Evaluating the safety of MuSK-CAART for treating MuSK Myasthenia Gravis
A Phase 1, Open-label, Safety and Dose-finding Study of Autologous Muscle-specific Tyrosine Kinase Chimeric Autoantibody Receptor T Cells (MuSK-CAART) in Subjects With Anti-MuSK-antibody-positive Myasthenia Gravis
This study is testing a new cell therapy called MuSK-CAART to see if it is safe and can help people with active MuSK Myasthenia Gravis feel better.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 24 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Cabaletta Bio Industry-sponsored |
| Drugs / interventions | Rituximab, Prednisone, cyclophosphamide, fludarabine |
| Locations | 5 sites (Orange, California and 4 other locations) |
| Trial ID | NCT05451212 on ClinicalTrials.gov |
What this trial studies
This phase 1 clinical trial aims to assess the safety of different dosing regimens of MuSK-CAART, an investigational cell therapy, for patients with MuSK Myasthenia Gravis who have active disease. Participants will receive MuSK-CAART either alone or in combination with cyclophosphamide and fludarabine. The study will monitor the safety and potential efficacy of these treatment regimens in achieving remission of the disease. The trial is designed for patients who have confirmed anti-MuSK antibody positive Myasthenia Gravis.
Who should consider this trial
Good fit: Ideal candidates include individuals with a confirmed diagnosis of MuSK-type Myasthenia Gravis and active disease who have tested positive for anti-MuSK antibodies.
Not a fit: Patients who have received rituximab in the last 12 months or have other autoimmune disorders requiring immunosuppressive therapies may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could lead to complete and durable remission for patients suffering from MuSK Myasthenia Gravis.
How similar studies have performed: While this approach is novel in the context of MuSK Myasthenia Gravis, similar CAR-T therapies have shown promise in other autoimmune conditions.
Eligibility criteria
Show full inclusion / exclusion criteria
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.
Where this trial is running
Orange, California and 4 other locations
- UC Irvine, Department of Neurology — Orange, California, United States (Recruiting)
- UC Davis, Department of Neurology — Sacramento, California, United States (Recruiting)
- University of Kansas Medical Center — Kansas City, Kansas, United States (Recruiting)
- Oregon Health & Science University (OHSU) — Portland, Oregon, United States (Recruiting)
- Houston Methodist Hospital — Houston, Texas, United States (Active_not_recruiting)
Study contacts
- Study coordinator: Cabaletta Bio
- Email: clinicaltrials@cabalettabio.com
- Phone: +1 267 759 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.