A Phase 1 interventional study of RJK-RT2831 dose-escalation phase Ia and RJK-RT2831 Dose Expansion Phase Ib in Hematologic Malignancies, sponsored by Nanjing RegeneCore Biotech Co., Ltd.. Recruiting at 16 sites in China. Open to participants aged 18 Years to 74 Years. Per ClinicalTrials.gov, last updated 2026-01-30.
Sponsored by Nanjing RegeneCore Biotech Co., Ltd. · Phase 1, Interventional, and Treatment
This study is a first-in-human (FIH) study which is required to understand the safety, tolerability, pharmacokinetics and preliminary efficacy of RJK-RT2831 injection in patients with hematologic malignancies
This is the first-in-human, Phase 1, open-label, multicenter, dose-escalation and dose expansion phase study to investigate the safety, Tolerability, pharmacokinetics and preliminary efficacy of RJK-RT2831 injection in patients with hematologic malignancies. About 70 subjects with malignant blood tumors will be recruited. The specific tumor type will be determined based on the results of the Phase Ia trial and may be expanded to other malignant blood tumor types.The primary goal of the study is to assess the safety, tolerability, maximum tolerated dose (MTD),determine the recommended doses for expansion (RDEs) and recommended phase 2 dose (RP2D) in patients with hematologic malignancies.
1,464 studies on the registry are indexed under Hematologic Neoplasms; 433 are open to participants now.
This study's planned enrollment of 71 is above the median of 45 across 1,068 interventional studies indexed under Hematologic Neoplasms.
Browse Hematologic Neoplasms studies →Nanjing RegeneCore Biotech Co., Ltd. is the lead sponsor of 5 studies on the registry; 3 are open to participants now.
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Note: The following diagnostic criteria must be met for relapsed/refractory AML: (1) Relapsed AML: Leukemia cells reappear in the peripheral blood or bone marrow blast cells exceed 5% after complete remission (CR) (excluding reasons such as bone marrow regeneration post-consolidation chemotherapy) or there is extramedullary infiltration by leukemia cells. (2) Refractory AML: Initial cases that are unresponsive after two cycles of standard regimen treatment; recurrence within 12 months after CR and consolidation therapy; recurrence beyond 12 months with ineffectiveness of conventional chemotherapy; those who have relapsed twice or more; or persistent extramedullary leukemia.;
-5. Phase Ib tentatively includes patients with hematologic malignancies that meet the following criteria: relapsed or refractory AML subjects who have received at least one previous treatment and have failed to current standard treatments that provide clinical benefit, and MDS subjects who are diagnosed according to the 5th edition of the WHO Classification of Haematolymphoid Tumours-Myeloid and Histiocytic/Dendritic Neoplasms (Khoury et al., 2022) and are classified as high-risk or very-high-risk according to the Revised International Prognostic Scoring System (IPSS-R) (Greenberg et al., 2012). The specific tumor type will be determined based on the results of the Phase Ia trial and may be extended to other hematologic malignancies.
Note: Subjects with myelodysplastic syndromes with excess blasts (MDS-EBs) type 1 (MDS-EB1) or type 2 (MDS-EB2) may be considered for inclusion. The EB type has a higher risk of transformation to AML than other subtypes and may have greater clinical benefits.
9. Substantial normal function of major organs with screening laboratory tests meeting the following criteria:
Exclusion Criteria:
2. Has received any anticancer therapy or investigational drug within the following specified time period prior to the first dose of RJK-RT2831:
3. Subjects with cardiovascular disease, including but not limited to:
4. Subjects with an evidence of infectious disease, including:
9. Subjects with uncontrolled co-morbidities such as:
Drug: RJK-RT2831 dose-escalation phase Ia · Drug: RJK-RT2831 Dose Expansion Phase Ib
there are seven doses(1mg-160mg) in this part. The subjects will receive RJK-RT2831 injection twice a week by intravenous push or intravenous infusion for 4 weeks until the end of one dosing cycle (28 days). After one dosing cycle, if the subject tolerates, the subject will continue to receive treatment until the researcher believes that the subject no longer benefits, or the subject has disease progression, unacceptable toxicity, withdraws informed consent, dies, is lost to follow-up, or starts new anti-tumor treatment (whichever occurs first).
