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

NCT ID: NCT00005900

Last Updated: 2005-06-24

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Total Enrollment

10 participants

Study Classification

OBSERVATIONAL

Study Start Date

1999-08-31

Brief Summary

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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.

Detailed Description

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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.

Conditions

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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

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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

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Inclusion Criteria

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

0 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Fairview University Medical Center

OTHER

Sponsor Role lead

Principal Investigators

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K. Scott Baker

Role: STUDY_CHAIR

Fairview University Medical Center

Locations

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Fairview University Medical Center

Minneapolis, Minnesota, United States

Site Status

Countries

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United States

Other Identifiers

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UMN-MT-1999-18

Identifier Type: -

Identifier Source: secondary_id

UMN-MT-9818

Identifier Type: -

Identifier Source: secondary_id

199/15111

Identifier Type: -

Identifier Source: org_study_id