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

Phase 2

Recruiting

• Conditions: Myasthenia Gravis, Generalized
• Interventions: Drug: Descartes-08

• Registration Number: NCT04146051
• Lead Sponsor: Cartesian Therapeutics

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

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2019-10-29
• Study First Submitted QC: 2019-10-29
• Study First Posted: 2019-10-31
• Study Start: 2019-12-04
• Status Verified: 2024-05
• Last Update Submitted: 2024-05-22
• Last Update Posted: 2024-05-24
• Primary Completion: 2025-03-31
• Study Completion: 2026-03-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 30

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

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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.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: CROSSOVER

Arm && Interventions

Group	Intervention	Description
Phase 1b Dose-Escalation	Descartes-08	Generalized Myasthenia Gravis
Phase IIa Expansion	Descartes-08	Generalized Myasthenia Gravis
Phase IIb Randomized Control Trial	Descartes-08	Generalized Myasthenia Gravis

Primary Outcome Measures

Name	Time	Method
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 Outcome Measures

Name	Time	Method
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.
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.
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).
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)

Trial Locations

Locations (15)

Profound Research; 🇺🇸 Carlsbad, California, United States

University of Kansas Medical Center Research Institute; 🇺🇸 Kansas City, Kansas, United States

University of California Irvine; 🇺🇸 Irvine, California, United States

SFM Clinical Research, LLC; 🇺🇸 Boca Raton, Florida, United States

University of Augusta; 🇺🇸 Augusta, Georgia, United States

Allegheny Health Network; 🇺🇸 Pittsburgh, Pennsylvania, United States

University of Pennsylvania; 🇺🇸 Philadelphia, Pennsylvania, United States

University of Alberta; 🇨🇦 Edmonton, Alberta, Canada

Dent Neurologic Institute; 🇺🇸 Amherst, New York, United States

University of Colorado Anschutz Medical Campus; 🇺🇸 Aurora, Colorado, United States

University of South Florida; 🇺🇸 Tampa, Florida, United States

Virginia Commonwealth University; 🇺🇸 Richmond, Virginia, United States

Neurology Associates, P.A.; 🇺🇸 Orlando, Florida, United States

University of North Carolina; 🇺🇸 Chapel Hill, North Carolina, United States

Oregon Health & Science University; 🇺🇸 Portland, Oregon, United States