AzTrials
AzTrials

Generalized Myasthenia Gravis (gMG)Phase 1/2NCT06359041

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

A Phase 1/2 study testing CABA-201 in people with Generalized Myasthenia Gravis (gMG), run by Cabaletta Bio at 17 sites in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial 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 Read more about this study

Who is it for?
Ages 18 to 70 years
Conditions the study is for
Generalized Myasthenia Gravis (gMG)
What is being tested?
CABA-201
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
24 participants
Where
17 sitesUnited States
Study dates
17 Dec 2024to Sep 2029
Phase
Phase 1/2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 70 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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

Who cannot join

  • 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.

Trial locations and contacts

17 sites in 1 country. Contact the study team or a site near you to ask about taking part.

Cabaletta Bio contact

267 759 3100 ext. 4444

clinicaltrials@cabalettabio.com

17 sites in 1 country

United States17 sites, 17 recruiting

  • University of Colorado

    Aurora, Colorado, 80045

    Recruitment Specialist, 303-724-4644, neuroresearch@cuanschutz.edu

    Alyssa Alviez, Alyssa.avilez@cuanschutz.edu

    Recruiting

  • Beth Israel Deaconess Medical Center

    Boston, Massachusetts, 02215

    Amy Lewandowski, 617-667-2545, alewand2@bidmc.harvard.edu

    Pushpa Narayanaswami, MD

    Recruiting

  • Northwestern Memorial Hospital

    Chicago, Illinois, 60611

    Kaitlin King, 312-921-2371, Kaitlin.King@nm.org

    Dr. Arjun Seth, MD

    Recruiting

  • Baylor College of Medicine Neurology Department

    Houston, Texas, 77030

    Robbie Henderson, 713-798-7486, rlhender@bcm.edu

    Milvia Pleitez, MD

    Recruiting

  • Houston Methodist Hospital

    Houston, Texas, 77030

    Amelia Nounes, 346-356-3639, aanounes@houstonmethodist.org

    Dr. Sheetal Shroff, MD

    Recruiting

  • University of Texas MD Anderson Cancer Center

    Houston, Texas, 77030

    Meredith del Rosario, 713-792-1416, mcdel@mdanderson.org

    Uday Popat, MD

    Recruiting

  • Mayo Clinic Florida

    Jacksonville, Florida, 32224

    Huy Tran CRC, 904-953-4503, Tran.Huy1@mayo.edu

    Dr. Jaimin Shah, MD

    Recruiting

  • University of Kansas Medical Center

    Kansas City, Kansas, 66160

    Lilli Saavedra, 913-945-9937, lsaavedra2@kumc.edu

    Mazen Dimachkie, MD

    Recruiting

  • Columbia University

    New York, New York, 10032

    CPDM Nursing Navigator, 212-342-5162, cpdm_celltherapy@lists.cumc.columbia.edu

    Dr. Christina Ulane, MD

    Recruiting

  • University of California Irvine

    Orange, California, 92868

    Alpha Clinic, 949-824-3990, alphaclinic@uci.edu

    Ali Habib, MD

    Recruiting

  • Oregon Health & Science University

    Portland, Oregon, 97239

    Katie Lewis, 503-494-7394, lewiskat@ohsu.edu

    Dr. Nizar Chahin, M.D.

    Recruiting

  • Mayo Clinic - Rochester

    Rochester, Minnesota, 55905

    Bridget Neja, 507-266-9150, neja.bridget@mayo.edu

    Elie Naddaf, M.D.

    Recruiting

  • University of Rochester Medical Center

    Rochester, New York, 14642

    Olivia Tauriello, 585-275-3707, Olivia_Tauriello@URMC.Rochester.edu

    Dr. Alexis Lizarraga, MD, MS

    Recruiting

  • UC Davis, Department of Neurology

    Sacramento, California, 95817

    Danielle Davis, 916-734-1798, ddavis4@health.ucdavis.edu

    David Richman, MD

    Recruiting

  • University of California, San Francisco

    San Francisco, California, 94143

    Zane Ashkar - Clinical Research Coordinator, 415-501-0671, Zane.Ashkar@ucsf.edu

    Dr. Min Kang, MD

    Recruiting

  • Swedish Neuroscience Research

    Seattle, Washington, 98122

    Desiree Iriarte, 206-320-2282, desiree.iriarte@swedish.org

    Mark Loreen, mark.loreen@swedish.org

    Recruiting

  • University of Massachusetts Chan Medical School

    Worcester, Massachusetts, 01655

    Catherine Douthwright, 508-856-1524, Catherine.Douthwright@umassmed.edu

    Dr. Margaret A. Owegi, DO

    Recruiting

Other trials in United States

About this study

Official title: 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

Read the 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.

Being tested
CABA-201
Keywords
CABA-201, autoimmune disease, anti-CD19 CAR-T therapy, cellular therapy, Myasthenia Gravis, Neuromuscular, Anti-AChR, Anti-LRP4, Anti-MuSK
Medical subject headings
Myasthenia Gravis, Autoimmune Diseases

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • CABA-201Biological

    Single intravenous infusion of CABA-201 at a single dose level following preconditioning with fludarabine and cyclophosphamide

  • CABA-201Biological

    Single intravenous infusion of CABA-201 at escalating dose levels without preconditioning

Study groups in NCT06359041
Study groupRoleWhat they receive
CABA-201ExperimentalAChR Antibody-Positive Cohort or AChR Antibody-Negative Cohort
Receives: Biological: CABA-201
CABA-201, No PreconditioningExperimentalInfusion of CABA-201 with no preconditioning in subjects with gMG
Receives: Biological: CABA-201

What is being measured

The primary outcome is the main question the study is designed to answer.

  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.

    Measured: Up to 28 days after CABA-201 infusion

Secondary outcomes (7)

  1. To evaluate the 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.

    Measured: Up to 156 weeks

  2. To characterize the pharmacodynamics (PD)

    Levels of B cells in the blood

    Measured: Up to 156 weeks

  3. To characterize the pharmacokinetics (PK)

    Levels of CABA-201-positive T cells in the blood

    Measured: Up to 156 weeks

  4. To evaluate disease-related biomarkers

    Levels of MG-specific autoantibodies in the serum

    Measured: Up to 156 weeks

  5. To evaluate efficacy by change in Myasthenia Gravis - Activities of Daily Living (MG-ADL) score over time.

    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.

    Measured: Up to 156 weeks

  6. To evaluate efficacy by change in Quantitative Myasthenia Gravis (QMG) score over time.

    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).

    Measured: Up to 156 weeks

  7. To evaluate efficacy by change in Myasthenia Gravis Composite (MGC) score over time.

    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).

    Measured: Up to 156 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted11 Apr 2024
  2. Study start17 Dec 2024
  3. Primary completionSep 2029 (estimated)
  4. Study completionSep 2029 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Single group
Masking
None (open label)
Enrolment
24 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06359041
Sponsor's protocol ID
CAB-201-004
Status last verified
Oct 2026
First submitted
21 Mar 2024
Last updated
19 Oct 2026
Results posted
No
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing CABA-201 in people with Generalized Myasthenia Gravis (gMG), run by Cabaletta Bio at 17 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 19 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 17 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Cabaletta Bio (267 759 3100 ext. 4444, clinicaltrials@cabalettabio.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

To evaluate incidence and severity of adverse events (AEs) (time frame: Up to 28 days after CABA-201 infusion). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06359041.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Oct 2026; AzTrials last refreshed its copy on 26 Oct 2026.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Keep searching with a free account

You’ve used your 3 free searches for this session. Log in or create a free AzTrials account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in