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 Chanarin-Dorfman syndrome CHMP2B-related frontotemporal dementia cholesteryl ester storage disease Farber lipogranulomatosis 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 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 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

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

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):

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

mannosidosis
metachromatic leukodystrophy
mucopolysaccharidosis
mucopolysaccharidosis I
mucopolysaccharidosis VI
oncologic disorders
pulmonary complications
rare disease
sphingolipidoses

Additional relevant MeSH terms:

Fucosidosis
Gaucher Disease
Mucolipidoses
Leukodystrophy, Globoid Cell
Leukodystrophy, Metachromatic
Mucopolysaccharidosis I
Alpha-Mannosidosis
Mannosidase Deficiency Diseases
Mucopolysaccharidoses
Mucopolysaccharidosis VI
Niemann-Pick Diseases
Niemann-Pick Disease, Type A
Niemann-Pick Disease, Type C
Wolman Disease
Cholesterol Ester Storage Disease

Adrenoleukodystrophy
Aphasia, Primary Progressive
Pick Disease of the Brain
Frontotemporal Dementia
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Carbohydrate Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases

ClinicalTrials.gov processed this record on May 16, 2013

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