Actively Recruiting
Safety and Efficacy of BAFF-R CART Cell Therapy for Refractory Neuroimmune Diseases Evaluating Dose Escalation and Preliminary Outcomes
Led by Tianjin Medical University General Hospital · Updated on 2025-06-15
27
Participants Needed
1
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the safety, tolerability, and preliminary effectiveness of autologous T cells engineered to express a chimeric antigen receptor targeting the B-cell activating factor receptor (BAFFR) in patients with refractory neuroimmune diseases. This phase Ib/IIa dose-escalation study aims to find the maximum tolerated dose (MTD) of BAFF-R CART cells and then further assess efficacy at that dose, enrolling 12 patients per disease for dose escalation and 15 patients per disease for efficacy evaluation. Participants will receive BAFF-R CART cell therapy in escalating dose levels: 0.5, 1, and 2 million CART cells per kilogram. Prior to the infusion of CART cells, patients undergo lymphodepletion with cyclophosphamide and fludarabine given intravenously over three days. The CART cells are infused intravenously on day 0 following the lymphodepletion. Dose escalation proceeds cautiously with close monitoring to determine the dose-limiting toxicity and maximum tolerated dose. Throughout the study, participants will be closely monitored for adverse events, vital signs, physical exams, lab tests, ECGs, and echocardiograms for up to two years after infusion. Researchers will assess disease-related clinical scores, MRI lesion changes, immune cell levels, and antibody changes at various points up to one year post-infusion. Long-term follow-up includes evaluation of CART cell levels and pharmacodynamics for up to 15 years. The total participation duration varies, with intensive early monitoring and extended long-term safety and efficacy assessments.
CONDITIONS
Brief Title
Safety and Efficacy of BAFF-R CART for Refractory Neuroimmune Diseases
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Adults aged 18 to 60 years
- Diagnosed with refractory neuroimmune disease, defined as poor symptom control on at least three immunosuppressive agents for over one year
- Clinical evidence of at least two relapses within 12 months or three relapses within 24 months with one relapse within 12 months prior to screening
- Male participants agree to use contraception during treatment and for 1 year after; sperm donation prohibited during study
- Female participants with childbearing potential agree to use contraception during treatment and for at least 1 year after; negative pregnancy tests required before treatment
You will not qualify if you...
- Any medical or psychiatric condition that may jeopardize safety or affect ability to participate
- History of drug or alcohol abuse within 12 months prior to baseline or poor adherence risk
- Women who are pregnant, breastfeeding, or planning pregnancy within 12 months post-treatment, or recent abortion within 4 weeks prior to screening
- Active or serious infections requiring hospitalization or antibiotics within 4 weeks prior to baseline
- Live attenuated vaccination within 8 weeks prior to baseline or planned live vaccination within 8 weeks after treatment
- Prior rituximab treatment within 6 months prior to baseline
- Prior tolizumab or eculizumab treatment within 3 months prior to baseline
- Intravenous immunoglobulin, plasma exchange, or immunotherapy within 4 weeks prior to baseline
- Serious underlying diseases such as severe liver or kidney impairment, hematologic disorders, severe cardiovascular disease, poorly controlled hypertension or diabetes
- Comorbid mental illness with recent suicidal ideation
- Abnormal laboratory values including elevated liver enzymes, bilirubin, low kidney function, or low B cell counts
- History or high risk of tuberculosis infection
- Known immunodeficiency including HIV infection
- Positive viral hepatitis B surface antigen
- Blood transfusion within 4 weeks prior to baseline
- Any other condition deemed inappropriate by investigator for participation
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 3 days
Participants undergo lymphodepletion treatment with cyclophosphamide and fludarabine before receiving BAFF-R CART cell infusion.
3 visits for lymphodepletion infusions
Duration - Single day
Participants receive one dose of BAFF-R CART cells by intravenous infusion to treat refractory neuroimmune diseases.
1 infusion visit (in-person)
Duration - Up to 2 years
Participants are monitored for safety, treatment response, and biological markers up to 2 years after BAFF-R CART infusion.
Multiple visits for safety and efficacy assessments over 2 years
Trial Site Locations
Total: 1 location
1
Tianjin Medical University General Hospital
Tianjin, Tianjin Municipality, China, 300052
Actively Recruiting
Research Team
Q
Qiang Liu, M.D.,Ph.D
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
BASIC_SCIENCE
Number of Arms
1
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