Myasthenia Gravis, Generalized
Conditions
Keywords
Descartes-08, CART, CAR-T, Autoimmune, Auto-antibody, Auto antibody, chimeric antigen receptor, T cells, MG, gMG, Myasthenia Gravis, Generalized Myasthenia gravis
Brief summary
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
Interventions
DRUGDescartes-08
Autogolous T-cells expressing a chimeric antigen receptor directed to BCMA
Sponsors
Cartesian Therapeutics
Study design
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Patient must be at least 18 years of age. * Patient must have Generalized Myasthenia Gravis at the time of screening. * Concomitant immunosuppressive drugs must be deemed necessary by the investigator. * Seronegative Patients are included
Exclusion criteria
* Major chronic illness that is not well managed at the time of study entry and in the opinion of the investigator may increase the risk to the patient. * Patient is pregnant or lactating.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Proportion of patients with MG Composite improvement of ≥5 points. | Day 0 to Day 85 | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Comparison on Descartes-08 versus placebo on QMG and MG ADL | Day 0 to Day 85 | 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. |
| Comparison on Descartes-08 versus placebo on MG assessments in crossover patients | Day 0 to Day 85 | 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. |
| Change in titer of myasthenia specific autoantibody titers | Day 0 to Day 168 | 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) |
| Effect of single or multiple infusions of Descartes-08 on patients by standard clinical assessment scales | Day 0 to Day 168 | 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). |
Countries
Canada, United States
Outcome results
None listed