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
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.