Safety Study of Unlicensed, Investigational Cord Blood Units Manufactured by the NCBP for Unrelated Transplantation

Study Overview
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Brief Summary
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This study will evaluate the safety of infusion of the investigational cord blood units by carefully documenting all infusion-related problems.

Detailed Description
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The primary aim of this study is to examine the safety of administration of the unlicensed investigational NCBP hematopoietic progenitor cell-cord blood (HPC-CORD BLOOD) products in a multi-institution setting. Therefore, the study will evaluate prospectively the incidence of serious adverse reactions as well as the incidence of all infusion related reactions after administration of the unlicensed, investigational NCBP CBU. Definitions of Infusion-related adverse reactions: Mild - Moderate: reactions during or after the infusion of the cord blood (CB) product that require some medical intervention but do not affect the overall patient status or outcome. Severe: serious, life-threatening or fatal infusion reactions, requiring major medical intervention. These include: anaphylactic shock, acute cardiac, pulmonary or renal failure, seizures, patient transfer to the Intensive Care Unit, or death within 48 hours of the CB infusion. Adverse Reactions will also be classified by grade, according to the Common Terminology Criteria for Adverse Events v4.0 (CTCAE).

Official Title
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A Multicenter Safety Study of Unlicensed, Investigational Cryopreserved Cord Blood Units Manufactured by the National Cord Blood Program and Provided for Unrelated Hematopoietic Stem Cell Transplantation of Pediatric and Adult Patients

Conditions
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Infusion Reactions

Intervention / Treatment
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* Biological: unlicensed CBU


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Diagnosis: Patients with disorders affecting the hematopoietic system that are inherited, acquired, or result from myeloablative treatment. Patients: Patients of any age and either gender Cord blood product manufactured by the NCBP (at least one, if the graft contains more than one units) Exclusion Criteria: Patients who are receiving licensed cord blood products (only) Patients who are receiving unlicensed cord blood products from other banks (only) Patients who are transplanted at non-US transplant centers Patients who are receiving cord blood products that will be manipulated post-thaw (e.g., ex vivo expansion, incubation in vitro, etc.)
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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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: unlicensed CBU<br>The Principal Investigators will be the transplant physicians at participating US transplant centers | Biological: unlicensed CBU<br>* infusion of unlicensed cord blood units<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of infusion-related reactions | The primary aim of this study is to examine the safety of administration of the unlicensed investigational cord blood products in a multi-institution setting. The study will evaluate prospectively the incidence of serious adverse reactions as well as the incidence of all infusion related reactions after administration of the unlicensed, investigational NCBP cord blood units. | within 48 hours of infusion | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| engraftment | This outcome will evaluate engraftment of unlicensed cord blood units | six months after transplant | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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cord blood, transplantation, stem cells, adverse event