Ignite Creation Date:
2025-12-24 @ 3:25 PM
Ignite Modification Date:
2026-01-03 @ 4:36 AM
Study NCT ID:
NCT00744692
Status:
COMPLETED
Last Update Posted:
2014-08-13
First Post:
2008-08-28
Is NOT Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders
Sponsor:
Duke University
Organization:
Study Overview
Official Title:
A Pilot Study of Reduced Intensity Conditioning in Pediatric Patients <21 Years of Age With Non-Malignant Disorders Undergoing Umbilical Cord Blood Transplantation
Status:
COMPLETED
Status Verified Date:
2014-07
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
The primary objective is to determine the feasibility of attaining acceptable rates of donor cell engraftment (\>25% donor chimerism at 180 days) following reduced intensity conditioning (RIC) regimens in pediatric patients \< 21 years receiving cord blood transplantation for non-malignant disorders.
Detailed Description:
Myeloablative doses of chemotherapy and/or radiation therapy are employed with the primary purpose of eradicating malignant cells. Additionally, these regimens exert varying degree of immunosuppression/immunoablation that aids in reducing the likelihood of rejection by host hematopoietic cells. However, myeloablative /immunoablative regimens have also been associated with significant regimen related toxicity (RRT) and regimen related mortality (RRM) that may cause death in up to 20% of patients and significantly higher rate of severe organ dysfunction or failure. While most of these RRT occur typically in the first 100 days \[ e.g. VOD (veno occlusive disease), pulmonary or intracranial hemorrhage, multiorgan failure (MOF)\], there are significant long term toxicities of TBI and/or chemotherapy including growth impairment, gonadal dysfunction/failure, hypothyroidism, cataracts, neurocognitive impairment, and second malignancies.
The primary objective is to determine the feasibility of attaining acceptable rates of donor cell engraftment (\>25% donor chimerism at 180 days) following reduced intensity conditioning (RIC) regimens in pediatric patients \< 21 years receiving cord blood transplantation for non-malignant disorders.
The secondary objectives are:
* To describe the pace of neutrophil and platelet recovery
* To evaluate the pace of immune reconstitution.
* To determine the treatment related mortality, overall survival and disease free survival by days 100 and 180 post-transplant
* To describe incidence of acute Graft Versus Host Disease (GVHD) (II - IV) and chronic extensive GVHD
* To describe the incidence of grade 3-4 organ toxicity
* To evaluate long-term complications, such as sterility, endocrinopathy, and growth failure
* To evaluate the incidence of late graft failures at 2 years post-transplant
The primary objective is to determine the feasibility of attaining acceptable rates of donor cell engraftment (\>25% donor chimerism at 180 days) following reduced intensity conditioning (RIC) regimens in pediatric patients \< 21 years receiving cord blood transplantation for non-malignant disorders.
The secondary objectives are:
* To describe the pace of neutrophil and platelet recovery
* To evaluate the pace of immune reconstitution.
* To determine the treatment related mortality, overall survival and disease free survival by days 100 and 180 post-transplant
* To describe incidence of acute Graft Versus Host Disease (GVHD) (II - IV) and chronic extensive GVHD
* To describe the incidence of grade 3-4 organ toxicity
* To evaluate long-term complications, such as sterility, endocrinopathy, and growth failure
* To evaluate the incidence of late graft failures at 2 years post-transplant
Study Oversight
Has Oversight DMC:
False
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: