PBSS1113 in Combination With Azacitidine to Treat Patients With AML/MDS (NCT07761533) | Clinical Trial Compass
RecruitingPhase 1/2
PBSS1113 in Combination With Azacitidine to Treat Patients With AML/MDS
China29 participantsStarted 2025-09-08
Plain-language 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. 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.
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* 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.
* 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 t…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Composite Complete Remission (cCR) Rate in Patients with Acute Myeloid Leukemia (AML)