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This study will have two Phases: Phase 1a and Phase 1b. The goals of this clinical study are to learn more about the study drug KITE-363, by evaluating its safety, tolerability and efficacy in participants with relapsed/refractory autoimmune neurologic diseases.
The primary objectives of this study are:
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Phase 1a: KITE-363 (Dose Escalation) | Experimental | Participants with relapsing forms of multiple sclerosis (RMS), progressive forms of multiple sclerosis (PMS), myasthenia gravis (MG), and/or chronic inflammatory demyelinating polyneuropathy (CIDP) will receive lymphodepleting chemotherapy with cyclophosphamide and fludarabine followed by infusion of KITE-363 chimeric antigen receptor (CAR) transduced autologous T cells at a single dose level, with different participants receiving sequential dose-escalation levels to find the Phase 1b recommended dose. |
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| Phase 1b: KITE-363 (Dose Expansion) | Experimental | Participants with RMS, PMS, MG, and/or CIDP will receive lymphodepleting chemotherapy with cyclophosphamide and fludarabine followed Phase 1a recommended dose of KITE-363 CAR T cells. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| KITE-363 | Biological | A single infusion of CAR-transduced autologous T cells administered as intravenous infusion. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Phase 1a: Percentage of Participants Experiencing Treatment-emergent Adverse Event (TEAEs) | Up to 2 years | |
| Phase 1a: Percentage of Participants Experiencing Dose-limiting Toxicities (DLTs) After the Infusion of KITE-363 | Up to 28 days | |
| Phase 1b: (All Cohorts) Percentage of Participants Experiencing TEAEs | Up to 2 years | |
| Phase 1b: Relapsing Forms of MS (RRMS) and (aSPMS): Number of New T1 Gadolinium Enhancing (GadE+) Lesions on Magnetic Resonance Imaging (MRI) at Week 12 | This will be reported in participants with relapsing forms of Multiple Sclerosis MS (RRMS) and (aSPMS). | Week 12 |
| Phase 1b: Relapsing Forms of MS (RRMS and aSPMS): Number of New and/or Enlarging T2 Lesions on MRI at Week 12 | Week 12 | |
| Phase 1b: Progressive Forms of MS (PPMS) and (naSPMS): Time to Onset of Confirmed Disability Progression Over 12 Weeks (CDP-12) | Up to 2 years | |
| Phase 1b: Myasthenia Gravis (MG): Proportion of Participants of MG Activities of Daily Living (MG-ADL) Responders | The MG-ADL is a scale to measure the functional impact of MG on daily activities. The total score ranges from 0 to 24, with higher scores indicating greater disability and disease burden. | Up to Week 24 |
| Phase 1b: Chronic Inflammatory Demyelinating Polyneuropathy (CIDP): Proportion of Participants with Confirmed Evidence of Clinical Improvement at Week 24 |
| Measure | Description | Time Frame |
|---|---|---|
| Relapsing forms of MS (RRMS and aSPMS): Annual Relapse Rate | Proportion of participants with annual relapse. | Up to 2 years |
| Relapsing Forms of MS (RRMS and aSPMS): Proportion of Participants With CDP-12 |
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Key Inclusion Criteria:
Reproductive status-related eligibility and contraception requirements:
Inclusion Criteria for multiple sclerosis (MS):
MS (Relapsing and progressive forms):
Relapsing forms of MS (relapsing-remitting multiple sclerosis (RRMS), active secondary-progressive multiple sclerosis (aSPMS)):
Progressive forms of MS (primary-progressive multiple sclerosis (PPMS) and non-active secondary-progressive multiple sclerosis (naSPMS)):
Inclusion Criteria for myasthenia gravis (MG):
Inclusion Criteria for chronic inflammatory demyelinating polyneuropathy (CIDP):
Key Exclusion Criteria:
Exclusion Criteria for MS:
Exclusion Criteria for MG:
Exclusion Criteria for CIDP:
Note: Other protocol defined Inclusion/Exclusion criteria may apply.
