Home › Trials › NCT06154252

Evaluating CABA-201 for treating active idiopathic inflammatory myopathy

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

Phase2; Phase3 Interventional Cabaletta Bio · NCT06154252

This study is testing a new cell therapy called CABA-201 to see if it can help people with active idiopathic inflammatory myopathy feel better by reducing muscle inflammation and weakness.

Quick facts

PhasePhase2; Phase3
Study typeInterventional
Enrollment74 (estimated)
Ages6 Years to 75 Years
SexAll
SponsorCabaletta Bio Industry-sponsored
Drugs / interventionsCAR T, cyclophosphamide, fludarabine
Locations35 sites (Orange, California and 34 other locations)
Trial IDNCT06154252 on ClinicalTrials.gov

What this trial studies

This study evaluates the safety and efficacy of CABA-201, an investigational cell therapy, in patients with active idiopathic inflammatory myopathy (IIM) or juvenile idiopathic inflammatory myopathy (JIIM). Participants will receive a single dose of CABA-201 following preconditioning with fludarabine and cyclophosphamide. The study aims to assess the treatment's effectiveness in reducing muscle inflammation and weakness associated with various subtypes of IIM. Patients will be monitored for safety and response to treatment through clinical assessments and laboratory tests.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18 to 75 with a clinical diagnosis of active idiopathic inflammatory myopathy, including dermatomyositis, anti-synthetase syndrome, or immune-mediated necrotizing myopathy.

Not a fit: Patients with contraindications to leukapheresis or severe uncontrolled comorbidities may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve muscle strength and reduce inflammation in patients with IIM.

How similar studies have performed: While the approach of using CAR-T therapy in autoimmune diseases is novel, similar studies have shown promise in other conditions, suggesting potential for success.

Eligibility criteria

Show full inclusion / exclusion 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, 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.

Where this trial is running

Orange, California and 34 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Idiopathic Inflammatory MyopathyDermatomyositisAnti-Synthetase SyndromeImmune-Mediated Necrotizing MyopathyJuvenile DermatomyositisJuvenile PolymyositisJuvenile Idiopathic Inflammatory MyopathyJuvenile Myositis
Last reviewed 2026-06-09 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.