Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation
![]() |
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT00005900 |
Recruitment Status
: Unknown
Verified October 2003 by Office of Rare Diseases (ORD).
Recruitment status was: Active, not recruiting
First Posted
: June 5, 2000
Last Update Posted
: June 24, 2005
|
- Study Details
- Tabular View
- No Results Posted
- Disclaimer
- How to Read a Study Record
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 or disease |
---|
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 |
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.
Study Type : | Observational |
Enrollment : | 10 participants |
Primary Purpose: | Screening |
Study Start Date : | August 1999 |


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, Learn About Clinical Studies.
Ages Eligible for Study: | Child, Adult, Senior |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
- Diagnosis of an inborn error of metabolism eligible for allogeneic hematopoietic stem cell transplantation on protocol UMN-MT-1995-01

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00005900
United States, Minnesota | |
Fairview University Medical Center | |
Minneapolis, Minnesota, United States, 55455 |
Study Chair: | K. Scott Baker | Fairview University Medical Center |
ClinicalTrials.gov Identifier: | NCT00005900 History of Changes |
Other Study ID Numbers: |
199/15111 UMN-MT-1999-18 UMN-MT-9818 |
First Posted: | June 5, 2000 Key Record Dates |
Last Update Posted: | June 24, 2005 |
Last Verified: | October 2003 |
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 Leukodystrophy, Metachromatic Niemann-Pick Diseases Niemann-Pick Disease, Type A Niemann-Pick Disease, Type C Mucopolysaccharidosis I Wolman Disease 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 |