Dose Escalation and Safety
Conditions
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.
Interventions
DRUGNXC-201 (formerly HBI0101)
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.
Sponsors
Nexcella Inc.
Hadassah Medical Organization
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Multiple Myeloma and AL amyloidosis Patients
Eligibility
Sex/Gender
ALL
Age
18 Years to 120 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Determination of MTD | 21 days | Part A: Determination of MTD Part B: Confirmation of selected dose tested (at or below MTD) ( safety ) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| The overall survival | 2 years | according to the IMWG Uniform Response Criteria for Multiple Myeloma |
| The progression-free survival | 2 years | according to the IMWG Uniform Response Criteria for Multiple Myeloma |
Countries
Israel
Outcome results
None listed