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

Primary Purpose

I Cell Disease, Fucosidosis, Globoid Cell Leukodystrophy

Status

Unknown status

Phase

Locations

United States

Study Type

Observational

Intervention

About this trial

This is an observational trial for I Cell Disease focused on measuring 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

0 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Fairview University Medical Center

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

NCT ID

NCT00005900

First Posted

June 2, 2000

Last Updated

June 23, 2005

Sponsor

Fairview University Medical Center

1. Study Identification

Unique Protocol Identification Number

NCT00005900

Brief Title

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

Study Type

Observational

2. Study Status

Record Verification Date

October 2003

Overall Recruitment Status

Unknown status

Study Start Date

August 1999 (undefined)

Primary Completion Date

undefined (undefined)

Study Completion Date

undefined (undefined)

3. Sponsor/Collaborators

Name of the Sponsor

Fairview University Medical Center

4. Oversight

5. Study Description

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.

Detailed 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

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

7. Study Design

Enrollment

10 (false)

10. Eligibility

Sex

All

Minimum Age & Unit of Time

0 Years

Accepts Healthy Volunteers

Eligibility Criteria

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

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

K. Scott Baker

Organizational Affiliation

Fairview University Medical Center

Official's Role

Study Chair

Facility Information:

Facility Name

Fairview University Medical Center

City

Minneapolis

State/Province

Minnesota

ZIP/Postal Code

55455

Country

United States

12. IPD Sharing Statement

Learn more about this trial

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

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