SLE (Systemic Lupus)Phase 1NCT06925542
A Safety and Efficacy Study Evaluating CTX112 in Adult Subjects With Refractory Autoimmune Disease
A Phase 1 study testing CTX112 in people with SLE (Systemic Lupus) and Lupus Erythematosus, Systemic, run by CRISPR Therapeutics at 17 sites in 3 countries.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- SLE (Systemic Lupus), Lupus Erythematosus, Systemic, Lupus Nephritis
- What is being tested?
- CTX112
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 80 participants
- Where
- 17 sitesin 3 countries
- Study dates
- 10 Mar 2025to 31 Dec 2031
- Phase
- Phase 1
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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).
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- 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.
Who cannot join
- 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.
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- 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.
Trial locations and contacts
17 sites in 3 countries. Contact the study team or a site near you to ask about taking part.
17 sites in 3 countries
United States9 sites, 9 recruiting
- Recruiting
Boston Medical Center
Boston, Massachusetts, 02118
Hanni Menn-Josephy, MD, Hanni.Menn@bmc.org
Hanni Menn-Josephy, MD
- Recruiting
University of North Carolina TraCS Institute - CTRC
Chapel Hill, North Carolina, 27599
Saira Sheikh, MD, saira.sheikh@unchealth.unc.edu
Saira Sheikh, MD
- Recruiting
Northwestern Medicine, Robert H. Lurie Comprehensive Cancer Center
Chicago, Illinois, 63110
Kaitlin King, 312-695-0990, Autoimmunesct@nm.org
George Georges, MD
- Recruiting
University of Iowa
Iowa City, Iowa, 52242
Ashley Pieper, 319-356-0765, ashley-pieper@uiowa.edu
Hanna Zembrzuska, MD
- Recruiting
UC San Diego Health
La Jolla, California, 92037
Brian Pedersen, MD
- Recruiting
Tulane University Medical Center
New Orleans, Louisiana, 70118
Anita Dhanrajani, MD, adhanrajani@tulane.edu
Anita Dhanrajani, MD
- Not yet recruiting
Oregon Health Sciences University
Portland, Oregon, 97239
Richard Maziarz, MD
- Recruiting
Stanford University Medical Center
Redwood City, California, 94063
Sally Arai, MD, sarai1@stanford.edu
Sally Arai, MD
- Recruiting
Washington University School of Medicine
St Louis, Missouri, 63130
Emily Green, 314-273-0339, green.e@wustl.edu
Alfred Kim, MD
Germany4 sites, 4 recruiting
- Recruiting
University of Augsburg
Augsburg, 86156
Christoph Schmid
- Recruiting
Universitätsklinikum Köln
Cologne, 50937
Philip Kohler
- Recruiting
Medizinische Hochschule Hannover (MHH)
Hanover, 30625
CART@mh-hannover.de
Christian Schultze-Florey, MD
- Recruiting
UCT University Medical Center Mainz
Mainz, 55131
KMT-Studie@unimedizin-mainz.de
Eva Maria Wagner-Drouet, MD
Spain4 sites, 4 recruiting
- Recruiting
Hospital Universitario Reina Sofia
Córdoba, 14004
Maria Angeles Aguirre, angeles.aguirre.sspa@juntadeandalucia.es
Maria del Carmen Ábalos, mcarmen.abalos@imibic.org
- Recruiting
Clinica Universidad de Navarra - Pamplona
Pamplona, 31008
Javier Garro Flamarique, fgarrofl@unav.es
Ornilla Ornilla, MD
- Recruiting
Hospital Universitario Virgen del Rocio
Seville, 41013
Elena Alonso Blanco-Morales, MD
- Recruiting
Hospital Clinico Universitario de Valencia
Valencia
Maria Jose Forner, MD
About this study
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
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).
Read the 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).
- Conditions
- SLE (Systemic Lupus), Lupus Erythematosus, Systemic, Lupus Nephritis, Systemic Sclerosis, Inflammatory Myopathy, Idiopathic, Myositis
- Being tested
- CTX112
- Keywords
- 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, SSc, IIM
- Medical subject headings
- Lupus Erythematosus, Systemic, Lupus Nephritis, Scleroderma, Systemic, Myositis, Scleroderma, Diffuse
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CTX112Biological
CTX112 (CD19-directed T-cell immunotherapy comprised of allogeneic T cells genetically modified ex vivo using CRISPR-Cas9 gene editing components)
| Study group | Role | What they receive |
|---|---|---|
| CTX112 | Experimental | Administered by IV infusion following lymphodepleting chemotherapy Receives: Biological: CTX112 |
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate the safety of CTX112 in adult subjects with refractory autoimmune diseases, including SLE, SSc or IIM
Incidence of dose-limiting toxicities
Measured: 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
Change from baseline in disease specific autoantibody markers
Measured: 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
Levels of CTX112 in blood over time
Measured: 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.
For SLE subjects: disease response rates based on SLEDAI-2K instrument; DORIS and LLDAS criteria
For SSc subjects: disease response rates based on rCRISS (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)
Measured: From CTX112 infusion up to 60 months post-infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Apr 2025
- Study start10 Mar 2025
- Primary completion31 Dec 2031 (estimated)
- Study completion31 Dec 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for SLE (Systemic Lupus)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 80 participants (estimated)
Sponsor and investigators
- Lead sponsor
- CRISPR Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06925542
- Sponsor's protocol ID
- CRSP-AID-500
- Status last verified
- Oct 2026
- First submitted
- 25 Mar 2025
- Last updated
- 23 Oct 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No