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ZILUCOPLAN

Clinical trials of ZILUCOPLAN are studying its safety, long-term tolerability, and how it works in people with generalized myasthenia gravis. The research includes children, teens, and adults, and some studies follow participants who already took part in earlier ZILUCOPLAN trials.

Table of Contents

Overview of the studies

These clinical trials are studying ZILUCOPLAN in people with generalized myasthenia gravis (gMG), a disease that causes muscle weakness in several parts of the body.[1][2]

The available trials focus on later stages of research, with Phase 3 and Phase 4 studies listed in the source data.[1][2]

Across the trials, researchers are mainly looking at safety, long-term tolerability, and in some studies how ZILUCOPLAN behaves in the body and how it affects disease-related tests.[1][2]

Who the trials include

One trial is for pediatric participants, meaning children and adolescents, and it includes patients from 2 to under 18 years of age with gMG.[2]

Another pediatric study follows children who already took part in a previous ZILUCOPLAN study, so it is designed as a follow-up for earlier participants.[1]

The adult extension study includes people with gMG who have already completed a qualifying ZILUCOPLAN clinical study.[3]

One additional study listed in the data is a broader myasthenia gravis study that includes ZILUCOPLAN among many other treatments, but its main focus is not ZILUCOPLAN alone.[4]

What the trials measure

The pediatric long-term safety study measures treatment-emergent adverse events (health problems that appear after treatment starts), serious adverse events, treatment stopping because of side effects, and infections.[1]

The pediatric Phase 4 study measures plasma concentrations of ZILUCOPLAN, which means how much of the study drug is found in the blood, at Week 4.[2]

That same study also measures change from baseline in sheep red blood cell (sRBC) lysis and complement component 5 (C5) levels at Week 4, which are laboratory tests used in the study to track biological effects.[2]

The adult extension study measures the incidence of TEAEs, which helps show how often new side effects happen during follow-up treatment.[3]

The broader study that lists ZILUCOPLAN among several treatments measures MG-ADL and QMG changes at 24 weeks in different phases, which are scores used to track daily function and muscle weakness in myasthenia gravis.[4]

Trial phases and status

The pediatric safety follow-up study is in Phase 3 and is marked Authorised with an enrollment of 10 participants.[1]

The pediatric study of blood levels and biological effects is in Phase 4, also marked Authorised, with an enrollment of 10 participants.[2]

The adult long-term extension study is in Phase 3, is Authorised, and has a planned enrollment of 190 participants.[3]

The broader study that includes ZILUCOPLAN alongside other treatments is listed as Phase 4, Authorised, with 66 participants.[4]

Study details by trial

NCT06435312 is a long-term safety study in children with gMG who already joined a previous ZILUCOPLAN study.[1] The brief summary says it is designed to assess safety and tolerability over an extra 52 weeks of daily subcutaneous treatment.[1]

NCT06055959 studies how ZILUCOPLAN moves through the body over time and how safe it is in children with gMG.[2] Its brief summary also says it looks at both pharmacokinetics and pharmacodynamics, which means it studies drug levels in the body and the body’s response to treatment.[2]

NCT04225871 is an adult extension study that looks at long-term safety and tolerability in people with gMG who finished a qualifying ZILUCOPLAN study.[3] Its only listed primary outcome is the incidence of TEAEs.[3]

NCT06193889 is a broader study of anti-CD19 chimeric antigen receptor T-cell therapy in generalized myasthenia gravis, and it lists ZILUCOPLAN among several study drugs.[4] Its primary outcomes in the source data focus on safety, MG-ADL change, and QMG change at 24 weeks for the main study treatment being tested.[4]

What the results could show

These trials may help show whether ZILUCOPLAN can be used safely over time in children and adults with generalized myasthenia gravis.[1][3]

They may also help researchers understand whether the treatment reaches the blood in expected amounts and whether it changes study markers linked to disease activity.[2]

Because the studies are in later phases, they are especially focused on real patient experience, follow-up safety, and longer-term observation rather than early proof-of-concept testing.[1][2][3]

Trial ID Phase Condition studied Status Enrollment
NCT06435312 Phase 3 Generalized Myasthenia Gravis Authorised 10
NCT06055959 Phase 4 Generalized Myasthenia Gravis Authorised 10
NCT04225871 Phase 3 Generalized Myasthenia Gravis Authorised 190
NCT06193889 Phase 4 Generalized Myasthenia Gravis Authorised 66

FAQ

  • What condition are the ZILUCOPLAN trials mainly studying? The main condition is generalized myasthenia gravis, often called gMG. This is a disease that causes muscle weakness and can affect daily activities.
  • Who can take part in these ZILUCOPLAN studies? Some studies are for children from 2 to under 18 years old, while others are for adults with gMG. One adult study includes people who already finished a qualifying ZILUCOPLAN study.
  • What are the main goals of the ZILUCOPLAN trials? The trials look at safety, tolerability, and long-term use. One pediatric study also measures how ZILUCOPLAN moves through the body and how it affects certain blood tests.
  • What phases are the ZILUCOPLAN trials in? The listed studies are in Phase 3 and Phase 4. These later phases usually focus on longer-term safety and on learning more about how well a treatment works in real patients.
  • What kinds of results are being measured in these studies? The studies measure treatment-emergent adverse events, serious adverse events, infections, and whether treatment has to be stopped. Some studies also measure plasma levels, C5 levels, sRBC lysis, and changes in MG-ADL or QMG scores.

Ongoing Clinical Trials on ZILUCOPLAN

  • Study of KYV-101 Therapy for Patients with Refractory Generalized Myasthenia Gravis

    Recruiting

    1 1 1 1
    Investigated diseases:
    Investigated drugs:
    Germany

  • Study on Zilucoplan Sodium for Children Aged 2 to 17 with Generalized Myasthenia Gravis

    Recruiting

    1 1 1 1
    Investigated diseases:
    Investigated drugs:
    Italy Poland

  • Study on the Long-Term Safety of Zilucoplan in Children with Generalized Myasthenia Gravis

    Recruiting

    1 1 1 1
    Investigated diseases:
    Investigated drugs:
    Italy Poland

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

    Not recruiting

    1 1 1 1
    Investigated diseases:
    Investigated drugs:
    Italy Norway Poland Spain

Glossary

  • Generalized myasthenia gravis (gMG): A disease that causes muscle weakness in more than one part of the body. It can affect the eyes, face, arms, legs, and breathing muscles.
  • Phase 3: A later stage of clinical research. It usually studies how safe a treatment is and how well it works in a larger group of patients.
  • Phase 4: A study done after a treatment is already being used more widely. It often looks at long-term safety and how the treatment performs in everyday care.
  • Interventional study: A study where researchers give a treatment or compare treatments to learn about effects on patients.
  • Enrollment: The number of people planned or included in a study.
  • Treatment-emergent adverse event (TEAE): A health problem that appears or gets worse after treatment starts, whether or not the treatment caused it.
  • Serious adverse event (SAE): A serious medical problem during a study, such as one that is life-threatening, causes hospital care, or leads to major disability.
  • Tolerability: How well patients can take a treatment without having too many problems.
  • Pharmacokinetics (PK): How a treatment moves through the body over time, including how much is in the blood.
  • Pharmacodynamics (PD): How a treatment affects the body. In these trials, it includes changes in blood tests linked to immune activity.
  • MG-ADL: A score that measures daily living problems caused by myasthenia gravis, such as speaking, swallowing, and breathing tasks.
  • QMG: A clinical score used to measure muscle weakness in myasthenia gravis.

References