RD06-05 Universal CD19/BCMA CAR-T for Refractory Pediatric Autoimmune Diseases

NCT07674147 | Not Yet Recruiting Early Phase 1 DMC

• 1 other identifier: BHCT-RD06-05-PAID
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 2

Brief Summary

This is a single-arm, open-label, phase I clinical study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of RD06-05, a universal CD19/BCMA dual-targeting chimeric antigen receptor T-cell (CAR-T), in pediatric and adolescent patients with refractory autoimmune diseases, including systemic lupus erythematosus/lupus nephritis (SLE/LN), systemic sclerosis (SSc), idiopathic inflammatory myopathy (IIM), multidrug-resistant nephrotic syndrome (MDR-NS), and refractory IgA nephropathy (IgAN). Approximately 30 eligible patients will be enrolled and receive a single intravenous infusion of RD06-05 at an initial dose of 6×10⁶ CAR+ T cells/kg, with a potential dose escalation to 10×10⁶ CAR+ T cells/kg following review by a Safety Review Committee (SRC).

Conditions

Autoimmune Diseases; SLE - Systemic Lupus Erythematosus; SSc-Systemic Sclerosis; IIM- Idiopathic Inflammatory Myopathies; IgAN - IgA Nephropathy; Multi-Drug Resistant Nephrotic Syndrome

Outcome Measures

Primary Outcomes (1)

• Incidence of Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Adverse Events of Special Interest (AESIs)

  Incidence of TEAEs, SAEs, and AESIs following RD06-05 infusion. AESIs include cytokine release syndrome (CRS) of grade ≥3, immune effector cell-associated neurotoxicity syndrome (ICANS) of any grade, graft-versus-host disease (GvHD) of any grade, infections of grade ≥3, secondary malignancies of any grade, and cardiac disorders of any grade.

  From signing of informed consent through 90 days post-infusion (for related AEs, up to 24 months post-infusion)

Secondary Outcomes (24)

• Proportion of SLE/LN Patients Achieving LLDAS and DORIS Remission

  2 years

• Renal Response in SLE/LN Patients with Renal Involvement

  2 years

• Change in SLE Disease Activity Scores

  2 years

• Change in SLE Disease Activity Scores

  2years

• Change in SLE Disease Activity Scores

  2years

Other Outcomes (2)

• Incidence of B-Cell Aplasia

  2 years

• Changes in B-Cell Subsets

  2 years

Study Arms (1)

RD06-05 Universal CAR-T Therapy

EXPERIMENTAL

Single-arm, open-label: participants receive a single intravenous infusion of RD06-05 (universal CD19/BCMA dual-targeting CAR-T cells) following a lymphodepleting regimen of fludarabine and cyclophosphamide.

Drug: RD06-05 CART Cell Infusion

Interventions

RD06-05 CART Cell Infusion DRUG

CAR T-cell therapy administered intravenously after a lymphodepleting therapy regimen consisting of fludarabine and cyclophosphamide.

