A Study to Examine the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Adult...
Generalized Myasthenia GravisThe primary objective is: To evaluate the effect of pozelimab + cemdisiran on daily functioning that is impacted by signs and symptoms in patients with symptomatic generalized myasthenia gravis (gMG) The secondary objectives of the study are: To evaluate the effect of pozelimab + cemdisiran (ie, combination) and cemdisiran monotherapy on: Clinician-assessed signs of myasthenia gravis (MG) and muscle strength Daily functioning that is impacted by signs and symptoms in patients with symptomatic gMG (cemdisiran monotherapy only). Proportion of patients with improvements in daily function that is impacted by signs and symptoms of MG Proportion of patients that have improvements in clinician-assessed signs of MG and muscle strength Health related quality of life Proportion of patients with minimal MG symptoms Patient- and clinician-reported signs and symptoms of MG To evaluate the safety and tolerability of pozelimab + cemdisiran and cemdisiran monotherapy To assess the concentration of total pozelimab in serum To assess the concentrations of cemdisiran and its metabolites in plasma To assess the immunogenicity of pozelimab To assess the concentration of total C5 in plasma To assess the immunogenicity of cemdisiran To study the effect of pozelimab + cemdisiran and cemdisiran monotherapy on complement activation
Evaluation of Pharmacokinetics, Pharmacodynamics, Efficacy, Safety, and Immunogenicity of Ravulizumab...
Generalized Myasthenia GravisgMGThe primary purpose of this study is to characterize the pharmacokinetics and pharmacodynamics of treatment with ravulizumab intravenous infusion in pediatric participants with gMG.
Phase 3 Study to Assess the Efficacy and Safety of Batoclimab as Induction and Maintenance Therapy...
Generalized Myasthenia GravisThe purpose of this 3-period study is to confirm the efficacy and safety of batoclimab in participants with gMG. In Period 1, participants will be randomized 1:1:1 to receive batoclimab 680 milligrams (mg) subcutaneously (SC) once a week (QW) or 340 mg SC QW or placebo. The primary efficacy endpoint will be assessed by change in the myasthenia gravis activities of daily living (MG- ADL) score in acetylcholine receptor antibody seropositive (AChRAb+) participants. In Period 2, participants previously treated with batoclimab will be re-randomized to stay on batoclimab (340 mg SC QW or 340 mg SC every two weeks) or receive placebo treatment. The secondary endpoint of maintenance of efficacy will be assessed by change in the MG- ADL score in AChRAb+ participants. Participants demonstrating a response to batoclimab during either Period 1 or 2 may enter the long-term extension (Period 3).
Open-Label Extension of Tocilizumab in Subjects With Generalized Myasthenia Gravis
Myasthenia GravisGeneralizedTo evaluate the safety and efficacy of tocilizumab in the treatment of generalized myasthenia gravis (gMG) as an extension study for the participants who previously completed Study tMG(NCT05067348).
A Phase 3 Open-Label Study of Eculizumab in Pediatric Participants With Refractory Generalized Myasthenia...
Myasthenia GravisMyasthenia Gravis3 moreThe purpose of this study is to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of eculizumab in the treatment of pediatric refractory gMG based on change from Baseline in the Quantitative Myasthenia Gravis (QMG) score for disease severity.
Open-Label Extension of Zilucoplan in Subjects With Generalized Myasthenia Gravis
Generalized Myasthenia GravisThe RAISE-XT study is an open-label extension study to evaluate the long-term efficacy, safety, and tolerability of zilucoplan in subjects with gMG who have previously participated in a qualifying Ra Pharmaceuticals sponsored zilucoplan study.
Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Myasthenia Gravis
Generalized Myasthenia GravisDuring this pilot study, we 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). We 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. We hypothesize that the WB-EMS exercise will improve fatigability and NMJ transmission. We also hypothesize that NMJ transmission dysfunction is related to fatigability.
Acupuncture in Myasthenia Gravis (AcuMG)
Myasthenia GravisThe purpose of the study is to determine the effect acupuncture treatment on individuals with a diagnosis of Myasthenia Gravis (MG). A total of 20 people with MG will be enrolled in this study to receive acupuncture treatment 2 times a week for 12 weeks. Participants will be randomized into two groups: 1) Immediate start and 2) Delayed start (12 weeks). The delayed start group will act as a control group for the first 12 weeks, but then receive acupuncture treatment for 12 weeks. It is hypothesized that patients with MG who receive acupuncture treatment will have improved quality of life and activities of daily living compared to no treatment.
Clinical Trial to Improve the Magnetic Levator Prosthesis
BlepharoptosisPtosis4 moreBlepharoptosis (incomplete opening of the eyelids) occurs because of a disruption in the normal agonist-antagonist neuro-muscular complex balance. An external device could restore eyelid movement. A newer class of permanent magnets made of alloys of neodymium (Nd), iron (Fe) and boron (B) might provide the technology needed to develop a feasible external magnetic device that could restore eyelid movement.
A Study to Evaluate Rozanolixizumab in Study Participants With Generalized Myasthenia Gravis
Generalized Myasthenia GravisThe purpose of this study is to assess the safety, tolerability and efficacy of additional 6-week treatment cycles with rozanolixizumab in study participants with generalized myasthenia gravis (gMG).