thymectomy \\\u003C12 months of screening or planned during the study\n8. Prior treatment with gene therapy product or cellular immunotherapy (eg, CAR T) requiring vector integration and directed at any target\n9. Patients requiring chronic anticoagulation therapy that cannot be discontinued for medical procedures",{"count":349,"type":21},66,[25,57],"A Study of the Anti-CD 19 Chimeric Antigen Receptor T Cell Therapy for Patients with Myasthenia Gravis",[88,28],[354,30,355,356,357,220,358,359,360,361],"KYV-101","autoimmune disease","anti-CD19 CAR-T Therapy","cellular therapy","KYSA-6","KYV101-006","mivocabtagene autoleucel","miv-cel","2026-05-13",{"date":364,"type":38},"2026-05-15",{"date":366,"type":38},"2024-08-28",{"date":368,"type":21},"2028-09",{"name":370,"class":45},"Kyverna Therapeutics",17,{"id":373,"slug":374,"hasResults":11,"nctId":375,"briefTitle":376,"officialTitle":377,"acronym":378,"eligibilityCriteria":379,"healthyVolunteers":11,"sex":16,"minAge":380,"maxAge":256,"enrollmentInfo":381,"targetDuration":4,"studyType":22,"phases":383,"briefSummary":384,"conditions":385,"keywords":386,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":391,"lastUpdatePostDateStruct":392,"startDateStruct":394,"completionDateStruct":396,"leadSponsor":398,"locationsCount":400},"100520391","phase-2-a-study-to-evaluate-subcutaneous-zilucoplan-in-pediatric-participants-with-generalized-myasthenia-gravis-100520391","NCT06055959","A Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia Gravis","A Multicenter Open-Label, Uncontrolled Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, Tolerability, and Activity of Zilucoplan in Pediatric Study Participants From 2 to Less Than 18 Years of Age With Acetylcholine Receptor Antibody Positive Generalized Myasthenia Gravis","ziMyG","Inclusion Criteria:\n\nUnited States of America (USA) specific inclusion criterion:\n\n\\- Participant must be 12 to \\\u003C18 years of age at the time of signing the Informed consent\u002Fassent according to local regulation\n\nRest of world (ROW) specific inclusion criterion:\n\n\\- Participant must be 2 to \\\u003C18 years of age at the time of signing the Informed consent\u002Fassent according to local regulation\n\nGlobal inclusion criteria:\n\n* Participant has a diagnosis of generalized myasthenia gravis (gMG) confirmed by a prior positive serologic test result to acetylcholine receptor (AChR) prior to Screening\n* Participant meets the criteria as defined by the Myasthenia Gravis Foundation of America (MGFA) Clinical Classification II to IV at Screening\n* Participants with gMG, including:\n* An MG-activities of daily living (MG-ADL) total score of 6 or more in adolescents from 12 years to \\\u003C18 years of age at Screening\n* Documented weakness in at least 1 limb, neck, or bulbar muscle in children from 2 years to \\\u003C12 years of age at Screening (does not apply to US)\n* Documented vaccination against meningococcal infections within 3 years prior to study start. If not fully vaccinated, participants must receive appropriate prophylactic antibiotic treatment until at least 2 weeks after the initial dose of vaccine(s)\n\nExclusion Criteria:\n\n* Participant has known positive serology for muscle-specific kinase\n* Participant has any medical or psychiatric condition that, in the opinion of the Investigator, could jeopardize or would compromise the participant's ability to participate in this study\n* Participant has had a thymectomy within 6 months prior to Baseline\n* Participant has minimal Manifestation Status of MG based on the clinical judgement of the Investigator\n* Current or recent systemic infection within 2 weeks prior to Baseline or infection requiring intravenous antibiotics within 4 weeks prior to Baseline","12 Years",{"count":382,"type":21},8,[25,57],"The purpose of this study is to assess the pharmacokinetics, pharmacodynamics, safety, tolerability, immunogenicity and activity of zilucoplan (ZLP) in pediatric study participants with generalized myasthenia gravis (gMG).",[28],[387,89,31,388,389,390],"RA101495","zilucoplan","pediatric","MG0014","2026-05-07",{"date":393,"type":38},"2026-05-08",{"date":395,"type":38},"2024-10-16",{"date":397,"type":21},"2026-12-25",{"name":399,"class":45},"UCB Biopharma SRL",9,{"id":402,"slug":403,"hasResults":11,"nctId":404,"briefTitle":405,"officialTitle":406,"acronym":4,"eligibilityCriteria":407,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":408,"enrollmentInfo":409,"targetDuration":4,"studyType":22,"phases":411,"briefSummary":412,"conditions":413,"keywords":4,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":414,"lastUpdatePostDateStruct":415,"startDateStruct":417,"completionDateStruct":419,"leadSponsor":421,"locationsCount":231},"100550478","phase-2-a-clinical-study-of-b007-in-the-treatment-of-generalized-myasthenia-gravis-100550478","NCT06447597","A Clinical Study of B007 in the Treatment of Generalized Myasthenia Gravis.","A Randomized, Double-blind, Placebo-controlled, Multicenter Phase II\u002FIII Clinical Study to Evaluate the Efficacy and Safety of B007 in Subjects With Generalized Myasthenia Gravis","Inclusion Criteria:\n\n1. Sign informed consent form;\n2. Subjects with generalized myasthenia gravis;\n3. Serum AchR(Acetylcholine receptor)-Ab or MUSK-Ab was positive during screening;\n4. MG-ADL ≥5 at screening and baseline;\n5. Before randomization, subjects received at least one gMG stable dose of SoC, and maintain a stable dose throughout the test;\n6. Subjects agree to use effective contraceptive methods for contraception from signing the informed consent form to 1 year after the last dose. Female subjects considered fertile by the investigator must have negative serum pregnancy tests before the first dose.