A Phase 1/2, Open-Label Study to Evaluate the Safety and Efficacy of Autologous CD19-specific Chimeric Antigen Receptor T Cells (CABA-201) in Subjects With Active Idiopathic Inflammatory Myopathy or Juvenile Idiopathic Inflammatory Myopathy
Cabaletta Bio
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
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 give…
Eligibility
- Age range
- 6–75 years
- Sex
- All
- Healthy volunteers
- No
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: * Contraindicatio…
Interventions
- BiologicalCABA-201 following preconditioning with fludarabine and cyclophosphamide
Single intravenous infusion of CABA-201 at a single dose level following preconditioning with fludarabine and cyclophosphamide
Locations (35)
- University of California Irvine - Accepting Adult PatientsOrange, California
- University of California, San Francisco Benioff Children's Hospital - Accepting Young Adult and Juvenile PatientsSan Francisco, California
- Children's Hospital Colorado - Accepting Juvenile PatientsAurora, Colorado
- Mayo Clinic Florida - Accepting Adult PatientsJacksonville, Florida
- Johns Hopkins All Children's Hospital - Accepting Juvenile PatientsSt. Petersburg, Florida
- Children's Healthcare of Atlanta - Accepting Juvenile PatientsAtlanta, Georgia