there are four doses in this part. The subjects will be randomized in a 1:1 ratio to receive one dose level of RJK-RT2831 intravenous infusion twice a week for 4 weeks until the end of one dosing cycle (28 days).After one dosing cycle, if the subject tolerates, the subject will continue to receive treatment until the researcher believes that the subject no longer benefits, or the subject has disease progression, unacceptable toxicity, withdraws informed consent, dies, is lost to follow-up, or starts new anti-tumor treatment (whichever occurs first).
phase Ia and phase Ib: Number of Participants with Adverse Events (AEs) as a Measure of Safety and Tolerability
All AEs (except for CRS and TLS) will be graded according to NCI CTCAE V5.0, a common standard term for AE. CRS and TLS will be graded according to ASCTC Consensus (2019) and Cairo-Bishop grading system (Howard revised) respectively.
Time frame: The summary of all AEs was dominated by adverse events that occurred during treatment, including any AE that occurred from the first administration of the study drug until 28 ± 7 days after the last administration.
phase Ia: Maximum Tolerated Dose (MTD)
To assess the safety, tolerability, and maximum tolerated dose (MTD) of RJK-RT2831 in patients with hematologic malignancies.
Time frame: From the first administration of the study drug until 28 days after the first dose
phase Ia: Recommended Doses for Expansion (RDEs)
To determine the recommended doses for expansion (RDEs) in patients with hematologic malignancies.
Time frame: From the first administration of the study drug until 28 ±7 days after the last administration and prior to the initiation of a new anti-tumor therapy
phase Ib: Recommended Phase II Dose (RP2D)
To determine the recommended Phase II dose (RP2D) in patients with hematologic malignancies.
Time frame: From the first administration of the study drug until 28 ±7 days after the last administration and prior to the initiation of a new anti-tumor therapy
Cmax
Pharmacokinetic (PK) analysis of RT2831
Time frame: The first 6 cycles (28 days/cycle)
Tmax
PK analysis of RT2831
Time frame: The first 6 cycles (28 days/cycle)
Area under the concentration-time curve (AUC)
PK analysis of RT2831
Time frame: The first 6 cycles (28 days/cycle)
Total Clearance (CL)
PK analysis of RT2831
Time frame: The first 6 cycles (28 days/cycle)
Volume of distribution (Vd)
PK analysis of RT2831
Time frame: The first 6 cycles (28 days/cycle)
Half-life (t1/2)
PK analysis of RT2831
Time frame: The first 6 cycles (28 days/cycle)
Clearance (CL)
PK analysis of RT2831
Time frame: The first 6 cycles (28 days/cycle)
volume of distribution (Vd)
PK analysis of RT2831
Time frame: The first 6 cycles (28 days/cycle)
elimination rate constant (Kel)
PK analysis of RT2831
Time frame: The first 6 cycles (28 days/cycle)
average concentration (Cave)
PK analysis of RT2831
Time frame: The first 6 cycles (28 days/cycle)
Anti-Drug Antibodies (ADA)
Monitor the production of anti-drug antibodies (ADA) during the dose escalation and dose expansion phases.
Time frame: On the first day of each cycle until the last cycle administrated (28 days/cycle)
overall remission rate (ORR)
Includes: complete remission (CR), complete remission with incomplete blood count recovery (CRi), partial remission (PR)
Time frame: On the first day of each cycle until the end of treatment (+ 3 days) and before starting new anti-tumor therapy (28 days/cycle)
duration of remission (DOR)
DOR is calculated from date of initial documentation of a response (complete response (CR) and complete remission with incomplete blood count recovery (CRi) or partial response (PR) to the date of first documented evidence of relapse, defined in disease-specific response criteria, or death, whichever occurs first.
Time frame: Throughout the study (from the first administration of the study drug until 28 ± 7 days after the last dose and before starting new anti-tumor therapy)
event-free survival (EFS)
time from the date of first study treatment administration to the date of treatment failure, hematologic relapse from CR/CRi/PR or death from any cause
Time frame: Throughout the study (until 28 ± 7 days after the last dose and before starting new anti-tumor therapy)
overall survival (OS)
time from the date of first study treatment administration to the date of death
Time frame: Throughout the study (every 3 months ±7 days after discontinuation to assess survival status until subject death or study termination)
Plan to share: No
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Nanjing RegeneCore Biotech Co., Ltd.