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    Clinical Trial Results:
    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

    Summary
    EudraCT number
    		 2014-002283-32
    Trial protocol
    		 DE   IT   GB   Outside EU/EEA   BE  
    Global end of trial date
    13 Sep 2018

    Results information
    Results version number
    		 v1(current)
    This version publication date
    16 Mar 2019
    First version publication date
    16 Mar 2019
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    GS-US-334-1112
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT02175758
    WHO universal trial number (UTN)
    		 -
    Sponsors
    Sponsor organisation name
    Gilead Sciences
    Sponsor organisation address
    333 Lakeside Drive, Foster City, CA, United States, 94404
    Public contact
    Gilead Clinical Study Information Center, Gilead Sciences, GileadClinicalTrials@gilead.com
    Scientific contact
    Gilead Clinical Study Information Center, Gilead Sciences, GileadClinicalTrials@gilead.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    Yes
    EMA paediatric investigation plan number(s)
    		 EMEA-001276-PIP01-12
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    Yes
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    13 Sep 2018
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    21 Jun 2018
    Global end of trial reached?
    Yes
    Global end of trial date
    13 Sep 2018
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    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.
    Protection of trial subjects
    The protocol and consent/assent forms were submitted by each investigator to a duly constituted Independent Ethics Committee (IEC) or Institutional Review Board (IRB) for review and approval before study initiation. All revisions to the consent/assent forms (if applicable) after initial IEC/IRB approval were submitted by the investigator to the IEC/IRB for review and approval before implementation in accordance with regulatory requirements. This study was conducted in accordance with recognized international scientific and ethical standards, including but not limited to the International Conference on Harmonization guideline for Good Clinical Practice (ICH GCP) and the original principles embodied in the Declaration of Helsinki.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    07 Jul 2014
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    United Kingdom: 12
    Country: Number of subjects enrolled
    Belgium: 2
    Country: Number of subjects enrolled
    Germany: 7
    Country: Number of subjects enrolled
    Italy: 11
    Country: Number of subjects enrolled
    United States: 46
    Country: Number of subjects enrolled
    New Zealand: 2
    Country: Number of subjects enrolled
    Russian Federation: 17
    Country: Number of subjects enrolled
    Australia: 9
    Worldwide total number of subjects
    106
    EEA total number of subjects
    32
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    54
    Adolescents (12-17 years)
    52
    Adults (18-64 years)
    0
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

    		 Close Top of page
    Recruitment
    Recruitment details
    		 Participants were enrolled at study sites in Australia, Europe, Russia, New Zealand, and the United States. The first participant was screened on 07 July 2014. The last study visit occurred on 13 September 2018.

    Pre-assignment
    Screening details
    		 135 participants were screened.

    Period 1
    Period 1 title
    Overall Study (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Non-randomised - controlled
    Blinding used
    		 Not blinded

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 12 to < 18 Years Old - SOF+RBV 12 Weeks
    Arm description
    		 Participants 12 to < 18 years of age with HCV genotype 2 received SOF 400 mg (1 x 400 mg tablet, 4 x 100 mg tablets, or 8 x 50 mg oral granules) once daily + RBV capsules or oral solution (up to 1400 mg, dose depending on weight) for 12 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Sofosbuvir
    Investigational medicinal product code
    Other name
    SOF, GS-7977, Sovaldi®
    Pharmaceutical forms
    Tablet, Granules
    Routes of administration
    Oral use
    Dosage and administration details
    400 mg (1 x 400 mg tablet, 4 x 100 mg tablets, or 8 x 50 mg oral granules) once daily

    Investigational medicinal product name
    Ribavirin
    Investigational medicinal product code
    Other name
    RBV, REBETOL®
    Pharmaceutical forms
    Oral solution, Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    Up to 1400 mg, oral solution or capsules administered orally in a divided daily dose based on weight

    Arm title
    		 12 to < 18 Years Old - SOF+RBV 24 Weeks
    Arm description
    		 Participants 12 to < 18 years of age with HCV genotype 3 received SOF 400 mg (1 x 400 mg tablet, 4 x 100 mg tablets, or 8 x 50 mg oral granules) once daily + RBV capsules or oral solution (up to 1400 mg, dose depending on weight) for 24 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Sofosbuvir
    Investigational medicinal product code
    Other name
    SOF, GS-7977, Sovaldi®
    Pharmaceutical forms
    Granules, Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    400 mg (1 x 400 mg tablet, 4 x 100 mg tablets, or 8 x 50 mg oral granules) once daily