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Medical Information | Contact | 844-454-5483(1-844-454-KITE) | medinfo@kitepharma.com |
| Name | Affiliation | Role |
|---|---|---|
| Kite Study Director | Kite, A Gilead Company | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Stanford Neuroscience Health Center | Recruiting | Palo Alto | California | 94304 | United States | |
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| Label | URL |
|---|---|
| Gilead Clinical Trials Website | View source |
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| Fludarabine | Drug | Administered intravenously |
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| Cyclophosphamide | Drug | Administered intravenously |
|
Clinical improvement will be analyzed using inflammatory neuropathy cause and treatment (INCAT) scale. The INCAT score is a clinician administered tool used to assess functional disability in participants with CIDP. The total scores range from 0 to 10, higher scores indicating greater disability and lower score would indicate clinical improvement. |
| Week 24 |
| Up to 2 years |
| Relapsing Forms of MS (RRMS and aSPMS): Proportion of Participants With CDP-24 | Up to 2 years |
| Relapsing Forms of MS (RRMS and aSPMS): Time to Onset of CDP-12 | Up to 2 years |
| Relapsing Forms of MS (RRMS and aSPMS): Time to Onset of CDP-24 | Up to 2 years |
| Progressive forms of MS (PPMS and naSPMS): Proportion of Participants with No Evidence of Disease Activity (NEDA) | Up to 2 years |
| Relapsing Forms of MS (RRMS and aSPMS): Change From Baseline in Expanded Disability Status Scale (EDSS) Score Over Time | EDSS is scale used to measure disability and disease progression in participants with MS. It ranges from 0 (normal neurological exam) to 10 (death due to MS) in 0.5-point increments. | Up to 2 years |
| Relapsing Forms of MS (RRMS and aSPMS): Change From Baseline in Timed 25-foot Walk (T25FW) Score Over Time | The T25FW is a performance-based measure used in people with MS to assess walking speed and mobility. Lower times indicate better walking ability, while longer times reflect greater impairment. | Up to 2 years |
| Relapsing Forms of MS (RRMS and aSPMS): Change From Baseline in 9-hole Peg Test Dominant/Non-dominant (9-HPT D/ND) Score Over Time | The 9-HPT assesses hand dexterity and fine motor function by timing how long it takes a participant to place and remove nine pegs from a board. Faster times indicate better function, while slower times reflect greater disability. | Up to 2 years |
| Relapsing Forms of MS (RRMS and aSPMS): Change From Baseline in Symbol Digit Modalities Test (SDMT) Score Over Time | SDMT is a performance-based cognitive assessment to measure information processing speed. The score is the number of correct responses completed in the time limit, with higher scores indicating better cognitive processing speed. | Up to 2 years |
| Progressive Forms of MS (PPMS and naSPMS): Proportion of Participants with CDP-12 | Up to 2 years |
| Progressive Forms of MS (PPMS and naSPMS): Proportion of Participants with CDP-24 | Up to 2 years |
| Progressive forms of MS (PPMS and naSPMS): Time to Onset of CDP-24 | Up to 2 years |
| Progressive forms of MS (PPMS and naSPMS): Number of new and Enlarging T2 Lesions on MRI | Up to 2 years |
| Relapsing forms of MS (RRMS and aSPMS): Percentage of Participants With NEDA | Up to 2 years |
| Progressive forms of MS (PPMS and naSPMS): Change From Baseline in EDSS Over Time | EDSS is scale used to measure disability and disease progression in participants with MS. It ranges from 0 (normal neurological exam) to 10 (death due to MS) in 0.5-point increments. | Up to 2 years |
| Progressive forms of MS (PPMS and naSPMS): Change From Baseline in T25FW Over Time | The T25FW is a performance-based measure used in people with MS to assess walking speed and mobility. Lower times indicate better walking ability, while longer times reflect greater impairment. | Up to 2 years |
| Progressive forms of MS (PPMS and naSPMS): Change From Baseline in 9-HPT D/ND Over Time | The 9-HPT assesses hand dexterity and fine motor function by timing how long it takes a participant to place and remove nine pegs from a board. Faster times indicate better function, while slower times reflect greater disability. | Up to 2 years |
