Hepatitis C Virus InfectionPhase 2NCT02175758

Safety and Efficacy of Sofosbuvir + Ribavirin in Adolescents and Children With Genotype 2 or 3 Chronic HCV Infection

A Phase 2 study testing SOF and RBV in people with Hepatitis C Virus Infection, run by Gilead Sciences at 37 sites in 8 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

This study will have two parts as follows: The PK Lead-in Phase of the study will evaluate the steady state pharmacokinetics (PK) and confirm the dose of sofosbuvir (SOF) in hepatitis C virus (HCV)-infected pediatric participants. Read more about this study

Who is it for?: Ages 3 to 17 years
Conditions the study is for: Hepatitis C Virus Infection
What is being tested?: SOF and RBV
Type of treatment: Pill or oral medicine
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 106 participants
Where: 37 sitesin 8 countries
Study dates: 7 Jul 2014to 13 Sep 2018
Phase: Phase 2
Are there results?: Yes, posted in the registrySummary in the doctors' view

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 3 to 17 years
All sexes
No healthy volunteers
Child

Who may be able to join

Consent of parent or legal guardian required
Chronic HCV infection genotype 2 or 3
Screening laboratory values within defined thresholds
PK Lead-in only: all individuals must be treatment naive

Who cannot join

History of clinically significant illness or any other medical disorder that may interfere with subject treatment, assessment or compliance with the protocol
Co-infection with HIV, acute hepatitis A virus, or hepatitis B virus
Clinical hepatic decompensation (ie, ascites, encephalopathy or variceal hemorrhage)
Pregnant or nursing females
Known hypersensitivity to study medication
Use of any prohibited concomitant medications as within 28 days of the Day 1 visit
Note: Other protocol defined Inclusion/Exclusion criteria may apply.

Trial locations and contacts

37 sites in 8 countries.

Country

City, postcode or hospital

37 sites in 8 countries

United States16 sites

Study site
Atlanta, Georgia
Study site
Baltimore, Maryland
Study site
Boston, Massachusetts
Study site
Cincinnati, Ohio
Study site
Dallas, Texas
Study site
Fort Worth, Texas
Study site
Gainesville, Florida
Study site
Indianapolis, Indiana
Study site
Los Angeles, California
Study site
Morgantown, West Virginia
Study site
Nashville, Tennessee
Study site
New York, New York
Study site
Philadelphia, Pennsylvania
Study site
San Francisco, California
Study site
Seattle, Washington
Study site
Washington D.C., District of Columbia

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Italy6 sites

Study site
Bologna
Study site
Florence
Study site
Milan
Study site
Padova
Study site
San Giovanni Rotondo
Study site
Torino

Other trials in Italy

Russia5 sites

Study site
Moscow
Study site
Novokuznetsk
Study site
Saint Petersburg, Russian Federation
Study site
Saint Petersburg
Study site
Tolyatti

Other trials in Russia

Australia3 sites

Study site
Melbourne, Victoria
Study site
New Lambton Heights
Study site
Westmead, New South Wales

Other trials in Australia

United Kingdom3 sites

Study site
Birmingham
Study site
Leeds
Study site
London

Other trials in United Kingdom

Germany2 sites

Study site
Berlin
Study site
Wuppertal, North Rhine-Westphalia

Other trials in Germany

Belgium1 site

Study site
Brussels

Other trials in Belgium

New Zealand1 site

Study site
Auckland

Other trials in New Zealand

About this study

Official title: A Phase 2, Open-Label, Multicenter, Multi-cohort, Single-Arm Study to Investigate the Safety and Efficacy of Sofosbuvir + Ribavirin in Adolescents and Children With Genotype 2 or 3 Chronic HCV Infection

This study will have two parts as follows:

The PK Lead-in Phase of the study will evaluate the steady state pharmacokinetics (PK) and confirm the dose of sofosbuvir (SOF) in hepatitis C virus (HCV)-infected pediatric participants. The PK Lead-in Phase will also evaluate the safety and tolerability of 7 days of dosing of SOF+ribavirin (RBV) in HCV-infected pediatric participants.

The Treatment Phase will be initiated by age cohort after confirmation of age-appropriate SOF dosage levels. Participants from the PK Lead-in Phase will immediately rollover into the Treatment Phase with no interruption of study drug administration. The Treatment Phase will evaluate the antiviral efficacy, safety, and tolerability of SOF+RBV for 12 or 24 weeks in pediatric participants with genotype 2 or 3 HCV infection, respectively.