    Investigational medicinal product name
    Ribavirin
    Investigational medicinal product code
    Other name
    RBV, REBETOL®
    Pharmaceutical forms
    Capsule, Oral solution
    Routes of administration
    Oral use
    Dosage and administration details
    Up to 1400 mg, oral solution or capsules administered orally in a divided daily dose based on weight

    Arm title
    		 6 to < 12 Years Old - SOF+RBV 12 Weeks
    Arm description
    		 Participants 6 to < 12 years of age with HCV genotype 2 received SOF 200 mg (2 x 100 mg tablets or 4 x 50 mg oral granules) once daily + RBV capsules or oral solution (up to 1400 mg, dose depending on weight) for 12 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Sofosbuvir
    Investigational medicinal product code
    Other name
    SOF, GS-7977, Sovaldi®
    Pharmaceutical forms
    Granules, Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    200 mg (2 x 100 mg tablets or 4 x 50 mg oral granules) once daily

    Investigational medicinal product name
    Ribavirin
    Investigational medicinal product code
    Other name
    RBV, REBETOL®
    Pharmaceutical forms
    Capsule, Oral solution
    Routes of administration
    Oral use
    Dosage and administration details
    Up to 1400 mg, oral solution or capsules administered orally in a divided daily dose based on weight

    Arm title
    		 6 to < 12 Years Old - SOF+RBV 24 Weeks
    Arm description
    		 Participants 6 to < 12 years of age with HCV genotype 3 received SOF 200 mg (2 x 100 mg tablets or 4 x 50 mg oral granules) once daily + RBV capsules or oral solution (up to 1400 mg, dose depending on weight) for 24 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Sofosbuvir
    Investigational medicinal product code
    Other name
    SOF, GS-7977, Sovaldi®
    Pharmaceutical forms
    Granules, Tablet
    Routes of administration
    Oral use
    Dosage and administration details
    200 mg (2 x 100 mg tablets or 4 x 50 mg oral granules) once daily

    Investigational medicinal product name
    Ribavirin
    Investigational medicinal product code
    Other name
    RBV, REBETOL®
    Pharmaceutical forms
    Capsule, Oral solution
    Routes of administration
    Oral use
    Dosage and administration details
    Up to 1400 mg, oral solution or capsules administered orally in a divided daily dose based on weight

    Arm title
    		 3 to < 6 Years Old - SOF+RBV 12 Weeks
    Arm description
    		 Participants 3 to < 6 years of age with HCV genotype 2 received SOF (weight ≥ 17 kg: 200 mg granules; weight < 17 kg: 150 mg granules) once daily + RBV capsules or oral solution (up to 1400 mg, dose depending on weight) for 12 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Sofosbuvir
    Investigational medicinal product code
    Other name
    SOF, GS-7977, Sovaldi®
    Pharmaceutical forms
    Granules
    Routes of administration
    Oral use
    Dosage and administration details
    Weight ≥ 17 kg: 200 mg granules; weight < 17 kg: 150 mg granules once daily

    Investigational medicinal product name
    Ribavirin
    Investigational medicinal product code
    Other name
    RBV, REBETOL®
    Pharmaceutical forms
    Capsule, Oral solution
    Routes of administration
    Oral use
    Dosage and administration details
    Up to 1400 mg, oral solution or capsules administered orally in a divided daily dose based on weight

    Arm title
    		 3 to < 6 Years Old - SOF+RBV 24 Weeks
    Arm description
    		 Participants 3 to < 6 years of age with HCV genotype 3 received SOF (weight ≥ 17 kg: 200 mg granules; weight < 17 kg: 150 mg granules) once daily + RBV capsules or oral solution (up to 1400 mg, dose depending on weight) for 24 weeks.
    Arm type
    		 Experimental

