RESET-MG: A Study to Evaluate the Safety and Efficacy of CABA-201 in Participants With 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
1 other identifier
interventional
12
1 country
17
Brief Summary
RESET-MG: A Phase 1/2 Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Participants with Generalized Myasthenia Gravis
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_1
Started Dec 2024
Longer than P75 for phase_1
17 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
March 21, 2024
CompletedFirst Posted
Study publicly available on registry
April 11, 2024
CompletedStudy Start
First participant enrolled
December 17, 2024
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 1, 2029
ExpectedStudy Completion
Last participant's last visit for all outcomes
September 1, 2029
February 20, 2026
February 1, 2026
4.7 years
March 21, 2024
February 19, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
To evaluate incidence and severity of adverse events (AEs)
An AE is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study treatment, whether or not considered related to the study treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal result of an investigation), symptom, or disease (new or exacerbated) temporally associated with the use of study treatment. The term AE is used to include both serious and non-serious AEs.
Up to 28 days after CABA-201 infusion
Secondary Outcomes (7)
To evaluate the incidence and severity of adverse events (AEs)
Up to 156 weeks
To characterize the pharmacodynamics (PD)
Up to 156 weeks
To characterize the pharmacokinetics (PK)
Up to 156 weeks
To evaluate disease-related biomarkers
Up to 156 weeks
To evaluate efficacy by change in Myasthenia Gravis - Activities of Daily Living (MG-ADL) score over time.
Up to 156 weeks
- +2 more secondary outcomes
Study Arms (1)
CABA-201
EXPERIMENTALAChR Antibody-Positive Cohort AChR Antibody-Negative Cohort
Interventions
Single intravenous infusion of CABA-201 at a single dose level following preconditioning with fludarabine and cyclophosphamide
Eligibility Criteria
You may qualify if:
- 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
You may not qualify if:
- 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
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Cabaletta Biolead
Study Sites (17)
University of California Irvine
Orange, California, 92868, United States
UC Davis, Department of Neurology
Sacramento, California, 95817, United States
University of California, San Francisco
San Francisco, California, 94143, United States
University of Colorado
Aurora, Colorado, 80045, United States
Mayo Clinic Florida
Jacksonville, Florida, 32224, United States
Northwestern Memorial Hospital
Chicago, Illinois, 60611, United States
University of Kansas Medical Center
Kansas City, Kansas, 66160, United States
Beth Israel Deaconess Medical Center
Boston, Massachusetts, 02215, United States
University of Massachusetts Chan Medical School
Worcester, Massachusetts, 01655, United States
Mayo Clinic - Rochester
Rochester, Minnesota, 55905, United States
Columbia University
New York, New York, 10032, United States
University of Rochester Medical Center
Rochester, New York, 14642, United States
Oregon Health & Science University
Portland, Oregon, 97239, United States
Baylor College of Medicine Neurology Department
Houston, Texas, 77030, United States
Houston Methodist Hospital
Houston, Texas, 77030, United States
University of Texas MD Anderson Cancer Center
Houston, Texas, 77030, United States
Swedish Neuroscience Research
Seattle, Washington, 98122, United States
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY CHAIR
Medical Director
Cabaletta Bio
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
March 21, 2024
First Posted
April 11, 2024
Study Start
December 17, 2024
Primary Completion (Estimated)
September 1, 2029
Study Completion (Estimated)
September 1, 2029
Last Updated
February 20, 2026
Record last verified: 2026-02
Data Sharing
- IPD Sharing
- Will not share