ClinicalTrials.gov record NCT06971380
Study of HBI0101 (NXC-201) CAR-T Therapy in Multiple Myeloma and Light-Chain Amyloidosis
A Phase 2 Trial to Study Efficacy and Safety of HBI0101 (NXC-201) CART in Subjects With Multiple Myeloma and Light-Chain Amyloidosis.
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
This Phase 2, single‑arm study will enroll up to 180 people with relapsed or refractory multiple myeloma or light‑chain (AL) amyloidosis. Participants provide cells by leukapheresis, receive lymphodepletion, then a single infusion of HBI0101 CAR‑T cells and are hospitalized for several days. Follow‑up visits occur periodically for up to 24 months after infusion.
What it is studying. The study is testing whether a single infusion of autologous HBI0101 (anti‑BCMA) CAR‑T cells is safe and produces clinical responses in people with relapsed/refractory multiple myeloma or light‑chain amyloidosis.
What the researchers hope to learn. Collect data on clinical response rates (partial response or better), safety and serious adverse events, overall and progression‑free survival, duration and persistence of CAR‑T cells, and organ responses in subjects with AL amyloidosis.
What this study is about
The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.
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.
Interventions being studied
Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.
- Biological
- HBI0101 CART
Eligibility
The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.
Inclusion criteria
- ≥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.
Exclusion criteria
- 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.
- 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.
Study description and what participation involves
The study's detailed description, as published on ClinicalTrials.gov.
Detailed description from the registry
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.
AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴
AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.
After consent and screening, participants will undergo leukapheresis (collection of their T cells) for manufacture of the investigational CAR‑T product, receive lymphodepleting chemotherapy, and then a single CAR‑T cell infusion. Participants are typically hospitalized for several days after the infusion, then return for scheduled follow‑up visits and testing (including blood tests and bone‑marrow aspirate for minimal residual disease assessments) for up to 24 months. Women of child‑bearing potential will have a pregnancy test before treatment, and all sexually active participants must agree to use effective contraception during the study. Routine and safety monitoring will occur throughout follow‑up.
How the study is structured. Interventional Phase 2, open‑label, single‑group trial. Each participant undergoes leukapheresis to make the product, receives lymphodepletion chemotherapy, then a single infusion of HBI0101 CAR‑T cells (one dose). Participants are observed in hospital for several days and return for scheduled follow‑up visits for up to 24 months.
Questions you could ask
These questions are suggestions for a conversation with the study team or your own clinician, not advice.
- The listed sites are in Israel. Is any of them reachable for me, and does the study help with travel?
- Which of the eligibility criteria would need to be checked with tests before I could join?
More on AL amyloidosis
Important dates
- Study start
- Mar 1, 2025
- Primary completion
- May 15, 2028 (estimated)
- Study completion
- May 15, 2030 (estimated)
- Target enrollment
- 180 participants (estimated)
- Last updated on registry
- May 14, 2025
- Data verified
- Oct 10, 2026
Sponsor and research team
The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.
- Lead sponsor
- Polina Stepensky
Central study contact
If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.
- Polina Stepensky, MD 📞 972-2-6778353 ✉ Fainak@hadassah.org.il
Participating sites (1)
- Hadassah MORecruiting
Jerusalem, 9574869 · Israel
- Polina Stepensky, Prof. · +97226776679 · Fainak@haddasah.org.il
- Polina Stepensky, prof.
Terms used on this page · explanations by MedicalUpdates.ai
- informed consent
- The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
- relapsed or refractory
- A condition that has not responded to a treatment. 'Relapsed' means it responded and then came back; 'refractory' means it did not respond in the first place.
- at screening
- The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
- prior lines
- Treatment a person has already had for the condition. A 'line of therapy' is one course of treatment; studies often require, or limit, the number of prior lines.
- relapse
- A condition that improved with treatment and has since returned or worsened.
- VGPR
- A response category used in some blood conditions meaning the disease marker has fallen a great deal but not disappeared. The exact definition depends on the condition and the study.
- ECOG
- A scale from 0 to 5 that describes how a person's condition affects daily activity. 0 means fully active; 1 means restricted in strenuous activity; 2 means able to self-care but unable to work; higher numbers mean more limitation.
- baseline
- Measurements taken at the start of the study, before any study treatment, so that later results can be compared with them.
- immunosuppressive
- A medicine that reduces the activity of the immune system. Studies of autoimmune conditions often require a stable dose, a prior failure, or a washout of such medicines.
- Contraindication
- A reason a particular test or treatment should not be used for a person, for example a metal implant that rules out an MRI scan.
- upper limit of normal
- The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
- creatinine clearance
- A measure of kidney function, calculated from a blood creatinine level together with age, weight and sex.
- stable dose
- A dose that has not changed for a defined period before the study starts. Studies may require this so that background treatment is relatively stable when study measurements begin.
- exclusion criteria
- The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
- absolute neutrophil count
- The number of neutrophils, a type of white blood cell that fights infection, in a blood sample. Studies often require it to be above a set level.
- Echocardiogram
- An ultrasound scan of the heart. A transesophageal echocardiogram is done through a probe passed into the food pipe for a closer view.
- complete remission
- No sign of the condition can be found with the tests used in the study. It does not always mean the condition is gone permanently, and the definition depends on the condition and the study.
- Inclusion Criteria
- The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
- single-arm
- All participants receive the same treatment or follow the same plan. There is no comparison group within the study.
- open-label
- No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
- investigational product
- A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
- follow-up
- The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.
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