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

The recruitment status of this study is unknown because the information has not been verified recently.

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: alpha-mannosidosis alpha-methylacyl-CoA racemase deficiency CASK-related intellectual disability Chanarin-Dorfman syndrome CHMP2B-related frontotemporal dementia cholesteryl ester storage disease D-bifunctional protein deficiency familial glucocorticoid deficiency frontotemporal dementia with parkinsonism-17 fucosidosis Gaucher disease GRN-related frontotemporal dementia inclusion body myopathy with early-onset Paget disease and frontotemporal dementia Krabbe disease Langerhans cell histiocytosis leukoencephalopathy with vanishing white matter MECP2 duplication syndrome megalencephalic leukoencephalopathy with subcortical cysts metachromatic leukodystrophy mucolipidosis II alpha/beta mucopolysaccharidosis type I mucopolysaccharidosis type VI Niemann-Pick disease peroxisomal acyl-CoA oxidase deficiency PPM-X syndrome Renpenning syndrome Schindler disease succinic semialdehyde dehydrogenase deficiency Wolman disease X-linked adrenoleukodystrophy Zellweger spectrum

MedlinePlus related topics: Gaucher's Disease Leukodystrophies

Genetic and Rare Diseases Information Center resources: Adrenoleukodystrophy X-linked Adrenomyeloneuropathy Cholesteryl Ester Storage Disease Frontotemporal Dementia Frontotemporal Dementia, Ubiquitin-positive Gaucher Disease Hand-Schuller-Christian Disease Hypoadrenalism I Cell Disease Krabbe Leukodystrophy Langerhans Cell Histiocytosis Leukodystrophy Lysosomal Acid Lipase Deficiency Metachromatic Leukodystrophy Mucopolysaccharidosis Mucopolysaccharidosis Type I Mucopolysaccharidosis Type VI Niemann-Pick Disease Niemann-Pick Disease Type C1 Pick's Disease Primary Progressive Aphasia Semantic Dementia Sphingolipidosis Wolman Disease

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


Genders Eligible for Study:	Both
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

No publications provided


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
Health Authority:	Unspecified

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:


Adrenoleukodystrophy Gaucher Disease Leukodystrophy, Globoid Cell Leukodystrophy, Metachromatic Mucopolysaccharidoses Mucopolysaccharidosis I Mucopolysaccharidosis VI Niemann-Pick Disease, Type A Niemann-Pick Disease, Type C Niemann-Pick Diseases Wolman Disease Adrenal Gland Diseases Adrenal Insufficiency Brain Diseases Brain Diseases, Metabolic	Brain Diseases, Metabolic, Inborn Carbohydrate Metabolism, Inborn Errors Central Nervous System Diseases Cholesterol Ester Storage Disease Connective Tissue Diseases Demyelinating Diseases Endocrine System Diseases Genetic Diseases, Inborn Genetic Diseases, X-Linked Hereditary Central Nervous System Demyelinating Diseases Heredodegenerative Disorders, Nervous System Histiocytosis Histiocytosis, Non-Langerhans-Cell Infant, Newborn, Diseases Intellectual Disability

Adrenoleukodystrophy
Gaucher Disease
Leukodystrophy, Globoid Cell
Leukodystrophy, Metachromatic
Mucopolysaccharidoses
Mucopolysaccharidosis I
Mucopolysaccharidosis VI
Niemann-Pick Disease, Type A
Niemann-Pick Disease, Type C
Niemann-Pick Diseases
Wolman Disease
Adrenal Gland Diseases
Adrenal Insufficiency
Brain Diseases
Brain Diseases, Metabolic

Brain Diseases, Metabolic, Inborn
Carbohydrate Metabolism, Inborn Errors
Central Nervous System Diseases
Cholesterol Ester Storage Disease
Connective Tissue Diseases
Demyelinating Diseases
Endocrine System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Hereditary Central Nervous System Demyelinating Diseases
Heredodegenerative Disorders, Nervous System
Histiocytosis
Histiocytosis, Non-Langerhans-Cell
Infant, Newborn, Diseases
Intellectual Disability

ClinicalTrials.gov processed this record on February 27, 2015

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