Idiopathic Inflammatory MyopathyPhase 2/3NCT06154252
RESET-Myositis: An Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects With Active Idiopathic Inflammatory Myopathy or Juvenile Idiopathic Inflammatory Myopathy
A Phase 2/3 study testing CABA-201 following preconditioning with fludarabine and cyclophosphamide in people with Idiopathic Inflammatory Myopathy and Dermatomyositis, run by Cabaletta Bio at 38 sites in 4 countries.
Trial summary
RESET-Myositis: Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects with Active Idiopathic Inflammatory Myopathy or Juvenile Idiopathic Inflammatory Myopathy Read more about this study
- Who is it for?
- Ages 6 to 75 years
- Conditions the study is for
- Idiopathic Inflammatory Myopathy, Dermatomyositis, Anti-Synthetase Syndrome
- What is being tested?
- CABA-201 following preconditioning with fludarabine and cyclophosphamide
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 74 participants
- Where
- 38 sitesin 4 countries
- Study dates
- 20 Dec 2023to Jul 2028
- 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 6 to 75 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Age ≥18 and ≤75
- A clinical diagnosis of IIM, based on the 2017 The European League Against Rheumatism/American College of Rheumatology classification criteria
- Diagnosis of DM, ASyS, or IMNM
- Evidence of active disease, despite prior or current treatment with standard of care treatments, as defined by the presence of elevated creatine kinase (CK), DM rash, or active disease on muscle biopsy, magnetic resonance imaging (MRI), or electromyography
- Presence of muscle weakness
- Other protocol-defined criteria apply.
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- Age ≥6 and ≤17 years at enrollment
- A clinical diagnosis of IIM, based on the 2017 The European League Against Rheumatism/American College of Rheumatology classification criteria
- Evidence of active disease, despite prior or current treatment with standard of care treatments, as defined by the presence of elevated muscle enzymes, DM rash, or active disease on muscle biopsy, magnetic resonance imaging (MRI), or electromyography
- Other protocol-defined criteria apply.
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
- 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
- Significant 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 criteria apply.
- Juvenile Cohort
- 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
- 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
- Significant lung or cardiac impairment
- Previous CAR T cell therapy
- Prior solid organ (heart, liver, kidney, lung) transplant or hematopoietic cell transplant
- Other protocol-defined criteria apply.
Trial locations and contacts
38 sites in 4 countries. Contact the study team or a site near you to ask about taking part.
38 sites in 4 countries
United States31 sites, 31 recruiting
- Recruiting
University of Michigan - Accepting Young Adult and Juvenile Patients
Ann Arbor, Michigan, 48105
Abigail Whalen, 734-615-4466, abigaiwh@med.umich.edu
Dr. Jennifer Agrusa, MD
- Recruiting
Children's Healthcare of Atlanta - Accepting Juvenile Patients
Atlanta, Georgia, 30029
Judson Russell, 404-785-7263, Judson.russell@choa.org
Shanmuganathan Chandrakasan, MD
- Recruiting
Emory University - Accepting Adult Patients
Atlanta, Georgia, 30032
John Varghese, john.varghese@emory.edu
Prateek Chandrashekar Gandiga, MD, FACP
- Recruiting
Children's Hospital Colorado - Accepting Juvenile Patients
Aurora, Colorado, 80046
Callie Strini - Primary Clinical Research Coordinator, 720-777-9845, callie.strini@childrenscolorado.org
Dr. Kentaro Yomogida, MD
- Recruiting
National Institutes of Health - Accepting Adult and Juvenile Patients
Bethesda, Maryland, 20892
Julie Thompson, 301-480-3191, Julie.thompson@nih.gov
Andrew Mammen, M.D., Ph.D.
