Descartes-08 CAR-T Cells in Generalized Myasthenia Gravis (MG)

Autologous T-Cells Expressing A Chimeric Antigen Receptor Directed To B-Cell Maturation Antigen (BCMA) In Patients With Generalized Myasthenia Gravis (MG)

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

Study Overview

• Status: Active, not recruiting
• Conditions: Myasthenia Gravis, Generalized
• Intervention / Treatment: Drug: Descartes-08

• Study Type: Interventional
• Enrollment (Estimated): 30
• Phase: Phase 2

Contacts and Locations

Study Locations

• Canada
  • Alberta
    • Edmonton, Alberta, Canada, T6G2C8
      • University of Alberta
• United States
  • California
    • Carlsbad, California, United States, 92011
      • Profound Research
    • Irvine, California, United States, 92697
      • University of California Irvine
  • Colorado
    • Aurora, Colorado, United States, 80045
      • University of Colorado Anschutz Medical Campus
  • Florida
    • Boca Raton, Florida, United States, 33487
      • SFM Clinical Research, LLC
    • Orlando, Florida, United States, 32751
      • Neurology Associates, P.A.
    • Tampa, Florida, United States, 33612
      • University of South Florida
  • Georgia
    • Augusta, Georgia, United States, 30912
      • University of Augusta
  • Kansas
    • Kansas City, Kansas, United States, 66160
      • University of Kansas Medical Center Research Institute
  • New York
    • Amherst, New York, United States, 14226
      • Dent Neurologic Institute
  • North Carolina
    • Chapel Hill, North Carolina, United States, 27599
      • University of North Carolina
  • Oregon
    • Portland, Oregon, United States, 97239
      • Oregon Health & Science University
  • Pennsylvania
    • Philadelphia, Pennsylvania, United States, 19104
      • University of Pennsylvania
    • Pittsburgh, Pennsylvania, United States, 15212
      • Allegheny Health Network
  • Virginia
    • Richmond, Virginia, United States, 23219
      • Virginia Commonwealth University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Crossover Assignment
• Masking: Quadruple

Number of Arms

3

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Phase 1b Dose-Escalation; Generalized Myasthenia Gravis	Drug: Descartes-08; Autogolous T-cells expressing a chimeric antigen receptor directed to BCMA
Experimental: Phase IIa Expansion; Generalized Myasthenia Gravis	Drug: Descartes-08; Autogolous T-cells expressing a chimeric antigen receptor directed to BCMA
Placebo Comparator: Phase IIb Randomized Control Trial; Generalized Myasthenia Gravis	Drug: Descartes-08; Autogolous T-cells expressing a chimeric antigen receptor directed to BCMA

What is the study measuring?

Primary Outcome Measures

Outcome Measure	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

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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
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
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

Collaborators and Investigators

• Sponsor: Cartesian Therapeutics

Publications and helpful links

Helpful Links

• Additional information can be found at Cartformg.org

Study record dates

Study Major Dates

• Study Start (Actual): December 4, 2019
• Primary Completion (Estimated): July 31, 2025
• Study Completion (Estimated): March 31, 2026

Study Registration Dates

• First Submitted: October 29, 2019
• First Submitted That Met QC Criteria: October 29, 2019
• First Posted (Actual): October 31, 2019

Study Record Updates

• Last Update Posted (Actual): May 28, 2025
• Last Update Submitted That Met QC Criteria: May 22, 2025
• Last Verified: May 1, 2025

More Information

Terms related to this study

• Keywords: CAR-T; Myasthenia Gravis; MG; T cells; CART; chimeric antigen receptor; Autoimmune; gMG; Descartes-08; Auto-antibody; Auto antibody; Generalized Myasthenia gravis
• Additional Relevant MeSH Terms: Neurologic Manifestations; Musculoskeletal Diseases; Nervous System Diseases; Muscular Diseases; Neuromuscular Manifestations; Pathologic Processes; Neoplasms by Site; Neoplasms; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases; Autoimmune Diseases of the Nervous System; Neurodegenerative Diseases; Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Paraneoplastic Syndromes; Neuromuscular Junction Diseases; Muscle Weakness; Myasthenia Gravis
• Other Study ID Numbers: MG-001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No