Could an antioxidant help gaucher disease? new study investigates
NCT ID NCT02583672
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study measured levels of brain chemicals related to oxidative stress and inflammation in people with type 1 Gaucher disease and healthy volunteers. Researchers gave 33 participants with Gaucher disease the antioxidant N-acetylcysteine (NAC) for about 90 days to see if it changed these chemical levels. The goal was to understand the role of oxidative stress in the disease, not to test a cure.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- N-acetylcysteine (NAC)
- What this could lead to
- If successful, this could point toward using antioxidants like NAC to reduce inflammation and oxidative stress in Gaucher disease.
- What could go wrong
- This is a small, early-phase study with only 33 participants, focused on measuring chemical changes rather than clinical improvement. Results may not lead to a new treatment.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 2
Tests whether the treatment actually works, and watches for side effects, in a larger group.
- Participants
-
33 people
The number who actually took part.
- Started
-
Sep 2015
- Finished
-
Dec 2025
- Lead sponsor
-
Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
-
18 years and older
- Sex
-
Anyone
- Healthy volunteers
-
Accepted
You do not need to have the condition being studied to take part.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: 1. All participants must be 18 years or older. 2. 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. 3. Individuals with GD1 who are medically stable for participation in the study in the opinion of the investigator. 4. 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. 5. 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. 6. Healthy subjects who will be frequency-matched for age. 7. 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: 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. Women 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. Patients enrolled in another interventional study. 6. Allergy to N-acetylcysteine. 7. Patients who cannot or are unwilling to have blood drawn. 8. 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. 9. Unable to adhere to study protocol for whatever reason.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
New York University
New York, New York, 10016, United States
-
University of Minnesota
Minneapolis, Minnesota, 55455, United States
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Other studies related to the condition(s) this trial covers.
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