Role of Oxidative Stress and Inflammation in Type 1 Gaucher Disease (GD1): Potential Use of Antioxidant/Anti-inflammatory Medications

Who is this study for? Patients with Gaucher Disease
What treatments are being studied? N-acetylcysteine
Status: Recruiting
Location: See all (2) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
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.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Healthy Volunteers: t
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• 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.

Locations
United States
Minnesota
University of Minnesota
RECRUITING
Minneapolis
New York
New York University
RECRUITING
New York
Contact Information
Primary
Reena V. Kartha, Ph.D.
rvkartha@umn.edu
612-626-2436
Time Frame
Start Date: 2015-09
Estimated Completion Date: 2026-01-31
Participants
Target number of participants: 50
Treatments
Experimental: N-acetylcysteine
The first 10 GD1 subjects will take 1800mg NAC twice daily (3600mg/day) orally for approximately 90 days. An interim analysis will be performed to determine if this dose produces changes in systemic redox status and brain glutathione (GSH) levels. If no signal of a significant change is observed, the remaining 20 subjects will receive up to 3600 mg NAC orally twice a day (7200 mg/day).
Authors
James C. Cloyd
Related Therapeutic Areas
Sponsors
Collaborators: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), National Center for Advancing Translational Sciences (NCATS), Rare Diseases Clinical Research Network, Lysosomal Disease Network, National Institute of Neurological Disorders and Stroke (NINDS)
Leads: University of Minnesota

This content was sourced from clinicaltrials.gov

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