Summary

Eligibility
for people ages 18-65 (full criteria)
Location
at UC Irvine
Dates
study started
completion around
Principal Investigator
by Tahseen Mozaffar, MD (uci)
Headshot of Tahseen Mozaffar
Tahseen Mozaffar

Description

Summary

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

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

Details

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.

Keywords

Idiopathic Inflammatory Myopathy, Dermatomyositis, Anti-Synthetase Syndrome, Immune-Mediated Necrotizing Myopathy, CABA-201, Autoimmune Disease, Anti-CD19 CAR-T therapy, Cellular Therapy, Myositis, Muscular Diseases, Cyclophosphamide, Fludarabine, CAB-201 following preconditioning with fludarabine and cyclophosphamide

Eligibility

You can join if…

Open to people ages 18-65

  • Age ≥18 and ≤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

You CAN'T join if...

  • 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

Locations

  • University of California Irvine accepting new patients
    Orange California 92868 United States
  • Oregon Health & Science University not yet accepting patients
    Portland Oregon 97239 United States
  • University of Kansas Medical Center accepting new patients
    Kansas City Kansas 66160 United States

Lead Scientist at University of California Health

  • Tahseen Mozaffar, MD (uci)
    Clinical Professor, Neurology, School of Medicine. Authored (or co-authored) 147 research publications

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Cabaletta Bio
ID
NCT06154252
Phase
Phase 1/2 research study
Study Type
Interventional
Participants
Expecting 18 study participants
Last Updated