\n\nExclusion Criteria:\n\n1. Subjects with MGFA I and V type;\n2. Subjects usingprescribed drugs;\n3. Subjects with a prescribed disease or history of disease;\n4. The subjects has prescribed examination abnormalities that are assessed as unsuitable for participation in the study by the investigator;\n5. Known history of severe allergic reaction to humanized monoclonal antibodies, or known allergy to any component of B007;\n6. Subjects who received live vaccine at the specified time before the first dose and are expected to receive the vaccine at the specified time after the last dose;\n7. Subjects who have received major surgery or participated in other clinical trials within the prescribed time before screening;\n8. Pregnant and lactating women;\n9. Fertile female subjects do not agree to use effective contraception from signing the informed consent form to 1 years after the last dose.\n10. Sexually active male subjects who do not intend to use an effective contraceptive method during the trial period or within1 years after the last dose, or male subjects who plan to donate sperm during the trial or within 1 year after the last dose;\n11. A history of alcohol or drug abuse within the past 12 months;\n12. Other conditions deemed unsuitable for participation in this study by the researchers.","70 Years",{"count":410,"type":21},104,[25,57],"The purpose of this study is to evaluate the efficacy and safety of B007 in subjects with generalized myasthenia gravis.",[28],"2026-04-29",{"date":416,"type":38},"2026-04-30",{"date":418,"type":38},"2024-07-01",{"date":420,"type":21},"2026-06-30",{"name":422,"class":45},"Shanghai Jiaolian Drug Research and Development Co., Ltd",{"id":424,"slug":425,"hasResults":11,"nctId":426,"briefTitle":427,"officialTitle":428,"acronym":4,"eligibilityCriteria":429,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":185,"enrollmentInfo":430,"targetDuration":4,"studyType":22,"phases":432,"briefSummary":433,"conditions":434,"keywords":435,"overallStatus":122,"whyStopped":4,"lastUpdateSubmitDate":437,"lastUpdatePostDateStruct":438,"startDateStruct":439,"completionDateStruct":441,"leadSponsor":443,"locationsCount":177},"100635702","phase-1-efficacy-and-safety-of-hn2301-in-patients-with-generalized-myasthenia-gravis-mg-100635702","NCT07556120","Efficacy and Safety of HN2301 in Patients With Generalized Myasthenia Gravis (MG)","An Exploratory Study to Evaluate Safety, Tolerability and Preliminary Efficacy of HN2301 in Patients With Generalized Myasthenia Gravis","Inclusion Criteria:\n\n* Age: 18-80 years, no gender restriction;\n* Confirmed diagnosis of generalized myasthenia gravis (MG) with positive AchR or MuSK antibodies, meeting at least one of the following conditions#(1) Repetitive nerve stimulation suggesting neuromuscular transmission defect; (2) Positive response to neostigmine test; (3) Clinically judged improvement of --MG symptoms after oral cholinesterase inhibitor therapy;\n* Clinical classification of MG according to MGFA types IIa-IVb (including IIa, IIb, IIIa, IIIb, IVa, IVb);\n* Baseline MG-ADL score ≥6, ocular-related score \\\u003C50%;\n* Poor response and\u002For lack of efficacy under standard therapies;\n* Minimum life expectancy \\> 12 weeks;\n* Adequate bone marrow, coagulation, cardiopulmonary, liver, and renal function.\n\nExclusion Criteria:\n\n* Subjects positive for hepatitis B surface antigen (HBsAg) and\u002For hepatitis B core antibody (HBcAb) with detectable or quantifiable HBV DNA, positive for hepatitis C antibody (HCV Ab) with detectable or quantifiable HCV RNA, positive for HIV antibody, positive CMV DNA, or CMV DNA above the lower limit of detection; positive for syphilis antigen or antibody;\n* Presence of other uncontrolled active infections;\n* History of major organ transplantation (e.g., heart, lung, liver, kidney) or bone marrow\u002Fhematopoietic stem cell transplantation;\n* Pregnant or breastfeeding women;\n* Receipt of any mRNA-LNP products or other LNP-based drugs within the past two years;\n* History of any of the following cardiovascular conditions within 6 months prior to screening: New York Heart Association (NYHA) Class III or IV heart failure, myocardial infarction, unstable angina, uncontrolled or symptomatic atrial arrhythmias, any ventricular arrhythmias, or other clinically significant cardiac disease;\n* History of ≥Grade 2 bleeding events within 30 days prior to screening, or requiring long-term continuous anticoagulation therapy (e.g., warfarin, low molecular weight heparin, Xa factor inhibitors);\n* History of live vaccination within 30 days prior to screening;\n* Severe central nervous system diseases or pathological changes, including but not limited to: cerebrovascular accident, aneurysm, epilepsy, seizures\u002Fconvulsions, aphasia, stroke, severe brain injury, dementia, Parkinson's disease, cerebellar disorders, organic brain syndromes, or psychosis;\n* History of asthma or severe allergies;\n* Patients combined with other malignant tumors;\n* Any condition that, in the investigator's opinion, may increase the patient's risk or interfere with study assessments.",{"count":431,"type":21},6,[24],"This is an open label, single arm study, to evaluate safety, tolerability and preliminary efficacy of HN2301 in patients with Generalized Myasthenia Gravis.",[28],[220,436],"in vivo CART","2026-04-26",{"date":414,"type":38},{"date":440,"type":21},"2026-05-05",{"date":442,"type":21},"2027-12-31",{"name":444,"class":45},"Shenzhen MagicRNA Biotechnology