Chronic Inflammatory Demyelinating PolyradiculoneuropathyPhase 1/2NCT07022197
Safety and Efficacy of BAFF-R CART for Refractory Neuroimmune Diseases
A Phase 1/2 study testing BAFF-R CART in people with Chronic Inflammatory Demyelinating Polyradiculoneuropathy and NMO Spectrum Disorder, run by Tianjin Medical University General Hospital at 1 site in China.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Chronic Inflammatory Demyelinating Polyradiculoneuropathy, NMO Spectrum Disorder, Myasthenia Gravis
- What is being tested?
- BAFF-R CART
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 27 participants
- Where
- 1 siteChina
- Study dates
- 10 Apr 2025to 30 Dec 2027
- Phase
- Phase 1/2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 1. Assessed by the investigator as having a refractory neuroimmune disease;
- Refractory neuroimmune diseases were defined as:
- Poor symptom control on at least three immunosuppressive agents for more than one year;
- Clinical evidence of at least two relapses within 12 months or three relapses within 24 months and one relapse within 12 months prior to screening.
- 2. Male study participants must agree to use contraception during the treatment period for 1 year after receiving study treatment, and sperm donation is prohibited throughout the study period;
- 3. In the case of females with childbearing potential, need to agree to use contraception during the treatment period and for at least 1 year after receiving study treatment. Participants must have a negative serum pregnancy test result at screening and a confirmed negative urine pregnancy test result prior to first CART treatment.
Who cannot join
- Any medical or psychiatric condition that, in the opinion of the investigator, may jeopardize the study participant or affect the study participant's ability to participate in this study;
- A history of drug or alcohol abuse within the 12 months prior to baseline, or any condition that in the opinion of the investigator is associated with poor adherence;
- Women who are breastfeeding or pregnant, or who plan to become pregnant at any time during the 12-month time period following treatment with CART, or a history of spontaneous or induced abortion within 4 weeks prior to screening;
- Study participants with a clinically relevant active infection (e.g., sepsis, pneumonia, or abscess) or serious infection (resulting in hospitalization or requiring antibiotic therapy) within 4 weeks prior to baseline;
- The study participant has received a live attenuated vaccination within 8 weeks prior to baseline; or is scheduled to receive a live vaccination (including COVID-19 vaccine) within 8 weeks after treatment;
- Study participants who have received prior treatment with rituximab within 6 months prior to baseline;
Show all 16Show fewer
- Study participants had received tolizumab, eculizumab within 3 months prior to baseline;
- Study participants who have received intravenous human immunoglobulin, plasma exchange, undergone immunotherapy within 4 weeks prior to baseline;
- Known concomitant serious underlying diseases, such as hepatic and renal impairment, hematologic disorders, previous severe cardiovascular disease, severe hypertension, diabetes mellitus, poor control of blood pressure and blood glucose;
- Comorbid mental illness, suicidal ideation (affirmative answer (yes) to question 4 or question 5 of the Colombian Suicide Severity Rating Scale (C-SSRS) indicating a suicide attempt within the last 6 months);
- Any of the following laboratory abnormalities during the screening period (a repeat measurement may be taken during the screening period prior to randomization to confirm results); (1) Elevated liver enzymes: aspartate aminotransferase (AST) or alanine aminotransferase (ALT) > 3 times the upper limit of normal (ULN); (2) Total bilirubin > 1.5 times the ULN; (3) Estimated glomerular filtration rate (eGFR) <45 mL/min/1.73 m2; (4) CD19 + B cell count <40 cells/µL;
- Presence of a history of tuberculosis infection, high risk of acquired tuberculosis infection;
- known immunodeficiency diseases, including human immunodeficiency virus (HIV) infection;
- Viral hepatitis B surface antigen (HBsAg) positivity during the screening period;
- Receiving blood transfusion therapy 4 weeks prior to baseline or during the screening period;
- Any other condition that the investigator deems inappropriate for participation in the study.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Tianjin Medical University General Hospital
Tianjin, Tianjin Municipality, 300052
Qiang Liu, M.D.,Ph.D., +8615022439149, qliu@tmu.edu.cn
Qiang Liu, M.D.,Ph.D.