- Recruiting
Boston Children's Hospital - Accepting Young Adults and Juvenile Patients
Boston, Massachusetts, 02115
Kyle McBrearty, 617-355-2780, kyle.mcbrearty@childrens.harvard.edu
Susan Prockop, MD
- Recruiting
University of North Carolina at Chapel Hill - Accepting Adult Patients
Chapel Hill, North Carolina, 27599
Leah Luizza, leah.luizza@med.unc.edu
Saira Sheikh, MD
- Recruiting
Ann & Robert H. Lurie Children's Hospital of Chicago - Accepting Young Adult and Juvenile Patients
Chicago, Illinois, 60611
Tyler Sorensen, 312-277-6828, tsorensen@luriechildrens.org
Pooja N. Patel, DO
- Recruiting
Northwestern Memorial Hospital - Accepting Adult Patients
Chicago, Illinois, 60611
Kaitlin King, 312-695-0990, autoimmunesct@nm.org
Dr. George Georges, MD
- Recruiting
The University of Chicago Medical Center - Accepting Adult and Juvenile Patients
Chicago, Illinois, 60637
Dr. Iazsmin Ventura, MD - Adult patient contact, 773-702-6619, iventura@uchicago.edu; Dequana.jones@bsd.uchicago.edu
Dr. Cuoghi Edens, MD, FAAP - Juvenile patient contact, cedens@bsd.uchicago.edu; Sydni.Hill@bsd.uchicago.edu
- Recruiting
University of Texas Southwestern Medical Center - Accepting Juvenile Patients
Dallas, Texas, 75390
Dr. Samuel John, dtpteam@childrens.com
Samuel John, MD
- Recruiting
Duke University Medical Center - Accepting Young Adults and Juvenile Patients
Durham, North Carolina, 27710
Lynn Rodgers, MS, 919-668-2953, Lynn.rodgers@duke.edu
Kaveh Ardalan, MD, MS
- Recruiting
Houston Methodist Hospital - Accepting Adult Patients
Houston, Texas, 77030
Amelia A. Nounes, MSN, RN, CCRP, 346-356-3639, aanounes@houstonmethodist.org
- Recruiting
University of Texas MD Anderson Cancer Center - Accepting Adult Patients
Houston, Texas, 77030
Christina Amos Briggs, BSN, RN, 713-563-5076, cbamos@mdanderson.org
Huifang (Linda) Lu, MD
- Recruiting
Mayo Clinic Florida - Accepting Adult Patients
Jacksonville, Florida, 32224
Gabrielle Pariseau, 904-953-5549, pariseau.gabrielle@mayo.edu
- Recruiting
University of Kansas Medical Center - Accepting Adult Patients
Kansas City, Kansas, 66160
Samantha Colgan, 913-945-9938, scolgan@kumc.edu
- Not yet recruiting
Children's Wisconsin Pediatric Rheumatology -Accepting Young Adult and Juvenile Patients
Milwaukee, Wisconsin, 53226
Erin Hammelev - Research Coordinator, 414-337-7064, ehammele@mcw.edu
Sara Sabbagh, DO
- Recruiting
Vanderbilt University Medical Center - Accepting Adult Patients
Nashville, Tennessee, 37232
Tofi Soneye, 817-883-4730, tofi.soneye@vumc.org
- Recruiting
Hospital for Special Surgery - Accepting Adult and Juvenile Patients
New York, New York, 10021
Janvi Bhatia, 212-774-2123, bhatiaj@hss.edu
David R. Fernandez, MD, PhD
- Recruiting
Memorial Sloan Kettering Cancer Center - Accepting Adult and Juvenile Patients
New York, New York, 10021
Atifah Osrat, 646-608-2091, osrata1@mskcc.org
Jae Park, MD
- Recruiting
University of California Irvine - Accepting Adult Patients
Orange, California, 92868
Alpha Clinic, 949-824-3990, alphaclinic@uci.edu
Tahseen Mozaffar, MD
- Recruiting
Children's Hospital of Philadelphia - Accepting Young Adult and Juvenile Patients
Philadelphia, Pennsylvania, 19104
CARTintake@chop.edu, CARTintake@chop.edu
Dr. Caitlin Elgarten, MD, MSCE
- Recruiting
UPMC Children's Hospital of Pittsburgh - Accepting Juvenile Patients
Pittsburgh, Pennsylvania, 15224
Vibha Chauhan, PhD, 412-692-7924, Vibha.chauhan@chp.edu
Kathryn Torok, MD
- Recruiting
UPMC Arthritis and Autoimmunity Center - Accepting Adult Patients
Pittsburgh, Pennsylvania, 15261
Carol Oriss, orissca@upmc.edu
Jeremy Tilstra, MD
