Recruiting
Phase 2/3
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
Condition(s) studied
Idiopathic Inflammatory MyopathyDermatomyositisAnti-Synthetase SyndromeImmune-Mediated Necrotizing MyopathyJuvenile DermatomyositisJuvenile PolymyositisJuvenile Idiopathic Inflammatory Myopathy (JIIM)Juvenile Myositis
Investigational drug(s) / intervention(s)
CABA-201 following preconditioning with fludarabine and cyclophosphamide
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
Study 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
Eligibility
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.
Primary outcome measure(s)
- Phase 1/2: Incidence and severity of adverse events (AEs) — 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 — 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 — 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
Trial sites (35)
| Facility | City | Region | Status |
| University of California Irvine - Accepting Adult Patients |
Orange |
California |
Recruiting |
| University of California, San Francisco Benioff Children's Hospital - Accepting Young Adult and Juvenile Patients |
San Francisco |
California |
Recruiting |
| Children's Hospital Colorado - Accepting Juvenile Patients |
Aurora |
Colorado |
Recruiting |
| Mayo Clinic Florida - Accepting Adult Patients |
Jacksonville |
Florida |
Recruiting |
| Johns Hopkins All Children's Hospital - Accepting Juvenile Patients |
St. Petersburg |
Florida |
Recruiting |
| Children's Healthcare of Atlanta - Accepting Juvenile Patients |
Atlanta |
Georgia |
Recruiting |
| Emory University - Accepting Adult Patients |
Atlanta |
Georgia |
Recruiting |
| Ann & Robert H. Lurie Children's Hospital of Chicago - Accepting Young Adult and Juvenile Patients |
Chicago |
Illinois |
Recruiting |
| Northwestern Memorial Hospital - Accepting Adult Patients |
Chicago |
Illinois |
Recruiting |
| The University of Chicago Medical Center - Accepting Adult and Juvenile Patients |
Chicago |
Illinois |
Recruiting |
| University of Kansas Medical Center - Accepting Adult Patients |
Kansas City |
Kansas |
Recruiting |
| National Institutes of Health - Accepting Adult and Juvenile Patients |
Bethesda |
Maryland |
Recruiting |
| Boston Children's Hospital - Accepting Young Adults and Juvenile Patients |
Boston |
Massachusetts |
Recruiting |
| University of Michigan - Accepting Young Adult and Juvenile Patients |
Ann Arbor |
Michigan |
Recruiting |
| Mayo Clinic - Accepting Adult Patients |
Rochester |
Minnesota |
Recruiting |
| Hospital for Special Surgery - Accepting Adult and Juvenile Patients |
New York |
New York |
Recruiting |
| Memorial Sloan Kettering Cancer Center - Accepting Adult and Juvenile Patients |
New York |
New York |
Recruiting |
| Children's Hospital at Montefiore - Accepting Young Adult and Juvenile Patients |
The Bronx |
New York |
Recruiting |
| University of North Carolina at Chapel Hill - Accepting Adult Patients |
Chapel Hill |
North Carolina |
Recruiting |
| Duke University Medical Center - Accepting Young Adults and Juvenile Patients |
Durham |
North Carolina |
Recruiting |
| Oregon Health & Science University - Accepting Adult Patients |
Portland |
Oregon |
Recruiting |
| Children's Hospital of Philadelphia - Accepting Young Adult and Juvenile Patients |
Philadelphia |
Pennsylvania |
Recruiting |
| UPMC Children's Hospital of Pittsburgh - Accepting Juvenile Patients |
Pittsburgh |
Pennsylvania |
Recruiting |
| UPMC Arthritis and Autoimmunity Center - Accepting Adult Patients |
Pittsburgh |
Pennsylvania |
Recruiting |
| Vanderbilt University Medical Center - Accepting Adult Patients |
Nashville |
Tennessee |
Recruiting |
| University of Texas Southwestern Medical Center - Accepting Juvenile Patients |
Dallas |
Texas |
Recruiting |
| Houston Methodist Hospital - Accepting Adult Patients |
Houston |
Texas |
Recruiting |
| University of Texas MD Anderson Cancer Center - Accepting Adult Patients |
Houston |
Texas |
Recruiting |
| Primary Children's Hospital/University of Utah - Accepting Juvenile Patients |
Salt Lake City |
Utah |
Recruiting |
| Seattle Children's Research Institute - Accepting Juvenile Patients |
Seattle |
Washington |
Recruiting |
| Children's Wisconsin Pediatric Rheumatology -Accepting Young Adult and Juvenile Patients |
Milwaukee |
Wisconsin |
Not Yet Recruiting |
| Kings College Hospital NHS Foundation Trust - Accepting Adult Patients |
London |
United Kingdom |
Recruiting |
| University College London Hospitals NHS Foundation Trust - Accepting Adult Patients |
London |
United Kingdom |
Recruiting |
| Manchester Royal Infirmary - Accepting Adult Patients |
Manchester |
United Kingdom |
Recruiting |
| Salford Royal Hospital - Accepting Adult Patients |
Salford |
United Kingdom |
Recruiting |
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