Nipocalimab for Myasthenia Gravis
Palo Alto (17 mi)Age: 18+
Sex: Any
Travel: May be covered
Time Reimbursement: Varies
Trial Phase: Phase 3
Recruiting
Sponsor: Janssen Research & Development, LLC
Pivotal Trial (Near Approval)
Prior Safety Data
Trial Summary
What is the purpose of this trial?This trial is testing nipocalimab, a medicine that blocks harmful proteins, in people with generalized myasthenia gravis to see if it can reduce their muscle weakness.
Is the drug Nipocalimab a promising treatment for Myasthenia Gravis?Yes, Nipocalimab is a promising drug for Myasthenia Gravis. It targets specific parts of the immune system involved in the disease, potentially offering more personalized and effective treatment options compared to traditional therapies.45679
What safety data is available for Nipocalimab in treating Myasthenia Gravis?The provided research does not directly mention safety data for Nipocalimab (also known as M281, Anti-FcRn monoclonal antibody - Johnson & Johnson, JNJ 80202135) in treating Myasthenia Gravis. However, it discusses the safety and efficacy of other FcRn inhibitors like rozanolixizumab and batoclimab, which are similar in function. These studies indicate that FcRn antagonists are being evaluated for their safety and tolerability in clinical trials for Myasthenia Gravis, suggesting a focus on reducing IgG levels without widespread immune suppression. For specific safety data on Nipocalimab, further research or direct clinical trial results would be needed.12469
Do I need to stop my current medications for this trial?The trial protocol does not specify whether you need to stop taking your current medications.
What data supports the idea that Nipocalimab for Myasthenia Gravis is an effective drug?The available research does not provide specific data on Nipocalimab for Myasthenia Gravis. However, it mentions that other similar drugs, like eculizumab and rozanolixizumab, have shown improvements in patients with this condition. This suggests that drugs targeting similar pathways might be effective, but more specific research on Nipocalimab is needed to confirm its effectiveness.34689
Eligibility Criteria
Adults with generalized myasthenia gravis (MG) who have a certain level of muscle weakness and can undergo infusions and blood sampling. Women must not be pregnant, and men agree to not donate sperm during the study. Participants should not have had recent thymectomy or MG crisis, nor should they have heart issues or allergies to the trial drug.Inclusion Criteria
I can receive medication through a vein and provide blood samples as needed.
I have been diagnosed with generalized myasthenia gravis.
Exclusion Criteria
I have mild or very severe myasthenia gravis, or had a crisis recently.
I had my thymus gland removed within the last year or plan to have it removed.
I have had a heart attack, unstable heart disease, or stroke in the last 3 months.
I do not have an immunodeficiency unrelated to my gMG treatment, nor a family history of it.
Treatment Details
The trial is testing nipocalimab's effectiveness compared to a placebo in improving symptoms of generalized myasthenia gravis. Patients will receive either the actual medication or a placebo without knowing which one they are getting.
2Treatment groups
Experimental Treatment
Placebo Group
Group I: NipocalimabExperimental Treatment1 Intervention
Double-blind Placebo-controlled Phase: Participants will receive nipocalimab intravenous (IV) infusions once every 2 weeks (q2w) up to 24 weeks during double-blind placebo-controlled phase.
Open-label Extension (OLE) Phase: Participants who complete the double-blind placebo-controlled phase will enter the OLE phase and continue to receive nipocalimab q2w IV infusion till study end.
Group II: PlaceboPlacebo Group1 Intervention
Double-blind Placebo-controlled Phase: Participants will receive matching placebo of nipocalimab IV infusion q2w up to 24 weeks during double-blind placebo-controlled phase.
Find a clinic near you
Research locations nearbySelect from list below to view details:
University of Florida Health Jacksonville - RheumatologyJacksonville, FL
Duke University School of MedicineDurham, NC
University of FloridaJacksonville, FL
Yale New Haven HospitalNew Haven, CT
More Trial Locations
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Who is running the clinical trial?
Janssen Research & Development, LLCLead Sponsor
References
Amifampridine phosphate in the treatment of muscle-specific kinase myasthenia gravis: a phase IIb, randomized, double-blind, placebo-controlled, double crossover study. [2022]The aim of this study is to determine the safety and the efficacy of amifampridine phosphate in muscle-specific kinase antibody-positive myasthenia gravis, in a 1:1 randomized, double-blind, placebo-controlled, switchback, double crossover study.
Antagonism of the Neonatal Fc Receptor as an Emerging Treatment for Myasthenia Gravis. [2020]Myasthenia gravis is an autoimmune disease in which immunoglobulin G (IgG) autoantibodies are formed against the nicotinic acetylcholine receptor (AChR) or other components of the neuromuscular junction. Though effective treatments are currently available, many commonly used therapies have important limitations and alternative therapeutic options are needed for patients. A novel treatment approach currently in clinical trials for myasthenia gravis targets the neonatal Fc receptor (FcRn). This receptor plays a central role in prolonging the half-life of IgG molecules. The primary function of FcRn is salvage of IgG and albumin from lysosomal degradation through the recycling and transcytosis of IgG within cells. Antagonism of this receptor causes IgG catabolism, resulting in reduced overall IgG and pathogenic autoantibody levels. This treatment approach is particularly intriguing as it does not result in widespread immune suppression, in contrast to many therapies in routine clinical use. Experience with plasma exchange and emerging phase 2 clinical trial data of FcRn antagonists provide proof of concept for IgG lowering in myasthenia gravis. Here we review the IgG lifecycle and the relevance of IgG lowering to myasthenia gravis treatment and summarize the available data on FcRn targeted therapeutics in clinical trials for myasthenia gravis.
