Not Signed In -
Niemann-Pick Disease |
Classical Niemann-Pick Disease; DAF syndrome; lipoid histiocytosis (classical phosphatide); Neuronal Cholesterol Lipidosis; NP; NPD; Ophthalmoplegia, Supraoptic Vertical; Sphingomyelin lipidosis; Sphingomyelin/cholesterol lipidosis; Sphingomyelinase deficiency |
Clinical Trial: Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation
This study is no longer recruiting patients.
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Purpose
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 |
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| 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 |
MedlinePlus related topics: Gaucher's Disease; Genetic Brain Disorders; Genetic Disorders; Infant and Toddler Health; Leukodystrophies; Metabolic Disorders; Skin Diseases
Genetics Home Reference related topics: Gaucher disease; Krabbe disease; Niemann-Pick disease
Study Type: Observational
Study Design: Screening
Expected Total Enrollment: 10
Study start: August 1999
PROTOCOL OUTLINE: 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.
Eligibility
Genders Eligible for Study: Both
Criteria
- Diagnosis of an inborn error of metabolism eligible for allogeneic hematopoietic stem cell transplantation on protocol UMN-MT-1995-01
Location Information
Minnesota
Fairview University Medical Center, Minneapolis, Minnesota, 55455, United States
K. Scott Baker, Study Chair, Fairview University Medical Center
More Information
Record last reviewed: October 2003
Last Updated: October 13, 2004
Record first received: June 2, 2000
ClinicalTrials.gov Identifier: NCT00005900
Health Authority: Unspecified
ClinicalTrials.gov processed this record on 2005-04-08
Source: ClinicalTrials.gov
Cache Date: April 9, 2005
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