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Multiple Myeloma, Refractory to Standard TreatmentPhase 2NCT06971380

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

A Phase 2 study testing HBI0101 CART in people with Multiple Myeloma, Refractory to Standard Treatment and Multiple Myeloma, Relapsed, run by Polina Stepensky at 1 site in Israel.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial 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. Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
HBI0101 CART
Could I receive a placebo?
No, everyone receives an active treatment
How many people
180 participants
Where
1 siteIsrael
Study dates
1 Mar 2025to 15 May 2030
Phase
Phase 2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • ≥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.
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  • 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.

Who cannot join

  • 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).
  • International ratio (INR) or partial thromboplastin time (PTT) > 2 x ULN, unless on a stable dose of anticoagulant for a thromboembolic event (provided this event is not an exclusion criteria).
  • 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.
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  • 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:
  • Have been on a stable dose of anticoagulation for < 1 month (except for acute line insertion induced thrombosis.)
  • Have had a Grade 2, 3, or 4 hemorrhage in the last 30 days
  • Are experiencing continued symptoms from their venous thromboembolic event (e.g. continued dyspnea or oxygen requirement).
  • Pregnant or lactating women.
  • Participation in another interventional clinical trial within 30 days prior to screening visit.

Trial locations and contacts

1 site in 1 country. Contact the study team or a site near you to ask about taking part.

Polina Stepensky, MD contact

972-2-6778353

Fainak@hadassah.org.il

Israel1 site, 1 recruiting

  • Hadassah MO

    Jerusalem, 9574869

    Polina Stepensky, Prof., +97226776679, Fainak@haddasah.org.il

    Polina Stepensky, prof.

    Recruiting

Other trials in Israel

About this study

Official title: A Phase 2 Trial to Study Efficacy and Safety of HBI0101 (NXC-201) CART in Subjects With Multiple Myeloma and Light-Chain Amyloidosis.

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.

Read the detailed description

Up to 180 subjects with relapsed/refractory (R/R) multiple myeloma (MM) or light chain amyloidosis (AL) will be enrolled in a single-arm, open-label, single-site Phase 2 study. Eligible subjects will undergo leukapheresis procedure to provide starting material for manufacturing of HBI0101 CART investigational product. Each eligible subject will receive a single dose of HBI0101 CART cells. Prior to administration of HBI0101 CART, the study subjects will undergo lymphodepletion. Following administration of HBI0101 CART the subjects will be hospitalized for several days and then will return for routine follow-up periodical visits until 24 months after infusion.

Being tested
HBI0101 CART
Keywords
Relapsed/Refractory Multiple Myeloma, Light-Chain Amyloidosis, B-cell maturation antigen (BCMA), Autologous CAR-T
Medical subject headings
Multiple Myeloma, Recurrence, Immunoglobulin Light-chain Amyloidosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • HBI0101 CARTBiological

    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.

Study groups in NCT06971380
Study groupRoleWhat they receive
CART BCMAExperimentalEach subject subjects will receive a single dose of 800-1200 (±20%) x 10^6 HBI0101 CART cells
Receives: Biological: HBI0101 CART

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Clinical response to HBI0101 CART

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

    Measured: 24 months

Secondary outcomes (8)

  1. Safety of HBI0101 CART

    Incidence of Serious Adverse Events and Adverse Events of Special Interest related to study treatment

    Measured: 24 months

  2. Overall response rate

    Overall Response Rate is defined by proportion of subjects with Partial Response or better per International Myeloma Working Group (IMWG) Criteria for MM, and Partial Response or better per Hematologic Response Criteria for AL.

    Measured: 24 months

  3. Overall survival

    Overall Survival is defined by the date of the initial infusion of HBI010 CART cells to the date of the patient's death due to any cause. If the patient is alive or the vital status is unknown, then their data will be censored at the date they were last known to be alive.

    Measured: 24 months

  4. Progression-free survival

    Progression-Free Survival is defined as the time from the date of the initial infusion of HBI0101 CART to the date of first documented disease progression, or death due to any cause, whichever occurs first. This is defined as Stable Disease (or better) per IMWG Criteria for MM, and Partial Response or better per Hematologic Response Criteria for AL.

    Measured: 24 months

  5. Duration of response

    Duration of response is defined as the first observation of partial response (for patients undergoing bridging therapy, this may be the date of the initial CAR T-cell infusion), to the time of disease progression, with deaths from causes other than progression censored.

    Measured: 24 months

  6. Disease-free survival

    Disease-Free Survival proportion of MRD evaluable subjects who are MRD negative is defined as the duration from start of complete response until the time of relapse from complete response.

    Minimal Residual Response (MRD) is defined as the absence of clonal plasma cells on bone marrow aspirate (minimum test sensitivity to detect 1 in 105 nucleated cells (10-5 threshold)).

    Measured: 24 months

  7. Persistence of HBI0101 CART cells

    Quantification of HBI0101 CART cells in the blood over time

    Measured: 24 months

  8. Organ response

    Frequency of organ response (including cardiac, renal, and hepatic responses) in subject with Light-chain Amyloidosis

    Measured: 24 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted14 May 2025
  2. Study start1 Mar 2025
  3. Primary completion15 May 2028 (estimated)
  4. Study completion15 May 2030 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
180 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06971380
Sponsor's protocol ID
HBI0101-02
Status last verified
Apr 2025
First submitted
23 Apr 2025
Last updated
14 May 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Undecided

Publications

  • 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.

    PubMed 36200421

  • 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.

    PubMed 36107221

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing HBI0101 CART in people with Multiple Myeloma, Refractory to Standard Treatment and Multiple Myeloma, Relapsed, run by Polina Stepensky at 1 site in Israel.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 10 inclusion and 21 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 14 May 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Israel. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Polina Stepensky, MD (972-2-6778353, Fainak@hadassah.org.il), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Clinical response to HBI0101 CART (time frame: 24 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06971380, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 May 2025; AzTrials last refreshed its copy on 26 Oct 2026.

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