A Study of KITE-363 in Participants With Refractory Autoimmune Diseases

NCT07038447 | Enrolling By Invitation Phase 1 DMC FDA Drug

• 1 other identifier: KT-US-720-0203
• Study Type: interventional
• Target: 52
• Locations: 3 countries
• Sites: 5

Brief Summary

This study will have two Phases: Phase 1a and Phase 1b. The goal of this clinical study is to learn more about the study drug KITE-363, to establish dosing, tolerability, safety, and preliminary efficacy of KITE-363 in participants with refractory autoimmune diseases. The primary objectives of this study are: Phase 1a: To evaluate the safety and tolerability of KITE-363 in participants with autoimmune disease. To determine the recommended dose for Phase 1b. Phase 1b: To evaluate the safety and efficacy of KITE-363 in participants with autoimmune disease.

Conditions

Lupus Nephritis; Systemic Sclerosis; Idiopathic Inflammatory Myopathy; Systemic Lupus Erythematosus

Outcome Measures

Primary Outcomes (7)

• Phase 1a: Percentage of Participants Experiencing Adverse Events Defined as Dose-limiting Toxicities (DLTs) After the Infusion of KITE-363

  Up to 2 years

• Phase 1b: All Cohorts Percentage of Participants Experiencing Treatment-emergent Adverse Event (TEAEs)

  Up to 2 years

• Phase 1b: Systemic lupus erythematosus (SLE): Proportion of participants meeting DORIS remission and Lupus low Disease Activity State (LLDAS) criteria at Month 6

  Month 6

• Phase 1b: Lupus Nephritis (LN): Proportion of Participants Meeting DORIS Remission

  Month 6

• Phase 1b: LN: Proportion of Participants Achieving a Complete Renal Response at Month 6

  Month 6

• Phase 1b: Systemic Sclerosis (SSc) Cohort: Proportion of Participants With Improvement in Disease Activity by the Revised Combined Response Index in Diffuse Cutaneous Systemic Sclerosis (rCRISS) at Month 6

  Month 6

• Phase 1b: Idiopathic Inflammatory Myopathy (IIM): Proportions of Participants Meeting European League Against Rheumatism (EULAR)-American College of Rheumatology (ACR) Moderate and Major response 2016 Criteria in Total Improvement Score (TIS) at Month 6

  Month 6

Secondary Outcomes (11)

• Characterization of Product, Including T-cell Phenotype as Assessed by Percent Change From Baseline in cluster of differentiation 3 (CD3)+ Cells and T Cells

  Baseline up to 2 years

• Pharmacokinetic parameter: Serum Concentration of KITE-363 CAR T-cells

  Up to 2 years

• Pharmacokinetic parameter: Peak Concentration (Cmax) for KITE-363 CAR T-cells

  Up to 2 years

• Pharmacokinetic parameter: AUC for KITE-363 CAR T-cells

  Up to 2 years

• Pharmacokinetic parameter: Time to Peak Serum Concentration (Tmax) for KITE-363 CAR T-cells

  Up to 2 years

Study Arms (2)

Phase 1a: KITE-363 (Dose Escalation)

EXPERIMENTAL

Participants with refractory autoimmune diseases will receive lymphodepleting chemotherapy with cyclophosphamide and fludarabine followed by 2 dose escalations of KITE-363 chimeric antigen receptor (CAR) transduced autologous T cells to find the Phase 1b recommended dose.

