Dose Escalation and SafetyPhase 1NCT04720313
NXC-201 (formerly HBI0101) Multiple Myeloma
A Phase 1 study testing NXC-201 (formerly HBI0101) in people with Dose Escalation and Safety, run by Hadassah Medical Organization at 1 site in Israel.
Trial summary
It is a phase one study with dose escalation and safety CART in BCMA- Expressing Multiple Myeloma and AL amyloidosis Patients Read more about this study
- Who is it for?
- Ages 18 to 120 years
- Conditions the study is for
- Dose Escalation and Safety
- What is being tested?
- NXC-201 (formerly HBI0101)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 160 participants
- Where
- 1 siteIsrael
- Study dates
- 1 Jan 2021to Jan 2027
- Phase
- Phase 1
- 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 to 120 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- ≥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
Show all 14Show fewer
- 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
Who cannot join
None listed.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: 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
Read the 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.
- Conditions
- Dose Escalation and Safety
- Being tested
- NXC-201 (formerly HBI0101)
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
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.
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: Drug: NXC-201 (formerly HBI0101) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Determination of MTD
Part A: Determination of MTD Part B: Confirmation of selected dose tested (at or below MTD) ( safety )
Measured: 21 days
Secondary outcomes (2)
The overall survival
according to the IMWG Uniform Response Criteria for Multiple Myeloma
Measured: 2 years
The progression-free survival
according to the IMWG Uniform Response Criteria for Multiple Myeloma
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jan 2021
- Study start1 Jan 2021
- Primary completionJan 2026 (estimated)
- Study completionJan 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (4)Papers the sponsor has linked to this study.
- Other trials for Dose Escalation and SafetyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Multiple Myeloma and AL amyloidosis Patients
- Masking
- None (open label)
- Enrolment
- 160 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hadassah Medical Organization
- Sponsor type
- University, hospital or other
- Collaborators
- Nexcella Inc.
- Responsible party
- Principal investigator, Polina Stepensky, Hadassah Medical Organization
- Investigators
- Polina Stepensky, prof., Principal investigator, Hadassah university hospital of Jerusalem
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04720313
- Sponsor's protocol ID
- MOH_2020-12-22_009584
- Status last verified
- Feb 2025
- First submitted
- 20 Jan 2021
- Last updated
- 14 Mar 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: There will be no IPD sharing
Publications
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.
PubMed 39653116Kfir-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.
PubMed 38768428Asherie 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 36200421Kfir-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
Study at a glance
From the registry record, last updated by the sponsor on 14 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Dose Escalation and Safety Study of NXC-201 (formerly HBI0101) CART in BCMA-Expressing Multiple Myeloma Patients
- ClinicalTrials.gov ID
- NCT04720313
- Acronym / sponsor ID
- MOH_2020-12-22_009584
- Conditions
- Dose Escalation and Safety
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 160 participants (estimated)
- Lead sponsor
- Hadassah Medical OrganizationUniversity, hospital or other
- Collaborators
- Nexcella Inc.
- Study start
- 1 Jan 2021 (actual)
- Primary completion
- Jan 2026 (estimated)
- Study completion
- Jan 2027 (estimated)
- First posted
- 22 Jan 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
It is a phase one study with dose escalation and safety CART in BCMA- Expressing Multiple Myeloma and AL amyloidosis Patients
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Multiple Myeloma and AL amyloidosis Patients
| Arm | Type | Receives |
|---|---|---|
| CART BCMA | Experimental | Drug: NXC-201 (formerly HBI0101)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 |
Who may qualify
- Ages
- 18 to 120 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (14 listed)
- ≥18 years of age
Key exclusion criteria (0 listed)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 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. | Drug | CART BCMA |
Primary outcome
Determination of MTD
Time frame: 21 days
Part A: Determination of MTD Part B: Confirmation of selected dose tested (at or below MTD) ( safety )
2 secondary outcomes
The overall survival
Time frame: 2 years
The progression-free survival
Time frame: 2 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Mar 2025.
- Derived from this study: PubMed 39653116Lebel 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.
- Derived from this study: PubMed 38768428Kfir-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.
- Derived from this study: PubMed 36200421Asherie 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.
- Derived from this study: PubMed 36107221Kfir-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.
- Individual participant dataSharing planned: No. There will be no IPD sharing
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Polina Stepensky, prof. principal investigator
Hadassah university hospital of Jerusalem
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing NXC-201 (formerly HBI0101) in people with Dose Escalation and Safety, run by Hadassah Medical Organization 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 14 inclusion and 0 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: under way, not looking for participants, as of the sponsor's last update on 14 Mar 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Determination of MTD (time frame: 21 days). 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 NCT04720313, which also lists 4 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 Mar 2025.