PBSS1113 in Combination With Azacitidine to Treat Patients With AML/MDS
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorChaser Therapeutics, Inc.
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Age and Gender: Age ≥ 18 years at the time of signing the informed consent form, male or female.
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.
Disqualifiers
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.
Trial design
Design model
Sequential
Treatments tested in this trial
PBSS1113
DrugPBSS1113 enteric tablet, 10/50 mg
Treatment groups
29 Participants
are divided into 1 treatment groupGroup A: PBSS1113Experimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
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-).
Time frame
Through study completion, an average of 1 yearSecondary 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).
Time frame
Through study completion, an average of 1 yearSponsors and contacts
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Source ClinicalTrials.gov