Co., Ltd",{"id":446,"slug":447,"hasResults":11,"nctId":448,"briefTitle":449,"officialTitle":450,"acronym":451,"eligibilityCriteria":452,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":453,"targetDuration":4,"studyType":22,"phases":455,"briefSummary":457,"conditions":458,"keywords":459,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":468,"lastUpdatePostDateStruct":469,"startDateStruct":471,"completionDateStruct":473,"leadSponsor":474,"locationsCount":258},"100609996","phase-4-a-study-to-investigate-ocs-tapering-in-adult-participants-with-generalized-myasthenia-gravis-treated-with-ravulizumab-100609996","NCT07221838","A Study to Investigate OCS Tapering in Adult Participants With Generalized Myasthenia Gravis Treated With Ravulizumab","OCTAGON: A Phase 4, Single Arm Study to Investigate Oral Corticosteroid Tapering in Adult Patients With Generalized Myasthenia Gravis Treated With Ravulizumab","OCTAGON","Inclusion Criteria:\n\n* Aged greater than 18 years and male or female\n* Clinical diagnosis of gMG\n* Receiving ravulizumab treatment prior to enrollment\n* Receipt of OCS therapy equivalent to a daily dose ≥ 7.5 mg of prednisone\u002Fprednisolone for ≥ 4 continuous weeks directly preceding enrollment\n* A participant of childbearing potential must have a negative highly sensitive pregnancy test (serum or urine, as required by local regulations) taken at Screening before OCS tapering begins.\n* Willing to sign informed consent\n\nExclusion Criteria:\n\n* Concurrent participation in an interventional clinical trial.\n* History of chronic hypoadrenalism (ie, Addison's disease).\n* Use of concomitant OCS for comorbid conditions other than gMG\n* Receipt of a biologic for gMG (e.g., efgartigimod, rozanolixizumab, inebilizumab, rituximab, intravenous immunoglobulin) within 5 half-lives of enrollment\n* Pregnant, breastfeeding, or intending to conceive during the course of the study",{"count":454,"type":21},75,[456],"PHASE4","This is a prospective, multicenter single arm study designed to evaluate the effectiveness and safety of a predefined oral corticosteroids (OCS) tapering schedule to reduce OCS use in adult participants with acetylcholine receptor positive (AChR+) generalized myasthenia gravis (gMG) being treated with intravenous ravulizumab.",[28,89],[460,89,461,462,463,464,121,465,466,467],"Generalized myasthenia gravis","Glucocorticoid toxicity","Complement C5 inhibitor","Prednisone reduction","Cortisol monitoring","corticosteroids","adrenal insufficiency","steroid sparing","2026-04-06",{"date":470,"type":38},"2026-04-09",{"date":472,"type":38},"2026-03-04",{"date":129,"type":21},{"name":131,"class":45},{"id":476,"slug":477,"hasResults":11,"nctId":478,"briefTitle":479,"officialTitle":480,"acronym":4,"eligibilityCriteria":481,"healthyVolunteers":11,"sex":16,"minAge":482,"maxAge":256,"enrollmentInfo":483,"targetDuration":4,"studyType":22,"phases":484,"briefSummary":485,"conditions":486,"keywords":487,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":496,"lastUpdatePostDateStruct":497,"startDateStruct":499,"completionDateStruct":501,"leadSponsor":503,"locationsCount":504},"100562781","phase-3-pk-pd-safety-and-efficacy-study-of-gefurulimab-in-pediatric-patients-with-achr-generalized-myasthenia-gravis-100562781","NCT06607627","PK, PD, Safety, and Efficacy Study of Gefurulimab in Pediatric Patients With AChR+ Generalized Myasthenia Gravis","An Open-Label, Single-arm Study to Evaluate the Pharmacokinetics (PK), Pharmacodynamics (PD), Safety, and Efficacy of Gefurulimab in Pediatric Patients (6 to \u003C 18 Years of Age) With Generalized Myasthenia Gravis (gMG) Who Express Acetylcholine Receptor Antibodies (AChR+)","Inclusion Criteria:\n\nUnited States of America (USA) specific inclusion criterion:\n\n* Participant must be 12 to \\\u003C 18 years of age at the time of signing the informed consent\u002Fassent.\n* All participants must be vaccinated against meningococcal infection from serogroups A, B, C, W, and Y within 3 years and at least 2 weeks prior to the first dose of study intervention administration.\n\nRest of World (ROW) specific inclusion criteria:\n\n* Participant must be 6 to \\\u003C 18 years of age at the time of signing the informed consent\u002Fassent.\n* All participants must be vaccinated against meningococcal infection from serogroups A, C, W, Y (and B where available) within 3 years prior to study intervention on Day 1. If vaccination occurs \\\u003C 2 weeks prior to Day 1, the participants will receive prophylactic antibiotics for at least 2 weeks after initial meningococcal vaccination for serogroups A,C,W,Y (and B, where available)\n\nGlobal inclusion criteria:\n\n* Diagnosis of MG with generalized muscle weakness meeting the clinical criteria defined by Myasthenia Gravis Foundation of America (MGFA) Class II, III or IV\n* Positive serological test for autoantibodies against AChR\n\nExclusion Criteria:\n\n* History of thymectomy, or any other thymic surgery within 12 months prior to Screening\n* Untreated thymic malignancy, carcinoma, or thymoma\n* History of Neisseria meningitidis infection\n* Pregnancy, breastfeeding, or intention to conceive during the course of the study","6 Years",{"count":258,"type":21},[57],"The primary objective of this study is to assess the pharmacokinetics and pharmacodynamics of gefurulimab in pediatric participants with AChR+ gMG for the duration of the study.",[28,89],[488,94,28,89,489,490,491,492,30,220,493,494,495,389],"Acetylcholine Receptor antibodies","ALXN1720","anti-acetylcholine receptor antibody-positive","acetylcholine receptor","AChR","complement component 5","C5","VHH