Idiopathic Inflammatory Myopathies(IIM), Systemic Lupus Erythematosus (SLE)
Conditions
Brief summary
This is a prospective, single-arm, open-label and dose-escalation investigator initialed study to evaluate LUCAR-G79D in adult subjects with r/r SLE and r/r IIM.
Detailed description
This is a prospective, single-arm, open-label clinical study to evaluate the safety, tolerability, pharmacokinetics and efficacy profiles of LUCAR-G79D, a chimeric antigen receptor (CAR) -T cell therapy in subjects with r/r SLE and r/r IIM. Patients who meet the eligibility criteria will receive LUCAR-G79D infusion. The study will include the following sequential stages: screening, pre-treatment (lymphodepleting chemotherapy), treatment (LUCAR-G79D infusion) and follow-up.
Interventions
BIOLOGICALLUCAR-G79D T cells
Prior to infusion of the LUCAR-G79D T cells, subjects will receive a conditioning premedication regimen consisting of cyclophosphamide and fludarabine.
Sponsors
Nanjing Legend Biotech Co.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 70 Years
Healthy volunteers
No
Inclusion criteria
1. Subjects voluntary agreement to provide written informed consent. 2. Aged 18 to 70 years, either sex. 3. Adequate organ function meet screening criteria. 4. Positive test for cluster of differentiation antigen 19 (CD19). SLE: * Have been diagnosed of SLE at least 6 months before screening. * At screening, antinuclear antibody, and/or anti-dsDNA antibody, and/or anti-Smith antibody should be positive. * Fulfill relapsed/refractory SLE conditions. IIM: * Have been diagnosed of IIM before screening. * Positive test for myositis-associated antibodies or myositis-specific antibodies at screening. * Fulfill relapsed/refractory IIM conditions.
Exclusion criteria
1. Active infections such as hepatitis and tuberculosis. 2. Other autoimmune diseases. 3. Serious underlying diseases such as tumor, uncontrolled diabetes. 4. Female subjects who were pregnant, breastfeeding. 5. Those with a history of major organ transplantation.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Maximum concentration (Cmax) in peripheral blood | 2 years after LUCAR-G79D infusion (Day 1) |
| Time of maximum concentration (Tmax) in peripheral blood | 2 years after LUCAR-G79D infusion (Day 1) |
| Incidence, severity, and type of treatment-emergent adverse events (TEAEs) | 2 years after LUCAR-G79D infusion (Day 1) |
| Incidence of dose-limiting toxicity (DLT) | 2 years after LUCAR-G79D infusion (Day 1) |
| Area under the concentration-time curve (AUC) in peripheral blood | 2 years after LUCAR-G79D infusion (Day 1) |
| Recommended Dose regimen finding | 2 years after LUCAR-G79D infusion (Day 1) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K) scores from baseline up to 52 weeks for r/r SLE subjects | 52 weeks after LUCAR-G79D infusion (Day 1) | SLEDAI-2K is a scale ranging from 0 to 105 points, with higher scores indicating stronger disease activity |
| Change in manual muscle testing (MMT-8) scores from baseline up to 52 weeks for r/r IIM subjects | 52 weeks after LUCAR-G79D infusion (Day 1) | MMT-8 is a scale ranging from 0 to 80 points, with higher scores indicating greater preserved muscle strength |
| Changes from baseline in peripheral blood immunoglobulins from baseline up to 52 weeks for all subjects | 52 weeks after LUCAR-G79D infusion (Day 1) | Including IgG、IgM、IgA、IgE |
| Changes from baseline in anti-drug antibody from baseline up to 52 weeks for all subjects | 52 weeks after LUCAR-G79D infusion (Day 1) | — |
Countries
China
Outcome results
None listed