    Investigational medicinal product name
    Sofosbuvir
    Investigational medicinal product code
    Other name
    SOF, GS-7977, Sovaldi®
    Pharmaceutical forms
    Granules
    Routes of administration
    Oral use
    Dosage and administration details
    Weight ≥ 17 kg: 200 mg granules; weight < 17 kg: 150 mg granules once daily

    Investigational medicinal product name
    Ribavirin
    Investigational medicinal product code
    Other name
    RBV, REBETOL®
    Pharmaceutical forms
    Capsule, Oral solution
    Routes of administration
    Oral use
    Dosage and administration details
    Up to 1400 mg, oral solution or capsules administered orally in a divided daily dose based on weight

    Number of subjects in period 1
    		12 to < 18 Years Old - SOF+RBV 12 Weeks 12 to < 18 Years Old - SOF+RBV 24 Weeks 6 to < 12 Years Old - SOF+RBV 12 Weeks 6 to < 12 Years Old - SOF+RBV 24 Weeks 3 to < 6 Years Old - SOF+RBV 12 Weeks 3 to < 6 Years Old - SOF+RBV 24 Weeks
    Started
    13
    39
    13
    28
    5
    8
    Completed
    13
    38
    13
    28
    4
    8
    Not completed
    0
    1
    0
    0
    1
    0
         Adverse event, non-fatal
    -
    -
    -
    -
    1
    -
         Lost to follow-up
    -
    1
    -
    -
    -
    -

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    12 to < 18 Years Old - SOF+RBV 12 Weeks
    Reporting group description
    		 Participants 12 to < 18 years of age with HCV genotype 2 received SOF 400 mg (1 x 400 mg tablet, 4 x 100 mg tablets, or 8 x 50 mg oral granules) once daily + RBV capsules or oral solution (up to 1400 mg, dose depending on weight) for 12 weeks.

    Reporting group title
    12 to < 18 Years Old - SOF+RBV 24 Weeks
    Reporting group description
    		 Participants 12 to < 18 years of age with HCV genotype 3 received SOF 400 mg (1 x 400 mg tablet, 4 x 100 mg tablets, or 8 x 50 mg oral granules) once daily + RBV capsules or oral solution (up to 1400 mg, dose depending on weight) for 24 weeks.

    Reporting group title
    6 to < 12 Years Old - SOF+RBV 12 Weeks
    Reporting group description
    		 Participants 6 to < 12 years of age with HCV genotype 2 received SOF 200 mg (2 x 100 mg tablets or 4 x 50 mg oral granules) once daily + RBV capsules or oral solution (up to 1400 mg, dose depending on weight) for 12 weeks.

    Reporting group title
    6 to < 12 Years Old - SOF+RBV 24 Weeks
    Reporting group description
    		 Participants 6 to < 12 years of age with HCV genotype 3 received SOF 200 mg (2 x 100 mg tablets or 4 x 50 mg oral granules) once daily + RBV capsules or oral solution (up to 1400 mg, dose depending on weight) for 24 weeks.

    Reporting group title
    3 to < 6 Years Old - SOF+RBV 12 Weeks
    Reporting group description
    		 Participants 3 to < 6 years of age with HCV genotype 2 received SOF (weight ≥ 17 kg: 200 mg granules; weight < 17 kg: 150 mg granules) once daily + RBV capsules or oral solution (up to 1400 mg, dose depending on weight) for 12 weeks.

    Reporting group title
    3 to < 6 Years Old - SOF+RBV 24 Weeks
    Reporting group description
    		 Participants 3 to < 6 years of age with HCV genotype 3 received SOF (weight ≥ 17 kg: 200 mg granules; weight < 17 kg: 150 mg granules) once daily + RBV capsules or oral solution (up to 1400 mg, dose depending on weight) for 24 weeks.

