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LeukemiaPhase 1NCT07347171

A Phase 1 Study of CG009301 for Injection in Adult Subjects With Recurrent or Refractory Haematological Malignancies

A Phase 1 study testing CG009301 for Injection in people with Leukemia and AML, run by Cullgen (Shanghai),Inc at 3 sites in China.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial summary

The goal of this clinical trial is to learn about the safety of drug CG009301. It also learns if drug CG009301 works to treat in Participants with relapsed or refractory adult haematological malignancies. The main question[s] it aims to answer are: 1. Read more about this study

Who is it for?
Ages 18 to 75 years
Conditions the study is for
Leukemia, AML, MDS
What is being tested?
CG009301 for Injection
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
45 participants
Where
3 sitesChina
Study dates
17 Apr 2025to 30 Dec 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 75 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Age ≥18 years and <75 years at the time of signing the informed consent form; no gender restrictions;
  • Patients with relapsed/refractory haematological malignancies who have received a definitive diagnosis by pathology and/or cytology, confirmed histologically, and who have failed prior standard treatment regimens. The investigator must deem that no standard treatment is available or that the patient cannot tolerate existing therapies. Dose-escalation phase: unrestricted haematological tumour types. Dose-expansion phase: must meet one of the following criteria: a. Subjects meeting AML diagnostic criteria based on WHO 2022 5th edition classification, confirmed by bone marrow cytomorphology, including AML evolving from early-stage MDS or MPN. Criteria for recurrent AML: Leukaemic cells reappearing in peripheral blood after CR, or >5% blast/immature cells in bone marrow, or extramedullary leukaemic infiltration. Criteria for refractory AML: - Treatment-naïve cases unresponsive to two standard-regimen cycles; - Relapse within 12 months after consolidation/intensification therapy following CR; - Relapse after 12 months unresponsive to conventional chemotherapy; Patients with two or more relapses; Persistent extramedullary leukaemia; b. Patients diagnosed with high-risk or very high-risk MDS according to the WHO 2022 5th edition classification, with a percentage of blasts in bone marrow smear or biopsy pathology < 20%, and deemed by the investigator to have no other appropriate treatment options. Diagnostic criteria for recurrent MDS: Following achievement of complete remission, partial remission, or haematological improvement, at least one of the following must occur: - Bone marrow blastic count returns to pre-treatment levels; - ANC or PLT decreases by ≥50% from best response; - HGB decreases by ≥15 g/L or becomes transfusion-dependent. Diagnostic criteria for refractory MDS: Following adequate treatment (at least four cycles of demethylating agent therapy), meeting the IWG 2023 response criteria for "stable disease", "failure", or "disease progression"; progression after demethylating agent or other drug therapy, or patient intolerance to toxicity (e.g., treatment-related grade 3 or higher hepatic or renal toxicity during therapy leading to permanent discontinuation); c. Subjects meeting ALL diagnostic criteria based on WHO 2022 5th edition classification, with ≥20% primitive/immature lymphocytes in bone marrow. Relapsed ALL diagnostic criteria: Patients who, after achieving CR following induction therapy, exhibit recurrence of leukaemic cells in peripheral blood, >5% primitive/immature lymphocytes in bone marrow, or development of extramedullary disease; Criteria for refractory ALL: Patients failing to achieve CR following standard induction therapy;
  • ECOG performance status score of 0-1;
  • Investigator-assessed expected survival ≥3 months;
  • Recovery of toxicities from prior treatment to ≤Grade 1 according to NCI-CTCAE v5.0 (excluding alopecia and long-term stable chronic conditions);
  • No prior autologous haematopoietic stem cell transplantation, or transplantation more than 2 months prior with toxicities resolved to ≤ Grade 1;
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  • Adequate organ function support, with screening laboratory tests meeting all criteria: a. Coagulation function prior to study drug administration: INR ≤ 1.5 × ULN or aPTT ≤ 1.5 × ULN; b. Hepatic function: serum total bilirubin ≤ 2× ULN; AST and/or ALT ≤ 2.5× ULN; c. Cr ≤ 2× ULN or CrCL > 30 mL/min (calculated using Cockcroft-Gault formula); d. LVEF ≥ 40%; and QTc ≤ 480 milliseconds; e. White blood cell count may decrease below 50.0 × 10⁹/L at baseline or following hydroxyurea administration
  • Non-pregnant and non-lactating: Infertile subjects; or subjects with potential for conception who agree to use effective contraception (hormonal, barrier, or abstinence). Male subjects must also abstain from sperm donation during study participation and for 90 days after the last dose of CG009301 injection. Women of childbearing potential must have a negative serum pregnancy test (serum-β-hCG) during the screening period;
  • Understand the study's purpose, process, nature, significance, potential benefits, and risks, and voluntarily sign the written informed consent form. Be able to comply with scheduled visits, treatment plans, laboratory tests, and other study instructions or procedures.

