SL-401

SL-401 works by targeting leukemia cells (blasts), and also possibly by stopping or slowing the growth of cancer stem cells, which are the undeveloped cells which can develop into cancer cells.

Venetoclax is a BH3-mimetic. Venetoclax blocks the anti-apoptotic B-cell lymphoma-2 (Bcl-2) protein, leading to programmed cell death of CLL cells. Overexpression of Bcl-2 in some lymphoid malignancies has sometimes shown to be linked with increased resistance to chemotherapy.

This research study is a Phase I clinical trial, which tests the safety of an investigational intervention and also tries to define the appropriate dose of the investigational intervention to use for further studies. "Investigational" means that the intervention is being studied. The FDA (the U.S. Food and Drug Administration) has approved azacitidine and venetoclax as a treatment option for AML.

This research study is studying a drug as a possible treatment for diagnosis of AML, BPDCN and high-risk MDS. The interventions involved in this study are: * SL-401 * Azacitidine * Venetoclax

Live record on ClinicalTrials.gov →

• Histologically confirmed diagnosis of acute myeloid leukemia (AML) \[Cohort B\] or myelodysplastic syndrome (MDS) \[Cohort A\] or BPDCN \[Cohort C\] per 2016 WHO criteria
• CD123 / IL3RA expression on the subject's AML or MDS blasts or BPDCN cells determined locally within 3 months of first protocol treatment
• Age \>= 18 years with relapsed or refractory AML (hydroxyurea is not considered a prior treatment regimen) \[Cohort B\]
• Age \>= 18 years with treatment-naïve AML who decline intensive induction chemotherapy or who are unfit due to co-morbidity or other factors (see APPENDIX A for unfitness definitions) (hydroxyurea is not considered a prior treatment regimen) \[Cohort B\]
• Age \>= 18 years with MDS and \> 10% myeloblasts in the bone marrow \[Cohort A\]
• Age \>= 18 years with relapsed or refractory BPDCN (hydroxyurea is not considered a prior treatment regimen) \[Cohort C\]

Inclusion criteria as written on the registry, trimmed. The full list, with exclusions, is on the live record.