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).
This study may enroll up to 80 subjects in total. CTX112 is a CD19 directed chimeric antigen receptor (CAR) T cell immunotherapy comprised of allogeneic T cells prepared for the treatment of refractory autoimmune diseases. The cells are from healthy adult volunteer donors that are genetically modified ex vivo using CRISPR-Cas9 (clustered regularly interspaced short palindromic repeats/ CRISPR-associated protein 9) gene editing components (single guide RNA and Cas9 nuclease).
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
80
CTX112 (CD19-directed T-cell immunotherapy comprised of allogeneic T cells genetically modified ex vivo using CRISPR-Cas9 gene editing components)
Research Site 4
Redwood City, California, United States
RECRUITINGResearch Site 2
Chicago, Illinois, United States
RECRUITINGResearch Site 8
Iowa City, Iowa, United States
RECRUITINGResearch Site 6
Boston, Massachusetts, United States
RECRUITINGResearch Site 1
St Louis, Missouri, United States
RECRUITINGResearch Site 5
Chapel Hill, North Carolina, United States
RECRUITINGResearch Site 7
Augsburg, Germany
NOT_YET_RECRUITINGResearch Site 3
Hanover, Germany
RECRUITINGTo evaluate the safety of CTX112 in adult subjects with refractory autoimmune diseases, including SLE, SSc or IIM
Incidence of dose-limiting toxicities
Time frame: From CTX112 infusion up to 28 days post-infusion
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
Time frame: From CTX112 infusion up to 60 months post-infusion
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
Time frame: From CTX112 infusion up to 60 months post-infusion
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)
Time frame: From CTX112 infusion up to 60 months post-infusion
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