- Recruiting
Oregon Health & Science University - Accepting Adult Patients
Portland, Oregon, 97239
Katie Lewis, 503-908-9724, lewiskat@ohsu.edu
- Recruiting
Mayo Clinic - Accepting Adult Patients
Rochester, Minnesota, 55902
Bridget Neja, 507-266-9150, neja.bridget@mayo.edu
- Recruiting
Primary Children's Hospital/University of Utah - Accepting Juvenile Patients
Salt Lake City, Utah, 84113
Morgan Badgley - Research Coordinator, 801-662-4812, morgan.badgley@hsc.utah.edu
Aimee Ogden Hersh, MD
- Recruiting
University of California, San Francisco Benioff Children's Hospital - Accepting Young Adult and Juvenile Patients
San Francisco, California, 94158
Zilan Zheng, 415-353-1301, Zilan.Zheng@ucsf.edu
Susan Kim, MD, MMSc
- Recruiting
Seattle Children's Research Institute - Accepting Juvenile Patients
Seattle, Washington, 98105
Dr. Susan Shenoi, MD, MS, 206-987-2193, susan.shenoi@SeattleChildrens.org
Dr. Susan Shenoi, MD, MS
- Recruiting
Johns Hopkins All Children's Hospital - Accepting Juvenile Patients
St. Petersburg, Florida, 33701
Laura Escobar, 727-767-2946, llabarr2@jh.edu
Natalie Booth, DO
- Recruiting
Children's Hospital at Montefiore - Accepting Young Adult and Juvenile Patients
The Bronx, New York, 10467
Emily Gillies, 718-696-2402, egillies@montefiore.org
Dr. Dawn Wahezi, MD, MS
United Kingdom5 sites, 5 recruiting
- Recruiting
Kings College Hospital NHS Foundation Trust - Accepting Adult Patients
London, SE5 9RS
Rheumatology Research Team, 0207 848 0214 ext. 0628, Kch-tr.CRDresearch@nhs.net
Patrick Gordon, PhD, FRCP, MBBS
- Recruiting
University College London Hospitals NHS Foundation Trust - Accepting Adult Patients
London, WC1N 3BG
Dr. Pedro Machado, FRCP, PhD, 020 3456 7890, uclh.car-ttrials@nhs.net
Dr. Pedro Machado, FRCP, PhD
- Recruiting
Great Ormond Street Hospital for Children NHS Foundation Trust - Accepting Juvenile Patients
London, WC1N 3JH
Professor Despina Eleftheriou, 020 7405 9200, Despina.Eleftheriou@gosh.nhs.uk
Professor Despina Eleftheriou
- Recruiting
Manchester Royal Infirmary - Accepting Adult Patients
Manchester, M13 9WL
Eleni Tholouli, MD, PhD, MRCP(UK), FRCPath, 01612765052, Eleni.Tholouli@mft.nhs.uk
Eleni Tholouli, MD, PhD, MRCP(UK), FRCPath
- Recruiting
Salford Royal Hospital - Accepting Adult Patients
Salford, M6 8HD
Michelle Beswick, 07759 136787, Rheumatology.Research@nca.nhs.uk
Hector Chinoy, PhD, FRCP, MSc, BMBS, BMedSci
Germany1 site, 1 recruiting
- Not yet recruiting
Universitätsmedizin Göttingen - Accepting Adult Patients
Göttingen, 37075
Dr. Jana Zschüntzsch, j.zschuentzsch@med.uni-goettingen.de
Dr. Jana Zschüntzsch
Italy1 site, 1 recruiting
- Not yet recruiting
Ospedale Pediatrico Bambino Gesù - Accepting Young Adult and Juvenile Patients
Roma, 00165
Dr. Marco Becilli, +39 0668592739, marco.becilli@opbg.net
Professor Franco Locatelli
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 Active Idiopathic Inflammatory Myopathy or Juvenile Idiopathic Inflammatory Myopathy
RESET-Myositis: Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects with Active Idiopathic Inflammatory Myopathy or Juvenile Idiopathic Inflammatory Myopathy
Read the detailed description
Idiopathic inflammatory myopathies (IIMs, or myositis) are a group of rare autoimmune diseases characterized by inflammation and muscle weakness. Though the cause of IIM is not well understood, some subtypes of IIM, including dermatomyositis (DM), anti-synthetase syndrome (ASyS), immune-mediated necrotizing myopathy (IMNM), and juvenile idiopathic inflammatory myopathy (JIIM), are thought to involve B cells that cause the body to attack different tissues in the body. 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 DM, ASyS, IMNM, or JIIM who have active disease. A single dose of CABA-201 in combination with cyclophosphamide (CY) and fludarabine (FLU) will be evaluated.
