UCAR-T Targeting CD19/BCMA in Subjects With Autoantibody-Mediated Autoimmune Benign Hematological Diseases

NCT07441525 | Recruiting Early Phase 1

• 1 other identifier: BHCT-RD06-05-BHD
• Study Type: interventional
• Target: 27
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Autoimmune Hemolytic Anemia; Primary Immune Thrombocytopenic Purpura; Evans Syndrome

Keywords

UCART; CD19/BCMA; ITP; AIHA; EVANS

Outcome Measures

Primary Outcomes (3)

• The incidence rates of Serious Adverse Events that occur during treatment

  From the day of cell infusion to months 24 after cell infusion

• The incidence rates of Treatment-Emergent Adverse Events that occur during treatment

  From the day of cell infusion to months 24 after infusion

• The incidence rates of Adverse Events of Special Interest that occur during treatment

  From the day of cell infusion to months 24 after cell infusion

Secondary Outcomes (15)

• ITP: The proportion of subjects who achieve a sustained platelet response

  From the day of cell infusion to months 24 after cell infusion

• ITP: Overall Response Rate (ORR)

  From the day of cell infusion to months 24 after cell infusion

• ITP: Complete Response Rate (CR)

  From the day of cell infusion to months 24 after cell infusion

• ITP: PartialResponse Rate (PR)

  From the day of cell infusion to months 24 after cell infusion

• ITP: Duration of Sustained Platelet Response

  From the day of cell infusion to months 24 after cell infusion

Study Arms (1)

CART Treatment Group

EXPERIMENTAL

All participants will recevie CAR+T cells infusion with dose groups of 6 × 10⁶ CAR+T cells/kg and 10 × 10⁶ CAR+T cells/kg.

Drug: CART Infusion

Interventions

CART Infusion DRUG

All participants will recevie CAR+T cells infusion with dose groups of 6 × 10⁶ CAR+T cells/kg and 10 × 10⁶ CAR+T cells/kg.

CART Treatment Group

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Subjects voluntarily participate in this trial and sign the informed consent form.
• Aged ≥ 18 years and ≤ 75 years, regardless of gender.
• Organ function and laboratory tests:
• 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.
• Oxygen saturation (SpO2) ≥ 92% in room air at rest.
• Echocardiography shows Left Ventricular Ejection Fraction (LVEF) ≥ 50%.
• Female subjects of childbearing potential must have a negative result in serum or urine pregnancy test at screening.
• Female subjects of childbearing potential must agree to use highly effective contraceptive methods from at least 28 days before the start of lymphodepletion to 12 months after reinfusion on RD06-05. Male subjects of childbearing potential must agree to use effective barrier contraceptive methods from the start of lymphodepletion to 12 months after reinfusion on RD06-05, and must not donate semen or sperm during the entire trial period.
• Subjects with primary Immune Thrombocytopenia (ITP), with a disease duration of at least \> 6 months.
• Refractory or relapsed ITP: Subjects show no response, unsustained response, or intolerance to at least 2 different categories of standard treatments (e.g., glucocorticoids, splenectomy, intravenous immunoglobulin (IVIg), thrombopoietin receptor agonists (TPO-RA), Bruton's tyrosine kinase (BTK) inhibitors, etc.); among which, subjects must have received at least one or more treatments from IVIg, TPO-RA, or BTK inhibitors. In addition, platelet counts must be \< 30,000/μL in two tests conducted within 15 days before the start of study treatment, with an interval of at least 7 days between the two tests.
• Complete blood count: Neutrophil count ≥ 1,000/µL, hemoglobin ≥ 60g/L.
• Subjects with Autoimmune Hemolytic Anemia (AIHA), including warm autoimmune hemolytic anemia (wAIHA), mixed autoimmune hemolytic anemia (mix-AIHA), and cold agglutinin disease (CAD), with a disease duration of at least \> 6 months.
• Refractory or relapsed AIHA: Subjects show no response, unsustained response, or intolerance to at least 3 lines of systemic treatments (e.g., glucocorticoids, rituximab, immunosuppressants, splenectomy, complement inhibitors, etc.).
• Laboratory evidence of hemolysis: At least one of the following conditions exists in either the screening period or any test within the past 3 months: haptoglobin below the lower limit of normal, or total bilirubin (especially indirect bilirubin) above the upper limit of normal, or lactate dehydrogenase (LDH) above the upper limit of normal, and/or elevated reticulocyte count.

You may not qualify if:

• 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).
• Has rapidly progressive glomerulonephritis (RPGN), defined as any of the following:
• 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.
• Subjects with the following cardiac diseases will be excluded:
• History of heart failure classified as New York Heart Association (NYHA) Class III or IV.
• History of myocardial infarction, cardiovascular angioplasty or stenting, unstable angina, or other serious cardiac diseases within 12 months before enrollment.
• Has a history of severe central nervous system (CNS) diseases that may affect the subject's ability to comply with the study protocol or interfere with the accuracy of study assessments, such as: traumatic brain injury, disturbance of consciousness, epilepsy, cerebral vascular ischemia, or cerebral vascular hemorrhage.
• Has a history of malignant tumors other than cured non-melanoma skin cancer or carcinoma in situ (e.g., carcinoma in situ of the cervix, bladder, or breast), unless the subject has been disease-free for at least 3 years.
• Primary immunodeficiency.
• Has uncontrolled infection; simple urinary tract infections and upper respiratory tract infections are permitted as judged by the investigator and the sponsor's medical monitor (or their designee).
• Has a known history of infection with human immunodeficiency virus (HIV), hepatitis C virus (HCV), or syphilis.
• Active or latent hepatitis B virus (HBV) infection.
• Positive results for Epstein-Barr virus (EBV) or cytomegalovirus (CMV) DNA or IgM antibodies during the screening period.

Sponsors & Collaborators

• Union Hospital, Tongji Medical College, Huazhong University of Science and Technology: lead
• Nanjing Bioheng Biotech Co., Ltd.: collaborator

Study Sites (1)

Union Hospital, Tongji Medical College, Huazhong University of Science & Technology

Wuhan, Hubei, China

RECRUITING

MeSH Terms

Conditions

Anemia, Hemolytic, Autoimmune; Evans Syndrome

Condition Hierarchy (Ancestors)

Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Autoimmune Diseases; Immune System Diseases

Central Study Contacts

Wei Xie, Attending Physician

CONTACT

027-8572600; sherryxw@163.com

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: All participanets will receive cell infusion.

Study Record Dates

• First Submitted: February 24, 2026
• First Posted: March 2, 2026
• Study Start: February 3, 2026
• Primary Completion (Estimated): January 10, 2029
• Study Completion (Estimated): June 30, 2029
• Last Updated: March 2, 2026

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)