Conditions: Hepatitis C Virus Infection
Being tested: SOF, RBV
Medical subject headings: Hepatitis C

What participants receive

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

SOFDrug
Also called Sovaldi®, GS-7977
SOF administered orally once daily
RBVDrug
Also called REBETOL®
RBV oral solution or capsules will be administered orally in a divided daily dose based on weight

Study groups in NCT02175758
Study group	Role	What they receive
12 to < 18 Years Old, SOF+RBV 12 Weeks (GT 2)	Experimental	Participants between 12 to < 18 years of age with genotype (GT) 2 HCV infection weighing ≥ 45 kg will receive SOF (1 x 400 mg tablet, 4 x 100 mg tablets, or 8 x 50 mg oral granules based on swallowability assessment during screening) plus RBV (up to 1400 mg) for 12 weeks. Receives: Drug: SOF; Drug: RBV
12 to < 18 Years Old, SOF+RBV 24 Weeks (GT 3)	Experimental	Participants between 12 to < 18 years of age with genotype 3 HCV infection weighing ≥ 45 kg will receive SOF (1 x 400 mg tablet, 4 x 100 mg tablets, or 8 x 50 mg oral granules based on swallowability assessment during screening) plus RBV (up to 1400 mg) for 24 weeks. Receives: Drug: SOF; Drug: RBV
6 to < 12 Years Old, SOF+RBV 12 Weeks (GT 2)	Experimental	Participants between 6 to < 12 years of age with genotype 2 HCV infection weighing ≥ 17 kg and < 45 kg will receive SOF (2 x 100 mg tablets or 4 x 50 mg oral granules based on swallowability assessment during screening) plus RBV (up to 1400 mg) for 12 weeks. Receives: Drug: SOF; Drug: RBV
6 to <12 Years Old, SOF+RBV 24 Weeks (GT 3)	Experimental	Participants between 6 to < 12 years of age with genotype 3 HCV infection weighing ≥ 17 kg and < 45 kg will receive SOF (2 x 100 mg tablets or 4 x 50 mg oral granules based on swallowability assessment during screening) plus RBV (up to 1400 mg) for 24 weeks. Receives: Drug: SOF; Drug: RBV
3 to < 6 Years Old, SOF+RBV 12 Weeks (GT 2)	Experimental	Participants between 3 to < 6 years of age with genotype 2 HCV infection weighing ≥ 17kg will receive SOF (4 x 50 mg oral granules) plus RBV (up to 1400 mg) for 12 weeks and those weighing < 17 kg will receive SOF (3 x 50 mg oral granules) + RBV (up to 1400 mg) for 12 weeks. Receives: Drug: SOF; Drug: RBV
3 to < 6 Years Old, SOF+RBV 24 Weeks (GT 3)	Experimental	Participants between 3 to < 6 years of age with genotype 2 HCV infection weighing ≥ 17kg will receive SOF (4 x 50 mg oral granules) plus RBV (up to 1400 mg) for 24 weeks and those weighing < 17 kg will receive SOF (3 x 50 mg oral granules) + RBV (up to 1400 mg) for 24 weeks. Receives: Drug: SOF; Drug: RBV

What is being measured

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

For Participants in the PK Lead-in Phase, Pharmacokinetic (PK) Parameter: AUCtau of GS-331007 (Metabolite of SOF)
AUCtau is defined as concentration of drug over time (the area under the concentration verses time curve over the dosing interval).
Measured: 6 to < 18 years of age: predose, 0.5, 1, 2, 3, 4, 8, and 12 hours postdose on Day 7; 3 to < 6 years of age: predose, 2, 4, 8, and 12 hours postdose on Day 7
Percentage of Participants Who Permanently Discontinued Any Study Drug Due to an Adverse Event During the PK Lead-in Phase or the Treatment Phase
Measured: Up to 24 weeks
For the Treatment Phase, Percentage of Participants With SVR at 12 Weeks After Discontinuation of Therapy (SVR12)
SVR12 was defined as HCV RNA < the lower limit of quantitation (LLOQ; ie, 15 IU/mL) at 12 weeks after stopping study treatment.
Measured: Posttreatment Week 12

Secondary outcomes (16)

