Newly Diagnosed High-Risk Cytogenetic Multiple Myeloma Patients Ineligible or Unwilling to Undergo ASCTPhase 1NCT07114432
RN1201 Injection in Newly Diagnosed High-Risk Cytogenetic Multiple Myeloma
A Phase 1 study testing BCMA/CD19-targeted allogeneic CAR-T in people with Newly Diagnosed High-Risk Cytogenetic Multiple Myeloma Patients Ineligible or Unwilling to Undergo ASCT and Newly Diagnosed Multiple Myeloma (NDMM), run by Tianjin Medical University Cancer Institute and Hospital at 2 sites in China.
Trial summary
This is a single-arm, dose-escalation exploratory study evaluating the safety and efficacy of RN1201, a BCMA/CD19-targeted allogeneic CAR-T cell therapy, in patients with newly diagnosed cytogenetically high-risk multiple myeloma who are ineligible or unwilling to undergo autologous stem cell transplantation (ASCT). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Newly Diagnosed High-Risk Cytogenetic Multiple Myeloma Patients Ineligible or Unwilling to Undergo ASCT, Newly Diagnosed Multiple Myeloma (NDMM)
- What is being tested?
- BCMA/CD19-targeted allogeneic CAR-T
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 19 participants
- Where
- 2 sitesChina
- Study dates
- Aug 2025to Aug 2028
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- A subject will be eligible for this trial only if all of the following criteria are met:
- Is willing and able to provide a written informed consent form before any trial-related activities are performed.
- Must have a diagnosis of Multiple Myeloma (MM) according to the World Health Organization (WHO) 2017 revised criteria.
- Must be diagnosed with active MM according to the International Multiple Myeloma Working Group (IMWG) criteria.
- Must meet the definition of genetic high-risk MM as per the "2024 Chinese Expert Consensus on the Diagnosis and Treatment of High-Risk Multiple Myeloma", defined as having one or more of the following high-risk cytogenetic abnormalities (HRCA) confirmed by FISH or mitotic karyotype analysis:
- del(17p) TP53 mutation t(4;14) t(14;16) t(14;20) 1q21 gain/amplification (defined as copy number ≥ 4) Del 1p Note: 1q21 amplification alone does not define cytogenetic high risk.
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- Must have a documented MM type that is B-cell maturation antigen (BCMA) positive and/or CD19 positive, confirmed at screening or from prior medical records.
- Age ≥ 18 years, male or female.
- Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2 at enrollment.
- Has a life expectancy of ≥ 12 weeks.
- Meets the following prior therapy requirements:
- Is currently receiving or has received an induction therapy regimen containing at least a proteasome inhibitor, an immunomodulatory agent, or a monoclonal antibody as a backbone therapy.
- Has not received more than 4 cycles of induction therapy at the time of enrollment.
- Has had an effective response to induction therapy without evidence of disease progression per IMWG criteria.
- Prior radiotherapy is permitted before enrollment.
- Is scheduled to initiate maintenance and/or consolidation therapy 3 months after cell reinfusion, following successful enrollment.
- Must have measurable disease to assess Progression-Free Survival (PFS). Measurable disease is defined by at least one of the following criteria:
- Serum M-protein (M-spike) ≥ 0.5 g/dL.
- 24-hour urine M-protein (M-spike) ≥ 200 mg.
- Abnormal serum free light chain (FLC) ratio with an involved FLC level ≥ 50 mg/L.
- For IgA MM: total serum IgA level outside the normal range.
- Note: The presence of measurable disease is not required at enrollment; it can be determined from historical lab data. For instance, a subject who achieved a complete response after induction and has no measurable disease at enrollment is still eligible if they met any of the above criteria after their initial diagnosis.
- Adequate organ function as defined by the following laboratory values:
- Total serum bilirubin < 2 times the upper limit of normal (ULN).
- Serum creatinine < ULN.
- Alanine aminotransferase (ALT) and Aspartate aminotransferase (AST) < 3 times ULN.
- Estimated or calculated creatinine clearance (CrCl) ≥ 40 mL/min, via CKD-EPI or Cockcroft-Gault formula.
- Diffusing capacity of the lungs for carbon monoxide (DLCO) ≥ 50% of predicted value (corrected or uncorrected for anemia/alveolar volume).
