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Legend discontinues the development of the IMP.
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| Name | Class |
|---|---|
| Nanjing Legend Biotech Co. | INDUSTRY |
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This is a prospective, single-arm, open-label, dose-exploration and expansion clinical study of LCAR-AIO in adult subjects with relapsed/refractory neurological autoimmune diseases.
This is a prospective, single-arm, open-label exploratory clinical study to evaluate the safety, tolerability, pharmacokinetics and efficacy profiles of LCAR-AIO, a chimeric antigen receptor (CAR) -T cell therapy in subjects with relapsed/refractory neurological autoimmune diseases. Patients who meet the eligibility criteria will receive LCAR-AIO infusion. The study will include the following sequential stages: screening, apheresis, pre-treatment (cell product preparation: lymphodepleting chemotherapy), treatment (LCAR AIO infusion) and follow-up.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| LCAR-AIO T Cells | Experimental | Chimeric antigen receptor T cells (LCAR-AIO) Each subject will be given a single-dose LCAR-AIO cells infusion at each dose level |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| LCAR-AIO T cells | Biological | Before treatment with LCAR-AIO T cells, subjects will receive a conditioning regimen. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Incidence, severity, and type of treatment-emergent adverse events (TEAEs) | An adverse event refers to any untoward medical occurrence in a clinical investigation subject administered a pharmaceutical product (investigational or non-investigational), which does not necessarily have a causal relationship with the treatment. | Baseline to 104 Weeks after last subject infusion |
| Incidence of dose-limiting toxicity (DLT) | DLTs are severe adverse events refers to any untoward medical occurred in a subject participated in a clinical investigation, which have a causal relationship with the treatment and will limit the dose escalation. | 30 days after LCAR-AIO infusion (Day 1) |
| Chimeric Antigen Receptor T (CAR-T) Positive Cell Concentration | Venous blood samples will be collected for measurement of CAR-T positive cellular concentration. | Baseline to 104 Weeks after last subject infusion |
| Recommended Phase 2 Dose (RP2D) regimen finding | RP2D established through dose exploratory. | Baseline to 104 Weeks after last subject infusion |
| Transgene Levels of LCAR-AIO CAR-T Cells | Transgene Levels of LCAR-AIO CAR-T Cells using sensitive assay methods will be assessed | Baseline to 104 Weeks after last subject infusion |
| Measure | Description | Time Frame |
|---|---|---|
| Annua Relapse Rate (ARR) | ARR refers to the number of relapses divided by observed year after LCAR-AIO infusion. | Baseline to 104 Weeks after last subject infusion |
| Change in Expanded Disability Status Scale (EDSS) scores from baseline up to 104 weeks |
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Inclusion Criteria:
MS;
NMOSD/MOGAD:
MG:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| The Affiliated Hospital of Fujian Medical University | Fuzhou | China | ||||
| The First Affiliated Hospital of SOOCHOW University |
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The EDSS is a method of quantifying disability and monitoring changes in the level of disability over time. EDSS score ranges from 0 (normal neurological exam) to 10 (death from MS). A negative change from baseline indicates improvement. |
| Baseline to 104 Weeks after last subject infusion |
| Changes in Visual Acuity from baseline up to 104 weeks | The Visual Acuity is determined by Snellen chart and visual field. | Baseline to 104 Weeks after last subject infusion |
| Changes in Visual analogue scale (VAS) pain score from baseline up to 104 weeks | VAS pain score is used to evaluate pain. Line from 0 = no pain to10 = worst pain. | Baseline to 104 Weeks after last subject infusion |
| Changes in MSE proportion from baseline up to 104 weeks | The proportion of patients who achieve Minimal Symptom Expression (MSE, defined as reaching an MG-ADL=0~1 or QMGS=0~2) after LCAR-AIO infusion. | Baseline to 104 Weeks after last subject infusion |
| Changes in Quantitative Myasthenia Gravis Score (QMGS) from baseline up to 104 weeks | The QMGS is a 13-item scale used to quantify disease severity in MG. The scale measures ocular, bulbar, respiratory, and limb function, grading each finding, and ranges from 0 (no myasthenic findings) to 39 (maximal myasthenic deficits). | Baseline to 104 Weeks after last subject infusion |
| Changes in Myasthenia Gravis Activities of Daily Living (MG-ADL) score from baseline up to 104 weeks | The MG-ADL is an eight-question survey of symptom severity. The average level of daily activity function in the last 7 days was assessed and the total score was calculated. Each question response graded from 0 (normal) to 3 (most severe). Cumulative MG-ADL scores range from 0 to 24. | Baseline to 104 Weeks after last subject infusion |
| Suzhou |
| China |
| Union Hospital Tongji Medical College HUAZHONG University of Science and Technology | Wuhan | China |
| ID | Term |
|---|---|
| D009103 | Multiple Sclerosis |
| D009157 | Myasthenia Gravis |
| D012008 | Recurrence |
| ID | Term |
|---|---|
| D020278 | Demyelinating Autoimmune Diseases, CNS |
| D020274 | Autoimmune Diseases of the Nervous System |
| D009422 | Nervous System Diseases |
| D003711 | Demyelinating Diseases |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
| D020361 | Paraneoplastic Syndromes, Nervous System |
| D009423 | Nervous System Neoplasms |
| D009371 | Neoplasms by Site |
| D009369 | Neoplasms |
| D010257 | Paraneoplastic Syndromes |
| D019636 | Neurodegenerative Diseases |
| D020511 | Neuromuscular Junction Diseases |
| D009468 | Neuromuscular Diseases |
| D020969 | Disease Attributes |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
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