Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders
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|ClinicalTrials.gov Identifier: NCT00744692|
Recruitment Status : Completed
First Posted : September 1, 2008
Results First Posted : August 13, 2014
Last Update Posted : August 13, 2014
|Condition or disease||Intervention/treatment||Phase|
|Non Malignant Disorders Immunodeficiencies Congenital Marrow Failures Hemoglobinopathies Inborn Errors of Metabolism Sickle Cell Thalassemia Lysosomal Storage Disease||Biological: Unrelated Umbilical Cord Blood Transplant Drug: Reduced Intensity Conditioning||Phase 1|
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 Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||22 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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|
|Study Start Date :||October 2008|
|Actual Primary Completion Date :||December 2012|
|Actual Study Completion Date :||April 2014|
Experimental: RIC Cord Blood Transplant
Reduced Intensity Conditioning for Umbilical Cord Blood Transplant
Biological: Unrelated Umbilical Cord Blood Transplant
Reduced Intensity Conditioning for unrelated umbilical cord blood transplant
Drug: Reduced Intensity Conditioning
- Determine the Feasibility of Attaining Acceptable Rates of Donor Cell Engraftment (>25% Donor Cells at 180 Days) Following RIC Regimens in Children < 21 Years Receiving UCBT for Non-malignant Disorders. [ Time Frame: 180 days post transplant ]Determine the feasibility of attaining acceptable rates of donor cell engraftment (>25% donor cells at 180 days) following reduced intensity conditioning regimens in children < 21 years receiving cord blood transplant for non-malignant disorders.
- To Describe the Pace of Neutrophil Recovery [ Time Frame: 42 days post transplant ]Neutrophil recovery was defined as the first day of an absolute neutrophil count (ANC) more than 500/uL for 3 consecutive days not secondary to granulocyte infusions
- To Evaluate the Pace of Immune Reconstitution. [ Time Frame: 1 year post transplant ]Immune reconstitution after RIC in UCBT was described. CD4 count is a standard measure of immune reconstitution and is described here. Additional data is available upon request.
- To Determine the Overall Survival at day180 Post-transplant [ Time Frame: 180 days ]To determine the overall survival at day180 post-transplant: determined by Kaplan Meier survival analysis
- To Describe Incidence of Acute Graft Versus Host Disease (GVHD) (II - IV) [ Time Frame: 100 days post transplant ]To describe incidence of acute Graft Versus Host Disease (GVHD) (II - IV) : measured by cumulative incidence analysis
- To Describe the Incidence of Grade 3-4 Organ Toxicity [ Time Frame: 2 years post transplant ]
- To Evaluate Long-term Complications, Such as Sterility, Endocrinopathy, and Growth Failure [ Time Frame: at least 2 years post transplant ]
- To Evaluate the Incidence of Late Graft Failures at 2 Years Post-transplant [ Time Frame: 2 years post transplant ]
- To Describe the Pace of Platelet Recovery [ Time Frame: 180 days post transplant ]Platelet engraftment was defined as the first day of platelet counts more than 50,000/uL for 7 consecutive days without transfusions
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00744692
|United States, North Carolina|
|Duke University Medical Center Pediatric Blood and Marrow Transplant Program|
|Durham, North Carolina, United States, 27705|
|Principal Investigator:||Suhag Parikh, MD||Duke Pediatric Blood and Marrow Transplant|