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

December 16, 2025 updated by: CRISPR Therapeutics

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

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).

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

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

Enrollment (Estimated)

80

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Germany
      • Augsburg, Germany, 86156
        • Not yet recruiting
        • Research Site 7
      • Hanover, Germany, 30625
        • Recruiting
        • Research Site 3
  • United States
    • California
      • Redwood City, California, United States, 94063
        • Recruiting
        • Research Site 4
    • Illinois
      • Chicago, Illinois, United States, 63110
        • Recruiting
        • Research Site 2
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • Recruiting
        • Research Site 8
    • Massachusetts
      • Boston, Massachusetts, United States, 02118
        • Recruiting
        • Research Site 6
    • Missouri
      • St Louis, Missouri, United States, 63130
        • Recruiting
        • Research Site 1
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27599
        • Recruiting
        • Research Site 5

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.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Key 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.

Key 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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: CTX112
Administered by IV infusion following lymphodepleting chemotherapy
Biological: CTX112
CTX112 (CD19-directed T-cell immunotherapy comprised of allogeneic T cells genetically modified ex vivo using CRISPR-Cas9 gene editing components)

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
To evaluate the safety of CTX112 in adult subjects with refractory autoimmune diseases, including SLE, SSc or IIM
Time Frame: From CTX112 infusion up to 28 days post-infusion
Incidence of dose-limiting toxicities
From CTX112 infusion up to 28 days post-infusion

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
To assess the pharmacodynamic response of CTX112 in adult subjects with refractory autoimmune diseases, including SLE, SSc or IIM
Time Frame: From CTX112 infusion up to 60 months post-infusion
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
Time Frame: From CTX112 infusion up to 60 months post-infusion
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.
Time Frame: From CTX112 infusion up to 60 months post-infusion

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

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 Major Dates

Study Start (Actual)

March 10, 2025

Primary Completion (Estimated)

December 31, 2031

Study Completion (Estimated)

December 31, 2031

Study Registration Dates

First Submitted

March 25, 2025

First Submitted That Met QC Criteria

April 10, 2025

First Posted (Actual)

April 13, 2025

Study Record Updates

Last Update Posted (Actual)

December 17, 2025

Last Update Submitted That Met QC Criteria

December 16, 2025

Last Verified

December 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CRSP-AID-500

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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