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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 |
| Health Authority: | Unspecified |
|
fucosidosis Gaucher's disease I cell disease Niemann-Pick disease Wolman disease adrenoleukodystrophy disease-related problem/condition 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 |
|
Pick Disease of the Brain Sphingolipidoses Frontotemporal dementia Adrenal Gland Diseases Demyelinating diseases Hypoadrenalism Brain Diseases Alpha-L-iduronidase deficiency Metabolism, Inborn Errors Mucopolysaccharidoses Heredodegenerative Disorders, Nervous System I cell disease Adrenoleukodystrophy Addison Disease Infant, Newborn, Diseases |
Metachromatic leukodystrophy Brain Diseases, Metabolic, Inborn 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 |
|
Reticuloendotheliosis Immune System Diseases Lysosomal Storage Diseases, Nervous System Nervous System Diseases Sulfatidosis |
Mental Retardation, X-Linked Histiocytosis, Non-Langerhans-Cell Mucinoses Hereditary Central Nervous System Demyelinating Diseases Carbohydrate Metabolism, Inborn Errors |
