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

Primary Purpose

Myasthenia Gravis, Generalized

Status
Recruiting
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
Descartes-08
Sponsored by
Cartesian Therapeutics
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Myasthenia Gravis, Generalized focused on measuring Descartes-08, CART, CAR-T, Autoimmune, Auto-antibody, Auto antibody, chimeric antigen receptor, T cells, MG, gMG, Myasthenia Gravis, Generalized Myasthenia gravis

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • The Research Center of Southern CaliforniaRecruiting
  • University of California IrvineRecruiting
  • University of Colorado Anschutz Medical CampusRecruiting
  • SFM Clinical Research, LLCRecruiting
  • Neurology Associates, P.A.Recruiting
  • University of South FloridaRecruiting
  • University of AugustaRecruiting
  • University of Kansas Medical Center Research InstituteRecruiting
  • Dent Neurologic InstituteRecruiting
  • University of North CarolinaRecruiting
  • Oregon Health & Science UniversityRecruiting
  • University of PennsylvaniaRecruiting
  • Allegheny Health NetworkRecruiting
  • Virginia Commonwealth UniversityRecruiting
  • University of AlbertaRecruiting

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

Placebo Comparator

Arm Label

Phase 1b Dose-Escalation

Phase IIa Expansion

Phase IIb Randomized Control Trial

Arm Description

Generalized Myasthenia Gravis

Generalized Myasthenia Gravis

Generalized Myasthenia Gravis

Outcomes

Primary Outcome Measures

Myasthenia Gravis -Activities of Daily Living Score
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

Comparison on Descartes-08 versus placebo on QMG, MG QoL 15R, MG Composite, MG PIS
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).
Comparison on Descartes-08 versus placebo on MG assessments in crossover patients
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).
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)
Effect of single or multiple infusions of Descartes-08 on patients by standard clinical assessment scales
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).

Full Information

First Posted
October 29, 2019
Last Updated
July 21, 2023
Sponsor
Cartesian Therapeutics
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1. Study Identification

Unique Protocol Identification Number
NCT04146051
Brief Title
Descartes-08 CAR-T Cells in Generalized Myasthenia Gravis (MG)
Official Title
Autologous T-Cells Expressing A Chimeric Antigen Receptor Directed To B-Cell Maturation Antigen (BCMA) In Patients With Generalized Myasthenia Gravis (MG)
Study Type
Interventional

2. Study Status

Record Verification Date
July 2023
Overall Recruitment Status
Recruiting
Study Start Date
December 4, 2019 (Actual)
Primary Completion Date
December 1, 2023 (Anticipated)
Study Completion Date
December 1, 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Cartesian Therapeutics

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Myasthenia Gravis, Generalized
Keywords
Descartes-08, CART, CAR-T, Autoimmune, Auto-antibody, Auto antibody, chimeric antigen receptor, T cells, MG, gMG, Myasthenia Gravis, Generalized Myasthenia gravis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Crossover Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
30 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Phase 1b Dose-Escalation
Arm Type
Experimental
Arm Description
Generalized Myasthenia Gravis
Arm Title
Phase IIa Expansion
Arm Type
Experimental
Arm Description
Generalized Myasthenia Gravis
Arm Title
Phase IIb Randomized Control Trial
Arm Type
Placebo Comparator
Arm Description
Generalized Myasthenia Gravis
Intervention Type
Drug
Intervention Name(s)
Descartes-08
Intervention Description
Autogolous T-cells expressing a chimeric antigen receptor directed to BCMA
Primary Outcome Measure Information:
Title
Myasthenia Gravis -Activities of Daily Living Score
Description
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.
Time Frame
Day 0 to Day 85
Secondary Outcome Measure Information:
Title
Comparison on Descartes-08 versus placebo on QMG, MG QoL 15R, MG Composite, MG PIS
Description
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).
Time Frame
Day 0 to Day 85
Title
Comparison on Descartes-08 versus placebo on MG assessments in crossover patients
Description
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).
Time Frame
Day 0 to Day 85
Title
Change in titer of myasthenia specific autoantibody titers
Description
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
Title
Effect of single or multiple infusions of Descartes-08 on patients by standard clinical assessment scales
Description
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).
Time Frame
Day 0 to Day 168

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Study Trial Central Mailbox
Phone
302-648-6497
Email
trials@cartesiantx.com
Facility Information:
Facility Name
The Research Center of Southern California
City
Carlsbad
State/Province
California
ZIP/Postal Code
92011
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Gregory Sahagian, MD
Facility Name
University of California Irvine
City
Irvine
State/Province
California
ZIP/Postal Code
92697
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Tahseen Mozaffar, MD
Facility Name
University of Colorado Anschutz Medical Campus
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Brianna Blume
First Name & Middle Initial & Last Name & Degree
Thomas Ragole, M.D.
Facility Name
SFM Clinical Research, LLC
City
Boca Raton
State/Province
Florida
ZIP/Postal Code
33487
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Marc Feinberg, MD
Facility Name
Neurology Associates, P.A.
City
Orlando
State/Province
Florida
ZIP/Postal Code
32751
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Adam Slansky, MD
Facility Name
University of South Florida
City
Tampa
State/Province
Florida
ZIP/Postal Code
33612
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Tuan H. Vu, MD
Facility Name
University of Augusta
City
Augusta
State/Province
Georgia
ZIP/Postal Code
30912
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Michael Rivner, MD
Facility Name
University of Kansas Medical Center Research Institute
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Ali Ciersdorff
First Name & Middle Initial & Last Name & Degree
Andrew Heim
First Name & Middle Initial & Last Name & Degree
Mamatha Pasnoor, M.D.
Facility Name
Dent Neurologic Institute
City
Amherst
State/Province
New York
ZIP/Postal Code
14226
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Bennett Myers, MD
Facility Name
University of North Carolina
City
Chapel Hill
State/Province
North Carolina
ZIP/Postal Code
27599
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
James F Howard, MD
Facility Name
Oregon Health & Science University
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Nizar Chahin, MD
Facility Name
University of Pennsylvania
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Kelsey Moulton
First Name & Middle Initial & Last Name & Degree
Chafic Karam, M.D.
Facility Name
Allegheny Health Network
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15212
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Dom Contestabile
First Name & Middle Initial & Last Name & Degree
George Small, M.D.
Facility Name
Virginia Commonwealth University
City
Richmond
State/Province
Virginia
ZIP/Postal Code
23219
Country
United States
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Melissa Hayes
First Name & Middle Initial & Last Name & Degree
Kelly Gwathmey, M.D.
Facility Name
University of Alberta
City
Edmonton
State/Province
Alberta
ZIP/Postal Code
T6G2C8
Country
Canada
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Faraz Hussain, Ph.D.
First Name & Middle Initial & Last Name & Degree
Zaeem Siddiqi, M.D.

12. IPD Sharing Statement

Plan to Share IPD
No
Links:
URL
https://cartformg.org/
Description
Additional information can be found at Cartformg.org

Learn more about this trial

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

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