Uproleselan With Pre-Transplant Conditioning in Hematopoietic Stem Cell Transplantation for AML

Acute Myeloid Leukemia Clinical Trial

Official title: A Phase 1/2 Trial of Uproleselan Combined With High Dose Busulfan Pre-Transplant Conditioning in Hematopoietic Stem Cell Transplantation for Patients With Chemotherapy Resistant Acute Myeloid Leukemia

Status Recruiting

Clinical Trial Summary

This research study is studying a new drug, uproleselan, to see if it is safe and effective in decreasing relapse after stem cell transplant and improving leukemia-free survival in pediatric patients with acute myeloid leukemia (AML).

The name of the study drugs involved in this study are:

• Uproleselan

• Busulfan

• Clofarabine

• Fludarabine

• Tacrolimus

• Methotrexate

• Mycophenolate Mofetil

Clinical Trial Description

This is a single arm, multi-center, phase 1/2 trial involving the use of the study drug, uproleselan, as part of the pre stem cell transplant conditioning regimen for pediatric patients with acute myeloid leukemia (AML). This study is looking to learn what dose of uproleselan should be given and the safety of uproleselan when combined with other drugs as part of the pre stem cell transplant conditioning regimen.

The U.S. Food and Drug Administration (FDA) has not approved uproleselan as a treatment for any disease. This is the first time that uproleselan will be given to children. Uproleselan is expected to treat acute myeloid leukemia (AML) by making AML cells sensitive to chemotherapy drugs that are part of standard of care pre-transplant conditioning regimen which could help make the transplant more effective..The standard of care conditioning regimen will include the drugs busulfan, clofarabine, and fludarabine. The standard of care drugs tacrolimus, and either methotrexate or mycophenolate mofetil will be used during the stem cell transplant course.

The research study procedures include screening for eligibility and study treatment including evaluations and follow up visits.

Participants will receive study drug doses for 7 days before their stem cell transplant and will be followed for 2 years following their stem cell transplant.

It is expected that about 28 people will take part in this research study.

GlycoMimetics, Inc., a pharmaceutical company, is supporting this research study by providing the study drug (uproleselan) and funding for some of the laboratory tests. ;

Related Conditions & MeSH terms

• Acute Myeloid Leukemia
• Leukemia
• Leukemia, Myeloid
• Leukemia, Myeloid, Acute
• Pediatric Cancer

• NCT number: NCT05569512
• Study type: Interventional
• Source: Dana-Farber Cancer Institute
• Contact: Mary Kate Czepiel
• Phone: 617-632-5915
• Email: UPROTRIAL@PARTNERS.ORG
• Status: Recruiting
• Phase: Phase 1/Phase 2
• Start date: October 6, 2022
• Completion date: December 1, 2028