RecruitingPHASE1INTERVENTIONAL

A Phase 1b Study to Investigate Safety and Tolerability of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes (CMS)

This research study is about a new medicine called ARGX-119 for adults who have a rare muscle condition called DOK7-Congenital Myasthenic Syndrome (CMS). The main goal is to check if ARGX-119 is safe and how well people can tolerate it. Researchers will also learn how the body handles the medicine and if it might improve health and daily activities for those with DOK7-CMS. Participants will either receive the new medicine or a dummy medicine (placebo) first, without knowing which one they are getting. After a period, everyone will have the chance to receive the new medicine. The study will last for about 38 months.

At a glance

Status

Recruiting

Phase

PHASE1

Sponsor

argenx

Enrolment target

Start

24 Sep 2024

Estimated completion

24 Jan 2028

Congenital Myasthenic Syndrome CMS

What is this study about?

You might be reading this because you, or someone you know, has a condition called DOK7-Congenital Myasthenic Syndrome (CMS). This is a rare, inherited condition that causes muscle weakness. Scientists are always working to find new and better ways to treat conditions like yours, and this study is part of that effort.

This study is testing a new medicine called ARGX-119. It's an early-stage study, meaning it's one of the first times this medicine is being given to people. The main things researchers want to find out are whether the medicine is safe and if people can take it without too many problems. They'll also be looking at how your body processes the medicine and if your immune system reacts to it. Importantly, the study will also try to see if ARGX-119 helps improve how you feel and what you can do each day.

Understanding how new medicines work is a long process, and studies like this are a vital step. By taking part, you could help researchers learn more about DOK7-CMS and contribute to future treatments for this condition.

Key takeaways

This is a study of a new medicine, ARGX-119, for DOK7-Congenital Myasthenic Syndrome (CMS).
Its main aim is to check if the medicine is safe and well-tolerated by adults.
Participants will receive either the study medicine or a dummy medicine (placebo) first.
The total study time will be about 38 months (just over 3 years).
Your participation could help advance understanding and treatment for DOK7-CMS.
You can stop participating at any time if you change your mind.

Who may be eligible?

To join this study, you need to be an adult, at least 18 years old, and have a confirmed diagnosis of DOK7-Congenital Myasthenic Syndrome. This diagnosis must be based on a genetic test showing changes in the DOK7 gene.

If you take certain oral medicines for your breathing, like albuterol or salbutamol (often called 'beta agonists'), you must have been taking them for at least three months and be willing to continue on the same dose throughout the study period.

There are also some reasons why you might not be able to join. For example, if your CMS is caused by a different genetic problem. You also can't take part if you have other serious medical conditions that might make it hard to tell if the study medicine is working, or if it could put you at risk. This includes having certain types of cancer that haven't been cured for more than five years. Pregnant or breastfeeding women, or those planning to become pregnant, cannot participate.

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.

Are you 18 years old or older?
Do you have a genetically confirmed diagnosis of DOK7-Congenital Myasthenic Syndrome?
If you take certain oral breathing medicines (like albuterol), have you been on the same dose for at least 3 months and can you stay on it?
Are you not pregnant, breastfeeding, or planning to become pregnant during the study?
Do you have any other serious medical conditions that a doctor might consider risky for a study?
Have you been clear of most cancers for at least 5 years (some minor cancers are allowed)?

Answer every question to see your result.

What does participation involve?

If you decide to join this study, you'll first go through a screening process to make sure the study is right for you. If you're eligible, you'll then be randomly assigned to receive either the new medicine, ARGX-119, or a dummy medicine called a placebo. Neither you nor your study doctor will know which you are receiving during this first part of the study; this is called 'double-blinded'. These medicines are given through a drip into your arm (intravenous infusions).

After this first treatment period, there will be a follow-up period. Once that's complete, you might be able to join an 'open-label' period, where everyone receives ARGX-119, and both you and the study team will know you're getting the active medicine. The full study, from start to finish, will take approximately 38 months, which is just over three years.

Potential risks and benefits

Taking part in any medical study has potential benefits and risks. You might feel better or experience an improvement in your symptoms with the study medicine, but there's no guarantee it will help you. There's also a chance that the medicine could cause side effects. These could be mild or, in rare cases, more serious. All risks and potential benefits will be explained in detail before you decide to join. Remember, taking part in a study is always voluntary, and you can change your mind and withdraw at any time, for any reason, without it affecting your usual medical care.

Locations (9)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

UC Davis Medical Center
Verified postcode
Sacramento, United States· Active not recruiting
Ann and Robert H Lurie Childrens Hospital of Chicago
Verified postcode
Chicago, United States· Recruiting
Ottawa Hospital Research Institute - Civic Campus
Verified postcode
Ottawa, Canada· Recruiting
CHU - Hospital de la Timone
Verified postcode
Marseille, France· Completed
Group Hospitalier Pitie-Salpetriere
Verified postcode
Paris, France· Recruiting
Fondazione IRCCS Istituto Neurologico Carlo Besta
Verified postcode
Milan, Italy· Recruiting
Universitat de Valencia - Hospital Universitari i Politecnic La Fe de Valencia (Hospital La Fe Bulevar Sur)
Verified postcode
Valencia, Spain· Active not recruiting
Clinical Trials Centre - South Eastern Health and Social Care Trust - The Ulster Hospital
Verified postcode
Belfast, United Kingdom· Recruiting
John Radcliffe Hospital - Oxford University Hospitals NHS Foundation Trust
Verified postcode
Oxford, United Kingdom· Active not recruiting

Common questions

What is DOK7-Congenital Myasthenic Syndrome (CMS)?

It's a rare, inherited condition that causes muscle weakness throughout the body, starting at or around birth.

What does 'Phase 1b Study' mean?

It means this is an early-stage study primarily focused on checking the safety of the new medicine and finding the right dose in people who have the condition.

Why is a placebo used in the study?

Using a placebo helps researchers fairly compare the new medicine's effects against not receiving the active medicine, giving a clearer picture of its true benefits and side effects.

Will I have to pay to be in the study?

No, you will not have to pay for the study medicine or study-related visits and tests.

What if I get pregnant during the study?

If you are or become pregnant, you cannot participate in this study. It's important to use effective birth control methods if you are able to become pregnant.

How to find out more

Sabine Coppieters, MD

clinicaltrials@argenx.com 857-350-4834 Official trial record

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