Systemic SclerosisPhase 2/3NCT06328777
RESET-SSc: An Open-Label Study to Evaluate the Safety and Efficacy of CABA-201, a CD19-CAR T Cell Therapy, in Subjects With Systemic Sclerosis
A Phase 2/3 study testing CABA-201 in people with Systemic Sclerosis and Scleroderma, run by Cabaletta Bio at 10 sites in United States.
Trial summary
RESET-SSc: A Phase 1/2 Open-Label Study to Evaluate the Safety and Efficacy of CABA-201, a CD19-CAR T cell therapy, in Subjects with Systemic Sclerosis Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Systemic Sclerosis, Scleroderma, Interstitial Lung Diseases
- 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
- 37 participants
- Where
- 10 sitesUnited States
- Study dates
- 2 Jul 2024to Jul 2029
- Phase
- Phase 2/3
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 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age ≥18 and ≤75
- A clinical diagnosis of SSc, based on the 2013 American College of Rheumatology and European League Against Rheumatism classification criteria.
- Early active disease
- Evidence of significant skin, pulmonary, or cardiac involvement
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 visit
- 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
- Severe lung or cardiac impairment
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- Previous CAR T cell therapy
- Prior solid organ (heart, liver, kidney, lung) transplant or hematopoietic cell transplant
- Other protocol-defined inclusion/exclusion criteria may apply.
Trial locations and contacts
10 sites in 1 country. Contact the study team or a site near you to ask about taking part.
10 sites in 1 country
United States10 sites, 10 recruiting
- Recruiting
University of Michigan
Ann Arbor, Michigan, 48109
Lynne Bischoff, lloisros@med.umich.edu
ssc-coordinator@umich.edu
- Recruiting
Northwestern Memorial Hospital
Chicago, Illinois, 60611
Kaitlin King, 312-695-0990, autoimmunesct@nm.org
Dr. George Georges, MD
- Recruiting
The University of Chicago Medical Center
Chicago, Illinois, 60637
Dequana Jones, 773-834-5726, Dequana.Jones@bsd.uchicago.edu
Dr. Michael Macklin, MD
- Recruiting
Duke University
Durham, North Carolina, 27710
Khalia Stewart, 919-660-2280, Khalia.stewart@duke.edu
Michelle Mack, 919-613-1107, Michelle.mack@duke.edu
- Recruiting
Mayo Clinic Florida
Jacksonville, Florida, 55905
Gabrielle Pariseau, 904-953-5549, Pariseau.Gabrielle@mayo.edu
Dr. Andy Abril, MD
- Recruiting
University of Kansas Medical Center
Kansas City, Kansas, 66160
Theresa Howard, 913-588-0653, Thoward2@kumc.edu
Dr. Paul Schmidt, MD
- Recruiting
Yale University
New Haven, Connecticut, 06519
Diane Documet, diane.documet@yale.edu
Dr. Monique Hinchcliff, MD, MS
- Recruiting
Columbia University Irving Medical Center
New York, New York, 10032
Gaby Ycaza, 212-342-2751, mgy2107@cumc.columbia.edu
Elana Bernstein, MD
- Recruiting
Mayo Clinic Rochester
Rochester, Minnesota, 55905
Christi Sagen, 507-422-3425, sagen.christine@mayo.edu
Ashima Makol, MD
- Recruiting
University of Rochester
Rochester, New York, 14642
Chelsey LoMonaco, 585-402-2916, Chelsey_lomonaco@urmc.rochester.edu
Benjamin Korman, M.D.
About this study
Official title: 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 Subjects With Systemic Sclerosis
RESET-SSc: A Phase 1/2 Open-Label Study to Evaluate the Safety and Efficacy of CABA-201, a CD19-CAR T cell therapy, in Subjects with Systemic Sclerosis
Read the detailed description
Systemic sclerosis (SSc), also known as scleroderma , is a rare autoimmune disorder characterized by autoantibody production and abnormal B cell function. Though the cause of SSc is not well understood, it is thought to involve B cells that cause the body to attack different tissues in one's own body, causing skin and organ fibrosis. 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 patients with SSc who have active disease. A single dose of CABA-201 in combination with cyclophosphamide (CY) and fludarabine (FLU) will be evaluated.
- Conditions
- Systemic Sclerosis, Scleroderma, Interstitial Lung Diseases
- Being tested
- CABA-201
- Keywords
- CABA-201, autoimmune disease, anti-CD19 CAR-T therapy, systemic sclerosis, scleroderma, Interstitial lung disease
- Medical subject headings
- Scleroderma, Systemic, Scleroderma, Diffuse, Lung Diseases, Interstitial, Autoimmune Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CABA-201Biological
Also called Rese-cel, Resecabtagene autoleucel
Single intravenous infusion of CABA-201 at a single dose level following preconditioning with fludarabine and cyclophosphamide
CABA-201Biological
Also called Rese-cel, Resecabtagene autoleucel
Single intravenous infusion of CABA-201 at a single dose level following preconditioning with fludarabine and cyclophosphamide
| Study group | Role | What they receive |
|---|---|---|
| CABA-201 Phase 1/2 | Experimental | 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 Receives: Biological: CABA-201 |
| CABA-201 Phase 2b - Sub-study | Experimental | Infusion of CABA-201 with preconditioning in subjects with SSc Receives: Biological: CABA-201 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Phase 1/2: To evaluate incidence of adverse events
Incidence and severity of AEs
Measured: Up to 28 days after CABA-201 infusion
Phase 2b Sub-study: To evaluate the efficacy of CABA-201 on % predicted forced vital capacity (FVC) in SSc-ILD subjects
Mean change from baseline in % predicted FVC without any immunomodulatory or anti-fibrotic medications and on no or low dose of steroids
Measured: Week 52
Secondary outcomes (5)
To evaluate adverse events and laboratory abnormalities
Incidence and severity of AEs, including changes in laboratory values and vital signs
Measured: Up to 156 weeks (Phase 1/2) and through 104 weeks (Sub-study)
To characterize the pharmacodynamics (PD)
Levels of B cells in the blood
Measured: Up to 156 weeks (Phase 1/2) and through 104 weeks (Sub-study)
To characterize the pharmacokinetics (PK)
Levels of CABA-201-positive T cells in the blood
Measured: Up to 156 weeks (Phase 1/2) and through 104 weeks (Sub-study)
To evaluate efficacy
Proportion of subjects achieving revised Composite Response Index for Systemic Sclerosis (CRISS) criteria
Measured: Up to 156 weeks (Phase 1/2) and at Week 52 (Sub-study)
Mean change from baseline in Modified Rodnan Skin Score (mRSS)
Measured: Week 52 (Sub-study)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Mar 2024
- Study start2 Jul 2024
- Primary completionJul 2029 (estimated)
- Study completionJul 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Systemic SclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 2/3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 37 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Cabaletta Bio
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Cabaletta Bio
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06328777
- Sponsor's protocol ID
- CAB-201-003
- Status last verified
- Oct 2026
- First submitted
- 19 Mar 2024
- Last updated
- 16 Oct 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No