NCT05569512

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

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: Terminated
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 1 (actual)
• Sponsor: Malika Kapadia · Academic / Other
• Sex: All
• Age: 12 Months – 39 Years
• Healthy volunteers: Not accepted

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

Detailed 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. On 7/26/24 the Sponsor-Investigator was notified GlycoMimetics, Inc. was terminating contracting for NCT05569512 following company restructuring. One subject was enrolled on the study prior to termination. The study did not proceed from Phase 1 to Phase 2.

Conditions

• Acute Myeloid Leukemia
• Pediatric Cancer

Interventions

Type	Name	Description
DRUG	Uproleselan	Administered by intravenous infusion
DRUG	Fludarabine	Administered by intravenous infusion
DRUG	Clofarabine	Administered by intravenous infusion
DRUG	Busulfan	Administered by intravenous infusion

Timeline

• Start date: 2022-10-06
• Primary completion: 2023-10-05
• Completion: 2023-12-06
• First posted: 2022-10-06
• Last updated: 2024-08-19

Locations

5 sites across 1 country: United States

Regulatory

• FDA-regulated drug study