CABA-201 for Lupus

Recruiting at 21 trial locations

Overseen By Elena Massarotti, MD

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Cabaletta Bio

Disqualifiers: Severe infections, Organ transplant, 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: Unknown

Duration: 1 day

28 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called CABA-201 for individuals with active systemic lupus erythematosus (SLE), an autoimmune disease where the immune system attacks healthy tissue. The trial evaluates the safety and effectiveness of this treatment, particularly for those with lupus nephritis (a type of kidney inflammation caused by lupus) or moderate to severe SLE that doesn't affect the kidneys. Participants must have a confirmed lupus diagnosis and experience symptoms that impact daily life, such as frequent flare-ups or kidney issues. As a Phase 1, Phase 2 trial, this research aims to understand how the treatment works in people and measure its effectiveness in an initial, smaller group, offering participants a chance to be among the first to benefit from this innovative therapy.

Will I have to stop taking my current medications?

The trial information does not specify whether 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 the treatment CABA-201 was safe in earlier studies. Most patients did not experience cytokine release syndrome (CRS) or neurotoxicity (ICANS), which are possible side effects. Specifically, only three out of eight patients had mild CRS. Overall, CABA-201 was well-tolerated by patients. These findings suggest that the treatment may offer a good balance between benefits and risks for people with lupus. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for lupus?

Researchers are excited about CABA-201 for lupus because it offers a novel approach compared to existing treatments. Unlike standard treatments, which often include immunosuppressants and corticosteroids that can have significant side effects, CABA-201 is engineered to target the immune system more precisely. This treatment involves an infusion with preconditioning, which might lead to better control of lupus symptoms with potentially fewer side effects. By focusing on specific immune pathways, CABA-201 could provide a more effective and safer option for patients, especially those with lupus nephritis (LN) and non-renal systemic lupus erythematosus (SLE).

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

Research has shown that CABA-201, the investigational treatment under study in this trial, holds promise for treating lupus. In an earlier study, three out of four lupus patients experienced significant symptom improvement or remission. Importantly, most patients did not encounter serious side effects, such as cytokine release syndrome, a potentially dangerous immune reaction. Early findings suggest that CABA-201 could be a safe and effective option for people with lupus. Overall, these initial results offer encouragement for those considering CABA-201 as a treatment. ^{1 2 3 6 7}

Who Is on the Research Team?

Medical Director

Principal Investigator

Cabaletta Bio

Are You a Good Fit for This Trial?

Adults aged 18-65 with active Systemic Lupus Erythematosus (SLE) can join. For lupus nephritis, a biopsy must confirm it. Participants need positive tests for specific antibodies and have moderate to severe SLE symptoms if not renal-related. Exclusions include other kidney diseases, previous CAR T cell therapy, reactions to certain drugs, ongoing serious infections or organ transplants.

Inclusion Criteria

You have positive test results for certain antibodies in your blood.

I have been diagnosed with lupus according to the 2019 EULAR/ACR criteria.

I have a confirmed diagnosis of class III or IV lupus nephritis, with or without class V.

Exclusion Criteria

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

I have received CAR T cell therapy before.

I have kidney disease, but it's not active lupus nephritis.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Preconditioning

Participants receive a standard regimen including cyclophosphamide (CY) and fludarabine (FLU) before the infusion of CABA-201

1-2 weeks

Treatment

Participants receive a single dose of CABA-201

1 day

Follow-up

Participants are monitored for safety and effectiveness after treatment

156 weeks

What Are the Treatments Tested in This Trial?

Interventions

• CABA-201

Trial Overview

The trial is testing CABA-201's safety and effectiveness in treating active SLE. It's an open-label study where everyone knows they're getting the same treatment without any comparison group.

How Is the Trial Designed?

Group I: CABA-201, No Preconditioning Experimental Treatment 1 Intervention

Infusion of CABA-201 with no preconditioning in subjects with LN and non-renal SLE

Group II: CABA-201 with FLU/CY Preconditioning Experimental Treatment 1 Intervention

LN Cohort: Infusion of CABA-201 with preconditioning in subjects with LN Non-renal SLE Cohort: Infusion of CABA-201 with preconditioning in subjects with non-renal SLE who do not meet criteria for inclusion in the LN cohort Expansion Cohort: Infusion of CABA-201 with preconditioning in subjects with LN and SLE

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.

jrheum.org

jrheum.org/content/52/Suppl_1/27

RESET SLE: CLINICAL TRIAL EVALUATING CABA-201, A ...

The primary endpoint is safety and tolerability within 28 days of infusion. Secondary endpoints include translational assessments (including CAR ...

2.

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 ...

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 ...

Three of 4 SLE patients with at least 3 months of follow-up achieved DORIS (definition of remission in SLE), and the fourth patient with pure ...

4.

lupus.org

lupus.org/news/update-investigational-therapy-caba201-for-lupus-treatment-shows-positive-initial-data

Investigational Therapy CABA-201 for Lupus Treatment ...

Initial Phase 1 of 2 RESET-Myositis and RESET-SLE clinical trial data of the investigational therapy, CABA-201, revealed positive outcomes.

5.

clinicaltrials.gov

clinicaltrials.gov/study/NCT06121297

RESET-SLE: A Phase 1/2 Open-Label Study to Evaluate ...

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 either LN or ...

6.

cabalettabio.com

cabalettabio.com/patients/phase-12-trial-in-lupus

RESET-SLE™ Trial :: Cabaletta Bio, Inc. (CABA)

This clinical trial evaluates the safety, tolerability and effectiveness of CABA-201 in the treatment of lupus. CABA-201 is an investigational chimeric ...

7.

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.