- Conditions
- Idiopathic Inflammatory Myopathy, Dermatomyositis, Anti-Synthetase Syndrome, Immune-Mediated Necrotizing Myopathy, Juvenile Dermatomyositis, Juvenile Polymyositis
- Being tested
- CABA-201 following preconditioning with fludarabine and cyclophosphamide
- Keywords
- CABA-201, Autoimmune Disease, Anti-CD19 CAR-T therapy, Cellular Therapy, Idiopathic Inflammatory Myopathy, Myositis, Dermatomyositis, Anti-synthetase Syndrome, Immune-mediated Necrotizing Myopathy, Juvenile Dermatomyositis, Juvenile Polymyositis, Juvenile Idiopathic Inflammatory Myopathy (JIIM), Juvenile Myositis
- Medical subject headings
- Myositis, Dermatomyositis, Autoimmune Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CABA-201 following preconditioning with fludarabine and cyclophosphamideBiological
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 | DM Cohort: Infusion of CABA-201 following preconditioning with fludarabine and cyclophosphamide in subjects with DM.
ASyS Cohort: Infusion of CABA-201 following preconditioning with fludarabine and cyclophosphamide in subjects with ASyS.
IMNM Cohort: Infusion of CABA-201 following preconditioning with fludarabine and cyclophosphamide in subjects with IMNM.
JIIM Cohort: Infusion of CABA-201 following preconditioning with fludarabine and cyclophosphamide in subjects with JIIM. Receives: Biological: CABA-201 following preconditioning with fludarabine and cyclophosphamide |
| CABA-201 Phase 2b - Sub-study 1 | Experimental | Infusion of CABA-201 following preconditioning with fludarabine and cyclophosphamide in subjects with DM and ASyS. Receives: Biological: CABA-201 following preconditioning with fludarabine and cyclophosphamide |
| CABA-201 Phase 2b - Sub-study 2 | Experimental | Infusion of CABA-201 following preconditioning with fludarabine and cyclophosphamide in subjects with IMNM. Receives: Biological: CABA-201 following preconditioning with fludarabine and cyclophosphamide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Phase 1/2: Incidence and severity of adverse events (AEs)
Incidence and severity of AEs
Measured: Up to 28 days after CABA-201 infusion
Phase 2b Sub-study 1: Proportion of DM & ASyS subjects achieving at least a moderate Total Improvement Score (TIS) without any immunomodulatory medications and no or low dose of steroids
≥40 on the TIS, a composite measure ranging from 0 to 100, derived from six Core Set Measures, with higher scores indicating greater clinical improvement
Measured: Within 16 weeks
Phase 2b Sub-study 2: Proportion of subjects with IMNM achieving at least a minimal TIS without any immunomodulatory medications and no or low dose of steroids
≥20 on the TIS, a composite measure ranging from 0 to 100, derived from six Core Set Measures, with higher scores indicating greater clinical improvement
Measured: Within 24 weeks
Secondary outcomes (12)
Incidence of 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 52 weeks (Sub-study 1 and 2)
Pharmacodynamics (PD)
Levels of B cells in the blood
Measured: Up to 156 weeks (Phase 1/2) and through 52 weeks (Sub-study 1 and 2)
Pharmacokinetics (PK)
Levels of CABA-201-positive T cells in the blood
Measured: Up to 156 weeks (Phase 1/2) and through 52 weeks (Sub-study 1 and 2)
Change in disease-related biomarkers of muscle inflammation
