A multicenter safety study of unlicensed investigational cryopreserved cord blood units (CBUs) manufactured by the National Cord Blood Program (NCBP) and provided for unrelated hematopoietic stem cell transplantation of pediatric and adult patients

Study on Investigational Cord Blood Units

J
Jodi Skiles

Primary Investigator

Enrolling By Invitation
100 years or below
All
Phase N/A
2 Locations

Brief description of study

The purpose of this study is to evaluate the safety of administration of the investigational cord blood units by carefully documenting all infusion-related problems.
 
THIS STUDY IS ENROLLING BY INVITATION ONLY - The study will enroll an unlimited number of transplant recipients at participating US transplant centers (up to 10 at Indiana University) based on well-defined inclusion and exclusion criteria, when the best graft source is determined to be CBU manufactured by the NCBP that does not meet FDA licensure requirements (HPC-CORD BLOOD product). 

Detailed description of study

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.
 
All recipients receiving at least one unlicensed investigational NCBP HPC-CORD BLOOD product will be enrolled in the study and will be assessed for: any infusion-related reaction, serious infusion adverse events, transmission of infectious diseases from the CBU, graft rejection, neutrophil engraftment, survival, acute and chronic GVHD, platelet engraftment, and CBU-derived engraftment.
 

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).

Eligibility of study

You may be eligible for this study if you meet the following criteria:

  • Conditions: Infusion Reactions, Blood Transfusion, Cancer, Riley
  • Age: 100 years or below
  • Gender: All

Inclusion Criteria:

  1. Diagnosis: Patients with disorders affecting the hematopoietic system that are inherited, acquired, or result from myeloablative treatment.
  2. Patients: Patients of any age and either gender
  3. Cord blood product manufactured by the NCBP (at least one, if the graft contains more than one units)

Exclusion Criteria:

  1. Patients who are receiving licensed cord blood products (only)
  2. Patients who are receiving unlicensed cord blood products from other banks (only)
  3. Patients who are transplanted at non-US transplant centers
  4. Patients who are receiving cord blood products that will be "manipulated" post-thaw (e.g., ex vivo expansion, incubation in vitro, etc.)
     

This study investigates the safety of using investigational cord blood units (CBUs) for hematopoietic stem cell transplants in both children and adults. Hematopoietic stem cells are important for creating new blood cells. The study will carefully look at any problems that happen during or after the infusion of these CBUs.

Participants will receive at least one investigational CBU and will be monitored for any infusion-related reactions. The study will track serious adverse reactions, infections from the CBUs, and various outcomes like graft rejection and survival. Infusion reactions are classified as mild to severe, with severe reactions needing major medical help.

  • Who can participate: This study is open to patients of any age with disorders affecting the blood system who must receive at least one cord blood product from the NCBP.
  • Study details: Participants will receive an investigational cord blood product and be observed for safety outcomes. The study will monitor for reactions and other health effects after receiving the product.
Updated on 11 Dec 2025. Study ID: 6637-01, 1207009172
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Interested in the study?

This study is accepting only persons who receive care at a certain clinic or doctor or who are part of an invited group. Questions about this study can be directed to the study team listed in the description or contact your doctor to see if you are eligible.

Accepting Referrals by Invitation Only