For Participants in the PK Lead-in Phase, Change From Baseline in HCV RNA
Measured: Baseline; Weeks 1, 2, 4, 8, and 12, 16 (24 Week groups only), 20 (24 Week groups only), and 24 (24 Week groups only)
Percentage of Participants Who Permanently Discontinued Any Study Drug Due to an Adverse Event During the PK Lead-in Phase
Measured: Up to Day 7
For the Treatment Phase, Percentage of Participants With Sustained Virologic Response (SVR) at 4 Weeks After Discontinuation of Therapy (SVR4)
SVR4 was defined as HCV RNA < LLOQ at 4 weeks after stopping study treatment.
Measured: Posttreatment Week 4
For the Treatment Phase, Percentage of Participants With SVR at 24 Weeks After Discontinuation of Therapy (SVR24)
SVR24 was defined as HCV RNA < LLOQ at 24 weeks after stopping study treatment.
Measured: Posttreatment Week 24
For the Treatment Phase, Percentage of Participants Experiencing Viral Breakthrough
Viral breakthrough was defined as having confirmed HCV RNA ≥ LLOQ after having previously had HCV RNA < LLOQ while on treatment.
Measured: Up to 24 weeks
For the Treatment Phase, Percentage of Participants Experiencing Viral Relapse
Viral relapse was defined as having confirmed HCV RNA ≥ LLOQ during the posttreatment period having achieved HCV RNA < LLOQ at last on-treatment visit.
Measured: Up to Posttreatment Week 24
For the Treatment Phase, Change From Baseline in HCV RNA
Measured: Baseline; Weeks 1, 2, 4, 8, 12, 16 (24 Week groups only), 20 (24 Week groups only), and 24 (24 Week groups only)
For the Treatment Phase, Percentage of Participants With HCV RNA < LLOQ While On Treatment
Measured: Weeks 1, 2, 4, 8, 12, 16 (24 Week groups only), 20 (24 Week groups only), and 24 (24 Week groups only)
For the Treatment Phase, Percentage of Participants With Alanine Aminotransferase (ALT) Normalization
ALT normalization was defined as ALT > the upper limit of normal (ULN) at baseline and ALT ≤ ULN at each visit. One participant in the 3 to < 6 Years Old 12 Weeks group had ALT > ULN at Baseline, but had no other available data.
Measured: Weeks 1, 2, 4, 8, 12, 16 (24 Week groups only), 20 (24 Week groups only), and 24 (24 Week groups only), and Posttreatment Week 4
For the Treatment Phase, Change From Baseline in Height
Measured: Baseline; Weeks 1, 2, 4, 8, 12, 16 (24 Week groups only), 20 (24 Week groups only), and 24 (24 Week groups only), and Posttreatment Weeks 4, 12, and 24
For the Treatment Phase, Change From Baseline in Weight
Measured: Baseline; Weeks 1, 2, 4, 8, 12, 16 (24 Week groups only), 20 (24 Week groups only), and 24 (24 Week groups only), and Posttreatment Weeks 4, 12, and 24
For the Treatment Phase, Number of Male Participants With a Change From Baseline in Tanner Stage for Pubic Hair
Tanner Stages is a scale that defines physical measurements of development based on external primary and secondary sex characteristics. It was used in this study to assess pubertal development with values ranging from Stage 1 (pre-pubertal characteristics) to Stage 5 (adult or mature characteristics). Any shifts (increase or decrease) in Tanner Stage from Baseline were analyzed and presented.
Measured: Baseline; End of Treatment (either Week 12 or 24), Posttreatment Week 12, and Posttreatment Week 24
For the Treatment Phase, Number of Male Participants With a Change From Baseline in Tanner Stage for Genitalia Development
Tanner Stages is a scale that defines physical measurements of development based on external primary and secondary sex characteristics. It was used in this study to assess pubertal development with values ranging from Stage 1 (pre-pubertal characteristics) to Stage 5 (adult or mature characteristics). Any shifts (increase or decrease) in Tanner Stage from Baseline were analyzed and presented.
Measured: Baseline; End of Treatment (either Week 12 or 24), Posttreatment Week 12, and Posttreatment Week 24
For the Treatment Phase, Number of Female Participants With a Change From Baseline in Tanner Stage for Pubic Hair
Tanner Stages is a scale that defines physical measurements of development based on external primary and secondary sex characteristics. It was used in this study to assess pubertal development with values ranging from Stage 1 (pre-pubertal characteristics) to Stage 5 (adult or mature characteristics). Any shifts (increase or decrease) in Tanner Stage from Baseline were analyzed and presented.
Measured: Baseline; End of Treatment (either Week 12 or 24), Posttreatment Week 12, and Posttreatment Week 24
For the Treatment Phase, Number of Female Participants With a Change From Baseline in Tanner Stage for Breast Development
Tanner Stages is a scale that defines physical measurements of development based on external primary and secondary sex characteristics. It was used in this study to assess pubertal development with values ranging from Stage 1 (pre-pubertal characteristics) to Stage 5 (adult or mature characteristics). Any shifts (increase or decrease) in Tanner Stage from Baseline were analyzed and presented.
Measured: Baseline; End of Treatment (either Week 12 or 24), Posttreatment Week 12, and Posttreatment Week 24
For the Treatment Phase, Palatability of SOF Granules at Day 1 as Assessed by the Percentage of Participants Able/Unable to Taste the SOF Oral Granules
Participants were asked if they were able to taste the SOF oral granules.
Measured: Day 1

Timeline

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

7 Jul 201413 Sep 2018

First posted26 Jun 2014 (estimated)
Study start7 Jul 2014
Primary completion21 Jun 2018
Study completion13 Sep 2018

