Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders

Thalassemia Clinical Trial

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

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

• Congenital Marrow Failures
• Hemoglobinopathies
• Immunodeficiencies
• Immunologic Deficiency Syndromes
• Inborn Errors of Metabolism
• Lysosomal Storage Disease
• Lysosomal Storage Diseases
• Metabolism, Inborn Errors
• Non Malignant Disorders
• Sickle Cell
• Thalassemia

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