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

Phase 1b: KITE-363 (Dose Expansion)

EXPERIMENTAL

Participants with refractory autoimmune diseases will receive lymphodepleting chemotherapy with cyclophosphamide and fludarabine followed Phase 1ab 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 intravenously

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

You may qualify if:

• Age ≥ 18 years
• Meet the European Alliance of Associations for Rheumatology (EULAR)- American College of Rheumatology (ACR) 2019 classification criteria for SLE
• Presence of either double-stranded deoxyribonucleic acid (DNA) anti- double-stranded DNA (anti-dsDNA) and/or anti-Smith antibodies at screening per local laboratory.
• Moderate to severe, active disease defined as at least one British Isles Lupus Assessment Group (BILAG-A) score or 2 BILAG B (excluding constitutional and/or neuropsychiatric organ system).
• Refractory to steroids and inadequate response or intolerance to at least 2 of the following treatments, used for at least 3 months each: cyclophosphamide, mycophenolate mofetil or its derivatives, belimumab, anifrolumab, rituximab, obinutuzumab, methotrexate, azathioprine, cyclosporin, tacrolimus, or voclosporin.
• For LN: Refractory to steroids and inadequate response or intolerance to at least 2 of the following treatments, used for at least 3 months each: cyclophosphamide, mycophenolate mofetil or its derivatives, belimumab, rituximab, obinutuzumab, azathioprine, cyclosporin, tacrolimus, or voclosporin
• Renal biopsy-proven Class III or intravenous (IV) ± V LN according to the revised International Society of Nephrology and Renal Pathology Society (ISN/RPS) criteria within 6 months prior to or during screening
• Evidence of active LN at screening
• Age ≥ 18 years
• Diffuse Systemic Sclerosis (SSc) according to ACR/EULAR 2013 classification criteria with active skin disease and/or progressive SSc-interstitial lung disease (ILD) OR limited SSc with progressive ILD.
• Refractory or intolerance to 1 of the following for a minimum of 3 months and/or contraindication: mycophenolate mofetil or its derivatives, methotrexate, tocilizumab (or other IL-6 inhibitor), rituximab (or other B-cell depleting agent), nintedanib (or other antifibrotic agents), cyclophosphamide.
• High-resolution computer tomography (HRCT) scan and pulmonary function test (PFT) within 3 months prior to screening.
• Age ≥ 18 years
• Active disease demonstrated by electromyography (EMG), magnetic resonance imaging (MRI) or muscle enzymes
• Moderate to severe disease activity

You may not qualify if:

• Females of childbearing potential who are pregnant or breast feeding.
• Dialysis within the past year.
• History of malignancy, within the last 5 years.
• Hypogammaglobulinemia requiring immunoglobulin replacement.
• History of autologous or allogeneic stem cell transplant and/or organ transplant.
• Prior treatment with cellular therapy, gene therapy and/or T-cell engager therapy.
• Known history of HIV infection, or hepatitis B or C virus infections.
• Active or untreated latent tuberculosis (TB).
• Active or uncontrolled infections.
• Nonspecific, overlap, mixed autoimmune diseases not clearly identified into any of the studied cohorts.
• Significant pre-existing damage or rapidly progressive glomerulonephritis (GN).
• Drug-induced SLE.
• Catastrophic antiphospholipid syndrome.
• Thrombotic thrombocytopenic purpura.
• Active or unstable lupus neuropsychiatric manifestations within last 6 months.

Sponsors & Collaborators

• Kite, A Gilead Company: lead

Study Sites (5)

City of Hope

Duarte, California, 91010, United States

Location

Stanford University

Stanford, California, 94305, United States

Location

Concord Repatriation General Hospital

Syndey, New South Wales, 2139, Australia

Location

St Vincent's Hospital

Fitzroy, Victoria, 3065, Australia

Location

Jewish General Hospital

Montreal, H3T1E2, Canada

Location

Related Links

• Gilead Clinical Trials Website

MeSH Terms

Conditions

Lupus Erythematosus, Systemic; Lupus Nephritis; Scleroderma, Systemic; Myositis

Interventions

fludarabine; Cyclophosphamide

Condition Hierarchy (Ancestors)

Connective Tissue Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases; Glomerulonephritis; Nephritis; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Skin Diseases; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases

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

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: June 18, 2025
• First Posted: June 26, 2025
• Study Start: July 2, 2025
• Primary Completion (Estimated): July 1, 2029
• Study Completion (Estimated): July 1, 2029
• Last Updated: March 16, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

CA (1); US (2); AU (2)