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

Center for International Blood and Marrow Transplant Research (CIBMTR)

Status: Enrolling

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

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

Study type: Observational

Funder types: Other; NETWORK

Identifiers

NCT01351545

10-CBA

Details and patient eligibility

About

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.

Full 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

Enrollment: 99,999 estimated patients

Sex: All

Volunteers: No Healthy Volunteers

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

Trial design

99,999 participants in 1 patient group

Unlicensed CBU

Description:

The cohort includes recipients of any age receiving unlicensed cryopreserved cord blood units (CBUs) for designated indications.

Treatment:

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