Oxidative Stress and Inflammatory Biomarkers in Gaucher Disease
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- This is an observational study: the protocol does not assign a study treatment.
- Who it may be relevant to
- Registry conditions: Gaucher Disease Type I, Oxidative Stress, Inflammation. Basic parameters: 18 years — 75 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
Novel Inflammatory Biomarkers Complement 5A and Hepcidin in Patients With Gaucher Disease (GD)
Overview
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.
Primary outcome measures
- Change in Plasma C5a Concentration [Time frame: 6 months]
- Change in Plasma Hepcidin Concentration [Time frame: 6 months]
Secondary outcome measures (2)
- Change in Blood Glutathione Concentration [Time frame: 6 months]
- Change in Plasma Tumor Necrosis Factor (TNF)-alpha Concentration [Time frame: 6 months]
Eligibility criteria
Inclusion criteria
- 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.
Exclusion criteria
- 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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Case-control
Study locations
United States · 1 center
- University of Minnesota — Minneapolis
Identifiers
NCT: NCT02437396 · 1305M34501