This Study is a FIH Study Which is Required to Understand the PK Characteristics, MTD, RP2D and Safety Profile.

Last updated: January 28, 2026
Sponsor: Nanjing RegeneCore Biotech Co., Ltd.
Overall Status: Active - Recruiting

Phase

1

Condition

Hematologic Neoplasms

Neoplasms

Treatment

RJK-RT2831 Dose Expansion Phase Ib

RJK-RT2831 dose-escalation phase Ia

Clinical Study ID

NCT06756451
RJK-RT2831-101
  • Ages 18-74
  • All Genders

Study Summary

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

Eligibility Criteria

Inclusion

Inclusion Criteria:

    1. A male or female aged ≥ 18 and <75 years old.
    1. The following three points are evaluated by the investigator and are deemedsuitable to participate in the study: a) The subject fully understands therequirements of this study and voluntarily signs a written informed consent form; b)Be able to comply with the medication requirements of this study as well as allstudy related procedures and assessments; c) Not deemed as potentially unreliableand/or uncooperative.
    1. An ECOG physical status score is ≤ 2 and an expected survival is ≥ 3 months.
    1. Relapsed or refractory acute myeloid leukemia (AML) that meets the followingcriteria will be included in Phase Ⅰa : Patients diagnosed with AML according to the 5th edition of the World Health Organization (WHO) Classification of HaematolymphoidTumours: -Myeloid and Histiocytic/Dendritic Neoplasms (Khoury et al., 2022),including any subtype except for acute promyelocytic leukemia (APL). These patientsmust have received at least one previous treatment and have failed to currentstandard treatments that provide clinical benefit.

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.

    1. The test of CD33 and/or CD123 for Phase Ia and Ib subjects in this study will berequired to be positive, and patients with any level of CD33 and/or CD123 positivitywere allowed to be included. Note: The positivity and expression levels of CD33 andCD123 were determined by each site.
    1. Consent to bone marrow biopsy and aspiration, and consent to implantation ofmedical devices such as peripherally inserted central catheter (PICC), or venousaccess port, or central venous catheter (CVC) (if applicable) for long-termintravenous drug administration.
    1. Patients have recovered from toxicity of prior first-line therapy, i.e., Grade 1toxicity according to the National Cancer Institute Common Terminology Criteria forAdverse Events (NCI CTCAE) V5.0, with the exception of toxicities such as alopecia,fatigue, and other toxicities deemed by the investigator to be of no safety risk tothe patient, as well as hematological toxicities related to the tumor.
    1. Substantial normal function of major organs with screening laboratory testsmeeting the following criteria:
  1. Bone marrow function: white blood cell count ≤ 30×10^9/L;

  2. Liver function: TBIL ≤ 1.5×ULN, TBIL ≤ 3×ULN if Gilbert's syndrome; ALT and AST ≤ 3 ×ULN;

  3. Renal function: creatinine clearance rate (CCr) > 50 mL/min calculatedaccording to the Cockcroft-Gault method

  4. Coagulation function: activated partial thromboplastin time (APTT) andprothrombin time (PT) ≤ 1.5×ULN.

    1. The serum pregnancy test for female patients of childbearing potential should benegative during the screening period. Male and female patients of childbearingpotential must agree to use effective methods of contraception from signing aninformed consent form during the screening period, throughout the study and for 3months after the last dose of the RJK-RT2831, including but not limited to:abstinence, vasectomy in males, female sterilization surgery, effective intrauterinecontraceptive devices or condoms, and effective contraceptive medications.. Note:Women of non-childbearing potential include permanent infertility (hysterectomy,bilateral oophorectomy, or bilateral salpingectomy) and postmenopausal women.

Exclusion

Exclusion Criteria:

    1. APL is suspected or confirmed based on morphology, immunophenotyping, moleculartesting, or chromosomal karyotyping, or other hematological tumors, such as myeloidsarcoma, mixed phenotype acute leukemia, accelerated phase and blast phase ofchronic myeloid leukemia, etc.;
    1. Has received any anticancer therapy or investigational drug within the followingspecified time period prior to the first dose of RJK-RT2831:
  1. Received cytotoxic chemotherapy (except hydroxyurea use for subjects withleukocytosis >10,000 cells/mm3 or rapid disease progression), radiotherapy (except local palliative radiotherapy), immunotherapy, targeted therapy andother anti-tumor treatments within 14 days;

  2. Received adoptive cell therapy, such as autologous or donor natural killer cellor T lymphocyte infusions (e.g., chimeric antigen receptor-T cells), unless > 60 days prior to the first dose of study treatment and treatment-relatedtoxicities have resolved to at least Grade 1;

  3. Received any investigational drug (for any indication) within 5 half-lives ofthe drug or 14 days, whichever is longer;

