Efficacy and Safety of Ledipasvir/Sofosbuvir Fixed Dose Combination Therapy in Treatment of Chronic Hepatitis C Infection in Egyptian Children With Gaucher Disease
试验速览
- 阶段
- 4 期
- 发起方
- 入组人数
- 10
- 试验地点
- 1
- 主要终点
- Proportion of patients with Sustained Virologic Response
研究概览
简要总结
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.
详细描述
This is a prospective open label study, all Gaucher disease pediatric patients (6-18 years), diagnosed at or referred to the HCV Egyptian treatment site, Mansoura University Children's Hospital Gastroenterology and hepatology unit, after positive HCV screen results by Anti HCV antibodies and confirmatory positive quantitative HCV polymerase chain reaction (PCR) are going to be enrolled. Study protocol had been approved by the Mansoura faculty of Medicine Institutional Review Board (IRB).
Study design: In this prospective study, patients will receive ledipasvir-sofosbuvir fixed dose combination tablet (90 mg ledipasvir, 400 mg sofosbuvir) once daily for 12 weeks for those 12-18 years and half the dose (45 mg ledipasvir, 200 mg sofosbuvir) once daily for 12 weeks for those 6-11 years. After the period of treatment, follow up visit are arranged at 4, 12 and 24 weeks post-treatment.
Methods:
Every patient will be subjected to the following:
A. History taking: time and route of acquisition, medications including previous antiviral therapy B. Comprehensive medical examination before study entry will be carried out for all participants and symptom-directed examination in every visit.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 6 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Aged 6 to <18 years
- •Parent or legal guardian must provide written informed consent
- •Treatment naïve or experienced children with chronic HCV infection
- •Chronic HCV infection (≥ 6 months) documented by medical history or liver biopsy
- •Screening laboratory values within defined thresholds
- •No History of solid organ or bone marrow transplantation
- •No history of clinical hepatic decompensation (eg, ascites, jaundice, encephalopathy, variceal hemorrhage)
排除标准
- •Patients with other sever comorbidities of chronic medical illness (e.g. decompensated heart disease, chronic kidney insufficiency)
- •Concomitant hepatitis B virus (HBV) or Human immunodeficiency virus (HIV) infection
- •Medications (systemic steroids, immunosuppressives)
- •Patients or guardians who are unwilling to participate or sign informed consent
研究组 & 干预措施
Treatment group
Ledipasvir/sofosbuvir fixed dose combination tablet (90 mg ledipasvir, 400 mg sofosbuvir) once daily for 12 weeks for those 12-18 years, Wt 35 kg or more and half the dose (45 mg ledipasvir, 200 mg sofosbuvir) once daily for 12 weeks for those 6-11 years, Wt Less than 35 kg.
干预措施: Ledipasvir/Sofosbuvir (Drug)
结局指标
主要结局
Proportion of patients with Sustained Virologic Response
时间窗: 18 months
Proportion of patients achieving negative HCV PCR 12 weeks after therapy
次要结局
- Prevalence of HCV infection among Gaucher disease children(12 months)
- Drug intolerability(18 months)
研究者
Ahmed Megahed, MD
Associate Professor of Pediatrics
Mansoura University Children Hospital