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 Active Juvenile Idiopathic Inflammatory Myopathy
This is a Phase 1/2, open-label study designed to evaluate the safety and efficacy of CABA-201 in patients with active IIM or JIIM and to identify an appropriate dose for future studies.
Why this Research Matters
Subjects will receive a single dose of CABA-201 and be evaluated for dose limiting toxicities (DLTs) during a 28-day DLT-assessment period. Subjects will also be followed for 156 following treatment for efficacy and safety.
Who can Participate
Subjects 6 to 17 years of age at the time of enrollment with a clinical diagnosis of Active Juvenile Idiopathic Inflammatory Myopathy. 1. Able to provide informed consent and assent (if applicable). 2. Age 6 to 17 years at enrollment. 3. A definite or probable clinical diagnosis of IIM. 4. Positive for at least 1 serum MSA or MAA prior to or during Screening. 5. Evidence of active disease. 7. Inadequately managed JIIM to and/or intolerant to at least 3 standard immunomodulatory therapies during a period of up to 5 years prior to Screening, either in combination or as monotherapy. 8. If a subject is currently receiving: a. Standard immunomodulatory therapy other than IVIg, SCIg, plasma exchange, or rituximab, the therapy must have been initiated at least 12 weeks prior to Screening and on a stable dose for at least 8 weeks prior to Screening, except for dose reduction due to safety or tolerability. b. An oral corticosteroid, the subject must be receiving less than or equal to 0.5 mg/kg/day or less than or equal to 20 mg per day prednisone or equivalent (whichever is lower) and on a stable dose for at least 2 weeks prior to Screening. c. IVIg or SCIg, the therapy must have been initiated at least 12 weeks prior to Screening. 10. Have received the recommended vaccinations per national and/or local/institutional guidelines for patients undergoing CAR T therapy. Medical monitor must review and approve enrollment of subjects with partially completed vaccination series. 10. Clinically stable by vital signs assessment for age at Screening based upon the Investigator's clinical judgement. 11. Female subjects of reproductive potential who are sexually active must agree to use 2 acceptable methods of birth control. Sexually active male subjects are required to use condoms with spermicide.
Study ID
Protocol Number: 25-0039
More information available at ClinicalTrials.gov: NCT06154252
Meet the Team
Principal Investigator