Active not recruitingPHASE2INTERVENTIONAL

A Clinical Trial to Investigate the Long-term Safety and Tolerability, Efficacy, Pharmacodynamics, Pharmacokinetics, and Immunogenicity of ARGX-117 in Adults With Multifocal Motor Neuropathy

Multifocal motor neuropathy (MMN) is a rare condition that causes muscle weakness, usually starting in the hands and arms. This Phase 2 clinical trial is looking at an experimental medicine called ARGX-117 to see if it is safe and works well over a longer period. The trial is an 'extension study'. This means it is only open to adults who have already taken part in and completed an earlier study testing the same medicine. During the trial, participants receive the treatment through a drip into a vein. The study includes a period where some people receive ARGX-117 and others receive a dummy medicine (placebo), followed by a stage where everyone receives ARGX-117. Doctors will track muscle strength, nerve health, and side effects to learn whether this treatment could offer a good long-term option for people living with MMN.

At a glance

Status

Active not recruiting

Phase

PHASE2

Sponsor

argenx

Enrolment target

Start

18 Jan 2023

Estimated completion

30 Sep 2029

Multifocal Motor Neuropathy (MMN)

What is this study about?

Multifocal motor neuropathy (MMN) is a rare condition affecting the nerves that control your muscles. It typically leads to weakness in the hands, wrists, and arms, which can make everyday actions like holding cutlery or writing difficult. The condition happens when the body's immune system mistakenly attacks its own motor nerves. Because there are currently very few treatments available, finding new ways to protect these nerves is essential.

This study tests an experimental drug called ARGX-117. The medicine is designed to calm down the specific part of the immune system causing nerve damage. Researchers want to know whether giving ARGX-117 over a longer period is safe, easy to tolerate, and helpful in keeping muscles strong.

Because this is an extension study, it builds directly on an earlier trial. It helps doctors see what happens when patients stay on the treatment for an extended period. By measuring how the drug moves through the body and checking nerve function regularly, scientists hope to learn if ARGX-117 could become a reliable long-term treatment.

Key takeaways

Tests the long-term safety and benefits of a new medicine called ARGX-117.
Open only to adults who completed the previous ARGX-117-2002 trial.
Given as a drip into a vein at regular specialist hospital appointments.
Includes a stage where every participant receives the active drug.
Participation is entirely voluntary and you can stop at any time.

Who may be eligible?

This study is only open to adults aged 18 or older who have already taken part in and completed the main treatment stage of the previous trial (called ARGX-117-2002). Participants must also be able to read, write, and provide their own informed consent.

You cannot join this study if you have a serious, active infection caused by bacteria, viruses, or fungi, or if you have recently had major surgery. It is also not suitable for people with other severe medical conditions that could make taking part unsafe.

In addition, you cannot take part if you are involved in any other clinical drug trials. For safety reasons, women who are pregnant, breastfeeding, or planning to become pregnant during the study or within 15 months after their final dose cannot take part.

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.

I am aged 18 or older.
I completed the treatment period of the earlier ARGX-117-2002 trial.
I do not have a serious active or long-term infection.
I have not recently had major surgery.
I am not pregnant, breastfeeding, or planning a pregnancy in the next 15 months.

Answer every question to see your result.

What does participation involve?

If you take part, you will receive your study medication through a drip into a vein (an intravenous infusion) at a hospital clinic. The trial begins with a period where neither you nor your doctor will know if you are receiving ARGX-117 or a dummy treatment (placebo). Later in the study, there is an open-label period where all participants receive the active medicine ARGX-117. You will attend regular clinic visits for blood tests, nerve and muscle checks, and general health reviews. After treatment finishes, there will be a safety follow-up period lasting several months to check on your health.

Potential risks and benefits

Taking part may help improve your muscle strength and nerve function, but there is no guarantee that you will directly benefit. There is also a chance of side effects from the study drug or mild discomfort from the drip and blood tests. You will be closely monitored by a specialist medical team throughout the study. Joining is completely voluntary, and you are free to leave the trial at any time without your regular NHS care being affected.

Locations (27)

HonorHealth Neurology
Verified postcode
Scottsdale, United States
NorthShore University HealthSystem
Verified postcode
Glenview, United States
University of Minnesota
Verified postcode
Minneapolis, United States
The Cleveland Clinic Foundation
Verified postcode
Cleveland, United States
University of Pennsylvania - Perelman Center for Advanced Medicine
Verified postcode
Philadelphia, United States
Austin Neuromuscular Center
Verified postcode
Austin, United States
Medizinische Universitat Wien (Medical University of Vienna)
Verified postcode
Vienna, Austria
AZ Sint-Lucas
Verified postcode
Ghent, Belgium
University Health Network
Verified postcode
Toronto, Canada
CHU Bordeaux - Groupe Hospitalier Pellegrin
Verified postcode
Bordeaux, France
Hôpital Roger Salengro (CHU de Lille)
Verified postcode
Lille, France
CHU de Nice
Verified postcode
Nice, France

Common questions

What is an extension study?

An extension study allows people who finished an earlier trial to continue receiving treatment and medical checks, helping doctors see how the medicine works over a longer time.

Will I definitely receive the real medicine?

During the first part, some people receive a dummy drug (placebo) and some receive ARGX-117. Later in the study, all participants receive the real medicine.

How is the treatment given?

The medicine is given as a liquid through a small tube into a vein in your arm (an intravenous drip) during your clinic visits.

Does taking part cost anything?

No. The study treatment and tests are provided free of charge, and travel expenses to and from the clinic are usually covered.

Can I leave the study if I change my mind?

Yes. You can leave the study at any time without giving a reason, and your normal medical treatment will continue as usual.