Safety and Efficacy of BAFF-R CART for Refractory Neuroimmune Diseases

Sponsor
Tianjin Medical University General Hospital
Study ID
NCT07022197
Phase
PHASE1/PHASE2
Status
Recruiting
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Conditions

  • Chronic Inflammatory Demyelinating Polyradiculoneuropathy
  • Idiopathic Inflammatory Myopathies
  • Myasthenia Gravis
  • NMO Spectrum Disorder

Eligibility Criteria

Sex
ALL
Age
18 Years - 60 Years
Healthy Volunteers
Not accepted

Interventions

  • BAFF-R CART — DRUG
    Biological: BAFF-R CART cells Subjects will undergo leukapheresis to isolate peripheral blood mononuclear cells (PBMCs) to manufacture BAFF-R CART cells, during which cyclophosphamide will be administered for the purpose of lymphocytes depletion. After lymphodepletion, subjects will receive one dose treatment with BAFF-R CART cells by intravenous (IV) infusion. The initial dose of 0.5×10\^6 CART cells/kg will be infused on day 0. Drug: Cyclophosphamide and fludarabine Subjects will receive one 3-day cycle of lymphodepletion starting 4 days prior to BAFF-R CART cells infusion on Day 0. Subjects will be given IV infusion of cyclophosphamide 250 mg/m2/day on day -5, -4 and -3, and fludarabine 30 mg/m2 over 30 minutes administered immediately after cyclophosphamide.

Study Details

This study is a phase Ib/IIa dose-escalation study designed to evaluate the safety, tolerability, and preliminary efficacy of autologous T cells expressing chimeric antigen receptor (CAR)-targeted B-cell activating factor receptor (BAFFR) in refractory neuroimmune diseases. The study design is divided into two parts, the first of which will be given to each patient at 3 incremental dose levels to establish the maximum tolerated dose (MTD). Each disease is expected to enroll 12 patients who meet the inclusion criteria. In the second part, 15 patients per disease will be recruited to further characterize the efficacy of the MTD.

Key Dates

First listed
Jun 15, 2025
Start date
Apr 10, 2025
Status verified
Jun 2025
Primary completion
Dec 30, 2027
Completion
Dec 30, 2027

Study Design

Enrollment
27 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
BASIC_SCIENCE

Arms

  • Experimental: Participant Group BAFF-R CART cells
    Experimental: CART cells therapy，Dose level 1: 0.5 × 10\^6 CART cells/Kg The tolerability and safety of BAFF-R CART cells will be assessed in an initial dose of 0.5×10\^6 CART cells/Kg and three subjects will be enrolled firstly. Experimental: CART cells therapy，Dose level 2: 1 × 10\^6 CART cells/Kg If neither DLT nor efficacy is shown in the first three subjects, the dose of CART cells will be increased to 1 × 10\^6 CART cells/kg to assess DLT. Experimental: CART cells therapy，Dose level 3: 2 × 10\^6 CART cells/Kg If DLT occurs in two subjects, whether to test the safety and efficacy in 2 × 10\^6 CART cells/kg group will be determined by the investigator based on the initial data of efficacy, PK and PD.

Primary Outcome Measure

Types and incidence of dose-limiting toxicity (DLT) after BAFF-R CART cells infusion [ Time Frame: Up to 3 months post BAFF-R CART cells infusion ]

Central Contacts

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