Inebilizumab for Myasthenia Gravis

Overseen By Amgen Call Center

Age: < 65

Sex: Any

Trial Phase: Phase 2

Sponsor: Amgen

Must be taking: Corticosteroids, Immunosuppressive therapies

Must not be taking: Biologic B-cell-depleting, Monoclonal antibodies

Disqualifiers: Thymectomy, Immunodeficiency, Hepatitis, Tuberculosis, 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: Unknown

15 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a medication called inebilizumab, an antibody treatment, to evaluate its effectiveness and safety for children with generalized myasthenia gravis (gMG), a condition that causes muscle weakness. Participants will receive the treatment through an IV to monitor how the body processes the drug and to check for side effects. Children aged 2 to 17 with a confirmed gMG diagnosis, who frequently experience muscle weakness and are on stable medication, may be eligible. As a Phase 2 trial, the research focuses on measuring the treatment's effectiveness in an initial, smaller group of participants.

Do I have to stop taking my current medications for the trial?

You may need to keep your current medications stable. If you're on corticosteroids or certain immunosuppressive therapies, your dose should not have increased recently. Acetylcholinesterase inhibitors should also be stable for at least 2 weeks before joining the study.

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

Research has shown that inebilizumab is generally well-tolerated by people with generalized myasthenia gravis (gMG). In a study with 238 participants, those who took inebilizumab demonstrated good safety results. Over 26 weeks, participants experienced improvements and manageable side effects.

This treatment is a monoclonal antibody, targeting specific cells in the immune system. Previous studies suggest that inebilizumab could be a safe option for treating gMG, even for younger patients. However, participants should discuss potential risks and benefits with their healthcare providers before joining a trial. ^{1 2 3 4 5}

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

Unlike the standard treatments for myasthenia gravis, which often include medications like anticholinesterase inhibitors and immunosuppressants, inebilizumab offers a fresh approach by targeting CD19, a protein on the surface of B cells. This mechanism helps reduce the number of B cells, which are believed to play a role in the autoimmune response that characterizes myasthenia gravis. Researchers are excited about inebilizumab because it is administered intravenously, which could potentially offer more controlled dosing and faster action compared to oral medications. This innovative approach might provide a more effective and efficient way to manage symptoms for patients.

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

Research has shown that inebilizumab, which participants in this trial will receive, effectively treats generalized myasthenia gravis (gMG). Studies found that patients taking inebilizumab experienced better muscle function and could perform daily activities more easily. For instance, one study revealed that 72.3% of patients taking the drug improved significantly in daily activities, compared to 45.2% of those not taking it. After 52 weeks, patients continued to benefit, especially those with specific antibodies (AChR+). This evidence suggests that inebilizumab could be a promising treatment option for people with gMG. ^{1 4 5 6 7}

Who Is on the Research Team?

MD

Principal Investigator

Amgen

Are You a Good Fit for This Trial?

This trial is for children with generalized Myasthenia Gravis (gMG) who have a positive test for specific antibodies, abnormal neuromuscular transmission tests, or improvement on oral cholinesterase inhibitors. They should be moderately to severely affected by gMG and may be taking certain stable medications.

Inclusion Criteria

* Quantitative Myasthenia Gravis score of 11 or greater at screening.

* Participant's legally authorized representative has provided informed consent when the participant is legally too young to provide informed consent and the participant has provided written assent based on local regulations and/or guidelines before any study-specific activities/procedures being initiated.

* Vital signs and laboratory parameters within the normal ranges at screening, or, if outside normal ranges, deemed not clinically significant by the investigator.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive inebilizumab administered intravenously to evaluate pharmacokinetics, pharmacodynamics, safety, and tolerability

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Inebilizumab

Trial Overview

The study aims to understand how the drug Inebilizumab behaves in the body (pharmacokinetics), its effects (pharmacodynamics), and its safety in pediatric patients with gMG.

How Is the Trial Designed?

Group I: Inebilizumab Experimental Treatment 1 Intervention

Inebilizumab will be administered intravenously (IV).

Who Is Running the Clinical Trial?

Amgen

Lead Sponsor

Trials

1,508

Recruited

1,433,000+

Founded

1980

Headquarters

Thousand Oaks, USA

Known For

Human Therapeutics

Top Products

Enbrel, Prolia, Neulasta, Otezla

Robert A. Bradway

Amgen

Chief Executive Officer since 2012

MBA from Harvard Business School

Paul Burton

Amgen

Chief Medical Officer since 2023

MD from University of London, PhD in Molecular and Cellular Biology from Imperial College London

Citations

1.

pubmed.ncbi.nlm.nih.gov

pubmed.ncbi.nlm.nih.gov/40202593/

A Phase 3 Trial of Inebilizumab in Generalized Myasthenia ...

Participants who received inebilizumab had a greater reduction in the MG-ADL score than those who received placebo (least-squares mean change, - ...

2.

ajmc.com

ajmc.com/view/mint-trial-26-week-data-show-inebilizumab-for-gmg-is-effective-and-safe

MINT Trial 26-Week Data Show Inebilizumab for gMG Is ...

Inebilizumab showed significant efficacy in improving MG-ADL and QMG scores in gMG patients compared to placebo over 26 weeks. The trial ...

3.

rheumatologyadvisor.com

rheumatologyadvisor.com/news/week-52-data-show-inebilizumab-continues-to-benefit-achr-myasthenia-gravis/

Week 52 Data Show Inebilizumab Continues to Benefit in ...

At week 52, results showed AChR+ patients treated with inebilizumab continued to improve based on the change from baseline in MG-ADL score vs ...

4.

neurology.org

neurology.org/doi/10.1212/WNL.0000000000211217

Phase 3 Myasthenia Gravis Inebilizumab Trial (MINT)

The primary endpoint was achieved demonstrating a clinically meaningful improvement in MG-ADL score change in the inebilizumab group (−4.2) as ...

5.

checkrare.com

checkrare.com/positive-results-from-mint-study-of-uplizna-for-myasthenia-gravis/

Positive Results From MINT Study of Uplizna for ...

Among the AChR+ population treated with inebilizumab, 72.3% had a 3 point or greater improvement in MG-ADL score, compared to 45.2% in the ...

6.

clinicaltrials.gov

clinicaltrials.gov/study/NCT04524273

NCT04524273 | Myasthenia Gravis Inebilizumab Trial

This study is a phase 3, randomized, double-blind, placebo-controlled study, to be conducted at approximately 120 study sites.

7.

amgen.com

amgen.com/newsroom/press-releases/2025/03/uplizna-inebilizumabcdon-significantly-improves-generalized-myasthenia-gravis-symptoms-in-acetylcholine-receptor-autoantibodypositive-patients-over-52-weeks

UPLIZNA® (INEBILIZUMAB-CDON) SIGNIFICANTLY ...

Patients Reported Improvement in Ability to Conduct Daily Activities with Twice-Yearly Dosing*. Late-Breaking Data to be Presented at AAN ...