Ireland
--:--IST
Recruiting Phase 1

Study of Anitocabtagene Autoleucel in Participants With Non-Oncology Plasma Cell-Related Diseases

NCT06626919 · tracked via the Priya Life Science USA tracker
Phase
Phase 1
Started
2025-04-30
Last updated
2026-10-22

Condition(s) studied

Muscular DiseasesNeuromuscular ManifestationsAutoimmuneAutoimmune DiseasesAutoimmune Diseases of the Nervous SystemMyasthenia GravisMuscle Weakness

Investigational drug(s) / intervention(s)

anito-cel →Cyclophosphamide →Fludarabine →

anito-cel: Anitocabtagene autoleucel BCMA directed CAR T-cell therapy using a novel, synthetic binding domain, called a D-Domain

Cyclophosphamide: Administered intravenously

Fludarabine: Administered intravenously

Study summary

The goal of this clinical study is to learn more about the study drug Anitocabtagene autoleucel (anito-cel), and its safety, tolerability, dosing, and preliminary effectiveness in participants with generalized myasthenia gravis (GMG). Anito-cel is a B-cell maturation antigen (BCMA)-directed CAR-T cell therapy.

The primary objective of this study is to assess the safety profile, including any dose-limiting toxicity (DLT) and identification of a maximum tolerated dose (MTD) (if applicable), to support selection of the recommended Phase 2 dose (RP2D) of anito-cel when administered to participants with GMG.

Eligibility

Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
Key Inclusion Criteria: * Individuals must be 18 years of age or older * Must have Myasthenia Gravis Foundation of America (MGFA) clinical classification Grades 2-4A at time of screening * Participant must have clinically active disease and requiring ongoing therapy for Generalized Myasthenia Gravis (GMG) * MG-ADL score ≥ 6 and QMG score ≥ 10 at screening * GMG specific autoantibodies must be above the reference laboratory upper limit of normal (ULN) Key Exclusion Criteria: * Individual 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 Note: Other protocol defined Inclusion/Exclusion criteria may apply.

Primary outcome measure(s)

  • Percentage of Participants Experiencing Treatment-Emergent Adverse Events (TEAE) — First dose date up to 24 months
  • Percentage of Participants Experiencing Dose-Limiting Toxicities (DLT) — Up to 28 days following anito-cel infusion
  • Maximum Tolerated Dose (MTD) of Anito-cel — First dose up to 30 days (Up to 24 months)
  • Percentage of Participants With Laboratory Abnormalities — First dose date up to 24 months
  • Recommended Phase 2 Dose (RP2D) of Anito-cel — Up to 24 months

Trial sites (14)

FacilityCityRegionStatus
Ronald Reagan UCLA Medical Center Los Angeles California Recruiting
UC Irvine Medical Center Orange California Recruiting
Stanford Blood and Marrow Transplant Palo Alto California Recruiting
Moffitt Cancer Center Tampa Florida Recruiting
University of South Florida - Carol and Frank Morsani Center for Advanced Healthcare Tampa Florida Recruiting
Karmanos Cancer Institute Detroit Michigan Recruiting
University of Minnesota Delaware Clinical Research Unit Minneapolis Minnesota Recruiting
Mayo Clinic Rochester Minnesota Recruiting
Columbia University Irving Medical Center New York New York Recruiting
The Ohio State University Wexner Medical Center Columbus Ohio Recruiting
Oregon Health & Science University Portland Oregon Recruiting
Lewis Katz School of Medicine at Temple University Philadelphia Pennsylvania Recruiting
UT Southwestern Medical Center Dallas Texas Recruiting
Houston Methodist Hospital Houston Texas Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06626919 on ClinicalTrials.gov ↗ ← All trials in the USA