Evaluating CABA-201 for treating generalized myasthenia gravis
RESET-MG: A Phase 1/2, Open-Label Study to Evaluate the Safety and Efficacy of Autologous CD19-specific Chimeric Antigen Receptor T Cells (CABA-201) in Participants With Generalized Myasthenia Gravis
PHASE1; PHASE2 · Cabaletta Bio · NCT06359041
This study is testing a new cell therapy called CABA-201 to see if it can help adults with generalized myasthenia gravis feel stronger and improve their daily function.
Quick facts
| Phase | PHASE1; PHASE2 |
|---|---|
| Study type | Interventional |
| Enrollment | 12 (estimated) |
| Ages | 18 Years to 70 Years |
| Sex | All |
| Sponsor | Cabaletta Bio (industry) |
| Drugs / interventions | cyclophosphamide, fludarabine |
| Locations | 13 sites (Orange, California and 12 other locations) |
| Trial ID | NCT06359041 on ClinicalTrials.gov |
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.
Who should consider this trial
Good fit: Ideal candidates are adults aged 18 to 70 diagnosed with generalized myasthenia gravis, either seropositive or seronegative.
Not a fit: Patients with contraindications to leukapheresis or severe uncontrolled medical conditions may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could offer a new therapeutic option for patients suffering from generalized myasthenia gravis.
How similar studies have performed: While there have been studies on CAR-T therapies for other conditions, this specific approach for generalized myasthenia gravis is novel and has not been extensively tested.
Eligibility criteria
Show full inclusion / exclusion criteria
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 this trial is running
Orange, California and 12 other locations
- 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 (NOT_YET_RECRUITING)
- Mayo Clinic Florida — Jacksonville, Florida, United States (RECRUITING)
- Northwestern Memorial Hospital — Chicago, Illinois, United States (RECRUITING)
- University of Kansas Medical Center — Kansas City, Kansas, United States (NOT_YET_RECRUITING)
- Mayo Clinic - Rochester — Rochester, Minnesota, United States (RECRUITING)
- Columbia University — New York, New York, United States (RECRUITING)
- University of Rochester Medical Center — Rochester, New York, United States (RECRUITING)
- Oregon Health & Science University — Portland, Oregon, United States (RECRUITING)
- Houston Methodist Hospital — Houston, Texas, United States (RECRUITING)
- Swedish Neuroscience Research — Seattle, Washington, United States (RECRUITING)
Study contacts
- Study coordinator: Cabaletta Bio
- Email: clinicaltrials@cabalettabio.com
- Phone: 267 759 3100
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions: Generalized Myasthenia Gravis, CABA-201, autoimmune disease, anti-CD19 CAR-T therapy, cellular therapy, Myasthenia Gravis, Neuromuscular, Anti-AChR