Evaluating CABA-201 for treating generalized myasthenia gravis

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This study aims to assess the safety and efficacy of CABA-201, an investigational cell therapy, in patients with generalized myasthenia gravis (gMG). Participants will receive a single dose of CABA-201 in combination with cyclophosphamide and fludarabine. The study will include adults aged 18 to 70 with a confirmed diagnosis of gMG, focusing on those with specific autoantibody profiles. The trial is designed to provide insights into the potential of this novel therapy to improve muscle strength and overall function in affected individuals.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Age ≥18 and ≤70 years of age
* Diagnosis of MG with generalized muscle weakness meeting criteria as defined by the MGFA class II, III , IVa, and IVb.
* Diagnosis of seropositive (autoantibodies AChR, MuSK and/or LRP4) or seronegative MG

Exclusion Criteria:

* Contraindication to leukapheresis
* History of anaphylactic or severe systemic reaction to fludarabine, cyclophosphamide or any of their metabolites
* Active infection requiring medical intervention at screening
* Current symptoms of severe, progressive, or uncontrolled renal, hepatic, hematological, gastrointestinal, pulmonary, psychiatric, cardiac, neurological, or cerebral disease, including severe and uncontrolled infections, such as sepsis and opportunistic infections.
* Concomitant medical conditions that, in the opinion of the investigator, might place the subject at unacceptable risk for participation in this study, interfere with the assessment of the effects or safety of the investigational product or with the study procedures
* Significant lung or cardiac impairment
* Prior solid organ (heart, liver, kidney, lung) transplant or hematopoietic cell transplant

Other protocol-defined inclusion/exclusion criteria may apply.

Where it's running

• University of California Irvine
  Orange, California, United States Recruiting
• UC Davis, Department of Neurology
  Sacramento, California, United States Recruiting
• University of California, San Francisco
  San Francisco, California, United States Recruiting
• University of Colorado
  Aurora, Colorado, United States Recruiting
• Mayo Clinic Florida
  Jacksonville, Florida, United States Recruiting
• Northwestern Memorial Hospital
  Chicago, Illinois, United States Recruiting

Study contacts

• Study coordinator: Cabaletta Bio
• Email: clinicaltrials@cabalettabio.com
• Phone: 267 759 3100

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Impact of efgartigimod on overall disease experience in adults with generalized myasthenia gravis in Italy

  This study looks at real-world use of efgartigimod in adults with generalized myasthenia gravis to see how it affects their daily symptoms and experiences.

  Recruiting at 30 sites argenx · Acquaviva delle Fonti and 29 other locations · NCT07595653

• ADAPT Forward master protocol testing multiple regimens for generalized myasthenia gravis

  This platform tests several drug regimens, including efgartigimod IV and empasiprubart IV, in adults with generalized myasthenia gravis to find safer and more effective treatment combinations.

  Recruiting at 37 sites argenx · Carlsbad, California and 36 other locations · NCT07294170

• Adding empasiprubart to efgartigimod for AChR-antibody positive generalized myasthenia gravis

  This trial will test whether adding empasiprubart to efgartigimod infusions helps people with AChR-antibody positive generalized myasthenia gravis who have only had a partial response to efgartigimod.

  Recruiting at 33 sites Phase 2 argenx · Carlsbad, California and 32 other locations · NCT07284420

• Treatment approaches for AChR-antibody positive generalized myasthenia gravis in Russia.

  This project will look at which treatments adults in Russia with AChR-antibody positive generalized myasthenia gravis receive and how their symptoms and hospitalizations change over time.

  Recruiting at 7 sites AstraZeneca · Kazan' and 6 other locations · NCT07247279

• Zilucoplan for severe gMG flares that require hospitalization.

  This trial will try daily subcutaneous zilucoplan in adults with AChR-positive generalized myasthenia gravis who are hospitalized for severe flares to see if it quickly improves breathing and bulbar symptoms.

  Recruiting Phase 3 Ohio State University · Tampa, Florida and 2 other locations · NCT07215949

• GC012F CAR‑T infusion for refractory generalized myasthenia gravis

  This trial tests a one-time GC012F CAR‑T cell infusion to see if it helps adults (18–75) with refractory generalized myasthenia gravis who have not responded to standard treatments.

  Recruiting Early Phase 1 Tongji Hospital · Wuhan, Hubei · NCT07058298

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.