Levels of muscle enzymes (CK, LDH, AST, ALT, and aldolase) in serum
Measured: Up to 156 weeks (Phase 1/2)
Change in autoantibody-related biomarkers
Levels of autoantibodies from the Myositis-Specific Autoantibody Panel (e.g., MDA-5, Jo-1, and HMGCR) in the serum
Measured: Up to 156 weeks (Phase 1/2)
Proportion of DM subjects achieving at least a moderate TIS without any immunomodulatory medications and no or low dose of steroids
≥40 on the TIS, a composite measure ranging from 0 to 100, derived from six Core Set Measures, with higher scores indicating greater clinical improvement
Measured: Week 16 (Sub-study 1)
Mean change from baseline on the MMT-8 in subjects with DM and ASyS without any immunomodulatory medications and no or low dose of steroids
MMT-8 measures muscle strength on a scale ranging from 0 to 150 points, where higher scores reflect greater muscle strength
Measured: Week 16 (Sub-study 1)
Proportion of DM and ASyS subjects achieving a major TIS response without any immunomodulatory medications and no or low dose of steroids
≥60 on the TIS, a composite measure ranging from 0 to 100, derived from six Core Set Measures, with higher scores indicating greater clinical improvement
Measured: Week 16 (Sub-study 1)
Proportion of subjects with DM & ASyS achieving moderate TIS without any immunomodulatory medications on no or low dose of steroids
≥40 on the TIS, a composite measure ranging from 0 to 100, derived from six Core Set Measures, with higher scores indicating greater clinical improvement
Measured: Week 52 (Sub-study 1)
Proportion of subjects with DM achieving moderate TIS without any immunomodulatory medications on no or low dose of steroids
≥40 on the TIS, a composite measure ranging from 0 to 100, derived from six Core Set Measures, with higher scores indicating greater clinical improvement
Measured: Week 52 (Sub-study 1)
Mean change from baseline on the MMT-8 in subjects with IMNM without any immunomodulatory medications and no or low dose of steroids
MMT-8 measures muscle strength on a scale ranging from 0 to 150 points, where higher scores reflect greater muscle strength
Measured: Week 24 (Sub-study 2)
Proportion of subjects with IMNM achieving at least minimal TIS response without immunomodulatory medications and no or low dose of steroids
≥20 on the TIS, a composite measure ranging from 0 to 100, derived from six Core Set Measures, with higher scores indicating greater clinical improvement
Measured: Week 52 (Sub-study 2)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Dec 2023
- Study start20 Dec 2023
- Primary completionJul 2028 (estimated)
- Study completionJul 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Idiopathic Inflammatory MyopathyStudies 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
- 74 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Cabaletta Bio
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study chair, Cabaletta Bio
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06154252
- Sponsor's protocol ID
- CAB-201-002
- Status last verified
- Oct 2026
- First submitted
- 16 Nov 2023
- Last updated
- 15 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Volkov J, Nunez D, Mozaffar T, Stadanlick J, Werner M, Vorndran Z, Ellis A, Williams J, Cicarelli J, Lam Q, Furmanak T, Schmitt C, Hadi-Nezhad F, Thompson D, Miller C, Little C, Chang D, Basu S. Case study of CD19 CAR T therapy in a subject with immune-mediate necrotizing myopathy treated in the RESET-Myositis phase I/II trial. Mol Ther. 2024 Nov 6;32(11):3821-3828. doi: 10.1016/j.ymthe.2024.09.009. Epub 2024 Sep 7.