- Adequate bone marrow function defined as: Absolute neutrophil count ≥ 0.5×10⁹/L and platelet count ≥ 20×10⁹/L.
- ECOG performance status of 0-2.
- Left ventricular ejection fraction (LVEF) ≥ 50% with no pericardial effusion.
- Must have recovered to ≤ Grade 1 (per CTCAE) from any Serious Adverse Event (SAE) before enrollment.
- Able to comply with the study visit schedule and other protocol requirements.
Who cannot join
- A subject will not be eligible for this trial if any of the following criteria are met:
- Known history of allergic reaction, hypersensitivity, intolerance, or contraindication to the BCMA/CD19 allogeneic CAR-T product or any of its excipients, including fludarabine, cyclophosphamide, and tocilizumab.
- Diagnosis of plasma cell leukemia.
- Presence of non-paraskeletal extramedullary disease.
- Relapse after allogeneic hematopoietic stem cell transplantation (allo-HSCT) with active graft-versus-host disease (GVHD) that requires treatment with steroids or immunosuppressive agents.
- Presence of a severe, active infection, including:
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- Human Immunodeficiency Virus (HIV) infection (subjects must have a negative HIV 1/2 antibody screen).
- Hepatitis C Virus (HCV) infection (subjects must have a negative anti-HCV antibody screen).
- Hepatitis B Virus (HBV) infection (subjects must have a negative HBsAg screen).
- History of prior gene therapy or genetically modified cell immunotherapy.
- Active autoimmune disease, including connective tissue disease, uveitis, sarcoidosis, inflammatory bowel disease, or multiple sclerosis; or a history of a severe autoimmune disease (as judged by the Principal Investigator) that required long-term immunosuppressive therapy.
- Diagnosis of an acquired or congenital immunodeficiency disease.
- History of Class III or IV heart failure as defined by the New York Heart Association (NYHA), unstable angina, myocardial infarction within the last 6 months, or sustained (>30 seconds) ventricular arrhythmia.
- History of seizure disorder or other central nervous system (CNS) disease; history or current evidence of CNS involvement with MM. Note: CNS screening (e.g., lumbar puncture) is not mandatory unless symptoms are present.
- History of other primary malignancies, except for the following:
- Non-melanoma skin cancer (e.g., basal cell carcinoma) cured by resection.
- Carcinoma in situ (e.g., cervical, bladder, or breast cancer) that has been cured.
- Is pregnant, breastfeeding, or, for female subjects, is planning a pregnancy within 6 months.
- Participation in another clinical trial within the last month.
- Any condition which, in the opinion of the investigator, would place the subject at increased risk or interfere with the trial results.
- Has undergone major surgery within 14 days prior to the first dose of the study drug.
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
China2 sites
Tianjin Medical University Cancer Institute and Hospital
Tianjin, Tianjin Municipality, 300060
Yafei Wang, PhD, +86-18622221250, drwang2005@163.com
Yafei Wang, PhD
Tianjin Cancer Hospital Airport Hospital
Tianjin, Tianjin Municipality
Yafei Wang, PhD, +86-18622221250, drwang2005@163.com
Qian Li, PhD, +86-13820801516, liqtj@163.com
About this study
Official title: An Exploratory Clinical Study on the Safety and Efficacy of BCMA/CD19 Allogeneic CAR-T Cell Injection in Newly Diagnosed High-Risk Cytogenetic Multiple Myeloma Patients Ineligible or Unwilling to Undergo ASCT
This is a single-arm, dose-escalation exploratory study evaluating the safety and efficacy of RN1201, a BCMA/CD19-targeted allogeneic CAR-T cell therapy, in patients with newly diagnosed cytogenetically high-risk multiple myeloma who are ineligible or unwilling to undergo autologous stem cell transplantation (ASCT). Patients will receive lymphodepletion followed by a single infusion of RN1201 across four dose levels. Primary endpoints include incidence and severity of treatment-emergent adverse events. Secondary endpoints assess response rate and minimal residual disease (MRD) status.