    Reporting group values
    		 12 to < 18 Years Old - SOF+RBV 12 Weeks 12 to < 18 Years Old - SOF+RBV 24 Weeks 6 to < 12 Years Old - SOF+RBV 12 Weeks 6 to < 12 Years Old - SOF+RBV 24 Weeks 3 to < 6 Years Old - SOF+RBV 12 Weeks 3 to < 6 Years Old - SOF+RBV 24 Weeks Total
    Number of subjects
    		 13 39 13 28 5 8 106
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 15 ( 1.9 ) 15 ( 1.9 ) 8 ( 2.1 ) 9 ( 1.7 ) 4 ( 0.8 ) 5 ( 0.8 ) -
    Gender categorical
    Units: Subjects
        Female
    		 5 16 10 20 4 6 61
        Male
    		 8 23 3 8 1 2 45
    Race
    Units: Subjects
        White
    		 11 36 9 20 3 6 85
        Asian
    		 0 1 2 6 0 1 10
        Other
    		 0 1 2 2 1 1 7
        Black or African American
    		 2 0 0 0 1 0 3
        Native Hawaiian or Pacific Islander
    		 0 1 0 0 0 0 1
    Ethnicity
    Units: Subjects
        Hispanic or Latino
    		 0 2 2 4 1 0 9
        Not Hispanic or Latino
    		 13 36 11 23 4 8 95
        Not Disclosed
    		 0 1 0 1 0 0 2
    HCV RNA Category
    Units: Subjects
        < 800,000 IU/mL
    		 5 13 7 15 2 8 50
        ≥ 800,000 IU/mL
    		 8 26 6 13 3 0 56

    End points

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    End points reporting groups
    Reporting group title
    12 to < 18 Years Old - SOF+RBV 12 Weeks
    Reporting group description
    		 Participants 12 to < 18 years of age with HCV genotype 2 received SOF 400 mg (1 x 400 mg tablet, 4 x 100 mg tablets, or 8 x 50 mg oral granules) once daily + RBV capsules or oral solution (up to 1400 mg, dose depending on weight) for 12 weeks.

    Reporting group title
    12 to < 18 Years Old - SOF+RBV 24 Weeks
    Reporting group description
    		 Participants 12 to < 18 years of age with HCV genotype 3 received SOF 400 mg (1 x 400 mg tablet, 4 x 100 mg tablets, or 8 x 50 mg oral granules) once daily + RBV capsules or oral solution (up to 1400 mg, dose depending on weight) for 24 weeks.

    Reporting group title
    6 to < 12 Years Old - SOF+RBV 12 Weeks
    Reporting group description
    		 Participants 6 to < 12 years of age with HCV genotype 2 received SOF 200 mg (2 x 100 mg tablets or 4 x 50 mg oral granules) once daily + RBV capsules or oral solution (up to 1400 mg, dose depending on weight) for 12 weeks.

    Reporting group title
    6 to < 12 Years Old - SOF+RBV 24 Weeks
    Reporting group description
    		 Participants 6 to < 12 years of age with HCV genotype 3 received SOF 200 mg (2 x 100 mg tablets or 4 x 50 mg oral granules) once daily + RBV capsules or oral solution (up to 1400 mg, dose depending on weight) for 24 weeks.

    Reporting group title
    3 to < 6 Years Old - SOF+RBV 12 Weeks
    Reporting group description
    		 Participants 3 to < 6 years of age with HCV genotype 2 received SOF (weight ≥ 17 kg: 200 mg granules; weight < 17 kg: 150 mg granules) once daily + RBV capsules or oral solution (up to 1400 mg, dose depending on weight) for 12 weeks.

    Reporting group title
    3 to < 6 Years Old - SOF+RBV 24 Weeks
    Reporting group description
    		 Participants 3 to < 6 years of age with HCV genotype 3 received SOF (weight ≥ 17 kg: 200 mg granules; weight < 17 kg: 150 mg granules) once daily + RBV capsules or oral solution (up to 1400 mg, dose depending on weight) for 24 weeks.

    Subject analysis set title
    PK Lead-in: 12 to < 18 Years Old - SOF +RBV 12 or 24 Weeks
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    Participants 12 to < 18 years of age received SOF 400 mg (1 x 400 mg tablet, 4 x 100 mg tablets, or 8 x 50 mg oral granules) once daily + RBV capsules or oral solution (up to 1400 mg, dose depending on weight) for 12 or 24 weeks.

    Subject analysis set title
    PK Lead-in: 6 to < 12 Years Old - SOF+RBV 12 or 24 Weeks
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    Participants 6 to < 12 years of age received SOF 200 mg (2 x 100 mg tablets or 4 x 50 mg oral granules) once daily + RBV capsules or oral solution (up to 1400 mg, dose depending on weight) for 12 or 24 weeks.