antibody","2026-03-31",{"date":498,"type":38},"2026-04-01",{"date":500,"type":38},"2024-11-13",{"date":502,"type":21},"2029-01-23",{"name":131,"class":45},13,{"id":506,"slug":507,"hasResults":11,"nctId":508,"briefTitle":509,"officialTitle":510,"acronym":4,"eligibilityCriteria":511,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":512,"targetDuration":514,"studyType":166,"phases":4,"briefSummary":515,"conditions":516,"keywords":517,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":524,"lastUpdatePostDateStruct":525,"startDateStruct":527,"completionDateStruct":529,"leadSponsor":531,"locationsCount":532},"100378037","registry-of-participants-with-generalized-myasthenia-gravis-treated-with-alexion-c5-inhibition-therapies-c5its-100378037","NCT04202341","Registry of Participants With Generalized Myasthenia Gravis Treated With Alexion C5 Inhibition Therapies (C5ITs)","Long-Term, Observational, Global Registry of Patients With Generalized Myasthenia Gravis Who Have Received Treatment With Complement C5 Inhibition Therapies (C5ITs)","Inclusion Criteria:\n\n1. Participants with a diagnosis of gMG who are treated with Alexion C5IT at the time of enrollment, including patients previously treated with Soliris or Ultomiris and withdrawn from treatment.\n2. Capable of giving signed informed consent, which includes compliance with the protocol requirements and restrictions.\n3. Participants must have myasthenia gravis historical data, such as MG-ADL and MGFA class, available to be enrolled in the Registry.\n\nExclusion Criteria:\n\n1\\. Participants currently enrolled in an Alexion-sponsored interventional clinical study for treatment of gMG cannot be enrolled in the Alexion gMG Registry while enrolled\u002Fparticipating in the clinical study for gMG therapy.",{"count":513,"type":21},500,"5 Years","Long-term, multicenter, multinational, observational, registry of patients with gMG that is designed to collect data on clinical outcomes and safety in patients prescribed Alexion C5 inhibitor therapies (C5IT) such as eculizumab (Soliris®) and ravulizumab (Ultomiris®).",[28],[89,518,519,520,521,522,28,523],"Soliris","Ultomiris","Alexion C5 Inhibition Therapy","Alexion C5IT","Patient Registry","Alexion Registry","2026-02-17",{"date":526,"type":38},"2026-02-18",{"date":528,"type":38},"2019-12-02",{"date":530,"type":21},"2029-10-01",{"name":131,"class":45},51,{"id":534,"slug":535,"hasResults":11,"nctId":536,"briefTitle":537,"officialTitle":538,"acronym":539,"eligibilityCriteria":540,"healthyVolunteers":11,"sex":16,"minAge":255,"maxAge":17,"enrollmentInfo":541,"targetDuration":4,"studyType":22,"phases":542,"briefSummary":543,"conditions":544,"keywords":4,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":545,"lastUpdatePostDateStruct":546,"startDateStruct":547,"completionDateStruct":549,"leadSponsor":551,"locationsCount":5},"100426506","phase-2-evaluating-the-pharmacokinetics-pharmacodynamics-and-safety-of-efgartigimod-administered-intravenously-in-children-with-generalized-myasthenia-gravis-100426506","NCT04833894","Evaluating the Pharmacokinetics, Pharmacodynamics, and Safety of Efgartigimod Administered Intravenously in Children With Generalized Myasthenia Gravis","Open-label Uncontrolled Trial to Evaluate Pharmacokinetics, Pharmacodynamics, Safety, and Activity of Efgartigimod in Children From 2 to Less Than 18 Years of Age With Generalized Myasthenia Gravis","ADAPT Jr","Inclusion Criteria:\n\n1. Ability of the participant and\u002For his\u002Fher legally authorized representative to understand the requirements of the trial and provide written informed consent\u002Fassent, if applicable (including consent\u002Fassent for the use and disclosure of research-related health information), willingness and ability to comply with the trial protocol procedures (including attending the required trial visits).\n2. Male or female participants between 2 to less than 18 years of age at the time of providing informed consent\u002Fassent. Age groups are enrolled in a staggered fashion respectively: 6 participants in the 12 to less than 18 years of age group followed by 6 participants in the 2 to less than 12 years of age group at the time of providing informed consent\u002Fassent.\n3. Diagnosed with Generalized Myasthenia Gravis (gMG) with confirmed documentation\n4. Meeting the clinical criteria as defined by the Myasthenia Gravis Foundation of America (MGFA) class II, III, and IVa.\n5. Eligible participants should have an unsatisfactory response (efficacy and\u002For safety) to immunosuppressants, steroids or acetylcholinesterase (AChE) inhibitors and should be on stable concomitant gMG therapy of adequate duration before screening.\n6. Positive serologic test for acetylcholine receptor (anti-AChR) antibodies at screening (for younger participants (\\\u003C15kg) historical values can be used).\n7. Contraceptive use should be consistent with local regulations regarding the methods of contraception for those participating in clinical trials. A subject is of childbearing potential if, in the opinion of the investigator, he\u002Fshe is biologically capable of having children and is sexually active.\n\n   1. Male participants: Male participants must agree to not donate sperm from of providing informed consent\u002Fassent until they have completed the trial.\n   2. Female participants: Female adolescents of childbearing potential must have a negative serum pregnancy test at screening and a negative urine pregnancy test at baseline before investigational medicinal product (IMP) can be administered.\n\nExclusion Criteria:\n\n1. Participants with MGFA class I, IVb, and V.