Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12625000707460
Ethics application status
Approved
Date submitted
2/06/2025
Date registered
3/07/2025
Date last updated
3/07/2025
Date data sharing statement initially provided
3/07/2025
Type of registration
Prospectively registered

Titles & IDs
Public title
Study of Kite-363 in participants with difficult to treat autoimmune diseases, evaluating efficacy and safety.
Scientific title
A Phase 1, Open-Label, Multiregional, Multicenter, Basket Study Evaluating the Safety & Efficacy of Kite-363, an Autologous Anti-CD19-CD20 CAR T-Cell Therapy in Participants with Refractory Autoimmune Diseases
Secondary ID [1] 314580 0
KT-US-720-0203
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Systemic Lupus Erythematosus, with or without lupus nephritis 337686 0
Systemic Sclerosis 337687 0
Idiopathic Inflammatory Myopathy 337688 0
Condition category
Condition code
Inflammatory and Immune System 334023 334023 0 0
Autoimmune diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
This is an open-label, basket study meaning this study includes participants who have been diagnosed with different illnesses but who will all receive the same treatment and have the same outcomes assessed. Safety and efficacy of Kite-363 will be evaluated in participants with Systemic Lupus Erythematosus with or without Lupus Nephritis; Systemic Sclerosis, or Idiopathic Inflammatory Myopathies.

Participants will firstly undergo leukapheresis to obtain leukocytes, from which the participant’s T cells will be used to manufacture KITE-363.
Participants will receive lymphodepleting chemotherapy with cyclophosphamide and fludarabine prior to infusion of KITE-363.
Treatment will consist of a single infusion of CAR-transduced autologous T cells (KITE-363) administered intravenously.
The dose escalation levels for Kite 363 are as follows: 0.2 x 106, 0.5 x 106, 1.0 x 106 Kite-363 Cells/kg.
Both the first and second cohorts of participants may include participants from any of the indicated diseases mentioned above.
Up to 12 participants will be enrolled in the first cohort and will be treated at sequential dose-escalation levels and assessed for dose limiting toxicities (DLTs). Administration of Kite-363 will be staggered to allow for observation of toxicities.
4 weeks after the first cohort is dosed with 0.2 x 106 Kite-363 Cells/kg, if the dose limiting toxicities observed do not cross the study-specific thresholds then the next dose level (0.5 x 106 Kite-363 Cells/kg) cohorts will be dosed. Similarly, the 1.0 x 106 Kite-363 Cells/kg dose will be administered 4 weeks after the second dose level if dose limiting toxicities are not observed.
Based on dose limiting toxicities (DLTs) observed in the first cohort of participants and in assessment of the recommended KITE-363 dose, additional participants will be enrolled and administered escalating doses of KITE-363 to further characterise the efficacy and safety profile. During this dose-expansion portion of the study, indication specific cohorts will also be used.
Participants will be dosed at treatment centres, by medical staff specially trained on the product.
Intervention code [1] 331205 0
Treatment: Drugs
Comparator / control treatment
No control group
Control group
Uncontrolled

Outcomes
Primary outcome [1] 341680 0
To assess the efficacy of KITE-363 on autoimmune disease activity - Systemic Lupus Erythematosus with or without Lupus Nephritis
Timepoint [1] 341680 0
6 months post-dose
Primary outcome [2] 341683 0
To evaluate the safety of KITE-363 in participants with autoimmune disease
Timepoint [2] 341683 0
There are 2 years of assessment in the study, All participants will be assessed daily for first 7 days post dose, once at 2 weeks post dose, monthly until 6 months post dose, then quarterly until month 24 post dose.
Secondary outcome [1] 448836 0
To evaluate pharmacodynamics of Kite-363
Timepoint [1] 448836 0
B-cell levels will be measured pre and post dose, then on days 7, 14 and 28 post dose, then monthly until month 6 post dose, then measured every 6 months until 24 months post dose.
Secondary outcome [2] 448837 0
This is another primary outcome - to assess efficacy of Kite-363 on Idiopathic Inflammatory Myopathy
Timepoint [2] 448837 0
6 months post dose
Secondary outcome [3] 449104 0
Levels of disease specific and associated autoantibodies, inflammatory and immune-modulatory markers, including cytokines and chemokines.
Timepoint [3] 449104 0
Analytes, including cytokines and chemokines will be measured pre and post dose, then on days 1, 3 and 7 post dose, 2 weeks post dose, monthly until 6 months post dose, then at 12 months post dose, 18 months post dose and 24 months post dose.

Eligibility
Key inclusion criteria
Inclusion criteria for Systemic Lupus Erythematosus (SLE) and Lupus Nephritis (LN):
1) Aged between 18 and 75 years
2) Meet the EULAR-ACR 2019 classification criteria for SLE
3) 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.

