RD06-05 Universal CD19/BCMA CAR-T for Refractory Pediatric Autoimmune Diseases
China30 participantsStarted 2026-07
Plain-language 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).
Who can participate
Age range
5 Years – 20 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. 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.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Incidence of Treatment-Emergent Adverse Events (TEAEs), Serious Adverse Events (SAEs), and Adverse Events of Special Interest (AESIs)
Timeframe: From signing of informed consent through 90 days post-infusion (for related AEs, up to 24 months post-infusion)
Trial details
NCT IDNCT07674147
SponsorThe Children's Hospital of Zhejiang University School of Medicine
. 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.
Exclusion criteria
. 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).