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

Sponsor:

Information provided by:

Office of Rare Diseases (ORD)

ClinicalTrials.gov Identifier:

NCT00005900

First received: June 2, 2000

Last updated: June 23, 2005

Last verified: October 2003

History of Changes

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
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:

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

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

U.S. FDA Resources

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


Estimated Enrollment:	10
Study Start Date:	August 1999

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.

Eligibility


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

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: 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
Study First Received:	June 2, 2000
Last Updated:	June 23, 2005

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 Wolman Disease Leukodystrophy, Metachromatic Niemann-Pick Diseases Niemann-Pick Disease, Type A Niemann-Pick Disease, Type C Mucopolysaccharidosis I 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

ClinicalTrials.gov processed this record on June 22, 2017

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