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

NCT06154252 | Recruiting Phase 2 FDA Drug

• 1 other identifier: CAB-201-002
• Study Type: interventional
• Target: 74
• Locations: 2 countries
• Sites: 35

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

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

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

Outcome Measures

Primary Outcomes (3)

• Phase 1/2: Incidence and severity of adverse events (AEs)

  Incidence and severity of AEs

  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

  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

  Within 24 weeks

Secondary Outcomes (12)

• Incidence of adverse events and laboratory abnormalities

  Up to 156 weeks (Phase 1/2) and through 52 weeks (Sub-study 1 and 2)

• Pharmacodynamics (PD)

  Up to 156 weeks (Phase 1/2) and through 52 weeks (Sub-study 1 and 2)

• Pharmacokinetics (PK)

  Up to 156 weeks (Phase 1/2) and through 52 weeks (Sub-study 1 and 2)

• Change in disease-related biomarkers of muscle inflammation

  Up to 156 weeks (Phase 1/2)

• Change in autoantibody-related biomarkers

  Up to 156 weeks (Phase 1/2)

Study Arms (3)

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.

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.

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.

Biological: CABA-201 following preconditioning with fludarabine and cyclophosphamide

Interventions

CABA-201 following preconditioning with fludarabine and cyclophosphamide BIOLOGICAL

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

Also known as: Rese-cel, Resecabtagene autoleucel

CABA-201 Phase 1/2; CABA-201 Phase 2b - Sub-study 1; CABA-201 Phase 2b - Sub-study 2

Eligibility Criteria

• Age: 6 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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
• 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.
• Contraindication to leukapheresis

Sponsors & Collaborators

• Cabaletta Bio: lead

Study Sites (35)

University of California Irvine - Accepting Adult Patients

Orange, California, 92868, United States

RECRUITING

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

San Francisco, California, 94158, United States

RECRUITING

Children's Hospital Colorado - Accepting Juvenile Patients

Aurora, Colorado, 80046, United States

RECRUITING

Mayo Clinic Florida - Accepting Adult Patients

Jacksonville, Florida, 32224, United States

RECRUITING

Johns Hopkins All Children's Hospital - Accepting Juvenile Patients

St. Petersburg, Florida, 33701, United States

RECRUITING

Children's Healthcare of Atlanta - Accepting Juvenile Patients

Atlanta, Georgia, 30029, United States

RECRUITING

Emory University - Accepting Adult Patients

Atlanta, Georgia, 30032, United States

RECRUITING

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

Chicago, Illinois, 60611, United States

RECRUITING

Northwestern Memorial Hospital - Accepting Adult Patients

Chicago, Illinois, 60611, United States

RECRUITING

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

Chicago, Illinois, 60637, United States

RECRUITING

University of Kansas Medical Center - Accepting Adult Patients

Kansas City, Kansas, 66160, United States

RECRUITING

National Institutes of Health - Accepting Adult and Juvenile Patients

Bethesda, Maryland, 20892, United States

NOT YET RECRUITING

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

Boston, Massachusetts, 02115, United States

RECRUITING

University of Michigan - Accepting Young Adult and Juvenile Patients

Ann Arbor, Michigan, 48105, United States

RECRUITING

Mayo Clinic - Accepting Adult Patients

Rochester, Minnesota, 55902, United States

RECRUITING

Hospital for Special Surgery - Accepting Adult and Juvenile Patients

New York, New York, 10021, United States

RECRUITING

Memorial Sloan Kettering Cancer Center - Accepting Adult and Juvenile Patients

New York, New York, 10021, United States

RECRUITING

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

The Bronx, New York, 10467, United States

RECRUITING

University of North Carolina at Chapel Hill - Accepting Adult Patients

Chapel Hill, North Carolina, 27599, United States

RECRUITING

Duke University Medical Center - Accepting Young Adults and Juvenile Patients

Durham, North Carolina, 27710, United States

RECRUITING

Oregon Health & Science University - Accepting Adult Patients

Portland, Oregon, 97239, United States

RECRUITING

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

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

UPMC Children's Hospital of Pittsburgh - Accepting Juvenile Patients

Pittsburgh, Pennsylvania, 15224, United States

NOT YET RECRUITING

UPMC Arthritis and Autoimmunity Center - Accepting Adult Patients

Pittsburgh, Pennsylvania, 15261, United States

NOT YET RECRUITING

Vanderbilt University Medical Center - Accepting Adult Patients

Nashville, Tennessee, 37232, United States

RECRUITING

University of Texas Southwestern Medical Center - Accepting Juvenile Patients

Dallas, Texas, 75390, United States

RECRUITING

Houston Methodist Hospital - Accepting Adult Patients

Houston, Texas, 77030, United States

RECRUITING

University of Texas MD Anderson Cancer Center - Accepting Adult Patients

Houston, Texas, 77030, United States

RECRUITING

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

Salt Lake City, Utah, 84113, United States

NOT YET RECRUITING

Seattle Children's Research Institute - Accepting Juvenile Patients

Seattle, Washington, 98105, United States

RECRUITING

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

Milwaukee, Wisconsin, 53226, United States

NOT YET RECRUITING

Kings College Hospital NHS Foundation Trust - Accepting Adult Patients

London, SE5 9RS, United Kingdom

RECRUITING

University College London Hospitals NHS Foundation Trust - Accepting Adult Patients

London, WC1N 3BG, United Kingdom

RECRUITING

Manchester Royal Infirmary - Accepting Adult Patients

Manchester, M13 9WL, United Kingdom

RECRUITING

Salford Royal Hospital - Accepting Adult Patients

Salford, M6 8HD, United Kingdom

RECRUITING

MeSH Terms

Conditions

Myositis; Dermatomyositis; Autoimmune Diseases

Interventions

fludarabine; Cyclophosphamide

Condition Hierarchy (Ancestors)

Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Polymyositis; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases; Immune System Diseases

Intervention Hierarchy (Ancestors)

Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Organic Chemicals; Phosphoramides; Organophosphorus Compounds

Study Officials

• Medical Director

  Cabaletta Bio

  STUDY CHAIR

Central Study Contacts

Cabaletta Bio

CONTACT

267-759-3100; clinicaltrials@cabalettabio.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 16, 2023
• First Posted: December 4, 2023
• Study Start: December 20, 2023
• Primary Completion (Estimated): July 1, 2028
• Study Completion (Estimated): July 1, 2028
• Last Updated: April 29, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (31); GB (4)