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NCT06154252PHASE2, PHASE3Recruiting

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

Cabaletta Bio

Start Date

12/20/2023

Completion Date

7/1/2028

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

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.

Eligibility Criteria

Age Range: 6 years to 75 years

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.

Interventions

BIOLOGICAL

CABA-201 following preconditioning with fludarabine and cyclophosphamide

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Conditions

Idiopathic Inflammatory MyopathyDermatomyositisAnti-Synthetase SyndromeImmune-Mediated Necrotizing MyopathyJuvenile DermatomyositisJuvenile PolymyositisJuvenile Idiopathic Inflammatory Myopathy (JIIM)Juvenile Myositis

Locations

University of California Irvine - Accepting Adult Patients

Orange, California 92868

United States

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

San Francisco, California 94158

United States

Children's Hospital Colorado - Accepting Juvenile Patients

Aurora, Colorado 80046

United States

Mayo Clinic Florida - Accepting Adult Patients

Jacksonville, Florida 32224

United States

Johns Hopkins All Children's Hospital - Accepting Juvenile Patients

St. Petersburg, Florida 33701

United States

Children's Healthcare of Atlanta - Accepting Juvenile Patients

Atlanta, Georgia 30029

United States

Emory University - Accepting Adult Patients

Atlanta, Georgia 30032

United States

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

Chicago, Illinois 60611

United States

Northwestern Memorial Hospital - Accepting Adult Patients

Chicago, Illinois 60611

United States

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

Chicago, Illinois 60637

United States

University of Kansas Medical Center - Accepting Adult Patients

Kansas City, Kansas 66160

United States

National Institutes of Health - Accepting Adult and Juvenile Patients

Bethesda, Maryland 20892

United States

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

Boston, Massachusetts 02115

United States

University of Michigan - Accepting Young Adult and Juvenile Patients

Ann Arbor, Michigan 48105

United States

Mayo Clinic - Accepting Adult Patients

Rochester, Minnesota 55902

United States

Hospital for Special Surgery - Accepting Adult and Juvenile Patients

New York, New York 10021

United States

Memorial Sloan Kettering Cancer Center - Accepting Adult and Juvenile Patients

New York, New York 10021

United States

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

The Bronx, New York 10467

United States

University of North Carolina at Chapel Hill - Accepting Adult Patients

Chapel Hill, North Carolina 27599

United States

Duke University Medical Center - Accepting Young Adults and Juvenile Patients

Durham, North Carolina 27710

United States

Oregon Health & Science University - Accepting Adult Patients

Portland, Oregon 97239

United States

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

Philadelphia, Pennsylvania 19104

United States

UPMC Children's Hospital of Pittsburgh - Accepting Juvenile Patients

Pittsburgh, Pennsylvania 15224

United States

UPMC Arthritis and Autoimmunity Center - Accepting Adult Patients

Pittsburgh, Pennsylvania 15261

United States

Vanderbilt University Medical Center - Accepting Adult Patients

Nashville, Tennessee 37232

United States

University of Texas Southwestern Medical Center - Accepting Juvenile Patients

Dallas, Texas 75390

United States

Houston Methodist Hospital - Accepting Adult Patients

Houston, Texas 77030

United States

University of Texas MD Anderson Cancer Center - Accepting Adult Patients

Houston, Texas 77030

United States

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

Salt Lake City, Utah 84113

United States

Seattle Children's Research Institute - Accepting Juvenile Patients

Seattle, Washington 98105

United States

Children's Wisconsin Pediatric Rheumatology -Accepting Young Adult and Juvenile Patients

Milwaukee, Wisconsin 53226

United States

Kings College Hospital NHS Foundation Trust - Accepting Adult Patients

London, SE5 9RS

United Kingdom

University College London Hospitals NHS Foundation Trust - Accepting Adult Patients

London, WC1N 3BG

United Kingdom

Manchester Royal Infirmary - Accepting Adult Patients

Manchester, M13 9WL

United Kingdom

Salford Royal Hospital - Accepting Adult Patients

Salford, M6 8HD

United Kingdom