Muscular DiseasesPhase 1NCT06626919
Study of Anitocabtagene Autoleucel in Participants With Non-Oncology Plasma Cell-Related Diseases
A Phase 1 study testing anito-cel, Cyclophosphamide and Fludarabine in people with Muscular Diseases and Neuromuscular Manifestations, run by Kite, A Gilead Company at 14 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Muscular Diseases, Neuromuscular Manifestations, Autoimmune
- What is being tested?
- anito-cel, Cyclophosphamide and Fludarabine
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 14 sitesUnited States
- Study dates
- 30 Apr 2025to Oct 2028
- Phase
- Phase 1
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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)
Who cannot join
- 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.
Trial locations and contacts
14 sites in 1 country. Contact the study team or a site near you to ask about taking part.
14 sites in 1 country
United States14 sites, 14 recruiting
- Recruiting
The Ohio State University Wexner Medical Center
Columbus, Ohio, 43210
- Recruiting
UT Southwestern Medical Center
Dallas, Texas, 75390
- Recruiting
Karmanos Cancer Institute
Detroit, Michigan, 48201
- Recruiting
Houston Methodist Hospital
Houston, Texas, 77030
- Recruiting
Ronald Reagan UCLA Medical Center
Los Angeles, California, 90095
- Recruiting
University of Minnesota Delaware Clinical Research Unit
Minneapolis, Minnesota, 55455
- Recruiting
Columbia University Irving Medical Center
New York, New York, 10032
- Recruiting
UC Irvine Medical Center
Orange, California, 92868
- Recruiting
Stanford Blood and Marrow Transplant
Palo Alto, California, 94305
- Recruiting
Lewis Katz School of Medicine at Temple University
Philadelphia, Pennsylvania, 19140
- Recruiting
Oregon Health & Science University
Portland, Oregon, 97239
- Recruiting
Mayo Clinic
Rochester, Minnesota, 55902
- Recruiting
Moffitt Cancer Center
Tampa, Florida, 33612
- Recruiting
University of South Florida - Carol and Frank Morsani Center for Advanced Healthcare
Tampa, Florida, 33612
About this study
Official title: A Phase 1 Study of Anitocabtagene Autoleucel for the Treatment of Subjects With Non-Oncology Plasma Cell-Related Diseases
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.
Read the detailed description
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.
- Conditions
- Muscular Diseases, Neuromuscular Manifestations, Autoimmune, Autoimmune Diseases, Autoimmune Diseases of the Nervous System, Myasthenia Gravis
- Being tested
- anito-cel, Cyclophosphamide, Fludarabine
- 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
- Medical subject headings
- Muscular Diseases, Neuromuscular Manifestations, Autoimmune Diseases, Autoimmune Diseases of the Nervous System, Myasthenia Gravis, Muscle Weakness
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
anito-celBiological
Also called anitocabtagene autoleucel, CART-ddBCMA
Anitocabtagene autoleucel BCMA directed CAR T-cell therapy using a novel, synthetic binding domain, called a D-Domain
CyclophosphamideDrug
Administered intravenously
FludarabineDrug
Administered intravenously
| Study group | Role | What they receive |
|---|---|---|
| Anito-cel (Dose Escalation) | Experimental | 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. Receives: Biological: anito-cel; Drug: Cyclophosphamide; Drug: Fludarabine |
| Anito-cel (Dose Expansion) | Experimental | 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. Receives: Biological: anito-cel; Drug: Cyclophosphamide; Drug: Fludarabine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants Experiencing Treatment-Emergent Adverse Events (TEAE)
Measured: First dose date up to 24 months
Percentage of Participants Experiencing Dose-Limiting Toxicities (DLT)
Measured: Up to 28 days following anito-cel infusion
Maximum Tolerated Dose (MTD) of Anito-cel
Measured: First dose up to 30 days (Up to 24 months)
Percentage of Participants With Laboratory Abnormalities
Measured: First dose date up to 24 months
Recommended Phase 2 Dose (RP2D) of Anito-cel
Measured: Up to 24 months
Secondary outcomes (14)
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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
Measured: Up to 24 months
Proportion of Participants Discontinuing Corticosteroids, and the Duration of Off-Treatment Period After Baseline
Measured: Up to 24 months
Proportion of Participants Achieving Minimal Manifestation Status (MMS)
Measured: Up to 24 months
Proportion of Participants Achieving ≥50% Reduction in Myasthenia-Specific Autoantibody Titers From Baseline at any Timepoint After Treatment.
Measured: 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
Measured: 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
Measured: 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.
Measured: Up to 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Oct 2024
- Study start30 Apr 2025
- Primary completionOct 2028 (estimated)
- Study completionOct 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Muscular DiseasesStudies of the same condition, open and under way.
- More trials run by Kite, A Gilead CompanyThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Kite, A Gilead Company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Kite Study Director, Study director, Kite, A Gilead Company
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06626919
- Sponsor's protocol ID
- ARC-311
- Status last verified
- Oct 2026
- First submitted
- 19 Sep 2024
- Last updated
- 22 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No