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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04146051
Recruitment Status : Recruiting
First Posted : October 31, 2019
Last Update Posted : February 6, 2024
Sponsor:
Information provided by (Responsible Party):
Cartesian Therapeutics

Study Description
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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

Condition or disease Intervention/treatment Phase
Myasthenia Gravis, Generalized Drug: Descartes-08 Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Autologous T-Cells Expressing A Chimeric Antigen Receptor Directed To B-Cell Maturation Antigen (BCMA) In Patients With Generalized Myasthenia Gravis (MG)
Actual Study Start Date : December 4, 2019
Estimated Primary Completion Date : March 31, 2025
Estimated Study Completion Date : March 31, 2026

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Arms and Interventions
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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


Outcome Measures
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Primary Outcome Measures :
  1. Myasthenia Gravis -Activities of Daily Living Score [ Time Frame: 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 MG ADL 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 :
  1. Comparison on Descartes-08 versus placebo on QMG, MG QoL 15R, MG Composite, MG PIS [ Time Frame: Day 0 to Day 85 ]
    Compare the effect of Descartes-08 versus placebo on QMG, MG QoL 15R, MG Composite, and MG PIS (change from baseline to Week 12) for all randomized patients (Part 3 only).

  2. Comparison on Descartes-08 versus placebo on MG assessments in crossover patients [ Time Frame: Day 0 to Day 85 ]
    Compare the effect of Descartes-08 versus placebo on MG ADL, QMG, MG QoL 15R, MG Composite, and MG PIS (change from baseline to Week 12) in patients who cross over from placebo to Descartes-08 (Part 3 only).

  3. Change in titer of myasthenia specific autoantibody titers [ Time Frame: 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)

  4. Effect of single or multiple infusions of Descartes-08 on patients by standard clinical assessment scales [ Time Frame: 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).


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04146051


Contacts
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Contact: Study Trial Central Mailbox 302-648-6497 trials@cartesiantx.com

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

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Responsible Party: Cartesian Therapeutics
ClinicalTrials.gov Identifier: NCT04146051    
Other Study ID Numbers: MG-001
First Posted: October 31, 2019    Key Record Dates
Last Update Posted: February 6, 2024
Last Verified: February 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Cartesian Therapeutics:
Descartes-08
CART
CAR-T
Autoimmune
Auto-antibody
Auto antibody
 chimeric antigen receptor
T cells
MG
gMG
Myasthenia Gravis
Generalized Myasthenia gravis
Additional relevant MeSH terms:
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Myasthenia Gravis
Muscle Weakness
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Pathologic Processes
Paraneoplastic Syndromes, Nervous System
Nervous System Neoplasms
 Neoplasms by Site
Neoplasms
Paraneoplastic Syndromes
Autoimmune Diseases of the Nervous System
Neurodegenerative Diseases
Neuromuscular Junction Diseases
Neuromuscular Diseases
Autoimmune Diseases
Immune System Diseases