Recruiting
Phase 2
Phase 3

CABA-201

Sponsor:

Cabaletta Bio

Code:

NCT06154252

Conditions

Idiopathic Inflammatory Myopathy

Dermatomyositis

Anti-Synthetase Syndrome

Immune-Mediated Necrotizing Myopathy

Juvenile Dermatomyositis

Eligibility Criteria

Sex: All

Age: 6 - 70+

Healthy Volunteers: Not accepted

Interventions

CABA-201 following preconditioning with fludarabine and cyclophosphamide

Study Details

Brief 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

Conditions

Idiopathic Inflammatory Myopathy

Dermatomyositis

Anti-Synthetase Syndrome

Immune-Mediated Necrotizing Myopathy

Juvenile Dermatomyositis

Study ID

NCT06154252

Start date

Dec 20, 2023

Status verified date

Sep, 2026

Completion date

Jul, 2028

Anticipated

Primary completion date

Jul, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 6 - 70+

Healthy Volunteers: Not accepted

Adult Cohorts

Inclusion Criteria:

  • 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.

Exclusion Criteria:

  • 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.

Juvenile Cohort

Inclusion Criteria:

  • 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.

Exclusion Criteria:

  • 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.

Study Design

Enrollment

74 participants

Anticipated

Allocation

Non randomized

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: CABA-201 Phase 1/2

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.

experimental: CABA-201 Phase 2b - Sub-study 1

Infusion of CABA-201 following preconditioning with fludarabine and cyclophosphamide in subjects with DM and ASyS.

experimental: CABA-201 Phase 2b - Sub-study 2

Infusion of CABA-201 following preconditioning with fludarabine and cyclophosphamide in subjects with IMNM.

Interventions

CABA-201 following preconditioning with fludarabine and cyclophosphamide

Single intravenous infusion of CABA-201 at a single dose level following preconditioning with fludarabine and cyclophosphamide

Primary outcome measure

  • Phase 1/2: Incidence and severity of adverse events (AEs) [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: Within 24 weeks ]

Central Contacts and Locations

Central contacts

Cabaletta Bio

267-759-3100clinicaltrials@cabalettabio.com

Locations

University of California Irvine - Accepting Adult Patients

Recruiting

Orange, California, United States, 92868

Contacts

Alpha Clinic

949-824-3990alphaclinic@uci.edu

Principal Investigator:

Tahseen Mozaffar, MD

University of California, San Francisco Benioff Children's Hospital - Accepting Young Adult and Juvenile Patients

Recruiting

San Francisco, California, United States, 94158

Contacts

Zilan Zheng

415-353-1301Zilan.Zheng@ucsf.edu

Principal Investigator:

Susan Kim, MD, MMSc

Children's Hospital Colorado - Accepting Juvenile Patients

Recruiting

Aurora, Colorado, United States, 80046

Contacts

Callie Strini - Primary Clinical Research Coordinator

720-777-9845callie.strini@childrenscolorado.org

Principal Investigator:

Dr. Kentaro Yomogida, MD

Mayo Clinic Florida - Accepting Adult Patients

Recruiting

Jacksonville, Florida, United States, 32224

Contacts

Gabrielle Pariseau

904-953-5549pariseau.gabrielle@mayo.edu

Johns Hopkins All Children's Hospital - Accepting Juvenile Patients

Recruiting

St. Petersburg, Florida, United States, 33701

Contacts

Laura Escobar

727-767-2946llabarr2@jh.edu

Principal Investigator:

Natalie Booth, DO

Children's Healthcare of Atlanta - Accepting Juvenile Patients

Recruiting

Atlanta, Georgia, United States, 30029

Contacts

Judson Russell

404-785-7263Judson.russell@choa.org

Principal Investigator:

Shanmuganathan Chandrakasan, MD

Emory University - Accepting Adult Patients

Recruiting

Atlanta, Georgia, United States, 30032

Contacts

John Varghese

john.varghese@emory.edu

Principal Investigator:

Prateek Chandrashekar Gandiga, MD, FACP

Ann & Robert H. Lurie Children's Hospital of Chicago - Accepting Young Adult and Juvenile Patients

Recruiting

Chicago, Illinois, United States, 60611

Contacts

Tyler Sorensen

312-277-6828tsorensen@luriechildrens.org

Principal Investigator:

Pooja N. Patel, DO

Northwestern Memorial Hospital - Accepting Adult Patients

Recruiting

Chicago, Illinois, United States, 60611

Contacts

Kaitlin King

312-695-0990autoimmunesct@nm.org

Principal Investigator:

Dr. George Georges, MD

The University of Chicago Medical Center - Accepting Adult and Juvenile Patients

Recruiting

Chicago, Illinois, United States, 60637

Contacts

Dr. Iazsmin Ventura, MD - Adult patient contact

773-702-6619iventura@uchicago.edu; Dequana.jones@bsd.uchicago.edu

Dr. Cuoghi Edens, MD, FAAP - Juvenile patient contact

cedens@bsd.uchicago.edu; Sydni.Hill@bsd.uchicago.edu

Principal Investigator:

Dr. Iazsmin Ventura, MD

University of Kansas Medical Center - Accepting Adult Patients

Recruiting

Kansas City, Kansas, United States, 66160

Contacts

Samantha Colgan

913-945-9938scolgan@kumc.edu

National Institutes of Health - Accepting Adult and Juvenile Patients

Recruiting

Bethesda, Maryland, United States, 20892

Contacts

Julie Thompson

301-480-3191Julie.thompson@nih.gov

Principal Investigator:

Andrew Mammen, M.D., Ph.D.

