Role of Oxidative Stress and Inflammation in Type 1 Gaucher Disease (GD1) (NCT02583672) | Clinical Trial Compass
CompletedPhase 2
Role of Oxidative Stress and Inflammation in Type 1 Gaucher Disease (GD1)
United States33 participantsStarted 2015-09
Plain-language summary
The purpose of this study is to measure levels of blood and brain chemicals related to oxidative stress and inflammation in healthy volunteers and individuals with Type 1 Gaucher disease (GD1) to see if these levels are altered by GD1.
Who can participate
Age range18 Years
SexALL
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Inclusion criteria
✓. All participants must be 18 years or older.
✓. All participants 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 GD1 who are medically stable for participation in the study in the opinion of the investigator.
✓. GD1 patients must be on a stable, specific ERT and/or SRT therapy at a specific dose (e.g. on a units/kg basis) for at least 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.
✓. Healthy subjects who will be frequency-matched for age.
✓. All participants must not have taken antioxidants coenzyme Q-10, vitamin C, or vitamin E for 3 weeks prior to the study and during the course of 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.
✕. Women who are pregnant or lactating or of child-bearing age who are not using acceptable forms of contraception.
What they're measuring
1
Change in subjects with Gaucher disease type 1, in concentration of glutathione in brain (μmol/g)
✕. History of asthma that is presently being treated.
✕. Patients enrolled in another interventional study.
✕. Allergy to N-acetylcysteine.
✕. Patients who cannot or are unwilling to have blood drawn.
✕. Inability to undergo MRI scanning, including but not limited to: unable to remain still in an MRI scanner for more than 30 minutes, claustrophobia, presence of paramagnetic substances or pacemakers in body, weight over 300 lbs.