PubMed 39245937
Study at a glance
From the registry record, last updated by the sponsor on 15 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 Active Idiopathic Inflammatory Myopathy or Juvenile Idiopathic Inflammatory Myopathy
- ClinicalTrials.gov ID
- NCT06154252
- Acronym / sponsor ID
- CAB-201-002
- Conditions
- Idiopathic Inflammatory Myopathy, Dermatomyositis, Anti-Synthetase Syndrome, Immune-Mediated Necrotizing Myopathy, Juvenile Dermatomyositis, Juvenile Polymyositis
- Keywords
- CABA-201, Autoimmune Disease, Anti-CD19 CAR-T therapy, Cellular Therapy, Idiopathic Inflammatory Myopathy, Myositis, Dermatomyositis, Anti-synthetase Syndrome
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 74 participants (estimated)
- Lead sponsor
- Cabaletta BioIndustry (pharma, biotech, devices)
- Study start
- 20 Dec 2023 (actual)
- Primary completion
- Jul 2028 (estimated)
- Study completion
- Jul 2028 (estimated)
- First posted
- 4 Dec 2023 (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-Myositis: Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects with Active Idiopathic Inflammatory Myopathy or Juvenile Idiopathic Inflammatory Myopathy
Detailed description
Idiopathic inflammatory myopathies (IIMs, or myositis) are a group of rare autoimmune diseases characterized by inflammation and muscle weakness. Though the cause of IIM is not well understood, some subtypes of IIM, including dermatomyositis (DM), anti-synthetase syndrome (ASyS), immune-mediated necrotizing myopathy (IMNM), and juvenile idiopathic inflammatory myopathy (JIIM), are thought to involve B cells that cause the body to attack different tissues in the body. 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 DM, ASyS, IMNM, or JIIM 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
- 3
| Arm | Type | Receives |
|---|---|---|
| CABA-201 Phase 1/2 | Experimental | Biological: CABA-201 following preconditioning with fludarabine and cyclophosphamideDM Cohort: Infusion of CABA-201 following preconditioning with fludarabine and cyclophosphamide in subjects with DM. ASyS Cohort: Infusion of CABA-201 following preconditioning with fludarabine and cyclophosphamide in subjects with ASyS. IMNM Cohort: Infusion of CABA-201 following preconditioning with fludarabine and cyclophosphamide in subjects with IMNM. JIIM Cohort: Infusion of CABA-201 following preconditioning with fludarabine and cyclophosphamide in subjects with JIIM. |
| CABA-201 Phase 2b - Sub-study 1 | Experimental | Biological: CABA-201 following preconditioning with fludarabine and cyclophosphamideInfusion of CABA-201 following preconditioning with fludarabine and cyclophosphamide in subjects with DM and ASyS. |
| CABA-201 Phase 2b - Sub-study 2 | Experimental | Biological: CABA-201 following preconditioning with fludarabine and cyclophosphamideInfusion of CABA-201 following preconditioning with fludarabine and cyclophosphamide in subjects with IMNM. |
Who may qualify
- Ages
- 6 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (10 listed)
- Age ≥18 and ≤75
- A clinical diagnosis of IIM, based on the 2017 The European League Against Rheumatism/American College of Rheumatology classification criteria
- Diagnosis of DM, ASyS, or IMNM
- Evidence of active disease, despite prior or current treatment with standard of care treatments, as defined by the presence of elevated creatine kinase (CK), DM rash, or active disease on muscle biopsy, magnetic resonance imaging (MRI), or electromyography
- Presence of muscle weakness
Key exclusion criteria (19 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
- 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-201 following preconditioning with fludarabine and cyclophosphamideAlso: 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-201 Phase 2b - Sub-study 1, CABA-201 Phase 2b - Sub-study 2 |
Primary outcome
Phase 1/2: Incidence and severity of adverse events (AEs)