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (7)Papers the sponsor has linked to this study.
Other trials for Hepatitis C Virus InfectionStudies of the same condition, open and under way.
More trials run by Gilead SciencesThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

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 2
Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Parallel groups
Masking: None (open label)
Enrolment: 106 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02175758
Sponsor's protocol ID: GS-US-334-1112
Other IDs: 2014-002283-32, 2014
Status last verified: Apr 2019
First submitted: 24 Jun 2014
Last updated: 30 Apr 2019
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No

Publications

Kirby B, German P, Kanwar B, Ni L, Lakatos I, Ling J, Mathias A. Pharmacokinetics of Once-Daily Sofosbuvir and Ledipasvir/Sofosbuvir in HCV-Infected Adolescents [Poster 1707]. American Association for the Study of Liver Diseases (AASLD); 2015 November 13-17, San Francisco, USA. Hepatology 2015;62 (S1): 1040A-1041A
Garrison KL, Mathias A, Kersey K, Kanwar B, Ni L, Jain A, et al. Pharmacokinetics of Once-Daily Sofosbuvir and Ledipasvir/Sofosbuvir in HCV-Infected Pediatrics Aged 6 to <12 Years Old [Poster 878]. American Association for the Study of Liver Diseases (AASLD); 2016 11-15 November; Boston, MA. Hepatology 2016;64 (S1): 436A
Schwarz KB, Rosenthal P, Gonzales-Peralta RP, Jonas MM, Balistreri WF, Lin CH, et al. Sofosbuvir + Ribavirin for 12 or 24 Weeks Is Safe and Effective in Adolescents with Genotype 2 or Genotype 3 Chronic Hepatitis C Infection. Hepatology 2016; 63 (Suppl 1): abstract 706.
Wirth S, Rosenthal P, Gonzalez-Peralta RP, Jonas MM, Balistreri WF, Lin CH, Hardikar W, Kersey K, Massetto B, Kanwar B, Brainard DM, Shao J, Svarovskaia E, Kirby B, Arnon R, Murray KF, Schwarz KB. Sofosbuvir and ribavirin in adolescents 12-17 years old with hepatitis C virus genotype 2 or 3 infection. Hepatology. 2017 Oct;66(4):1102-1110. doi: 10.1002/hep.29278. Epub 2017 Aug 26.
PubMed 28543053
Younossi ZM, Stepanova M, Schwarz KB, Wirth S, Rosenthal P, Gonzalez-Peralta R, Murray K, Henry L, Hunt S. Quality of life in adolescents with hepatitis C treated with sofosbuvir and ribavirin. J Viral Hepat. 2018 Apr;25(4):354-362. doi: 10.1111/jvh.12830. Epub 2017 Dec 26.
PubMed 29193603
Rosenthal P, Schwarz KB, Gonzales-Peralta RP, Lin CH, Kelly DA, Nightingale S, et al. Sofosbuvir + Ribavirin for 12 or 24 Weeks Is Safe and Effective in Children 3 to <12 Years Old with Genotype 2 or Genotype 3 Chronic Hepatitis C Infection. Hepatology 2018; 68 (Suppl 1): abstract 1844.
Rosenthal P, Schwarz KB, Gonzalez-Peralta RP, Lin CH, Kelly DA, Nightingale S, Balistreri WF, Bansal S, Jonas MM, Massetto B, Brainard DM, Hsueh CH, Shao J, Parhy B, Davison S, Feiterna-Sperling C, Gillis LA, Indolfi G, Sokal EM, Murray KF, Wirth S. Sofosbuvir and Ribavirin Therapy for Children Aged 3 to <12 Years With Hepatitis C Virus Genotype 2 or 3 Infection. Hepatology. 2020 Jan;71(1):31-43. doi: 10.1002/hep.30821. Epub 2019 Aug 13.
PubMed 31222783

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

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Study at a glance

From the registry record, last updated by the sponsor on 30 Apr 2019. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase 2, Open-Label, Multicenter, Multi-cohort, Single-Arm Study to Investigate the Safety and Efficacy of Sofosbuvir + Ribavirin in Adolescents and Children With Genotype 2 or 3 Chronic HCV Infection
ClinicalTrials.gov ID: NCT02175758
Acronym / sponsor ID: GS-US-334-1112
Conditions: Hepatitis C Virus Infection
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 106 participants (actual)
Lead sponsor: Gilead SciencesIndustry (pharma, biotech, devices)
Study start: 7 Jul 2014 (actual)
Primary completion: 21 Jun 2018 (actual)
Study completion: 13 Sep 2018 (actual)
First posted: 26 Jun 2014 (estimated)
Results: Posted 30 Apr 2019

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

This study will have two parts as follows:

Study design

Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Parallel groups
Masking: None (open label)
Number of arms: 6

