Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation

NCT00005900 | Unknown

• 3 other identifiers: 199/15111UMN-MT-1999-18UMN-MT-9818
• Study Type: observational
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

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

Keywords

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

Eligibility Criteria

• Age: 0 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

* Diagnosis of an inborn error of metabolism eligible for allogeneic hematopoietic stem cell transplantation on protocol UMN-MT-1995-01

Contact the study team to discuss eligibility requirements. They can help determine if this study is right for you.

Sponsors & Collaborators

• Fairview University Medical Center: lead

Study Sites (1)

Fairview University Medical Center

Minneapolis, Minnesota, 55455, United States

Location

MeSH Terms

Conditions

Mucolipidoses; Fucosidosis; Leukodystrophy, Globoid Cell; Adrenoleukodystrophy; Mannosidase Deficiency Diseases; Niemann-Pick Diseases; Mucopolysaccharidosis I; Mucopolysaccharidosis VI; Leukodystrophy, Metachromatic; Gaucher Disease; Wolman Disease; Genetic Diseases, Inborn; Metabolism, Inborn Errors; Mucopolysaccharidoses; Rare Diseases; Sphingolipidoses

Condition Hierarchy (Ancestors)

Bone Diseases, Metabolic; Bone Diseases; Musculoskeletal Diseases; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Carbohydrate Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Hereditary Central Nervous System Demyelinating Diseases; Leukoencephalopathies; Demyelinating Diseases; Lipidoses; Lipid Metabolism, Inborn Errors; Lipid Metabolism Disorders; X-Linked Intellectual Disability; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Genetic Diseases, X-Linked; Heredodegenerative Disorders, Nervous System; Peroxisomal Disorders; Adrenal Insufficiency; Adrenal Gland Diseases; Endocrine System Diseases; Histiocytosis, Non-Langerhans-Cell; Histiocytosis; Lymphatic Diseases; Hemic and Lymphatic Diseases; Mucinoses; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Sulfatidosis; Cholesterol Ester Storage Disease; Infant, Newborn, Diseases; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• K. Scott Baker

  Fairview University Medical Center

  STUDY CHAIR

Study Design

• Study Type: observational
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: June 2, 2000
• First Posted: June 5, 2000
• Study Start: August 1, 1999
• Last Updated: June 24, 2005

Record last verified: 2003-10

Locations

US (1)