    Subject analysis set title
    PK Lead-in: 3 to < 6 Years Old - SOF+RBV 12 or 24 Weeks
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    Participants 3 to < 6 years of age received SOF (weight ≥ 17 kg: 200 mg granules; weight < 17 kg: 150 mg granules) once daily + RBV capsules or oral solution (up to 1400 mg, dose depending on weight) for 12 or 24 weeks.

    Subject analysis set title
    12 to < 18 Years Old (Total) - SOF+RBV 12 or 24 Weeks
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Participants 12 to < 18 years of age received SOF 400 mg (1 x 400 mg tablet, 4 x 100 mg tablets, or 8 x 50 mg oral granules) once daily + RBV capsules or oral solution (up to 1400 mg, dose depending on weight) for 12 or 24 weeks.

    Subject analysis set title
    3 to < 12 Years Old (Total) - SOF+RBV 12 or 24 Weeks
    Subject analysis set type
    		 Full analysis
    Subject analysis set description
    Participants 6 to < 12 years of age received SOF 200 mg (2 x 100 mg tablets or 4 x 50 mg oral granules) once daily + RBV capsules or oral solution (up to 1400 mg, dose depending on weight) for 12 or 24 weeks. Participants 3 to < 6 years of age received SOF (weight ≥ 17 kg: 200 mg granules; weight < 17 kg: 150 mg granules) once daily + RBV capsules or oral solution (up to 1400 mg, dose depending on weight) for 12 or 24 weeks.

    Subject analysis set title
    Males 12 to < 18 Years Old - SOF+RBV 12 or 24 Weeks
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Male participants 12 to < 18 years of age received SOF 400 mg (1 x 400 mg tablet, 4 x 100 mg tablets, or 8 x 50 mg oral granules) once daily + RBV capsules or oral solution (up to 1400 mg, dose depending on weight) for 12 or 24 weeks.

    Subject analysis set title
    Males 6 to < 12 Years Old - SOF+RBV 12 or 24 Weeks
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Male participants 6 to < 12 years of age received SOF 200 mg (2 x 100 mg tablets or 4 x 50 mg oral granules) once daily + RBV capsules or oral solution (up to 1400 mg, dose depending on weight) for 12 or 24 weeks.

    Subject analysis set title
    Males 3 to < 6 Years Old - SOF+RBV 12 or 24 Weeks
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Male participants 3 to < 6 years of age received SOF (weight ≥ 17 kg: 200 mg granules; weight < 17 kg: 150 mg granules) once daily + RBV capsules or oral solution (up to 1400 mg, dose depending on weight) for 12 or 24 weeks.

    Subject analysis set title
    Females 12 to < 18 Years Old - SOF+RBV 12 or 24 Weeks
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Female participants 12 to < 18 years of age received SOF 400 mg (1 x 400 mg tablet, 4 x 100 mg tablets, or 8 x 50 mg oral granules) once daily + RBV capsules or oral solution (up to 1400 mg, dose depending on weight) for 12 or 24 weeks.

    Subject analysis set title
    Females 6 to < 12 Years Old - SOF+RBV 12 or 24 Weeks
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Female participants 6 to < 12 years of age received SOF 200 mg (2 x 100 mg tablets or 4 x 50 mg oral granules) once daily + RBV capsules or oral solution (up to 1400 mg, dose depending on weight) for 12 or 24 weeks.

    Subject analysis set title
    Females 3 to < 6 Years Old - SOF+RBV 12 or 24 Weeks
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Female participants 3 to < 6 years of age received SOF (weight ≥ 17 kg: 200 mg granules; weight < 17 kg: 150 mg granules) once daily + RBV capsules or oral solution (up to 1400 mg, dose depending on weight) for 12 or 24 weeks.

    Subject analysis set title
    12 to < 18 Years Old - SOF+RBV 12 or 24 Weeks
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Participants 12 to < 18 years of age received SOF 400 mg (1 x 400 mg tablet, 4 x 100 mg tablets, or 8 x 50 mg oral granules) once daily + RBV capsules or oral solution (up to 1400 mg, dose depending on weight) for 12 or 24 weeks.

