drugset / Trial / NCT02583672
Role of Oxidative Stress and Inflammation in Type 1 Gaucher Disease (GD1)
Phase 2
Completed
33 enrolled
University of Minnesota
Lysosomal Disease Network · collabNational Center for Advancing Translational Sciences (NCATS) · collabNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) · collabNational Institute of Neurological Disorders and Stroke (NINDS) · collabRare Diseases Clinical Research Network · collab
NaSingle-groupOpen-labelTreatment
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.
Timeline
- Start
- 2015-09
- Primary completion
- 2025-12-31
- Completion
- 2025-12-31
Publications
- Background Zhou J, Coles LD, Kartha RV, Nash N, Mishra U, Lund TC, Cloyd JC. Intravenous Administration of Stable-Labeled N-Acetylcysteine Demonstrates an Indirect Mechanism for Boosting Glutathione and Improving Redox Status. J Pharm Sci. 2015 Aug;104(8):2619-26. doi: 10.1002/jps.24482. Epub 2015 Jun 5.
- Background Radtke KK, Coles LD, Mishra U, Orchard PJ, Holmay M, Cloyd JC. Interaction of N-acetylcysteine and cysteine in human plasma. J Pharm Sci. 2012 Dec;101(12):4653-9. doi: 10.1002/jps.23325. Epub 2012 Sep 27.
- Background Coles LD, Tuite PJ, Oz G, Mishra UR, Kartha RV, Sullivan KM, Cloyd JC, Terpstra M. Repeated-Dose Oral N-Acetylcysteine in Parkinson's Disease: Pharmacokinetics and Effect on Brain Glutathione and Oxidative Stress. J Clin Pharmacol. 2018 Feb;58(2):158-167. doi: 10.1002/jcph.1008. Epub 2017 Sep 22.
- Results Holmay MJ, Terpstra M, Coles LD, Mishra U, Ahlskog M, Oz G, Cloyd JC, Tuite PJ. N-Acetylcysteine boosts brain and blood glutathione in Gaucher and Parkinson diseases. Clin Neuropharmacol. 2013 Jul-Aug;36(4):103-6. doi: 10.1097/WNF.0b013e31829ae713.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | N-acetylcysteine | Small molecule | 1800 mg | Oral |
| Subject | N-acetylcysteine | Small molecule | 3600 mg | Oral |