Not yet recruitingRare disease

Study of TOL2 safety and immune response in patients with generalized myasthenia gravis

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What is this trial about?

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

This study focuses on patients with generalized myasthenia gravis, a condition that causes muscle weakness throughout the body. The main treatment being tested is TOL2, which is given through intravenous administration. The study will also use other medications including prednisolone, human albumin solution, glucose, and human normal immunoglobulin as part of the treatment plan.

The purpose of this research is to evaluate if TOL2 is safe and well-tolerated by patients who have myasthenia gravis with positive acetylcholine receptor antibodies. This is the first time this medication is being tested in humans. The study will test both single doses and multiple doses of the medication, comparing it to placebo.

During the study, participants will receive either TOL2 or placebo through intravenous infusions. The study will monitor various aspects of patients' health, including their daily activities, quality of life, and any changes in their condition. Doctors will regularly check blood tests, vital signs, and perform physical examinations to ensure patient safety throughout the treatment period.

The research process

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

Step 1
Initial evaluation
You will undergo evaluation to confirm your diagnosis of generalized myasthenia gravis
Your current MG-ADL score (a measure of daily living activities) must be 5 or higher
Your blood pressure and heart rate will be checked to ensure they are within normal ranges
Step 2
Treatment groups assignment
You will be randomly assigned to receive either TOL2 (the study medication) or a placebo
Neither you nor your doctor will know which treatment you are receiving
The study will test both single doses and multiple doses of the medication
Step 3
Medication administration
You will receive the study medication or placebo through intravenous infusion
You will continue your regular myasthenia gravis medications throughout the study
The medications may include: prednisolone tablets (2.5 mg, 5 mg, or 10 mg), intravenous immunoglobulin, and other standard treatments
Step 4
Monitoring and assessments
Regular blood tests will be performed to monitor your safety
Your heart activity will be checked through ECG (heart rhythm recording)
Physical examinations will be conducted throughout the study
Your myasthenia gravis symptoms will be evaluated using several rating scales
The study team will monitor for any side effects or reactions to the treatment
Step 5
Study completion
The study is expected to run from January 2025 to November 2026
You will be monitored for any changes in your condition throughout the study period
The study will assess if your regular medication doses can be reduced based on your response to treatment

Who can join the trial?

15 criteria

Must provide signed and dated informed consent to participate
Must have diagnosed generalized myasthenia gravis (a condition affecting muscle strength) with:
- Positive blood test for anti-AChR antibodies (proteins that indicate the presence of the condition)
- At least one of these:
  - Previous abnormal nerve and muscle testing
  - Positive response to medication that helps with muscle weakness
  - Shown improvement in symptoms with oral medications
  - Must have a MG-ADL score (a measure of daily living activities) of 5 or higher
  - Must be between 18 and 80 years old
  - Must have normal blood pressure and heart rate, or variations that doctor considers safe
  - Must have suitable veins for blood sampling
  - Must have stable condition and be on stable treatment
  - For women who can become pregnant:
    
    Must use highly effective birth control (less than 1% failure rate) or abstain from sex
    Must continue birth control from 2 weeks before to 4 weeks after treatment
    Cannot donate eggs during treatment and for 3 months after
    For men:
    
    Must use condoms or abstain from sex during treatment and for 3 months after
    Cannot donate sperm during treatment and for 3 months after

Who cannot join the trial?

15 criteria

Patients under 18 years of age
Patients who have had thymectomy (surgical removal of the thymus gland) within the last 12 months
Active or chronic infections including hepatitis B, hepatitis C, HIV, or tuberculosis
Pregnant or breastfeeding women
History of cancer within the past 5 years
Severe kidney disease (patients on dialysis)
Severe liver disease
Uncontrolled heart conditions
Participation in other clinical trials within the past 30 days
Known allergies to the study medication or its components
Use of immunosuppressive medications (medicines that weaken the immune system) other than those allowed in the study
History of substance abuse within the past year
Mental health conditions that could interfere with study participation
Unable to provide informed consent
Any condition that, in the opinion of the study doctor, would make participation unsafe

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Verified sites

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

Karolinska University Hospital
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Solna, Sweden
Klinikum der Technischen Universitaet Muenchen (TUM Klinikum)
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Munich, Germany
Rigshospitalet
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Copenhagen, Denmark

Trial locations

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Investigated drugs

TOL2
is an experimental immune-tolerizing agent being studied for the first time in humans. It is designed to help patients with generalized myasthenia gravis, an autoimmune condition that causes muscle weakness. The medication aims to modify how the immune system responds in patients who have antibodies against acetylcholine receptors (AChR), which are important for muscle function.
Placebo
is an inactive substance that looks identical to the real medication but contains no active ingredients. It is used as a control to help determine if TOL2 is effective.

What is already known about the treatment

TOL2 - An investigational immune tolerising agent being studied in clinical trials for the treatment of generalized myasthenia gravis, specifically in patients who are seropositive for acetylcholine receptor antibodies. The medication is being evaluated in a Phase I/IIa first-in-human trial to assess its safety profile and ability to modulate immune responses. TOL2 represents a novel therapeutic approach aimed at developing immune tolerance in patients with this autoimmune neuromuscular disorder, though its exact mechanism of action and administration route are still under investigation in the current clinical trials. As this is a first-in-human study, the medication is currently experimental and not yet approved for general medical use.

Investigated diseases

Generalized Myasthenia Gravis - A chronic autoimmune disorder that causes muscle weakness and fatigue throughout the body. The condition occurs when the immune system mistakenly attacks the connection between nerves and muscles, specifically targeting acetylcholine receptors at the neuromuscular junction. Symptoms typically include drooping eyelids, double vision, difficulty speaking and swallowing, and weakness in the arms and legs. The weakness tends to worsen with activity and improve with rest. The disease can affect people of any age, but it most commonly begins in women under 40 and men over 60.