Actively Recruiting
A Phase 1 Dose Evaluation Study of the Safety and Preliminary Efficacy of Anti-CD19 Allogeneic CRISPR-Cas9-Engineered T Cells (CTX112) in Adult Subjects With Refractory Autoimmune Disease
Led by CRISPR Therapeutics · Updated on 2025-12-17
80
Participants Needed
8
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and early effectiveness of CTX112, an investigational treatment for adults with difficult-to-treat autoimmune diseases such as systemic lupus erythematosus (SLE), systemic sclerosis (SSc), and idiopathic inflammatory myopathy (IIM). This Phase 1, open-label study involves multiple centers and focuses on patients whose conditions have not responded well to previous therapies. CTX112 is a special type of immunotherapy using genetically modified T cells designed to target CD19, a protein involved in these autoimmune diseases. CTX112 is made from healthy donor T cells that are modified outside the body using CRISPR-Cas9 gene editing technology. Participants will receive CTX112 through an intravenous (IV) infusion after undergoing chemotherapy to reduce their existing immune cells. The study will enroll up to 80 adults aged 18 to under 70 years and will follow them for up to 60 months after infusion to monitor responses to the treatment and its behavior in the body. During the study, participants will attend scheduled visits for medical evaluations, laboratory tests, and safety monitoring. The main focus is to assess the safety of CTX112 within 28 days after infusion, but the study will also track how the treatment affects the immune system and disease activity over several years. Researchers will collect data on pharmacodynamics, pharmacokinetics, and preliminary efficacy to better understand the potential of this new therapy for autoimmune diseases.
CONDITIONS
Brief Title
A Safety and Efficacy Study Evaluating CTX112 in Adult Subjects With Refractory Autoimmune Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 18 years or older and under 70 years
- Voluntarily sign informed consent and willing to follow all study requirements
- Adequate hematologic, kidney, liver, heart, and lung function
- Agree to use acceptable contraception methods
- Willing and able to comply with visits, treatment plan, lab tests, and other procedures
- Diagnosed with systemic lupus erythematosus (SLE), systemic sclerosis (SSc), or idiopathic inflammatory myopathy (IIM)
- For SLE: diagnosis per 2019 ACR/EULAR criteria; active biopsy-proven proliferative lupus nephritis Class III or IV with or without Class V
- For SSc: diagnosis of diffuse cutaneous systemic sclerosis or SSc-ILD per 2013 ACR/EULAR criteria with active skin or lung disease
- For IIM: diagnosis of dermatomyositis, polymyositis, or related myositis per 2017 ACR/EULAR criteria with moderate to severe skin or lung involvement
You will not qualify if you...
- Prior anti-CD19 therapy or any gene therapy/genetically modified cell therapy
- Prior solid organ transplant (heart, liver, kidney, lung) or hematopoietic cell transplant
- Severe active or history of central nervous system involvement
- History of seizure disorder, stroke, dementia, cerebellar disease, or autoimmune disease with CNS involvement other than SLE, SSc, or IIM
- Mixed connective tissue disease without clear predominant disease
- Conditions posing increased safety risks or confounding assessments for CAR T cell therapy
- History of primary or secondary immunodeficiency
- Presence or history of certain bacterial, viral, or fungal infections
- Malignancy within past 5 years except low recurrence risk cancers
- Genetic disorders linked to bone marrow failure or myelodysplastic syndrome
- History or current catastrophic or ongoing anticoagulated anti-phospholipid syndrome
- Pregnant or lactating
- Diseases or conditions requiring incompatible treatments or conflicting with study protocol
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - From CTX112 infusion up to 28 days post-infusion
Participants receive CTX112 by IV infusion following lymphodepleting chemotherapy as treatment for refractory autoimmune diseases.
1 infusion visit and multiple follow-up visits during 28 days post-infusion
Duration - Up to 60 months post-infusion
Participants are monitored for safety, pharmacodynamics, pharmacokinetics, and preliminary efficacy up to 60 months after CTX112 infusion.
Regular follow-up visits over 60 months
Trial Site Locations
Total: 8 locations
1
Research Site 4
Redwood City, California, United States, 94063
Actively Recruiting
2
Research Site 2
Chicago, Illinois, United States, 63110
Actively Recruiting
3
Research Site 8
Iowa City, Iowa, United States, 52242
Actively Recruiting
4
Research Site 6
Boston, Massachusetts, United States, 02118
Actively Recruiting
5
Research Site 1
St Louis, Missouri, United States, 63130
Actively Recruiting
6
Research Site 5
Chapel Hill, North Carolina, United States, 27599
Actively Recruiting
7
Research Site 7
Augsburg, Germany, 86156
Not Yet Recruiting
8
Research Site 3
Hanover, Germany, 30625
Actively Recruiting
Research Team
C
Clinical Trials
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
1
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