Batoclimab for Myasthenia Gravis

Not currently recruiting at 124 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a treatment called batoclimab (an anti-FcRn monoclonal antibody) to determine its effectiveness for people with generalized myasthenia gravis (gMG), a condition that causes muscle weakness. The trial aims to confirm batoclimab's effectiveness and safety compared to a placebo (a substance with no active medication) by assessing participants' ability to handle daily activities. Participants may receive different doses of batoclimab or a placebo in various trial phases. Individuals with gMG who frequently struggle with tasks like walking or lifting due to muscle weakness might be suitable for this trial. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking treatment for gMG.

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.

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

Research has shown that batoclimab, the treatment being tested for myasthenia gravis, has been generally safe in earlier studies. Most patients tolerated the treatment well. These studies reported only mild side effects, such as headaches and tiredness, with serious side effects being rare.

Batoclimab targets a specific protein involved in the immune system, a method also used in other autoimmune diseases. Its progression to phase 3 trials indicates that earlier studies found it generally safe for people. While no treatment is without risks, available data suggests batoclimab is relatively well-tolerated by participants in past research. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for myasthenia gravis?

Researchers are excited about Batoclimab for treating Myasthenia Gravis because it offers a new approach by targeting the neonatal Fc receptor (FcRn). Unlike traditional treatments that often focus on suppressing the immune system broadly, Batoclimab specifically aims to reduce the levels of harmful antibodies by interfering with their recycling process, potentially leading to fewer side effects. Additionally, Batoclimab's mechanism offers a promising alternative for patients who may not respond well to standard treatments like acetylcholinesterase inhibitors, corticosteroids, or immunosuppressants. This targeted action could mean more efficient management of symptoms with a potentially quicker onset of relief.

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

Research has shown that batoclimab may help treat generalized myasthenia gravis (gMG), a condition where muscles weaken due to nerve-muscle communication issues. This trial will evaluate different doses of batoclimab and a placebo to assess its effectiveness and safety. Studies have found that batoclimab can ease symptoms by blocking a specific receptor in the immune system. This action helps lower harmful antibody levels, potentially improving muscle strength. Patients who used batoclimab reported better scores in daily activities, making everyday tasks easier. Early results suggest that batoclimab is safe and well-tolerated, offering hope for people with gMG. ^{1 2 3 4 5}

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Published Research Related to This Trial

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]

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]

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]

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]

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10806862/

Batoclimab as induction and maintenance therapy in ... The FLEX study is expected to provide pivotal data supporting batoclimab as a treatment option in patients with gMG, as well as valuable ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT05403541

NCT05403541 | Phase 3 Study to Assess the Efficacy and ... The purpose of this 4-period study is to confirm the efficacy and safety of batoclimab in participants with gMG.

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10791011/

Subcutaneous batoclimab in generalized myasthenia gravis To assess the safety, tolerability, and key pharmacodynamic effects of subcutaneous batoclimab, a fully human anti‐neonatal Fc receptor monoclonal antibody,

4. clinicaltrials.eu clinicaltrials.eu/inn/batoclimab/

Batoclimab – Application in Therapy and Current Clinical ... Batoclimab, also known as IMVT-1401, is a novel drug currently being studied in clinical trials for various autoimmune disorders.

5. academic.oup.com academic.oup.com/jcem/article/108/12/3122/7214053

Proof-of-concept and Randomized, Placebo-controlled Trials ... These results provide insight into the efficacy and safety of batoclimab and support its further investigation as a potential therapy for TED. neonatal fragment ...