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
Overview
- Phase
- Not Applicable
- Status
- Recruiting
- Enrollment
- 99,999
- Locations
- 257
- Primary Endpoint
- Neutrophil recovery of ≥500/mm3 after cord blood transplantation
Overview
Brief Summary
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
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
Study Design
- Study Type
- Observational
- Observational Model
- Other
- Time Perspective
- Prospective
Eligibility Criteria
- Sex
- All
- Accepts Healthy Volunteers
- No
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
Arms & Interventions
Unlicensed CBU
The cohort includes recipients of any age receiving unlicensed cryopreserved cord blood units (CBUs) for designated indications.
Intervention: A multicenter access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs) (Drug)
Outcomes
Primary Outcomes
Neutrophil recovery of ≥500/mm3 after cord blood transplantation
Time Frame: Cord blood engraftment occurs in weeks following transplant is defined as defined as ANC 500/mm3 and is analyzed at 100 days post-transplant.
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.
Neutrophil recovery of ≥500/mm3 after cord blood transplantation
Time Frame: Cord blood engraftment occurs in weeks following transplant is defined as defined as ANC 500/mm3 and is analyzed at 60 days post-transplant.
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.
Secondary Outcomes
No secondary outcomes reported