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

Launched by CABALETTA BIO · Nov 30, 2023

Keywords

ClinConnect Summary

The RESET-Myositis trial is studying a new treatment called CABA-201 for people with active idiopathic inflammatory myopathy, a group of diseases that cause muscle weakness and inflammation. This study aims to find out if CABA-201 is safe and effective for adults aged 18 to 75 who have been diagnosed with conditions like dermatomyositis or immune-mediated necrotizing myopathy and are still experiencing symptoms despite other treatments. Participants will need to have specific signs of active disease, such as elevated muscle enzymes or muscle weakness.

If you join this trial, you will receive CABA-201 and be closely monitored by the research team to see how well the treatment works and if there are any side effects. Before enrolling, doctors will check that you meet certain criteria, like not having serious infections or certain health conditions that could make participation risky. This trial is currently recruiting participants, and it offers a chance to help researchers learn more about potential new therapies for these challenging conditions.

Gender

ALL

Eligibility criteria

• • 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, IMNM based on the presence of serum myositis-specific antibodies
• • 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
• 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
• • 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
• • Presence of serum myositis-specific antibodies or myositis-associated autoantibody
• • 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
• 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