Time frame: Up to 28 days after CABA-201 infusion
Incidence and severity of AEs
Phase 2b Sub-study 1: Proportion of DM & ASyS subjects achieving at least a moderate Total Improvement Score (TIS) without any immunomodulatory medications and no or low dose of steroids
Time frame: Within 16 weeks
≥40 on the TIS, a composite measure ranging from 0 to 100, derived from six Core Set Measures, with higher scores indicating greater clinical improvement
Phase 2b Sub-study 2: Proportion of subjects with IMNM achieving at least a minimal TIS without any immunomodulatory medications and no or low dose of steroids
Time frame: Within 24 weeks
≥20 on the TIS, a composite measure ranging from 0 to 100, derived from six Core Set Measures, with higher scores indicating greater clinical improvement
12 secondary outcomes
Incidence of adverse events and laboratory abnormalities
Time frame: Up to 156 weeks (Phase 1/2) and through 52 weeks (Sub-study 1 and 2)
Pharmacodynamics (PD)
Time frame: Up to 156 weeks (Phase 1/2) and through 52 weeks (Sub-study 1 and 2)
Pharmacokinetics (PK)
Time frame: Up to 156 weeks (Phase 1/2) and through 52 weeks (Sub-study 1 and 2)
Change in disease-related biomarkers of muscle inflammation
Time frame: Up to 156 weeks (Phase 1/2)
Change in autoantibody-related biomarkers
Time frame: Up to 156 weeks (Phase 1/2)
Proportion of DM subjects achieving at least a moderate TIS without any immunomodulatory medications and no or low dose of steroids
Time frame: Week 16 (Sub-study 1)
Mean change from baseline on the MMT-8 in subjects with DM and ASyS without any immunomodulatory medications and no or low dose of steroids
Time frame: Week 16 (Sub-study 1)
Proportion of DM and ASyS subjects achieving a major TIS response without any immunomodulatory medications and no or low dose of steroids
Time frame: Week 16 (Sub-study 1)
Proportion of subjects with DM & ASyS achieving moderate TIS without any immunomodulatory medications on no or low dose of steroids
Time frame: Week 52 (Sub-study 1)
Proportion of subjects with DM achieving moderate TIS without any immunomodulatory medications on no or low dose of steroids
Time frame: Week 52 (Sub-study 1)
Mean change from baseline on the MMT-8 in subjects with IMNM without any immunomodulatory medications and no or low dose of steroids
Time frame: Week 24 (Sub-study 2)
Proportion of subjects with IMNM achieving at least minimal TIS response without immunomodulatory medications and no or low dose of steroids
Time frame: Week 52 (Sub-study 2)
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 15 Oct 2026.
- Derived from this study: PubMed 39245937Volkov J, Nunez D, Mozaffar T, Stadanlick J, Werner M, Vorndran Z, Ellis A, Williams J, Cicarelli J, Lam Q, Furmanak T, Schmitt C, Hadi-Nezhad F, Thompson D, Miller C, Little C, Chang D, Basu S. Case study of CD19 CAR T therapy in a subject with immune-mediate necrotizing myopathy treated in the RESET-Myositis phase I/II trial. Mol Ther. 2024 Nov 6;32(11):3821-3828. doi: 10.1016/j.ymthe.2024.09.009. Epub 2024 Sep 7.
- 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 chair
Cabaletta Bio
38 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing CABA-201 following preconditioning with fludarabine and cyclophosphamide in people with Idiopathic Inflammatory Myopathy and Dermatomyositis, run by Cabaletta Bio at 38 sites in 4 countries.
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 10 inclusion and 19 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 15 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 38 sites in United States, United Kingdom, Germany, Italy. 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: Incidence and severity of adverse events (AEs) (time frame: Up to 28 days after CABA-201 infusion). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06154252, which also lists 1 linked publication.
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 15 Oct 2026.