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

Study Overview
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Brief Summary
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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
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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

Official Title
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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

Conditions
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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

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


Participation Criteria
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Eligibility Criteria
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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
Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Unlicensed CBU<br>The cohort includes recipients of any age receiving unlicensed cryopreserved cord blood units (CBUs) for designated indications. | Drug: A multicenter access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs)<br>* 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<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Neutrophil recovery of ≥500/mm3 after cord blood transplantation | 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. | Cord blood engraftment occurs in weeks following transplant is defined as defined as ANC 500/mm3 and is analyzed at 60 days post-transplant. | 
| Neutrophil recovery of ≥500/mm3 after cord blood transplantation | 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. | Cord blood engraftment occurs in weeks following transplant is defined as defined as ANC 500/mm3 and is analyzed at 100 days post-transplant. |