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

NCT ID: NCT06925542

Last Updated: 2025-12-17

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1

Total Enrollment

80 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-03-10

Study Completion Date

2031-12-31

Brief Summary

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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).

Detailed Description

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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).

Conditions

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

Study Design

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Allocation Method

NA

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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CTX112

Administered by IV infusion following lymphodepleting chemotherapy

Group Type EXPERIMENTAL

CTX112

Intervention Type BIOLOGICAL

CTX112 (CD19-directed T-cell immunotherapy comprised of allogeneic T cells genetically modified ex vivo using CRISPR-Cas9 gene editing components)

Interventions

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CTX112

CTX112 (CD19-directed T-cell immunotherapy comprised of allogeneic T cells genetically modified ex vivo using CRISPR-Cas9 gene editing components)

Intervention Type BIOLOGICAL

Eligibility Criteria

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Inclusion 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).

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.

Exclusion Criteria

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.
Minimum Eligible Age

18 Years

Maximum Eligible Age

70 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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CRISPR Therapeutics

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Research Site 4

Redwood City, California, United States

Site Status RECRUITING

Research Site 2

Chicago, Illinois, United States

Site Status RECRUITING

Research Site 8

Iowa City, Iowa, United States

Site Status RECRUITING

Research Site 6

Boston, Massachusetts, United States

Site Status RECRUITING

Research Site 1

St Louis, Missouri, United States

Site Status RECRUITING

Research Site 5

Chapel Hill, North Carolina, United States

Site Status RECRUITING

Research Site 7

Augsburg, , Germany

Site Status NOT_YET_RECRUITING

Research Site 3

Hanover, , Germany

Site Status RECRUITING

Countries

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United States Germany

Central Contacts

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Clinical Trials

Role: CONTACT

1 877-214-4634

Other Identifiers

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CRSP-AID-500

Identifier Type: -

Identifier Source: org_study_id