Nipocalimab for Myasthenia Gravis

Recruiting at 22 trial locations

Overseen By Study Contact

Age: < 18

Sex: Any

Trial Phase: Phase 2 & 3

Sponsor: Janssen Research & Development, LLC

Must be taking: Standard-of-care therapy

Disqualifiers: Severe hepatic, renal, cardiovascular, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 12

Duration: 24 weeks

12 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores the effectiveness of nipocalimab, an anti-FcRn monoclonal antibody, for children and teens with generalized myasthenia gravis (gMG), a condition that causes muscle weakness. Researchers aim to assess how nipocalimab affects certain blood proteins and its safety and tolerability for young patients. The trial seeks children and teens with gMG who experience muscle weakness and have not responded well to current treatments. Participants will receive the treatment every two weeks for 24 weeks, with an option to continue longer. As a Phase 2, Phase 3 trial, this research measures the treatment's effectiveness in an initial, smaller group and represents the final step before FDA approval, offering participants a chance to contribute to potentially groundbreaking advancements in gMG treatment.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications. However, if you are using herbal, naturopathic, traditional Chinese remedies, ayurvedic or nutritional supplements, or medical marijuana, you can continue them if they are stable and approved by the investigator.

Is there any evidence suggesting that nipocalimab is likely to be safe for humans?

Research has shown that nipocalimab is generally safe for people with generalized myasthenia gravis (gMG). In previous studies, patients reported no major side effects from the treatment. Nipocalimab reduces certain proteins made by the immune system, helping to manage the disease. These studies suggest the treatment is safe, with no major safety issues identified. Participants in these studies experienced good symptom control over a 24-week period, which is encouraging for its use in treating gMG. ^{1 2 3 4 5}

Why are researchers excited about this possible treatment for myasthenia gravis?

Nipocalimab is unique because it targets the neonatal Fc receptor (FcRn), which is different from most current treatments for myasthenia gravis that often focus on modulating the immune system or improving neuromuscular transmission. This mechanism allows nipocalimab to reduce the levels of harmful antibodies more directly and potentially with fewer side effects. Researchers are excited about nipocalimab because it offers the possibility of a more targeted approach, which could lead to improved outcomes and a better quality of life for young patients with myasthenia gravis.

What evidence suggests that nipocalimab might be an effective treatment for myasthenia gravis?

Research has shown that nipocalimab, the treatment being studied in this trial, may help treat generalized myasthenia gravis (gMG). In one study, patients started feeling better within just one week. The treatment was generally well-tolerated, although it did not significantly improve a specific measure called MG-ADL, which assesses daily activities. Another study found that many patients experienced positive results with nipocalimab. These findings suggest that nipocalimab could be a good option for managing gMG symptoms. ^{1 5 6 7 8}

Who Is on the Research Team?

Janssen Research & Development, LLC Clinical Trial

Principal Investigator

Janssen Research & Development, LLC

Are You a Good Fit for This Trial?

This trial is for children aged 2 to less than 18 with generalized myasthenia gravis (gMG), who have not responded well to standard treatments. They must meet specific clinical criteria, have certain antibodies present, and maintain a stable use of any herbal or nutritional supplements. Participants need proper venous access for infusions and blood sampling, be within the normal weight range for their age and sex, and females able to bear children must test negative for pregnancy.

Inclusion Criteria

My weight and BMI are within the normal range for my age and sex.

I use herbal or traditional remedies, supplements, or medical marijuana with my doctor's approval and can keep the dose the same during the study.

I have been diagnosed with generalized myasthenia gravis.

Exclusion Criteria

I had a heart attack, unstable heart disease, or stroke in the last 3 months.

I do not have an immunodeficiency unrelated to my myasthenia gravis treatment.

I have had or will have surgery to remove my thymus gland within a year.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive nipocalimab once every two weeks for 24 weeks

24 weeks

12 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Open-label extension (optional)

Participants may opt into continuation of treatment long-term

Long-term

What Are the Treatments Tested in This Trial?

Interventions

• Nipocalimab

Trial Overview

The study tests Nipocalimab's effects on total serum immunoglobulin G (IgG) levels in pediatric patients with gMG. It aims to assess the safety, tolerability, and pharmacokinetics of Nipocalimab in these young participants who are already receiving standard care but need additional treatment.

How Is the Trial Designed?

Group I: Nipocalimab Experimental Treatment 1 Intervention

Participants age 2 to less than (\<) 18 years of age (globally) and 8 to \<18 years of age (for US sites only) will be divided into 2 cohorts as per their age-adolescents 12 to \<18 years and children 2 to \<12 years and will receive nipocalimab once every two weeks for 24 weeks. After Week 24, all participants will have the option to enroll in long term extension (LTE).

Who Is Running the Clinical Trial?

Janssen Research & Development, LLC

Lead Sponsor

Trials

1,022

Recruited

6,408,000+

Joaquin Duato

Janssen Research & Development, LLC

Chief Executive Officer since 2022

MBA from ESADE, Master of International Management from Thunderbird School of Global Management

Dr. Jijo James, MD

Janssen Research & Development, LLC

Chief Medical Officer since 2014

MD from St. Johns Medical College, MPH from Columbia University

Published Research Related to This Trial

In a phase 2a trial involving 43 patients with generalized myasthenia gravis, rozanolixizumab showed a trend towards clinical benefit, particularly in secondary measures like the MG-Activities of Daily Living and MG-Composite scores, although the primary endpoint (QMG score) did not reach statistical significance.

