Investigating an mRNA CAR T-cell Therapy, Known as Descartes-08, as a Potential Approach to Treat Myasthenia Gravis

NCT06799247 | Recruiting Phase 3 FDA Drug

• 1 other identifier: RNAC-MG-002 (AURORA)
• Study Type: interventional
• Target: 100
• Locations: 8 countries
• Sites: 34

Brief Summary

The AURORA Study is evaluating the safety, tolerability, and efficacy of an investigational mRNA CAR T-cell therapy known as Descartes-08 in adults with acetylcholine receptor autoantibody -positive generalized myasthenia gravis. Part 1 of the study will last around 6 months. For eligible participants, Part 2 will last around 8 months.

Conditions

Myasthaenia Gravis

Keywords

myasthenia gravis; CAR-T therapy; Cell Therapy; Decartes-8; BMCA; B cell maturation antigen

Outcome Measures

Primary Outcomes (1)

• Myasthenia Gravis Activities of Daily Living (MG-ADL)

  To evaluate the efficacy of Descartes-08 as assessed by the proportion of Myasthenia Gravis Activities of Daily Living (MG-ADL) responders at Month 4.

  assessment at 4 months of study

Study Arms (2)

Decartes-08

EXPERIMENTAL

This group will undergo leukapheresis and receive manufactured Decartes-08

Biological: Decartes-08

Placebo

PLACEBO COMPARATOR

This group will receive placebo

Other: Placebo Drug

Interventions

Placebo Drug OTHER

infusion without Decartes-08

Placebo

Decartes-08 BIOLOGICAL

Autologous mRNA CAR T-cell therapy

Decartes-08

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patient must be at least 18 years of age.
• Patient must have generalized myasthenia gravis (gMG), Myasthenia Gravis Foundation of America (MGFA) clinical classification grades 2-4 at the time of Sscreening.
• MG-Activities of Daily Living (MG ADL) total score ≥ 6.
• Concomitant immunosuppressive drugs must be deemed necessary by the investigator. The dose must be stable for a minimum of 8 weeks prior to Baseline visit.
• If a patient is using corticosteroids, the daily dose should not exceed 40 mg/day of prednisone equivalent. The dose must have been stable for a minimum of 8 weeks prior to Baseline visit.
• Acetylcholine receptor autoantibody (anti-nAChR) titer or anti-AChR cluster antibody must be above the reference laboratory upper normal limit (UNL) and documented within the past 10 years of screening.
• Patient must be willing to return for all study visits.
• Patient must be able to give written informed consent.
• Women of childbearing potential must agree to use highly effective birth control from Screening until 14 days post last dose of Descartes-08,

You may not qualify if:

• 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.
• Diagnosis of gMG within 12 months of screening.
• No history of systemic treatment for gMG other than acetylcholine esterase inhibitors.
• Diagnosis of a neuromuscular disease other than gMG.
• Patient is pregnant or lactating.
• Treatment with intravenous immunoglobulin (IVIG) or plasma exchange within 4 weeks prior to the Baseline visit.
• Treatment with rituximab or ocrelizumab within 12 months prior to Baseline visit; treatment with calcineurin inhibitors (e.g. tacrolimus, cyclosporine, cyclophosphamide), Neonatal Fc receptor antagonists, and/or other biologics within 3 weeks prior to planned leukapheresis and within 8 weeks prior to Baseline visit.
• The patient has started treatment with a complement 5a (C5a) inhibitor, such as eculizumab, within 8 weeks of Baseline visit. (NOTE: patients who have been receiving a C5a inhibitor for more than 8 weeks and meet other criteria for enrollment are eligible for treatment).
• Prior treatment with B-cell maturation antigen (BCMA)-directed therapy (e.g. monoclonal antibody, T-cell engager, or chimeric antigen receptor T-cell \[CAR-T\]).
• Absolute neutrophil count (ANC) \< 1000 cells/microliter.
• Hemoglobin \< 8.0 g/dL.
• Platelets \< 50,000/mm3.
• Alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) \> 3x above normal.
• Creatine clearance less than 30 mL/min.
• History of primary immunodeficiency, organ, or allogeneic bone marrow transplant.

Sponsors & Collaborators

• Cartesian Therapeutics: lead

Study Sites (34)

A40

Tucson, Arizona, 85718, United States

RECRUITING

A13

Carlsbad, California, 92011, United States

RECRUITING

A46

Los Angeles, California, 90095, United States

RECRUITING

A14

Orange, California, 92868, United States

RECRUITING

A21

Aurora, Colorado, 80045, United States

RECRUITING

A50

Washington D.C., District of Columbia, 20007, United States

RECRUITING

A48

Maitland, Florida, 32751, United States

RECRUITING

A10

Tampa, Florida, 33612, United States

RECRUITING

A53

O'Fallon, Illinois, 62269, United States

RECRUITING

A20

Fairway, Kansas, 66205, United States

RECRUITING

A16

Lexington, Kentucky, 40536, United States

RECRUITING

A38

Boston, Massachusetts, 02111, United States

RECRUITING

A12

Amherst, New York, 14226, United States

RECRUITING

A52

New York, New York, 10027, United States

RECRUITING

A47

New York, New York, 10065, United States

RECRUITING

A22

Chapel Hill, North Carolina, 27599, United States

RECRUITING

A39

Charlotte, North Carolina, 28204, United States

RECRUITING

A15

Portland, Oregon, 97239, United States

RECRUITING

A11

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

A49

Pittsburgh, Pennsylvania, 15213, United States

RECRUITING

A43

Houston, Texas, 77030, United States

RECRUITING

A41

Seattle, Washington, 98195, United States

RECRUITING

A54

Milwaukee, Wisconsin, 53215, United States

RECRUITING

A18

Toronto, Canada

RECRUITING

A23

Rome, Italy

RECRUITING

A30

Krakow, Poland

RECRUITING

A24

Belgrade, Serbia

RECRUITING

A25

Barcelona, Spain

RECRUITING

A26

Barcelona, Spain

RECRUITING

A31

Madrid, Spain

RECRUITING

A32

Ankara, Turkey (Türkiye)

RECRUITING

A17

Istanbul, Turkey (Türkiye)

RECRUITING

A33

Birmingham, United Kingdom

RECRUITING

A51

Sheffield, United Kingdom

RECRUITING

MeSH Terms

Conditions

Myasthenia Gravis

Condition Hierarchy (Ancestors)

Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases

Central Study Contacts

Cartesian Clinical Trials

CONTACT

617-231-8102; trials@cartesiantx.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: first part of the study is double blinided and the second part of the study is open label
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: This is a randomized, double-blind, placebo-controlled, multi-center phase 3 trial to evaluate the efficacy, safety and tolerability of autologous T-cells expressing a chimeric antigen receptor (CAR) directed to B-Cell maturation antigen (BCMA) in patients with antibody positive gMG. The cell product will be referred to as "Descartes-08".

Study Record Dates

• First Submitted: January 23, 2025
• First Posted: January 29, 2025
• Study Start: May 6, 2025
• Primary Completion (Estimated): November 30, 2026
• Study Completion (Estimated): September 30, 2027
• Last Updated: March 24, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

SE (1); ES (3); TU (2); PL (1); US (23); GB (2); IT (1); CA (1)