NCT05451212CompletedPhase 1
Open-label Study to Evaluate the Safety of Various Dosing Regimens of MuSK-CAART for 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
Source: ClinicalTrials.gov · Last updated 10 Nov 2025
Brief summary (registry, in English)
Muscle-specific tyrosine kinase (MuSK) myasthenia gravis (MG) is a rare but potentially severe disease, in which patients develop pathogenic autoantibodies that specifically target the MuSK protein in the neuromuscular junction. This phase 1 study is being conducted to evaluate the safety of various dosing regimens of an investigational cell therapy, MuSK-CAART, that can be given to patients with anti-MuSK antibody positive Myasthenia Gravis (MuSK MG), who have active disease. Various dosing regimens of MuSK-CAART alone, in combination with cyclophosphamide (CY), and in combination with CY and fludarabine (FLU) will be evaluated. Treatment with MuSK-CAART may potentially lead to complete and durable remission of disease.
- Status (registry)
- Completed
- Phase (registry)
- Phase 1
- Study type
- Interventional
- Enrollment
- 7 (actual)
- First submitted
- 30 Jun 2022
- Start date
- 23 Nov 2022
- Primary completion date
- 24 Jul 2025
- Lead sponsor
- Cabaletta Bio
- Diseases
- Myasthenia gravis
- NCT number
- NCT05451212
What you commit to
- Study type: Interventional · Phase 1
- Participants enrolled: 7
- From 23 Nov 2022 to the primary completion date, 24 Jul 2025
- 5 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Other illness
- Other autoimmune disorder requiring immunosuppressive therapies.
Lab values
- Absolute lymphocyte count \< 500/µL at screening.
Other
- Rituximab in the last 12 months.
- Prednisone \> 0.25mg/kg/day \[in Part A\]
- Investigational treatment for MG in the past 12 weeks.
Who the study is looking for
- 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
Age in the registry: 18 years to - · Sex: All · Does not accept healthy volunteers
Sites (5)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Houston Methodist Hospital Coordinator of this site? Confirm its status | Houston | United States | Unknown status |
| University of Kansas Medical Center Coordinator of this site? Confirm its status | Kansas City | United States | Unknown status |
| UC Irvine, Department of Neurology Coordinator of this site? Confirm its status | Orange | United States | Unknown status |
| Oregon Health & Science University (OHSU) Coordinator of this site? Confirm its status | Portland | United States | Unknown status |
| UC Davis, Department of Neurology Coordinator of this site? Confirm its status | Sacramento | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.