Rozanolixizumab was generally well tolerated, with headache being the most common adverse event, occurring in 57% of patients receiving the treatment compared to 14% in the placebo group.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy and Safety of Rozanolixizumab in Moderate to Severe Generalized Myasthenia Gravis: A Phase 2 Randomized Control Trial. Bril, V., Benatar, M., Andersen, H., et al.[2021]

A novel treatment for myasthenia gravis is being developed that targets the neonatal Fc receptor (FcRn), which helps prolong the lifespan of IgG antibodies; antagonizing this receptor can reduce harmful autoantibody levels without broadly suppressing the immune system.

Clinical trials, including phase 2 studies, show promise for FcRn antagonists in lowering IgG levels, providing a new therapeutic option for patients with myasthenia gravis who may not respond well to existing treatments.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Antagonism of the Neonatal Fc Receptor as an Emerging Treatment for Myasthenia Gravis. Gable, KL., Guptill, JT.[2020]

In a study of 30 patients with refractory myasthenia gravis (MG) treated with rituximab for an average of 33.3 months, significant improvements were observed in disease severity, as indicated by a decrease in the quantitative myasthenia gravis (QMG) score from 11 to 4.3 (p < 0.001).

Rituximab was found to be safe and well-tolerated, with no crises or deaths reported, and 93.1% of patients were able to reduce their daily steroid dose to 10 mg or less, highlighting its potential as a long-term treatment option for anti-AChR+ and double seronegative MG patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Rituximab as a sole steroid-sparing agent in generalized myasthenia gravis: Long-term outcomes. Kefalopoulou, ZM., Veltsista, D., Germeni, A., et al.[2023]

In a prospective study of 22 patients with treatment-refractory myasthenia gravis, rituximab treatment led to significant clinical improvement, with manual muscle testing scores decreasing from an average of 10.6 to 3.3 over a mean follow-up of 28.8 months.

Rituximab also allowed for a substantial reduction in prednisone dosage, from 25.2 mg/d to 7.3 mg/d, indicating its potential to reduce reliance on steroids while managing the disease.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Rituximab in refractory myasthenia gravis: Extended prospective study results. Beecher, G., Anderson, D., Siddiqi, ZA.[2019]

The FDA has recently approved new therapies, including complement and Fc receptor inhibitors, for treating generalized myasthenia gravis, highlighting advancements in treatment options for this condition.

There is ongoing uncertainty regarding which patients will benefit most from these expensive therapies, prompting discussions on the need for better clinical trial readiness and biomarker development to guide future research.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The best and worst of times in therapy development for myasthenia gravis. Benatar, M., Cutter, G., Kaminski, HJ.[2023]

Myasthenia gravis (MG) is driven by IgG antibodies that attack acetylcholine receptors, leading to muscle weakness, and current treatments like corticosteroids are not very specific.

New therapies targeting IgG Fc mechanisms, such as FcRn-targeting agents, are being developed and show promise for more effective and personalized treatments that could lead to sustained remission in MG patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Fc-Receptor Targeted Therapies for the Treatment of Myasthenia gravis. Keller, CW., Pawlitzki, M., Wiendl, H., et al.[2021]

In a Phase 2a trial involving 17 patients with generalized myasthenia gravis, batoclimab significantly reduced total immunoglobulin G and anti-acetylcholine receptor antibodies compared to placebo, indicating its potential efficacy in targeting the underlying immune response.

The study found no safety issues with batoclimab, supporting its tolerability and suggesting it could be a viable option for patient-administered therapy in this condition.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Subcutaneous batoclimab in generalized myasthenia gravis: Results from a Phase 2a trial with an open-label extension. Nowak, RJ., Breiner, A., Bril, V., et al.[2023]

In a phase 2 study involving 44 patients with moderate-to-severe myasthenia gravis (MG), iscalimab demonstrated significant improvements in the MG Composite score and MG-Activities of Daily Living score compared to placebo, particularly in non-thymectomized patients.

The safety profile of iscalimab was comparable to placebo, with similar rates of adverse events, indicating that it may be a safe option for patients with MG despite not showing a protective effect overall.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy and safety of iscalimab, a novel anti-CD40 monoclonal antibody, in moderate-to-severe myasthenia gravis: A phase 2 randomized study. GomezMancilla, B., Meriggioli, MN., Genge, A., et al.[2023]

Myasthenia gravis (MG) is primarily caused by antibodies against the nicotinic acetylcholine receptor, leading to muscle weakness, and current treatments like intravenous immunoglobulin and prednisone have significant side effects.

Monoclonal antibody therapies, particularly eculizumab, which targets the complement system, show promise in specifically modulating the disease mechanisms of MG, potentially transforming treatment options for patients.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Monoclonal Antibody-Based Therapies for Myasthenia Gravis. Alabbad, S., AlGaeed, M., Sikorski, P., et al.[2022]

Monoclonal antibodies (mAbs) are emerging as targeted therapies for myasthenia gravis (MG), a condition traditionally treated with non-specific immunosuppression, offering a more precise approach to treatment.

Eculizumab and efgartigimod have been approved for generalized MG, showing that targeting specific pathways can lead to better treatment outcomes, with ongoing research into additional mAbs showing promising results.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Antibody Therapies in Autoimmune Neuromuscular Junction Disorders: Approach to Myasthenic Crisis and Chronic Management. Vanoli, F., Mantegazza, R.[2023]

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