Response to eculizumab in patients with myasthenia gravis recently treated with chronic IVIg: a subgroup analysis of REGAIN and its open-label extension study. [2022]Label="BACKGROUND" NlmCategory="BACKGROUND">In the phase III eculizumab for refractory generalized myasthenia gravis REGAIN study [ClinicalTrials.gov identifier: NCT01997229] and its open-label extension (OLE) [ClinicalTrials.gov identifier: NCT02301624], patients with treatment-refractory antiacetylcholine receptor antibody-positive generalized myasthenia gravis had clinically meaningful improvements with eculizumab versus placebo. This subgroup analysis evaluated data from patients with a recent history of chronic intravenous immunoglobulin (IVIg) use before study entry.
Efficacy and Safety of Rozanolixizumab in Moderate to Severe Generalized Myasthenia Gravis: A Phase 2 Randomized Control Trial. [2021]To explore the clinical efficacy and safety of subcutaneous (SC) rozanolixizumab, an anti-neonatal Fc receptor humanized monoclonal antibody, in patients with generalized myasthenia gravis (gMG).
Fc-Receptor Targeted Therapies for the Treatment of Myasthenia gravis. [2021]Myasthenia gravis (MG) is an autoimmune disease in which immunoglobulin G (IgG) antibodies (Abs) bind to acetylcholine receptors (AChR) or to functionally related molecules in the postsynaptic membrane at the neuromuscular junction. IgG crystallizable fragment (Fc)-mediated effector functions, such as antibody-dependent complement deposition, contribute to disease development and progression. Despite progress in understanding Ab-mediated disease mechanisms, immunotherapy of MG remained rather unspecific with corticosteroids and maintenance with immunosuppressants as first choice drugs for most patients. More specific therapeutic IgG Fc-based platforms that reduce serum half-life or effector functions of pathogenic MG-related Abs are currently being developed, tested in clinical trials or have recently been successfully translated into the clinic. In this review, we illustrate mechanisms of action and clinical efficacies of emerging Fc-mediated therapeutics such as neonatal Fc receptor (FcRn)-targeting agents. Furthermore, we evaluate prospects of therapies targeting classical Fc receptors that have shown promising therapeutic efficacy in other antibody-mediated conditions. Increased availability of Fc- and Fc receptor-targeting biologics might foster the development of personalized immunotherapies with the potential to induce sustained disease remission in patients with MG.
The best and worst of times in therapy development for myasthenia gravis. [2023]Within the last 5 years, the US Food and Drug Administration (FDA) has approved complement and neonatal Fc receptor (FcRN) inhibitors for treatment of generalized myasthenia gravis, and several other therapies are in late-stage clinical trials or under regulatory review. However, questions about which patients are most likely to benefit from which therapies, and the relative effectiveness of these very expensive drugs, has resulted in uncertainty around the place that they should occupy in the existing therapeutic armamentarium. MGNet (a Rare Diseases Clinical Research Consortium funded by the National Institute of Neurological Diseases and Stroke) held two meetings during the 14th International Conference of the Myasthenia Gravis Foundation of America to discuss the most critical needs for clinical trial readiness and biomarker development in the context of therapy development for myasthenia gravis. Herein we provide a summary of these discussions, but not a consensus opinion, and offer a series of recommendations to guide focused research in the most critical areas. We welcome ongoing discussion through comments on this work.
Rituximab as a sole steroid-sparing agent in generalized myasthenia gravis: Long-term outcomes. [2023]Rituximab, a B-cell depleting monoclonal antibody, represents an option for the treatment of refractory myasthenia gravis (MG). Its use is more established in muscle-specific tyrosine kinase positive (MuSK +) patients, while its role in managing acetylcholine receptor positive (AChR +), or double seronegative (DSN) patients, remains less clear. This study evaluates the long-term effectiveness and safety of rituximab in MG of various serotypes.
Efficacy and safety of iscalimab, a novel anti-CD40 monoclonal antibody, in moderate-to-severe myasthenia gravis: A phase 2 randomized study. [2023]Increased morbidity in many patients with myasthenia gravis (MG) on long-term immunosuppression highlights the need for improved treatments. The aim of this study is to investigate the safety and efficacy of iscalimab (CFZ533), a fully human anti-CD40 monoclonal antibody, in patients with moderate-to-severe MG receiving standard-of-care (SoC) therapies.
Subcutaneous batoclimab in generalized myasthenia gravis: Results from a Phase 2a trial with an open-label extension. [2023]To assess the safety, tolerability, and key pharmacodynamic effects of subcutaneous batoclimab, a fully human anti-neonatal Fc receptor monoclonal antibody, in patients with generalized myasthenia gravis and anti-acetylcholine receptor antibodies.