Acute Myeloid Leukemia (AML)Phase 1/2NCT07761533
PBSS1113 in Combination With Azacitidine to Treat Patients With AML/MDS
A Phase 1/2 study testing PBSS1113 in people with Acute Myeloid Leukemia (AML) and MDS (Myelodysplastic Syndrome), run by Chaser Therapeutics, Inc. at 1 site in China.
Trial summary
Acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS) are highly heterogeneous myeloid malignancies where few effective targeted therapies are available. While venetoclax has significantly advanced the treatment landscape, therapeutic resistance remains a critical challenge. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acute Myeloid Leukemia (AML), MDS (Myelodysplastic Syndrome)
- What is being tested?
- PBSS1113
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 29 participants
- Where
- 1 siteChina
- Study dates
- 8 Sep 2025to Sep 2027
- Phase
- Phase 1/2
- 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
- Age and Gender: Age ≥ 18 years at the time of signing the informed consent form, male or female.
- Disease Status: Must meet one of the following diagnostic criteria:
- Relapsed or refractory (R/R) acute myeloid leukemia (AML).
- Newly diagnosed AML in patients who are ineligible for intensive induction chemotherapy (including primary AML or secondary AML arising from myelodysplastic syndromes [MDS]).
- Higher-risk MDS, defined per the Revised International Prognostic Scoring System (IPSS-R) as Intermediate risk (score > 3.5 points), High risk, or Very High risk. Prior therapy with hypomethylating agents (HMAs, e.g., decitabine or azacitidine) is permitted.
- Performance Status: Eastern Cooperative Oncology Group (ECOG) performance status score of 0, 1, or 2.
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- Life Expectancy: Expected survival period ≥ 3 months.
- Organ Function: Baseline organ function meeting the following laboratory criteria:
- Hepatic: Total bilirubin (TBIL) ≤ 1.5 × upper limit of normal (ULN); aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 3 × ULN.
- Renal: Serum creatinine ≤ 1.5 × ULN.
- Coagulation: Activated partial thromboplastin time (APTT) ≤ 1.5 × ULN; prothrombin time (PT) ≤ 1.5 × ULN; International Normalized Ratio (INR) ≤ 1.5 × ULN.
- Cardiac: Fridericia-corrected QT interval (QTcF) < 450 ms for males and < 470 ms for females.
- Compliance: Able and willing to adhere to the study procedures, scheduling, and follow-up examinations outlined in the protocol.
- Informed Consent: Capable of understanding and voluntarily providing written informed consent prior to the initiation of any trial-specific screening procedures.
Who cannot join
- Leukemia Subtypes: Diagnosis of acute promyelocytic leukemia (APL), classified as AML-M3 per the French-American-British (FAB) criteria or APL with PML-RARA fusion gene per standard diagnostic classifications.
- Concomitant Malignancies: Diagnosis of another active malignant tumor within the screening period, except for the target AML or MDS indications (as evaluated by the investigator).
- Hyperleukocytosis: White blood cell (WBC) count > 25 × 10⁹/L during screening. Note: Hydroxyurea use is permitted to stabilize the WBC count below this threshold prior to first dose.
- Contraception: Female patients of childbearing potential, male patients, and their partners who are unwilling to practice a medically accepted, highly effective method of contraception during the treatment period and for at least 6 months following the final dose of the investigational drug. Note: Women are considered postmenopausal if they have experienced at least 12 consecutive months of amenorrhea with no alternative pathological cause.
- Prior/Concomitant Therapies:
- Received radiotherapy, immunotherapy, hormonal therapy, targeted therapy, biologic therapy, traditional Chinese medicine indicated for oncology, chemotherapy, or any other investigational agent within 14 days prior to the first dose of the study drug.
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- Prior allogeneic hematopoietic stem cell transplantation (HSCT) with evidence of active graft-versus-host disease (GVHD), or requiring systemic immunosuppressive therapy for GVHD.
- Use of strong CYP3A inhibitors or inducers within 14 days prior to the first dose of the study drug (unless on a stable, clinically justified regimen approved by the investigator).
- Residual Toxicity: Unresolved toxicities from prior anti-cancer therapies that have not recovered to ≤ Grade 1 per CTCAE v5.0 (excluding alopecia and stable Grade 2 peripheral neuropathy, if applicable).
