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

Sponsor:

Information provided by:

Office of Rare Diseases (ORD)

Study Description

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:

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


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

Resource links provided by the National Library of Medicine

Genetics Home Reference related topics: CHMP2B-related frontotemporal dementia GRN-related frontotemporal dementia Gaucher disease Niemann-Pick disease RNAse T2-deficient leukoencephalopathy Alpha-mannosidosis Frontotemporal dementia with parkinsonism-17 Fucosidosis Leukoencephalopathy with vanishing white matter Lysosomal acid lipase deficiency Megalencephalic leukoencephalopathy with subcortical cysts Mucolipidosis II alpha/beta Multiple sulfatase deficiency

Genetic and Rare Diseases Information Center resources: Frontotemporal Dementia Frontotemporal Dementia, Ubiquitin-positive Pick's Disease Primary Progressive Aphasia Semantic Dementia Mucopolysaccharidosis Mucopolysaccharidosis Type I Gaucher Disease Niemann-Pick Disease Niemann-Pick Disease Type A Leukodystrophy Metachromatic Leukodystrophy Mucopolysaccharidosis Type VI X-linked Adrenoleukodystrophy Adrenomyeloneuropathy Alpha-mannosidosis Krabbe Disease Wolman Disease Cholesteryl Ester Storage Disease Lysosomal Acid Lipase Deficiency Sphingolipidosis I Cell Disease Fucosidosis Niemann-Pick Disease Type C1 Peroxisomal Biogenesis Disorders Hypoadrenalism

U.S. FDA Resources

Groups and Cohorts

Outcome Measures

Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:	Child, Adult, Senior
Sexes Eligible for Study:	All
Accepts Healthy Volunteers:	No

Criteria

Diagnosis of an inborn error of metabolism eligible for allogeneic hematopoietic stem cell transplantation on protocol UMN-MT-1995-01

Contacts and Locations

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 ClinicalTrials.gov identifier (NCT number): NCT00005900

Locations


United States, Minnesota
Fairview University Medical Center
Minneapolis, Minnesota, United States, 55455

Sponsors and Collaborators

Fairview University Medical Center

Investigators


Study Chair:	K. Scott Baker	Fairview University Medical Center

More Information


ClinicalTrials.gov Identifier:	NCT00005900 History of Changes
Other Study ID Numbers:	199/15111 UMN-MT-1999-18 UMN-MT-9818
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):


mannosidosis Gaucher's disease I cell disease Niemann-Pick disease Wolman disease adrenoleukodystrophy disease-related problem/condition fucosidosis genetic diseases and dysmorphic syndromes globoid cell leukodystrophy	inborn errors of metabolism metachromatic leukodystrophy mucopolysaccharidosis mucopolysaccharidosis I mucopolysaccharidosis VI oncologic disorders pulmonary complications rare disease sphingolipidoses

Additional relevant MeSH terms:


Mannosidase Deficiency Diseases alpha-Mannosidosis Mucopolysaccharidoses Gaucher Disease Adrenoleukodystrophy 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 Wolman Disease Leukodystrophy, Globoid Cell	Mucopolysaccharidosis VI Fucosidosis Mucolipidoses Carbohydrate Metabolism, Inborn Errors Metabolism, Inborn Errors Genetic Diseases, Inborn Lysosomal Storage Diseases Mucinoses Connective Tissue Diseases Metabolic Diseases Sphingolipidoses Lysosomal Storage Diseases, Nervous System Brain Diseases, Metabolic, Inborn Brain Diseases, Metabolic Brain Diseases

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