A Study Evaluating the Safety and Efficacy of KITE-363 in Relapsed/Refractory Autoimmune Neurologic Diseases

A Phase 1 Open-label, Multiregional, Multicenter, Basket Study Evaluating the Safety and Efficacy of KITE-363, an Autologous Anti-CD19/CD20 CAR T-cell Therapy in Participants With Relapsed/Refractory Autoimmune Neurologic Diseases

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:

• To evaluate the safety and tolerability of KITE-363 in participants with autoimmune neurologic diseases
• To determine the recommended dose for Phase 1b.
• To evaluate the preliminary efficacy of KITE-363 in participants with autoimmune neurologic diseases.

Study Overview

• Status: Recruiting
• Conditions: Multiple Sclerosis; Myasthenia Gravis; Chronic Inflammatory Demyelinating Polyneuropathy
• Intervention / Treatment: Drug: Cyclophosphamide; Drug: Fludarabine; Biological: KITE-363

• Study Type: Interventional
• Enrollment (Estimated): 52
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Medical Information; Phone Number: 844-454-5483(1-844-454-KITE); Email: medinfo@kitepharma.com

Study Locations

• Australia
  • New South Wales
    • Sydney, New South Wales, Australia, 2139
      • Recruiting
      • Concord Repatriation General Hospital
• United States
  • Utah
    • Salt Lake City, Utah, United States, 84143
      • Recruiting
      • LDS Hospital - Intermountain Health
  • Washington
    • Seattle, Washington, United States, 98109
      • Recruiting
      • Fred Hutchinson Cancer Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Key Inclusion Criteria:

• Reproductive status-related eligibility and contraception requirements:

  • Participants must agree to use protocol-specified method(s) of contraception where applicable

Inclusion Criteria for multiple sclerosis (MS):

MS (Relapsing and progressive forms):

• Diagnosed with MS according to the 2017 revision of the McDonald diagnostic criteria

Relapsing forms of MS (relapsing-remitting multiple sclerosis (RRMS), active secondary-progressive multiple sclerosis (aSPMS)):

• Inadequate response to previous therapies is defined as evidence of breakthrough disease activity within 12 months prior to screening while on high efficacy disease-modifying therapy (DMT) OR Inadequate response to previous therapies defined as intolerance to ≥ 2 DMTs due to side effects prohibiting the chronic use of the DMT.
• Expanded Disability Status Scale (EDSS) 0 to 5.5

Progressive forms of MS (primary-progressive multiple sclerosis (PPMS) and non-active secondary-progressive multiple sclerosis (naSPMS)):

• Inadequate response to previous therapies is defined as evidence of disease progression within 12 months prior to screening despite standard of care therapy for naSPMS or despite ocrelizumab, where available, for PPMS
• Absence of clinical relapses for at least 24 months
• No evidence of Gadolinium enhancing (GadE+) on magnetic resonance imaging (MRI) brain at screening or baseline
• EDSS of 3 to 6.5 who are ambulatory

Inclusion Criteria for myasthenia gravis (MG):

• Documentation of autoantibodies against acetylcholine receptor (AChR), muscle-specific kinase (MuSK), or low-density lipoprotein receptor-related protein 4 (LRP4)
• Diagnosis of MG with generalized weakness meeting criteria as defined by the Myasthenia Gravis Foundation of American (MGFA) classification of II- IV at screening
• Myasthenia Gravis Activities of Daily Living (MG-ADL) score ≥ 6 (> 50% of the total score due to non-ocular symptoms)
• Quantitative Myasthenia Gravis (QMG) score ≥ 10
• Inadequate response to previous therapies while taking at least 2 classes of immunosuppressants (ie, steroids, azathioprine (AZA), mycophenolate mofetil (MMF), intravenous immunoglobulin (IVIg), biologics (eg, rituximab, anti-neonatal fragment crystallizable (Fc) receptor (FcRN) class, and anti-complement class))
• Thymectomy allowed if completed ≥ 12 months prior to screening

Inclusion Criteria for chronic inflammatory demyelinating polyneuropathy (CIDP):