    Subject analysis set title
    6 to < 12 Years Old - SOF+RBV 12 or 24 Weeks
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Participants 6 to < 12 years of age received SOF 200 mg (2 x 100 mg tablets or 4 x 50 mg oral granules) once daily + RBV capsules or oral solution (up to 1400 mg, dose depending on weight) for 12 or 24 weeks.

    Subject analysis set title
    3 to < 6 Years Old - SOF+RBV 12 or 24 Weeks
    Subject analysis set type
    		 Sub-group analysis
    Subject analysis set description
    Participants 3 to < 6 years of age received SOF (weight ≥ 17 kg: 200 mg granules; weight < 17 kg: 150 mg granules) once daily + RBV capsules or oral solution (up to 1400 mg, dose depending on weight) for 12 or 24 weeks.

    Primary: For Participants in the PK Lead-in Phase, PK Parameter: AUCtau of GS-331007

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    End point title
    		 For Participants in the PK Lead-in Phase, PK Parameter: AUCtau of GS-331007 [1]
    End point description
    AUCtau is defined as concentration of drug over time (the area under the concentration verses time curve over the dosing interval). 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.
    End point type
    Primary
    End point timeframe
    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
    Notes
    [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: The statistical analysis of this primary endpoint is provided in the attachment. AUCtau of GS-331007 was compared against historical data collected in adult Phase 2/3 studies. Equivalence was determined if the 90% confidence intervals (CI) were within the predefined equivalence boundaries of 50% to 200% for all age groups.
    End point values
    		 PK Lead-in: 12 to < 18 Years Old - SOF +RBV 12 or 24 Weeks PK Lead-in: 6 to < 12 Years Old - SOF+RBV 12 or 24 Weeks PK Lead-in: 3 to < 6 Years Old - SOF+RBV 12 or 24 Weeks
    Number of subjects analysed
    10
    10
    10
    Units: h*ng/mL
        arithmetic mean (standard deviation)
    9106.0 ( 2601.96 )
    7651.2 ( 1723.32 )
    10293.7 ( 1860.57 )
    Attachments
    		 Statistical Analysis
    No statistical analyses for this end point

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

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    End point title
    		 Percentage of Participants Who Permanently Discontinued Any Study Drug Due to an Adverse Event During the PK Lead-in Phase or the Treatment Phase [2]
    End point description
    Safety Analysis Set included all participants who took at least 1 dose of study drug.
    End point type
    Primary
    End point timeframe
    Up to 24 weeks
    Notes
    [2] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: No statistical comparison was planned or performed.
    End point values
    		 12 to < 18 Years Old - SOF+RBV 12 Weeks 12 to < 18 Years Old - SOF+RBV 24 Weeks 6 to < 12 Years Old - SOF+RBV 12 Weeks 6 to < 12 Years Old - SOF+RBV 24 Weeks 3 to < 6 Years Old - SOF+RBV 12 Weeks 3 to < 6 Years Old - SOF+RBV 24 Weeks
    Number of subjects analysed
    13
    39
    13
    28
    5
    8
    Units: percentage of participants
        number (not applicable)
    0
    0
    0
    0
    20.0
    0
    No statistical analyses for this end point

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

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    End point title
    		 For the Treatment Phase, Percentage of Participants With SVR at 12 Weeks After Discontinuation of Therapy (SVR12) [3]
    End point description
    SVR12 was defined as HCV RNA < the lower limit of quantitation (LLOQ; ie, 15 IU/mL) at 12 weeks after stopping study treatment.
    End point type
    Primary
    End point timeframe
    Posttreatment Week 12
    Notes
    [3] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point.
    Justification: The statistical analysis of this primary endpoint is provided in the attachment. The SVR12 rate for the 12 to < 18 Years Old group was compared with the historical SVR12 rate of 80% using a 2-sided exact 1-sample binomial test at the 0.05 significance level. If superiority was demonstrated in the 12 to < 18 Years Old group, then the SVR12 rate for participants aged 3 to < 12 years would be compared with 80% at the 0.05 significance level.
    End point values
    		 12 to < 18 Years Old - SOF+RBV 12 Weeks 12 to < 18 Years Old - SOF+RBV 24 Weeks 6 to < 12 Years Old - SOF+RBV 12 Weeks 6 to < 12 Years Old - SOF+RBV 24 Weeks 3 to < 6 Years Old - SOF+RBV 12 Weeks 3 to < 6 Years Old - SOF+RBV 24 Weeks 12 to < 18 Years Old (Total) - SOF+RBV 12 or 24 Weeks 3 to < 12 Years Old (Total) - SOF+RBV 12 or 24 Weeks
    Number of subjects analysed
    13
    39
    13
    28
    5
    8
    52
    54
    Units: percentage of participants
        number (confidence interval 95%)
    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)
    98.1 (89.7 to 100.0)
    98.1 (90.1 to 100.0)
    Attachments
    		 Statistical Analysis
    No statistical analyses for this end point

