A Phase 1 Study of Anitocabtagene Autoleucel for the Treatment of Subjects With Non-oncology Plasma Cell-related Diseases

NCT06626919 | Recruiting Phase 1 FDA Drug

• 1 other identifier: ARC-311
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 13

Brief Summary

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

Autoimmune; Muscular Diseases; Neuromuscular Manifestations; Myasthenia Gravis; Muscle Weakness; Autoimmune Diseases; Autoimmune Diseases of the Nervous System

Keywords

ARC-311; CART; CAR-T; BCMA; Anito-cel; Generalized Myasthenia Gravis; gMG; MG; Myasthenia Gravis; non-oncology plasma cell; autoimmune; auto-antibody; chimeric antigen receptor; D-Domain chimeric antigen receptor; B-cell maturation antigen (BCMA); anitocabtagene autoleucel

Outcome Measures

Primary Outcomes (2)

• Assess safety profile, including any DLT and MTD (if applicable)

  Type, incidence, and severity of treatment-emergent adverse events (TEAEs), including DLT(s) and laboratory abnormalities

  24 months

• Selection of RP2D

  Evaluate the MTD and establish the RP2D

  24 months

Secondary Outcomes (11)

• Quantify Clinical Effect of Anito-cel in the Myasthenia Gravis Activities of Daily Living (MG ADL) score

  24 months

• Quantify Clinical Effect of Anito-cel in the Quantitative Myasthenia Gravis (QMG) score

  24 months

• Quantify Clinical Effect of Anito-cel in the Myasthenia Gravis Composite (MGC) scale.

  24 months

• Mean change in QMG score

  24 months

• Mean change in MG-ADL score

  24 months

Study Arms (1)

anito-cel

EXPERIMENTAL

Single dose of anito-cel cells infused intravenously

Biological: anito-cel; Drug: Standard Lymphodepletion regimen

Interventions

anito-cel BIOLOGICAL

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

Also known as: anitocabtagene autoleucel, CART-ddBCMA

anito-cel

Standard Lymphodepletion regimen DRUG

Standard lymphodepletion regimen subject receive 5 days prior to CAR T infusion

Also known as: Cyclophosphamide, Fludarabine

anito-cel

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Arcellx, Inc.: lead

Study Sites (13)

UCLA Medical Center

Los Angeles, California, 90095, United States

RECRUITING

University of California, Irvine

Orange, California, 92602, United States

RECRUITING

Stanford Hospital

Palo Alto, California, 94305, United States

RECRUITING

University of South Florida - Carol and Frank Morsani Center of Advanced Healthcare

Tampa, Florida, 33612, United States

RECRUITING

Karmanos Cancer Institute

Detroit, Michigan, 48201, United States

RECRUITING

University of Minnesota Delaware Clinical Research Unit

Minneapolis, Minnesota, 55414, United States

RECRUITING

Mayo Clinic

Rochester, Minnesota, 55905, United States

RECRUITING

Columbia University Irving Medical Center

New York, New York, 10032, United States

RECRUITING

Ohio State University

Columbus, Ohio, 43221, United States

RECRUITING

Oregon Health & Science University (OHSU)

Portland, Oregon, 97239, United States

RECRUITING

Temple University Hospital

Philadelphia, Pennsylvania, 19140, United States

RECRUITING

UT Southwestern Medical Center

Dallas, Texas, 75390, United States

RECRUITING

Houston Methodist Hospital

Houston, Texas, 77030, United States

RECRUITING

MeSH Terms

Conditions

Muscular Diseases; Neuromuscular Manifestations; Autoimmune Diseases; Autoimmune Diseases of the Nervous System; Myasthenia Gravis; Muscle Weakness

Interventions

Cyclophosphamide; fludarabine

Condition Hierarchy (Ancestors)

Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Neurologic Manifestations; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Immune System Diseases; Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Pathologic Processes

Intervention Hierarchy (Ancestors)

Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Organic Chemicals; Phosphoramides; Organophosphorus Compounds

Central Study Contacts

Clinical Information

CONTACT

240-327-0379; clinical@arcellx.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 19, 2024
• First Posted: October 4, 2024
• Study Start: April 30, 2025
• Primary Completion (Estimated): March 1, 2027
• Study Completion (Estimated): April 1, 2028
• Last Updated: February 25, 2026

Record last verified: 2026-02

Locations

US (13)