Nipocalimab for Myasthenia Gravis
Recruiting in Palo Alto (17 mi)
+114 other locations
Age: 18+
Sex: Any
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Phase 3
Recruiting
Sponsor: Janssen Research & Development, LLC
Disqualifiers: Immunodeficiency, MGFA Class I/V, others
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.
Will I have to stop taking my current medications?
The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.
What data supports the effectiveness of the drug Nipocalimab for treating myasthenia gravis?
What makes the drug Nipocalimab unique for treating myasthenia gravis?
Nipocalimab is unique because it targets the neonatal Fc receptor (FcRn), which plays a role in prolonging the lifespan of antibodies that contribute to myasthenia gravis. This approach is different from traditional treatments that use corticosteroids or immunosuppressants, offering a more specific way to reduce harmful antibodies in the body.12567
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
You have difficulty with everyday activities due to Myasthenia Gravis, with a score of 6 or higher on the MG-ADL test.
A woman of childbearing potential must have a negative highly sensitive serum (beta-human chorionic gonadotropin [beta-hCG]) at screening and a negative urine pregnancy test at Day 1 prior to administration of study intervention
I can receive medication through a vein and provide blood samples as needed.
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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.
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Trial Timeline
Screening
Participants are screened for eligibility to participate in the trial
2-4 weeks
Double-blind Placebo-controlled Phase
Participants receive either nipocalimab or placebo via IV infusion every 2 weeks
24 weeks
12 visits (in-person)
Open-label Extension (OLE) Phase
Participants who complete the double-blind phase continue to receive nipocalimab every 2 weeks
24 weeks
12 visits (in-person)
Follow-up
Participants are monitored for safety and effectiveness after treatment
4 weeks
Treatment Details
Interventions
- Nipocalimab (Monoclonal Antibodies)
- Placebo (Other)
Trial OverviewThe 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.
Participant Groups
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
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.
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.
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.
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).
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.
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.