Study on Long-Term Safety and Effectiveness of Zilucoplan in Adults with Generalized Myasthenia Gravis

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What is this study about?

This clinical trial is focused on studying the long-term safety and effectiveness of a treatment called zilucoplan sodium for individuals with generalized myasthenia gravis. Generalized myasthenia gravis is a chronic autoimmune disease that causes muscle weakness. The treatment being tested, zilucoplan sodium, is administered as a solution for injection using a pre-filled syringe. It is designed to be self-administered by patients through a subcutaneous injection, which means it is injected under the skin.

The purpose of this study is to evaluate how safe and tolerable zilucoplan sodium is for patients who have already participated in a previous study involving this medication. Participants will receive the treatment over an extended period to monitor its effects and any potential side effects. The study will not involve any new medications or diseases, focusing solely on zilucoplan sodium and generalized myasthenia gravis.

Throughout the study, participants will be regularly assessed to ensure their well-being and to gather information on how the treatment impacts their daily activities and quality of life. The study aims to provide valuable insights into the long-term use of zilucoplan sodium for managing symptoms of generalized myasthenia gravis.

1 joining the study

Participation begins after completing a previous study involving zilucoplan.

Informed consent is required, which involves signing and dating a consent form.

Vaccination against meningococcal infections is necessary, including a quadrivalent vaccine and, if available, a serotype B vaccine. A booster may be needed based on local guidelines.

2 initial health checks

Female participants of childbearing potential must undergo a urine pregnancy test within 24 hours before the first dose of the study drug.

All participants must agree to use effective contraception during the study and for 40 days after the last dose.

3 medication administration

Zilucoplan sodium is administered as a solution for injection using a pre-filled syringe.

The route of administration is subcutaneous, meaning the injection is given under the skin.

4 monitoring and assessments

The primary focus is on the incidence of treatment-emergent adverse events (TEAEs).

Secondary assessments include changes from baseline to Week 12 in various scores related to myasthenia gravis, such as the Myasthenia Gravis-Activities of Daily Living (MG-ADL) Score, Quantitative Myasthenia Gravis (QMG) Score, Myasthenia Gravis Composite (MGC) Score, and Myasthenia Gravis – Quality of Life revised (MG-QOL15r) Score.

5 study duration

The study is an open-label extension, meaning it continues from a previous study and participants know they are receiving zilucoplan.

The estimated end date for the study is May 20, 2026.

Who Can Join the Study?

You must have completed a previous study involving the drug zilucoplan.
You need to be able to understand and agree to the study details, which includes signing a form called the informed consent form.
You must have received a vaccine that protects against a type of bacteria called meningococcal, which can cause serious infections. This includes a specific vaccine for a type called serotype B, if available. If you have been vaccinated before, you might need a booster shot, which is an additional dose to keep you protected.
If you are a woman who can have children, you must have a negative pregnancy test within 24 hours before taking the first dose of the study drug.
If you are a woman who can have children or a man who can father children, you must agree to use effective birth control during the study and for 40 days after the last dose of the study drug. This is to prevent pregnancy during the study.

Who Cannot Join the Study?

Patients who have not completed a previous clinical study with the medication called zilucoplan.
Patients who are part of a vulnerable population, which means groups of people who might need special protection or care.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
ClinHouse Centrum Medyczne	Zabrze	Poland
Oslo Universitetssykehus HF	Oslo	Norway
Galen Clinic	Lublin	Poland
Neurologia Slaska Centrum Medyczne	Katowice	Poland
NZOZ Neuro-Kard Ilkowski i Partnerzy Spółka Partnerska Lekarzy	Poznan	Poland

Other Sites

Site Name	City	Country
Novo-Med Zielinski I Wspolnicy Sp. j.	Katowice	Poland
Amicare Sp. z o.o. S.K.	Lodz	Poland
Mtz Clinical Research Powered By Pratia	Warsaw	Poland
Hospital Universitario Basurto	Bilbao	Spain
Twoja Przychodnia Nowosolskie Centrum Medyczne Sp. z o.o.	Nowa Sol	Poland
Specjalistyczne Gabinety Sp. z o.o.	Cracow	Poland
Krakowska Akademia Neurologii Sp. z o.o.	Cracow	Poland
Fondazione I.R.C.C.S. Istituto Neurologico Besta	Milan	Italy
Mjtqtlrgr i Pcwadnbnu Lqpyjvl Sgrqru Pvgkhlqmdyf	Cracow	Poland
Hmjqbuvt Dr Lx Sxgba Cmdv I Shaz Pzq	Barcelona	Spain
Hgvzhjxd Vyxw dufhxmaz	Barcelona	Spain

Trial status

Country	Status	Recruitment Start
Italy	Not recruiting	23.12.2019
Norway	Not recruiting	23.12.2019
Poland	Not recruiting	23.12.2019
Spain	Not recruiting	23.12.2019

Trial locations

Investigated drugs:

ZILUCOPLAN

Zilucoplan is a medication being studied for its long-term safety and tolerability in patients with generalized myasthenia gravis (gMG). It is designed to help manage the symptoms of this autoimmune disorder, which affects the communication between nerves and muscles, leading to muscle weakness. This study is an extension of previous research to further understand how well patients tolerate the medication over an extended period.

Investigated diseases:

Myasthenia gravis

Generalized Myasthenia Gravis – Generalized Myasthenia Gravis is a chronic autoimmune disorder that affects the communication between nerves and muscles, leading to muscle weakness. It occurs when the immune system mistakenly attacks the body’s own tissues, specifically targeting the receptors that receive nerve signals. This results in fluctuating muscle weakness and fatigue, which can affect various muscle groups, including those responsible for eye movement, facial expression, and swallowing. The severity of symptoms can vary throughout the day, often worsening with activity and improving with rest. Over time, the condition may lead to more persistent muscle weakness, impacting daily activities and quality of life.

Trial ID:

2024-512399-37-00

Protocol code:

RA101495-02.302

NCT ID:

NCT04225871

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on Long-Term Safety and Effectiveness of Zilucoplan in Adults with Generalized Myasthenia Gravis

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