  4. Received an autologous haematopoietic stem cell transplant within 12 weeks.

    1. Subjects with cardiovascular disease, including but not limited to:
  1. Resting state, mean QTcF > 480 ms in men/women derived from 12-leadelectrocardiogram (ECG) (repeated 3 times); Note: QTcF = QTcB ×RR^0.17, QTcB isthe corrected QT interval obtained using the Bazetts correction formula;

  2. Echocardiogram or multigated acquisition (MUGA) scan showing a left ventricularejection fraction of less than 50%;

  3. Symptomatic or treatment-requiring abnormalities in rhythm, conduction, andresting ECG morphology, such as arrhythmias, degree II or III atrioventricularblock, congestive heart failure (New York Heart Association cardiac functionclass III or IV)

  4. New onset of angina pectoris and myocardial infarction within 6 months prior toinitiation of study treatment;

  5. Various factors that may increase the risk of prolonged QTc, such as congenitallong QT syndrome, having long QT syndrome in the immediate family history, andbeing on any medications known to prolong the QT interval.

  6. History of cerebral perfusion (e.g., carotid artery stenosis) or stroke ortransient ischemic attack within 6 months prior to screening.

    1. Subjects with an evidence of infectious disease, including:
  1. Hepatitis B surface antigen (HBs Ag) positive, and hepatitis B virusdeoxyribonucleic acid (HBV DNA) copy number greater than the normal upperlimit;

  2. Hepatitis C virus antibody (HCV Ab) positive and hepatitis C virus ribonucleicacid (HCV RNA) levels greater than the normal upper limit;

  3. infected with human immunodeficiency virus (HIV);

  4. Treponema pallidum (Tp) antibody positive;

  5. Known active tuberculosis.

    1. Subjects with leukemic extramedullary infiltration.
    1. Subjects with long-term steroid therapy, except for the following: 10 mg ofprednisone daily (or equivalent) or lower dose steroids for the control of nausea,vomiting, seasonal allergies, or for the prevention of adrenocortical insufficiency.Subjects who require > 10 mg of prednisone for a long time (e.g., more than 2months) need to be excluded.Note: Topical steroids or inhaled steroids arepermitted.
    1. Subjects with autoimmune hemocytopenia that the investigator assessed asrequiring active intervention, e.g. autoimmune haemolytic anaemia, idiopathicthrombocytopenic purpura.
    1. Subjects who have undergone major surgical procedures (craniotomy, thoracotomy,laparotomy, vascular intervention, other as defined by the investigator) or haveunhealed wounds, ulcers or fractures within 4 weeks prior to the first dose ofRJK-RT2831. Note: For palliative care purposes, local surgical treatment of isolatedlesions is acceptable.
    1. Subjects with uncontrolled co-morbidities such as:
  1. Severe infection, including but not limited to hospitalization due toinfection, bacteremia, or severe pneumonia complications, occurs within 4 weeksprior to initiation of study treatment; or patients who have receivedtherapeutic oral or intravenous antibiotics within two weeks prior to startingstudy treatment, except who have received prophylactic antibiotics (e.g., forthe prevention of urinary tract infection or chronic obstructive pulmonarydisease). If it is not an active infection and the infection is controllable,the subjects can be enrolled;

  2. Other malignancies (other than non-melanoma basal cell carcinoma or squamouscell carcinoma of the skin, breast/cervical carcinoma in situ, superficialbladder carcinoma that have received radical treatment and no evidence ofdisease recurrence) within 5 years prior to initiation of RJK-RT2831 treatmentor concurrently;

  3. Subjects with current cancerous meningitis, spinal cord compression, etc.;

  4. A history of poorly controlled hypertension (abnormal blood pressure measured 3times to take the mean, systolic blood pressure ≥ 160 mmHg and/or diastolicblood pressure ≥ 100 mmHg);

  5. Other acute or chronic illnesses or abnormal laboratory test values that mayresult in an increase in the risk associated with study participation orinvestigational drug administration, or interfere with the interpretation ofstudy results;

    1. Subjects who are expected to receive other anti-tumor therapy during the study (palliative radiotherapy is allowed).
    1. Subjects with prior therapy using gemtuzumab ozogamicin or other CD33-targetingagents, or who have experience with CD33-targeting compounds in the clinical trialphase.
  • 12.Subjects with prior therapy using tagraxofusp or other CD123-targeting agents, orwho have experience with CD123-targeting compounds in the clinical trial phase.

  • 13.Subjects who are allergic to prescription components of the RJK-RT2831formulation (histidine, histidine hydrochloride, sucrose, Poloxam 188).

  • 14.Subjects who are pregnant or lactating women.

  • 15.Subjects who have received other investigational drugs within 2 cycles prior tomedication, or are concurrently participating in another interventional clinicalstudy (unless they are participating in an observational study or are in thefollow-up phase of an interventional study).

