200 Participants Needed

Zilucoplan for Myasthenia Gravis

(RAISE-XT Trial)

Recruiting at 145 trial locations
Age: 18+
Sex: Any
Trial Phase: Phase 3
Sponsor: UCB Biopharma SRL
Must be taking: Zilucoplan
No Placebo GroupAll 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 DataThis treatment has passed at least one previous human trial
Approved in 2 JurisdictionsThis treatment is already approved in other countries

Trial Summary

What is the purpose of this trial?

This trial is testing zilucoplan, a medication for muscle weakness, in patients with generalized Myasthenia Gravis who were in previous studies. It aims to see if the drug is safe and effective over time by calming the immune system to prevent it from attacking muscles. Zilucoplan is currently being tested for its potential to improve disease control in generalized myasthenia gravis.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It only mentions that you must have completed a previous zilucoplan study to participate.

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

Zilucoplan has been approved in Japan, the USA, and the EU for treating generalized myasthenia gravis in adults who are positive for anti-acetylcholine receptor antibodies and do not respond well to other treatments. A study showed that Zilucoplan is effective and well-tolerated in these patients, providing a new option for those with this challenging condition.12345

What safety data exists for Zilucoplan in humans?

Zilucoplan has been studied for safety in patients with generalized myasthenia gravis, showing it is generally well tolerated when self-administered under the skin. It has been approved in Japan, the USA, and the EU, indicating regulatory bodies have found its safety profile acceptable for use in adults with this condition.12678

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

Zilucoplan is unique because it is a self-administered injection that specifically targets and inhibits complement component 5 (C5), a part of the immune system involved in the disease process, offering a new option for patients who do not respond well to traditional treatments like steroids or other immunosuppressants.1291011

Research Team

UC

UCB Cares

Principal Investigator

001 844 599 2273

Eligibility Criteria

This trial is for individuals with generalized Myasthenia Gravis (gMG) who have already completed a qualifying study involving zilucoplan. They should not be part of any other clinical trials testing new treatments, although observational studies are okay.

Inclusion Criteria

You have participated in a previous study involving zilucoplan.

Exclusion Criteria

With the exception of a prior zilucoplan trial, participation in another concurrent clinical trial involving an experimental therapeutic intervention (participation in observational studies and/or registry studies is permitted)

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive zilucoplan and are monitored for efficacy, safety, and tolerability

12 weeks
Regular visits as per study protocol

Follow-up

Participants are monitored for safety and effectiveness after treatment

up to 36 months
Safety Follow-Up Visit

Open-label extension

Participants continue to receive zilucoplan to evaluate long-term efficacy, safety, and tolerability

Long-term

Treatment Details

Interventions

  • Zilucoplan
Trial Overview The RAISE-XT study is testing the long-term effects of zilucoplan on people with gMG. It's an open-label extension, meaning everyone knows they're getting zilucoplan and there's no placebo comparison.
Participant Groups
1Treatment groups
Experimental Treatment
Group I: 0.3 mg/kg zilucoplan (RA101495)Experimental Treatment1 Intervention

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

🇺🇸
Approved in United States as Zilbrysq for:
  • Generalized myasthenia gravis in adults who are anti-acetylcholine receptor (AChR) antibody positive
🇪🇺
Approved in European Union as Zilbrysq for:
  • Generalized myasthenia gravis in adults whose immune system produces antibodies against the acetylcholine receptor

Find a Clinic Near You

Who Is Running the Clinical Trial?

UCB Biopharma SRL

Lead Sponsor

Trials
118
Recruited
23,200+

Jean-Christophe Tellier

UCB Biopharma SRL

Chief Executive Officer since 2015

MD from University of Reims Champagne-Ardenne, Rheumatology specialization from University of Paris V, Executive business programs at Harvard and INSEAD

Dr. Iris Loew-Friedrich

UCB Biopharma SRL

Chief Medical Officer since 2014

MD from University of Leuven, PhD in Medical Sciences from University of Leuven

Ra Pharmaceuticals

Lead Sponsor

Trials
8
Recruited
660+

Ra Pharmaceuticals, Inc.

Lead Sponsor

Trials
3
Recruited
390+

Findings from Research

Zilucoplan, a C5 inhibitor, has been approved in Japan, the USA, and the EU for treating generalized myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) antibody positive and have not responded adequately to other treatments.
This medication represents a significant advancement in gMG treatment options, particularly for patients who do not respond well to steroids or immunosuppressants, and is currently under review in Australia and Canada.
Zilucoplan: First Approval.Shirley, M.[2023]
In a phase 3 trial involving 174 patients with AChR-positive generalized myasthenia gravis, zilucoplan demonstrated a significant reduction in the myasthenia gravis activities of daily living (MG-ADL) score compared to placebo, indicating its efficacy as a treatment option.
Zilucoplan was generally well tolerated, with a safety profile similar to placebo, and the most common side effect was injection-site bruising, suggesting it may be a safe option for patients with this condition.
Safety and efficacy of zilucoplan in patients with generalised myasthenia gravis (RAISE): a randomised, double-blind, placebo-controlled, phase 3 study.Howard, JF., Bresch, S., Genge, A., et al.[2023]
In a phase III trial involving 176 patients with myasthenia gravis, mycophenolate mofetil (MMF) did not show superior efficacy compared to placebo in achieving minimal manifestations or pharmacologic remission while tapering corticosteroids over 36 weeks.
Both MMF and placebo groups experienced similar rates of adverse events, indicating that MMF is well tolerated, but it did not provide additional benefits in disease control compared to placebo.
An international, phase III, randomized trial of mycophenolate mofetil in myasthenia gravis.Sanders, DB., Hart, IK., Mantegazza, R., et al.[2022]

References

Zilucoplan: First Approval. [2023]
Safety and efficacy of zilucoplan in patients with generalised myasthenia gravis (RAISE): a randomised, double-blind, placebo-controlled, phase 3 study. [2023]
An international, phase III, randomized trial of mycophenolate mofetil in myasthenia gravis. [2022]
When myasthenia gravis is deemed refractory: clinical signposts and treatment strategies. [2022]
Long-term effects of combined immunosuppressive treatment on myasthenic crisis. [2015]
A Sum Score to Define Therapy-Refractory Myasthenia Gravis: A German Consensus. [2022]
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]
Measuring treatment adverse event burden in myasthenia gravis: Single-center prospective evaluation utilizing the Adverse Event Unit (AEU). [2023]
[Efficacy and safety of low-dose cyclophosphamide plus corticosteroids for type I/II myasthenia gravis]. [2013]
[Randomized controlled clinical trial of middle-dose cyclophosphamide plus methylprednisolone for myasthenia gravis patients in crisis]. [2013]
[Actual aspects of myasthenia gravis treatment]. [2006]