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

NCT ID: NCT06626919

Last Updated: 2025-08-01

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1

Total Enrollment

30 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-04-30

Study Completion Date

2028-04-30

Brief Summary

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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.

Detailed Description

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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 CD3ΞΎ, 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.

Conditions

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

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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anito-cel

Single dose of anito-cel cells infused intravenously

Group Type EXPERIMENTAL

anito-cel

Intervention Type BIOLOGICAL

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

Standard Lymphodepletion regimen

Intervention Type DRUG

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

Interventions

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anito-cel

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

Intervention Type BIOLOGICAL

Standard Lymphodepletion regimen

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

Intervention Type DRUG

Other Intervention Names

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anitocabtagene autoleucel CART-ddBCMA Cyclophosphamide Fludarabine

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Arcellx, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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UCLA Medical Center

Los Angeles, California, United States

Site Status RECRUITING

University of California, Irvine

Orange, California, United States

Site Status RECRUITING

Stanford Hospital

Palo Alto, California, United States

Site Status RECRUITING

Karmanos Cancer Institute

Detroit, Michigan, United States

Site Status RECRUITING

Columbia University Irving Medical Center

New York, New York, United States

Site Status RECRUITING

Ohio State University

Columbus, Ohio, United States

Site Status RECRUITING

Oregon Health & Science University (OHSU)

Portland, Oregon, United States

Site Status RECRUITING

Temple University Hospital

Philadelphia, Pennsylvania, United States

Site Status RECRUITING

Houston Methodist Hospital

Houston, Texas, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Clinical Information

Role: CONTACT

240-327-0379

Other Identifiers

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ARC-311

Identifier Type: -

Identifier Source: org_study_id

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