NCT00692926

Unrelated Umbilical Cord Blood Transplantation Augmented With ALDHbr Umbilical Cord Blood Cells

A Pilot Trial of Unrelated Umbilical Cord Blood Transplantation Augmented With Ex Vivo CytokinePrimed ALDHbr Umbilical Cord Blood Cells

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 37 (actual)
• Sponsor: Joanne Kurtzberg, MD · Academic / Other
• Sex: All
• Age: 55 Years
• Healthy volunteers: Not accepted

Summary

The main purpose of this investigational (not approved by the FDA) Phase I research is to test whether transplantation of umbilical cord blood cells can be safely supplemented with a transfusion of a portion of these cells that have been sorted (collected from a special machine called a cell sorter) and then either infused a few hours after the standard transplant or for some patients grown in a special system in the laboratory prior to the transplant, designed to increase the number of stem cells transplanted. This system is currently in the early phases of testing.

Detailed description

The main purpose of this study is to test whether transplantation of umbilical cord blood cells can be safely supplemented with transfusion of a portion of these cells that have been grown in a special system (designed to increase the number of cells transplanted) in the laboratory prior to the transplant. This system is currently in the early phases of testing in a FDA-IND-sponsored clinical trial. If the patient consents to participate in this study, approximately 1/5th (20%) of the cord blood unit selected for the transplant will be treated per protocol. The first 3 patients will receive ALDHbr sorted cells but not primed in culture. This is to test the safety of the ALDHbr cells. The treated cells will be given to the patient on the day of transplant approximately 4 hours after the standard or conventional transplant which will be given from the 80% fraction of the cord blood unit. A total of 26 evaluable patients are to be enrolled as outlined below (protocol has been amended to allow this enrollment): * 10 evaluable patients who received ALDHbr freshly sorted cells (20% portion) * 10 evaluable patients who received ALDHbr sorted and cytokine primed cells (20% portion) * 3 evaluable patients who received a conventional cord blood unit and a cord blood unit that has been ALDHbr sorted (sort of the UCB unit will be done on Day -1 due to the time it takes to actually perform the sort) * 3 evaluable patients who received a conventional cord blood unit and a cord blood unit that has been ALDHbr sorted and cytokine primed (sort and priming will be done on day -5 as described later in the protocol)

Conditions

• MDS
• Anemia, Aplastic
• Inborn Errors of Metabolism
• Congenital Marrow Failure
• Congenital Immunodeficiency Syndrome

Interventions

Type	Name	Description
BIOLOGICAL	ALDHbr Umbilical Cord Blood Cells	ALDHbr sorted Umbilical Cord Blood Cells

Timeline

• Start date: 2005-10-01
• Primary completion: 2012-07-01
• Completion: 2012-07-01
• First posted: 2008-06-06
• Last updated: 2017-06-29

Locations

1 site across 1 country: United States