RecruitingPHASE3INTERVENTIONAL

Efficacy and Safety of a New Formulation of Oral Cladribine Compared With Placebo in Participants With Generalized Myasthenia Gravis (MyClad)

This important study is looking at a new pill form of a medicine called cladribine for people who have generalized myasthenia gravis. Myasthenia gravis is a condition that causes muscles to weaken. The main goal is to find out if this new cladribine pill works better than a dummy pill (placebo) to improve symptoms, and if it's safe to use. Researchers are also interested in understanding if the positive effects of the medicine last over time, if people might need to take it again, and what the long-term safety looks like. We'll also be studying how the body handles this new medicine. The study involves different stages, including a period where some people get the cladribine and others get a placebo, followed by longer-term follow-up periods. Some participants will also be asked about their experiences with the treatment and the study.

At a glance

Status

Recruiting

Phase

PHASE3

Sponsor

Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, Germany

Enrolment target

264

Start

25 Jun 2024

Estimated completion

12 Nov 2030

Generalized Myasthenia Gravis

What is this study about?

This research study, called MyClad, is focused on a health condition called generalized myasthenia gravis (gMG). If you have gMG, your muscles can become weak and tired, which can affect things like your eyes, face, throat, and limbs. Researchers are testing a new version of a medicine called cladribine, which comes as a pill. They want to find out two main things: first, if this new pill helps to improve the symptoms of gMG more effectively than a dummy pill (placebo), and second, if it's generally safe for people to take.

Beyond just looking at how well it works and its safety in the short term, the study also aims to understand the longer-term picture. This includes seeing if the positive effects of cladribine continue over time, whether people might need to take the medicine again later on, and how safe it is when used for a longer period. There's also a part of the study that looks at how your body processes and uses this new cladribine pill. This helps scientists understand the medicine better.

The study is set up in a few parts. Initially, some people will receive the cladribine pill, while others will receive a placebo. Neither you nor your doctor will know which one you are getting. After this initial period, there will be longer follow-up phases to continue observing the medicine's effects. Additionally, a small group of participants will be invited for interviews to share their personal experiences with the treatment and what it's like to be part of the study. This helps researchers understand the human side of the treatment.

Key takeaways

This study is testing a new pill for generalized myasthenia gravis.
It aims to see if the medicine, cladribine, is safe and effective compared to a dummy pill.
Researchers are also looking at long-term benefits and how the medicine works in your body.
Participation involves taking medication, regular check-ups, and potentially sharing experiences.
You might receive the active medicine or a placebo, and neither you nor your doctor will know which during the initial phase.

Who may be eligible?

To join this study, you must be at least 18 years old and have been diagnosed with generalized myasthenia gravis, where your muscles throughout your body are affected. This diagnosis needs to fit certain medical classifications, and your myasthenia gravis should involve general muscle weakness, not just in your eyes.

You'll also need certain symptom scores for myasthenia gravis that show your condition is stable and within a specific range, with a good portion of your symptoms not being just eye-related. If you're currently taking certain medications like steroids or medicines for myasthenia gravis (like pyridostigmine), you need to have been on a steady dose for at least three months, and those doses must be below a certain level. You also need to weigh at least 40 kilograms.

However, there are reasons you might not be able to join. For example, if you have other immune system conditions besides myasthenia gravis, or if you need to take steroid medicines for a long time for another health issue (except for a well-managed thyroid condition), you might not be eligible. The study also has other specific criteria that your doctor will review to make sure it's the right fit for you.

Quick self-check

Are you 18 years old or older?
Have you been diagnosed with generalized myasthenia gravis (not just eye symptoms)?
Are your myasthenia gravis symptoms stable (not getting much worse or better suddenly)?
If you take steroids or other MG medications, have you been on a steady dose for 3 months, and is that dose within specific limits?
Do you weigh at least 40 kilograms?

This is a guide only — the research team will confirm whether you can take part.

What does participation involve?

If you decide to join this study, you will go through different periods. Initially, you'll be in the 'double-blind' phase, meaning neither you nor your study doctor will know if you're receiving the new cladribine medication or a dummy pill (placebo). This helps make sure the study results are fair. This main part of the study will involve regular visits to the clinic, where you'll have check-ups, answer questions about your symptoms, and have blood tests. You will be taking the study medication, which is a pill, according to a specific schedule.

After this initial period, there will be further extension periods where the study continues, and more long-term effects are monitored. You might have continued clinic visits and assessments, and some participants might have the opportunity to receive cladribine if they were initially on the placebo. The study will look at your progress, how your symptoms change, and your general health over time. There will also be a focus on understanding how your body handles the medicine through specific tests (pharmacokinetics). Some participants will also be asked to share their experiences in interviews. The total duration of your involvement could be several years, given the focus on long-term safety and effectiveness.

Potential risks and benefits

Taking part in any medical study has potential benefits and risks. A potential benefit of joining this study is that you might receive access to a new medication, cladribine, which could potentially improve your myasthenia gravis symptoms if it proves to be effective. You would also be contributing to important medical research that could help others with myasthenia gravis in the future. However, there's also a chance you might receive the placebo, which means you wouldn't get the active medicine. As with any medication, cladribine may have side effects, which the study will carefully monitor. These risks will be explained in detail by the study team. You have the right to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (138)

Arizona Neuroscience Research, LLC
Phoenix, United States· Recruiting
Advanced Neurosciences Research LLC
Longmont, United States· Recruiting
University of Connecticut Health Center - Department of Medicine
Farmington, United States· Recruiting
The George Washington University Medical Faculty Associates Foggy Bottom South Pavilion
Washington D.C., United States· Recruiting
Neurology of Central Florida Research Center, LLC
Altamonte Springs, United States· Recruiting
SFM Clinical Research, LLC
Boca Raton, United States· Recruiting
University of Florida Health Science Center - 300120311
Jacksonville, United States· Recruiting
Neurology Associates, P. A.
Maitland, United States· Recruiting
University of South Florida - PARENT - PARENT
Tampa, United States· Recruiting
University of Kansas Medical Center Research Institute, Inc. - 3901 Rainbow (MAIN)
Kansas City, United States· Recruiting
UNC Hospitals - PARENT
Chapel Hill, United States· Recruiting
Atrium Health Wake Forest Baptist
Winston-Salem, United States· Recruiting

+126 more sites — see the official record for the full list.

Common questions

What is generalized myasthenia gravis (gMG)?

It's a condition where your immune system mistakenly attacks connections between your nerves and muscles, causing muscle weakness throughout your body.

What is cladribine?

Cladribine is a medication that affects your immune system, and in this study, it's being tested in a new pill form for gMG to see if it can help reduce muscle weakness.

What does 'placebo' mean?

A placebo is a dummy pill that looks like the active medicine but contains no active ingredients. It's used to fairly compare the new medicine's effects.

Will I know if I'm getting the actual medicine or the dummy pill?

No, during the main part of the study, neither you nor your doctor will know. This is called 'double-blind' and helps ensure the study results are unbiased.

How long will I be in the study?

The study involves different periods, including initial treatment and longer-term follow-up, which could mean you participate for several years to track long-term effects.

How to find out more

US Medical Information

eMediUSA@emdserono.com 888-275-7376 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.