CRISPR-Edited Allogeneic Anti-CD19 CAR-T Cell Therapy, in Patients with Refractory Systemic Lupus Erythematosus (GALLOP)

Phase 1

Not yet recruiting

• Conditions: Systemic Lupus Erythematosus; Lupus; Lupus Erythematosus; Lupus Nephritis
• Interventions: Drug: CB-010

• Registration Number: NCT06752876
• Lead Sponsor: Caribou Biosciences, Inc.

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.

Study Timeline

• Status Verified: 2024-12
• Study First Submitted: 2024-12-18
• Study First Submitted QC: 2024-12-20
• Last Update Submitted: 2024-12-20
• Study First Posted: 2024-12-31
• Last Update Posted: 2024-12-31
• Study Start: 2025-01-01
• Primary Completion: 2029-04-04
• Study Completion: 2029-04-04

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 20

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

• Up to date on recommended vaccinations

• 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

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Cohort 1 Lupus Nephritis (LN)	CB-010	-
Cohort 2 Extrarenal Lupus (ERL)	CB-010	-

Primary Outcome Measures

Name	Time	Method
Incidence of critical safety events (CSEs) ≤ 28days after CB-010 infusion	Through 28 days
Incidence of treatment emergent adverse events (TEAEs), serious adverse events (SAEs), and adverse events of special interest (AESIs)	Through end of study (approximately 2 years)

Secondary Outcome Measures

Name	Time	Method
To characterize the PK profile of a single infusion of CB-010 (i.e., CB-010 expansion and persistence)	Through end of study (approximately 2 years)	Concentration of CB-010 in blood samples over time