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

Recruiting

• 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
• Interventions: Drug: A multicenter access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs)

• Registration Number: NCT01351545
• Lead Sponsor: Center for International Blood and Marrow Transplant Research

Brief 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

Study Timeline

• Study First Submitted: 2011-05-09
• Study First Submitted QC: 2011-05-10
• Study First Posted: 2011-05-11
• Study Start: 2011-10
• Status Verified: 2025-01
• Last Update Submitted: 2025-04-15
• Last Update Posted: 2025-04-17
• Primary Completion: 2041-10
• Study Completion: 2041-10

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 99999

Inclusion Criteria

• Disorders affecting the hematopoietic system that are inherited, acquired, or result from myeloablative treatment
• Signed informed consent (and signed assent, if applicable) obtained prior to study enrollment
• Pediatric and adult patients of any age

Exclusion Criteria

• Patients who are receiving only licensed CBUs
• Cord blood transplant recipients at international transplant centers
• Patients who are enrolled on another IND protocol to access the unlicensed CBU(s)
• Patients whose selected unlicensed CBU(s) will be more than minimally manipulated

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Unlicensed CBU	A multicenter access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs)	The cohort includes recipients of any age receiving unlicensed cryopreserved cord blood units (CBUs) for designated indications.

Primary Outcome Measures

Name	Time	Method
Neutrophil recovery of ≥500/mm3 after cord blood transplantation	Cord blood engraftment occurs in weeks following transplant is defined as defined as ANC 500/mm3 and is analyzed at 100 days post-transplant.	The primary objective of this access and distribution protocol 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 FDA licensed.

Trial Locations

Locations (141)

University of Alabama at Birmingham; 🇺🇸 Birmingham, Alabama, United States

Banner MD Anderson Cancer Center; 🇺🇸 Gilbert, Arizona, United States

Phoenix Children's Hospital; 🇺🇸 Phoenix, Arizona, United States

Mayo Clinic Arizona (adults); 🇺🇸 Phoenix, Arizona, United States

University of Arizona Medical Center - Tucson; 🇺🇸 Tucson, Arizona, United States

Arkansas Children's Hospital; 🇺🇸 Little Rock, Arkansas, United States

City of Hope; 🇺🇸 Duarte, California, United States

Children's Hospital of Los Angeles; 🇺🇸 Glendale, California, United States

University of California San Diego; 🇺🇸 La Jolla, California, United States

Scripps Green Hospital; 🇺🇸 LaJolla, California, United States

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