NXC-201 (formerly HBI0101) Multiple Myeloma

NCT04720313 | Active Not Recruiting Phase 1 DMC

• 1 other identifier: MOH_2020-12-22_009584
• Study Type: interventional
• Target: 160
• Locations: 1 country
• Sites: 1

Brief Summary

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

Conditions

Dose Escalation and Safety

Outcome Measures

Primary Outcomes (1)

• Determination of MTD

  Part A: Determination of MTD Part B: Confirmation of selected dose tested (at or below MTD) ( safety )

  21 days

Secondary Outcomes (2)

• The overall survival

  2 years

• The progression-free survival

  2 years

Study Arms (1)

CART BCMA

EXPERIMENTAL

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)

Interventions

NXC-201 (formerly HBI0101) DRUG

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.

CART BCMA

Eligibility Criteria

• Age: 18 Years - 120 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• ≥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

Sponsors & Collaborators

• Hadassah Medical Organization: lead
• Nexcella Inc.: collaborator

Study Sites (1)

Hadassah University Hospital

Jerusalem, Jerusalem, 91120, Israel

Location

Related Publications (4)

• Lebel E, Asherie N, Kfir-Erenfeld S, Grisariu S, Avni B, Elias S, Assayag M, Dubnikov-Sharon T, Pick M, Alexander-Shani R, Bessig N, Herr S, Shehadeh A, Ishtay A, Pimienta S, Vainstein V, Zimran E, Cohen Y, Avivi I, Cohen C, Stepensky P, Gatt ME. Efficacy and Safety of Anti-B-Cell Maturation Antigen Chimeric Antigen Receptor T-Cell for the Treatment of Relapsed and Refractory AL Amyloidosis. J Clin Oncol. 2025 Jun 10;43(17):2007-2016. doi: 10.1200/JCO-24-02252. Epub 2024 Dec 9.

  PMID: 39653116 DERIVED

• Kfir-Erenfeld S, Asherie N, Lebel E, Vainstein V, Assayag M, Dubnikov Sharon T, Grisariu S, Avni B, Elias S, Alexander-Shani R, Bessig N, Shehadeh A, Ishtay A, Zelmanovich V, Zimran E, Pick M, Roziner I, Kenett RS, Cohen Y, Avivi I, Cohen CJ, Gatt ME, Stepensky P. Clinical evaluation and determinants of response to HBI0101 (BCMA CART) therapy in relapsed/refractory multiple myeloma. Blood Adv. 2024 Aug 13;8(15):4077-4088. doi: 10.1182/bloodadvances.2024012967.

  PMID: 38768428 DERIVED

• Asherie N, Kfir-Erenfeld S, Avni B, Assayag M, Dubnikov T, Zalcman N, Lebel E, Zimran E, Shaulov A, Pick M, Cohen Y, Avivi I, Cohen C, Gatt ME, Grisariu S, Stepensky P. Development and manufacture of novel locally produced anti-BCMA CAR T cells for the treatment of relapsed/refractory multiple myeloma: results from a phase I clinical trial. Haematologica. 2023 Jul 1;108(7):1827-1839. doi: 10.3324/haematol.2022.281628.

  PMID: 36200421 DERIVED

• 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.

  PMID: 36107221 DERIVED

Study Officials

• Polina Stepensky, prof.

  Hadassah university hospital of Jerusalem

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Model Details: Multiple Myeloma and AL amyloidosis Patients

Study Record Dates

• First Submitted: January 20, 2021
• First Posted: January 22, 2021
• Study Start: January 1, 2021
• Primary Completion: January 1, 2026
• Study Completion (Estimated): January 1, 2027
• Last Updated: March 14, 2025

Record last verified: 2025-02

Data Sharing

• IPD Sharing: Will not share

Locations

IL (1)