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
Purpose
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
- Juvenile Polymyositis
- Juvenile Idiopathic Inflammatory Myopathy (JIIM)
- Juvenile Myositis
Eligibility
- Eligible Ages
- Between 6 Years and 75 Years
- Eligible Sex
- All
- Accepts Healthy Volunteers
- No
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
- Phase
- Phase 2/Phase 3
- Study Type
- Interventional
- Allocation
- Non-Randomized
- Intervention Model
- Single Group Assignment
- Primary Purpose
- Treatment
- Masking
- None (Open Label)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
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. |
|
Recruiting Locations
University of California Irvine - Accepting Adult Patients
Orange, California 92868
Orange, California 92868
University of California, San Francisco Benioff Children's Hospital - Accepting Young Adult and Juvenile Patients
San Francisco, California 94158
San Francisco, California 94158
Children's Hospital Colorado - Accepting Juvenile Patients
Aurora, Colorado 80046
Aurora, Colorado 80046
Mayo Clinic Florida - Accepting Adult Patients
Jacksonville, Florida 32224
Jacksonville, Florida 32224
Johns Hopkins All Children's Hospital - Accepting Juvenile Patients
St. Petersburg, Florida 33701
St. Petersburg, Florida 33701
Children's Healthcare of Atlanta - Accepting Juvenile Patients
Atlanta, Georgia 30029
Atlanta, Georgia 30029
Emory University - Accepting Adult Patients
Atlanta, Georgia 30032
Atlanta, Georgia 30032
Ann & Robert H. Lurie Children's Hospital of Chicago - Accepting Young Adult and Juvenile Patients
Chicago, Illinois 60611
Chicago, Illinois 60611
Northwestern Memorial Hospital - Accepting Adult Patients
Chicago, Illinois 60611
Chicago, Illinois 60611
The University of Chicago Medical Center - Accepting Adult and Juvenile Patients
Chicago, Illinois 60637
Chicago, Illinois 60637
Contact:
Dr. Iazsmin Ventura, MD - Adult patient contact
773-702-6619
iventura@uchicago.edu; Dequana.jones@bsd.uchicago.edu
Dr. Iazsmin Ventura, MD - Adult patient contact
773-702-6619
iventura@uchicago.edu; Dequana.jones@bsd.uchicago.edu
University of Kansas Medical Center - Accepting Adult Patients
Kansas City, Kansas 66160
Kansas City, Kansas 66160
National Institutes of Health - Accepting Adult and Juvenile Patients
Bethesda, Maryland 20892
Bethesda, Maryland 20892
Boston Children's Hospital - Accepting Young Adults and Juvenile Patients
Boston, Massachusetts 02115
Boston, Massachusetts 02115
University of Michigan - Accepting Young Adult and Juvenile Patients
Ann Arbor, Michigan 48105
Ann Arbor, Michigan 48105
Mayo Clinic - Accepting Adult Patients
Rochester, Minnesota 55902
Rochester, Minnesota 55902
Hospital for Special Surgery - Accepting Adult and Juvenile Patients
New York, New York 10021
New York, New York 10021
Memorial Sloan Kettering Cancer Center - Accepting Adult and Juvenile Patients
New York, New York 10021
New York, New York 10021
Children's Hospital at Montefiore - Accepting Young Adult and Juvenile Patients
The Bronx, New York 10467
The Bronx, New York 10467
University of North Carolina at Chapel Hill - Accepting Adult Patients
Chapel Hill, North Carolina 27599
Chapel Hill, North Carolina 27599
Duke University Medical Center - Accepting Young Adults and Juvenile Patients
Durham, North Carolina 27710
Durham, North Carolina 27710
Oregon Health & Science University - Accepting Adult Patients
Portland, Oregon 97239
Portland, Oregon 97239
Children's Hospital of Philadelphia - Accepting Young Adult and Juvenile Patients
Philadelphia, Pennsylvania 19104
Philadelphia, Pennsylvania 19104
UPMC Arthritis and Autoimmunity Center - Accepting Adult Patients
Pittsburgh, Pennsylvania 15261
Pittsburgh, Pennsylvania 15261
Vanderbilt University Medical Center - Accepting Adult Patients
Nashville, Tennessee 37232
Nashville, Tennessee 37232
University of Texas Southwestern Medical Center - Accepting Juvenile Patients
Dallas, Texas 75390
Dallas, Texas 75390
Houston Methodist Hospital - Accepting Adult Patients
Houston, Texas 77030
Houston, Texas 77030
University of Texas MD Anderson Cancer Center - Accepting Adult Patients
Houston, Texas 77030
Houston, Texas 77030
Primary Children's Hospital/University of Utah - Accepting Juvenile Patients
Salt Lake City, Utah 84113
Salt Lake City, Utah 84113
Seattle Children's Research Institute - Accepting Juvenile Patients
Seattle, Washington 98105
Seattle, Washington 98105
More Details
- Status
- Recruiting
- Sponsor
- Cabaletta Bio
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.