RESET-MG: A Study to Evaluate the Safety and Efficacy of CABA-201 in Participants With Generalized Myasthenia Gravis

August 12, 2026 updated by: Cabaletta Bio

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

RESET-MG: A Phase 1/2 Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Participants with Generalized Myasthenia Gravis

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

Myasthenia gravis (MG) is a rare autoimmune disorder characterized by autoantibody responses that cause defective transmission of signals at the neuromuscular junction, resulting in a distinctive pattern of weakness. Patients with generalized MG (gMG) typically experience symptoms associated with ocular disease in addition to weakness of many other voluntary muscle groups, including extremity, bulbar, and respiratory muscles. MG is considered a classic example of a B-cell mediated autoimmune disease. Currently, there are no curative treatments for MG. This study is being conducted to evaluate the safety and efficacy of an investigational cell therapy, CABA-201, that can be given to patients with gMG. A single dose of CABA-201 in combination with cyclophosphamide (CY) and fludarabine (FLU) will be evaluated. In addition, escalating doses of CABA-201 will be evaluated in patients without CY and FLU preconditioning.

Study Type

Interventional

Enrollment (Estimated)

24

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • United States
    • California
      • Orange, California, United States, 92868
        • Recruiting
        • University of California Irvine
        • Principal Investigator:
          • Ali Habib, MD
        • Contact:
      • Sacramento, California, United States, 95817
        • Recruiting
        • UC Davis, Department of Neurology
        • Principal Investigator:
          • David Richman, MD
        • Contact:
      • San Francisco, California, United States, 94143
        • Recruiting
        • University of California, San Francisco
        • Contact:
        • Principal Investigator:
          • Dr. Min Kang, MD
    • Colorado
    • Florida
      • Jacksonville, Florida, United States, 32224
        • Recruiting
        • Mayo Clinic Florida
        • Principal Investigator:
          • Dr. Jaimin Shah, MD
        • Contact:
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Recruiting
        • Northwestern Memorial Hospital
        • Principal Investigator:
          • Dr. Arjun Seth, MD
        • Contact:
    • Kansas
      • Kansas City, Kansas, United States, 66160
        • Recruiting
        • University of Kansas Medical Center
        • Principal Investigator:
          • Mazen Dimachkie, MD
        • Contact:
    • Massachusetts
      • Boston, Massachusetts, United States, 02215
        • Recruiting
        • Beth Israel Deaconess Medical Center
        • Contact:
        • Principal Investigator:
          • Pushpa Narayanaswami, MD
      • Worcester, Massachusetts, United States, 01655
        • Recruiting
        • University of Massachusetts Chan Medical School
        • Principal Investigator:
          • Dr. Margaret A. Owegi, DO
        • Contact:
    • Minnesota
      • Rochester, Minnesota, United States, 55905
        • Recruiting
        • Mayo Clinic - Rochester
        • Contact:
        • Principal Investigator:
          • Elie Naddaf, M.D.
    • New York
      • New York, New York, United States, 10032
      • Rochester, New York, United States, 14642
        • Recruiting
        • University of Rochester Medical Center
        • Principal Investigator:
          • Dr. Alexis Lizarraga, MD, MS
        • Contact:
    • Oregon
      • Portland, Oregon, United States, 97239
        • Recruiting
        • Oregon Health & Science University
        • Contact:
        • Principal Investigator:
          • Dr. Nizar Chahin, M.D.
    • Texas
      • Houston, Texas, United States, 77030
        • Recruiting
        • Houston Methodist Hospital
        • Contact:
        • Principal Investigator:
          • Dr. Sheetal Shroff, MD
      • Houston, Texas, United States, 77030
        • Recruiting
        • University of Texas MD Anderson Cancer Center
        • Principal Investigator:
          • Uday Popat, MD
        • Contact:
      • Houston, Texas, United States, 77030
        • Recruiting
        • Baylor College of Medicine Neurology Department
        • Principal Investigator:
          • Milvia Pleitez, MD
        • Contact:
    • Washington
      • Seattle, Washington, United States, 98122

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: CABA-201
AChR Antibody-Positive Cohort or AChR Antibody-Negative Cohort
Biological: CABA-201
Single intravenous infusion of CABA-201 at a single dose level following preconditioning with fludarabine and cyclophosphamide
Biological: CABA-201
Single intravenous infusion of CABA-201 at escalating dose levels without preconditioning
Experimental: CABA-201, No Preconditioning
Infusion of CABA-201 with no preconditioning in subjects with gMG
Biological: CABA-201
Single intravenous infusion of CABA-201 at a single dose level following preconditioning with fludarabine and cyclophosphamide
Biological: CABA-201
Single intravenous infusion of CABA-201 at escalating dose levels without preconditioning

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
To evaluate incidence and severity of adverse events (AEs)
Time Frame: Up to 28 days after CABA-201 infusion
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 Outcome Measures

Outcome Measure
Measure Description
Time Frame
To characterize the pharmacodynamics (PD)
Time Frame: Up to 156 weeks
Levels of B cells in the blood
Up to 156 weeks
To characterize the pharmacokinetics (PK)
Time Frame: Up to 156 weeks
Levels of CABA-201-positive T cells in the blood
Up to 156 weeks
To evaluate the incidence and severity of adverse events (AEs)
Time Frame: Up to 156 weeks
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 156 weeks
To evaluate disease-related biomarkers
Time Frame: Up to 156 weeks
Levels of MG-specific autoantibodies in the serum
Up to 156 weeks
To evaluate efficacy by change in Myasthenia Gravis - Activities of Daily Living (MG-ADL) score over time.
Time Frame: Up to 156 weeks
The MG-ADL scale is a validated instrument administered by a qualified assessor with 8 areas of activities of daily living as reported by patients: talking, chewing, swallowing, breathing, ability to brush teeth or comb hair, ability to rise from a chair, double vision, and eyelid droop. In each area, the score ranges from 0 (normal) to 3 (most severe). The total score is the sum of all subscores.
Up to 156 weeks
To evaluate efficacy by change in Quantitative Myasthenia Gravis (QMG) score over time.
Time Frame: Up to 156 weeks
The QMG score is a 13-item scale conducted by a qualified assessor to evaluate disease severity in patients with MG. The total score ranges from a minimum of 0 (no myasthenic findings) to 39 (maximum myasthenic deficits).
Up to 156 weeks
To evaluate efficacy by change in Myasthenia Gravis Composite (MGC) score over time.
Time Frame: Up to 156 weeks
The MGC scale is a physician-reported instrument for the assessment of MG patients' symptoms and impairments. It includes 10 domains, and the total score ranges from 0 (normal) to 50 (most severe).
Up to 156 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Cabaletta Bio

Investigators

  • Study Chair: Medical Director, Cabaletta Bio

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

December 17, 2024

Primary Completion (Estimated)

September 1, 2029

Study Completion (Estimated)

September 1, 2029

Study Registration Dates

First Submitted

March 21, 2024

First Submitted That Met QC Criteria

April 5, 2024

First Posted (Actual)

April 11, 2024

Study Record Updates

Last Update Posted (Actual)

August 13, 2026

Last Update Submitted That Met QC Criteria

August 12, 2026

Last Verified

August 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CAB-201-004

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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