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Clinical Trial 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.


Clinical Trial 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 ;


Study Design

Allocation: Non-Randomized, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment


Related Conditions & MeSH terms


NCT number NCT00744692
Study type Interventional
Source Duke University
Contact
Status Completed
Phase Phase 1
Start date October 2008
Completion date April 2014

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