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RECRUITING

NCT06154252

PHASE2/PHASE3

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

Sponsor: Cabaletta Bio

View on ClinicalTrials.gov

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

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

Key Details

Gender

All

Age Range

6 Years - 75 Years

Study Type

INTERVENTIONAL

Enrollment

Start Date

2023-12-20

Completion Date

2028-07

Last Updated

2026-04-09

Healthy Volunteers

Conditions

Idiopathic Inflammatory Myopathy Dermatomyositis Anti-Synthetase Syndrome Immune-Mediated Necrotizing Myopathy Juvenile Dermatomyositis Juvenile Polymyositis Juvenile Idiopathic Inflammatory Myopathy (JIIM)Juvenile Myositis

Interventions

BIOLOGICAL

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

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.

Locations (32)

University of California Irvine - Accepting Adult Patients

Orange, California, United States

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

San Francisco, California, United States

Children's Hospital Colorado - Accepting Juvenile Patients

Aurora, Colorado, United States

Mayo Clinic Florida - Accepting Adult Patients

Jacksonville, Florida, United States

Johns Hopkins All Children's Hospital - Accepting Juvenile Patients

St. Petersburg, Florida, United States

Children's Healthcare of Atlanta - Accepting Juvenile Patients

Atlanta, Georgia, United States

Emory University - Accepting Adult Patients

Atlanta, Georgia, United States

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

Chicago, Illinois, United States

Northwestern Memorial Hospital - Accepting Adult Patients

Chicago, Illinois, United States

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

Chicago, Illinois, United States

University of Kansas Medical Center - Accepting Adult Patients

Kansas City, Kansas, United States

National Institutes of Health - Accepting Adult and Juvenile Patients

Bethesda, Maryland, United States

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

Boston, Massachusetts, United States

University of Michigan - Accepting Young Adult and Juvenile Patients

Ann Arbor, Michigan, United States

Mayo Clinic - Accepting Adult Patients

Rochester, Minnesota, United States

Hospital for Special Surgery - Accepting Adult and Juvenile Patients

New York, New York, United States

Memorial Sloan Kettering Cancer Center - Accepting Adult and Juvenile Patients

New York, New York, United States

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

The Bronx, New York, United States

University of North Carolina at Chapel Hill - Accepting Adult Patients

Chapel Hill, North Carolina, United States

Duke University Medical Center - Accepting Young Adults and Juvenile Patients

Durham, North Carolina, United States

Oregon Health & Science University - Accepting Adult Patients

Portland, Oregon, United States

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

Philadelphia, Pennsylvania, United States

UPMC Arthritis and Autoimmunity Center - Accepting Adult Patients

Pittsburgh, Pennsylvania, United States

Vanderbilt University Medical Center - Accepting Adult Patients

Nashville, Tennessee, United States

University of Texas Southwestern Medical Center - Accepting Juvenile Patients

Dallas, Texas, United States

Houston Methodist Hospital - Accepting Adult Patients

Houston, Texas, United States

University of Texas MD Anderson Cancer Center - Accepting Adult Patients

Houston, Texas, United States

Seattle Children's Research Institute - Accepting Juvenile Patients

Seattle, Washington, United States

Kings College Hospital NHS Foundation Trust - Accepting Adult Patients

London, United Kingdom

University College London Hospitals NHS Foundation Trust - Accepting Adult Patients

London, United Kingdom

Manchester Royal Infirmary - Accepting Adult Patients

Manchester, United Kingdom

Salford Royal Hospital - Accepting Adult Patients

Salford, United Kingdom

Clinical Research Directory

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

Summary

Key Details

Conditions

Interventions

Eligibility Criteria

Locations (32)