Read the detailed description
This is a Phase 1, single-arm, dose-escalation exploratory study to evaluate the safety, feasibility, and preliminary efficacy of RN1201, a BCMA/CD19-targeted allogeneic CAR-T cell therapy, in patients with newly diagnosed high-risk cytogenetic multiple myeloma (MM) who are ineligible or unwilling to undergo autologous stem cell transplantation (ASCT). Patients will receive lymphodepletion chemotherapy followed by a single infusion of RN1201 cells, across four dose levels. The primary endpoint will assess the incidence and severity of treatment-emergent adverse events (TEAEs), while secondary endpoints will evaluate the objective response rate (ORR), complete response (CR), minimal residual disease (MRD) status, and pharmacokinetics (PK) and pharmacodynamics (PD) of RN1201.
- Conditions
- Newly Diagnosed High-Risk Cytogenetic Multiple Myeloma Patients Ineligible or Unwilling to Undergo ASCT, Newly Diagnosed Multiple Myeloma (NDMM)
- Being tested
- BCMA/CD19-targeted allogeneic CAR-T
- Keywords
- Newly Diagnosed, High-Risk Cytogenetic, Multiple Myeloma
- Medical subject headings
- Multiple Myeloma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BCMA/CD19-targeted allogeneic CAR-TDrug
Also called RN1201
Lymphodepletion chemotherapy followed by allogeneic CAR-T cell (RN1201) infusion
| Study group | Role | What they receive |
|---|---|---|
| Allogeneic CAR-T cell therapy | Experimental | Newly Diagnosed High-Risk Cytogenetic Multiple Myeloma Patients Ineligible or Unwilling to Undergo ASCT are treated with allogeneic CAR-T cell therapy Receives: Drug: BCMA/CD19-targeted allogeneic CAR-T |
What is being measured
The primary outcome is the main question the study is designed to answer.
The incidence and severity of dose-limiting toxicities (DLTs)
Measured: DLTs: Within 28 days after CAR-T cell infusion
The incidence and severity of treatment-emergent adverse events (TEAEs)
Measured: TEAEs: From infusion up to 24 months post-treatment.
Secondary outcomes (5)
Overall effectiveness and duration of efficacy
Overall response rate (ORR) and complete response rate (CR), negative rate of MRD (detected by flow cytometry or NGS)
Measured: 4 weeks, 3 months, 6 months, and 12 months
Pharmacokinetic (PK) of RN1201
Levels of RN1201 CAR-positive T cells in the blood and/or bone marrow
Measured: From RN1201 infusion up to 12 months
Levels of Peripheral blood M protein
Levels of Peripheral blood M protein
Measured: From RN1201 infusion up to 12 months
Levels of urine M protein
Levels of urine M protein
Measured: From RN1201 infusion up to 12 months
Levels of Peripheral blood cytokine
Levels of Peripheral blood cytokine, including IL-2, IL-6, IL-10, TNFa et. al.
Measured: From RN1201 infusion up to 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Aug 2025
- Study startAug 2025 (estimated)
- Primary completionDec 2027 (estimated)
- Study completionAug 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Newly Diagnosed High-Risk Cytogenetic Multiple Myeloma Patients Ineligible or Unwilling to Undergo ASCTStudies 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
- Masking
- None (open label)
- Enrolment
- 19 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Tianjin Medical University Cancer Institute and Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Allorunning Therapeutics
- Responsible party
- Sponsor
- Investigators
- Yafei Wang, PhD, Principal investigator, Tianjin Medical University Cancer Institute and Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07114432
- Sponsor's protocol ID
- NDHR
- Status last verified
- Jul 2025
- First submitted
- 28 Jul 2025
- Last updated
- 11 Aug 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Data sharing can be permitted after approval by the study leader due to patient privacy concerns.
Study at a glance
From the registry record, last updated by the sponsor on 11 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Exploratory Clinical Study on the Safety and Efficacy of BCMA/CD19 Allogeneic CAR-T Cell Injection in Newly Diagnosed High-Risk Cytogenetic Multiple Myeloma Patients Ineligible or Unwilling to Undergo ASCT
- ClinicalTrials.gov ID
- NCT07114432
- Acronym / sponsor ID
- NDHR
- Conditions
- Newly Diagnosed High-Risk Cytogenetic Multiple Myeloma Patients Ineligible or Unwilling to Undergo ASCT, Newly Diagnosed Multiple Myeloma (NDMM)
- Keywords
- Newly Diagnosed, High-Risk Cytogenetic, Multiple Myeloma
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 19 participants (estimated)
- Lead sponsor
- Tianjin Medical University Cancer Institute and HospitalUniversity, hospital or other
- Collaborators
- Allorunning Therapeutics
- Study start
- Aug 2025 (estimated)
- Primary completion
- Dec 2027 (estimated)
- Study completion
- Aug 2028 (estimated)
- First posted
- 11 Aug 2025 (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.
