Oxidative Stress and Inflammatory Biomarkers in Gaucher Disease
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|ClinicalTrials.gov Identifier: NCT02437396|
Recruitment Status : Recruiting
First Posted : May 7, 2015
Last Update Posted : July 13, 2022
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|Condition or disease|
|Gaucher Disease Type I Oxidative Stress Inflammation|
|Study Type :||Observational|
|Estimated Enrollment :||35 participants|
|Official Title:||Novel Inflammatory Biomarkers Complement 5A and Hepcidin in Patients With Gaucher Disease (GD)|
|Actual Study Start Date :||October 2015|
|Estimated Primary Completion Date :||June 2023|
|Estimated Study Completion Date :||June 2023|
Treatment naive GD1
Type 1 Gaucher disease subjects who are naive to any treatment
Type 1 Gaucher disease who are stable on therapy (on the specific ERT and/or SRT and specific dose for at least 2 years)
Healthy Volunteers (No longer recruiting)
Age matched healthy controls. No new participants will be enrolled to this arm.
- Change in Plasma C5a Concentration [ Time Frame: 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
- Change in Plasma Hepcidin Concentration [ Time Frame: 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 Blood Glutathione Concentration [ Time Frame: 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 [ Time Frame: 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
Biospecimen Retention: Samples Without DNA
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|Ages Eligible for Study:||18 Years to 75 Years (Adult, Older Adult)|
|Sexes Eligible for Study:||All|
|Accepts Healthy Volunteers:||No|
|Sampling Method:||Non-Probability Sample|
- 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).
- GD1 patients, who have had a change in therapy i.e. a change in dose or switch from one drug to another, can be enrolled after at least 6 months have elapsed since the change and is considered stable in the opinion of the clinician providing care to the patient.
- All participants must not have taken antioxidants coenzyme Q-10, vitamin C, or vitamin E for 3 weeks prior to the study.
- 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
- Subjects who cannot or are unwilling to have blood drawn
- Unable to adhere to study protocol for whatever reason
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02437396
|Contact: Reena Kartha, PhDfirstname.lastname@example.org|
|United States, Minnesota|
|University of Minnesota||Recruiting|
|Minneapolis, Minnesota, United States, 55455|
|Contact: Reena Kartha, PhD 612-626-2436 email@example.com|
|Principal Investigator: Reena Kartha, PhD|
|Principal Investigator:||Reena Kartha, PhD||University of Minnesota|
|Responsible Party:||University of Minnesota|
|Other Study ID Numbers:||
|First Posted:||May 7, 2015 Key Record Dates|
|Last Update Posted:||July 13, 2022|
|Last Verified:||July 2022|
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