All studies
Active not recruitingPHASE3INTERVENTIONAL

A Study to Assess the Efficacy and Safety of Empasiprubart Versus IVIg in Adults With Multifocal Motor Neuropathy

This research study is investigating a new medicine, empasiprubart, for adults who have Multifocal Motor Neuropathy, also known as MMN. MMN is a rare condition that affects the muscles, leading to weakness. The study wants to compare empasiprubart with a common treatment for MMN, called IVIg (Intravenous Immunoglobulin). Researchers want to find out if empasiprubart is effective and safe to use. The study will involve two main parts: an initial period where participants might receive either empasiprubart or IVIg in a 'blinded' way (meaning neither they nor their doctors know which they're getting), followed by an 'open-label' part where everyone knows they are receiving empasiprubart. Participants could be in the study for up to 49 months.

At a glance

Status
Active not recruiting
Phase
PHASE3
Sponsor
argenx
Enrolment target
154
Start
18 Dec 2024
Estimated completion
01 Dec 2029

What is this study about?

This study is designed to explore a new treatment called empasiprubart for adults diagnosed with Multifocal Motor Neuropathy (MMN). MMN is a long-term condition where the body's immune system mistakenly attacks the nerves that control muscle movement, leading to weakness, typically in the hands and arms, and sometimes the legs. Currently, a common treatment for MMN is IVIg, which helps to calm down the immune system.

The main goal of this study is to compare how well empasiprubart works and how safe it is compared to IVIg. Researchers hope to find out if empasiprubart could be a good new option for people living with MMN. By comparing it to an existing treatment, they can get a clear picture of its potential benefits and any side effects.

The study is split into two parts. In the first part, called 'double-blinded', some participants will receive empasiprubart, and others will receive IVIg. Neither the participants nor their doctors will know which treatment they are getting. This helps to make sure the results are fair and unbiased. The second part is 'open-label', which means everyone involved will know that participants are receiving empasiprubart. The study could last for more than four years for some individuals.

Key takeaways

  • This study is comparing a new medicine (empasiprubart) with a standard one (IVIg) for adults with MMN.
  • It aims to see if the new medicine is safe and effective.
  • The study involves two parts: a 'blinded' comparison and an 'open-label' period.
  • Participants could be in the study for up to 49 months.
  • You must have responded to IVIg in the past and be on a stable IVIg dose to qualify.
  • You can withdraw from the study at any time.

Who may be eligible?

To join this study, you need to be an adult, at least 18 years old, and have a clear diagnosis of Multifocal Motor Neuropathy (MMN). A key requirement is that you have responded well to IVIg treatment for MMN in the past five years. Also, you should currently be receiving regular IVIg infusions, specifically every 2, 3, 4, or 5 weeks, and have been on a stable dose for at least 8 to 10 weeks before the study starts. You'll also need to have certain vaccinations up to date, or be willing to get them before starting the study medicine.

There are also reasons why someone might not be able to join. For example, if you have other serious autoimmune conditions (like lupus) or other medical problems that might make the study results unclear or put you at unnecessary risk. You also wouldn't be able to join if your doctors suspect you have other nerve conditions that are not MMN, such as motor neuron disease (like ALS) or other types of nerve inflammation that only affect sensation.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

  1. Are you at least 18 years old?
  2. Have you been diagnosed with Multifocal Motor Neuropathy (MMN)?
  3. Have you responded to IVIg treatment for your MMN in the last 5 years?
  4. Are you currently receiving regular IVIg infusions every 2, 3, 4, or 5 weeks?
  5. Have you been on a stable dose of IVIg for at least 8 to 10 weeks?
  6. Do you have up-to-date vaccinations for certain bacterial infections, or are you willing to get them?
Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will have regular visits to the clinic. During these visits, doctors and nurses will carry out checks to see how you are doing, including various tests and assessments of your muscle strength and overall health. You will receive either empasiprubart or IVIg as part of a treatment plan. For the first part of the study, you won't know which one you are getting, and neither will your local doctor. There will be continuous follow-up to monitor your health and how the treatment is affecting you. The total time you could be involved in the study can be up to 49 months, which is over four years.

Potential risks and benefits

Taking part in any medical study has potential benefits and risks. Potential benefits of joining this study might include access to a new treatment (empasiprubart) that is not yet widely available, and careful monitoring of your health by a medical team. However, there are also potential risks, such as side effects from the study medicines, which will be fully explained to you. It's important to remember that you can decide to leave the study at any time, for any reason, without it affecting your usual medical care.

Locations (109)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.
  • HonorHealth Neurology - Bob Bove Neuroscience Institute - Neurology
    Verified postcode
    Scottsdale, United States
  • The Neurology Group
    Verified postcode
    Pomona, United States
  • Samir Macwan, M.D., Inc.
    Verified postcode
    Rancho Mirage, United States
  • University of California San Francisco
    Verified postcode
    San Francisco, United States
  • University of Colorado - Anschutz Cancer Pavilion (Neuro Onc Clinic)
    Verified postcode
    Aurora, United States
  • Yale University School of Medicine
    Verified postcode
    New Haven, United States
  • Medstar Health Research Institute
    Verified postcode
    Washington D.C., United States
  • EZR Research LLC
    Verified postcode
    Boca Raton, United States
  • Healthcare Innovations Institute, LLC
    Verified postcode
    Coral Springs, United States
  • University of Miami Miller School of Medicine
    Verified postcode
    Miami, United States
  • University of South Florida
    Verified postcode
    Tampa, United States
  • Northwestern University
    Verified postcode
    Chicago, United States

Common questions

What is Multifocal Motor Neuropathy (MMN)?

MMN is a rare condition where your body's immune system affects nerves that control muscles, causing weakness, often in the hands and arms.

What is IVIg?

IVIg stands for Intravenous Immunoglobulin. It's a common treatment for MMN that helps calm the immune system.

What does 'double-blinded' mean in a study?

It means neither you nor your doctors will know if you're receiving the new medicine or the standard treatment, which helps keep the study fair.

How long will I be in the study?

The maximum time a participant could be in this study is up to 49 months, which is a little over four years.

Can I leave the study early?

Yes, you are free to stop participating in the study at any time, without it affecting your regular medical care.

How to find out more

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

Discussion

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