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A Phase 1 Study of Anitocabtagene Autoleucel for the Treatment of Subjects With Non-oncology Plasma Cell-related Diseases
Trial Status: active
A Phase 1 dose-escalation study designed to evaluate the safety, tolerability, and
preliminary efficacy of anito-cel in subjects with generalized myasthenia gravis (GMG).
Anitocabtagene autoleucel (anito-cel) is a BCMA-directed CAR-T cell therapy.
Inclusion Criteria
Subject must be 18 years of age or older
Must have MGFA clinical classification Grades 2-4A at time of screening
Subject must have clinically active disease and requiring ongoing therapy for GMG
MG-ADL score 6 and QMG score >10 at screening
GMG specific autoantibodies must be above the reference laboratory ULN
Exclusion Criteria
Subject is pregnant or breastfeeding
Treatment with Anti-CD20 agents, calcineurin inhibitors, FcRN inhibitors, azathioprine, mycophenolate mofetil, methotrexate, or cyclophosphamide within the specified time frame prior to leukapheresis or prior to anito-cel infusion
Previous treatment with any gene therapy, chimeric antigen receptor therapy or T cell engager
Previous thymectomy within 6 months of screening
Major chronic illness that is not well managed at the time of study entry and in the opinion of the investigator
Study sponsor and potential other locations can be found on ClinicalTrials.gov for NCT06626919.
Locations matching your search criteria
United States
Michigan
Detroit
Wayne State University/Karmanos Cancer Institute
Status: Active
Name Not Available
New York
New York
NYP/Columbia University Medical Center/Herbert Irving Comprehensive Cancer Center
Status: Active
Name Not Available
This is a Phase 1 open-label, multi-center safety and dose-escalation study of anito-cel*
in adult subjects with GMG (MGFA Grade 2 to 4a), in whom immunosuppressive therapy is
clinically indicated in the judgement of the treating neurologist. The primary objective
of this study is to assess the safety profile, including any DLT and identification of a
MTD (if applicable), to support selection of the RP2D of anito-cel when administered to
subjects with GMG.
The study will have the following sequential phases: screening, enrollment
(leukapheresis), pretreatment with lymphodepletion (LD) chemotherapy, treatment with
anito-cel and follow-up. Optional bridging therapy is allowed at investigator discretion
while anito-cel is being manufactured.
Following a single infusion of anito-cel both safety and efficacy data will be assessed.
The DLTs will be assessed in the first 28 days following anito-cel administration, and
safety data will be collected throughout the study.
*Anitocabtagene autoleucel (anito-cel) drug product consists of autologous T cells that
have been genetically modified ex vivo to express a D-domain Chimeric Antigen Receptor
(CAR), followed by a cluster of differentiation 8 (CD8) hinge and transmembrane region
that is fused to the intracellular signaling domains for 4-1BB and CD3ξ, that
specifically recognizes B-cell maturation antigen (BCMA). The active substance of
anitocabtagene autoleucel is CAR+ CD3+ T cells that have undergone ex vivo T-cell
activation, gene transfer by replication-deficient lentiviral vector, and expansion.
