Evaluating CABA-201 for treating active idiopathic inflammatory myopathy

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• University of California Irvine - Accepting Adult Patients
  Orange, California, United States Recruiting
• University of California, San Francisco Benioff Children's Hospital - Accepting Young Adult and Juvenile Patients
  San Francisco, California, United States Recruiting
• Children's Hospital Colorado - Accepting Juvenile Patients
  Aurora, Colorado, United States Recruiting
• Mayo Clinic Florida - Accepting Adult Patients
  Jacksonville, Florida, United States Recruiting
• Johns Hopkins All Children's Hospital - Accepting Juvenile Patients
  St. Petersburg, Florida, United States Recruiting
• Children's Healthcare of Atlanta - Accepting Juvenile Patients
  Atlanta, Georgia, United States Recruiting

Study contacts

• Study coordinator: Cabaletta Bio
• Email: clinicaltrials@cabalettabio.com
• Phone: 267-759-3100

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.