Trial Review

Did you know?

The ANZCTR now automatically displays published trial results and simplifies the addition of trial documents such as unpublished protocols and statistical analysis plans.

These enhancements will offer a more comprehensive view of trials, regardless of whether their results are positive, negative, or inconclusive.

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
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
Date last updated
25/01/2024

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)
Timepoint [1] 0 0
28 days
Primary outcome [2] 0 0
Nature of Dose limiting toxicities (DLT)
Timepoint [2] 0 0
28 days
Primary outcome [3] 0 0
Incidence and severity of adverse events (AEs) and serious adverse events (SAEs)
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)
Timepoint [1] 0 0
24 Months
Secondary outcome [2] 0 0
Overall Response Rate (ORR) in Part A
Timepoint [2] 0 0
24 months
Secondary outcome [3] 0 0
ORR in Part B
Timepoint [3] 0 0
24 months
Secondary outcome [4] 0 0
Response rate at 3 and 6 months in Part A
Timepoint [4] 0 0
3 months, 6 months
Secondary outcome [5] 0 0
Overall response rate at 3 and 6 months in Part B
Timepoint [5] 0 0
3 months, 6 months
Secondary outcome [6] 0 0
Overall Complete Response Rate (CRR) in Part A
Timepoint [6] 0 0
24 months
Secondary outcome [7] 0 0
Overall CRR in Part B
Timepoint [7] 0 0
24 months
Secondary outcome [8] 0 0
CRR at months 3 and 6 in Part A
Timepoint [8] 0 0
3 months, 6 months
Secondary outcome [9] 0 0
CRR at months 3 and 6 in Part B
Timepoint [9] 0 0
3 months, 6 months
Secondary outcome [10] 0 0
DOR (duration of response) in Part A
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
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
Timepoint [12] 0 0
24 months
Secondary outcome [13] 0 0
Tmax of BCMA CAR-T cells
Timepoint [13] 0 0
24 months
Secondary outcome [14] 0 0
AUC of BCMA CAR-T cells
Timepoint [14] 0 0
24 months
Secondary outcome [15] 0 0
Clast of BCMA CAR-T cells
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
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 hospital [2] 0 0
Novartis Investigative Site - Camperdown
Recruitment postcode(s) [1] 0 0
3181 - Prahran
Recruitment postcode(s) [2] 0 0
NSW - Camperdown
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
Kansas
Country [3] 0 0
United States of America
State/province [3] 0 0
Massachusetts
Country [4] 0 0
United States of America
State/province [4] 0 0
Wisconsin
Country [5] 0 0
Israel
State/province [5] 0 0
Haifa
Country [6] 0 0
Israel
State/province [6] 0 0
Ramat Gan
Country [7] 0 0
Israel
State/province [7] 0 0
Tel Aviv
Country [8] 0 0
Singapore
State/province [8] 0 0
Singapore

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

Ethics approval
Ethics application status

Summary
Brief summary
This is a first-in-human study to evaluate the feasibility, safety and preliminary antitumor efficacy of autologous T cells genetically engineered with a novel B-cell Maturation Antigen (BCMA)-specific chimeric antigen receptor (CAR) and manufactured with a new process. CAR-T cells will be investigated as a single agent in multiple myeloma
Trial website
https://clinicaltrials.gov/study/NCT04318327
Trial related presentations / publications
Public notes

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



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT04318327