Back to resultsNXC-201 (Formerly HBI0101) Multiple Myeloma
Primary Purpose
Dose Escalation and Safety
Status
Recruiting
Phase
Phase 1
Locations
Israel
Study Type
Interventional
Intervention
NXC-201 (formerly HBI0101)
Sponsored by
About this trial
This is an interventional treatment trial for Dose Escalation and Safety
Eligibility Criteria
Inclusion Criteria:
≥18 years of age
- Voluntarily signed informed consent form (ICF)
- Eastern Cooperative Oncology Group (ECOG) performance status 0 - 2
- Diagnosis of MM with relapsed or refractory disease and have had at least 3 different prior lines of therapy including proteasome inhibitor, immunomodulatory therapy and at least one antibody therapy.
Subjects must have measurable disease, including at least one of the criteria below:
- Serum M-protein greater or equal to 0.5 g/dL
- Urine M-protein greater or equal to 200 mg/24 h
- Serum free light chain (FLC) assay: involved FLC level greater or equal to 5 mg/dL (50 mg/L) provided serum FLC ratio is abnormal
- A biopsy-proven evaluable plasmacytoma
- Bone marrow plasma cells > 20% of total bone marrow cells
- Non secretory patient will be allowed provided they have measurable disease by PET-CT or bone marrow aspiration, as designated.
- Women of child-bearing potential (WCBP), must have a negative serum pregnancy test prior to treatment. All sexually active WCBP and all sexually active male subjects must agree to use effective methods of birth control throughout the study
- Recovery to ≤Grade 2 or baseline of any non-hematologic toxicities due to prior treatments, excluding alopecia and Grade 3 neuropathy
- Ability and willingness to adhere to the study visit schedule and all protocol requirements
Sites / Locations
- Hadassah University HospitalRecruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
CART BCMA
Arm Description
The dose escalation phase (Part A) will include the following doses of CAR-positive (CAR+) T cells: 150×10^6, 450×10^6, 800×10^6 or 1200 ×10^6 The expansion phase (Part B) will include a dose between 450×10^6 to 800×10^6 CAR-positive (CAR+) T cells
Outcomes
Primary Outcome Measures
Determination of MTD
Part A: Determination of MTD Part B: Confirmation of selected dose tested (at or below MTD) ( safety )
Secondary Outcome Measures
The overall survival
according to the IMWG Uniform Response Criteria for Multiple Myeloma
The progression-free survival
according to the IMWG Uniform Response Criteria for Multiple Myeloma
Full Information
NCT ID
NCT04720313
First Posted
January 20, 2021
Last Updated
April 30, 2023
Sponsor
Hadassah Medical Organization
Collaborators
Nexcella Inc.
1. Study Identification
Unique Protocol Identification Number
NCT04720313
Brief Title
NXC-201 (Formerly HBI0101) Multiple Myeloma
Official Title
A Phase 1 Dose Escalation and Safety Study of NXC-201 (Formerly HBI0101) CART in BCMA-Expressing Multiple Myeloma Patients
Study Type
Interventional
2. Study Status
Record Verification Date
April 2023
Overall Recruitment Status
Recruiting
Study Start Date
January 1, 2021 (Actual)
Primary Completion Date
January 2025 (Anticipated)
Study Completion Date
January 2026 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Hadassah Medical Organization
Collaborators
Nexcella Inc.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
It is a phase one study with dose escalation and safety CART in BCMA- Expressing Multiple Myeloma and AL amyloidosis Patients
Detailed Description
The intention with NXC-201 (formerly HBI0101) CART is to follow the chimeric antigen receptor T-cells (CART) approach, as for approved products, but target the B cell maturation antigen (BCMA) rather than the CD19 antigen targeted by KYMRIAHTM (tisagenlecleucel) and YESCARTATM (axicabtagene ciloleucel).
