- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT03721627
Chronic Hepatitis C Treatment in Egyptian Children With Gaucher Disease.
Efficacy and Safety of Ledipasvir/Sofosbuvir Fixed Dose Combination Therapy in Treatment of Chronic Hepatitis C Infection in Egyptian Children With Gaucher Disease
Study Overview
Detailed Description
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.
C. In every visit, adverse events and concurrent medication will be reported for safety issue.
D. Tanner staging for pubertal assessment will be done for all patients prior to enrollment then at the end of therapy and end of post-treatment follow up.
E. Laboratory tests: All patients had positive (Anti-HCV Ab) and HCV-RNA PCR for more than 6 months. HCV-RNA will be measured Basal pretreatment at week 12 on treatment then at week 12, 24 post-treatment. Complete blood count (CBC),liver function tests (LFTs), international normalized ratio (INR), and serum creatinine will be done in the same time frame. Lab investigations are going to be done as a part of the routine work, using commercially available kits.
F. Percutaneous liver biopsy: Histological examination of liver biopsy will be done for all patients when feasible (Hemoglobin more than 10gm/dl, Platlet count more than 100 x103/mm2, Prothrombin time (PT) less than 3 seconds prolongation) and agreed by the patients and legal guardians. Liver fibrosis and necroinflammatory injury are going to be reported by a single expert pathologist according to the Modified Knodell score by Ishak, in which inflammatory activity is graded from 0-18 and fibrosis is graded from 0-6. Alternatively transient hepatic elastography by fibroscan is going to be done for those patients with contraindication or refusing liver biopsy.
Study Type
Enrollment (Anticipated)
Phase
- Phase 4
Contacts and Locations
Study Contact
- Name: Ahmed MH Megahed, MD
- Phone Number: +201224642996
- Email: megahed732000@gmail.com
Study Locations
-
-
Dakahliya
-
Mansoura, Dakahliya, Egypt, 35516
- Recruiting
- Mansoura University Children Hospital, Ped Gastroenteroloy and Hepatology Unit
-
Contact:
- Ahmed Megahed, MD
- Phone Number: +201224642996
- Email: megahed732000@gmail.com
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
- 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)
Exclusion Criteria:
- 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
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: 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.
|
Direct acting anti HCV drugs
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Proportion of patients with Sustained Virologic Response
Time Frame: 18 months
|
Proportion of patients achieving negative HCV PCR 12 weeks after therapy
|
18 months
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Prevalence of HCV infection among Gaucher disease children
Time Frame: 12 months
|
Number of HCV PCR positive Gaucher disease patients/Number of screened Gaucher Disease patients
|
12 months
|
Drug intolerability
Time Frame: 18 months
|
Proportion of patients who permanently discontinue study drug due to an adverse event.
|
18 months
|
Collaborators and Investigators
Publications and helpful links
General Publications
- 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.
- 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.
- 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.
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Anticipated)
Study Completion (Anticipated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Digestive System Diseases
- Metabolic Diseases
- Brain Diseases
- Central Nervous System Diseases
- Nervous System Diseases
- RNA Virus Infections
- Virus Diseases
- Infections
- Blood-Borne Infections
- Communicable Diseases
- Liver Diseases
- Genetic Diseases, Inborn
- Flaviviridae Infections
- Hepatitis, Viral, Human
- Hepatitis, Chronic
- Metabolism, Inborn Errors
- Lysosomal Storage Diseases
- Lipid Metabolism Disorders
- Brain Diseases, Metabolic
- Brain Diseases, Metabolic, Inborn
- Sphingolipidoses
- Lysosomal Storage Diseases, Nervous System
- Lipidoses
- Lipid Metabolism, Inborn Errors
- Hepatitis
- Hepatitis C
- Hepatitis C, Chronic
- Gaucher Disease
- Anti-Infective Agents
- Antiviral Agents
- Sofosbuvir
- Ledipasvir, sofosbuvir drug combination
- Ledipasvir
Other Study ID Numbers
- R.18.03.130.R1
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
product manufactured in and exported from the U.S.
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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