CABA-201 for Myasthenia Gravis

Recruiting at 13 trial locations

Overseen By Dr. Jaimin Shah, MD

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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a treatment called CABA-201 for individuals with generalized myasthenia gravis (MG), a condition that causes muscle weakness. The goal is to determine if CABA-201 is safe and effective for those with MG. Participants should have MG with muscle weakness, with or without certain antibodies in their blood. The trial seeks individuals experiencing significant muscle weakness due to MG. As a Phase 1, Phase 2 trial, this research aims to understand how the treatment works and measure its effectiveness in an initial, smaller group.

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.

Is there any evidence suggesting that CABA-201 is likely to be safe for humans?

Research has shown that CABA-201 has a good safety record from previous studies. Most patients did not experience serious side effects. Importantly, severe conditions like cytokine release syndrome (CRS) or immune effector cell-associated neurotoxicity syndrome (ICANS) were not reported in most cases. However, mild CRS occurred in three out of eight patients. This indicates that while some minor reactions were observed, the treatment was generally well-tolerated. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard of care for myasthenia gravis, which typically involves immunosuppressants and acetylcholinesterase inhibitors, CABA-201 is a novel treatment that targets the acetylcholine receptor (AChR) antibodies. This approach is unique because it directly addresses the underlying cause of the disease by focusing on the immune system's role in attacking the body's own receptors. Researchers are excited about CABA-201 because it has the potential to provide more targeted relief for patients, potentially reducing symptoms more effectively and with fewer side effects compared to traditional treatments.

What evidence suggests that CABA-201 might be an effective treatment for Myasthenia Gravis?

Research shows that CABA-201 could help treat myasthenia gravis, a condition causing muscle weakness. CABA-201 alters certain immune cells. Early research in similar areas has shown positive results for patients with severe autoimmune diseases. In other studies, patients receiving similar treatments experienced significant improvements. Although specific data for myasthenia gravis is still being gathered, altering immune cells has proven effective in related conditions. This suggests that CABA-201 might improve symptoms for people with myasthenia gravis. ^{1 2 4 6 7}

Who Is on the Research Team?

Medical Director

Principal Investigator

Cabaletta Bio

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT06359041

NCT06359041 | RESET-MG: A Study to Evaluate the ... 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 ...

2. cabalettabio.com cabalettabio.com/patients/phase-12-trial-in-myasthenia-gravis

RESET-MG™ Phase 1/2 Trial This clinical trial evaluates the safety, tolerability and effectiveness of CABA-201 in the treatment of myasthenia gravis. CABA-201 is an investigational ...

3. cabalettabio.com cabalettabio.com/news-media/press-releases/detail/137/cabaletta-bio-presents-positive-clinical-data-and

Cabaletta Bio Presents Positive Clinical Data and ... All myositis patients in the Phase 1/2 DM/ASyS cohort with sufficient follow-up who met key registrational inclusion criteria exceeded the ...

4. clinicaltrials.ucsf.edu clinicaltrials.ucsf.edu/trial/NCT06359041

UCSF Myasthenia Gravis Trial → RESET-MG: A Study to ... 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.

5. cabalettabio.com cabalettabio.com/pipeline/caba-201

CABA-201 Learn more about Cabaletta Bio's lead product candidate, CABA-201, a CD19-CAR T cell therapy under development for patients with severe autoimmune diseases.

6. cabalettabio.com cabalettabio.com/investors/news-events/press-releases/detail/119/cabaletta-bio-presents-positive-clinical-safety-and

Cabaletta Bio Presents Positive Clinical Safety and Efficacy ... CABA-201 safety profile suggests favorable risk-benefit with no CRS or ICANS in the majority of patients; low-grade CRS in three of eight ...

7. ucihealth.org ucihealth.org/clinical-trials/ascc-23-25

CABA-201 for Generalized Myasthenia Gravis This is a Phase 1/2, open-label study designed to evaluate the safety, tolerability, and efficacy of CABA-201 in adult participants with generalized MG.