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.
Brief Title
A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
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
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
Categories
Central Contacts
Central Contact Role
Contact
Central Contact Email
10-CBA@ndmp.org
Completion Date
Completion Date Type
Estimated
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
Eligibility Criteria
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
* 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
Inclusion Criteria
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
* 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
Gender
All
Gender Based
false
Healthy Volunteers
No
Last Update Post Date
Last Update Post Date Type
Actual
Last Update Submit Date
NCT Id
NCT01351545
Org Class
Network
Org Full Name
Center for International Blood and Marrow Transplant Research
Org Study Id
10-CBA
Overall Status
Recruiting
Primary Completion Date
Primary Completion Date Type
Estimated
Official Title
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
Primary Outcomes
Outcome Description
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.
Outcome Measure
Neutrophil recovery of ≥500/mm3 after cord blood transplantation
Outcome 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.
Outcome Description
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.
Outcome Measure
Neutrophil recovery of ≥500/mm3 after cord blood transplantation
Outcome 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.
Start Date
Status Verified Date
First Post Date
First Post Date Type
Estimated
First Submit Date
First Submit QC Date
Study Population
Recipients of unlicensed cryopreserved cord blood units who are being treated as U.S. transplant centers.
Std Ages
Child
Adult
Older Adult
Locked Fields
Render the field
Maximum Age Number (converted to Years and rounded down)
999
Minimum Age Number (converted to Years and rounded down)
0
Investigators
Investigator Type
Principal Investigator
Investigator Name
David Loeb
Investigator Email
david.loeb@einsteinmed.org
Investigator Phone
718-741-2342