Batoclimab for Myasthenia Gravis

Recruiting at 108 trial locations

Overseen By Central Study Contact

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Immunovant Sciences GmbH

Must not be taking: Immunosuppressives, Anti-FcRn

Disqualifiers: Myasthenic crisis, Thymectomy, Malignant thymoma, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

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

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

240 Participants Needed

Trial Summary

What is the purpose of this trial?

This trial is testing batoclimab, a medication given by injection, in patients with generalized Myasthenia Gravis who have specific antibodies. The goal is to see if it can improve their muscle strength and daily activities by lowering harmful antibodies. The study will also check if the benefits last over time. Batoclimab is being tested for its safety and potential efficacy in Chinese patients with generalized myasthenia gravis.

Will I have to stop taking my current medications?

The trial information does not specify if you need to stop taking your current medications. However, if you have used treatments with immunosuppressive properties or anti-FcRn treatment recently, you may not be eligible to participate.

What data supports the effectiveness of the drug Batoclimab for treating myasthenia gravis?

Research shows that Batoclimab, which targets a specific receptor involved in prolonging the life of harmful antibodies, may help reduce these antibodies in myasthenia gravis patients. Similar drugs like efgartigimod have shown improvements in patient quality of life and symptoms, suggesting Batoclimab could have similar benefits. ^{1 2 3 4 5}

Is Batoclimab safe for humans?

Research on Batoclimab, used for treating generalized myasthenia gravis, shows it has been evaluated for safety and tolerability in clinical trials. These studies suggest that Batoclimab is generally safe for human use, although specific side effects or risks were not detailed in the available summaries. ^{1 3 4 5 6}

How is the drug Batoclimab different from other treatments for myasthenia gravis?

Batoclimab is unique because it is a fully human monoclonal antibody that targets the neonatal Fc receptor, which helps reduce harmful antibodies in the body. It is administered subcutaneously (under the skin), offering a different route compared to some other treatments like efgartigimod, which is given intravenously (through a vein). ^{1 3 4 5 7}

Eligibility Criteria

Adults over 18 with generalized Myasthenia Gravis (gMG) who experience muscle weakness. They should have specific scores on the QMG and MG-ADL scales, indicating mild to severe gMG. Those who've had a recent myasthenic crisis, thymectomy, untreated malignant thymoma, or certain immunosuppressive treatments are excluded.

Inclusion Criteria

My myasthenia gravis symptoms are moderate to severe.

Have a QMG score ≥ 11 at the Screening and Baseline Visits

Additional inclusion criteria are defined in the protocol.

Exclusion Criteria

I haven't had treatments that weaken my immune system in the past year.

I had my thymus gland removed less than 6 months ago or will have it removed during the study.

I have used anti-FcRn treatment in the last 3 months or it didn't work for me before.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Induction Treatment (Period 1)

Participants are randomized to receive batoclimab 680 mg SC QW, 340 mg SC QW, or placebo. Efficacy is assessed by change in MG-ADL score.

12 weeks

Maintenance Treatment (Period 2)

Participants previously treated with batoclimab are re-randomized to continue batoclimab or receive placebo. Efficacy maintenance is assessed.

Up to 64 weeks

Long-term Extension (Period 3)

Participants demonstrating a response to batoclimab may enter the long-term extension.

Duration not specified

Optional Long-Term Extension (Period 4)

Participants who complete Period 3 are eligible for an optional long-term extension according to their treatment assignment.

Duration not specified

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Batoclimab

Trial Overview The trial is testing Batoclimab's effectiveness for gMG at different doses: 680 mg weekly or 340 mg weekly/bi-weekly versus placebo. It has three periods: initial treatment randomization, dose adjustment re-randomization for responders, and long-term extension for sustained response.

Participant Groups

Group I: Batoclimab Maintenance Dose 2 (Period 2) Experimental Treatment 1 Intervention

Group II: Batoclimab Maintenance Dose 1 (Period 2) Experimental Treatment 1 Intervention

Group III: Batoclimab Induction Dose 2 (Period 1) Experimental Treatment 1 Intervention

Group IV: Batoclimab Induction Dose 1 (Period 1) Experimental Treatment 1 Intervention

Group V: Placebo Induction Dose (Period 1) Placebo Group 1 Intervention

Group VI: Placebo Maintenance Dose (Period 2) Placebo Group 1 Intervention

Batoclimab is already approved in European Union, United States for the following indications:

Approved in European Union as Batoclimab for:

• Thyroid Eye Disease (TED)

Approved in United States as Batoclimab for:

• Thyroid Eye Disease (TED)

Who Is Running the Clinical Trial?

Immunovant Sciences GmbH

Lead Sponsor

Trials

12

Recruited

1,400+

Findings from Research

Efgartigimod, a first-in-class neonatal Fc receptor antagonist, received its first approval in the USA in December 2021 for treating generalized myasthenia gravis in adults who are positive for anti-acetylcholine receptor antibodies.

The drug is also being evaluated for other autoimmune diseases and has been approved in Japan for generalized myasthenia gravis patients regardless of antibody status, indicating its potential broad application in autoimmune conditions.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efgartigimod: First Approval. Heo, YA.[2022]

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 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]

Ravulizumab demonstrated sustained efficacy in treating generalized myasthenia gravis over 60 weeks, with significant improvements in Myasthenia Gravis-Activities of Daily Living (MG-ADL) scores for patients who continued treatment from the randomized controlled period.

The treatment was well tolerated with no reported cases of meningococcal infections, indicating a favorable safety profile for long-term use.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Long-term efficacy and safety of ravulizumab in adults with anti-acetylcholine receptor antibody-positive generalized myasthenia gravis: results from the phase 3 CHAMPION MG open-label extension. Meisel, A., Annane, D., Vu, T., et al.[2023]

Efgartigimod alfa is the first neonatal Fc receptor antagonist approved for treating generalized myasthenia gravis (gMG), showing significant and rapid improvements in muscle strength and quality of life in a phase 3 trial with a placebo group.

The treatment was generally well tolerated, with most side effects being mild to moderate, indicating a favorable safety profile for patients with gMG.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efgartigimod Alfa in Generalised Myasthenia Gravis: A Profile of Its Use. Heo, YA.[2023]

References

1. New Zealand pubmed.ncbi.nlm.nih.gov

Efgartigimod: First Approval. [2022]

2. Switzerland pubmed.ncbi.nlm.nih.gov

Antagonism of the Neonatal Fc Receptor as an Emerging Treatment for Myasthenia Gravis. [2020]

3. United States pubmed.ncbi.nlm.nih.gov

Subcutaneous batoclimab in generalized myasthenia gravis: Results from a Phase 2a trial with an open-label extension. [2023]

4. Germany pubmed.ncbi.nlm.nih.gov

Efgartigimod improved health-related quality of life in generalized myasthenia gravis: results from a randomized, double-blind, placebo-controlled, phase 3 study (ADAPT). [2023]

5. New Zealand pubmed.ncbi.nlm.nih.gov

Therapeutic Effects of Batoclimab in Chinese Patients with Generalized Myasthenia Gravis: A Double-Blinded, Randomized, Placebo-Controlled Phase II Study. [2022]

6. Germany pubmed.ncbi.nlm.nih.gov

Long-term efficacy and safety of ravulizumab in adults with anti-acetylcholine receptor antibody-positive generalized myasthenia gravis: results from the phase 3 CHAMPION MG open-label extension. [2023]

7. New Zealand pubmed.ncbi.nlm.nih.gov

Efgartigimod Alfa in Generalised Myasthenia Gravis: A Profile of Its Use. [2023]