This is a single-arm, open-label, multicenter, ascending dose Phase 1 study evaluating the safety and preliminary efficacy of CTX112 in adult subjects with refractory autoimmune diseases, including active systemic lupus erythematosus (SLE), systemic sclerosis (SSc), or idiopathic inflammatory myopathy (IIM).
Age ≥18 years and < 70 years of age.
Subjects must voluntarily sign a written informed consent and be willing and able to comply with all study requirements.
Adequate hematologic, renal, liver, cardiac and pulmonary organ function.
Subjects must agree to use acceptable methods of contraception.
Prior anti-CD19 therapy or any gene therapy/genetically modified cell therapy.
Prior solid organ (heart, liver, kidney, lung) transplant or hematopoietic cell transplant.
Severe active or history of central nervous (CNS) involvement.
History of a seizure disorder, cerebrovascular ischemia/hemorrhage, dementia, cerebellar disease or any autoimmune disease with CNS involvement other than SLE, SSc or IIM.
Sequential
CTX112
Biological/VaccineCTX112 (CD19-directed T-cell immunotherapy comprised of allogeneic T cells genetically modified ex vivo using CRISPR-Cas9 gene editing components)
To evaluate the safety of CTX112 in adult subjects with refractory autoimmune diseases, including SLE, SSc or IIM
Incidence of dose-limiting toxicities
To assess the pharmacodynamic response of CTX112 in adult subjects with refractory autoimmune diseases, including SLE, SSc or IIM
Change from baseline in disease specific autoantibody markers
To assess the pharmacokinetics (PK) of CTX112 in adult subjects with refractory autoimmune diseases, including SLE, SSc or IIM
Levels of CTX112 in blood over time
To assess the preliminary efficacy of CTX112 in adult subjects with refractory autoimmune diseases, including active SLE, SSc or IIM.
For SLE subjects: disease response rates based on SLEDAI-2K instrument; DORIS and LLDAS criteria For SSc subjects: disease response rates based on ACR-CRISS (including mRSS, forced vital capacity, HAQ-DI, Physician Global Assessment, Patient Global Assessment) For IIM subjects: disease response rates based on ACR/EULAR total Improvement score (including MMT8, EMDA, forced vital capacity, Physician Global Assessment, Patient Global Assessment, muscle enzyme level)
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