    Secondary: For Participants in the PK Lead-in Phase, Change From Baseline in HCV RNA

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    End point title
    		 For Participants in the PK Lead-in Phase, Change From Baseline in HCV RNA
    End point description
    Participants who were enrolled in the PK lead-in phase with available data were analyzed. 999 = Not Applicable; Participants from the 12 Weeks groups were not analyzed for Change at Weeks 16, 20, and 24 because they were only treated for 12 weeks.
    End point type
    Secondary
    End point timeframe
    Baseline; Weeks 1, 2, 4, 8, and 12, 16 (24 Week groups only), 20 (24 Week groups only), and 24 (24 Week groups only)
    End point values
    		 12 to < 18 Years Old - SOF+RBV 12 Weeks 12 to < 18 Years Old - SOF+RBV 24 Weeks 6 to < 12 Years Old - SOF+RBV 12 Weeks 6 to < 12 Years Old - SOF+RBV 24 Weeks 3 to < 6 Years Old - SOF+RBV 12 Weeks 3 to < 6 Years Old - SOF+RBV 24 Weeks
    Number of subjects analysed
    4
    6
    2
    10
    4
    8
    Units: log10 IU/mL
    arithmetic mean (standard deviation)
        Change at Week 1 (N = 4, 6, 2, 10, 4, 8)
    -3.98 ( 1.056 )
    -4.23 ( 0.765 )
    -3.82 ( 0.447 )
    -3.78 ( 1.234 )
    -4.12 ( 0.632 )
    -3.53 ( 0.347 )
        Change at Week 2 (N = 4, 6, 2, 9, 4, 8)
    -4.84 ( 0.743 )
    -4.34 ( 0.758 )
    -4.92 ( 0.674 )
    -4.56 ( 1.553 )
    -4.31 ( 0.846 )
    -3.94 ( 0.422 )
        Change at Week 4 (N = 4, 6, 2, 10, 4, 8)
    -4.84 ( 0.743 )
    -4.34 ( 0.758 )
    -4.92 ( 0.674 )
    -4.60 ( 1.686 )
    -4.42 ( 1.006 )
    -3.97 ( 0.453 )
        Change at Week 8 (N = 4, 6, 2, 10, 4, 8)
    -4.84 ( 0.743 )
    -4.34 ( 0.758 )
    -4.92 ( 0.674 )
    -4.61 ( 1.700 )
    -4.52 ( 1.177 )
    -3.97 ( 0.453 )
        Change at Week 12 (N = 4, 6, 2, 10, 4, 8)
    -4.84 ( 0.743 )
    -4.34 ( 0.758 )
    -4.92 ( 0.674 )
    -4.61 ( 1.700 )
    -4.52 ( 1.177 )
    -3.97 ( 0.453 )
        Change at Week 16 (N = NA, 6, NA, 10, NA, 8)
    999 ( 999 )
    -4.34 ( 0.758 )
    999 ( 999 )
    -4.61 ( 1.700 )
    999 ( 999 )
    -3.97 ( 0.453 )
        Change at Week 20 (N = NA, 6, NA, 10, NA, 8)
    999 ( 999 )
    -4.34 ( 0.758 )
    999 ( 999 )
    -4.61 ( 1.700 )
    999 ( 999 )
    -3.97 ( 0.453 )
        Change at Week 24 (N = NA, 6, NA, 10, NA, 8)
    999 ( 999 )
    -4.34 ( 0.758 )
    999 ( 999 )
    -4.61 ( 1.700 )
    999 ( 999 )
    -3.97 ( 0.453 )
    No statistical analyses for this end point

