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Clinical Trial Summary

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.


Clinical Trial Description

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. ;


Study Design

Primary Purpose: Screening


Related Conditions & MeSH terms

  • Adrenoleukodystrophy
  • Alpha-Mannosidosis
  • Aphasia, Primary Progressive
  • Frontotemporal Dementia
  • Fucosidosis
  • Gaucher Disease
  • Gaucher's Disease
  • Globoid Cell Leukodystrophy
  • I Cell Disease
  • Leukodystrophy, Globoid Cell
  • Leukodystrophy, Metachromatic
  • Mannosidosis
  • Metachromatic Leukodystrophy
  • Mucopolysaccharidoses
  • Mucopolysaccharidosis I
  • Mucopolysaccharidosis VI
  • Niemann-Pick Disease
  • Niemann-Pick Disease, Type C
  • Niemann-Pick Diseases
  • Pick Disease of the Brain
  • Pulmonary Complications
  • Wolman Disease

NCT number NCT00005900
Study type Observational
Source Office of Rare Diseases (ORD)
Contact
Status Active, not recruiting
Phase N/A
Start date August 1999

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