This is a single-arm, dose-escalation exploratory study evaluating the safety and efficacy of RN1201, a BCMA/CD19-targeted allogeneic CAR-T cell therapy, in patients with newly diagnosed cytogenetically high-risk multiple myeloma who are ineligible or unwilling to undergo autologous stem cell transplantation (ASCT). Patients will receive lymphodepletion followed by a single infusion of RN1201 across four dose levels. Primary endpoints include incidence and severity of treatment-emergent adverse events. Secondary endpoints assess response rate and minimal residual disease (MRD) status.
Detailed description
This is a Phase 1, single-arm, dose-escalation exploratory study to evaluate the safety, feasibility, and preliminary efficacy of RN1201, a BCMA/CD19-targeted allogeneic CAR-T cell therapy, in patients with newly diagnosed high-risk cytogenetic multiple myeloma (MM) who are ineligible or unwilling to undergo autologous stem cell transplantation (ASCT). Patients will receive lymphodepletion chemotherapy followed by a single infusion of RN1201 cells, across four dose levels. The primary endpoint will assess the incidence and severity of treatment-emergent adverse events (TEAEs), while secondary endpoints will evaluate the objective response rate (ORR), complete response (CR), minimal residual disease (MRD) status, and pharmacokinetics (PK) and pharmacodynamics (PD) of RN1201.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Allogeneic CAR-T cell therapy | Experimental | Drug: BCMA/CD19-targeted allogeneic CAR-TNewly Diagnosed High-Risk Cytogenetic Multiple Myeloma Patients Ineligible or Unwilling to Undergo ASCT are treated with allogeneic CAR-T cell therapy |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (33 listed)
- A subject will be eligible for this trial only if all of the following criteria are met:
- del(17p) TP53 mutation t(4;14) t(14;16) t(14;20) 1q21 gain/amplification (defined as copy number ≥ 4) Del 1p Note: 1q21 amplification alone does not define cytogenetic high risk.
Key exclusion criteria (21 listed)
- A subject will not be eligible for this trial if any of the following criteria are met:
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 |
|---|---|---|
| BCMA/CD19-targeted allogeneic CAR-TAlso: RN1201Lymphodepletion chemotherapy followed by allogeneic CAR-T cell (RN1201) infusion | Drug | Allogeneic CAR-T cell therapy |
Primary outcome
The incidence and severity of dose-limiting toxicities (DLTs)
Time frame: DLTs: Within 28 days after CAR-T cell infusion
The incidence and severity of treatment-emergent adverse events (TEAEs)
Time frame: TEAEs: From infusion up to 24 months post-treatment.
5 secondary outcomes
Overall effectiveness and duration of efficacy
Time frame: 4 weeks, 3 months, 6 months, and 12 months
Pharmacokinetic (PK) of RN1201
Time frame: From RN1201 infusion up to 12 months
Levels of Peripheral blood M protein
Time frame: From RN1201 infusion up to 12 months
Levels of urine M protein
Time frame: From RN1201 infusion up to 12 months
Levels of Peripheral blood cytokine
Time frame: From RN1201 infusion up to 12 months
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 11 Aug 2025.
- Individual participant dataSharing planned: No. Data sharing can be permitted after approval by the study leader due to patient privacy concerns.
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.
Yafei Wang, PhD principal investigator
Tianjin Medical University Cancer Institute and Hospital
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing BCMA/CD19-targeted allogeneic CAR-T in people with Newly Diagnosed High-Risk Cytogenetic Multiple Myeloma Patients Ineligible or Unwilling to Undergo ASCT and Newly Diagnosed Multiple Myeloma (NDMM), run by Tianjin Medical University Cancer Institute and Hospital at 2 sites in China.
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 33 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: opening soon, as of the sponsor's last update on 11 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in China. 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?
Contact Yafei Wang, PhD (+86-18622221250, drwang2005@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The incidence and severity of dose-limiting toxicities (DLTs) (time frame: DLTs: Within 28 days after CAR-T cell infusion). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 11 Aug 2025; AzTrials last refreshed its copy on 26 Oct 2026.