A Phase 1 interventional study of anito-cel and Cyclophosphamide in Muscular Diseases, Neuromuscular Manifestations and Autoimmune, sponsored by Kite, A Gilead Company. Recruiting at 14 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-10-22.
Sponsored by Kite, A Gilead Company (part of Gilead Sciences) · Phase 1, Interventional, and Treatment
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.
This is a Phase 1 open-label, multi-center safety and dose-escalation study of anito-cel in adult participants 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 participants 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.
477 studies on the registry are indexed under Muscle Weakness; 73 are open to participants now.
This study's planned enrollment of 30 is below the median of 44 across 362 interventional studies indexed under Muscle Weakness.
Browse Muscle Weakness studies →Kite, A Gilead Company is the lead sponsor of 40 studies on the registry; 8 are open to participants now.
Of its 19 completed or terminated interventional studies of FDA-regulated products, 15 (79%) have results posted.
First submitted after Kite became part of Gilead Sciences.
Compared within Muscle Weakness, this study's primary condition as ClinicalTrials.gg classifies it; the registry's first condition term is Muscular Diseases. The counts are of registry records indexed under each term, refreshed daily. How we classify →
The criteria were revised on the registry on Oct 22, 2026. See what changed
Key Inclusion Criteria:
Key Exclusion Criteria:
Note: Other protocol defined Inclusion/Exclusion criteria may apply.
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participant will receive lymphodepleting chemotherapy with cyclophosphamide and fludarabine followed by a single IV infusion of anito-cel. Anito-cel will be given at one of several possible doses in dose escalation until the optimal dose is identified.
Biological: anito-cel · Drug: Cyclophosphamide · Drug: Fludarabine
Participant will receive lymphodepleting chemotherapy with cyclophosphamide and fludarabine followed by a single IV infusion of anito-cel. Anito-cel will be given at the optimal dose identified in dose escalation.
Biological: anito-cel · Drug: Cyclophosphamide · Drug: Fludarabine
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
Administered intravenously
Administered intravenously
Percentage of Participants Experiencing Treatment-Emergent Adverse Events (TEAE)
Time frame: First dose date up to 24 months
Percentage of Participants Experiencing Dose-Limiting Toxicities (DLT)
Time frame: Up to 28 days following anito-cel infusion
Maximum Tolerated Dose (MTD) of Anito-cel
Time frame: First dose up to 30 days (Up to 24 months)
Percentage of Participants With Laboratory Abnormalities
Time frame: First dose date up to 24 months
Recommended Phase 2 Dose (RP2D) of Anito-cel
Time frame: Up to 24 months
Proportion of Participants Achieving a ≥2-Point Change in the Myasthenia Gravis Activities of Daily Living (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.
Time frame: Baseline, Up to 24 months
Proportion of Participants Achieving a ≥3-Point Change in the Quantitative Myasthenia Gravis (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.
Time frame: Baseline, Up to 24 months
Proportion of Participants Achieving a ≥3-Point Change in the Myasthenia Gravis Composite (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.
Time frame: Baseline, Up to 24 months
Mean Change in Myasthenia Gravis Activities of Daily Living (MG-ADL) Score From Baseline at any Timepoint After Treatment
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.
Time frame: Baseline, Up to 24 months
Mean Change in Quantitative Myasthenia Gravis (QMG) Score From Baseline at any Timepoint After Treatment
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.
Time frame: Baseline, Up to 24 months
Mean Change in Myasthenia Gravis Quality of Life (MG-QoL15R) Score From Baseline at any Timepoint After Treatment
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.
Time frame: Baseline, Up to 24 months
Mean Change in Myasthenia Gravis Composite (MCG) Score From Baseline at any Timepoint After Treatment
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.
Time frame: Baseline, Up to 24 months
Proportion of Participants not Requiring Non-Steroidal Treatments, Including Immunosuppressive Therapies and the Duration of Off-Treatment Period After Baseline
Time frame: Up to 24 months
Proportion of Participants Discontinuing Corticosteroids, and the Duration of Off-Treatment Period After Baseline
Time frame: Up to 24 months
Proportion of Participants Achieving Minimal Manifestation Status (MMS)
Time frame: Up to 24 months
Proportion of Participants Achieving ≥50% Reduction in Myasthenia-Specific Autoantibody Titers From Baseline at any Timepoint After Treatment.
Time frame: Baseline, Up to 24 months
Pharmacokinetics (PK) Parameters: Cmax of Anito-cel
Cmax is defined as the maximum observed concentration of anito-cel measured/quantified using vector copy number (VCN) on peripheral blood mononuclear cells
Time frame: Up to 24 months
PK Parameters: Tmax of Anito-cel
Tmax is defined as the time (observed time point) of Cmax of anito-cel measured/quantified using vector copy number (VCN) on peripheral blood mononuclear cells
Time frame: Up to 24 months
PK Parameters: Area Under the Curve (AUC) of Anito-cel
AUC is a measure of the anito-cel concentration in the blood measured/quantified using VCN on peripheral blood mononuclear cells over time.
Time frame: Up to 24 months
Plan to share: No
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
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Studies of the drug tested here: Anitocabtagene autoleucel