Summary

Eligibility
for people ages 18-70 (full criteria)
Location
at UCSD
Dates
study started
study ends around
Principal Investigator
by Brian Pedersen, MD (ucsd)
Headshot of Brian Pedersen
Brian Pedersen

Description

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).

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

Details

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).

Keywords

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, SSc, IIM, Systemic Lupus Erythematosus, Systemic Scleroderma, Diffuse Scleroderma, CTX112

Eligibility

You can join if…

Open to people ages 18-70

  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:

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 CAN'T join if...

  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.

Locations

  • UC San Diego Health not yet accepting patients
    La Jolla California 92037 United States
  • Stanford University Medical Center accepting new patients
    Redwood City California 94063 United States
  • University of Iowa accepting new patients
    Iowa City Iowa 52242 United States

Lead Scientist at University of California Health

  • Brian Pedersen, MD (ucsd)
    Associate Clinical Professor, Medicine, Vc-health Sciences-schools. Authored (or co-authored) 11 research publications

Details

Status
accepting new patients at some sites,
but this study is not currently recruiting here
Start Date
Completion Date
(estimated)
Sponsor
CRISPR Therapeutics
ID
NCT06925542
Phase
Phase 1 research study
Study Type
Interventional
Participants
Expecting 80 study participants
Last Updated