Clinical trial NCT04146051: 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
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE2
Conditions: 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
Lead sponsor: Cartesian Therapeutics
Sponsor class: INDUSTRY
First posted: 2019-10-31
Enrollment: 30
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

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


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.

Primary outcomes:
measure: Proportion of patients with MG Composite improvement of ≥5 points.
timeFrame: Day 0 to Day 85
Secondary outcomes:
measure: Comparison on Descartes-08 versus placebo on QMG and MG ADL
timeFrame: Day 0 to Day 85
measure: Comparison on Descartes-08 versus placebo on MG assessments in crossover patients
timeFrame: Day 0 to Day 85
measure: Change in titer of myasthenia specific autoantibody titers
timeFrame: Day 0 to Day 168
measure: Effect of single or multiple infusions of Descartes-08 on patients by standard clinical assessment scales
timeFrame: Day 0 to Day 168
Other outcomes:
Locations:
city: Carlsbad
country: United States
facility: Profound Research
state: California
city: Irvine
country: United States
facility: University of California Irvine
state: California
city: Aurora
country: United States
facility: University of Colorado Anschutz Medical Campus
state: Colorado
city: Boca Raton
country: United States
facility: SFM Clinical Research, LLC
state: Florida
city: Orlando
country: United States
facility: Neurology Associates, P.A.
state: Florida
city: Tampa
country: United States
facility: University of South Florida
state: Florida
city: Augusta
country: United States
facility: University of Augusta
state: Georgia
city: Kansas City
country: United States
facility: University of Kansas Medical Center Research Institute
state: Kansas
city: Amherst
country: United States
facility: Dent Neurologic Institute
state: New York
city: Chapel Hill
country: United States
facility: University of North Carolina
state: North Carolina
city: Portland
country: United States
facility: Oregon Health & Science University
state: Oregon
city: Philadelphia
country: United States
facility: University of Pennsylvania
state: Pennsylvania
city: Pittsburgh
country: United States
facility: Allegheny Health Network
state: Pennsylvania
city: Richmond
country: United States
facility: Virginia Commonwealth University
state: Virginia
city: Edmonton
country: Canada
facility: University of Alberta
state: Alberta
Study officials:
References: