NCT03113643

SL-401 in Combination With Azacitidine or Azacitidine/Venetoclax in Acute Myeloid Leukemia (AML), High-Risk Myelodysplastic Syndrome (MDS) or Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)

Phase 1 Study of SL-401 in Combination With Azacitidine and Venetoclax in Relapsed/Refractory Acute Myeloid Leukemia (AML) and in Treatment-Naive Subjects With AML Not Eligible for Standard Induction and in Subjects With Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) or SL-401 in Combination With Azacitidine in Subjects With High-Risk Myelodysplastic Syndrome (MDS)

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 72 (estimated)
• Sponsor: Dana-Farber Cancer Institute · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

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

Detailed description

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. However, the combination of these two drugs with SL-401 has not been FDA approved. The combination of SL-401, azacitidine and venetoclax has not been FDA approved for BPDCN. However, SL-401 has been FDA approved for BPDCN. The combination of SL-401 and azacitidine has not been FDA approved for BPDCN. In this research study, the study drug SL-401 will be combined with the standard dose of azacitidine (for MDS patients) or azacitidine/venetoclax (for AML and BPDCN patients). The goal of this research study is to try and determine the safest, highest dose of study drug, SL-401, in combination with azacitidine or azacitidine/venetoclax that can be given to patients with AML, BPDCN or high-risk MDS. SL-401 works by stopping or slowing the growth of cancer stem cells, which are the undeveloped cells which can develop into cancer cells. The goals of this research study are to look at if this combination works to help treat your cancer and if there is any lasting effect of this combination. This study will also look at how the SL-401, in combination with azacitidine or azacitidine/venetoclax, affects certain proteins in your blood and bone marrow. SL-401 has been given to patients with AML, and MDS in the past, but this is the first time it will be given in combination with another drug.

Conditions

• Acute Myeloid Leukemia
• Myelodysplastic Syndrome
• Blastic Plasmacytoid Dendritic Cell Neoplasm

Interventions

Type	Name	Description
DRUG	Azacitidine	Chemotherapy
DRUG	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.
DRUG	Venetoclax	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.

Timeline

• Start date: 2017-06-26
• Primary completion: 2026-05-31
• Completion: 2027-05-31
• First posted: 2017-04-13
• Last updated: 2025-11-04

Locations

3 sites across 1 country: United States

Regulatory

• FDA-regulated drug study