This trial is active, not recruiting.

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
Sponsor Fairview University Medical Center
Start date August 1999
Trial size 10 participants
Trial identifier NCT00005900, 199/15111, UMN-MT-1999-18, UMN-MT-9818


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.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Primary purpose screening

Eligibility Criteria

Male or female participants of any age.

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

Additional Information

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.
Trial information was received from ClinicalTrials.gov and was last updated in June 2005.
Information provided to ClinicalTrials.gov by Office of Rare Diseases (ORD).