Study of HBI0101 (NXC-201) CAR-T Therapy in Multiple Myeloma and Light-Chain Amyloidosis

NCT06971380 | Recruiting Phase 2 DMC

• 1 other identifier: HBI0101-02
• Study Type: interventional
• Target: 180
• Locations: 1 country
• Sites: 1

Brief Summary

A Phase II study of HBI0101 (NXC-201) BCMA-CART in Multiple Myeloma and Light-chain Amyloidosis Patients. The goal of the study is to evaluate the efficacy and safety of HBI0101 CART.

Conditions

Multiple Myeloma, Refractory to Standard Treatment; Multiple Myeloma, Relapsed; Light Chain Amyloidosis

Keywords

Relapsed/Refractory Multiple Myeloma; Light-Chain Amyloidosis; B-cell maturation antigen (BCMA); Autologous CAR-T

Outcome Measures

Primary Outcomes (1)

• Clinical response to HBI0101 CART

  Percentage of subjects who achieved partial response (PR) or better

  24 months

Secondary Outcomes (8)

• Safety of HBI0101 CART

  24 months

• Overall response rate

  24 months

• Overall survival

  24 months

• Progression-free survival

  24 months

• Duration of response

  24 months

Study Arms (1)

CART BCMA

EXPERIMENTAL

Each subject subjects will receive a single dose of 800-1200 (±20%) x 10\^6 HBI0101 CART cells

Biological: HBI0101 CART

Interventions

HBI0101 CART BIOLOGICAL

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 HBI0101 CART may be provided fresh or cryopreserved.

CART BCMA

Eligibility Criteria

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

You may qualify if:

• ≥18 years of age at the time of signing informed consent.
• Voluntarily signed informed consent form.
• Diagnosis of multiple myeloma and/or light-chain amyloidosis with relapsed or refractory disease, with measurable disease at screening visit
• Subject suffering from multiple myeloma must have been exposed to at least two prior lines of therapy including proteasome inhibitor, immunomodulatory (IMiDs) therapy or anti-CD38 antibody, or functionally high-risk patients (i.e. first relapse within 18 months of treatment initiation) may be included.
• Subject with amyloidosis must have been exposed to at least one prior line of therapy which includes proteasome inhibitor or anti-CD38 antibody, or subjects with insufficient response (i.e. not achieving a VGPR or CR after exposure to at least an anti-CD38 antibody and a proteasome inhibitor) may be included.
• Eastern Cooperative Oncology Group (ECOG) performance status 0 - 2.
• Women of child-bearing potential (WCBP), defined as a sexually mature woman who has not undergone a hysterectomy or tubal ligation or who has not been naturally postmenopausal for at least 24 consecutive months, 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.
• Subjects with relapsed multiple myeloma who have previously undergone allogenic stem cell transplantation must have no evidence of graft versus host disease after cessation of any immunosuppressive therapy for at least one month before recruitment to the study.

You may not qualify if:

• Contraindication to a study treatment/procedure or is anticipated to receive treatment/procedure that may preclude performance of study procedures.
• Known bulky central nervous system disease.
• Inadequate hepatic function defined by aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) \> 2.5 x upper limit of normal (ULN) and direct bilirubin \> 4x ULN.
• Inadequate renal function defined by serum creatinine clearance/estimated clearance of \<20(ml/min).
• Inadequate bone marrow function defined by absolute neutrophil count (ANC) \< 1000 cells/mm\^3, platelet count \< 30,000 mm\^3, or hemoglobin \< 8 g/dL. Subjects with absolute lymphocyte count \< 300 cells/mm\^3 may be excluded (due to potential challenges with producing CART cells), per investigator judgement.
• Echocardiogram with left ventricular ejection fraction \< 40%.
• Ongoing treatment with chronic immunosuppressant such as cyclosporine or systemic steroids (physiological replacement doses of steroids are allowed up to 12 mg/m\^2/d hydrocortisone or equivalent)
• Significant co-morbid condition or disease which in the judgment of the Investigator would place the subject at undue risk or interfere with the study; examples include, but are not limited to, cirrhotic liver disease, sepsis, recent significant traumatic injury, and other conditions.
• Known human immunodeficiency virus (HIV) positive status.
• Active Hepatitis B or Hepatitis C active infection.
• Active CMV infection.
• Known history of stroke, unstable angina, myocardial infarction, or ventricular arrhythmia requiring medication or mechanical control within 3 months.
• Chronic atrial fibrillation with uncontrolled heart rate.
• Second primary malignancies that has required therapy in the last 2 years or is not in complete remission.
• Subjects who have had a venous thromboembolic event (e.g., pulmonary embolism or deep vein thrombosis) requiring anticoagulation and who meet any of the following criteria:

Sponsors & Collaborators

• Polina Stepensky: lead

Study Sites (1)

Hadassah MO

Jerusalem, 9574869, Israel

RECRUITING

Related Publications (2)

• 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 BACKGROUND

• 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 BACKGROUND

MeSH Terms

Conditions

Multiple Myeloma; Recurrence; Immunoglobulin Light-chain Amyloidosis

Condition Hierarchy (Ancestors)

Neoplasms, Plasma Cell; Neoplasms by Histologic Type; Neoplasms; Hemostatic Disorders; Vascular Diseases; Cardiovascular Diseases; Paraproteinemias; Blood Protein Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemorrhagic Disorders; Lymphoproliferative Disorders; Immunoproliferative Disorders; Immune System Diseases; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Amyloidosis; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Central Study Contacts

Polina Stepensky, MD

CONTACT

972-2-6778353; Fainak@hadassah.org.il

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Prof. Polina Stepensky, Director Department of Bone Marrow Transplantation and Cancer Immunotherapy, Hadassah Medical Organization

Study Record Dates

• First Submitted: April 23, 2025
• First Posted: May 14, 2025
• Study Start: March 1, 2025
• Primary Completion (Estimated): May 15, 2028
• Study Completion (Estimated): May 15, 2030
• Last Updated: May 14, 2025

Record last verified: 2025-04

Locations

IL (1)