  • 16.Subjects with a previous history of receiving allogeneic organ transplantationand allogeneic haematopoietic stem cell transplantation.

    1. In the opinion of the investigator, the subject has other factors that mayaffect the results of the study and interfere with his/her participation throughoutthe study, including previous or existing medical conditions, laboratoryabnormalities, and the subject's unwillingness to comply with the processes,restrictions, and requirements of the study.

Study Design

Total Participants: 71
Treatment Group(s): 2
Primary Treatment: RJK-RT2831 Dose Expansion Phase Ib
Phase: 1
Study Start date:
March 25, 2025
Estimated Completion Date:
July 14, 2028

Study Description

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.

Connect with a study center

  • The First Affiliated Hospital of USTC

    Hefei, Anhui 230001
    China

    Site Not Available

  • The Second Hospital of Anhui Medical University

    Hefei, Anhui 230601
    China

    Active - Recruiting

  • The First Affiliated Hospital of USTC

    Hefei 1808722, Anhui 1818058 230001
    China

    Active - Recruiting

  • The Second Hospital of Anhui Medical University

    Hefei 1808722, Anhui 1818058 230601
    China

    Active - Recruiting

  • Guangdong Provincial People's Hospital

    Guangzhou, Guangdong 510080
    China

    Active - Recruiting

  • Southern Medical University Nanfang Hospital

    Guangzhou, Guangdong 510515
    China

    Site Not Available

  • Shenzhen People's Hospital

    Shenzhen, Guangdong 518020
    China

    Site Not Available

  • Guangdong Provincial People's Hospital

    Guangzhou 1809858, Guangdong 1809935 510080
    China

    Active - Recruiting

  • Southern Medical University Nanfang Hospital

    Guangzhou 1809858, Guangdong 1809935 510515
    China

    Active - Recruiting

  • Shenzhen People's Hospital

    Shenzhen 1795565, Guangdong 1809935 518020
    China

    Active - Recruiting

  • The Affiliated Hospital of Guilin Medical University

    Guilin, Guangxi 541001
    China

    Site Not Available

  • The First Affiliated Hospital of Guangxi Medical University

    Nanning, Guangxi 530021
    China

    Site Not Available

  • The Affiliated Hospital of Guilin Medical University

    Guilin 1809498, Guangxi 1809867 541001
    China

    Active - Recruiting

  • The First Affiliated Hospital of Guangxi Medical University

    Nanning 1799869, Guangxi 1809867 530021
    China

    Active - Recruiting

  • Affiliated Hospital of Hebei University

    Baoding, Hebei 071000
    China

    Site Not Available

  • Tangshan Central Hospital

    Tangshan, Hebei 063008
    China

    Site Not Available

  • Affiliated Hospital of Hebei University

    Baoding 1816971, Hebei 1808773 071000
    China

    Active - Recruiting

  • Tangshan Central Hospital

    Tangshan 1793346, Hebei 1808773 063008
    China

    Active - Recruiting

  • Henan Cancer Hospital

    Zhengzhou, Henan 450003
    China

    Site Not Available

  • Henan Cancer Hospital

    Zhengzhou 1784658, Henan 1808520 450003
    China

    Active - Recruiting

  • Zhongda Hospital Southeast University

    Nanjing, Jiangsu 210009
    China

    Site Not Available

  • The First Affiliated Hospital of Soochow University

    Suzhou, Jiangsu 215006
    China

    Site Not Available

  • Zhongda Hospital Southeast University

    Nanjing 1799962, Jiangsu 1806260 210009
    China

    Active - Recruiting

  • The First Affiliated Hospital of Soochow University

    Suzhou 1886760, Jiangsu 1806260 215006
    China

    Active - Recruiting

  • The First Affiliated Hospital of Nanchang University

    Nanchang, Jiangxi 330006
    China

    Site Not Available

  • The First Affiliated Hospital of Nanchang University

    Nanchang 1800163, Jiangxi 1806222 330006
    China

    Active - Recruiting

  • The First Hospital of China Medical University

    Shenyang, Liaoning 110001
    China

    Site Not Available

  • The First Hospital of China Medical University

    Shenyang 2034937, Liaoning 2036115 110001
    China

    Active - Recruiting

  • The First Affiliated Hospital of Xi 'an Jiaotong University

    Xi'an, Shaanxi 710061
    China

    Site Not Available

  • The First Affiliated Hospital of Xi 'an Jiaotong University

    Xi'an 1790630, Shaanxi 1796480 710061
    China

    Active - Recruiting

  • Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College

    Tianjin, Tianjin 300020
    China

    Site Not Available

  • Institute of Hematology and Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College

    Tianjin 1792947, Tianjin Municipality 1792943 300020
    China

    Active - Recruiting

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