Who cannot join

  • Central nervous system leukaemia presenting with neurological and/or psychiatric symptoms;
  • Receipt of antitumour therapy (excluding hydroxyurea and prophylactic intrathecal injections) such as chemotherapy, immunotherapy, targeted therapy, or biological therapy within 4 weeks or 5 half-lives (whichever is shorter) prior to the first study drug administration; receipt of radiotherapy within 2 weeks; receipt of traditional Chinese herbal medicine within 2 weeks;
  • Major surgery within 4 weeks prior to the first study dose, or anticipated need for major surgery during the study period;
  • Active infection deemed uncontrolled by the investigator following treatment with antibiotics, antiviral agents, or antifungal medications;
  • Severe or uncontrolled underlying medical conditions deemed ineligible for inclusion by the investigator, including but not limited to respiratory disorders (e.g., chronic obstructive pulmonary disease requiring oxygen therapy, moderate or higher asthma, moderate or higher pulmonary fibrosis, recurrent pulmonary oedema), cardiovascular disorders (e.g., prior coronary artery bypass grafting or coronary stent implantation, myocardial infarction within the past 6 months, NYHA Class III-IV heart failure), unstable angina within the past 6 months, uncontrolled hypertension (systolic >160 mmHg or diastolic >100 mmHg), arrhythmias requiring ongoing medical or interventional management), endocrine disorders (severe hyperthyroidism/hypothyroidism, uncontrolled diabetes mellitus), and neurological/psychiatric conditions affecting cognition, compliance, or personal safety (e.g., unstable epilepsy, dementia, schizophrenia, depression); psychiatric disorders (e.g., unstable epilepsy, dementia, schizophrenia, depression);
  • Active autoimmune diseases (e.g., inflammatory bowel disease, idiopathic thrombocytopenic purpura, lupus erythematosus, autoimmune haemolytic anaemia, scleroderma, severe psoriasis, rheumatoid arthritis), or allergy to the study drug or excipients;
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  • Significant non-leukaemia-related bleeding risk (e.g., anticoagulant or antiplatelet therapy, arteriovenous malformation), or recent history of major bleeding (e.g., gastrointestinal haemorrhage, intracranial haemorrhage, disseminated intravascular coagulation);
  • Grade 2 or higher central nervous system or peripheral neuropathy (excluding stable Grade 3 conditions lasting over 6 months that do not impair daily functioning);
  • Allogeneic haematopoietic stem cell transplantation within 12 months prior to initial administration;
  • History of deep vein thrombosis, pulmonary embolism, or any other severe thromboembolic event within 6 months prior to initial administration (thrombosis originating from implanted venous access ports or catheters, superficial vein thrombosis, or lacunar cerebral infarction are not considered "severe" thromboembolic events); Known familial and/or acquired thrombotic predisposition, such as hereditary or acquired defects in anticoagulant proteins, coagulation factors, fibrinolytic proteins, or presence of acquired risk factors conferring high thrombotic propensity;
  • HIV, HBV, and HCV infection: positive HIV antibody and PCR tests; HBsAg positive or viral DNA ≥100 IU/mL; positive HCV antibody with HCV-RNA quantification exceeding the upper limit of normal;
  • Individuals who received (attenuated) live virus vaccination within 4 weeks prior to first dosing;
  • Individuals with a documented history of alcohol or substance abuse;
  • Any past or current medical condition, treatment, or laboratory abnormality that may interfere with study results or affect the subject's ability to complete the study, or if the investigator deems the subject unsuitable for participation in this study.

Trial locations and contacts

3 sites in 1 country. Contact the study team or a site near you to ask about taking part.

Xiangyu Jin, MS contact

+86 13858065096

xiangyu.jin@cullgen.com

Qiao Yi contact

qiao.yi@cullgen.com

China3 sites, 3 recruiting

  • The First Affiliated Hospital of Nanchang University

    Nanchang

    Fei Li, MD, +8613970038386, yx021021@sina.com

    Recruiting

  • The Haematology Hospital of the Chinese Academy of Medical Sciences

    Tianjin

    Jianxiang Wang, MS, 86-22-23909120, wangjx@medmail.com.cn

    Junyuan Qi, MS

    Recruiting

  • Tongji Hospital Affiliated to Tongji Medical College, Huazhong University of Science and Technology

    Wuhan

    Dengju Li, MD, +8613237100810, lidengju@163.com

    Recruiting

Other trials in China

About this study

Official title: A Phase 1, Open-label, Multicentre Study Evaluating the Safety, Tolerability, Pharmacokinetic/Pharmacodynamic Characteristics, and Preliminary Efficacy of CG009301 for Injection in Adult Subjects With Relapsed or Refractory Haematological Malignancies

The goal of this clinical trial is to learn about the safety of drug CG009301. It also learns if drug CG009301 works to treat in Participants with relapsed or refractory adult haematological malignancies.

