NCT04146051

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

Trial Health

58
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
30

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Dec 2019

Longer than P75 for phase_2

Geographic Reach
2 countries

15 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

October 29, 2019

Completed
2 days until next milestone

First Posted

Study publicly available on registry

October 31, 2019

Completed
1 month until next milestone

Study Start

First participant enrolled

December 4, 2019

Completed
5.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 31, 2025

Completed
8 months until next milestone

Study Completion

Last participant's last visit for all outcomes

March 31, 2026

Completed
Last Updated

May 28, 2025

Status Verified

May 1, 2025

Enrollment Period

5.7 years

First QC Date

October 29, 2019

Last Update Submit

May 22, 2025

Conditions

Myasthenia Gravis, Generalized

Keywords

Descartes-08CARTCAR-TAutoimmuneAuto-antibodyAuto antibodychimeric antigen receptorT cellsMGgMGMyasthenia GravisGeneralized Myasthenia gravis

Outcome Measures

Primary Outcomes (1)

  • 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 Outcomes (4)

  • Comparison on Descartes-08 versus placebo on QMG and MG ADL

    Day 0 to Day 85

  • Comparison on Descartes-08 versus placebo on MG assessments in crossover patients

    Day 0 to Day 85

  • Change in titer of myasthenia specific autoantibody titers

    Day 0 to Day 168

  • Effect of single or multiple infusions of Descartes-08 on patients by standard clinical assessment scales

    Day 0 to Day 168

Study Arms (3)

Phase 1b Dose-Escalation

EXPERIMENTAL

Generalized Myasthenia Gravis

Drug: Descartes-08

Phase IIa Expansion

EXPERIMENTAL

Generalized Myasthenia Gravis

Drug: Descartes-08

Phase IIb Randomized Control Trial

PLACEBO COMPARATOR

Generalized Myasthenia Gravis

Drug: Descartes-08

Interventions

Descartes-08DRUG

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

Phase 1b Dose-EscalationPhase IIa ExpansionPhase IIb Randomized Control Trial

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

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.
  • Patient is pregnant or lactating.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (15)

Profound Research

Carlsbad, California, 92011, United States

Location

University of California Irvine

Irvine, California, 92697, United States

Location

University of Colorado Anschutz Medical Campus

Aurora, Colorado, 80045, United States

Location

SFM Clinical Research, LLC

Boca Raton, Florida, 33487, United States

Location

Neurology Associates, P.A.

Orlando, Florida, 32751, United States

Location

University of South Florida

Tampa, Florida, 33612, United States

Location

University of Augusta

Augusta, Georgia, 30912, United States

Location

University of Kansas Medical Center Research Institute

Kansas City, Kansas, 66160, United States

Location

Dent Neurologic Institute

Amherst, New York, 14226, United States

Location

University of North Carolina

Chapel Hill, North Carolina, 27599, United States

Location

Oregon Health & Science University

Portland, Oregon, 97239, United States

Location

University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

Location

Allegheny Health Network

Pittsburgh, Pennsylvania, 15212, United States

Location

Virginia Commonwealth University

Richmond, Virginia, 23219, United States

Location

University of Alberta

Edmonton, Alberta, T6G2C8, Canada

Location

Related Publications (4)

  • Fedak RR, Ruggerie RN, Shan Y, Curvino EJ, de Sousa JF, Daniel S, Ngo-Casi M, Kamboh H, Vu T, Durmus H, Mozaffar T, Howard JF Jr, English EP, Benson A, Duvernay MT, Singer MS, Kalayoglu MV, Brunn C, Bodansky A, Anderson MS, DeRisi JL, Garcia ST, Yu DJL, Zorn KC, Kurtoglu M, Miljkovic MD, Stewart CA, Jewell CM; MG-001 Study Team. BCMA-directed mRNA CAR-T cell therapy for myasthenia gravis: exploratory biomarker analysis of a placebo-controlled phase 2b trial. Nat Med. 2026 Jan 9. doi: 10.1038/s41591-025-04170-z. Online ahead of print.

    PMID: 41514039DERIVED

  • Vu T, Durmus H, Rivner M, Shroff S, Ragole T, Myers B, Pasnoor M, Small G, Karam C, Vullaganti M, Peltier A, Sahagian G, Feinberg MH, Slanksy A, Barnett-Tapia C, Siddiqi Z, Gwathmey K, Badruddoja MA, Kamboh H, Ruggerie RN, Fedak RR, Stewart CA, Kurtoglu M, Kalayoglu M, Singer M, Jewell CM, Miljkovic MD, Dimachkie M, Mozaffar T, Howard JF Jr; MG-001 Study Team. BCMA-directed mRNA CAR T cell therapy for myasthenia gravis: a randomized, double-blind, placebo-controlled phase 2b trial. Nat Med. 2026 Jan 9. doi: 10.1038/s41591-025-04171-y. Online ahead of print.

    PMID: 41514038DERIVED

  • Granit V, Benatar M, Kurtoglu M, Miljkovic MD, Chahin N, Sahagian G, Feinberg MH, Slansky A, Vu T, Jewell CM, Singer MS, Kalayoglu MV, Howard JF Jr, Mozaffar T; MG-001 Study Team. Safety and clinical activity of autologous RNA chimeric antigen receptor T-cell therapy in myasthenia gravis (MG-001): a prospective, multicentre, open-label, non-randomised phase 1b/2a study. Lancet Neurol. 2023 Jul;22(7):578-590. doi: 10.1016/S1474-4422(23)00194-1.

    PMID: 37353278DERIVED

  • Meisel A. Are CAR T cells the answer to myasthenia gravis therapy? Lancet Neurol. 2023 Jul;22(7):545-546. doi: 10.1016/S1474-4422(23)00211-9. No abstract available.

    PMID: 37353270DERIVED

MeSH Terms

Conditions

Myasthenia Gravis

Condition Hierarchy (Ancestors)

Paraneoplastic Syndromes, Nervous SystemNervous System NeoplasmsNeoplasms by SiteNeoplasmsParaneoplastic SyndromesAutoimmune Diseases of the Nervous SystemNervous System DiseasesNeurodegenerative DiseasesNeuromuscular Junction DiseasesNeuromuscular DiseasesAutoimmune DiseasesImmune System Diseases

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
CROSSOVER
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 29, 2019

First Posted

October 31, 2019

Study Start

December 4, 2019

Primary Completion

July 31, 2025

Study Completion

March 31, 2026

Last Updated

May 28, 2025

Record last verified: 2025-05

Data Sharing

IPD Sharing
Will not share

Locations

CA(1)US(14)