Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12625001031459p
Ethics application status
Submitted, not yet approved
Date submitted
12/08/2025
Date registered
17/09/2025
Date last updated
17/09/2025
Date data sharing statement initially provided
17/09/2025
Type of registration
Prospectively registered

Titles & IDs
Public title
Place-of-care manufactured anti-BCMA chimeric antigen receptor (CAR) T-cells (ARI0002h) in patients with relapsed/refractory multiple myeloma.
Scientific title
Place-of-care manufactured anti-BCMA chimeric antigen receptor (CAR) T-cells (ARI0002h) in patients with relapsed/refractory multiple myeloma.
Secondary ID [1] 315107 0
None
Universal Trial Number (UTN)
U1111-1326-6194
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Multiple Myeloma 338515 0
Relapsed refractory myeloma 338638 0
Condition category
Condition code
Cancer 334821 334821 0 0
Myeloma

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
ARI0002h is an autologous CAR-T cell product modified to specifically target BCMA on B cells.

Leukapheresis for collection of the T cells is planned for 2-4 weeks prior to the infusion to allow for manufacturing of the ARI0002h. Five days prior to infusion of the ARI0002h cells, all patients will undergo lymphodepletive chemotherapy with fludarabine, given intravenously at 30mg/m^2/day, and cyclophosphamide, given intravenously at 300mg/m^2/day, for 3 consecutive days.

ARI0002h will be given over a week with 3 separate intravenous infusions to make at a total dose of 3 x 10^6 CARTBCMA/kg. The dose of each infusion will be increased step-wise to minimise side effects (10% on day 0, 30% on day 3 and 60% on day 7

A second dose may be given between 3-4 months after the first dose consisting of one infusion at a total dose of 3 x 10^6 CARTBCMA/kg. The second dose of ARI0002h will only be given if some degree of response has been achieved (at least minimal response), without progression and in the absence of cytokine release syndrome grade III-IV.

The intervention is given in hospital and will be recorded on the patient's electronic medical record. Adherence to dosing, per protocol, will be documented on chemotherapy and cell infusion charts in the participant's medical record. Prior to administration, both lymphodepletive chemotherapy and ARI0002h will be subject to a 2-person label check by appropriately qualified individuals.

Response to treatment will be assessed using the International Myeloma Working Group criteria 2016.
Intervention code [1] 331730 0
Treatment: Drugs
Comparator / control treatment
No control group
Control group
Uncontrolled

Outcomes
Primary outcome [1] 342458 0
To assess the efficacy of CARTBCMA ARI0002h in patients with relapsed/refractory multiple myeloma who have been previously treated with a proteasome inhibitor, an immunomodulator, and an anti-CD38 monoclonal antibody.
Timepoint [1] 342458 0
Month 3 / Day +100 after the first infusion
Primary outcome [2] 342463 0
To assess the safety of CARTBCMA ARI0002h in patients with relapsed/refractory multiple myeloma who have been previously treated with a proteasome inhibitor, an immunomodulator, and an anti-CD38 monoclonal antibody.
Timepoint [2] 342463 0
30 days after CARTBCMA ARI0002h infusion
Secondary outcome [1] 450890 0
Assess the duration of response after administration of ARI0002h
Timepoint [1] 450890 0
6 months post infusion
Secondary outcome [2] 450891 0
Evaluate the overall survival after the administration of ARI0002h
Timepoint [2] 450891 0
Month 36 after the first infusion or death
Secondary outcome [3] 450892 0
Progression-free survival, defined as the time between administration of ARI0002h and disease progression or death.
Timepoint [3] 450892 0
Month 12 and Month 36 after the first infusion
Secondary outcome [4] 450893 0
Minimal residual disease (MRD) outcomes in the bone marrow at 3months
Timepoint [4] 450893 0
Month 3 after the first infusion
Secondary outcome [5] 450894 0
Overall response rate 6 months after the first infusion
Timepoint [5] 450894 0
6 months after the first infusion
Secondary outcome [6] 451455 0
Minimal residual disease (MRD) outcomes in the bone marrow at 6 months
Timepoint [6] 451455 0
Month 6 after first infusion

