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Chronic Hepatitis C Treatment in Egyptian Children With Gaucher Disease.

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ClinicalTrials.gov Identifier: NCT03721627
Recruitment Status : Recruiting
First Posted : October 26, 2018
Last Update Posted : October 30, 2018
Sponsor:
Information provided by (Responsible Party):
Ahmed Megahed, MD, Mansoura University Children Hospital

Tracking Information
First Submitted Date  ICMJE October 25, 2018
First Posted Date  ICMJE October 26, 2018
Last Update Posted Date October 30, 2018
Actual Study Start Date  ICMJE April 3, 2018
Estimated Primary Completion Date April 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: October 26, 2018)
Proportion of patients with Sustained Virologic Response [ Time Frame: 18 months ]
Proportion of patients achieving negative HCV PCR 12 weeks after therapy
Original Primary Outcome Measures  ICMJE
 (submitted: October 25, 2018)
Proportion of patients with Sustained Virologic Response [ Time Frame: 18 months ]
Proportion of Patients Achiving Negative HCV PCR 12 weeks after therapy
Change History Complete list of historical versions of study NCT03721627 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: October 25, 2018)
  • 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.
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Chronic Hepatitis C Treatment in Egyptian Children With Gaucher Disease.
Official Title  ICMJE Efficacy and Safety of Ledipasvir/Sofosbuvir Fixed Dose Combination Therapy in Treatment of Chronic Hepatitis C Infection in Egyptian Children With Gaucher Disease
Brief 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.
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  ICMJE Interventional
Study Phase  ICMJE Phase 4
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE
  • Gaucher Disease
  • HCV
Intervention  ICMJE Drug: Ledipasvir/Sofosbuvir
Direct acting anti HCV drugs
Study Arms  ICMJE 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.
Intervention: Drug: Ledipasvir/Sofosbuvir
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: October 25, 2018)
10
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE October 2019
Estimated Primary Completion Date April 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 6 Years to 18 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Ahmed MH Megahed, MD +201224642996 megahed732000@gmail.com
Listed Location Countries  ICMJE Egypt
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03721627
Other Study ID Numbers  ICMJE R.18.03.130.R1
Has Data Monitoring Committee No
U.S. FDA-regulated Product
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
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Ahmed Megahed, MD, Mansoura University Children Hospital
Study Sponsor  ICMJE Mansoura University Children Hospital
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Mansoura University Children Hospital
Verification Date October 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP