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NCT Number: NCT06626919

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

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.

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Key information

Conditions

Muscular Diseases Autoimmune Autoimmune Diseases Autoimmune Diseases of the Nervous System Immune System Diseases Muscle Weakness Musculoskeletal Diseases Myasthenia Gravis Neoplasms Neoplasms by Site Nervous System Diseases Nervous System Neoplasms Neurodegenerative Diseases Neurologic Manifestations Neuromuscular Diseases Neuromuscular Junction Diseases Neuromuscular Manifestations Paraneoplastic Syndromes Paraneoplastic Syndromes, Nervous System Pathologic Processes Pathological Conditions, Signs and Symptoms Signs and Symptoms

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

UCLA Medical Center, Los Angeles, California, United States

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

anito-cel

Biological

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

Other names: anitocabtagene autoleucel, CART-ddBCMA

Standard lymphodepletion regimen

Drug

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

Other names: Cyclophosphamide, Fludarabine

Primary outcomes

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

    Time frame: 24 months

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

  2. Selection of RP2D

    Time frame: 24 months

    Evaluate the MTD and establish the RP2D

Secondary outcomes

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

    Time frame: 24 months

    The proportion of subjects achieving a ≥2-point change in the MG ADL score from Baseline at any timepoint after treatment. The MG-ADL is an 8-item patient-reported outcome measure assessing GMG symptoms and functional activities related to activities of daily living and producing a total score ranging from 0 to 24, where higher scores indicate greater severity of disease symptoms.

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

    Time frame: 24 months

    The proportion of subjects achieving a ≥3-point change in the QMG from Baseline at any timepoint after treatment. The QMG score is a 13-item direct physician assessment scoring system that quantifies disease severity, based on impairments of body functions and structures. The total QMG score ranges from 0 to 39, where higher scores indicate greater disease severity.

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

    Time frame: 24 months

    The proportion of subjects achieving a ≥3-point change in the MGC score from Baseline at any timepoint after treatment. The MGC is a 10-item assessment that measures signs and symptoms of GMG based on physical examination findings and patient history. The total score ranges from 0 to 50, where higher scores indicate more severe impairment.

  4. Mean change in QMG score

    Time frame: 24 months

    The mean change in the QMG score from Baseline at any timepoint after treatment. The QMG score is a 13-item direct physician assessment scoring system that quantifies disease severity, based on impairments of body functions and structures. The total QMG score ranges from 0 to 39, where higher scores indicate greater disease severity.

  5. Mean change in MG-ADL score

    Time frame: 24 months

    The mean change in the MG-ADLscore from Baseline at any timepoint after treatment. The MG-ADL is an 8-item patient-reported outcome measure assessing GMG symptoms and functional activities related to activities of daily living and producing a total score ranging from 0 to 24, where higher scores indicate greater severity of disease symptoms.

  6. Mean change in MCG score

    Time frame: 24 months

    The mean change in the MGC score from Baseline at any timepoint after treatment. The MGC is a 10-item assessment that measures signs and symptoms of GMG based on physical examination findings and patient history. The total score ranges from 0 to 50, where higher scores indicate more severe impairment.

  7. Mean change in MG-QoL15R score

    Time frame: 24 months

    The mean change in the MG QoL15R score from Baseline at any timepoint after treatment. The MG-QoL15 is a 15-item questionnaire that allows clinicians to estimate a patient's quality of life relevant to GMG. The cumulative scores range from 0 to 60, with higher scores representing decreased quality of life.

  8. Change in titer of myasthenia specific autoantibodies

    Time frame: 24 months

    The proportion of subjects achieving ≥50% reduction in myasthenia-specific autoantibody titers from Baseline at any timepoint after treatment

  9. PK Parameter for anito-cel: Cmax

    Time frame: Day 1 up to 24 months

    Cmax is defined as the maximum observed concentration of anitocel measured/quantified using vector copy number (VCN) on peripheral blood mononuclear cells

  10. PK Parameter for anito-cel: Tmax

    Time frame: Day 1 up to 24 months

    Tmax is defined as the time (observed time point) of Cmax of anitocel measured/quantified using vector copy number (VCN) on peripheral blood mononuclear cells

  11. PK Parameter for anito-cel: Area under the curve (AUC)

    Time frame: Day 1 up to 24 months

    AUC is a measure of the anitocel concentration in the blood measured/quantified using VCN on peripheral blood mononuclear cells over time.

Study contacts

Contact information is provided by the study sponsor or research team.

Clinical Information

CONTACT

[email protected]

240-327-0379

Sponsors and collaborators

Lead sponsor

Arcellx, Inc.

Industry

Registry information

Important dates

Study start
2025
Primary completion
2027
Study completion
2028
First posted
Oct 4, 2024
Registry last updated
Feb 25, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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