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Experimental cell therapy targets rare Muscle-Weakening disease

NCT ID NCT05451212

Disease control Sponsor: Cabaletta Bio 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
Running, but no longer taking on new participants.
Completed This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Jun 27, 2026

Summary

This early-stage study tested a new cell therapy called MuSK-CAART in 7 people with a rare, severe form of myasthenia gravis (MuSK MG). The treatment uses a patient's own immune cells, modified to attack the harmful antibodies causing the disease. The main goal was to check safety and find the right dose, with the hope of achieving long-term remission.

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

7 people

The number who actually took part.

Started

Nov 2022

Finished

Jul 2025

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: * Confirmed diagnosis of MuSK-type MG with at least 1 prior positive anti-MuSK antibody test. * History of a negative anti-AChR (acetylcholine receptor) antibody test. * Positive anti-MuSK antibody test at screening * MG severity Class I to IVa on the MGFA (Myasthenia Gravis Foundation of America) Clinical Classification Exclusion Criteria: * Rituximab in the last 12 months. * Prednisone \> 0.25mg/kg/day \[in Part A\] * Other autoimmune disorder requiring immunosuppressive therapies. * Investigational treatment for MG in the past 12 weeks. * Absolute lymphocyte count \< 500/µL at screening.

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Conditions

The condition(s) this trial relates to.

autoimmune disease immune system disorder Muscle Weakness musculoskeletal system disorder myasthenia gravis neuromuscular disease

As listed by the trial registrant

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

Musk myasthenia gravis

Contacts and locations

Locations

  • Houston Methodist Hospital

    Houston, Texas, 77030, United States

  • Oregon Health & Science University (OHSU)

    Portland, Oregon, 97239, United States

  • UC Davis, Department of Neurology

    Sacramento, California, 95817, United States

  • UC Irvine, Department of Neurology

    Orange, California, 92868, United States

  • University of Kansas Medical Center

    Kansas City, Kansas, 66160, United States

More trials for these conditions

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