This is an open-label, investigator-initiated clinical trial (IIT) designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of RD06-04 in patients with refractory IIM and AAV. The study plans to enroll a total of 12 participants, with 6 cases each for IIM and AAV. Enrollment for both diseases will proceed in parallel. The dose will be 6×10\^6 CAR+T cells/kg (±30%), and patients will receive a single infusion of RD06-04.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
12
CAR T-cell therapy administered intravenously after a lymphodepleting therapy regimen consisting of fludarabine and cyclophosphamide.
Peking University People's Hospital
Beijing, China
Adverse Events (TEAE), Serious Adverse Events (SAE), Adverse Events of Special Interest (AESI)
Time frame: Up to 2 years
The proportion of patients with kidney involvement achieving complete kidney remission (CRR)
Time frame: At weeks 12, and months 6, 12, 18, and 24 following CAR-T infusion.
The change in UPCR from baseline in patients with kidney involvement.
Time frame: At weeks 2, 4, 8, 12, and at months 6, 9, 12, 18, and 24 following CAR-T infusion.
The change in eGFR (estimated glomerular filtration rate) from baseline in patients with kidney involvement.
Time frame: At weeks 2, 4, 8, 12, and at months 6, 9, 12, 18, and 24 following CAR-T infusion.
The change in BVAS score from baseline in AAV patients
Birmingham Vasculitis Activity Score (BVAS) has a minimum score of 0 and a theoretical maximum score exceeding 100 (depending on the number and severity of affected organ systems). A higher score indicates stronger disease activity and a worse prognosis, while a decrease in score (especially to \<5) typically suggests effective treatment.
Time frame: At 6, 12, 18, and 24 months following CAR-T infusion.
IIM patients were assessed for major clinical remission (Total Improvement Score, TIS) according to the 2016 ACR /EULAR criteria for myocarditis remission.
Total Improvement Score (TIS) has a minimum score of 0 (indicating no improvement) and a maximum score of 100 (indicating maximal improvement). A higher score represents more significant disease improvement and better treatment efficacy.
Time frame: At 6, 12, 18, and 24 months following CAR-T infusion.
The change in FACIT-Fatigue from baseline.
Functional Assessment of Chronic Illness Therapy-Fatigue Scale (FACIT-Fatigue) has a minimum score of 0 and a maximum score of 52. A higher score indicates less severe fatigue and better quality of life.
Time frame: At 6, 12, 18, and 24 months following CAR-T infusion.
Assessment of the peak amplification level (Cmax), area under the concentration-time curve (AUC0-28), and persistence profile of RD06-04.
Time frame: Up to 2 years
The change in HAQ-DI from baseline.
Health Assessment Questionnaire-Disability Index (HAQ-DI) has a minimum score of 0 and a maximum score of 3. A higher score indicates more severe physical dysfunction and worse quality of life.
Time frame: At 6, 12, 18, and 24 months following CAR-T infusion.
Assessment of the incidence and titer levels of anti-RD06-04 specific anti-drug antibodies (ADA).
Time frame: Up to 2 years
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