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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).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| CTX112 | Experimental | Administered by IV infusion following lymphodepleting chemotherapy |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| CTX112 | Biological | CTX112 (CD19-directed T-cell immunotherapy comprised of allogeneic T cells genetically modified ex vivo using CRISPR-Cas9 gene editing components) |
|
| Measure | Description | Time Frame |
|---|---|---|
| To evaluate the safety of CTX112 in adult subjects with refractory autoimmune diseases, including SLE, SSc or IIM | Incidence of dose-limiting toxicities | From CTX112 infusion up to 28 days post-infusion |
| Measure | Description | Time Frame |
|---|---|---|
| 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 | 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 |
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Key Inclusion Criteria:
For systemic lupus erythematosus (SLE) subjects:
- Diagnosis of SLE by a board-certified rheumatologist that conforms with 2019 ACR/EULAR criteria. For lupus nephritis subjects, active, biopsy-proven proliferative lupus nephritis Class III or IV, either with or without the presence of Class V, and appropriate National Institutes of Health index activity score using the 2018 International Society of Nephrology/Renal Pathology Society criteria.
For Systemic Sclerosis (SSc) subjects:
- Diagnosis of diffuse cutaneous systemic sclerosis (dcSSC) or SSc-ILD that conforms with 2013 ACR/EULAR criteria. Subjects should meet active skin or lung disease criteria.
For Idiopathic Inflammatory Myopathy (IIM) subjects:
- Diagnosis with dermatomyositis (DM), polymyositis (PM) or myositis as part of rheumatologic overlap syndrome, antisynthetase (ASyS), or immune-mediated necrotizing myopathy (IMNM) that conforms with 2017 ACR/EULAR criteria for inflammatory myopathies. Subjects must meet moderate severe, skin, or lung involvement criteria.
Key Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Clinical Trials | Contact | 1 877-214-4634 | medicalaffairs@crisprtx.com |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Research Site 4 | Recruiting | Redwood City | California | 94063 | United States | |
| Research Site 2 |
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Levels of CTX112 in blood over time |
| 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) | From CTX112 infusion up to 60 months post-infusion |
| Recruiting |
| Chicago |
| Illinois |
| 63110 |
| United States |
| Research Site 8 | Recruiting | Iowa City | Iowa | 52242 | United States |
| Research Site 14 | Recruiting | New Orleans | Louisiana | 70118 | United States |
| Research Site 6 | Recruiting | Boston | Massachusetts | 02118 | United States |
| Research Site 1 | Recruiting | St Louis | Missouri | 63130 | United States |
| Research Site 5 | Recruiting | Chapel Hill | North Carolina | 27599 | United States |
| Research Site 7 | Recruiting | Augsburg | 86156 | Germany |
| Research Site 13 | Recruiting | Cologne | 50937 | Germany |
| Research Site 3 | Recruiting | Hanover | 30625 | Germany |
| Research Site 12 | Recruiting | Mainz | 55131 | Germany |
| Research Site 10 | Recruiting | Córdoba | 14004 | Spain |
| Research Site 9 | Recruiting | Pamplona | 31008 | Spain |
| Research Site 11 | Recruiting | Seville | 41013 | Spain |
| ID | Term |
|---|---|
| D008180 | Lupus Erythematosus, Systemic |
| D008181 | Lupus Nephritis |
| D012595 | Scleroderma, Systemic |
| D009220 | Myositis |
| D045743 | Scleroderma, Diffuse |
| ID | Term |
|---|---|
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
| D005921 | Glomerulonephritis |
| D009393 | Nephritis |
| D007674 | Kidney Diseases |
| D014570 | Urologic Diseases |
| D052776 | Female Urogenital Diseases |
| D005261 | Female Urogenital Diseases and Pregnancy Complications |
| D000091642 | Urogenital Diseases |
| D052801 | Male Urogenital Diseases |
| D012871 | Skin Diseases |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
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