A Phase 1 Study of Anitocabtagene Autoleucel for the Treatment of Subjects With Non-oncology Plasma Cell-related Diseases
Purpose
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.
Conditions
- Muscular Diseases
- Neuromuscular Manifestations
- Autoimmune
- Autoimmune Diseases
- Autoimmune Diseases of the Nervous System
- Myasthenia Gravis
- Muscle Weakness
Eligibility
- Eligible Ages
- Over 18 Years
- Eligible Sex
- All
- Accepts Healthy Volunteers
- No
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 Design
- Phase
- Phase 1
- Study Type
- Interventional
- Allocation
- N/A
- Intervention Model
- Single Group Assignment
- Primary Purpose
- Treatment
- Masking
- None (Open Label)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental anito-cel |
Single dose of anito-cel cells infused intravenously |
|
Recruiting Locations
Los Angeles 5368361, California 5332921 90095
Orange 5379513, California 5332921 92602
Palo Alto 5380748, California 5332921 94305
Detroit 4990729, Michigan 5001836 48201
New York 5128581, New York 5128638 10032
Columbus 4509177, Ohio 5165418 43221
Portland 5746545, Oregon 5744337 97239
Philadelphia 4560349, Pennsylvania 6254927 19140
Houston 4699066, Texas 4736286 77030
More Details
- NCT ID
- NCT06626919
- Status
- Recruiting
- Sponsor
- Arcellx, Inc.
Detailed Description
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.