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
- Are there results?
- Not yet: the study is under way
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
Show all 9Show fewer
- 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
Study at a glance
From the registry record, last updated by the sponsor on 16 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT06328777
- Acronym / sponsor ID
- CAB-201-003
- Conditions
- Systemic Sclerosis, Scleroderma, Interstitial Lung Diseases
- Keywords
- CABA-201, autoimmune disease, anti-CD19 CAR-T therapy, systemic sclerosis, scleroderma, Interstitial lung disease
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 37 participants (estimated)
- Lead sponsor
- Cabaletta BioIndustry (pharma, biotech, devices)
- Study start
- 2 Jul 2024 (actual)
- Primary completion
- Jul 2029 (estimated)
- Study completion
- Jul 2029 (estimated)
- First posted
- 25 Mar 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| CABA-201 Phase 1/2 | Experimental | Biological: CABA-201Severe 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 Phase 2b - Sub-study | Experimental | Biological: CABA-201Infusion of CABA-201 with preconditioning in subjects with SSc |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (9 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CABA-201Also: Rese-cel, Resecabtagene autoleucelSingle intravenous infusion of CABA-201 at a single dose level following preconditioning with fludarabine and cyclophosphamide | Biological | CABA-201 Phase 1/2 |
| CABA-201Also: Rese-cel, Resecabtagene autoleucelSingle intravenous infusion of CABA-201 at a single dose level following preconditioning with fludarabine and cyclophosphamide | Biological | CABA-201 Phase 2b - Sub-study |
Primary outcome
Phase 1/2: To evaluate incidence of adverse events
Time frame: Up to 28 days after CABA-201 infusion
Incidence and severity of AEs
Phase 2b Sub-study: To evaluate the efficacy of CABA-201 on % predicted forced vital capacity (FVC) in SSc-ILD subjects
Time frame: Week 52
Mean change from baseline in % predicted FVC without any immunomodulatory or anti-fibrotic medications and on no or low dose of steroids
5 secondary outcomes
To evaluate adverse events and laboratory abnormalities
Time frame: Up to 156 weeks (Phase 1/2) and through 104 weeks (Sub-study)
To characterize the pharmacodynamics (PD)
Time frame: Up to 156 weeks (Phase 1/2) and through 104 weeks (Sub-study)
To characterize the pharmacokinetics (PK)
Time frame: Up to 156 weeks (Phase 1/2) and through 104 weeks (Sub-study)
To evaluate efficacy
Time frame: Up to 156 weeks (Phase 1/2) and at Week 52 (Sub-study)
Mean change from baseline in Modified Rodnan Skin Score (mRSS)
Time frame: Week 52 (Sub-study)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Oct 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Medical Director study director
Cabaletta Bio
10 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
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.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 16 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Cabaletta Bio (267 759 3100 ext. 4444, clinicaltrials@cabalettabio.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Phase 1/2: To evaluate incidence of adverse events (time frame: Up to 28 days after CABA-201 infusion). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Oct 2026.