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This is a Phase IIb study to evaluate the safety and preliminary efficacy of Descartes-08 CAR T-cells in patients with Generalized Myasthenia Gravis
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Phase 1b Dose-Escalation | Experimental | Generalized Myasthenia Gravis |
|
| Phase IIa Expansion | Experimental | Generalized Myasthenia Gravis |
|
| Phase IIb Randomized Control Trial | Placebo Comparator | Generalized Myasthenia Gravis |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Descartes-08 | Drug | Autogolous T-cells expressing a chimeric antigen receptor directed to BCMA |
|
| Measure | Description | Time Frame |
|---|---|---|
| Proportion of patients with MG Composite improvement of ≥5 points. | In Part 3, the primary objective is to compare the effect of Descartes-08 versus placebo, as measured by the change in MGC score from baseline to Week 12. In Part 1, up to three ascending doses of Descartes-08 will be administered to each of 3 to 6 patients; the patients will be staggered at least 21 days apart and will complete a safety review between each dose. In Part 2, patients will receive up to 6 doses in three different schedules depending on the arm they were enrolled into and will be monitored for up to 1 year to assess the safety and tolerability of a repeated dosing schedule. | Day 0 to Day 85 |
| Measure | Description | Time Frame |
|---|---|---|
| Comparison on Descartes-08 versus placebo on QMG and MG ADL | The proportion of patients achieving a ≥6-point change from Baseline at each posttreatment follow-up in the MG ADL score and QMG score (all parts), and the difference between this proportion in the treatment arm compared to placebo at Day 85. | Day 0 to Day 85 |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Profound Research | Carlsbad | California | 92011 | United States | ||
| University of California Irvine |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 41514039 | Derived | Fedak RR, Ruggerie RN, Shan Y, Curvino EJ, de Sousa JF, Daniel S, Ngo-Casi M, Kamboh H, Vu T, Durmus H, Mozaffar T, Howard JF Jr, English EP, Benson A, Duvernay MT, Singer MS, Kalayoglu MV, Brunn C, Bodansky A, Anderson MS, DeRisi JL, Garcia ST, Yu DJL, Zorn KC, Kurtoglu M, Miljkovic MD, Stewart CA, Jewell CM; MG-001 Study Team. BCMA-directed mRNA CAR-T cell therapy for myasthenia gravis: exploratory biomarker analysis of a placebo-controlled phase 2b trial. Nat Med. 2026 Mar;32(3):1118-1130. doi: 10.1038/s41591-025-04170-z. Epub 2026 Jan 9. | |
| 41514038 |
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| Comparison on Descartes-08 versus placebo on MG assessments in crossover patients |
The proportion of patients initially randomized to placebo who then cross over to Descartes-08 and achieve a ≥5-point reduction in MGC score from Crossover Day 1 to Crossover Day 85. |
| Day 0 to Day 85 |
| Change in titer of myasthenia specific autoantibody titers | Determine the change from baseline over a period of 24 weeks in the titer of myasthenia specific autoantibody titers, e.g. nicotinic acetylcholine receptor autoantibody (anti-nAChR) or muscle-specific tyrosine kinase autoantibody (anti- MusK or anti-Low-Density-Lipoprotein-Related-Protein4 (anti-LRP4) or anti-AChR cluster antibody and the 4 Immunoglobulin types (IgG, IgM, IgA, IgE) following single or multiple infusions of Descartes-08 in MG auto-antibody seropositive patients (Parts 1 and 2 only) | Day 0 to Day 168 |
| Effect of single or multiple infusions of Descartes-08 on patients by standard clinical assessment scales | Quantify the clinical effect of single or multiple infusions of Descartes-08 on patients by standard clinical assessment scales (MG ADL, QMG, MG QoL 15R, MG PIS, MG Composite) over a period of 24 weeks in MG auto-antibody seropositive and seronegative patients together and separately (all parts). | Day 0 to Day 168 |
| Irvine |
| California |
| 92697 |
| United States |
| University of Colorado Anschutz Medical Campus | Aurora | Colorado | 80045 | United States |
| SFM Clinical Research, LLC | Boca Raton | Florida | 33487 | United States |
| Neurology Associates, P.A. | Orlando | Florida | 32751 | United States |
| University of South Florida | Tampa | Florida | 33612 | United States |
| University of Augusta | Augusta | Georgia | 30912 | United States |
| University of Kansas Medical Center Research Institute | Kansas City | Kansas | 66160 | United States |
| Dent Neurologic Institute | Amherst | New York | 14226 | United States |
| University of North Carolina | Chapel Hill | North Carolina | 27599 | United States |
| Oregon Health & Science University | Portland | Oregon | 97239 | United States |
| University of Pennsylvania | Philadelphia | Pennsylvania | 19104 | United States |
| Allegheny Health Network | Pittsburgh | Pennsylvania | 15212 | United States |
| Virginia Commonwealth University | Richmond | Virginia | 23219 | United States |
| University of Alberta | Edmonton | Alberta | T6G2C8 | Canada |
| Derived |
| Vu T, Durmus H, Rivner M, Shroff S, Ragole T, Myers B, Pasnoor M, Small G, Karam C, Vullaganti M, Peltier A, Sahagian G, Feinberg MH, Slanksy A, Barnett-Tapia C, Siddiqi Z, Gwathmey K, Badruddoja MA, Kamboh H, Ruggerie RN, Fedak RR, Stewart CA, Kurtoglu M, Kalayoglu M, Singer M, Jewell CM, Miljkovic MD, Dimachkie M, Mozaffar T, Howard JF Jr; MG-001 Study Team. BCMA-directed mRNA CAR T cell therapy for myasthenia gravis: a randomized, double-blind, placebo-controlled phase 2b trial. Nat Med. 2026 Mar;32(3):1131-1141. doi: 10.1038/s41591-025-04171-y. Epub 2026 Jan 9. |
| 37353278 | Derived | Granit V, Benatar M, Kurtoglu M, Miljkovic MD, Chahin N, Sahagian G, Feinberg MH, Slansky A, Vu T, Jewell CM, Singer MS, Kalayoglu MV, Howard JF Jr, Mozaffar T; MG-001 Study Team. Safety and clinical activity of autologous RNA chimeric antigen receptor T-cell therapy in myasthenia gravis (MG-001): a prospective, multicentre, open-label, non-randomised phase 1b/2a study. Lancet Neurol. 2023 Jul;22(7):578-590. doi: 10.1016/S1474-4422(23)00194-1. |
| 37353270 | Derived | Meisel A. Are CAR T cells the answer to myasthenia gravis therapy? Lancet Neurol. 2023 Jul;22(7):545-546. doi: 10.1016/S1474-4422(23)00211-9. No abstract available. |
| ID | Term |
|---|---|
| D009157 | Myasthenia Gravis |
| ID | Term |
|---|---|
| D020361 | Paraneoplastic Syndromes, Nervous System |
| D009423 | Nervous System Neoplasms |
| D009371 | Neoplasms by Site |
| D009369 | Neoplasms |
| D010257 | Paraneoplastic Syndromes |
| D020274 | Autoimmune Diseases of the Nervous System |
| D009422 | Nervous System Diseases |
| D019636 | Neurodegenerative Diseases |
| D020511 | Neuromuscular Junction Diseases |
| D009468 | Neuromuscular Diseases |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
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