Evaluate the Safety & Efficacy of CABA-201 in Subjects with Active Idiopathic Inflammatory Myopathy

UCI Specialty Area: Rheumatology

Principal Investigator: Tahseen Mozaffar

Official Title: 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

A Study On:

Idiopathic Inflammatory Myopathies

Myositis

Study Description

Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects with Active Idiopathic Inflammatory Myopathy

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), and immune-mediated necrotizing myopathy (IMNM), 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, and IMNM who have active disease. A single dose of CABA-201 in combination with cyclophosphamide (CY) and fludarabine (FLU) will be evaluated.

Eligibility

Age greater than or equal to 18 and less than or equal to 65

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

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

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University of California, Irvine - Study Information Sheet

UCI Center for Clinical Research Potential Participant Database

Lead Researcher
Maheswari Senthil, MD
UCI Center for Clinical Research

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