Chronic Hepatitis C Treatment in Egyptian Children With Gaucher Disease.
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| ClinicalTrials.gov Identifier: NCT03721627 |
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Recruitment Status : Unknown
Verified October 2018 by Ahmed Megahed, MD, Mansoura University Children Hospital.
Recruitment status was: Recruiting
First Posted : October 26, 2018
Last Update Posted : October 30, 2018
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| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Gaucher Disease HCV | Drug: Ledipasvir/Sofosbuvir | Phase 4 |
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 : | Interventional (Clinical Trial) |
| Estimated Enrollment : | 10 participants |
| Allocation: | N/A |
| Intervention Model: | Single Group Assignment |
| Masking: | None (Open Label) |
| Primary Purpose: | Treatment |
| Official Title: | Efficacy and Safety of Ledipasvir/Sofosbuvir Fixed Dose Combination Therapy in Treatment of Chronic Hepatitis C Infection in Egyptian Children With Gaucher Disease |
| Actual Study Start Date : | April 3, 2018 |
| Estimated Primary Completion Date : | April 2019 |
| Estimated Study Completion Date : | October 2019 |
| Arm | Intervention/treatment |
|---|---|
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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.
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Drug: Ledipasvir/Sofosbuvir
Direct acting anti HCV drugs |
- Proportion of patients with Sustained Virologic Response [ Time Frame: 18 months ]Proportion of patients achieving negative HCV PCR 12 weeks after therapy
- 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
- Drug intolerability [ Time Frame: 18 months ]Proportion of patients who permanently discontinue study drug due to an adverse event.
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| Ages Eligible for Study: | 6 Years to 18 Years (Child, Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03721627
| Contact: Ahmed MH Megahed, MD | +201224642996 | megahed732000@gmail.com |
| Egypt | |
| Mansoura University Children Hospital, Ped Gastroenteroloy and Hepatology Unit | Recruiting |
| Mansoura, Dakahliya, Egypt, 35516 | |
| Contact: Ahmed Megahed, MD +201224642996 megahed732000@gmail.com | |
| Responsible Party: | Ahmed Megahed, MD, Associate Professor of Pediatrics, Mansoura University Children Hospital |
| ClinicalTrials.gov Identifier: | NCT03721627 |
| Other Study ID Numbers: |
R.18.03.130.R1 |
| First Posted: | October 26, 2018 Key Record Dates |
| Last Update Posted: | October 30, 2018 |
| Last Verified: | October 2018 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | No |
| Studies a U.S. FDA-regulated Drug Product: | No |
| Studies a U.S. FDA-regulated Device Product: | No |
| Product Manufactured in and Exported from the U.S.: | No |
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Ledipasvir/Sofosbuvir Gaucher disease HCV |
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Hepatitis C Hepatitis C, Chronic Gaucher Disease Hepatitis Liver Diseases Digestive System Diseases Blood-Borne Infections Communicable Diseases Infections Hepatitis, Viral, Human Virus Diseases Flaviviridae Infections RNA Virus Infections Hepatitis, Chronic Sphingolipidoses |
Lysosomal Storage Diseases, Nervous System Brain Diseases, Metabolic, Inborn Brain Diseases, Metabolic Brain Diseases Central Nervous System Diseases Nervous System Diseases Metabolism, Inborn Errors Genetic Diseases, Inborn Lipidoses Lipid Metabolism, Inborn Errors Lysosomal Storage Diseases Metabolic Diseases Lipid Metabolism Disorders Sofosbuvir Ledipasvir |