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Gaucher DiseasePhase 4NCT03721627

Chronic Hepatitis C Treatment in Egyptian Children With Gaucher Disease.

A Phase 4 study testing Ledipasvir/Sofosbuvir in people with Gaucher Disease and HCV, run by Mansoura University Children Hospital at 1 site in Egypt.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 6 to 18 years
Conditions the study is for
Gaucher Disease, HCV
What is being tested?
Ledipasvir/Sofosbuvir
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
10 participants
Where
1 siteEgypt
Study dates
3 Apr 2018to Oct 2019
Phase
Phase 4
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 6 to 18 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • 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)

Who cannot join

  • 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

Trial locations and contacts

1 site in 1 country.

Ahmed MH Megahed, MD contact

+201224642996

megahed732000@gmail.com

Egypt1 site, 1 recruiting

  • Mansoura University Children Hospital, Ped Gastroenteroloy and Hepatology Unit

    Al Mansurah, Dakahliya, 35516

    Ahmed Megahed, MD, +201224642996, megahed732000@gmail.com

    Recruiting

Other trials in Egypt

About this study

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

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.

Read the 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:

  1. 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.
  2. In every visit, adverse events and concurrent medication will be reported for safety issue.
  3. 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.
  4. 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.
  5. 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.
Conditions
Gaucher Disease, HCV
Being tested
Ledipasvir/Sofosbuvir
Keywords
Ledipasvir/Sofosbuvir, Gaucher disease, HCV
Medical subject headings
Gaucher Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Ledipasvir/SofosbuvirDrug

    Direct acting anti HCV drugs

Study groups in NCT03721627
Study groupRoleWhat they receive
Treatment groupExperimentalLedipasvir/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.
Receives: Drug: Ledipasvir/Sofosbuvir

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Proportion of patients with Sustained Virologic Response

    Proportion of patients achieving negative HCV PCR 12 weeks after therapy

    Measured: 18 months

Secondary outcomes (2)

  1. Prevalence of HCV infection among Gaucher disease children

    Number of HCV PCR positive Gaucher disease patients/Number of screened Gaucher Disease patients

    Measured: 12 months

  2. Drug intolerability

    Proportion of patients who permanently discontinue study drug due to an adverse event.

    Measured: 18 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted26 Oct 2018
  2. Study start3 Apr 2018
  3. Primary completionApr 2019 (estimated)
  4. Study completionOct 2019 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 4
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
10 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03721627
Sponsor's protocol ID
R.18.03.130.R1
Status last verified
Oct 2018
First submitted
25 Oct 2018
Last updated
30 Oct 2018
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

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.

    PubMed 27997679

  • 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.

    PubMed 27842801

Questions about this trial

What is this clinical trial studying?

A Phase 4 study testing Ledipasvir/Sofosbuvir in people with Gaucher Disease and HCV, run by Mansoura University Children Hospital at 1 site in Egypt.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: status not confirmed, as of the sponsor's last update on 30 Oct 2018. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Egypt. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

Contact Ahmed MH Megahed, MD (+201224642996, megahed732000@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Proportion of patients with Sustained Virologic Response (time frame: 18 months). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03721627, which also lists 3 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Oct 2018.

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