RECRUITING

A Safety and Efficacy Study Evaluating CTX112 in Adult Subjects With Refractory Autoimmune Disease

Conditions

SLE (Systemic Lupus)Lupus Erythematosus, Systemic Lupus Nephritis Systemic Sclerosis Inflammatory Myopathy, Idiopathic Myositis Diffuse Cutaneous Systemic Sclerosis CAR T Lupus SLE Allogeneic CD19 Cell Therapy Scleroderma Idiopathic Inflammatory Myopathy Inflammatory Myopathy Diffused Cutaneous Systemic Sclerosis Gene Therapy Autoimmune

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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).

Official Title

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

Quick Facts

Study Start:2025-03-10

Study Completion:2031-12-31

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06925542

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years to 70 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion Criteria
1. Age ≥18 years and \< 70 years of age. 2. Subjects must voluntarily sign a written informed consent and be willing and able to comply with all study requirements. 3. Adequate hematologic, renal, liver, cardiac and pulmonary organ function. 4. Subjects must agree to use acceptable methods of contraception. 5. Willing and able to comply with scheduled visits, treatment plan, laboratory tests, contraceptive guidelines, and other study procedures. 6. Diagnosis of systemic lupus erythematosus (SLE), systemic sclerosis (SSc) or idiopathic inflammatory myopathy (IIM).	1. Prior anti-CD19 therapy or any gene therapy/genetically modified cell therapy. 2. Prior solid organ (heart, liver, kidney, lung) transplant or hematopoietic cell transplant. 3. Severe active or history of central nervous (CNS) involvement. 4. History of a seizure disorder, cerebrovascular ischemia/hemorrhage, dementia, cerebellar disease or any autoimmune disease with CNS involvement other than SLE, SSc or IIM. 5. Mixed connective tissue disease with no clear predominant disease. 6. Presence of study disease manifestations or other conditions that are likely to pose increase safety risks and/or confound disease assessments, or pose significant risk to those receiving CAR T cell therapy. 7. History of primary or secondary immunodeficiency. 8. Presence or history of certain bacterial, viral or fungal infection. 9. Malignancy in the last 5 years (with the exception of cancers deemed to be low likelihood for recurrence). 10. Diagnosis of a genetic disorder associated with bone marrow failure or myelodysplastic syndrome. 11. History or current diagnosis of catastrophic anti-phospholipid syndrome or anti phospholipid syndrome that requires ongoing anticoagulation. 12. Pregnant or lactating. 13. Presence or history of disease requiring treatment that is not compatible with the study protocol; presence or history of other conditions that are not compatible with the study protocol.

Inclusion Criteria

Exclusion Criteria

1. Age ≥18 years and \< 70 years of age.
2. Subjects must voluntarily sign a written informed consent and be willing and able to comply with all study requirements.
3. Adequate hematologic, renal, liver, cardiac and pulmonary organ function.
4. Subjects must agree to use acceptable methods of contraception.
5. Willing and able to comply with scheduled visits, treatment plan, laboratory tests, contraceptive guidelines, and other study procedures.
6. Diagnosis of systemic lupus erythematosus (SLE), systemic sclerosis (SSc) or idiopathic inflammatory myopathy (IIM).

1. Prior anti-CD19 therapy or any gene therapy/genetically modified cell therapy.
2. Prior solid organ (heart, liver, kidney, lung) transplant or hematopoietic cell transplant.
3. Severe active or history of central nervous (CNS) involvement.
4. History of a seizure disorder, cerebrovascular ischemia/hemorrhage, dementia, cerebellar disease or any autoimmune disease with CNS involvement other than SLE, SSc or IIM.
5. Mixed connective tissue disease with no clear predominant disease.
6. Presence of study disease manifestations or other conditions that are likely to pose increase safety risks and/or confound disease assessments, or pose significant risk to those receiving CAR T cell therapy.
7. History of primary or secondary immunodeficiency.
8. Presence or history of certain bacterial, viral or fungal infection.
9. Malignancy in the last 5 years (with the exception of cancers deemed to be low likelihood for recurrence).
10. Diagnosis of a genetic disorder associated with bone marrow failure or myelodysplastic syndrome.
11. History or current diagnosis of catastrophic anti-phospholipid syndrome or anti phospholipid syndrome that requires ongoing anticoagulation.
12. Pregnant or lactating.
13. Presence or history of disease requiring treatment that is not compatible with the study protocol; presence or history of other conditions that are not compatible with the study protocol.

Contacts and Locations

Study Contact

Clinical Trials

CONTACT

1 877-214-4634

medicalaffairs@crisprtx.com

Study Locations (Sites)

Research Site 4

Redwood City, California, 94063

United States

Research Site 2

Chicago, Illinois, 63110

United States

Research Site 6

Boston, Massachusetts, 02118

United States

Research Site 1

Saint Louis, Missouri, 63130

United States

Research Site 5

Chapel Hill, North Carolina, 27599

United States

Collaborators and Investigators

Sponsor: CRISPR Therapeutics

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2025-03-10

Study Completion Date2031-12-31

Study Record Updates

Study Start Date2025-03-10

Study Completion Date2031-12-31

Terms related to this study

Keywords Provided by Researchers

CAR T
Lupus
SLE
Lupus Nephritis
Allogeneic
CD19
Cell Therapy
Scleroderma
Myositis
Systemic sclerosis
Idiopathic Inflammatory Myopathy
Inflammatory Myopathy
Diffused Cutaneous Systemic Sclerosis
Gene Therapy
Autoimmune

Additional Relevant MeSH Terms

SLE (Systemic Lupus)
Lupus Erythematosus, Systemic
Lupus Nephritis
Systemic Sclerosis
Inflammatory Myopathy, Idiopathic
Myositis
Diffuse Cutaneous Systemic Sclerosis