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

Starting soon · Early Phase 1

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

In brief

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).

Key facts

Study ID

NCT07674147

Run by

The Children's Hospital of Zhejiang University School of Medicine

People needed

30

Starts

2026-07-01

Expected to finish

2030-07-01

Last updated by the study team

2026-06-29

Who can join

Age: 5 and older, up to 20. Sex: any. Healthy volunteers: not accepted.

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.
• Disease-Specific Inclusion Criteria for SLE/LN:
• 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).
• Disease-Specific Inclusion Criteria for SSc:
• 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).
• Disease-Specific Inclusion Criteria for IIM:
• Diagnosis of IIM (dermatomyositis, antisynthetase syndrome, IMNM) by 2017 ACR/EULAR criteria (probability >=55%).
• Active disease: at least 2 of 6 core set abnormalities (MMT-8<142, PhGA >=2 cm, PtGA >=2 cm, extra-muscular MDAAT >=2 cm, PedsQL >=60, CK >=1.5xULN).
• Positive myositis-specific autoantibody.
• Failed or relapsed on conventional therapy (glucocorticoid >1 mg/kg/d prednisone equivalent + at least 2 immunomodulators for >=6 months).
• Disease-Specific Inclusion Criteria for IgAN:
• Biopsy-confirmed IgA nephropathy.
• On ACEi/ARB for >=3 months, and at least one of: a) proteinuria >=500 mg/24h or UPCR >=0.5 mg/mg after >=3 months of steroid + at least one immunosuppressant/biologic; b) eGFR decline >50% within 3 months; c) 22.intolerance to conventional therapy with benefit > risk.

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.
• Hypersensitivity to tacrolimus or prior grade >=3 tacrolimus-related toxicity requiring hospitalization (exceptions may be approved).
• Participation in another clinical trial within 30 days before screening.
• Pregnancy, breastfeeding, or unwillingness to use effective contraception.
• Any other condition judged by investigator as unsuitable for study.
• Disease-Specific Exclusion Criteria for SLE:
• Active/unstable neuropsychiatric lupus (seizures, psychosis, organic brain syndrome, CVA, encephalitis, CNS vasculitis) within 90 days requiring intervention.
• Prior treatments: belimumab/telitacicept within 4 weeks; ianalumab within 8 weeks unless B-cells > LLN; >1 systemic NSAID within 14 days; inability to wash out NSAID before disease activity assessment; intra-articular/IM glucocorticoid within 6 weeks; immunosuppressant doses above specified limits; initiation or dose change of hydroxychloroquine within 8 weeks; ACEi/ARB/SGLT2 inhibitor dose change within 4 weeks.
• Disease flare requiring increased corticosteroids (>20 mg/day prednisone equivalent) or new immunosuppression during screening.
• Disease-Specific Exclusion Criteria for IIM:
• Severe rhabdomyolysis or CK >=20xULN.

Where it is running

• Nanjing Children's Hospital — Nanjing, Jiangsu, China
• Children's Hospital, Zhejiang University School of Medicine — Hangzhou, Zhejiang, China

Full record on ClinicalTrials.gov

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