Zilucoplan for Myasthenia Gravis

Overseen By Julie Agriesti, MACPR, CCRC

Age: 18+

Sex: Any

Trial Phase: Phase 3

Sponsor: Miriam Freimer

Must not be taking: C5 inhibitors, Rituximab

Disqualifiers: Meningococcal disease, Intubation, Pregnancy, others

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

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

Compensation: Varies

Number of Visits: Unknown

Duration: 12 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial examines how well zilucoplan, a daily injection, works for people with myasthenia gravis (MG), a condition that causes muscle weakness. It targets individuals experiencing a severe worsening of symptoms that require hospitalization. This study suits those diagnosed with MG, experiencing severe muscle weakness affecting areas like breathing or swallowing, and testing positive for AChR antibodies, a specific protein linked to MG. The goal is to determine if zilucoplan can manage these severe symptoms over a 12-week period. As a Phase 3 trial, it represents the final step before FDA approval, offering participants a chance to contribute to potentially bringing a new treatment to market.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop your current medications, but it does exclude those who have used certain treatments like C5 inhibitors recently. It's best to discuss your specific medications with the trial team.

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

Research has shown that zilucoplan is generally safe for people. One study found that patients quickly felt better without major safety problems. Long-term data indicates that while most patients (95.5%) experienced some side effects, these were not serious for most. This suggests that zilucoplan is well-tolerated for treating myasthenia gravis, a condition that causes muscle weakness. ^{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 pyridostigmine or immunosuppressants, Zilucoplan acts uniquely by targeting the complement system, a part of the immune system involved in the disease process. This mechanism of action is promising because it may prevent the immune attacks on the neuromuscular junctions more effectively. In addition to its novel approach, Zilucoplan is administered subcutaneously, offering potentially more convenient and comfortable dosing compared to some existing treatments that require intravenous administration. Researchers are excited about Zilucoplan because these differences could lead to improved symptom control and quality of life for patients.

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

Research has shown that zilucoplan can help treat myasthenia gravis, a condition that causes muscle weakness. Studies found that patients taking zilucoplan experienced noticeable improvements in daily activities and overall muscle strength compared to those on a placebo. By the 12th week of treatment, patients showed an improvement of more than four points in handling everyday tasks. Zilucoplan was well-tolerated, with no major unexpected safety concerns. These findings suggest that zilucoplan could be a promising treatment option for people with myasthenia gravis. ^{4 5 6 7 8}

Who Is on the Research Team?

Miriam Freimer, MD

Principal Investigator

Ohio State University

Are You a Good Fit for This Trial?

This trial is for individuals with a condition called Myasthenia Gravis (gMG) who have severe symptoms requiring hospitalization. Participants must test positive for acetylcholine receptor antibodies (AChR+). Specific inclusion and exclusion criteria details are not provided, but typically these would outline health requirements and any conflicting medications or conditions.

Inclusion Criteria

MGFA class II - IVb

If female of child-bearing potential, negative serum pregnancy test at screening

Women of child-bearing potential or men with sexual partners of childbearing potential must be willing to use an acceptable method of birth control for the duration of the study and for 40 days after the last dose of study drug therapy. Acceptable methods of birth control include abstinence, oral contraceptives, the contraceptive patch, intra-uterine device, the contraceptive ring, and or barrier contraception such as condoms with spermicide

Exclusion Criteria

History of meningococcal disease

Participants requiring intubation prior to study start

Pregnancy or lactating

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive subcutaneous zilucoplan injections daily for 12 weeks

12 weeks

Daily visits (in-hospital initially, then outpatient)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Zilucoplan

Trial Overview

The study is testing Zilucoplan®, administered through daily subcutaneous injections over a period of 12 weeks. The trial aims to evaluate its effectiveness in treating severe exacerbations of gMG in hospitalized patients. It's an open-label phase 3b study, meaning both the researchers and participants know what treatment is being given.

How Is the Trial Designed?

Group I: Zilucoplan Experimental Treatment 1 Intervention

Subcutaneous zilucoplan

Who Is Running the Clinical Trial?

Miriam Freimer

Lead Sponsor

UCB Pharma

Industry Sponsor

Trials

345

Recruited

110,000+

Jean-Christophe Tellier

UCB Pharma

Chief Executive Officer since 2015

MD specialized in Rheumatology

Prof. Dr. Iris Löw-Friedrich

UCB Pharma

Chief Medical Officer since 2004

MD, PhD

Citations

1.

ncbi.nlm.nih.gov

ncbi.nlm.nih.gov/books/NBK616632/

Clinical Review - Zilucoplan (Zilbrysq) - NCBI - NIH

In the overall gMG population, the 1 patient (1.2%) in the zilucoplan group who died experienced an SAE leading to death due to COVID-19 and COVID-19 pneumonia, ...

2.

sciencedirect.com

sciencedirect.com/science/article/pii/S0022510X25001674

Efficacy of zilucoplan in patients with generalised ...

Zilucoplan showed clinically meaningful improvements in myasthenia gravis (MG)-specific outcomes versus placebo in the overall population. We assessed the ...

3.

zilbrysqhcp.com

zilbrysqhcp.com/clinical-trial

ZILBRYSQ® Clinical Trial Data For gMG

In the pivotal Phase 3 RAISE trial, ZILBRYSQ delivered a >4-point improvement in the ability to manage activities of daily living at Week 12 for adults with ...

4.

ucb.com

ucb.com/newsroom/press-releases/article/ucb-announces-positive-data-in-myasthenia-gravis-with-zilucoplan-phase-3-study-results

UCB announces positive data in myasthenia gravis with ...

The results show zilucoplan was well-tolerated and no major unexpected safety findings were identified compared to earlier zilucoplan studies. The incidence of ...

5.

neurology.org

neurology.org/doi/10.1212/WNL.0000000000208755

Effect of Zilucoplan on Myasthenia Gravis–Specific ...

Zilucoplan demonstrated rapid and clinically meaningful improvements in MG-ADL and QMG total scores vs placebo in adults with acetylcholine receptor ...

6.

pubmed.ncbi.nlm.nih.gov

pubmed.ncbi.nlm.nih.gov/37059508/

Safety and efficacy of zilucoplan in patients with ...

Zilucoplan treatment showed rapid and clinically meaningful improvements in myasthenia gravis-specific efficacy outcomes, had a favourable safety profile, and ...

7.

neurology.org

neurology.org/doi/10.1212/WNL.0000000000204989

Long-term Safety and Efficacy of Zilucoplan in Myasthenia ...

At data cut-off (May 11, 2023), median (range) exposure to zilucoplan was 1.8 (0.11–5.1) years. TEAEs occurred in 191 (95.5%) patients and 71 ( ...

8.

ucb.com

ucb.com/newsroom/press-releases/article/ucb-presents-efficacy-and-safety-results-for-zilucoplan-and-rozanolixizumab-in-generalized-myasthenia-gravis

UCB presents efficacy and safety results for zilucoplan ...

The study met its primary endpoint with zilucoplan showing a placebo-corrected mean improvement of 2.12 points in the Myasthenia Gravis Activities of Daily ...