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

Additional relevant MeSH terms:
Leukodystrophy, Globoid Cell
Leukodystrophy, Metachromatic
Adrenoleukodystrophy
Alpha-Mannosidosis
Aphasia, Primary Progressive
Frontotemporal Dementia
Fucosidosis
Gaucher Disease
Mannosidase Deficiency Diseases
Mucopolysaccharidoses
Mucopolysaccharidosis I
Mucopolysaccharidosis VI
Niemann-Pick Disease, Type A
Niemann-Pick Disease, Type C
Niemann-Pick Diseases
Pick Disease of the Brain
Wolman Disease
Adrenal Gland Diseases
Adrenal Insufficiency
Aphasia
Brain Diseases
Brain Diseases, Metabolic
Brain Diseases, Metabolic, Inborn
Carbohydrate Metabolism, Inborn Errors
Central Nervous System Diseases
Cholesterol Ester Storage Disease
Communication Disorders
Connective Tissue Diseases
Delirium, Dementia, Amnestic, Cognitive Disorders
Dementia

ClinicalTrials.gov processed this record on November 25, 2014