\n2. Female adolescents of childbearing potential: Pregnancy or lactation, or the participant intends to become pregnant during the trial or within 90 days after the last dose of IMP.\n3. Has any of the following medical conditions:\n\n   1. Clinically significant uncontrolled active or chronic bacterial, viral, or fungal infection at screening.\n   2. Any other known autoimmune disease that, in the opinion of the investigator, would interfere with an accurate assessment of clinical symptoms of myasthenia gravis or put the participant at undue risk.\n   3. History of malignancy unless deemed cured by adequate treatment with no evidence of recurrence for ≥3 years before the first administration of IMP. Participants with the following cancers can be included at any time: Adequately treated basal cell or squamous cell skin cancer; Carcinoma in situ of the cervix; Carcinoma in situ of the breast; Incidental histological findings of prostate cancer\n   4. Clinical evidence of other significant serious diseases, or have had a recent major surgery, or who have any other condition that, in the opinion of the investigator, could confound the results of the trial or put the participant at undue risk\n4. Worsening muscle weakness secondary to concurrent infections or medications (aminoglycosides, fluoro-quinolones, beta-blockers, etc).\n5. A documented lack of clinical response to plasma exchange (PLEX).\n6. Received a live or live-attenuated vaccine fewer than 28 days before screening. Receiving an inactivated, subunit, polysaccharide, or conjugate vaccine any time before screening is not exclusionary.\n7. Received a thymectomy \\\u003C3 months before screening or 1 is planned to be performed during the trial period.\n8. The following results from these diagnostic assessments will be considered exclusionary:\n\n   a. Positive serum test at screening for an active viral infection with any of the following conditions: Hepatitis B virus (HBV) that is indicative of an acute or chronic infection; Hepatitis C virus (HCV) based on HCV antibody assay; Positive HIV serology at screening; Positive nasopharyngeal swab polymerase chain reaction (PCR) test for severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) at screening.\n9. Using the following prior or concomitant therapies: Use of an investigational product within 3 months or 5 half-lives (whichever is longer) before the first dose of IMP, Use of any monoclonal antibody within the 6 months before the first dose of IMP, Use of intravenous immunoglobulin (IVIg), administered subcutaneously or intramuscularly, or PLEX within 4 weeks before screening.\n10. Total immunoglobulin (IgG) levels \\\u003C6 g\u002FL below the lower limit of normal (LLN) according to the reference ranges of the central laboratory for participant by sex and age at screening.\n11. A known hypersensitivity reaction to efgartigimod or any of its excipients.\n12. Current participation in another interventional clinical trial or previous participation in an efgartigimod trial with at least 1 dose of IMP received.\n13. History (within 12 months of screening) of current alcohol, drug, or medication abuse as assessed by the investigator.",{"count":258,"type":21},[25,57],"The purpose of this trial is to investigate the PK, PD, safety, and activity of efgartigimod IV in children and adolescents aged from 2 to less than 18 years of age with gMG.\n\nTrial details include:\n\n* The maximum trial duration for each individual participant will be approximately 28 weeks\n* The treatment duration will be 8 weeks for the dose-confirmatory part (Part A) and 18 weeks for the treatment response-confirmatory part (Part B)",[28],"2026-02-16",{"date":526,"type":38},{"date":548,"type":38},"2021-10-26",{"date":550,"type":21},"2027-03",{"name":230,"class":45},{"id":553,"slug":554,"hasResults":11,"nctId":555,"briefTitle":556,"officialTitle":557,"acronym":4,"eligibilityCriteria":558,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":559,"targetDuration":4,"studyType":166,"phases":4,"briefSummary":561,"conditions":562,"keywords":564,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":567,"lastUpdatePostDateStruct":568,"startDateStruct":570,"completionDateStruct":572,"leadSponsor":574,"locationsCount":575},"100590443","early-ravulizumab-treatment-of-anti--achr-antibody-positive-generalized-myasthenia-gravis-100590443","NCT06967480","Early Ravulizumab Treatment Of Anti- AChR Antibody-Positive Generalized Myasthenia Gravis","MG-EVOLUTION. Early Ravulizumab Treatment Of Anti- AChR Antibody-Positive Generalized Myasthenia Gravis: A Real-World Study With Biomarker Analysis","Inclusion Criteria:\n\n* Diagnosis of MG confirmed by:\n\n  * History of a positive serologic test for anti-AChR antibodies, and\n  * One of the following:\n\n    * History of abnormal neuromuscular transmission test demonstrated by singlefibre electromyography or repetitive nerve stimulation\n    * History of positive anticholinesterase test (e.g., edrophonium chloride test); or\n    * Patient demonstrated improvement in MG signs on oral cholinesterase inhibitors, as assessed by the treating physician\n* Disease duration from MG onset ≤ 3 years before T-4 (Enrolment);\n* MGFA class IIb to IV;\n* Patient eligible for Ravulizumab treatment based on AIFA reimbursement criteria\n* Vaccination cycle for Neisseria meningitidis completed at least two weeks before Ravulizumab initiation or antibiotics chemoprophylaxis according to the SmPC.