Importantly, successful results from at least three clinical trials of a BCMA targeted CAR T therapy were published (Zhao 2018, Brundo 2018, Raje 2019), with excellent results obtained for relapsed or refractory multiple myeloma (MM) patients, that validate the approach.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Dose Escalation and Safety
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Model Description
Multiple Myeloma and AL amyloidosis Patients
Masking
None (Open Label)
Allocation
N/A
Enrollment
160 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
CART BCMA
Arm Type
Experimental
Arm Description
The dose escalation phase (Part A) will include the following doses of CAR-positive (CAR+) T cells: 150×10^6, 450×10^6, 800×10^6 or 1200 ×10^6 The expansion phase (Part B) will include a dose between 450×10^6 to 800×10^6 CAR-positive (CAR+) T cells
Intervention Type
Drug
Intervention Name(s)
NXC-201 (formerly HBI0101)
Intervention Description
NXC-201 (formerly HBI0101) CART is defined as autologous T cells transduced ex-vivo with anti-BCMA CAR retroviral vector encoding the chimeric antigen receptor (CAR) targeted to human BCMA. The NXC-201 (formerly HBI0101) CART is provided fresh without cryopreservation.
Primary Outcome Measure Information:
Title
Determination of MTD
Description
Part A: Determination of MTD Part B: Confirmation of selected dose tested (at or below MTD) ( safety )
Time Frame
21 days
Secondary Outcome Measure Information:
Title
The overall survival
Description
according to the IMWG Uniform Response Criteria for Multiple Myeloma
Time Frame
2 years
Title
The progression-free survival
Description
according to the IMWG Uniform Response Criteria for Multiple Myeloma
Time Frame
2 years
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
120 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
≥18 years of age
Voluntarily signed informed consent form (ICF)
Eastern Cooperative Oncology Group (ECOG) performance status 0 - 2
Diagnosis of MM with relapsed or refractory disease and have had at least 3 different prior lines of therapy including proteasome inhibitor, immunomodulatory therapy and at least one antibody therapy.
Subjects must have measurable disease, including at least one of the criteria below:
Serum M-protein greater or equal to 0.5 g/dL
Urine M-protein greater or equal to 200 mg/24 h
Serum free light chain (FLC) assay: involved FLC level greater or equal to 5 mg/dL (50 mg/L) provided serum FLC ratio is abnormal
A biopsy-proven evaluable plasmacytoma
Bone marrow plasma cells > 20% of total bone marrow cells
Non secretory patient will be allowed provided they have measurable disease by PET-CT or bone marrow aspiration, as designated.
Women of child-bearing potential (WCBP), must have a negative serum pregnancy test prior to treatment. All sexually active WCBP and all sexually active male subjects must agree to use effective methods of birth control throughout the study
Recovery to ≤Grade 2 or baseline of any non-hematologic toxicities due to prior treatments, excluding alopecia and Grade 3 neuropathy
Ability and willingness to adhere to the study visit schedule and all protocol requirements
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Polina Stepensky, Prof
Phone
972-26777803
Email
polina@hadassah.org.il
First Name & Middle Initial & Last Name or Official Title & Degree
Ella Dardac, BSc
Phone
+972585852136
Email
ellad@hadassah.org.il
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Polina Stepensky, prof.
Organizational Affiliation
Hadassah university hospital of Jerusalem
Official's Role
Principal Investigator
Facility Information:
Facility Name
Hadassah University Hospital
City
Jerusalem
ZIP/Postal Code
91120
Country
Israel
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Polina Stepensky, Prof
Phone
972-26777803
Email
polina@hadassah.org.il
First Name & Middle Initial & Last Name & Degree
liliane Dray, NSC
Phone
972-26777260
Email
lilane@hadassah.org.il
12. IPD Sharing Statement
Plan to Share IPD
No
IPD Sharing Plan Description
There will be no IPD sharing
Citations:
PubMed Identifier
36107221
Citation
Kfir-Erenfeld S, Asherie N, Grisariu S, Avni B, Zimran E, Assayag M, Sharon TD, Pick M, Lebel E, Shaulov A, Cohen YC, Avivi I, Cohen CJ, Stepensky P, Gatt ME. Feasibility of a Novel Academic BCMA-CART (HBI0101) for the Treatment of Relapsed and Refractory AL Amyloidosis. Clin Cancer Res. 2022 Dec 1;28(23):5156-5166. doi: 10.1158/1078-0432.CCR-22-0637.
Results Reference
derived
Learn more about this trial
NXC-201 (Formerly HBI0101) Multiple Myeloma
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