The main question[s] it aims to answer are:

  1. To determine the maximum tolerated dose (MTD) and/or objective best dose (OBD) of CG009301 for injection in subjects with relapsed or refractory adult haematological malignancies.
  2. To establish subsequent dosing regimens for CG009301 for injection.
  3. To characterise the safety profile and tolerability of CG009301 for injection. Participants will Receive treatment with CG009301 until disease progression.
Read the detailed description

This study will employ a multicentre, open-label design to evaluate the safety, tolerability, pharmacokinetic/pharmacodynamic characteristics, and preliminary efficacy of CG009301 for injection in adult subjects with relapsed or refractory haematological malignancies.

This Phase I trial will comprise two phases, corresponding to the following indications:

Dose-escalation study - Relapsed/refractory haematological malignancies, regardless of tumour type; Dose-expansion phase: Relapsed/refractory (R/R) acute myeloid leukaemia (AML), high-risk myelodysplastic syndromes (HR-MDS), and R/R acute lymphoblastic leukaemia (ALL).

Being tested
CG009301 for Injection
Keywords
Leukemia, GSPT1, CG009301, AML, MDS
Medical subject headings
Leukemia, Leukemia, Myeloid, Acute, Anemia, Refractory, with Excess of Blasts

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • CG009301 for InjectionDrug

    0.9% Sodium Chloride Injection diluted to 250mL，Cycle 1 and subsequent cycles, IV, infusion duration: 2 hours, once daily (QD) administration for 7 days continuously(28 days constituting one cycle)

Study groups in NCT07347171
Study groupRoleWhat they receive
0.25mg QDExperimentalCG009301 for Injection
Receives: Drug: CG009301 for Injection
0.5mg QDExperimentalCG009301 for Injection
Receives: Drug: CG009301 for Injection
1.0mg QDExperimentalCG009301 for Injection
Receives: Drug: CG009301 for Injection
1.6mg QDExperimentalCG009301 for Injection
Receives: Drug: CG009301 for Injection
2.5mg QDExperimentalCG009301 for Injection
Receives: Drug: CG009301 for Injection
3.5mg QDExperimentalCG009301 for Injection
Receives: Drug: CG009301 for Injection
5.0mg QDExperimentalCG009301 for Injection
Receives: Drug: CG009301 for Injection
6.5mg QDExperimentalCG009301 for Injection
Receives: Drug: CG009301 for Injection
8.25mg QDExperimentalCG009301 for Injection
Receives: Drug: CG009301 for Injection
10.0mg QDExperimentalCG009301 for Injection
Receives: Drug: CG009301 for Injection

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. The RDE(Recommended Dose for Expansion)of CG009301 for injection.

    RDE refer to recommended dose for expansion

    Measured: up to 8 months

  2. Duration of continuous administration and dosing cycle for CG009301 for injection

    Measured: up to 20 months

  3. Safety profile of CG009301 for injection: Incidence, severity, duration, outcome, and relationship to the study drug for adverse events (AEs), serious adverse events (SAEs), dose-limiting toxicity (DLT), and clinically significant laboratory abnormalitie

    Measured: up to 20 months

Secondary outcomes (15)

  1. Cmax

    Cmax refer to Peak concentration

    Measured: up to 20 months

  2. Tmax

    Tmax refer to Time to peak concentration

    Measured: up to 20 months

  3. AUC0-t

    AUC0-t refer to Area under the concentration-time curve from zero to the last quantifiable time point

    Measured: up to 20 months

  4. AUCinf

    AUCinf refer to Area under the concentration-time curve from zero extrapolated to infinity