RD06-05 Universal CAR-T Therapy

Eligibility Criteria

• Age: 5 Years - 20 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Voluntary participation with signed informed consent from patient or legal guardian.
• Age \>=5 to \<20 years, male or female.
• Important organ function meeting the following requirements (excluding abnormalities related to autoimmune disease activity): a) Bone marrow: ANC \>=1.0x10\^9/L, hemoglobin \>=60 g/L, platelets \>=30x10\^9/L; b) Liver: ALT \<=3xULN (except IIM-related elevation), AST \<=3xULN, total bilirubin \<=2xULN (\<=3xULN for Gilbert syndrome); c) Kidney: eGFR \>=30 mL/min/1.73m\^2 (lower eGFR or on renal replacement may be allowed if benefit \> risk by investigator judgment); d) Cardiac: LVEF \>=55% by echocardiogram; e) Pulmonary: No severe lung disease, SpO2 \>=92%.
• Negative serum or urine pregnancy test for females of childbearing potential at screening.
• Females of childbearing potential must use highly effective contraception from at least 28 days before lymphodepletion through 12 months post-infusion. Males must use effective barrier contraception and not donate sperm from start of lymphodepletion through 12 months post-infusion.
• Diagnosis of SLE by 2019 EULAR/ACR or 2012 SLICC criteria.
• If renal involvement: kidney biopsy within 2 years showing active nephritis (class III, IV, V, or combination). Renal involvement defined as proteinuria \>0.15g/24h, or hematuria, or eGFR \<90.Inadequate response to standard therapy: high-dose glucocorticoid (\>=1 mg/kg/d prednisone equivalent) + hydroxychloroquine + at least 2 DMARDs for 3 months, or intolerance, or unable to taper steroid to \<=5 mg/day at 6 months.
• Positive ANA, anti-dsDNA, or anti-Smith antibody.
• SLEDAI-2K \>=8 and clinical SLEDAI-2K \>=4 (renal proteinuria \>0.5g/24h or UPCR \>500 mg/g or active urinary sediment may waive the clinical SLEDAI-2K requirement).
• Physician Global Assessment (PGA) \>=1.0 (0-3 VAS).
• Diagnosis of SSc by 2013 ACR/EULAR criteria.
• Diffuse cutaneous SSc.
• Evidence of active disease (e.g., new SSc within 2 years, new skin involvement or worsening mRSS within 6 months, tendon friction rubs, lung function decline, ILD progression).
• FVC \>=50% and DLCO \>=45% predicted.
• Failed or relapsed on conventional therapy (glucocorticoid \>0.5 mg/kg/d prednisone equivalent + at least two immunomodulators for \>6 months).

You may not qualify if:

• Co-existing autoimmune disease that may interfere with disease activity attribution or add safety risk (unless stable \>=3 months and approved).
• Prior B-cell/ASC depletion therapy: a) Anti-CD20 or T-cell engager within 3 months (allowed if \>3-6 months and CD19+ B-cells \> LLN); b) Prior CD19 and BCMA dual-targeted therapy, or CD19 or BCMA targeted therapy within 6 months (allowed if \>6 months and B-cells \> LLN); c) Other B-cell/ASC targeted therapies require approval.
• Rapidly progressive glomerulonephritis (RPGN): \>=50% crescents on biopsy, or doubling of serum creatinine within 2 months, or investigator judgment.
• Cardiac disease: NYHA class III/IV heart failure, MI, angioplasty/stent, unstable angina, or other severe cardiac disease within 12 months.
• Severe CNS disease (traumatic brain injury, impaired consciousness, epilepsy, cerebrovascular ischemia/hemorrhage) that may affect compliance or assessment.
• Malignancy history except cured non-melanoma skin cancer or carcinoma in situ, unless disease-free for \>=3 years.
• Primary immunodeficiency.
• Uncontrolled infection (simple UTI or upper respiratory infection allowed).
• Known history of HIV, hepatitis C, or syphilis infection.
• Active or latent hepatitis B infection.
• Positive EBV or CMV DNA or IgM at screening.
• History of recurrent tuberculosis.
• Prior CAR-T or other transgenic immune cell therapy.
• Live attenuated vaccine within 4 weeks before enrollment.
• Allergy to any component of the cell therapy product.

Sponsors & Collaborators

• The Children's Hospital of Zhejiang University School of Medicine: lead

Study Sites (2)

Nanjing Children's Hospital

Nanjing, Jiangsu, 210000, China

Location

Children's Hospital, Zhejiang University School of Medicine

Hangzhou, Zhejiang, 310052, China

Location

MeSH Terms

Conditions

Autoimmune Diseases; Lupus Erythematosus, Systemic; Glomerulonephritis, IGA; Nephrotic Syndrome

Condition Hierarchy (Ancestors)

Immune System Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Glomerulonephritis; Nephritis; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Nephrosis

Study Officials

• Jianhua Mao

  The Children's Hospital of Zhejiang University School of Medicine

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Jianhua Mao

CONTACT

+86 13516819071; maojh88@zju.edu.cn

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: June 5, 2026
• First Posted: June 29, 2026
• Study Start: July 1, 2026
• Primary Completion (Estimated): July 1, 2029
• Study Completion (Estimated): July 1, 2030
• Last Updated: June 29, 2026

Record last verified: 2026-06

Data Sharing

• IPD Sharing: Will not share

Locations

CN (2)