Treatment of generalized myasthenia gravis with a new CAR-T therapy
A Phase 1 Study of Anitocabtagene Autoleucel for the Treatment of Subjects With Non-oncology Plasma Cell-related Diseases
This study is testing a new CAR-T therapy to see if it can safely help adults with generalized myasthenia gravis feel better.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 30 (estimated) |
| Ages | 18 Years and up |
| Sex | All |
| Sponsor | Arcellx, Inc. Industry-sponsored |
| Drugs / interventions | chimeric antigen receptor, methotrexate, cyclophosphamide, CAR-T, chemotherapy |
| Locations | 13 sites (Los Angeles, California and 12 other locations) |
| Trial ID | NCT06626919 on ClinicalTrials.gov |
What this trial studies
This Phase 1 open-label, multi-center study evaluates the safety, tolerability, and preliminary efficacy of anitocabtagene autoleucel (anito-cel) in adults with generalized myasthenia gravis (GMG). Participants will undergo leukapheresis followed by lymphodepletion chemotherapy and a single infusion of anito-cel. The study aims to identify the maximum tolerated dose and assess any dose-limiting toxicities within the first 28 days post-infusion. Safety data will be collected throughout the study to support further development of this CAR-T cell therapy.
Who should consider this trial
Good fit: Ideal candidates are adults aged 18 and older with clinically active generalized myasthenia gravis requiring ongoing therapy.
Not a fit: Patients who have received prior gene therapy or CAR-T therapy, or those with certain chronic illnesses may not benefit from this study.
Why it matters
Potential benefit: If successful, this therapy could provide a novel treatment option for patients with generalized myasthenia gravis, potentially improving their quality of life.
How similar studies have performed: While CAR-T therapies have shown success in oncology, this application in non-oncology plasma cell-related diseases is novel and untested.
Eligibility criteria
Show full inclusion / exclusion criteria
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
Where this trial is running
Los Angeles, California and 12 other locations
- UCLA Medical Center — Los Angeles, California, United States (Recruiting)
- University of California, Irvine — Orange, California, United States (Recruiting)
- Stanford Hospital — Palo Alto, California, United States (Recruiting)
- University of South Florida - Carol and Frank Morsani Center of Advanced Healthcare — Tampa, Florida, United States (Recruiting)
- Karmanos Cancer Institute — Detroit, Michigan, United States (Recruiting)
- University of Minnesota Delaware Clinical Research Unit — Minneapolis, Minnesota, United States (Recruiting)
- Mayo Clinic — Rochester, Minnesota, United States (Recruiting)
- Columbia University Irving Medical Center — New York, New York, United States (Recruiting)
- Ohio State University — Columbus, Ohio, United States (Recruiting)
- Oregon Health & Science University (OHSU) — Portland, Oregon, United States (Recruiting)
- Temple University Hospital — Philadelphia, Pennsylvania, United States (Recruiting)
- UT Southwestern Medical Center — Dallas, Texas, United States (Recruiting)
- Houston Methodist Hospital — Houston, Texas, United States (Recruiting)
Study contacts
- Study coordinator: Clinical Information
- Email: clinical@arcellx.com
- Phone: 240-327-0379
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.