This is a single-arm, open-label, dose-escalation and dose-expansion clinical trial designed to evaluate the safety, efficacy, and cellular metabolism kinetics of GT801 in the treatment of moderate-to-severe refractory autoimmune diseases. Interim analysis will be conducted when 5 patients complete primary endpoint measurement.
1. The participant or their legal representative voluntarily signs a written informed consent form and is willing and able to comply with the study procedures.
2. Aged 18 to 65 years old (inclusive) at the time of signing the informed consent, regardless of gender.
a) Fulfill the 2019 European League Against Rheumatism/American College of Rheumatology (EULAR/ACR) classification criteria for SLE.
b) Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2000) score ≥ 6, with at least one British Isles Lupus Assessment Group Index (BILAG-2004) Grade A (severe manifestation) or two Grade B (moderate manifestation) organ scores, or both; or SLEDAI-2000 score ≥ 8.
1. Participants with SLE
a. Drug-induced systemic lupus erythematosus.
b. Participants with lupus crisis; or those with concurrent diseases requiring the use of protocol-prohibited drugs, who are deemed ineligible for enrollment by the investigator.
2. Participants with IIM
Single group
GT801 Injection
Biological/VaccineGT801 Injection
Proportion of participants experiencing dose limiting toxicity
The proportion of participants with dose-limiting toxicity (DLT) occurring within 28 days after infusion
Adverse Events (AEs) occurring after infusion and their proportions
Adverse Events (AEs) occurring after infusion and their proportions
Efficacy outcomes for Systemic Lupus Erythematosus (SLE)
SLE Response index 4(SRI-4) response: Min/Max Value: Not specified; a decrease in score indicates improvement, higher scores indicate worse outcome
Efficacy outcomes for Idiopathic Inflammatory Myopathies (IIM)
Total lmprovement Score (TlS): Min/Max Value: Not specified; an increase in score indicates improvement, higher scores indicate better outcome.
Efficacy outcomes for Systemic Sclerosis (SSc)
Combined Response Index in Systemic Sclerosis (CRISS): the score ranges from 0 to 1 point; a score ≥ 0.6 indicates treatment improvement, while a score \< 0.6 indicates no disease improvement.
Efficacy Outcomes for ANCA-Associated Vasculitis (AAV)
The proportion of subjects achieving complete remission and partial remission
Exploration of Peripheral Blood Lymphocyte Subsets and B Lymphocyte Subsets: Changes in T Cells and NK Cells
Determination of the counts of T cells and NK cells
Exploration of Peripheral Blood Lymphocyte Subsets and B Lymphocyte Subsets: Duration of B-Cell Depletion
Determination of B-cell counts and calculation of the duration of B-cell count reduction
Exploration and analysis of BCR repertoire diversity via BCR sequencing
Detection of BCR diversity on B cells via sequencing
Pathological changes of involved organs before and after treatment
Pathological examination of lesion tissue biopsies was performed to observe the changes in histopathological characteristics after treatment compared with those before treatment, such as fibrosis, vascular lesions, and inflammatory cell infiltration.
Click on the lead sponsor to view all of their trials.
Grit Biotechnology
Lead sponsor
Shanghai Changzheng Hospital
Collaborator
