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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04318327




Registration number
NCT04318327
Ethics application status
Date submitted
20/03/2020
Date registered
23/03/2020

Titles & IDs
Public title
BCMA-directed CAR-T Cell Therapy in Adult Patients With Multiple Myeloma
Scientific title
Phase I, Open Label, Study of B-cell Maturation Antigen (BCMA)-Directed CAR-T Cells in Adult Patients With Multiple Myeloma
Secondary ID [1] 0 0
CADPT07A12101
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Multiple Myeloma 0 0
Condition category
Condition code
Cancer 0 0 0 0
Other cancer types

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Other - PHE885

Experimental: PHE885 (Part A) - Relapsed and/or refractory multiple myeloma (r/r MM) patients will receive PHE885.

Experimental: PHE885 (Part B) - Newly diagnosed multiple myeloma (NDMM) patients will receive PHE885.


Treatment: Other: PHE885
Infusion
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Incidence of Dose limiting toxicities (DLT)
Assessment method [1] 0 0
Incidence of Dose Limiting Toxicities (DLTs) during the first 28 days after anti-BCMA CAR-T cell administration
Timepoint [1] 0 0
28 days
Primary outcome [2] 0 0
Nature of Dose limiting toxicities (DLT)
Assessment method [2] 0 0
Nature of Dose Limiting Toxicities (DLTs) during the first 28 days after anti-BCMA CAR-T cell administration
Timepoint [2] 0 0
28 days
Primary outcome [3] 0 0
Incidence and severity of adverse events (AEs) and serious adverse events (SAEs)
Assessment method [3] 0 0
Timepoint [3] 0 0
24 months
Secondary outcome [1] 0 0
Manufacture success rate (defined as number of subjects treated with planned target dose divided by total number of subjects treated)
Assessment method [1] 0 0
evaluate the feasibility of the manufacturing process
Timepoint [1] 0 0
24 Months
Secondary outcome [2] 0 0
Overall Response Rate (ORR) in Part A
Assessment method [2] 0 0
Proportion of subjects with the best overall response (BOR) of PR (partial response) or better, as determined by local investigator using the IMWG Criteria
Timepoint [2] 0 0
24 months
Secondary outcome [3] 0 0
ORR in Part B
Assessment method [3] 0 0
Proportion of subjects with VGPR (very good partial response) or PR to induction therapy who achieve the BOR of PR or better, as determined by local investigator using the IMWG Criteria
Timepoint [3] 0 0
24 months
Secondary outcome [4] 0 0
Response rate at 3 and 6 months in Part A
Assessment method [4] 0 0
Proportion of subjects with the overall response of PR or better at months 3 and 6 after infusion respectively, as determined by local investigator using the IMWG Criteria
Timepoint [4] 0 0
3 months, 6 months
Secondary outcome [5] 0 0
Overall response rate at 3 and 6 months in Part B
Assessment method [5] 0 0
Proportion of subjects with VGPR or PR to induction therapy who achieve the overall response of PR or better at months 3 and 6 after infusion respectively, as determined by local investigator using the IMWG Criteria
Timepoint [5] 0 0
3 months, 6 months
Secondary outcome [6] 0 0
Overall Complete Response Rate (CRR) in Part A
Assessment method [6] 0 0
Proportion of subjects with the BOR of CR or better, as determined by local investigator using the IMWG Criteria
Timepoint [6] 0 0
24 months
Secondary outcome [7] 0 0
Overall CRR in Part B
Assessment method [7] 0 0
Proportion of subjects with a response of VGPR or PR to induction therapy with the BOR of CR or better, as determined by local investigator using the IMWG Criteria
Timepoint [7] 0 0
24 months
Secondary outcome [8] 0 0
CRR at months 3 and 6 in Part A
Assessment method [8] 0 0
Proportion of subjects with the overall response of CR or better at months 3 and 6 respectively, as determined by local investigator using the IMWG Criteria
Timepoint [8] 0 0
3 months, 6 months
Secondary outcome [9] 0 0
CRR at months 3 and 6 in Part B
Assessment method [9] 0 0
Proportion of subjects with a response of VGPR or PR to induction therapy with the overall response of CR or better at months 3 and 6 after infusion, respectively, as determined by local investigator using the IMWG Criteria
Timepoint [9] 0 0
3 months, 6 months
Secondary outcome [10] 0 0
DOR (duration of response) in Part A
Assessment method [10] 0 0
DOR as assessed by local investigator: the time from achievement of PR or better to relapse or death due to MM (multiple myeloma)
Timepoint [10] 0 0
from disease response to disease progression, assessed up to approximately 24 months
Secondary outcome [11] 0 0
DOR in Part B
Assessment method [11] 0 0
DOR as assessed by local investigator: * The time from the first documented disease response after infusion of CR or better to the date of the first documented progression as assessed by IMGW or death due to MM, for subjects with a response of VGPR or PR to induction therapy, and * The time from the first documented disease response after infusion of PR or better to the date of the first documented progression as assessed by IMGW or death due to MM, for subjects with a response of VGPR or PR to induction therapy
Timepoint [11] 0 0
from disease response to disease progression, assessed up to approximately 24 months
Secondary outcome [12] 0 0
Cmax of BCMA CAR-T cells
Assessment method [12] 0 0
through qPCR-detected transgene of CART concentrations over time in peripheral blood and bone marrow
Timepoint [12] 0 0
24 months
Secondary outcome [13] 0 0
Tmax of BCMA CAR-T cells
Assessment method [13] 0 0
through qPCR-detected transgene of CART concentrations over time in peripheral blood and bone marrow
Timepoint [13] 0 0
24 months
Secondary outcome [14] 0 0
AUC of BCMA CAR-T cells
Assessment method [14] 0 0
through qPCR-detected transgene of CART concentrations over time in peripheral blood and bone marrow
Timepoint [14] 0 0
24 months
Secondary outcome [15] 0 0
Clast of BCMA CAR-T cells
Assessment method [15] 0 0
through qPCR-detected transgene of CART concentrations over time in peripheral blood and bone marrow
Timepoint [15] 0 0
24 months
Secondary outcome [16] 0 0
number of patients with pre-existing and treatment induced immunogenicity (cellular and humoral) of BCMA CAR-T cell therapy
Assessment method [16] 0 0
Timepoint [16] 0 0
24 Months

