This is a single-arm, open-label, investigator-initiated trial (IIT) designed to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of RD06-05 in subjects with autoantibody-mediated autoimmune hematological diseases. The enrolled population consists of patients with active autoimmune hematological diseases, including primary immune thrombocytopenic purpura (ITP), autoimmune hemolytic anemia (AIHA), and Evans syndrome.
This study sets two dose groups: 6 × 10⁶ CAR⁺T cells/kg and 10 × 10⁶ CAR⁺T cells/kg, with the initial dose being 6 × 10⁶ CAR⁺T cells/kg. To reduce efficacy risks, the dose may be escalated to 10 × 10⁶ CAR⁺T cells/kg following evaluation and recommendation by the Safety Review Committee (SRC). The SRC's recommendation on dose escalation will be based on a comprehensive assessment of all available safety, pharmacokinetic (PK), pharmacodynamic (PD), and preliminary efficacy data.
Subjects voluntarily participate in this trial and sign the informed consent form.
Aged ≥ 18 years and ≤ 75 years, regardless of gender.
Liver function: Alanine Aminotransferase (ALT) and Aspartate Aminotransferase (AST) ≤ 3 × Upper Limit of Normal (ULN); Total Bilirubin (TBIL) ≤ 2 × ULN (except for Gilbert's Syndrome).
Renal function: Creatinine ≤ 1.5 × ULN or Creatinine Clearance Rate ≥ 40 ml/min.
Has a coexisting autoimmune disease that may seriously interfere with the attribution of study disease activity or pose additional safety risks. However, if the subject's condition has been clinically stable for ≥ 3 months, and it is expected not to interfere with study assessments, the subject may be enrolled after confirmation by the investigator and approval by the sponsor's medical monitor (or their designee).
Renal biopsy shows crescent formation in ≥ 50% of glomeruli.
Sustained doubling of serum creatinine level within 2 months before screening.
The investigator assesses that the subject has RPGN.
Union Hospital, Tongji Medical College, Huazhong University of Science and Technology
Lead sponsor
Nanjing Bioheng Biotech Co., Ltd.
Collaborator