| Progressive forms of MS (PPMS and naSPMS): Change From Baseline in SDMT Over Time | SDMT is a performance-based cognitive assessment to measure information processing speed. The score is the number of correct responses completed in the time limit, with higher scores indicating better cognitive processing speed. | Up to 2 years |
| MG: Proportion of Participants With at Least a 2-Point or 5 Point Improvement in MG-ADL Score up to Week 24 | The MG-ADL is a scale to measure the functional impact of MG on daily activities. The total score ranges from 0 to 24, with higher scores indicating greater disability and disease burden. | Up to 24 weeks |
| MG: Change From Baseline in MG-ADL Score at Week 24 | The MG-ADL is a scale to measure the functional impact of MG on daily activities. The total score ranges from 0 to 24, with higher scores indicating greater disability and disease burden. | Baseline, Week 24 |
| MG: Proportion of Participants With Minimal Symptom Expression (MG-ADL Score of 0 or 1 Point) | The MG-ADL is a scale to measure the functional impact of MG on daily activities. The total score ranges from 0 to 24, with higher scores indicating greater disability and disease burden. | Up to 2 years |
| MG: Proportion of Participants With at Least a 3-point or 5-point Improvement in Medical Research Council (QMG) Score up to Week 24 | The QMG test is a standardized quantitative strength scoring system developed specifically for MG. The total QMG score is the sum of the scores for 13 items, with a possible maximum score of 39. A higher score indicates more severe disease involvement, while a lower score suggests less functional impairment. | Baseline, Week 24 |
| MG: Change From Baseline in QMG Score to Week 24 | The QMG test is a standardized quantitative strength scoring system developed specifically for MG. The total QMG score is the sum of the scores for 13 items, with a possible maximum score of 39. A higher score indicates more severe disease involvement, while a lower score suggests less functional impairment. | Baseline, Week 24 |
| MG: Proportion of Participants With at Least a 3-Point Improvement in MG-C Score up to Week 24 | MG-C scale is a tool that measures disease severity in MG. The total score ranges from 0 to 50, with higher scores indicating greater impairment. | Baseline, Up to 24 weeks |
| Change From Baseline in MG-C Scale Score at Week 24 | MG-C scale is a tool that measures disease severity in MG. The total score ranges from 0 to 50, with higher scores indicating greater impairment. | Baseline, Week 24 |
| MG: Number of Participants With Changes in Myasthenia Gravis Foundation of American Post-Intervention Status (MGFA-PIS), Including Minimal Manifestation, Complete Stable Remission, Pharmacologic Remission | The MGFA-PIS is a standardized classification system which categorizes outcomes into distinct states such as complete stable remission (CSR), pharmacologic remission (PR), minimal manifestations (MM), improved, unchanged, worse, and exacerbation. CSR indicates no symptoms or signs for at least one year without therapy, while MM reflects minimal weakness without functional limitation. | Up to 2 years |
| CIDP: Time to First Adjusted INCAT Deterioration | INCAT is a clinician-administered tool used to assess functional disability in participants with CIDP. The total score ranges from 0 to 10, where higher scores indicate greater disability and lower score would indicate clinical improvement. | Up to 2 years |
| CIDP: Proportion of Participants With Confirmed Evidence of Clinical Improvement | Clinical improvement will be analyzed using INCAT scale. The INCAT score is a clinician administered tool used to assess functional disability in participants with CIDP. The total scores range from 0 to 10, higher scores indicating greater disability and lower score would indicate improvement. | Week 48 |
| CIDP: Proportion of Participants With Evidence of Clinical Improvement | Clinical improvement will be analyzed using inflammatory Rasch-built overall disability scale (I-RODS) scale. I-RODS is a scale that assesses activity and participation limitations in people with CIDP. The score ranges from 0 to 48, with higher scores indicating better functional ability. | Up to week 48 |
