NCT03721627

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.

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
10

participants targeted

Target at below P25 for phase_4

Timeline
Completed

Started Apr 2018

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

April 3, 2018

Completed
7 months until next milestone

First Submitted

Initial submission to the registry

October 25, 2018

Completed
1 day until next milestone

First Posted

Study publicly available on registry

October 26, 2018

Completed
5 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 1, 2019

Completed
6 months until next milestone

Study Completion

Last participant's last visit for all outcomes

October 1, 2019

Completed
Last Updated

October 30, 2018

Status Verified

October 1, 2018

Enrollment Period

12 months

First QC Date

October 25, 2018

Last Update Submit

October 26, 2018

Conditions

Gaucher DiseaseHCV

Keywords

Ledipasvir/SofosbuvirGaucher diseaseHCV

Outcome Measures

Primary Outcomes (1)

  • Proportion of patients with Sustained Virologic Response

    Proportion of patients achieving negative HCV PCR 12 weeks after therapy

    18 months

Secondary Outcomes (2)

  • Prevalence of HCV infection among Gaucher disease children

    12 months

  • Drug intolerability

    18 months

Study Arms (1)

Treatment group

EXPERIMENTAL

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.

Drug: Ledipasvir/Sofosbuvir

Interventions

Ledipasvir/SofosbuvirDRUG

Direct acting anti HCV drugs

Treatment group

Eligibility Criteria

Age6 Years - 18 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • 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)

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Mansoura University Children Hospital, Ped Gastroenteroloy and Hepatology Unit

Al Mansurah, Dakahliya, 35516, Egypt

RECRUITING

Related Publications (3)

  • 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.

    PMID: 27997679BACKGROUND

  • 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.

    PMID: 27842801BACKGROUND

MeSH Terms

Conditions

Gaucher Disease

Interventions

ledipasvir, sofosbuvir drug combination

Condition Hierarchy (Ancestors)

SphingolipidosesLysosomal Storage Diseases, Nervous SystemBrain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesLipidosesLipid Metabolism, Inborn ErrorsLysosomal Storage DiseasesMetabolic DiseasesNutritional and Metabolic DiseasesLipid Metabolism Disorders

Central Study Contacts

Ahmed MH Megahed, MD

CONTACT

+201224642996megahed732000@gmail.com

Study Design

Study Type
interventional
Phase
phase 4
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Associate Professor of Pediatrics

Study Record Dates

First Submitted

October 25, 2018

First Posted

October 26, 2018

Study Start

April 3, 2018

Primary Completion

April 1, 2019

Study Completion

October 1, 2019

Last Updated

October 30, 2018

Record last verified: 2018-10

Data Sharing

IPD Sharing
Will not share

Locations

EG(1)