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Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation

The recruitment status of this study is unknown. The completion date has passed and the status has not been verified in more than two years.
Verified October 2003 by Office of Rare Diseases (ORD).
Recruitment status was:  Active, not recruiting
Information provided by:
Office of Rare Diseases (ORD) Identifier:
First received: June 2, 2000
Last updated: June 23, 2005
Last verified: October 2003

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.

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

Study Type: Observational
Study Design: Primary Purpose: Screening

Resource links provided by NLM:

Further study details as provided by Office of Rare Diseases (ORD):

Estimated Enrollment: 10
Study Start Date: August 1999
Detailed Description:


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.


Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
  • Diagnosis of an inborn error of metabolism eligible for allogeneic hematopoietic stem cell transplantation on protocol UMN-MT-1995-01
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Please refer to this study by its identifier: NCT00005900

United States, Minnesota
Fairview University Medical Center
Minneapolis, Minnesota, United States, 55455
Sponsors and Collaborators
Fairview University Medical Center
Study Chair: K. Scott Baker Fairview University Medical Center
  More Information Identifier: NCT00005900     History of Changes
Other Study ID Numbers: 199/15111
Study First Received: June 2, 2000
Last Updated: June 23, 2005

Keywords provided by Office of Rare Diseases (ORD):
Gaucher's disease
I cell disease
Niemann-Pick disease
Wolman disease
disease-related problem/condition
genetic diseases and dysmorphic syndromes
globoid cell leukodystrophy
inborn errors of metabolism
metachromatic leukodystrophy
mucopolysaccharidosis I
mucopolysaccharidosis VI
oncologic disorders
pulmonary complications
rare disease

Additional relevant MeSH terms:
Gaucher Disease
Pick Disease of the Brain
Aphasia, Primary Progressive
Frontotemporal Dementia
Leukodystrophy, Metachromatic
Niemann-Pick Diseases
Niemann-Pick Disease, Type A
Niemann-Pick Disease, Type C
Mucopolysaccharidosis I
Mannosidase Deficiency Diseases
Wolman Disease
Leukodystrophy, Globoid Cell
Mucopolysaccharidosis VI
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Connective Tissue Diseases
Metabolic Diseases
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases processed this record on September 19, 2017