NCT06925542

Brief Summary

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).

Trial Health

80
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
80

participants targeted

Target at P75+ for phase_1

Timeline
68mo left

Started Mar 2025

Longer than P75 for phase_1

Geographic Reach
2 countries

8 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress19%
Mar 2025Dec 2031

Study Start

First participant enrolled

March 10, 2025

Completed
15 days until next milestone

First Submitted

Initial submission to the registry

March 25, 2025

Completed
19 days until next milestone

First Posted

Study publicly available on registry

April 13, 2025

Completed
6.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 31, 2031

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 31, 2031

Last Updated

December 17, 2025

Status Verified

December 1, 2025

Enrollment Period

6.8 years

First QC Date

March 25, 2025

Last Update Submit

December 16, 2025

Conditions

SLE (Systemic Lupus)MyositisLupus Erythematosus, SystemicLupus NephritisSystemic SclerosisInflammatory Myopathy, IdiopathicDiffuse Cutaneous Systemic Sclerosis

Keywords

CAR TLupusSLELupus NephritisAllogeneicCD19Cell TherapySclerodermaMyositisSystemic sclerosisIdiopathic Inflammatory MyopathyInflammatory MyopathyDiffused Cutaneous Systemic SclerosisGene TherapyAutoimmune

Outcome Measures

Primary Outcomes (1)

  • 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

Secondary Outcomes (3)

  • To assess the pharmacodynamic response of CTX112 in adult subjects with refractory autoimmune diseases, including SLE, SSc or IIM

    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

    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.

    From CTX112 infusion up to 60 months post-infusion

Study Arms (1)

CTX112

EXPERIMENTAL

Administered by IV infusion following lymphodepleting chemotherapy

Biological: CTX112

Interventions

CTX112BIOLOGICAL

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

CTX112

Eligibility Criteria

Age18 Years - 70 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Age ≥18 years and \< 70 years of age.
  • Subjects must voluntarily sign a written informed consent and be willing and able to comply with all study requirements.
  • Adequate hematologic, renal, liver, cardiac and pulmonary organ function.
  • Subjects must agree to use acceptable methods of contraception.
  • Willing and able to comply with scheduled visits, treatment plan, laboratory tests, contraceptive guidelines, and other study procedures.
  • 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.

You may not qualify if:

  • Prior anti-CD19 therapy or any gene therapy/genetically modified cell therapy.
  • Prior solid organ (heart, liver, kidney, lung) transplant or hematopoietic cell transplant.
  • Severe active or history of central nervous (CNS) involvement.
  • History of a seizure disorder, cerebrovascular ischemia/hemorrhage, dementia, cerebellar disease or any autoimmune disease with CNS involvement other than SLE, SSc or IIM.
  • Mixed connective tissue disease with no clear predominant disease.
  • 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.
  • History of primary or secondary immunodeficiency.
  • Presence or history of certain bacterial, viral or fungal infection.
  • Malignancy in the last 5 years (with the exception of cancers deemed to be low likelihood for recurrence).
  • Diagnosis of a genetic disorder associated with bone marrow failure or myelodysplastic syndrome.
  • History or current diagnosis of catastrophic anti-phospholipid syndrome or anti phospholipid syndrome that requires ongoing anticoagulation.
  • Pregnant or lactating.
  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (8)

Research Site 4

Redwood City, California, 94063, United States

RECRUITING

Research Site 2

Chicago, Illinois, 63110, United States

RECRUITING

Research Site 8

Iowa City, Iowa, 52242, United States

RECRUITING

Research Site 6

Boston, Massachusetts, 02118, United States

RECRUITING

Research Site 1

St Louis, Missouri, 63130, United States

RECRUITING

Research Site 5

Chapel Hill, North Carolina, 27599, United States

RECRUITING

Research Site 7

Augsburg, 86156, Germany

NOT YET RECRUITING

Research Site 3

Hanover, 30625, Germany

RECRUITING

MeSH Terms

Conditions

Lupus Erythematosus, SystemicLupus NephritisScleroderma, SystemicMyositisScleroderma, Diffuse

Condition Hierarchy (Ancestors)

Connective Tissue DiseasesSkin and Connective Tissue DiseasesAutoimmune DiseasesImmune System DiseasesGlomerulonephritisNephritisKidney DiseasesUrologic DiseasesFemale Urogenital DiseasesFemale Urogenital Diseases and Pregnancy ComplicationsUrogenital DiseasesMale Urogenital DiseasesSkin DiseasesMuscular DiseasesMusculoskeletal DiseasesNeuromuscular DiseasesNervous System Diseases

Central Study Contacts

Clinical Trials

CONTACT

1 877-214-4634medicalaffairs@crisprtx.com

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SEQUENTIAL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 25, 2025

First Posted

April 13, 2025

Study Start

March 10, 2025

Primary Completion (Estimated)

December 31, 2031

Study Completion (Estimated)

December 31, 2031

Last Updated

December 17, 2025

Record last verified: 2025-12

Data Sharing

IPD Sharing
Will not share

Locations

US(6)DE(2)