Not yet recruitingRare disease

Study on the Safety and Effects of YTB323 and Drug Combination for Patients with Treatment-Resistant Generalized Myasthenia Gravis

Verified siteRegistered 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 treatment-resistant generalized myasthenia gravis (gMG). This is a chronic autoimmune disease where the body's immune system mistakenly attacks the connections between nerves and muscles, leading to muscle weakness. The study is testing a new treatment called YTB323, which is a type of cell therapy. This treatment involves using a patient's own immune cells, which are modified to better fight the disease, and then infused back into the patient.

The purpose of the study is to assess the safety of YTB323 in patients with gMG who have antibodies against specific proteins called AChR or MuSK. The study will also look at how the treatment affects the body over time. Participants will receive the treatment through an infusion, which is a way of delivering medication directly into the bloodstream. The study will monitor participants for any side effects and changes in their health, including vital signs and laboratory tests.

In addition to YTB323, the study involves other medications such as Fludarabine Phosphate, Tocilizumab, Cyclophosphamide, and Flebogamma DIF, which are used to prepare the body for the main treatment or manage symptoms. The study will take place over several years, with regular check-ups to track the progress and effects of the treatment. Participants will be closely monitored to ensure their safety and to gather information on how well the treatment works in managing the symptoms of gMG.

The research process

The trial runs in 7 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Initial assessment
Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes reviewing medical history and conducting necessary tests to ensure adequate renal, hepatic, cardiac, hematological, and pulmonary function.
A confirmed diagnosis of treatment-resistant generalized myasthenia gravis (gMG) with positive antibodies against AChR or MuSK is required.
Step 2
Pre-treatment phase
Participants may need to adjust current medications, such as tapering corticosteroids to a maximum dose at least one week before leukapheresis.
Leukapheresis is a procedure to collect white blood cells, which is necessary for the preparation of the study treatment.
Step 3
Conditioning regimen
Participants receive a conditioning regimen to prepare the body for the study treatment. This includes the administration of fludarabine phosphate and cyclophosphamide via IV infusion.
Step 4
Administration of study treatment
The study treatment, YTB323, is administered through intravenous use. This is a key part of the study aimed at assessing the safety and efficacy of the treatment.
Step 5
Monitoring and follow-up
Participants are closely monitored for any adverse events, including changes in vital signs, laboratory parameters, ECG, and neurological status.
Regular assessments are conducted to measure the levels of YTB323 in the blood and to evaluate the immune response to the treatment.
Step 6
Supportive care
Supportive care may include the administration of tocilizumab and human normal immunoglobulin (IV) as a solution for infusion to manage potential side effects and support the immune system.
Step 7
Evaluation of outcomes
The effectiveness of the treatment is evaluated by measuring changes in the MG-ADL score and QMG total score over time.
The study aims to determine if participants achieve a significant reduction in symptoms and maintain this improvement for six months.

Who can join the trial?

6 criteria

Must have signed an informed consent form and be able to communicate well with the study doctor and follow the study requirements.
Must be a male or female between 18 and 65 years old with good kidney, liver, heart, blood, and lung function.
Must have a confirmed diagnosis of **generalized myasthenia gravis (gMG)**, which is a condition that affects the muscles, with positive antibodies against **AChR** (acetylcholine receptor) or **MuSK** (muscle-specific kinase) at the start of the study. Additionally, must have one of the following: a history of abnormal muscle tests, a positive test for acetylcholinesterase (a substance that helps muscles work), or improvement in muscle weakness with an oral acetylcholinesterase inhibitor as judged by the doctor.
Must have generalized myasthenia gravis classified as **MGFA Class III-IVa**, which indicates a certain level of muscle weakness, at the start of the study.
Must have treatment-resistant gMG, meaning the condition does not improve with standard treatments. This is defined by a **MG-ADL score** of 6 or higher, which measures daily life activities, despite trying at least two different non-steroidal immunosuppressive drugs. Must have failed treatment with at least one oral immunomodulatory or steroid-sparing drug and either one approved anti-C5 complement antibody, an approved FcRn antagonist, or rituximab, or needed two or more rescue therapies like plasma exchange or IVIg for worsening symptoms in the past year.
If taking long-term corticosteroids, must be able and willing to reduce the dose to a certain level at least one week before a procedure called **leukapheresis**, which involves removing white blood cells from the blood.

Who cannot join the trial?

6 criteria

Patients who do not have treatment-resistant generalized Myasthenia Gravis cannot participate. This means the study is only for those whose condition does not improve with standard treatments.
Patients without antibodies against AChR (acetylcholine receptor) or MuSK (muscle-specific kinase) are excluded. These are specific proteins related to the condition.
Patients who are not within the specified age range for the study cannot participate. The study is for adults only.
Patients who are not part of the specified clinical trial group are excluded. This means the study is for a specific group of patients with certain characteristics.
Patients who are not male or female are excluded, as the study includes both genders.
Patients who are not considered part of a vulnerable population are excluded. This refers to groups that may need special protection in research.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Centre Hospitalier Regional Et Universitaire De Brest
Verified
Brest, France
Centre Hospitalier Universitaire De Bordeaux
Verified
Bordeaux, France
Centre Hospitalier Universitaire De Lille
Verified
Lille, France

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Investigated drugs

YTB323 is an investigational therapy being studied for its safety and effectiveness in patients with treatment-resistant generalized myasthenia gravis. This condition is characterized by muscle weakness and fatigue due to the body's immune system attacking its own muscle receptors. YTB323 is designed to help manage these symptoms in patients who have not responded well to other treatments. The study aims to understand how this therapy works in the body and its potential benefits for patients with this challenging condition.

What is already known about the treatment

YTB323 – YTB323 is an investigational medication currently being studied in clinical trials for its potential use in treating treatment-resistant generalized Myasthenia Gravis, a condition characterized by muscle weakness. It is administered through infusion, allowing the medication to enter the bloodstream directly. The main focus of current research is to evaluate its safety and effectiveness in patients who have antibodies against AChR or MuSK, which are proteins involved in muscle function. YTB323 works by targeting specific immune cells, aiming to reduce the abnormal immune response that contributes to the symptoms of Myasthenia Gravis. As a part of its pharmacological classification, it is considered a type of cellular therapy, which involves modifying or using cells to treat diseases.

Investigated diseases

Myasthenia Gravis – 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 connections between nerves and muscles, specifically targeting receptors that receive nerve signals. This results in symptoms such as drooping eyelids, difficulty swallowing, and general muscle fatigue. The severity of muscle weakness can vary, often worsening with activity and improving with rest. In treatment-resistant generalized Myasthenia Gravis, standard therapies are ineffective, and symptoms persist despite treatment efforts. The condition can significantly impact daily activities and quality of life.