CABA-201 for Scleroderma

Recruiting at 9 trial locations

Overseen By Cabaletta Bio

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 week

12 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment, CABA-201, for people with systemic sclerosis (SSc), a condition where the immune system attacks its own tissues, causing hardening and tightening of the skin and other organs. The goal is to determine if CABA-201, a therapy using modified immune cells, is safe and effective for those with severe skin or organ involvement due to SSc. Individuals with early active SSc and significant skin or organ issues, such as lung, heart, or kidney problems, might be suitable for this trial.

As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the chance to be among the first to receive this new therapy.

Do I need to stop my current medications to join 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, a type of cell therapy, appears safe. In earlier studies, most patients did not experience cytokine release syndrome (CRS) or immune cell-associated neurotoxicity syndrome (ICANS), common issues with cell therapies. Only a few patients experienced mild CRS, which was easy to manage. This suggests that CABA-201 is generally well-tolerated. However, since this treatment remains under study, more information is needed to fully understand its safety in humans. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about CABA-201 for scleroderma because it offers a novel approach distinct from current treatments like immunosuppressants and corticosteroids. CABA-201 is an experimental therapy that targets severe skin and organ involvement in scleroderma by utilizing a specific infusion technique with preconditioning, which may enhance its effectiveness. This innovative approach could potentially address the underlying disease mechanisms more directly, offering new hope for improved outcomes in patients with severe scleroderma.

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

Research has shown that CABA-201, a type of cell therapy, may help treat systemic sclerosis, also known as scleroderma. In earlier studies, all four patients monitored for at least three months showed significant improvements without needing other immune-modulating drugs or steroids. This trial will evaluate CABA-201 in participants with severe skin and organ involvement in scleroderma. Early results in similar conditions, such as systemic lupus erythematosus and myositis, suggest it could be effective as a one-time treatment. Although more research is needed, these initial findings are promising. ^{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?

This trial is for adults aged 18-70 with Systemic Sclerosis, an autoimmune condition affecting the skin and internal organs. Participants should have early active disease and signs of significant involvement in skin, lungs, kidneys, or heart.

Inclusion Criteria

A clinical diagnosis of SSc, based on the 2013 American College of Rheumatology and European League Against Rheumatism classification criteria

My condition is in its early stages.

My condition significantly affects my skin, lungs, kidneys, or heart.

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 cannot undergo leukapheresis due to health reasons.

I have an infection that needs treatment.

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 week

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 study tests CABA-201, a new type of cell therapy targeting CD19 on immune cells. It's an open-label trial meaning everyone knows they're getting the treatment to see if it's safe and effective against Systemic Sclerosis.

How Is the Trial Designed?

Group I: CABA-201 Experimental Treatment 1 Intervention

Severe Skin Cohort: Infusion of CABA-201 with preconditioning in subjects with SSc with severe skin involvement Organ Cohort: Infusion of CABA-201 with preconditioning in subjects with SSc with organ involvement

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. 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 4 patients with at least 3 months of follow-up achieved an rCRISS-25 response off immunomodulators and steroids. These initial data suggest ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT06328777

NCT06328777 | RESET-SSc: An Open-Label Study to ... This study is being conducted to evaluate the safety and efficacy of an investigational therapy, CABA-201, a CD19-CAR T cell therapy, that can be given to ...

3. cabalettabio.com cabalettabio.com/news-media/press-releases/detail/130/cabaletta-bio-announces-new-rese-cel-safety-and-efficacy

Cabaletta Bio Announces New Rese-cel Safety and ... Cabaletta is presenting new clinical and translational data from 18 evaluable patients who were dosed with rese-cel across the RESET-Myositis, RESET-SLE and ...

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

CABA-201 Emerging data in patients with systemic lupus erythematosus, myositis and systemic sclerosis suggests the potential of a one-time treatment with a 4-1BB ...

5. duke-research.dukehealth.org duke-research.dukehealth.org/studies/pro00115528

DRA01: CABA-201 for Systemic Sclerosis (Scleroderma) We are doing this study to find out if an experimental drug called CABA-201 (the study drug) is a safe and effective option for people with systemic sclerosis.

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. cabalettabio.com cabalettabio.com/patients/phase-12-trial-in-systemic-sclerosis

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