Clinical trial · Interventional
A Study of APTO-253 in Patients With Relapsed or Refractory AML or MDS
A Phase Ia/b Dose Escalation and Expansion, Multicenter, Open-label, Safety, Pharmacokinetic and Pharmacodynamic Study of APTO-253 in Patients With Relapsed or Refractory Acute Myelogenous Leukemia or High-Risk Myelodysplasia
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Why stopped (as posted): Drug manufacturing process and procedure review
Summary
Brief summary (as posted)
This study is being done to evaluate the safety and effectiveness of APTO-253 for the treatment of patients with the condition of acute myelogenous leukemia (AML) or myelodysplastic syndrome (MDS) for which either the standard treatment has failed, is no longer effective, or can no longer be administered safely or poses a risk for your general well being.
Conditions
Conditions (5)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Acute Myelogenous Leukemia | Acute Myeloid Leukemia | ALIAS | 0.90 |
| Acute Myelogenous Leukemia, Adult | Adult Acute Myeloid Leukemia | ALIAS | 0.90 |
| Acute Myelogenous Leukemia in Relapse | Acute Myeloid Leukemia | CURATED_BROADER | 0.78 |
| Acute Myelogenous Leukemia, Relapsed, Adult | Acute Myeloid Leukemia | ALIAS | 0.85 |
| High Risk Myelodysplasia | — | UNRESOLVED | — |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| APTO-253 | Drug | — | UNRESOLVED |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- Dose Escalation and Expansion
- description
- APTO-253 will be given in ascending doses in patients with relapsed or refractory AML or high risk MDS (escalation cohort), until the maximum tolerated dose or recommended dose is reached. Followed by up to 30 patients enrolled in the expansion cohort at the recommended dose.
- interventionNames
- Drug: APTO-253
Primary outcomes (3)
- measure
- Incidence of treatment-emergent adverse events of APTO-253
- timeFrame
- Cycle 1 (28 days)
- description
- To determine the safety and tolerability of APTO-253 by assessing treatment-related adverse events as assessed by CTCAE v4.0.
- measure
- Maximum tolerated dose and dose limiting toxicities
- timeFrame
- Cycle 1 (28 days)
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
Show eligibility criteria text
Inclusion Criteria: * Patients ≥18 years old * Life expectancy of at least 2 months * Off previous cancer therapy for at least 14 days, or 5 half-lives for noncytotoxic agents prior to first study treatment administration * Patients must have a calculated creatinine clearance \>60 mL/min * Acceptable hematologic, renal and liver functions and coagulation status parameters Exclusion Criteria: * Patients with GVHD requiring systemic immunosuppressive therapy * Uncontrolled leptomeningeal disease, auto-immune hemolytic anemia and uncontrolled and clinical significant disease related metabolic disorder * Clinically significant intravascular coagulation * Treatment with other investigational drugs within 14 days prior to first study treatment administration
References
Publications (0)
Data not yet available