Oxidative Stress and Inflammatory Biomarkers in Gaucher Disease
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age18-75
SponsorUniversity of Minnesota
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
All participants must be 18 years or older.
All enrollees must understand and cooperate with requirements of the study in the opinion of the investigators and must be able to provide written informed consent.
Individuals with Gaucher disease who are medically stable for participation in study in the opinion of the investigator.
GD subjects must be stable on a specific ERT and/or SRT therapy at a specific dose (for e.g. on a units/kg basis) for at least 2 years or be naïve to these therapies (no therapy for 2 years).
Disqualifiers
Medically unstable conditions in any group as determined by the investigators
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.
Females who are pregnant or lactating or of child-bearing age who are not using acceptable forms of contraception
History of asthma that is presently being treated
Trial population
Type 1 Gaucher disease subjects who are either naive to treatment or are stable on therapy (specific Enzyme Replacement Therapy (ERT) / Substrate Reduction Therapy (SRT) formulation at a specific dose) for at least 2 years.
Trial design
Design model
Case-control
Time perspective
Prospective
Treatments tested in this trial
Not listed
Trial groups
34 Participants
are grouped into 3 trial groupsGroup A: Treatment naive GD1
Group B: Treated GD1
Group C: Healthy Volunteers (No longer recruiting)
Trial outcomes
Primary outcomes
1
Change in Plasma C5a Concentration
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
Time frame
6 months2
Change in Plasma Hepcidin Concentration
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
Time frame
6 monthsSecondary outcomes
1
Change in Blood Glutathione Concentration
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
Time frame
6 months2
Change in Plasma Tumor Necrosis Factor (TNF)-alpha Concentration
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
Time frame
6 monthsSponsors and contacts
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Source ClinicalTrials.gov