Boston Children's Hospital - Accepting Young Adults and Juvenile Patients

Recruiting

Boston, Massachusetts, United States, 02115

Contacts

Kyle McBrearty

617-355-2780kyle.mcbrearty@childrens.harvard.edu

Principal Investigator:

Susan Prockop, MD

University of Michigan - Accepting Young Adult and Juvenile Patients

Recruiting

Ann Arbor, Michigan, United States, 48105

Contacts

Abigail Whalen

734-615-4466abigaiwh@med.umich.edu

Principal Investigator:

Dr. Jennifer Agrusa, MD

Mayo Clinic - Accepting Adult Patients

Recruiting

Rochester, Minnesota, United States, 55902

Contacts

Bridget Neja

507-266-9150neja.bridget@mayo.edu

Hospital for Special Surgery - Accepting Adult and Juvenile Patients

Recruiting

New York, New York, United States, 10021

Contacts

Janvi Bhatia

212-774-2123bhatiaj@hss.edu

Principal Investigator:

David R. Fernandez, MD, PhD

Memorial Sloan Kettering Cancer Center - Accepting Adult and Juvenile Patients

Recruiting

New York, New York, United States, 10021

Contacts

Atifah Osrat

646-608-2091osrata1@mskcc.org

Principal Investigator:

Jae Park, MD

Children's Hospital at Montefiore - Accepting Young Adult and Juvenile Patients

Recruiting

The Bronx, New York, United States, 10467

Contacts

Emily Gillies

718-696-2402egillies@montefiore.org

Principal Investigator:

Dr. Dawn Wahezi, MD, MS

University of North Carolina at Chapel Hill - Accepting Adult Patients

Recruiting

Chapel Hill, North Carolina, United States, 27599

Contacts

Leah Luizza

leah.luizza@med.unc.edu

Principal Investigator:

Saira Sheikh, MD

Duke University Medical Center - Accepting Young Adults and Juvenile Patients

Recruiting

Durham, North Carolina, United States, 27710

Contacts

Lynn Rodgers, MS

919-668-2953Lynn.rodgers@duke.edu

Principal Investigator:

Kaveh Ardalan, MD, MS

Oregon Health & Science University - Accepting Adult Patients

Recruiting

Portland, Oregon, United States, 97239

Contacts

Katie Lewis

503-908-9724lewiskat@ohsu.edu

Children's Hospital of Philadelphia - Accepting Young Adult and Juvenile Patients

Recruiting

Philadelphia, Pennsylvania, United States, 19104

Contacts

CARTintake@chop.edu

CARTintake@chop.edu

Principal Investigator:

Dr. Caitlin Elgarten, MD, MSCE

UPMC Children's Hospital of Pittsburgh - Accepting Juvenile Patients

Recruiting

Pittsburgh, Pennsylvania, United States, 15224

Contacts

Vibha Chauhan, PhD

412-692-7924Vibha.chauhan@chp.edu

Principal Investigator:

Kathryn Torok, MD

UPMC Arthritis and Autoimmunity Center - Accepting Adult Patients

Recruiting

Pittsburgh, Pennsylvania, United States, 15261

Contacts

Carol Oriss

orissca@upmc.edu

Principal Investigator:

Jeremy Tilstra, MD

Vanderbilt University Medical Center - Accepting Adult Patients

Recruiting

Nashville, Tennessee, United States, 37232

Contacts

Jon Williams

jon.williams@vumc.org

University of Texas Southwestern Medical Center - Accepting Juvenile Patients

Recruiting

Dallas, Texas, United States, 75390

Contacts

Dr. Samuel John

dtpteam@childrens.com

Principal Investigator:

Samuel John, MD

Houston Methodist Hospital - Accepting Adult Patients

Recruiting

Houston, Texas, United States, 77030

Contacts

Amelia A. Nounes, MSN, RN, CCRP

346-356-3639aanounes@houstonmethodist.org

University of Texas MD Anderson Cancer Center - Accepting Adult Patients

Recruiting

Houston, Texas, United States, 77030

Contacts

Christina Amos Briggs, BSN, RN

713-563-5076cbamos@mdanderson.org

Principal Investigator:

Huifang (Linda) Lu, MD

Primary Children's Hospital/University of Utah - Accepting Juvenile Patients

Recruiting

Salt Lake City, Utah, United States, 84113

Contacts

Morgan Badgley - Research Coordinator

801-662-4812morgan.badgley@hsc.utah.edu

Principal Investigator:

Aimee Ogden Hersh, MD

Seattle Children's Research Institute - Accepting Juvenile Patients

Recruiting

Seattle, Washington, United States, 98105

Contacts

Dr. Susan Shenoi, MD, MS

206-987-2193susan.shenoi@SeattleChildrens.org

Principal Investigator:

Dr. Susan Shenoi, MD, MS

More Information

Sponsor

Cabaletta Bio

Last update posted

Sep 2, 2026

Last verified

Sep, 2026

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

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by Cabaletta Bio on 2026-09-02.