- Surgery: Major surgical procedures or significant traumatic injuries within 28 days prior to the first dose of the study drug, or an anticipated requirement for major surgery during the trial.
- Gastrointestinal Disorders: Difficulty swallowing or any clinically significant gastrointestinal disease or malabsorption syndrome that would significantly impair the oral absorption of the investigational drug.
- Infections and Comorbidities:
- Active, clinically significant, or poorly controlled fungal, bacterial, or viral infections at baseline.
- Known human immunodeficiency virus (HIV) infection (anti-HIV positive).
- Active hepatitis B virus (HBV; HBsAg positive and HBV DNA ≥ 2000 copies/mL or ≥ 500 IU/mL) or active hepatitis C virus (HCV; HCV antibody positive and HCV RNA positive).
- Active syphilis infection.
- Any severe, poorly controlled systemic comorbidities (e.g., unstable psychiatric, neurological, cardiovascular, respiratory, hepatic, or renal diseases).
- Hypersensitivity: Known hypersensitivity or allergy to the investigational drug (PBSS1113), azacitidine, or any of their excipients.
- Investigator Discretion: Any other clinical condition or social circumstance that, in the opinion of the investigator, would compromise patient safety or interfere with the scientific integrity of the study data.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
The First Affiliated Hospital of Zhejiang University School of Medicine
Hangzhou, Zhejiang, 310003
Yiming Zhu, +86 13071862292, yiming_zhu@chasertherapeutics.com
Jie Jin, MD
About this study
Official title: A Phase Ib/II, Open-Label, Dose-Escalation and Cohort Expansion Clinical Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PBSS1113 in Combination With Azacitidine in Patients With Acute Myeloid Leukemia or Myelodysplastic Syndrome
Acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS) are highly heterogeneous myeloid malignancies where few effective targeted therapies are available. While venetoclax has significantly advanced the treatment landscape, therapeutic resistance remains a critical challenge. Consequently, patients with relapsed or refractory (R/R) AML/MDS face a profound unmet medical need for novel therapeutic options. PBSS1113 has demonstrated robust anti-leukemic activity in both in vitro and in vivo models, alongside an acceptable safety profile in a Phase I study. This Phase Ib/II trial will evaluate the safety, tolerability, pharmacokinetics (PK), and preliminary efficacy of PBSS1113 in combination with azacitidine for treatment-naive unfit AML, R/R AML, and higher-risk MDS populations.
- Being tested
- PBSS1113
- Keywords
- AML, MDS, PBSS1113
- Medical subject headings
- 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.
PBSS1113Drug
PBSS1113 enteric tablet, 10/50 mg
| Study group | Role | What they receive |
|---|---|---|
| PBSS1113 | Experimental | PBSS1113 in combination with azacitidine Receives: Drug: PBSS1113 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Composite Complete Remission (cCR) Rate in Patients with Acute Myeloid Leukemia (AML)
The composite complete remission (cCR) rate is defined as the proportion of AML subjects who achieve a best overall response of complete remission (CR), complete remission with partial hematologic recovery (CRh), complete remission with incomplete hematologic recovery (CRi), or a morphological leukemia-free state (MLFS). The analysis will further evaluate the subset of patients achieving measurable residual disease (MRD) negativity within these categories (CRMRD-, CRhMRD-, or CRiMRD-).
Measured: Through study completion, an average of 1 year
Secondary outcomes (1)
Overall Response Rate (ORR) in Patients with Myelodysplastic Syndrome (MDS)
The overall response rate (ORR) is defined as the proportion of MDS subjects who achieve a best overall response according to the revised International Working Group (IWG) 2023 response criteria. This includes complete remission (CR), CR-equivalent (CRequiv), partial remission (PR), CR with limited count recovery (CRL), CR with partial hematologic recovery (CRh), or hematologic improvement (HI).
Measured: Through study completion, an average of 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Aug 2026
- Study start8 Sep 2025
- Primary completionSep 2027 (estimated)
- Study completionSep 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid Leukemia (AML)Studies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 29 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Chaser Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07761533
- Sponsor's protocol ID
- CT-102
- Other IDs
- CTR20253847
- Status last verified
- Aug 2026
- First submitted
- 5 Aug 2026
- Last updated
- 14 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase Ib/II, Open-Label, Dose-Escalation and Cohort Expansion Clinical Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PBSS1113 in Combination With Azacitidine in Patients With Acute Myeloid Leukemia or Myelodysplastic Syndrome
- ClinicalTrials.gov ID
- NCT07761533
- Acronym / sponsor ID
- CT-102
- Keywords
- AML, MDS, PBSS1113
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 29 participants (estimated)
- Lead sponsor
- Chaser Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 8 Sep 2025 (actual)
- Primary completion
- Sep 2027 (estimated)
- Study completion
- Sep 2027 (estimated)
- First posted
- 12 Aug 2026 (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.
Acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS) are highly heterogeneous myeloid malignancies where few effective targeted therapies are available. While venetoclax has significantly advanced the treatment landscape, therapeutic resistance remains a critical challenge. Consequently, patients with relapsed or refractory (R/R) AML/MDS face a profound unmet medical need for novel therapeutic options. PBSS1113 has demonstrated robust anti-leukemic activity in both in vitro and in vivo models, alongside an acceptable safety profile in a Phase I study. This Phase Ib/II trial will evaluate the safety, tolerability, pharmacokinetics (PK), and preliminary efficacy of PBSS1113 in combination with azacitidine for treatment-naive unfit AML, R/R AML, and higher-risk MDS populations.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| PBSS1113 | Experimental | Drug: PBSS1113PBSS1113 in combination with azacitidine |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (14 listed)
- Age and Gender: Age ≥ 18 years at the time of signing the informed consent form, male or female.
- Disease Status: Must meet one of the following diagnostic criteria:
- Relapsed or refractory (R/R) acute myeloid leukemia (AML).
- Newly diagnosed AML in patients who are ineligible for intensive induction chemotherapy (including primary AML or secondary AML arising from myelodysplastic syndromes [MDS]).
- Higher-risk MDS, defined per the Revised International Prognostic Scoring System (IPSS-R) as Intermediate risk (score > 3.5 points), High risk, or Very High risk. Prior therapy with hypomethylating agents (HMAs, e.g., decitabine or azacitidine) is permitted.
Key exclusion criteria (19 listed)
- Leukemia Subtypes: Diagnosis of acute promyelocytic leukemia (APL), classified as AML-M3 per the French-American-British (FAB) criteria or APL with PML-RARA fusion gene per standard diagnostic classifications.
- Concomitant Malignancies: Diagnosis of another active malignant tumor within the screening period, except for the target AML or MDS indications (as evaluated by the investigator).
- Hyperleukocytosis: White blood cell (WBC) count > 25 × 10⁹/L during screening. Note: Hydroxyurea use is permitted to stabilize the WBC count below this threshold prior to first dose.
- Contraception: Female patients of childbearing potential, male patients, and their partners who are unwilling to practice a medically accepted, highly effective method of contraception during the treatment period and for at least 6 months following the final dose of the investigational drug. Note: Women are considered postmenopausal if they have experienced at least 12 consecutive months of amenorrhea with no alternative pathological cause.
- Prior/Concomitant Therapies:
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 |
|---|---|---|
| PBSS1113PBSS1113 enteric tablet, 10/50 mg | Drug | PBSS1113 |
Primary outcome
Composite Complete Remission (cCR) Rate in Patients with Acute Myeloid Leukemia (AML)
Time frame: Through study completion, an average of 1 year
The composite complete remission (cCR) rate is defined as the proportion of AML subjects who achieve a best overall response of complete remission (CR), complete remission with partial hematologic recovery (CRh), complete remission with incomplete hematologic recovery (CRi), or a morphological leukemia-free state (MLFS). The analysis will further evaluate the subset of patients achieving measurable residual disease (MRD) negativity within these categories (CRMRD-, CRhMRD-, or CRiMRD-).
1 secondary outcome
Overall Response Rate (ORR) in Patients with Myelodysplastic Syndrome (MDS)
Time frame: Through study completion, an average of 1 year
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 14 Aug 2026.
- Individual participant dataSharing planned: No
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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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing PBSS1113 in people with Acute Myeloid Leukemia (AML) and MDS (Myelodysplastic Syndrome), run by Chaser Therapeutics, Inc. at 1 site 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 14 inclusion and 19 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 14 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 combined 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.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 Trial Manager (+86 13071862292, CT_clinicaltrials@chasertherapeutics.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Composite Complete Remission (cCR) Rate in Patients with Acute Myeloid Leukemia (AML) (time frame: Through study completion, an average of 1 year). 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 14 Aug 2026.