NCT01351545

A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)

A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs) for Transplantation in Pediatric and Adult Patients With Hematologic Malignancies and Other Indications

• Status: Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 99,999 (estimated)
• Sponsor: Center for International Blood and Marrow Transplant Research · Network
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

This study is an access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs) in pediatric and adult patients with hematologic malignancies and other indications.

Detailed description

Principal Investigators: The principal investigators (PIs) will be transplant physicians at all participating U.S. transplant centers. Study Design: This study is an access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs) in pediatric and adult patients with hematologic malignancies and other indications. Primary Objective: The primary objective of this study is to examine the incidence of neutrophil recovery of ≥500/mm3 after cord blood transplantation in a multi-institution setting using CBUs that are not Food and Drug Administration (FDA) licensed. Secondary Objectives: In patients receiving a non-licensed CBU: * Assess incidence of transmission of infection * Assess incidence of serious infusion reaction * Determine 1 year overall survival after cord blood transplantation * Assess cumulative incidence of acute graft vs. host disease (GVHD) grades II to IV and grades III to IV * Assess cumulative incidence of chronic GVHD * Determine platelet engraftment of \>20,000 mcL and \>50,000 mcL

Conditions

• Hematologic Malignancies
• Inherited Disorders of Metabolism
• Inherited Abnormalities of Platelets
• Histiocytic Disorders
• Acute Myelogenous Leukemia (AML or ANLL)
• Acute Lymphoblastic Leukemia (ALL)
• Other Acute Leukemia
• Chronic Myelogenous Leukemia (CML)
• Myelodysplastic (MDS) / Myeloproliferative (MPN) Diseases
• Other Leukemia
• Hodgkin Lymphoma
• Non-hodgkin Lymphoma
• Multiple Myeloma/ Plasma Cell Disorder (PCD)
• Inherited Abnormalities of Erythrocyte Differentiation or Function
• Disorders of the Immune System
• Autoimmune Diseases
• Severe Aplastic Anemia

Interventions

Type	Name	Description
DRUG	A multicenter access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs)	A multicenter access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs) for transplantation in pediatric and adult patients with hematologic malignancies and other indications

Timeline

• Start date: 2011-10-01
• Primary completion: 2041-10-01
• Completion: 2041-10-01
• First posted: 2011-05-11
• Last updated: 2026-04-13

Locations

142 sites across 1 country: United States

Regulatory

• FDA-regulated drug study