Clinical trial NCT06752876: CRISPR-Edited Allogeneic Anti-CD19 CAR-T Cell Therapy, in Patients With Refractory Systemic Lupus Erythematosus (GALLOP)
Official title: A Phase 1, Multicenter, Open-Label Study of CB-010, a CRISPR-Edited Allogeneic Anti-CD19 CAR-T Cell Therapy, in Patients With Refractory Systemic Lupus Erythematosus (GALLOP)
Study type: INTERVENTIONAL
Overall status: NOT_YET_RECRUITING
Phase: PHASE1
Conditions: Systemic Lupus Erythematosus
Lupus
Lupus Erythematosus
Lupus Nephritis
Keywords: CB-010
Lupus nephritis
Lupus
Extrarenal lupus
Autoimmune disease
Anti-CD19 CAR-T therapy
Cell therapy
Lead sponsor: Caribou Biosciences, Inc.
Sponsor class: INDUSTRY
First posted: 2024-12-31
Enrollment: 20
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This is a Phase 1 study to evaluate the safety and efficacy of a single infusion of CB-010 in patients with refractory Systemic Lupus Erythematosus (SLE) with cohorts for lupus nephritis (LN) and extrarenal lupus (ERL).

Detailed description: Participants enrolled can expect to be on the study for a total duration of approximately 2 years, during which there will be a screening period followed by a single administration of CB-010 and then 24 months of safety follow-up and monitoring.

Eligibility criteria: Inclusion Criteria:

* Clinical diagnosis of SLE according to 2019 EULAR/ACR classification criteria for at least 6 months
* Cohort 1 LN:

  1. Class III or IV lupus nephritis
  2. Urine protein-to-creatinine ratio (UPCR) ≥ 0.8 mg/mg
  3. Refractory to glucocorticoids and at least 2 immunosuppressive therapies
* Cohort 2 ERL (Patients with class I and II LN may be included in the ERL cohort if their SLEDAI-2K is ≥ 8):

  1. SLEDAI-2K ≥ 8
  2. Refractory to glucocorticoids, and at least 2 immunosuppressive therapies
* Adequate renal, hepatic, pulmonary, and cardiac function, with specific laboratory criteria
* Females must be either of nonchildbearing potential, defined as postmenopausal or surgically sterile or agree to use a highly effective double barrier method of contraception or vasectomized partner

Exclusion Criteria:

* Has active severe central nervous system (CNS) lupus in the previous 3 months from planned LD start date
* Has received any other investigational treatment for any indication within the 4 weeks or 5 half-lives
* Prior treatment with cellular therapy (genetically modified cells), gene therapy directed at any target, allogenic or autologous stem cell transplant or organ transplant
* History of infection with human immunodeficiency virus or evidence of hepatitis B or C virus infection
* History of hypersensitivity to Cyclophosphamide, Fludarabine, or any of the components of CB-010
* Received a live vaccine ≤ 6 weeks prior to start of LD
* Patients for whom magnetic resonance imaging (MRI) studies are contraindicated or who cannot tolerate them

Primary outcomes:
measure: Incidence of critical safety events (CSEs) ≤ 28days after CB-010 infusion
timeFrame: Through 28 days
measure: Incidence of treatment emergent adverse events (TEAEs), serious adverse events (SAEs), and adverse events of special interest (AESIs)
timeFrame: Through end of study (approximately 2 years)
Secondary outcomes:
measure: To characterize the PK profile of a single infusion of CB-010 (i.e., CB-010 expansion and persistence)
timeFrame: Through end of study (approximately 2 years)
Other outcomes:
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Study officials:
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