Eligibility
Key inclusion criteria
1. Patients between 18 and 80 years old diagnosed with multiple myeloma,
2. Disease measurable by serum or urine monoclonal component, or by serum free light chains, according to the eligibility criteria for clinical trials of the International Myeloma Working Group (IMWG) (Kumar, S., B. Paiva, et al. 2016).
3. Having completed two or more prior lines of treatment and having received at least one proteasome inhibitor (such as bortezomib or carfilzomib), one immunomodulator (lenalidomide or pomalidomide) and one anti-CD38 monoclonal antibody (such as daratumumab).
4. Refractory to the last line of treatment (defined as progression during treatment or within 60 days after the end of treatment).
5. ECOG performance status from 0 to 2
6. Life expectancy greater than 3 months.
7. Patients who, after being informed, give their consent by signing the Informed Consent Document.
Minimum age
18 Years
Maximum age
80 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Prior allogeneic transplant in the 6 months prior to inclusion or GVHD requiring active systemic immunosuppressive treatment.
2. Patients who have previously received any treatment with CART or with anti-BCMA antibodies.
3. Absolute lymphocyte count 3 years and skin carcinomas (non-melanoma)
5. Active infection requiring treatment.
6. Active HIV, HBV, or HCV infection.
7. Uncontrolled medical disease,
8. Severe organ involvement that meets any of the following criteria: EF<40%, DLCO <40%, GFR 3 times the upper limit of normality (unless Gilbert syndrome)
9. Previous diagnosis of symptomatic AL amyloidosis,
10. Pregnant or lactating women. Women of childbearing potential must have a negative pregnancy test at the screening phase.
11. Women of childbearing potential, including those whose last menstrual cycle was in the year prior to screening, who are unable or unwilling to use highly effective contraceptive methods from the beginning of the study to completion of the study.
12. Men who are unable or unwilling to use highly effective contraceptive methods* from the beginning of the study to completion of the study.
13. Contraindication to receive lymphodepletive chemotherapy.

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
Single group
Other design features
Phase
Phase 2
Type of endpoint/s
Safety/efficacy
Statistical methods / analysis

Recruitment
Recruitment status
Not yet recruiting
Date of first participant enrolment
Anticipated
2/02/2026
Actual
Date of last participant enrolment
Anticipated
2/02/2028
Actual
Date of last data collection
Anticipated
2/02/2031
Actual
Sample size
Target
15
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
WA
Recruitment hospital [1] 28325 0
Royal Perth Hospital - Perth
Recruitment postcode(s) [1] 44538 0
6000 - Perth

Funding & Sponsors
Funding source category [1] 319681 0
Charities/Societies/Foundations
Name [1] 319681 0
RPH Research Foundation and WA Future Health Research and Innovation Fund
Country [1] 319681 0
Australia
Primary sponsor type
Government body
Name
Cell and Tissue Therapies WA, Royal Perth Hospital, East Metropolitan Health Service
Address
Country
Australia
Secondary sponsor category [1] 322188 0
None
Name [1] 322188 0
Address [1] 322188 0
Country [1] 322188 0

Ethics approval
Ethics application status
Submitted, not yet approved
Ethics committee name [1] 318244 0
Western Sydney Local Health District Human Research Ethics Committee
Ethics committee address [1] 318244 0
Ethics committee country [1] 318244 0
Australia
Date submitted for ethics approval [1] 318244 0
12/08/2025
Approval date [1] 318244 0
Ethics approval number [1] 318244 0

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

Contacts
Principal investigator
Name 143550 0
A/Prof Agnes Yong
Address 143550 0
Royal Perth Hospital ,Wellington Street, Perth, 6000, Western Australia
Country 143550 0
Australia
Phone 143550 0
+61 08 9224 4503
Fax 143550 0
Email 143550 0
[email protected]
Contact person for public queries
Name 143551 0
Kate Maslen
Address 143551 0
Royal Perth Hospital ,Wellington Street, Perth, 6000, Western Australia
Country 143551 0
Australia
Phone 143551 0
+61 08 9224 4503
Fax 143551 0
Email 143551 0
[email protected]
Contact person for scientific queries
Name 143552 0
Agnes Yong
Address 143552 0
Royal Perth Hospital ,Wellington Street, Perth, 6000, Western Australia
Country 143552 0
Australia
Phone 143552 0
+61 08 9224 4503
Fax 143552 0
Email 143552 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.