UPCC 04711 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

Investigating Cord Blood Transplantation for Blood Conditions

Enrolling By Invitation
All
Phase N/A
99999 participants needed
1 Location

Brief description of study

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 of study

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

Eligibility of study

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

  • 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, 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
  • Gender: All

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

This study investigates the use of unlicensed cryopreserved cord blood units (CBUs) for transplantation in pediatric and adult patients with hematologic malignancies and other conditions. The purpose of this study is to understand how well patients recover neutrophil levels, a type of white blood cell important for fighting infections, after receiving these CBUs.

Participants in this study will undergo cord blood transplantation using CBUs that are not licensed by the Food and Drug Administration (FDA). The study will monitor various outcomes, including the incidence of infection transmission, serious infusion reactions, overall survival rates one year post-transplant, and the development of graft vs. host disease (GVHD). Additionally, the study will assess the recovery of platelet levels, which are crucial for blood clotting.

  • Who can participate: This study is open to pediatric and adult patients of any age with disorders affecting the blood system, whether inherited, acquired, or due to myeloablative treatment. Participants must provide signed informed consent.
  • Study details: Participants will receive cord blood transplants with unlicensed CBUs. The study will track health outcomes such as infection rates and survival. It will also evaluate the development of graft vs. host disease and the recovery of blood cell levels.
Updated on 06 Jul 2026. Study ID: 11-0050
If you need assistance finding a non-cancer clinical research study or if you have any questions, please email psom-ocr@pobox.upenn.edu For Cancer trials contact Penn Medicine's Cancer Trial Navigator at: PMCancerResearch@pennmedicine.upenn.edu or 215-349-8245

Study is selecting its participants from a population, or group of people, decided on by the researchers in advance.

Contact Abramson Cancer Center Navigator