• Probable or definite CIDP as defined by the 2010 European Federation of Neurological Societies/Peripheral Nerve Society (EFNS/PNS) criteria, relapsing or progressive forms
• CIDP Disease Activity Status (CDAS) score ≥ 3 at screening
• Inflammatory neuropathy cause and treatment (INCAT) score ≥ 3
• Inadequate response to previous therapies despite standard of care therapy (ie, steroids, IVIg, subcutaneous immunoglobulin (SCIg), plasmapheresis exchange (PLEX), rituximab, or anti FcRN) OR Unable to tolerate standard of care due to side effects with ongoing disease activity
• Except for nodal/paranodal CIDP, historical documentation of objective improvement in the past 24 months while on IVIg, SCIg, PLEX, or anti-FcRN OR Historical documentation of objective disease worsening in the past 24 months when IVIg, SCIg, PLEX, or anti-FcRN has been reduced or interrupted

Key Exclusion Criteria:

• History or presence of central nervous system (CNS) or peripheral nervous system disorders before enrollment that may impact cognition, strength, or cause weakness
• History of autologous or allogeneic stem cell transplant and/or organ transplant

Exclusion Criteria for MS:

• Cohort 1 or 2; inability to complete 9-hole Peg Test (9-HPT) in < 240 seconds and Timed 25 foot Walk (T25FW) < 150 seconds
• History of hypersensitivity to parenteral administration of gadolinium-based contrast agents
• Any renal condition that would preclude the administration of gadolinium (for the relapsing forms of MS and progressive forms of MS)
• Any contraindication to lumbar puncture (LP) (for the relapsing forms of MS and progressive forms of MS)

Exclusion Criteria for MG:

• Current myasthenic crisis not effectively controlled within 2 weeks before enrollment
• Thymectomy performed within 12 months of baseline

Exclusion Criteria for CIDP:

• Pure sensory CIDP and focal CIDP
• Polyneuropathy of other causes

Note: Other protocol defined Inclusion/Exclusion criteria may apply.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Phase 1a: KITE-363 (Dose Escalation); 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.	Drug: Cyclophosphamide; Administered intravenously; Drug: Fludarabine; Administered intravenously; Biological: KITE-363; A single infusion of CAR-transduced autologous T cells administered as intravenous infusion.
Experimental: Phase 1b: KITE-363 (Dose Expansion); 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.	Drug: Cyclophosphamide; Administered intravenously; Drug: Fludarabine; Administered intravenously; Biological: KITE-363; A single infusion of CAR-transduced autologous T cells administered as intravenous infusion.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	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	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

Secondary Outcome Measures

Outcome Measure	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		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
Proportion of Participants With of Antibodies Against the KITE-363 CAR T cells 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

Collaborators and Investigators

• Sponsor: Kite, A Gilead Company
• Investigators: Study Director: Kite Study Director, Kite, A Gilead Company

Publications and helpful links

Helpful Links

• Gilead Clinical Trials Website

Study record dates

Study Major Dates

• Study Start (Actual): April 10, 2026
• Primary Completion (Estimated): June 1, 2029
• Study Completion (Estimated): June 1, 2029

Study Registration Dates

• First Submitted: December 12, 2025
• First Submitted That Met QC Criteria: December 12, 2025
• First Posted (Actual): December 26, 2025

Study Record Updates

• Last Update Posted (Actual): April 21, 2026
• Last Update Submitted That Met QC Criteria: April 16, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Pathologic Processes; Neoplasms by Site; Neoplasms; Neuromuscular Diseases; Chronic Disease; Disease Attributes; Autoimmune Diseases; Immune System Diseases; Peripheral Nervous System Diseases; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Demyelinating Diseases; Neurodegenerative Diseases; Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Paraneoplastic Syndromes; Neuromuscular Junction Diseases; Polyneuropathies; Polyradiculoneuropathy; Pathological Conditions, Signs and Symptoms; Multiple Sclerosis; Myasthenia Gravis; Polyradiculoneuropathy, Chronic Inflammatory Demyelinating; Organic Chemicals; Hydrocarbons; Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Phosphoramides; Organophosphorus Compounds; Cyclophosphamide; fludarabine
• Other Study ID Numbers: KT-US-728-0204

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No