NCT02007863

Umbilical Cord Blood (UCB) Transplantation in Pediatric Patients With High Risk Leukemia and Myelodysplasia

Treatment Protocol: Umbilical Cord Blood (UCB) Transplantation in Pediatric Patients With High Risk Leukemia and Myelodysplasia Using Conditioning Regimen Without Radiation

• Status: Completed
• Phase: N/A
• Study type: Interventional
• Enrollment: 2 (actual)
• Sponsor: University of Miami · Academic / Other
• Sex: All
• Age: 21 Years
• Healthy volunteers: Not accepted

Summary

Unrelated Cord Blood (UCB) transplant in children is a viable stem cell transplant modality for patients with leukemia and myelodysplasia. UCB is now considered "Standard Of Care" in cases where a suitable living bone marrow donor is not available. The survival of UCB is similar to Matched Unrelated Marrow Transplant. This study is considered "Research" since UCB is not a licensed product and requires investigational new drug (IND). THERE ARE NO SPECIFIC RESEARCH QUESTIONS IN THIS PROTOCOL. This protocol merely provides UCB as a stem cell treatment modality to pediatric patients who may require it after a conditioning regimen that excludes Total Body Irradiation.

Detailed description

The preparative regimen will consist of: * Fludarabine: 25 mg/m2/day IV x 5 doses on Days -13, to -9 * Busulfan 1mg/kg IV every 6 hrs x 16 doses on Days -8 to -5 * Melphalan 45 mg/m2/day IV x 3 doses on days -4 to -2 * ATGAM 30mg/kg/day x 3 doses on Days -3 to -1 * Day 0 will be the day of the UCB Transplant * The graft-versus-host-disease (GVHD) prophylaxis will be Cyclosporin A to maintain level 200-400 beginning on Day -3, through 200-400. Solumedrol at 1mg/kg/day on Day 1 until D+4, then solumedrol 2mg/kg/day until Day +19 or till absolute neutrophil count (ANC) reaches 500/mm2, then taper by 0.2 mg/kg/week.

Conditions

• Leukemia
• Myelodysplasia
• Acute Lymphocytic Leukemia
• Acute Myelogenous Leukemia

Interventions

Type	Name	Description
PROCEDURE	Umbilical Cord Blood Transfusion	Following the administration of the preparative therapy, all subjects will undergo UCB transplantation. Umbilical Cord Blood Transfusion will occur on Day 0
DRUG	Fludarabine	Fludarabine 25 mg/m2/day will be administered over 30-60 minutes intravenous infusion on Days -13 through -9 for a total of 5 doses. Fludarabine will not be dose adjusted for body weight.
DRUG	Busulfan	Busulfan IV (Busulfex) will be administered IV every 6 hours on days -8 through -5 for a total of 16 doses. Seizure prophylaxis prior to first dose of busulfan till Day -3 will be administered.
DRUG	Melphalan	Melphalan 45 mg/m2/day will be administered over 60 minutes intravenous infusion on Days -4 through -2 for a total of 3 doses.

Timeline

• Start date: 2008-08-01
• Primary completion: 2014-06-01
• Completion: 2014-06-01
• First posted: 2013-12-11
• Last updated: 2015-12-28
• Results posted: 2015-12-28

Locations

2 sites across 1 country: United States