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| Sponsored by: |
Fairview University Medical Center |
|---|---|
| Information provided by: | Office of Rare Diseases (ORD) |
| ClinicalTrials.gov Identifier: | NCT00005900 |
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: | Screening |
| Estimated Enrollment: | 10 |
| Study Start Date: | August 1999 |
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 |
Contacts and Locations| United States, Minnesota | |
| Fairview University Medical Center | |
| Minneapolis, Minnesota, United States, 55455 | |
| Study Chair: | K. Scott Baker | Fairview University Medical Center |
More Information
| Study ID Numbers: | 199/15111, UMN-MT-1999-18, UMN-MT-9818 |
| Study First Received: | June 2, 2000 |
| Last Updated: | June 23, 2005 |
| ClinicalTrials.gov Identifier: | NCT00005900 History of Changes |
| Health Authority: | Unspecified |
|
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 |
|
Metachromatic Leukodystrophy Pick Disease of the Brain Sphingolipidoses I Cell Disease Adrenal Gland Diseases Hypoadrenalism Brain Diseases X-linked Adrenoleukodystrophy Metabolism, Inborn Errors Mucopolysaccharidoses Heredodegenerative Disorders, Nervous System Frontotemporal Dementia Adrenoleukodystrophy Addison Disease Infant, Newborn, Diseases |
Brain Diseases, Metabolic, Inborn Metabolic Disorder Niemann-Pick Disease Delirium Speech Disorders Adrenal Insufficiency Cholesterol Ester Storage Disease Metabolic Diseases Demyelinating Diseases Lysosomal Storage Diseases Aphasia Language Disorders Endocrine System Diseases Cognition Disorders Fucosidosis |
|
Pick Disease of the Brain Sphingolipidoses Adrenal Gland Diseases Brain Diseases Mucinoses Hereditary Central Nervous System Demyelinating Diseases Metabolism, Inborn Errors Mucopolysaccharidoses Heredodegenerative Disorders, Nervous System Adrenoleukodystrophy Addison Disease Infant, Newborn, Diseases Brain Diseases, Metabolic, Inborn Speech Disorders Adrenal Insufficiency |
Cholesterol Ester Storage Disease Metabolic Diseases Reticuloendotheliosis Immune System Diseases Demyelinating Diseases Aphasia Lysosomal Storage Diseases Nervous System Diseases Sulfatidosis Language Disorders Endocrine System Diseases Fucosidosis Mental Retardation Niemann-Pick Disease, Type C Delirium, Dementia, Amnestic, Cognitive Disorders |
