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NCT00005900 Clinical Trial

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

Mucopolysaccharidosis I Clinical Trial

Clinical Trial Details — Status: Active, not recruiting

Administrative data
NCT number NCT00005900
Other study ID # 199/15111
Secondary ID UMN-MT-1999-18UM
Status Active, not recruiting
Phase N/A
First received June 2, 2000
Last updated June 23, 2005
Start date August 1999

Study information
Verified date October 2003
Source Office of Rare Diseases (ORD)
Contact n/a
Is FDA regulated No
Health authority Unspecified
Study type Observational

Clinical Trial 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.

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.

Recruitment information / eligibility
Status Active, not recruiting
Enrollment 10
Est. completion date
Est. primary completion date
Accepts healthy volunteers No
Gender Both
Age group N/A and older
Eligibility - Diagnosis of an inborn error of metabolism eligible for allogeneic hematopoietic stem cell transplantation on protocol UMN-MT-1995-01

Study Design

Primary Purpose: Screening

Related Conditions & MeSH terms

  • Adrenoleukodystrophy
  • Alpha-Mannosidosis
  • Aphasia, Primary Progressive
  • Frontotemporal Dementia
  • Fucosidosis
  • Gaucher Disease
  • Gaucher's Disease
  • Globoid Cell Leukodystrophy
  • I Cell Disease
  • Leukodystrophy, Globoid Cell
  • Leukodystrophy, Metachromatic
  • Mannosidosis
  • Metachromatic Leukodystrophy
  • Mucopolysaccharidoses
  • Mucopolysaccharidosis I
  • Mucopolysaccharidosis VI
  • Niemann-Pick Disease
  • Niemann-Pick Disease, Type C
  • Niemann-Pick Diseases
  • Pick Disease of the Brain
  • Pulmonary Complications
  • Wolman Disease

Locations
Country Name City State
United States Fairview University Medical Center Minneapolis Minnesota

Sponsors (1)
Lead Sponsor Collaborator
Fairview University Medical Center

Country where clinical trial is conducted

United States, 

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