Trial Review

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Trial registered on ANZCTR


Registration number
Help: Registration number
ACTRN12625001346460p
Ethics application status
Help: Ethics application status
Submitted, not yet approved
Date submitted
Help: Date submitted
14/11/2025
Date registered
Help: Date registered
2/12/2025
Date last updated
Help: Date last updated
2/12/2025
Date data sharing statement initially provided
Help: Data sharing statement date
2/12/2025
Type of registration
Help: Type of registration
Prospectively registered

Titles & IDs
Public title
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
Scientific title
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
Additional trial identifiers [1] 315945 0
KT-US-728-0204
Universal Trial Number (UTN)
Trial acronym
Related trial records

Health condition
Health condition(s) studied:
Multiple Sclerosis 339746 0
Myasthenia Gravis 339747 0
Chronic Inflammatory Demyelinating Polyneuropathy 339748 0
Condition category
Condition code
Neurological 335998 335998 0 0
Multiple sclerosis
Inflammatory and Immune System 335999 335999 0 0
Autoimmune diseases
Neurological 336000 336000 0 0
Other neurological disorders

Intervention/exposure
Study type
Interventional
Description of the intervention or 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 Multiple Sclerosis, Myasthenia Gravis or Chronic Inflammatory Demyelinating Polyneuropathy.

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 on Day 5 through Day 3 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: 0.5 x 10^6 & 1.0 x 10^6 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 0.5 x 10^6 Kite-363 Cells/kg, if the dose limiting toxicities observed do not cross the study-specific thresholds then the next dose level (1.0 x 10^6 Kite-363 Cells/kg) cohorts will be dosed.
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] 332558 0
Treatment: Drugs
Description of the comparator or control
No control group
Control group
Uncontrolled

Outcomes
Primary outcome [1] 343528 0
To evaluate the safety and tolerability of KITE-363 in participants with autoimmune neurologic diseases
Timepoint [1] 343528 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.
Primary outcome [2] 343529 0
To evaluate the preliminary efficacy of KITE-363 in patients with Multiple Sclerosis
Timepoint [2] 343529 0
Baseline and 12 weeks after treatment
Primary outcome [3] 343531 0
To evaluate the preliminary efficacy of KITE-363 in patients with Myasthenia Gravis
Timepoint [3] 343531 0
Baseline and weekly up until week 24 post-treatment
Secondary outcome [1] 454715 0
This is a primary outcome. To evaluate the preliminary efficacy of KITE-363 in participants with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP).
Timepoint [1] 454715 0
Baseline, daily from days 1-7 post-treatment, at 14 days post-treatment, at 28 days post treatment and then monthly up until Week 24 post-treatment.

Eligibility
Key inclusion criteria
Diagnosed with MS according to the 2017 revision of the McDonald diagnostic criteria
or
Diagnosis of MG with generalized weakness meeting criteria as defined by the MGFA
classification of II-IV at screening
or
Diagnosed with probable or definite CIDP as defined by the 2010 European Federation of Neurological Societies/Peripheral Nerve Society (EFNS/PNS)

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.
Minimum age
18 Years
Maximum age
75 Years
Sex
Both males and females
Healthy volunteers
No
Key exclusion criteria
1) Bone marrow insufficiency within 28 days of lymphodepletion
2) History of autologous or allogeneic stem cell transplant and/or organ transplant
3) 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
18/02/2026
Actual
Date of last participant enrolment
Anticipated
7/06/2027
Actual
Date of last data collection
Anticipated
27/06/2029
Actual
Sample size
Target
52
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,VIC
Recruitment outside Australia
Country [1] 27606 0
Canada
State/province [1] 27606 0
Country [2] 27607 0
United States of America
State/province [2] 27607 0

Funding & Sponsors
Funding source category [1] 320555 0
Commercial sector/Industry
Name [1] 320555 0
Kite, A Gilead Company
Country [1] 320555 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] 323169 0
None
Name [1] 323169 0
Address [1] 323169 0
Country [1] 323169 0

Ethics approval
Ethics application status
Submitted, not yet approved
Ethics committee name [1] 319096 0
Bellberry Human Research Ethics Committee A
Ethics committee address [1] 319096 0
Ethics committee country [1] 319096 0
Australia
Date submitted for ethics approval [1] 319096 0
27/08/2025
Approval date [1] 319096 0
Ethics approval number [1] 319096 0

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

Contacts
Principal investigator
Name 146238 0
Dr Janlyn Falconer
Address 146238 0
Concord Hospital, Hospital Road, Concord, NSW 2139
Country 146238 0
Australia
Phone 146238 0
+61 297676233
Fax 146238 0
Email 146238 0
[email protected]
Contact person for public queries
Name 146239 0
Janlyn Falconer
Address 146239 0
Concord Hospital, Hospital Road, Concord, NSW 2139
Country 146239 0
Australia
Phone 146239 0
+61 297676233
Fax 146239 0
Email 146239 0
[email protected]
Contact person for scientific queries
Name 146240 0
Ryan Capps
Address 146240 0
Kite Pharma Inc, 2400 Broadway, Santa Monica, CA 90404
Country 146240 0
United States of America
Phone 146240 0
+18444545483
Fax 146240 0
Email 146240 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.