Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation
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|ClinicalTrials.gov Identifier: NCT00005900|
Recruitment Status : Unknown
Verified October 2003 by Office of Rare Diseases (ORD).
Recruitment status was: Active, not recruiting
First Posted : June 5, 2000
Last Update Posted : June 24, 2005
OBJECTIVES: I. Evaluate bronchoalveolar lavage fluid and serum obtained from pediatric patients with storage disorders prior to allogeneic hematopoietic stem cell transplantation (HSCT) for the presence of proinflammatory cytokines and for the production of nitric oxide by alveolar macrophages to identify possible risk factors for pulmonary complications.
II. Investigate the underlying mechanism for the development of significant pulmonary complications in these patients during HSCT.
III. Evaluate bronchoalveolar lavage fluid and serum obtained from these same patients at the time a pulmonary complication develops post-HSCT, or at 60 days post-HSCT if there has been no pulmonary complications.
|Condition or disease|
|I Cell Disease Fucosidosis Globoid Cell Leukodystrophy Adrenoleukodystrophy Mannosidosis Niemann-Pick Disease Pulmonary Complications Mucopolysaccharidosis I Mucopolysaccharidosis VI Metachromatic Leukodystrophy Gaucher's Disease Wolman Disease|
Patients undergo bronchoscopy with bronchoalveolar lavage (BAL) prior to allogeneic hematopoietic stem cell transplantation (HSCT). ELISA assays for cytokines are performed. Patients are followed post-HSCT for the development of transplant related pulmonary complications. A repeat bronchoscopy with BAL is performed at the time pulmonary complications develop or at day 60 post-HSCT if no complications develop. Cytokine assays are repeated.
|Study Type :||Observational|
|Estimated Enrollment :||10 participants|
|Study Start Date :||August 1999|
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00005900
|United States, Minnesota|
|Fairview University Medical Center|
|Minneapolis, Minnesota, United States, 55455|
|Study Chair:||K. Scott Baker||Fairview University Medical Center|