Arm	Type	Receives
12 to < 18 Years Old, SOF+RBV 12 Weeks (GT 2)	Experimental	Drug: SOF, Drug: RBVParticipants between 12 to < 18 years of age with genotype (GT) 2 HCV infection weighing ≥ 45 kg will receive SOF (1 x 400 mg tablet, 4 x 100 mg tablets, or 8 x 50 mg oral granules based on swallowability assessment during screening) plus RBV (up to 1400 mg) for 12 weeks.
12 to < 18 Years Old, SOF+RBV 24 Weeks (GT 3)	Experimental	Drug: SOF, Drug: RBVParticipants between 12 to < 18 years of age with genotype 3 HCV infection weighing ≥ 45 kg will receive SOF (1 x 400 mg tablet, 4 x 100 mg tablets, or 8 x 50 mg oral granules based on swallowability assessment during screening) plus RBV (up to 1400 mg) for 24 weeks.
6 to < 12 Years Old, SOF+RBV 12 Weeks (GT 2)	Experimental	Drug: SOF, Drug: RBVParticipants between 6 to < 12 years of age with genotype 2 HCV infection weighing ≥ 17 kg and < 45 kg will receive SOF (2 x 100 mg tablets or 4 x 50 mg oral granules based on swallowability assessment during screening) plus RBV (up to 1400 mg) for 12 weeks.
6 to <12 Years Old, SOF+RBV 24 Weeks (GT 3)	Experimental	Drug: SOF, Drug: RBVParticipants between 6 to < 12 years of age with genotype 3 HCV infection weighing ≥ 17 kg and < 45 kg will receive SOF (2 x 100 mg tablets or 4 x 50 mg oral granules based on swallowability assessment during screening) plus RBV (up to 1400 mg) for 24 weeks.
3 to < 6 Years Old, SOF+RBV 12 Weeks (GT 2)	Experimental	Drug: SOF, Drug: RBVParticipants between 3 to < 6 years of age with genotype 2 HCV infection weighing ≥ 17kg will receive SOF (4 x 50 mg oral granules) plus RBV (up to 1400 mg) for 12 weeks and those weighing < 17 kg will receive SOF (3 x 50 mg oral granules) + RBV (up to 1400 mg) for 12 weeks.
3 to < 6 Years Old, SOF+RBV 24 Weeks (GT 3)	Experimental	Drug: SOF, Drug: RBVParticipants between 3 to < 6 years of age with genotype 2 HCV infection weighing ≥ 17kg will receive SOF (4 x 50 mg oral granules) plus RBV (up to 1400 mg) for 24 weeks and those weighing < 17 kg will receive SOF (3 x 50 mg oral granules) + RBV (up to 1400 mg) for 24 weeks.

Who may qualify

Ages: 3 to 17 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child

Key inclusion criteria (4 listed)

Consent of parent or legal guardian required
Chronic HCV infection genotype 2 or 3
Screening laboratory values within defined thresholds
PK Lead-in only: all individuals must be treatment naive

Key exclusion criteria (7 listed)

History of clinically significant illness or any other medical disorder that may interfere with subject treatment, assessment or compliance with the protocol
Co-infection with HIV, acute hepatitis A virus, or hepatitis B virus
Clinical hepatic decompensation (ie, ascites, encephalopathy or variceal hemorrhage)
Pregnant or nursing females
Known hypersensitivity to study medication

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
SOFAlso: Sovaldi®, GS-7977SOF administered orally once daily	Drug	12 to < 18 Years Old, SOF+RBV 12 Weeks (GT 2), 12 to < 18 Years Old, SOF+RBV 24 Weeks (GT 3), 3 to < 6 Years Old, SOF+RBV 12 Weeks (GT 2), 3 to < 6 Years Old, SOF+RBV 24 Weeks (GT 3), 6 to < 12 Years Old, SOF+RBV 12 Weeks (GT 2), 6 to <12 Years Old, SOF+RBV 24 Weeks (GT 3)
RBVAlso: REBETOL®RBV oral solution or capsules will be administered orally in a divided daily dose based on weight	Drug	12 to < 18 Years Old, SOF+RBV 12 Weeks (GT 2), 12 to < 18 Years Old, SOF+RBV 24 Weeks (GT 3), 3 to < 6 Years Old, SOF+RBV 12 Weeks (GT 2), 3 to < 6 Years Old, SOF+RBV 24 Weeks (GT 3), 6 to < 12 Years Old, SOF+RBV 12 Weeks (GT 2), 6 to <12 Years Old, SOF+RBV 24 Weeks (GT 3)

Primary outcome

For Participants in the PK Lead-in Phase, Pharmacokinetic (PK) Parameter: AUCtau of GS-331007 (Metabolite of SOF)
Time frame: 6 to < 18 years of age: predose, 0.5, 1, 2, 3, 4, 8, and 12 hours postdose on Day 7; 3 to < 6 years of age: predose, 2, 4, 8, and 12 hours postdose on Day 7
AUCtau is defined as concentration of drug over time (the area under the concentration verses time curve over the dosing interval).
Percentage of Participants Who Permanently Discontinued Any Study Drug Due to an Adverse Event During the PK Lead-in Phase or the Treatment Phase
Time frame: Up to 24 weeks
For the Treatment Phase, Percentage of Participants With SVR at 12 Weeks After Discontinuation of Therapy (SVR12)
Time frame: Posttreatment Week 12
SVR12 was defined as HCV RNA < the lower limit of quantitation (LLOQ; ie, 15 IU/mL) at 12 weeks after stopping study treatment.

16 secondary outcomes

For Participants in the PK Lead-in Phase, Change From Baseline in HCV RNA
Time frame: Baseline; Weeks 1, 2, 4, 8, and 12, 16 (24 Week groups only), 20 (24 Week groups only), and 24 (24 Week groups only)
Percentage of Participants Who Permanently Discontinued Any Study Drug Due to an Adverse Event During the PK Lead-in Phase
Time frame: Up to Day 7
For the Treatment Phase, Percentage of Participants With Sustained Virologic Response (SVR) at 4 Weeks After Discontinuation of Therapy (SVR4)
Time frame: Posttreatment Week 4
For the Treatment Phase, Percentage of Participants With SVR at 24 Weeks After Discontinuation of Therapy (SVR24)
Time frame: Posttreatment Week 24
For the Treatment Phase, Percentage of Participants Experiencing Viral Breakthrough
Time frame: Up to 24 weeks
For the Treatment Phase, Percentage of Participants Experiencing Viral Relapse
Time frame: Up to Posttreatment Week 24
For the Treatment Phase, Change From Baseline in HCV RNA
Time frame: Baseline; Weeks 1, 2, 4, 8, 12, 16 (24 Week groups only), 20 (24 Week groups only), and 24 (24 Week groups only)
For the Treatment Phase, Percentage of Participants With HCV RNA < LLOQ While On Treatment
Time frame: Weeks 1, 2, 4, 8, 12, 16 (24 Week groups only), 20 (24 Week groups only), and 24 (24 Week groups only)
For the Treatment Phase, Percentage of Participants With Alanine Aminotransferase (ALT) Normalization
Time frame: Weeks 1, 2, 4, 8, 12, 16 (24 Week groups only), 20 (24 Week groups only), and 24 (24 Week groups only), and Posttreatment Week 4
For the Treatment Phase, Change From Baseline in Height
Time frame: Baseline; Weeks 1, 2, 4, 8, 12, 16 (24 Week groups only), 20 (24 Week groups only), and 24 (24 Week groups only), and Posttreatment Weeks 4, 12, and 24
For the Treatment Phase, Change From Baseline in Weight
Time frame: Baseline; Weeks 1, 2, 4, 8, 12, 16 (24 Week groups only), 20 (24 Week groups only), and 24 (24 Week groups only), and Posttreatment Weeks 4, 12, and 24
For the Treatment Phase, Number of Male Participants With a Change From Baseline in Tanner Stage for Pubic Hair
Time frame: Baseline; End of Treatment (either Week 12 or 24), Posttreatment Week 12, and Posttreatment Week 24
For the Treatment Phase, Number of Male Participants With a Change From Baseline in Tanner Stage for Genitalia Development
Time frame: Baseline; End of Treatment (either Week 12 or 24), Posttreatment Week 12, and Posttreatment Week 24
For the Treatment Phase, Number of Female Participants With a Change From Baseline in Tanner Stage for Pubic Hair
Time frame: Baseline; End of Treatment (either Week 12 or 24), Posttreatment Week 12, and Posttreatment Week 24
For the Treatment Phase, Number of Female Participants With a Change From Baseline in Tanner Stage for Breast Development
Time frame: Baseline; End of Treatment (either Week 12 or 24), Posttreatment Week 12, and Posttreatment Week 24
For the Treatment Phase, Palatability of SOF Granules at Day 1 as Assessed by the Percentage of Participants Able/Unable to Taste the SOF Oral Granules
Time frame: Day 1

Results and safety

Results posted to the registry on 30 Apr 2019, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
12 to < 18 Years Old - SOF+RBV 12 Weeks	4	4
12 to < 18 Years Old - SOF+RBV 24 Weeks	6	6
6 to < 12 Years Old - SOF+RBV 12 Weeks	2	2
6 to < 12 Years Old - SOF+RBV 24 Weeks	10	10
3 to < 6 Years Old - SOF+RBV 12 Weeks	4	4
3 to < 6 Years Old - SOF+RBV 24 Weeks	8	8

Primary outcome: For Participants in the PK Lead-in Phase, Pharmacokinetic (PK) Parameter: AUCtau of GS-331007 (Metabolite of SOF)