\n\nExclusion Criteria:\n\n* Patient unable to understand and sign the informed consent\n* Hypersensitivity to the active substance or to any of the excipients of the study product\n* Patient for whom the study product is contraindicated according to SmPC\n* Previous treatment with C5 inhibitors\n* Last rituximab infusion performed \\\u003C 6 months before T-4 (Enrolment)\n* Last infusion with FcRn blockers performed \\\u003C 3 months before T-4 (Enrollment)\n* Pregnant or lactating or planning a pregnancy during the study\n* Patient who plan to relocate during the study\n* Patient who are unsure of following the visit schedule\n* Patient unable to complete questionnaires\n* Previous or current participation to other interventional studies",{"count":560,"type":21},40,"The primary objective of the study is to evaluate the effectiveness of Ravulizumab in improving MG-ADL in an early-stage AChR+ gMG population.",[28,563],"Anti-AChR Antibody Positive",[565,28,89,566],"Anti-AChR Antibody-Positive","Ravulizumab","2026-02-04",{"date":569,"type":38},"2026-02-05",{"date":571,"type":38},"2025-07-31",{"date":573,"type":21},"2027-06-30",{"name":131,"class":45},23,{"id":577,"slug":578,"hasResults":11,"nctId":579,"briefTitle":580,"officialTitle":581,"acronym":4,"eligibilityCriteria":582,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":583,"targetDuration":4,"studyType":22,"phases":585,"briefSummary":586,"conditions":587,"keywords":4,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":588,"lastUpdatePostDateStruct":589,"startDateStruct":591,"completionDateStruct":593,"leadSponsor":595,"locationsCount":177},"100609520","phase-1-clinical-study-of-cizutamig-in-generalized-myasthenia-gravis-gmg-100609520","NCT07215650","Clinical Study of Cizutamig in Generalized Myasthenia Gravis (gMG)","A Phase 1b, Open-Label, Multicenter Study Evaluating the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, Immunogenicity, and Preliminary Clinical Activity of Cizutamig in Patients With Generalized Myasthenia Gravis (gMG)","Inclusion Criteria:\n\n1. At least 18 years old at the time of signing the Informed Consent Form (ICF);\n2. Diagnosed with MG, classified as MGFA Class II-IVa, and judged by the investigator as unlikely to require respiratory support during the study;\n3. At screening, the Myasthenia Gravis Activities of Daily Living (MG-ADL) score ≥ 5, with non-ocular items accounting for ≥ 50% of the total score, and GMG ≥ 11;\n4. Inadequate response to conventional therapies or lack of effective treatment options, defined as disease recurrence or progression despite treatment with corticosteroids, immunosuppressants (e.g., azathioprine, mycophenolate mofetil, tacrolimus, cyclosporine A, methotrexate), or biologics (e.g., rituximab), and\u002For lack of effective treatment methods.\n\nExclusion Criteria:\n\n1. Any history of CAR-T or TCE therapy targeting any antigen or BCMA-targeted therapy;\n2. Use of any approved immunosuppressive drugs not listed here within 12 weeks or 5 half-lives (whichever is longer) before screening, unless approved by the medical monitor;\n3. Participation in any investigational trial involving non-biological agents within 4 weeks or 5 half-lives (whichever is longer) of the investigational product (IP) before screening;\n4. Participation in any investigational trial involving biological agents within 12 weeks or 5 half-lives (whichever is longer) of the IP before screening;\n5. Administration of live vaccines within 4 weeks before screening;\n6. History of progressive multifocal leukoencephalopathy;\n7. History of primary immunodeficiency (e.g., hypogammaglobulinemia) or hereditary complement deficiency;\n8. Presence of one or more significant concurrent diseases, as judged by the investigator, including but not limited to:\n\n   1. Poorly controlled diabetes\n   2. Chronic kidney disease stages IIIb, IV, or V\n   3. Severe chronic pulmonary disease (e.g., requiring supplemental oxygen) or respiratory failure\n9. Any severe medical condition or clinically significant laboratory abnormality that, in the judgment of the investigator or medical monitor, would compromise the patient's safe participation and completion of the study or may affect protocol compliance or interpretation of study results.",{"count":584,"type":21},44,[24],"The purpose of this study is to assess the safety, tolerability, PK, PD, immunogenicity, and preliminary clinical activity of Cizutamig in patients with Generalized Myasthenia Gravis.",[28],"2025-11-13",{"date":590,"type":38},"2025-11-17",{"date":592,"type":38},"2025-09-16",{"date":594,"type":21},"2028-04",{"name":596,"class":45},"Candid Therapeutics",{"id":598,"slug":599,"hasResults":11,"nctId":600,"briefTitle":601,"officialTitle":601,"acronym":4,"eligibilityCriteria":602,"healthyVolunteers":603,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":604,"targetDuration":4,"studyType":22,"phases":606,"briefSummary":608,"conditions":609,"keywords":610,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":615,"lastUpdatePostDateStruct":616,"startDateStruct":618,"completionDateStruct":620,"leadSponsor":622,"locationsCount":316},"100521061","effects-of-whole-body-electrical-muscle-stimulation-exercise-on-adults-with-myasthenia-gravis-100521061","NCT06064695","Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Myasthenia Gravis","Inclusion Criteria for Participants with MG:\n\n* Age 18 or older\n* Diagnosed with Generalized Myasthenia Gravis (gMG) via seropositive test for acetylcholine receptor (AChR) antibodies or seronegative test with clinical symptoms consistent with gMG\n* On stable MG therapy for at least 1 month\n* Ability to stand for approximately 15 minutes continuously with or without an assistive device (i.e. the length of time to stand to take a shower, complete meal preparation, wait in line at the bank, etc.)