| CIDP: Time to Disease Progression by I-RODS | I-RODS is a scale that assesses activity and participation limitations in people with CIDP. The score ranges from 0 to 48, with higher scores indicating better functional ability. | Baseline, Up to 48 Weeks |
| CIDP: Change From Baseline Over Time in 24-Item I-RODS Score | I-RODS is a scale that assesses activity and participation limitations in people with CIDP. The score ranges from 0 to 48, with higher scores indicating better functional ability. | Up to 2 years |
| CIDP: Change From Baseline Over Time in Medical Research Council (MRC) Sum Score | MRC Sum Score is a measure to assess muscle strength for people with CIDP. The score ranges from 0 to 60. Higher scores indicate better muscle strength, while lower scores reflect greater weakness. | Up to 2 years |
| CIDP: Change From Baseline Over Time in Adjusted INCAT Score | INCAT is a tool used to assess functional disability in participants with CIDP. The total score ranges from 0 to 10, where higher scores indicate greater disability. | Up to 2 years |
| CIDP: Change From Baseline Over Time in Timed Up and Go (TUG) Score | TUG test is a measure of mobility, balance, and fall risk. The score is recorded in seconds; shorter times indicate better mobility, while longer times suggest impaired balance or gait. | Up to 2 years |
| CIDP: Change From Baseline Over Time in Mean grip strength assessed by Martin Vigorimeter | Mean Grip Strength is a quantitative measure of hand and forearm muscle strength. Higher values indicate better muscle strength, while lower values reflect weakness or functional impairment. | Up to 2 years |
| Levels of T-cells in Blood | Up to 2 years |
| Pharmacokinetics: Levels of Chimeric Antigen Receptor (CAR) T-cells in Blood | Up to 2 years |
| Pharmacodynamics Parameters: Levels of B cells in Blood | Up to 2 years |
| Pharmacodynamics Parameters: Levels of Disease-Specific Biomarkers and Autoantibodies in Blood | Up to 2 years |
| Pharmacodynamics Parameters: Levels of Cytokines and Chemokines in Blood | A panel of inflammatory, immuno-modulatory and effector molecules, including cytokines and chemokines, will be measured as units of concentration in serum (e.g. pg/ml) using ligand-based immunoassays such as MSD, Ella and O-link. | Up to 2 years |
| Proportion of Participants With of Antibodies Against the KITE-363 CAR T cells in Blood | Up to 2 years |
| Columbia University Irving Medical Center |
| Recruiting |
| New York |
| New York |
| 10032 |
| United States |
| LDS Hospital - Intermountain Health | Recruiting | Salt Lake City | Utah | 84143 | United States |
| Fred Hutchinson Cancer Center | Recruiting | Seattle | Washington | 98109 | United States |
| Concord Repatriation General Hospital | Recruiting | Sydney | New South Wales | 2139 | Australia |
| Corner Hawkesbury Road and Darcy Road | Recruiting | Westmead | New South Wales | 2145 | Australia |
| ID | Term |
|---|---|
| D020277 | Polyradiculoneuropathy, Chronic Inflammatory Demyelinating |
| D009157 | Myasthenia Gravis |
| D009103 | Multiple Sclerosis |
| ID | Term |
|---|---|
| D011129 | Polyradiculoneuropathy |
| D020274 | Autoimmune Diseases of the Nervous System |
| D009422 | Nervous System Diseases |
| D003711 | Demyelinating Diseases |
| D011115 | Polyneuropathies |
| D010523 | Peripheral Nervous System Diseases |
| D009468 | Neuromuscular Diseases |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
| D002908 | Chronic Disease |
| D020969 | Disease Attributes |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D020361 | Paraneoplastic Syndromes, Nervous System |
| D009423 | Nervous System Neoplasms |
| D009371 | Neoplasms by Site |
| D009369 | Neoplasms |
| D010257 | Paraneoplastic Syndromes |
| D019636 | Neurodegenerative Diseases |
| D020511 | Neuromuscular Junction Diseases |
| D020278 | Demyelinating Autoimmune Diseases, CNS |
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| ID | Term |
|---|---|
| C024352 | fludarabine |
| D003520 | Cyclophosphamide |
| ID | Term |
|---|---|
| D010752 | Phosphoramide Mustards |
| D009588 | Nitrogen Mustard Compounds |
| D009150 | Mustard Compounds |
| D006846 | Hydrocarbons, Halogenated |
| D006838 | Hydrocarbons |
| D009930 | Organic Chemicals |
| D063088 | Phosphoramides |
| D009943 | Organophosphorus Compounds |
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