CABA-201 for Myasthenia Gravis

Recruiting at 10 trial locations

Overseen By Cabaletta Bio

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Cabaletta Bio

Disqualifiers: Severe infections, Cardiac impairment, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: 1

Duration: 1 day

12 Participants Needed

Trial Summary

What is the purpose of this trial?

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

Do I need to stop my current medications for the trial?

The trial information does not specify if you need to stop taking your current medications. It's best to discuss this with the trial team or your doctor.

Research Team

Medical Director

Principal Investigator

Cabaletta Bio

Eligibility Criteria

This trial is for adults aged 18 to 70 with Generalized Myasthenia Gravis (MG), a condition causing muscle weakness. Participants must have been diagnosed as seropositive or seronegative MG and exhibit certain levels of muscle weakness, according to the MGFA classification.

Inclusion Criteria

I have been diagnosed with Myasthenia Gravis and experience widespread muscle weakness.

I have been diagnosed with myasthenia gravis.

Exclusion Criteria

I have had a transplant of a solid organ or bone marrow.

History of anaphylactic or severe systemic reaction to fludarabine, cyclophosphamide or any of their metabolites

I am currently being treated for an infection.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single dose of CABA-201 in combination with cyclophosphamide and fludarabine

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment, including evaluation of adverse events and pharmacodynamics

28 days

Multiple visits (in-person and virtual)

Long-term Follow-up

Participants are monitored for long-term safety and efficacy, including evaluation of disease-related biomarkers and efficacy scores

156 weeks

Treatment Details

Interventions

• CABA-201

Trial Overview The study is testing CABA-201, a new potential treatment for Generalized Myasthenia Gravis. It's an open-label trial, meaning both researchers and participants know what treatment is being given, aimed at assessing how safe and effective this medication is.

Participant Groups

Group I: CABA-201 Experimental Treatment 1 Intervention

AChR Antibody-Positive Cohort AChR Antibody-Negative Cohort

CABA-201 is already approved in United States for the following indications:

Approved in United States as CABA-201 for:

• None approved; under investigation for systemic lupus erythematosus, myositis, systemic sclerosis, myasthenia gravis, and pemphigus vulgaris

Who Is Running the Clinical Trial?

Cabaletta Bio

Lead Sponsor

Trials

6

Recruited

140+