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Study on the Safety and Effectiveness of Rozanolixizumab in Children with Generalized Myasthenia Gravis

Fast replyRegistered drugNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a condition called Generalized Myasthenia Gravis, which is a chronic autoimmune disease that causes muscle weakness. The study is investigating a treatment called Rozanolixizumab, also known by its code name UCB7665. Rozanolixizumab is given as a solution for injection under the skin, a method known as subcutaneous use. The purpose of the study is to assess the safety and how well this treatment is tolerated in children aged 2 to under 18 years who have moderate to severe generalized myasthenia gravis.

Participants in the study will receive Rozanolixizumab over a period of six weeks. During this time, researchers will monitor how the drug moves through the body and its effects on the disease. The study will also look at any side effects that may occur, including serious ones that might lead to stopping the treatment. Additionally, the study will measure changes in certain proteins in the blood, known as immunoglobulins, and specific antibodies related to myasthenia gravis. These measurements will help determine if the treatment is working and how it affects the participants' daily activities and overall condition.

The study is designed to last up to 18 weeks, including the treatment and observation periods. Throughout the study, researchers will evaluate the local tolerability of the treatment, which means they will check how the skin and body react to the injections. The study aims to provide valuable information on the potential benefits and risks of using Rozanolixizumab in young patients with generalized myasthenia gravis, contributing to better understanding and management of this condition.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, the participant is required to have a documented diagnosis of generalized myasthenia gravis (gMG).

    The participant must be between 2 and 18 years old and have previously received conventional treatments for gMG.

  2. Step 2

    Treatment administration

    The participant will receive rozanolixizumab, a solution for injection, administered subcutaneously.

    The treatment is designed to assess the safety and tolerability of rozanolixizumab in pediatric participants.

  3. Step 3

    Treatment period

    The treatment period lasts for 6 weeks, during which the participant will receive regular doses of rozanolixizumab.

    The participant's response to the treatment will be monitored, including any changes in symptoms and potential side effects.

  4. Step 4

    Observation period

    Following the treatment period, there is an observation period to monitor the participant's condition and any delayed effects of the treatment.

    The participant will be assessed for any adverse events and changes in their condition.

  5. Step 5

    End of study visit

    At the end of the study, a final visit will be conducted to evaluate the overall impact of the treatment.

    The occurrence of any serious treatment-emergent adverse events will be documented up to this point.

Who can join the trial?

5 criteria

  • The participant must be between the ages of 2 and 18 years old at the time of signing the consent form.
  • The participant must have a confirmed diagnosis of generalized Myasthenia Gravis (gMG). This includes having specific antibodies related to the condition, which are proteins in the blood that can affect muscle function.
  • The participant must be classified as having Myasthenia Gravis Foundation of America (MGFA) Clinical Classification II to IVa. This is a way to describe the severity of the condition, with II to IVa indicating moderate to more severe symptoms.
  • The participant must have tried existing treatments for gMG, such as pyridostigmine, corticosteroids, or immune suppressants, before joining the study.
  • The participant must have experienced unsatisfactory results or worsening symptoms from their current treatment and need additional therapy. This could include treatments like plasma exchange (PEX) or intravenous immunoglobulin g (IVIg), which are methods to help manage symptoms.

Who cannot join the trial?

8 criteria

  • Participants who are not within the age range of 2 to 18 years old.
  • Participants who do not have a diagnosis of Generalized Myasthenia Gravis, a condition that affects the muscles and causes weakness.
  • Participants who have any other medical condition that might interfere with the study or the treatment being tested.
  • Participants who are unable to follow the study procedures or instructions.
  • Participants who have taken certain medications that might affect the study results.
  • Participants who have a history of allergic reactions to similar treatments.
  • Participants who are pregnant or breastfeeding.
  • Participants who have participated in another clinical trial recently.
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Investigated drugs

Rozanolixizumab is a medication being studied for its effects on children and teenagers with moderate to severe generalized myasthenia gravis, a condition that causes muscle weakness. The study aims to understand how safe and tolerable this medication is when given as an injection under the skin.

What is already known about the treatment

Rozanolixizumab – This medication is administered subcutaneously, meaning it is injected under the skin. It is currently being studied in clinical trials for its safety and effectiveness in treating generalized myasthenia gravis, particularly in pediatric patients. Rozanolixizumab works by targeting and reducing the activity of certain antibodies that are involved in the autoimmune response associated with myasthenia gravis. It is classified as a monoclonal antibody, which is a type of medication designed to specifically target and neutralize specific proteins in the body.

Investigated diseases

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 for a neurotransmitter called acetylcholine. This disruption impairs the transmission of signals from nerves to muscles, causing symptoms such as drooping eyelids, difficulty swallowing, and general muscle fatigue. The severity of muscle weakness can vary throughout the day, often worsening with activity and improving with rest. Over time, the condition can affect various muscle groups, including those responsible for breathing and limb movement.
Trial detailsLast updated 7 Oct 2026
Age0-17PhasePhase IVTrial ID2022-502074-16-00Protocol codeMG0006Estimated enrolment12 patientsSponsorUCB Biopharma

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