Actively Recruiting
A Clinical Study Evaluating the Safety and Preliminary Efficacy of Universal Allogeneic CAR T-cell Therapy Targeting CD19 and BCMA in Patients with Autoimmune Hemolytic Anemia Who Have Failed 3 or More Lines of Therapy
Led by Institute of Hematology & Blood Diseases Hospital, China · Updated on 2024-12-13
15
Participants Needed
1
Research Sites
43 weeks
Total Duration
On this page
Sponsors
I
Institute of Hematology & Blood Diseases Hospital, China
Lead Sponsor
X
Xi'niao Biotech
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating the safety and preliminary effectiveness of universal allogeneic anti-CD19/BCMA CAR T-cells in adults with autoimmune hemolytic anemia (AIHA) who have not responded to three or more prior treatments. This Phase 1 clinical study focuses on patients who have undergone multiple therapies without success, aiming to understand the potential of this cellular therapy in difficult-to-treat AIHA cases. The treatment involves a single intravenous infusion of universal allogeneic CAR T-cells targeting CD19 and BCMA after patients receive lymphodepleting chemotherapy with fludarabine and cyclophosphamide. Participants will be monitored closely, with an interim analysis planned at 12 weeks following the CAR T-cell infusion to assess safety and response. Participants will undergo assessments to track side effects, clinical response, and overall safety up to 24 weeks after treatment. Key measures include the number and severity of dose-limiting toxicities within 28 days, adverse events up to 12 months, and clinical response of AIHA up to 24 weeks post-infusion. The study requires informed consent and cooperation with follow-up evaluations throughout the monitoring period.
CONDITIONS
Brief Title
CAR T-cell Therapy Targeting CD19 and BCMA in Patients with AIHA Who Have Failed ≥3 Lines of Therapy.
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 18 years or older
- Positive B cell CD19 or BCMA detected by flow cytometry in peripheral blood
- Diagnosed with AIHA, including warm antibody type, cold agglutinin disease, mixed type, or other types per Chinese Adult AIHA Diagnosis and Treatment Guidelines (2023 Edition)
- Recurrent or refractory AIHA with symptomatic anemia (hemoglobin <100g/L) after at least 3 failed lines of treatment including glucocorticoids, rituximab, and immunomodulatory drugs
- ECOG performance status of 2 or less
- Adequate bone marrow function: neutrophil count ≥ 1.0 × 10^9/L; platelets ≥ 30 × 10^9/L
- Adequate liver function: ALT ≤ 3× upper limit; AST ≤ 3× upper limit; total bilirubin ≤ 2.0× upper limit (≤ 3.0× for Gilbert syndrome)
- Adequate renal function: creatinine clearance rate ≥ 30 ml/min (Cockcroft/Gault formula)
- Use of medically approved contraception or abstinence during treatment and for 6 months after
- Negative pregnancy test for women of childbearing potential within 7 days before enrollment and not lactating
- Willingness to participate, provide informed consent, and comply with follow-up
You will not qualify if you...
- History of severe drug allergies or allergic tendencies
- Uncontrolled or treatment-required fungal, bacterial, viral, or other infections
- Central nervous system diseases caused by autoimmune or non-autoimmune causes (e.g., epilepsy, psychosis, brain syndrome, stroke, encephalitis, CNS vasculitis)
- Insufficient cardiac function
- Congenital immunoglobulin deficiencies
- Malignancy history within five years
- End-stage renal failure
- Positive for hepatitis B (HBsAg or HBcAb with HBV DNA above normal), hepatitis C, HIV, or syphilis infections
- Psychiatric disorders or severe cognitive impairments
- Use of immunosuppressive agents or biologics within five half-lives before enrollment
- Pregnant or planning to conceive
- Active infections, active rheumatic or immune diseases, drug-induced or lymphoproliferative tumor-associated secondary AIHA
- Other investigator-determined reasons making the participant unsuitable for the study
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 - Single infusion with monitoring for 12 weeks
Participants receive a single infusion of universal allogeneic anti-CD19/BCMA CAR T-cells after lymphodepleting therapy.
1 infusion visit and weekly visits for up to 12 weeks
Duration - Up to 24 weeks
Participants are monitored for safety and clinical response for up to 24 weeks after infusion.
Visits up to 24 weeks after infusion
Trial Site Locations
Total: 1 location
1
Regenerative Medicine Center and Red Blood Cell Disorders Center
Tianjin, Tianjin Municipality, China
Actively Recruiting
Research Team
J
Jun Shi, PhD
L
Lele Zhang, PhD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
Similar Trials
Frequently Asked Questions
Have more questions? Get in touch with our team for quick support
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here