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

NCT07304154 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: KT-US-728-0204
• Study Type: interventional
• Target: 52
• Locations: 2 countries
• Sites: 3

Brief Summary

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.

Conditions

Chronic Inflammatory Demyelinating Polyneuropathy; Myasthenia Gravis; Multiple Sclerosis

Outcome Measures

Primary Outcomes (8)

• 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 Outcomes (42)

• Relapsing forms of MS (RRMS and aSPMS): Annual Relapse Rate

  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

Study Arms (2)

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.

Biological: KITE-363; Drug: Fludarabine; Drug: Cyclophosphamide

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.

Biological: KITE-363; Drug: Fludarabine; Drug: Cyclophosphamide

Interventions

KITE-363 BIOLOGICAL

A single infusion of CAR-transduced autologous T cells administered as intravenous infusion.

Phase 1a: KITE-363 (Dose Escalation); Phase 1b: KITE-363 (Dose Expansion)

Fludarabine DRUG

Administered intravenously

Phase 1a: KITE-363 (Dose Escalation); Phase 1b: KITE-363 (Dose Expansion)

Cyclophosphamide DRUG

Administered intravenously

Phase 1a: KITE-363 (Dose Escalation); Phase 1b: KITE-363 (Dose Expansion)

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Reproductive status-related eligibility and contraception requirements:
• Participants must agree to use protocol-specified method(s) of contraception where applicable
• 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
• 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)

You may not qualify if:

• 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
• 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)
• Current myasthenic crisis not effectively controlled within 2 weeks before enrollment
• Thymectomy performed within 12 months of baseline
• Pure sensory CIDP and focal CIDP
• Polyneuropathy of other causes

Sponsors & Collaborators

• Kite, A Gilead Company: lead

Study Sites (3)

LDS Hospital - Intermountain Health

Salt Lake City, Utah, 84143, United States

RECRUITING

Fred Hutchinson Cancer Center

Seattle, Washington, 98109, United States

RECRUITING

Concord Repatriation General Hospital

Sydney, New South Wales, 2139, Australia

RECRUITING

Related Links

• Gilead Clinical Trials Website

MeSH Terms

Conditions

Polyradiculoneuropathy, Chronic Inflammatory Demyelinating; Myasthenia Gravis; Multiple Sclerosis

Interventions

fludarabine; Cyclophosphamide

Condition Hierarchy (Ancestors)

Polyradiculoneuropathy; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Demyelinating Diseases; Polyneuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Demyelinating Autoimmune Diseases, CNS

Intervention Hierarchy (Ancestors)

Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Organic Chemicals; Phosphoramides; Organophosphorus Compounds

Study Officials

• Kite Study Director

  Kite, A Gilead Company

  STUDY DIRECTOR

Central Study Contacts

Medical Information

CONTACT

844-454-5483(1-844-454-KITE); medinfo@kitepharma.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 12, 2025
• First Posted: December 26, 2025
• Study Start: April 10, 2026
• Primary Completion (Estimated): June 1, 2029
• Study Completion (Estimated): June 1, 2029
• Last Updated: April 21, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

AU (1); US (2)