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

Phase 2Active Not RecruitingNCT04146051

Cartesian Therapeutics30 enrolled

Overview

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 Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Myasthenia Gravis, Generalized

Interventions

Descartes-08DRUG

Autogolous T-cells expressing a chimeric antigen receptor directed to BCMA

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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.

Locations (15)

Profound Research

Carlsbad, California, United States

University of California Irvine

Irvine, California, United States

Outcomes

Primary Outcomes

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.

Time frame: Day 0 to Day 85

Secondary Outcomes

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.

Time frame: 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.

Time frame: 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)

Time frame: Day 0 to Day 168

Data from ClinicalTrials.gov

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