Time frame: 6 to < 18 years of age: predose, 0.5, 1, 2, 3, 4, 8, and 12 hours postdose on Day 7; 3 to < 6 years of age: predose, 2, 4, 8, and 12 hours postdose on Day 7. Mean (Standard Deviation). Unit: h*ng/mL

Measure	PK Lead-in: 12 to < 18 Years Old - SOF+RBV 12 or 24 Weeks n = 10	PK Lead-in: 6 to < 12 Years Old - SOF+RBV 12 or 24 Weeks n = 10	PK Lead-in: 3 to < 6 Years Old - SOF+RBV 12 or 24 Weeks n = 10
Value	9106.0 (2601.96)	7651.2 (1723.32)	10293.7 (1860.57)

PK Lead-in: 12 to < 18 Years Old - SOF+RBV 12 or 24 Weeks: Percentage Geometric Mean Ratio: 129.48 (90% CI 109.96 to 152.48)
PK Lead-in: 6 to < 12 Years Old - SOF+RBV 12 or 24 Weeks: Percentage Geometric Mean Ratio: 109.80 (90% CI 93.25 to 129.29)
PK Lead-in: 3 to < 6 Years Old - SOF+RBV 12 or 24 Weeks: Percentage Geometric Mean Ratio: 149.67 (90% CI 127.12 to 176.21)

Population analysed: Intensive PK Analysis Set included all participants in the PK lead-in phase who received at least 1 dose of study drug and for whom at least 1 nonmissing PK concentration value, during the intensive sampling period, was reported by the PK laboratory.

Primary outcome: Percentage of Participants Who Permanently Discontinued Any Study Drug Due to an Adverse Event During the PK Lead-in Phase or the Treatment Phase

Time frame: Up to 24 weeks. Number. Unit: percentage of participants

Measure	12 to < 18 Years Old - SOF+RBV 12 Weeks n = 13	12 to < 18 Years Old - SOF+RBV 24 Weeks n = 39	6 to < 12 Years Old - SOF+RBV 12 Weeks n = 13	6 to < 12 Years Old - SOF+RBV 24 Weeks n = 28	3 to < 6 Years Old - SOF+RBV 12 Weeks n = 5	3 to < 6 Years Old - SOF+RBV 24 Weeks n = 8
Value	0	0	0	0	20.0	0

Population analysed: Safety Analysis Set included all participants who took at least 1 dose of study drug.

Primary outcome: For the Treatment Phase, Percentage of Participants With SVR at 12 Weeks After Discontinuation of Therapy (SVR12)

Time frame: Posttreatment Week 12. Number (95% Confidence Interval). Unit: percentage of participants

Measure	12 to < 18 Years Old (Total) - SOF+RBV 12 or 24 Weeks n = 52	3 to < 12 Years Old (Total) - SOF+RBV 12 or 24 Weeks n = 54	12 to < 18 Years Old - SOF+RBV 12 Weeks n = 13	12 to < 18 Years Old - SOF+RBV 24 Weeks n = 39	6 to < 12 Years Old - SOF+RBV 12 Weeks n = 13	6 to < 12 Years Old - SOF+RBV 24 Weeks n = 28	3 to < 6 Years Old - SOF+RBV 12 Weeks n = 5	3 to < 6 Years Old - SOF+RBV 24 Weeks n = 8
Value	98.1 (89.7 to 100.0)	98.1 (90.1 to 100.0)	100.0 (75.3 to 100.0)	97.4 (86.5 to 99.9)	100.0 (75.3 to 100.0)	100.0 (87.7 to 100.0)	80.0 (28.4 to 99.5)	100.0 (63.1 to 100.0)

12 to < 18 Years Old (Total) - SOF+RBV 12 or 24 Weeks: p <0.001, 2-sided exact 1-sample binomial test
3 to < 12 Years Old (Total) - SOF+RBV 12 or 24 Weeks: p <0.001, 2-sided exact 1-sample binomial test

Population analysed: Full Analysis Set included all participants who were enrolled into the study and received at least 1 dose of study drug.

16 secondary outcomes are reported in the full results.