\n* Score of 1 (Mild) or 2 (Moderate) on at least one side of the \"arm outstretched\" and \"leg outstretched\" items of the QMG, demonstrating fatigability\n* At least some anti-gravity strength in major muscle groups as assessed by manual muscle testing (i.e. 2+\u002F5 strength or better)\n* Medical clearance to participate in an exercise program\n* Ability to provide informed consent\n* Ability to conform to the requirements of the study (i.e. attendance at assessment and intervention visits, maintain current level of non-study physical activity for the duration of the study, no intention to relocate mid-study)\n\nExclusion Criteria for Participants with MG:\n\n* Concurrent participation in another interventional research study\n* Unable to tolerate 15 minutes of continuous standing with or without an assistive device\n* Regular participation in strength training (2x per week or more over the past 6 months)\n* Score of 0 (None) on all \"arm outstretched\" and \"leg outstretched\" items of the QMG\n* Presence of a pacemaker, metal implants, or other implanted medical devices that could impact participant safety during WB-EMS intervention\n* Presence of unstable acute or chronic disease (i.e. renal failure, rheumatologic disease, cardia arrhythmia, neoplasm, uncontrolled hypertension)\n* Known pregnancy at time of screening\n* Presence of a terminal disease (i.e. receiving hospice services)\n* Current or previous use of any drugs known to influence muscle mass or performance within 6 months; these may include but are not limited to anabolic steroids, IGF01, growth hormone, replacement androgen therapy, anti-androgen therapy\n* Presence of an additional neuromuscular or neurologic condition affecting somatosensory or motor function\u002Fcontrol (i.e. motor neuron disease, muscle disease, peripheral neuropathy, other NMJ condition, Parkinson's disease, Multiple Sclerosis, h\u002Fo stroke, traumatic brain injury, spinal cord injury, ataxia, apraxia, hemiplegia, etc.)\n* Musculoskeletal condition or surgery in the past year that would confound results of exercise interventions (i.e. knee replacement, hip replacement, rotator cuff repair, spinal fusion)\n* Other medical conditions, signs, or symptoms that would interfere with study conduct or interpretation of results as determined by an investigator\n\nInclusion Criteria for Healthy Controls:\n\n* Age 18-39 years\n* Ability to provide informed consent\n\nExclusion Criteria for Healthy Controls:\n\n* Concurrent participation in an interventional research study\n* Presence of unstable acute or chronic disease (i.e. renal failure, rheumatologic disease, cardia arrhythmia, neoplasm, uncontrolled hypertension)\n* Presence of a terminal disease (i.e. receiving hospice services)\n* Current or previous use of any drugs known to influence muscle mass or performance within 6 months; these may include but are not limited to anabolic steroids, IGF01, growth hormone, replacement androgen therapy, anti-androgen therapy\n* Presence of an additional neuromuscular or neurologic condition affecting somatosensory or motor function\u002Fcontrol (i.e. motor neuron disease, muscle disease, peripheral neuropathy, other NMJ condition, Parkinson's disease, Multiple Sclerosis, h\u002Fo stroke, TBI, SCI, ataxia, apraxia, hemiplegia, etc.)\n* Musculoskeletal condition or surgery in the past year that would confound results of exercise interventions (i.e. TKA, THA, RTC repair, spinal fusion)\n* Other medical conditions, signs, or symptoms that would interfere with study conduct or interpretation of results as determined by an investigator\n\nHealthy controls will complete dEMG testing only during a one-time session.",true,{"count":605,"type":21},16,[607],"NA","During this pilot study, the investigators will examine the effects of whole-body electrical muscle stimulation exercise (WB-EMS Exercise) on neuromuscular junction (NMJ) transmission and fatigability in adults with Generalized Myasthenia Gravis (gMG). The investigators will also test whether a relationship exists between NMJ transmission dysfunction and fatigability in gMG, which has long been presumed but never directly assessed. Participants will undergo clinical and electrophysiologic testing before and after the WB-EMS Exercise intervention. The WB-EMS Exercise intervention will be delivered 2 times per week for 4 weeks. Long-term follow up is optional. The hypotheses are (a) that the WB-EMS exercise will improve fatigability and NMJ transmission, and (b) that NMJ transmission dysfunction is related to fatigability.",[28],[611,612,613,614],"Exercise","Electrical Stimulation","Fatigue","Neuromuscular Junction Pathology","2025-04-09",{"date":617,"type":38},"2025-04-10",{"date":619,"type":38},"2023-07-12",{"date":621,"type":21},"2025-08",{"name":623,"class":176},"University of Missouri-Columbia",{"id":625,"slug":626,"hasResults":11,"nctId":627,"briefTitle":628,"officialTitle":629,"acronym":4,"eligibilityCriteria":630,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":631,"targetDuration":4,"studyType":22,"phases":633,"briefSummary":634,"conditions":635,"keywords":637,"overallStatus":34,"whyStopped":4,"lastUpdateSubmitDate":639,"lastUpdatePostDateStruct":640,"startDateStruct":642,"completionDateStruct":644,"leadSponsor":646,"locationsCount":177},"100561261","phase-3-seronegative-myasthenia-gravis---efgartigimod-iv-100561261","NCT06587867","Seronegative Myasthenia Gravis - Efgartigimod IV","Efficacy, Safety and Tolerability of Efgartigimod in Patients with Seronegative Generalized Myasthenia Gravis: an Open-Label Study","Inclusion Criteria:\n\n* Evidence of signed and dated informed consent document(s) indicating that the subject has been informed of all pertinent aspects of the trial. Subjects must be willing and able to comply with the protocol, complete study assessments, and return for follow-up visits.