    Measured: up to 20 months

  5. T1/2

    T1/2 refer to Half-life

    Measured: up to 20 months

  6. CL

    CL refer to Apparent clearance

    Measured: up to 20 months

  7. Vd

    Vd refer to Apparent volume of distribution

    Measured: up to 20 months

  8. Cmax,ss

    Cmax,ss refer to Steady-state peak concentration

    Measured: up to 20 months

  9. Cmin,ss

    Cmin,ss refer to Steady-state trough concentration

    Measured: up to 20 months

  10. drug accumulation ratio

    Measured: up to 20 months

  11. ORR

    ORR refer to objective response rate, the proportion of subjects whose best overall response is CR(complete response),CRi(complete response with incomplete hematologic recovery),CRh(complete remission with partial hematologic recovery), MLFS(morphologic leukemia-free state) or PR(partial response) in the study assessed by investigator according to response criteria of hematological malignancy,which AML(Acute Myeloid Leukemia) according to 2022 European LeukemiaNet (ELN) Response Criteria for Acute Myeloid Leukemia(Döhner et al., Blood 2022),ALL(Acute Lymphoblastic Leukemia) according to National Comprehensive Cancer Network. NCCN Clinical Practice Guidelines in Oncology: Acute Lymphoblastic Leukemia(Version 4.2023).,MDS(Myelodysplastic Syndromes) according to International Working Group 2023 response criteria for higher-risk myelodysplastic syndromes(Zeidan AM et al., Blood 2023).

    Measured: up to 20 months

  12. CRR

    CRR refer to complete response rate, the proportion of subjects whose best overall response is CR(complete response),CRi(complete response with incomplete hematologic recovery) or CRh(complete remission with partial hematologic recovery)in the study assessed by investigator according to response criteria of hematological malignancy,which AML(Acute Myeloid Leukemia) according to 2022 European LeukemiaNet (ELN) Response Criteria for Acute Myeloid Leukemia(Döhner et al., Blood 2022),ALL(Acute Lymphoblastic Leukemia) according to National Comprehensive Cancer Network. NCCN Clinical Practice Guidelines in Oncology: Acute Lymphoblastic Leukemia(Version 4.2023).,MDS(Myelodysplastic Syndromes) according to International Working Group 2023 response criteria for higher-risk myelodysplastic syndromes(Zeidan AM et al., Blood 2023).

    Measured: up to 20 months

  13. EFS

    EFS refer to Event-Free Survival. Defined as the time from the first administration of CG009301 to the first occurrence of trail failure,disease relapse after CR(complete response),CRi(complete response with incomplete hematologic recovery) or CRh(complete remission with partial hematologic recovery), or death (whichever occurs first), as assessed by the investigator according to response criteria of hematological malignancy,which AML according to 2022 European LeukemiaNet (ELN) Response Criteria for Acute Myeloid Leukemia(Döhner et al., Blood 2022),ALL according to National Comprehensive Cancer Network. NCCN Clinical Practice Guidelines in Oncology: Acute Lymphoblastic Leukemia(Version 4.2023).,MDSaccording to International Working Group 2023 response criteria for higher-risk myelodysplastic syndromes(Zeidan AM et al., Blood 2023).

    Trail failure defined as didn't achieve CR,CRi or CRh after 6 period treatment.

    Measured: up to 92 months

  14. RFS

    RFS refer to Relapse-Free Survival. Only uesd to evaluate the subjects which achieved CR(complete response),CRi(complete response with incomplete hematologic recovery) or CRh(complete remission with partial hematologic recovery),Defined as the time from the day subject achieve CR,CRi or CRh,to the hematologic relapse or death (whichever occurs first),as assessed by the investigator according to response criteria of hematological malignancy,which AML according to 2022 European LeukemiaNet (ELN) Response Criteria for Acute Myeloid Leukemia(Döhner et al., Blood 2022),ALL according to National Comprehensive Cancer Network. NCCN Clinical Practice Guidelines in Oncology: Acute Lymphoblastic Leukemia(Version 4.2023).,MDSaccording to International Working Group 2023 response criteria for higher-risk myelodysplastic syndromes(Zeidan AM et al., Blood 2023).

    Measured: up to 92 months

  15. Determination of the RP2D for CG009301 for Injection

    RP2D refer to recommended phase 2 dose

    Measured: up to 20 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted16 Jan 2026
  2. Study start17 Apr 2025
  3. Primary completion30 Dec 2027 (estimated)
  4. Study completion30 Dec 2027 (estimated)

Resources

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
Non-randomised
Intervention model
Single group
Masking
None (open label)
Enrolment
45 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07347171
Sponsor's protocol ID
CG-GSPT1-9301001
Other IDs
CTR20244643
Status last verified
Jan 2026
First submitted
7 Dec 2025
Last updated
16 Jan 2026
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing CG009301 for Injection in people with Leukemia and AML, run by Cullgen (Shanghai),Inc at 3 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 9 inclusion and 14 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: looking for participants now, as of the sponsor's last update on 16 Jan 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 3 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 Xiangyu Jin, MS (+86 13858065096, xiangyu.jin@cullgen.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

The RDE(Recommended Dose for Expansion)of CG009301 for injection. (time frame: up to 8 months). The study lists 3 primary outcomes in all. 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 NCT07347171.

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 16 Jan 2026.

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