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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. 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
Information provided by:
Office of Rare Diseases (ORD)

Brief 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.

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

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.

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Study Type : Observational
Enrollment : 10 participants
Primary Purpose: Screening
Study Start Date : August 1999

Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00005900

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United States, Minnesota
Fairview University Medical Center
Minneapolis, Minnesota, United States, 55455
Sponsors and Collaborators
Fairview University Medical Center
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Study Chair: K. Scott Baker Fairview University Medical Center
Layout table for additonal information Identifier: NCT00005900    
Other Study ID Numbers: 199/15111
First Posted: June 5, 2000    Key Record Dates
Last Update Posted: June 24, 2005
Last Verified: October 2003
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:
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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
Leukodystrophy, Globoid Cell
Mucopolysaccharidosis I
Mannosidase Deficiency Diseases
Wolman Disease
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