\n* Male or female subjects ≥ 18 years old.\n* Diagnosis of SN MG defined as: (a) clinical syndrome consistent with a diagnosis of MG, and not otherwise explained by another condition, (b) abnormal neuromuscular transmission test results demonstrated by single-fiber electromyography or repetitive nerve stimulation; and (c) negative serologic test for anti-AChR and anti- MuSK antibodies as confirmed at screening, (d) limited, if any, response to therapy with immunotherapy and\u002For antiacetylcholinesterase (AChE) treatment. Further testing for low affinity antibodies to rapsyn-clustered AChR by cell-based assays will be done at baseline and the results included as part of subgroup analysis. All patients will have a negative genetic test for congenital myasthenic syndromes by history or at baseline to exclude the possibility of congenital myasthenic syndrome mimicking SN MG.\n* MGFA Clinical Classification Class II, III, or IV at the time of screening and baseline.\n* Moderate to severe myasthenia gravis as defined by a generalized myasthenia gravis impairment index score \\> 11 or MG-ADL score of at least 5 (with \\>50% of the score due to non-ocular symptoms) and a PASS response of \"No\" and a SSQ of \\\u003C 70% with at least 6 months of historical data as the baseline.\n* Stable or worsening MG as defined by MGII remaining stable or increasing in the 4 week run-in interval.\n* Patients are required to be on a stable dose of their MG treatment (Standard of care-SoC) for at least one month prior to screening. The SoC is limited to AChE inhibitors, steroids and NSISTs (e.g., azathioprine, methotrexate, cyclosporine, tacrolimus, and mycophenolate mofetil. There is no requirement for specific generalised myasthenia gravis therapies.\n* Patients who discontinued early from previous trials of efgartigimod for reasons other than pregnancy, rescue therapy or a SAE can be included.\n* Females of childbearing potential who are sexually active with a non-sterilized male partner must be willing to use at least one highly effective contraception method from the time of screening and for 3 months after the final dose of efgartigimod.\n* Non-sterilized males who are sexually active with a female partner of childbearing potential must be willing to use a condom for the duration of the study and for 3 months after the last dose of efgartigimod. Because male condom is not a highly effective contraception method, it is strongly recommended that female partners of a male study subject also use a highly effective method of contraception throughout this period.\n* Vital signs, electrocardiogram (ECG), and laboratory parameters within the normal ranges at screening, or, if outside normal ranges, deemed not clinically significant by the Investigator.\n* Patient has documented IgG \\>6 g\u002FL within one month of screening\n* Vaccinated for COVID-19 at least 2 weeks prior to screening visit.\n\nExclusion Criteria:\n\n* Patients who discontinued early from trials of efgartigimod for pregnancy or rescue reasons or an SAE that was likely to result in a life-threatening situation or pose a serious safety risk.\n* Pregnant and lactating women, and those intending to become pregnant during the trial or within 3 months after the last dosing. Women of childbearing potential should have a negative urine pregnancy test at screening and baseline.\n* Male patients who are sexually active and do not intend to use effective methods of contraception (as mentioned above) during the trial or within 3 months after the last dosing or male patients who plan to donate sperm during the trial or within 3 months after the last dosing.\n* Patients with known hepatitis B virus (HBV), hepatitis C virus (HCV) or human immunodeficiency virus (HIV) seropositivity.\n* Patients with known autoimmune disease other than MG (e.g., rheumatoid arthritis) which in the investigator opinion would interfere with an accurate assessment of clinical symptoms.\n* Patients with clinical evidence of other significant disease or patients who underwent a recent major surgery, which could confound the results of the trial or put the patient at undue risk.\n* Patients with renal\u002Fhepatic function impairment as defined by (Cr\\>1.5 x elevated) and\u002For (transaminases \\> 2.5 x elevation) at screening.\n* Patients with known medical history of hypersensitivity to any of the ingredients of efgartigimod.\n* Patients who have received rituximab or eculizumab in the 6 months before screening.\n* Patients who have undergone thymectomy within 3 months of screening.\n* Patients who had intravenous immunoglobulin or plasma exchange within 4 weeks of screening.\n* Patient who has clinically significant uncontrolled active or chronic bacterial, viral, or fungal infection at screening\n* Patient has received a live or a live-attenuated vaccination during the month before screening",{"count":632,"type":21},30,[57],"This is to study the efficacy, safety and tolerability of efgartigimod in patients with seronegative generalized myasthenia gravis. This is an open label study. There will be 30 participants to enroll at University Health Network Toronto General Hospital. Study duration is 43 weeks from screening to end of study.",[636,28],"Efgartigimod",[638,31],"seronegative","2024-09-04",{"date":641,"type":38},"2024-09-19",{"date":643,"type":38},"2023-06-01",{"date":645,"type":21},"2025-05",{"name":647,"class":176},"University Health Network, Toronto"]