Clinical trial NCT00744692
Conditions: Non Malignant Disorders Immunodeficiencies Congenital Marrow Failures Hemoglobinopathies Inborn Errors of Metabolism Sickle Cell Thalassemia Lysosomal Storage Disease
Keywords: Immunodeficiencies Congenital Marrow Failures Hemoglobinopathies Inborn Errors of Metabolism SCIDS Wiskott Aldrich FEL HLH IPEX LAD Sickle Cell Thalassemia Omenn's Syndrome Hurler's Syndrome MLD ALD Sanfilippo Krabbe Hunter's syndrome TaySachs Diamond Blackfan Anemia transplant MPS Gaucher
Sponsor: Duke University
Start date: 2008-10-01; Completion date: 2014-04-01
Enrollment: 22 participants; Allocation: NON_RANDOMIZED; Intervention model: SINGLE_GROUP

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

Arms: arm 1: Reduced Intensity Conditioning for Umbilical Cord Blood Transplant
Interventions: intervention 1: Unrelated Umbilical Cord Blood Transplant intervention 2: Reduced Intensity Conditioning
Intervention descriptions: intervention 1: Reduced Intensity Conditioning for unrelated umbilical cord blood transplant intervention 2: None

Protocol documents: