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Can a new infusion ease muscle weakness in rare genetic disorders?

NCT ID NCT07746089

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 This study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Aug 04, 2026 · Last updated Aug 05, 2026 · Updated 1 time

Summary

This Phase 3 trial is testing whether an experimental drug called adimanebart can help people aged 12 and older with certain congenital myasthenic syndromes (CMS) feel better and do daily activities more easily. Participants are randomly assigned to receive either adimanebart or a placebo, and after that, everyone can receive the drug in an open-label phase. The study measures changes in walking distance and physical function over about 24 weeks.

What this could mean

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

Active substance
Adimanebart given as an intravenous infusion
What this could lead to
If it works, adimanebart could become a new treatment to ease muscle weakness and improve daily activities for people with certain congenital myasthenic syndromes.
What could go wrong
This is an early-stage test in a small group, and results may not hold up. The drug could also cause side effects, and not everyone may respond.

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 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 105 people

The number the study aims to enrol. It can still change while the study runs.

Expected to start

Sep 2026

An estimate. Start dates often move.

Expected to finish

Oct 2030

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

12 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: DBTP: * At least 12 years of age. * Has a diagnosis of DOK7-, MUSK-, AGRN-, or LRP4-CMS with documented mutations. * Participants taking oral beta agonists (eg, albuterol, salbutamol, ephedrine) or other CMS medication must have been receiving the medication for at least 6 months and agree to remain on a same stable dosing regimen of the same medication unless directed to change their CMS medication(s) by their treating physician. OLE: * Completed part of the active-treatment period of ARGX-119-2302. Exclusion Criteria: DBTP: * 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. OLE: * Investigational study drug discontinuation in ARGX-119-2302.

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Conditions

The condition(s) this trial relates to.

congenital myasthenic syndrome

As listed by the trial registrant

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

CMS Congenital myasthenic syndrome

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

More trials for these conditions

Other studies related to the condition(s) this trial covers.