Inclusion Criteria for Systemic sclerosis:
1) Age between 18 and 60 years old
2) Systemic sclerosis according to ACR/EULAR 2013 classification criteria
3) Disease duration greater than 5 years
4)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

Inclusion Criteria for Idiopathic inflammatory myopathy (IIM):
1) Aged 18 years or over
2) Idiopathic inflammatory myopathy (IIM) based on EULAR/ACR 2017 classification
3) Active disease by electromyography (EMG) or magnetic resonance imaging (MRI) within 3 months of lymphodepletion
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1) Females of childbearing potential who are pregnant or breastfeeding because of the potentially dangerous effects of the preparative chemotherapy on the fetus or infant.
2) Unstable cardiac disease within 12 months before enrollment:
A) Cardiac ejection fraction < 50% via echocardiogram (ECHO) or multigated acquisition
scan (MUGA)
B) Haemodynamic instability secondary to large pericardial effusion or cardiac tamponade
C) Unstable arrythmia, valvular dysfunction, myocarditis, coronary vasculitis, or clinically
significant electrocardiogram (ECG) findings, and/or
D) Myocardial infarction, cardiac angioplasty or stenting, unstable angina
3) Clinically significant pulmonary disease within 12 months before enrollment:
A) Baseline oxygen saturation < 92% on room air
B) Pulmonary hemorrhage
4) eGFR < 40 mL/min/1.73 m2 using the Chronic Kidney Disease (CKD) Epidemiology
Collaboration (CKD-EPI) formula at screening
5) Dialysis within the past year
6) History of symptomatic deep vein thrombosis or pulmonary embolism requiring therapeutic anticoagulation within 6 months before enrollment
7) History of autologous or allogeneic stem cell transplant and/or organ transplant
8) Prior treatment with cellular therapy, gene therapy and/or T-cell engager therapy

These criteria are not exhaustive, other protocol defined Inclusion/Exclusion criteria may apply. A member of the research team will provide further details at the time of screening and enrolment.

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Other
Other design features
Only one investigational treatment available to participants. As this is a dose escalation study, participants may be assigned to different dose levels.
Phase
Phase 1
Type of endpoint/s
Safety/efficacy
Statistical methods / analysis

Recruitment
Recruitment status
Not yet recruiting
Date of first participant enrolment
Anticipated
30/07/2025
Actual
Date of last participant enrolment
Anticipated
22/06/2027
Actual
Date of last data collection
Anticipated
19/06/2029
Actual
Sample size
Target
52
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment outside Australia
Country [1] 27096 0
United States of America
State/province [1] 27096 0
Country [2] 27097 0
Korea, Republic Of
State/province [2] 27097 0

Funding & Sponsors
Funding source category [1] 319128 0
Commercial sector/Industry
Name [1] 319128 0
Kite, A Gilead Company
Country [1] 319128 0
United States of America
Primary sponsor type
Commercial sector/Industry
Name
Kite, A Gilead Company
Address
Country
United States of America
Secondary sponsor category [1] 321592 0
Commercial sector/Industry
Name [1] 321592 0
Gilead Sciences Pty Ltd
Address [1] 321592 0
Country [1] 321592 0
Australia

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 317722 0
St Vincent's Hospital Melbourne Human Research Ethics Committee
Ethics committee address [1] 317722 0
Ethics committee country [1] 317722 0
Australia
Date submitted for ethics approval [1] 317722 0
17/04/2025
Approval date [1] 317722 0
Ethics approval number [1] 317722 0
Ethics committee name [2] 317725 0
Bellberry Human Research Ethics Committee A
Ethics committee address [2] 317725 0
Ethics committee country [2] 317725 0
Australia
Date submitted for ethics approval [2] 317725 0
01/04/2025
Approval date [2] 317725 0
13/05/2025
Ethics approval number [2] 317725 0

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 141934 0
Dr Janlyn Falconer
Address 141934 0
Concord Hospital, Hospital Road, Concord, NSW 2139
Country 141934 0
Australia
Phone 141934 0
+612 9767 6233
Fax 141934 0
Email 141934 0
[email protected]
Contact person for public queries
Name 141935 0
Janlyn Falconer
Address 141935 0
Concord Hospital, Hospital Road, Concord, NSW 2139
Country 141935 0
Australia
Phone 141935 0
+61297675000
Fax 141935 0
Email 141935 0
[email protected]
Contact person for scientific queries
Name 141936 0
Ryan Capps
Address 141936 0
Kite Pharma Inc, 2400 Broadway, Santa Monica, CA 90404
Country 141936 0
United States of America
Phone 141936 0
+18444545483
Fax 141936 0
Email 141936 0
[email protected]

Data sharing statement
Will the study consider sharing individual participant data?
No


What supporting documents are/will be available?

No Supporting Document Provided


Results publications and other study-related documents

Documents added manually
No documents have been uploaded by study researchers.

Documents added automatically
No additional documents have been identified.