Adverse events

Time frame: Adverse Events (AEs): First dose date up to Week 12 or 24 (depending on group) plus 30 days (includes AEs occurring during the PK Lead-in Phase); All-Cause Mortality: Up to Posttreatment Week 24. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
12 to < 18 Years Old - SOF+RBV 12 Weeks	0 of 13 (0%)	12 of 13 (92.3%)	0 of 13
12 to < 18 Years Old - SOF+RBV 24 Weeks	0 of 39 (0%)	27 of 39 (69.2%)	0 of 39
6 to < 12 Years Old - SOF+RBV 12 Weeks	0 of 13 (0%)	9 of 13 (69.2%)	0 of 13
6 to < 12 Years Old - SOF+RBV 24 Weeks	0 of 28 (0%)	24 of 28 (85.7%)	0 of 28
3 to < 6 Years Old - SOF+RBV 12 Weeks	0 of 5 (0%)	5 of 5 (100%)	0 of 5
3 to < 6 Years Old - SOF+RBV 24 Weeks	1 of 8 (12.5%)	8 of 8 (100%)	0 of 8

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 30 Apr 2019.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Results publicationKirby B, German P, Kanwar B, Ni L, Lakatos I, Ling J, Mathias A. Pharmacokinetics of Once-Daily Sofosbuvir and Ledipasvir/Sofosbuvir in HCV-Infected Adolescents [Poster 1707]. American Association for the Study of Liver Diseases (AASLD); 2015 November 13-17, San Francisco, USA. Hepatology 2015;62 (S1): 1040A-1041A
Results publicationGarrison KL, Mathias A, Kersey K, Kanwar B, Ni L, Jain A, et al. Pharmacokinetics of Once-Daily Sofosbuvir and Ledipasvir/Sofosbuvir in HCV-Infected Pediatrics Aged 6 to <12 Years Old [Poster 878]. American Association for the Study of Liver Diseases (AASLD); 2016 11-15 November; Boston, MA. Hepatology 2016;64 (S1): 436A
Results publicationSchwarz KB, Rosenthal P, Gonzales-Peralta RP, Jonas MM, Balistreri WF, Lin CH, et al. Sofosbuvir + Ribavirin for 12 or 24 Weeks Is Safe and Effective in Adolescents with Genotype 2 or Genotype 3 Chronic Hepatitis C Infection. Hepatology 2016; 63 (Suppl 1): abstract 706.
Results publication: PubMed 28543053Wirth S, Rosenthal P, Gonzalez-Peralta RP, Jonas MM, Balistreri WF, Lin CH, Hardikar W, Kersey K, Massetto B, Kanwar B, Brainard DM, Shao J, Svarovskaia E, Kirby B, Arnon R, Murray KF, Schwarz KB. Sofosbuvir and ribavirin in adolescents 12-17 years old with hepatitis C virus genotype 2 or 3 infection. Hepatology. 2017 Oct;66(4):1102-1110. doi: 10.1002/hep.29278. Epub 2017 Aug 26.
Results publication: PubMed 29193603Younossi ZM, Stepanova M, Schwarz KB, Wirth S, Rosenthal P, Gonzalez-Peralta R, Murray K, Henry L, Hunt S. Quality of life in adolescents with hepatitis C treated with sofosbuvir and ribavirin. J Viral Hepat. 2018 Apr;25(4):354-362. doi: 10.1111/jvh.12830. Epub 2017 Dec 26.
Results publicationRosenthal P, Schwarz KB, Gonzales-Peralta RP, Lin CH, Kelly DA, Nightingale S, et al. Sofosbuvir + Ribavirin for 12 or 24 Weeks Is Safe and Effective in Children 3 to <12 Years Old with Genotype 2 or Genotype 3 Chronic Hepatitis C Infection. Hepatology 2018; 68 (Suppl 1): abstract 1844.
Derived from this study: PubMed 31222783Rosenthal P, Schwarz KB, Gonzalez-Peralta RP, Lin CH, Kelly DA, Nightingale S, Balistreri WF, Bansal S, Jonas MM, Massetto B, Brainard DM, Hsueh CH, Shao J, Parhy B, Davison S, Feiterna-Sperling C, Gillis LA, Indolfi G, Sokal EM, Murray KF, Wirth S. Sofosbuvir and Ribavirin Therapy for Children Aged 3 to <12 Years With Hepatitis C Virus Genotype 2 or 3 Infection. Hepatology. 2020 Jan;71(1):31-43. doi: 10.1002/hep.30821. Epub 2019 Aug 13.

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Gilead Study Director study director

Gilead Sciences

37 sites in 8 countries: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing SOF and RBV in people with Hepatitis C Virus Infection, run by Gilead Sciences at 37 sites in 8 countries.

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 4 inclusion and 7 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: finished, as of the sponsor's last update on 30 Apr 2019. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 37 sites in United States, Italy, Russia, Australia, United Kingdom and 3 more countries. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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

Are results available for this study?

Yes. Results are posted in the registry (first posted 30 Apr 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

For Participants in the PK Lead-in Phase, Pharmacokinetic (PK) Parameter: AUCtau of GS-331007 (Metabolite of SOF) (time frame: 6 to < 18 years of age: predose, 0.5, 1, 2, 3, 4, 8, and 12 hours postdose on Day 7; 3 to < 6 years of age: predose, 2, 4, 8, and 12 hours postdose on Day 7). The study lists 3 primary outcomes in all. 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 NCT02175758, which also lists 7 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 Apr 2019.