Eligibility
Key inclusion criteria
* Part A: Subjects with MM who are relapsed and/or refractory to at least 2 prior treatment regimens, including an IMiD (e.g. lenalidomide or pomalidomide), a proteasome inhibitor (e.g. bortezomib, carfilzomib), and an approved anti-CD38 antibody (e.g. daratumumab), if available, and have documented evidence of disease progression (IMWG criteria)
* Part A: ECOG performance status that is either 0 or 1 at screening
* Part B: Subjects with newly diagnosed multiple myeloma (NDMM) who have received a minimum of 4 and up to 6 cycles of standard induction therapy with VRd, D-VRd, or D-Rd, and have achieved a response of PR or better. One cycle of CyBorDex is allowed prior to induction.
* Part B: ECOG performance status that is either 0,1 or 2 at screening
* Measurable disease as defined by the protocol
* Adequate hematological values
* Must have a leukapheresis material of non-mobilized cells accepted for manufacturing
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Prior administration of a genetically modified cellular product including prior BCMA CAR-T therapy. Patients who have received prior BCMA-directed bi-specific antibodies or antibody-drug conjugates (ADC) are not excluded.
* Autologous HSCT within 6 weeks prior to enrollment or any prior history of allogeneic hematopoietic stem cell transplant (HSCT)
* Chemotherapy or any concomitant anti-cancer therapies (other than protocol prescribed lymphodepletion (LD) chemotherapy) within 2 weeks prior to apheresis
* Treatment with small molecule targeted antineoplastics within 2 weeks of apheresis collection or 5 half-lives whichever is shorter
* Have received antibodies or immunotherapies (other than daratumumab) within 4 weeks prior to apheresis collection. Daratumumab within 3 weeks prior to apheresis collection.

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
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
23/07/2020
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
19/02/2026
Actual
Sample size
Target
96
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Novartis Investigative Site - Prahran
Recruitment postcode(s) [1] 0 0
3181 - Prahran
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Illinois
Country [2] 0 0
United States of America
State/province [2] 0 0
Massachusetts
Country [3] 0 0
United States of America
State/province [3] 0 0
Wisconsin
Country [4] 0 0
Israel
State/province [4] 0 0
Ramat Gan
Country [5] 0 0
Israel
State/province [5] 0 0
Tel Aviv
Country [6] 0 0
Singapore
State/province [6] 0 0
Singapore

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Novartis Pharmaceuticals
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT04318327