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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
  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: CASK-related intellectual disability CHMP2B-related frontotemporal dementia Chanarin-Dorfman syndrome D-bifunctional protein deficiency GRN-related frontotemporal dementia Gaucher disease Krabbe disease MECP2 duplication syndrome Niemann-Pick disease PPM-X syndrome Renpenning syndrome SYNGAP1-related intellectual disability Schindler disease Wolman disease X-linked adrenoleukodystrophy alpha-mannosidosis alpha-methylacyl-CoA racemase deficiency cholesteryl ester storage disease familial glucocorticoid deficiency frontotemporal dementia with parkinsonism-17 fucosidosis inclusion body myopathy with early-onset Paget disease and frontotemporal dementia leukoencephalopathy with vanishing white matter megalencephalic leukoencephalopathy with subcortical cysts metachromatic leukodystrophy mucolipidosis II alpha/beta mucopolysaccharidosis type I mucopolysaccharidosis type VI peroxisomal acyl-CoA oxidase deficiency succinic semialdehyde dehydrogenase deficiency
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
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

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:
Brain Diseases, Metabolic, Inborn
Mucopolysaccharidoses
Gaucher Disease
Adrenoleukodystrophy
Pick Disease of the Brain
Aphasia, Primary Progressive
Frontotemporal Dementia
Leukodystrophy, Metachromatic
Mannosidase Deficiency Diseases
Alpha-Mannosidosis
Wolman Disease
Mucopolysaccharidosis VI
Leukodystrophy, Globoid Cell
Fucosidosis
Niemann-Pick Diseases
Niemann-Pick Disease, Type A
Niemann-Pick Disease, Type C
Mucopolysaccharidosis I
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
Brain Diseases

ClinicalTrials.gov processed this record on September 28, 2016