About this study
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.
Read the detailed description
- Phase I: Frequency, type, and severity of adverse events (according to the National Cancer Institute Common Terminology Criteria for Adverse Events CTCAE V5.0) occurring within 4 weeks after BAFF-R CART infusion;
- Phase II: Number of recurrences at 52 weeks after BAFF-R CART infusion;
- Characterization of BAFF-R CART cell expansion levels over time in subjects (peripheral blood, cerebrospinal fluid, bone marrow, etc.);
- Characteristics of BAFF-R+ B cell and antibody secreting cell (ASC) lymphocyte ablation in subjects;
- Changes in disease-related clinical scores at baseline, 24 and 52 weeks after BAFF-R CART infusion;
- Changes in the cumulative total number of MRI active lesions in MS and NMOSD patients at 24 and 52 weeks after BAFF-R CART infusion;
- Frequency, type, and severity of treatment-related laboratory index abnormalities (according to the National Cancer Institute Common Terminology Criteria for Adverse Events CTCAE V5.0);
- Characterization of changes in disease-associated antibody titers in the subject;
- Changes in vital signs (blood pressure, pulse rate, weight, ECG).
- Conditions
- Chronic Inflammatory Demyelinating Polyradiculoneuropathy, NMO Spectrum Disorder, Myasthenia Gravis, Idiopathic Inflammatory Myopathies
- Being tested
- BAFF-R CART
- Medical subject headings
- Polyradiculoneuropathy, Chronic Inflammatory Demyelinating, Neuromyelitis Optica, Myasthenia Gravis, Myositis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BAFF-R CARTDrug
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 group | Role | What they receive |
|---|---|---|
| Participant Group BAFF-R CART cells | Experimental | 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. Receives: Drug: BAFF-R CART |
What is being measured
The primary outcome is the main question the study is designed to answer.
Types and incidence of dose-limiting toxicity (DLT) after BAFF-R CART cells infusion
To evaluate the DLT occurred within 3 months after BAFF-R CART cells infusion.
Measured: Up to 3 months post BAFF-R CART cells infusion
Incidence and severity of AEs, including changes in vital signs, physical examination, laboratory parameters, Electrocardiograms and Echocardiograms.
To evaluate the AEs occurred within 2 years after BAFF-R CART cells infusion.
Measured: Up to 2 years post BAFF-R CART cells infusion
Secondary outcomes (5)
PD-Soluble BAFF
The changes of concentration of soluble BAFF in the peripheral blood after BAFF-R CART cells infusion.
Measured: Up to 2 years post BAFF-R CART cells infusion
PD-Pathogenic antibody
The changes of pathogenic antibody titers in peripheral blood or cerebrospinal fluid.
Measured: Up to 2 years post BAFF-R CART cells infusion
PD-Nfl（MS）
The changes of peripheral blood Neurofilament Light chain (NfL) concentration in patients with MS.
Measured: Up to 2 years post BAFF-R CART cells infusion
PK-VCN
The number of BAFF-R CAR gene copies (VCN, copies/μg DNA) in peripheral blood and cerebrospinal fluid after administration.
Measured: Up to 15 years post BAFF-R CART cells infusion
PK-BAFF-R CAR-T cells
The concentration of BAFF-R CAR T cells (cells/mL) in peripheral blood after administration was detected by flow cytometry.
Measured: Up to 2 years post BAFF-R CART cells infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Jun 2025
- Study start10 Apr 2025
- Primary completion30 Dec 2027 (estimated)
- Study completion30 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Chronic Inflammatory Demyelinating PolyradiculoneuropathyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Basic science
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 27 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Tianjin Medical University General Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Qiang Liu, Tianjin Medical University General Hospital
- Investigators
- Qiang Liu, M.D.,Ph.D, Principal investigator, Tianjin Medical University General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07022197
- Sponsor's protocol ID
- IRB2024-YX-274-01
- Status last verified
- Oct 2026
- First submitted
- 6 Jun 2025
- Last updated
- 16 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No