Oxidative Stress and Inflammatory Biomarkers in Gaucher Disease

Recruiting

• Conditions: Inflammation; Oxidative Stress; Gaucher Disease Type I

• Registration Number: NCT02437396
• Lead Sponsor: University of Minnesota

Brief Summary

The objective of this study is to evaluate oxidative stress and/or inflammation in patients with Gaucher disease type I using a series of biomarkers and correlate with measurements of currently used diagnostic biomarkers.

Detailed Description

We will determine oxidative stress and/or inflammation related biomarkers in whole blood and/or plasma in adult subjects with Gaucher disease. Fifteen milliliter blood sample will be collected during three independent visits over a period of approximately 3 months. These samples will be processed to separate plasma from red blood cells and frozen until assays are performed. Standardized immunoassay methods and LC/MS based methods will be adopted to assay a series of biomarkers in these samples. These data will be correlated with currently used diagnostic biomarkers.

Study Timeline

• Study First Submitted: 2015-05-04
• Study First Submitted QC: 2015-05-04
• Study First Posted: 2015-05-07
• Study Start: 2015-10
• Status Verified: 2025-01
• Last Update Submitted: 2025-01-02
• Last Update Posted: 2025-01-03
• Primary Completion: 2025-06-30
• Study Completion: 2025-06-30

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 35

Inclusion Criteria

Not provided

Read More

Exclusion Criteria

1. Medically unstable conditions in any group as determined by the investigators
2. Concurrent disease; medical condition; or an extenuating circumstance that, in the opinion of the investigator, might compromise subject safety, study compliance, completion of the study, or the integrity of the data collected for the study.
3. Females who are pregnant or lactating or of child-bearing age who are not using acceptable forms of contraception
4. History of asthma that is presently being treated
5. Subjects who cannot or are unwilling to have blood drawn
6. Unable to adhere to study protocol for whatever reason

Read More

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Change in Plasma Hepcidin Concentration	6 months	Outcome will be reported as the change in plasma concentration of hepcidin (ng/ml) from baseline to 6 months in participants with Gaucher disease type 1
Change in Plasma C5a Concentration	6 months	Outcome will be reported as the change in plasma concentration of C5a (ng/ml) from baseline to 6 months in participants with Gaucher disease type 1

Secondary Outcome Measures

Name	Time	Method
Change in Blood Glutathione Concentration	6 months	Outcome will be reported as the change in blood concentration of glutathione (umol/g) from baseline to 6 months in participants with Gaucher disease type 1
Change in Plasma Tumor Necrosis Factor (TNF)-alpha Concentration	6 months	Outcome will be reported as the change in blood concentration of TNF-alpha (pg/ml) from baseline to 6 months in participants with Gaucher disease type 1

Trial Locations

Locations (1)

University of Minnesota; 🇺🇸 Minneapolis, Minnesota, United States