drugset / Trial / NCT03721627
Chronic Hepatitis C Treatment in Egyptian Children With Gaucher Disease.
NaSingle-groupOpen-labelTreatment
Summary
This prospective open label study is designed to screen all available Gaucher disease patients \[either on enzyme replacement therapy (ERT) or not\] for hepatitis C virus (HCV) infection. Furthermore to evaluate the safety and effectiveness of combined Sofosbuvir/Ledipasvir regimen given for 12 weeks in chronically infected patients aged 6-18 years.
Timeline
- Start
- 2018-04-03
- Primary completion
- 2019-04
- Completion
- 2019-10
Publications
- Background Balistreri WF, Murray KF, Rosenthal P, Bansal S, Lin CH, Kersey K, Massetto B, Zhu Y, Kanwar B, German P, Svarovskaia E, Brainard DM, Wen J, Gonzalez-Peralta RP, Jonas MM, Schwarz K. The safety and effectiveness of ledipasvir-sofosbuvir in adolescents 12-17 years old with hepatitis C virus genotype 1 infection. Hepatology. 2017 Aug;66(2):371-378. doi: 10.1002/hep.28995. Epub 2017 Jun 19.
- Background KF Murray, W Balistreri, S Bansal, et al. Ledipasvir/sofosbuvir ± ribavirin for 12 or 24 weeks is safe and effective in children 6-11 years old with chronic hepatitis C infection. EASL International Liver Congress. Amsterdam, April 19-23, 2017. Abstract GS-010.
- Background Adar T, Ilan Y, Elstein D, Zimran A. Liver involvement in Gaucher disease - Review and clinical approach. Blood Cells Mol Dis. 2018 Feb;68:66-73. doi: 10.1016/j.bcmd.2016.10.001. Epub 2016 Oct 19.
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Ledipasvir | Small molecule | 45 mg | Oral |
| Subject | Ledipasvir | Small molecule | 90 mg | Oral |
| Subject | Sofosbuvir | Small molecule | 200 mg | Oral |
| Subject | Sofosbuvir | Small molecule | 400 mg | Oral |