NXC-201 (formerly HBI0101) Multiple Myeloma

A Phase 1 Dose Escalation and Safety Study of NXC-201 (formerly HBI0101) CART in BCMA-Expressing Multiple Myeloma Patients

It is a phase one study with dose escalation and safety CART in BCMA- Expressing Multiple Myeloma and AL amyloidosis Patients

Study Overview

• Status: Active, not recruiting
• Conditions: Dose Escalation and Safety
• Intervention / Treatment: Drug: NXC-201 (formerly HBI0101)

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.

• Study Type: Interventional
• Enrollment (Estimated): 160
• Phase: Phase 1

Contacts and Locations

Study Locations

• Israel
  • Jerusalem, Israel, 91120
    • Hadassah University Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 120 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: CART BCMA; 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	Drug: NXC-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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Determination of MTD	Part A: Determination of MTD Part B: Confirmation of selected dose tested (at or below MTD) ( safety )	21 days

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
The overall survival	according to the IMWG Uniform Response Criteria for Multiple Myeloma	2 years
The progression-free survival	according to the IMWG Uniform Response Criteria for Multiple Myeloma	2 years

Collaborators and Investigators

• Sponsor: Hadassah Medical Organization
• Collaborators: Nexcella Inc.
• Investigators: Principal Investigator: Polina Stepensky, prof., Hadassah university hospital of Jerusalem

Publications and helpful links

General Publications

• 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.

Study record dates

Study Major Dates

• Study Start (Actual): January 1, 2021
• Primary Completion (Estimated): January 1, 2026
• Study Completion (Estimated): January 1, 2027

Study Registration Dates

• First Submitted: January 20, 2021
• First Submitted That Met QC Criteria: January 21, 2021
• First Posted (Actual): January 22, 2021

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: March 12, 2025
• Last Verified: February 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Vascular Diseases; Cardiovascular Diseases; Neoplasms; Immune System Diseases; Neoplasms by Histologic Type; Hematologic Diseases; Lymphoproliferative Disorders; Immunoproliferative Disorders; Neoplasms, Plasma Cell; Hemostatic Disorders; Paraproteinemias; Blood Protein Disorders; Hemorrhagic Disorders; Multiple Myeloma
• Other Study ID Numbers: MOH_2020-12-22_009584

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: There will be no IPD sharing

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No