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New hope for rare muscle disease: ARGX-119 enters human testing

NCT ID NCT06436742

Disease control Sponsor: argenx Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 24, 2026 · Last updated Jul 24, 2026 · Updated 2 times

Summary

This early-stage trial tests a new biologic drug called ARGX-119 in 16 adults with a rare genetic muscle weakness condition called DOK7-congenital myasthenic syndrome (CMS). The study aims to see if the drug is safe and how the body processes it. Participants will receive either ARGX-119 or a placebo by IV infusion, and researchers will monitor side effects and measure changes in walking distance and muscle strength.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
ARGX-119 (a biologic drug given by IV infusion)
What this could lead to
If it works, this could point toward a treatment that improves muscle strength and daily function for people with DOK7-CMS.
What could go wrong
This is a very early Phase 1b trial with only 16 participants, so it is primarily testing safety. It may not show clear benefit, and results may not apply to all CMS types.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

15 people

The number who actually took part.

Started

Sep 2024

Expected to finish

Jan 2028

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * At least 18 years of age. * Has genetically confirmed congenital myasthenic syndromes due to mutation of downstream of kinase 7 (DOK7-CMS). * Participants taking oral beta agonists (eg, albuterol, salbutamol, ephedrine) must have been receiving the medication for more than 3 months and agree to remain on a same stable dosing regimen of the same medication until the end of the study. Exclusion Criteria: * Diagnosis of CMS due to mutation of any gene other than DOK7. * Known medical condition that would interfere with an accurate assessment of CMS, confound the results of the study, or put the patient at undue risk, as assessed by the investigator. * History of malignancy, cancer, unless considered cured by adequate treatment with no evidence of recurrence for more than 5 years. Adequately treated participants with the following cancers can be included at any time: Basal cell or squamous cell skin cancer, Carcinoma in situ of the cervix, Carcinoma in situ of the breast, Incidental histological findings of prostate cancer. * Pregnant or lactating state or intention to become pregnant during the study.

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Conditions

The condition(s) this trial relates to.

congenital myasthenic syndrome congenital myasthenic syndrome 10

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

CMS Congenital myasthenic syndrome

Contacts and locations

Locations

  • Ann and Robert H Lurie Childrens Hospital of Chicago

    Chicago, Illinois, 60611, United States

  • CHU - Hospital de la Timone

    Marseille, 13385, France

  • Clinical Trials Centre - South Eastern Health and Social Care Trust - The Ulster Hospital

    Belfast, BT16 1RH, United Kingdom

  • Fondazione IRCCS Istituto Neurologico Carlo Besta

    Milan, 20133, Italy

  • Group Hospitalier Pitie-Salpetriere

    Paris, 75013, France

  • John Radcliffe Hospital - Oxford University Hospitals NHS Foundation Trust

    Oxford, OX3 9DU, United Kingdom

  • Ottawa Hospital Research Institute - Civic Campus

    Ottawa, K1Y 4E9, Canada

  • UC Davis Medical Center

    Sacramento, California, 95817, United States

  • Universitat de Valencia - Hospital Universitari i Politecnic La Fe de Valencia (Hospital La Fe Bulevar Sur)

    Valencia, 46026, Spain

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