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

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    End point title
    		 Percentage of Participants Who Permanently Discontinued Any Study Drug Due to an Adverse Event During the PK Lead-in Phase
    End point description
    Participants who were enrolled in the PK lead-in phase were analyzed.
    End point type
    Secondary
    End point timeframe
    Up to Day 7
    End point values
    		 PK Lead-in: 12 to < 18 Years Old - SOF +RBV 12 or 24 Weeks PK Lead-in: 6 to < 12 Years Old - SOF+RBV 12 or 24 Weeks PK Lead-in: 3 to < 6 Years Old - SOF+RBV 12 or 24 Weeks
    Number of subjects analysed
    10
    12
    12
    Units: percentage of participants
        number (not applicable)
    0
    0
    0
    No statistical analyses for this end point

    Secondary: For the Treatment Phase, Percentage of Participants With Sustained Virologic Response (SVR) at 4 Weeks After Discontinuation of Therapy (SVR4)

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    End point title
    		 For the Treatment Phase, Percentage of Participants With Sustained Virologic Response (SVR) at 4 Weeks After Discontinuation of Therapy (SVR4)
    End point description
    SVR4 was defined as HCV RNA < LLOQ at 4 weeks after stopping study treatment. Participants in the Full Analysis Set were analyzed.
    End point type
    Secondary
    End point timeframe
    Posttreatment Week 4
    End point values
    		 12 to < 18 Years Old - SOF+RBV 12 Weeks 12 to < 18 Years Old - SOF+RBV 24 Weeks 6 to < 12 Years Old - SOF+RBV 12 Weeks 6 to < 12 Years Old - SOF+RBV 24 Weeks 3 to < 6 Years Old - SOF+RBV 12 Weeks 3 to < 6 Years Old - SOF+RBV 24 Weeks
    Number of subjects analysed
    13
    39
    13
    28
    5
    8
    Units: percentage of participants
        number (confidence interval 95%)
    100.0 (75.3 to 100.0)
    100.0 (91.0 to 100.0)
    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)
    No statistical analyses for this end point

    Secondary: For the Treatment Phase, Percentage of Participants With SVR at 24 Weeks After Discontinuation of Therapy (SVR24)

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    End point title
    		 For the Treatment Phase, Percentage of Participants With SVR at 24 Weeks After Discontinuation of Therapy (SVR24)
    End point description
    SVR24 was defined as HCV RNA < LLOQ at 24 weeks after stopping study treatment. Participants in the Full Analysis Set were analyzed.
    End point type
    Secondary
    End point timeframe
    Posttreatment Week 24
    End point values
    		 12 to < 18 Years Old - SOF+RBV 12 Weeks 12 to < 18 Years Old - SOF+RBV 24 Weeks 6 to < 12 Years Old - SOF+RBV 12 Weeks 6 to < 12 Years Old - SOF+RBV 24 Weeks 3 to < 6 Years Old - SOF+RBV 12 Weeks 3 to < 6 Years Old - SOF+RBV 24 Weeks
    Number of subjects analysed
    13
    39
    13
    28
    5
    8
    Units: percentage of participants
        number (confidence interval 95%)
    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)
    No statistical analyses for this end point

    Secondary: For the Treatment Phase, Percentage of Participants Experiencing Viral Breakthrough

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    End point title
    		 For the Treatment Phase, Percentage of Participants Experiencing Viral Breakthrough
    End point description
    Viral breakthrough was defined as having confirmed HCV RNA ≥ LLOQ after having previously had HCV RNA < LLOQ while on treatment. Participants in the Full Analysis Set were analyzed.
    End point type
    Secondary
    End point timeframe
    Up to 24 weeks
    End point values
    		 12 to < 18 Years Old - SOF+RBV 12 Weeks 12 to < 18 Years Old - SOF+RBV 24 Weeks 6 to < 12 Years Old - SOF+RBV 12 Weeks 6 to < 12 Years Old - SOF+RBV 24 Weeks 3 to < 6 Years Old - SOF+RBV 12 Weeks 3 to < 6 Years Old - SOF+RBV 24 Weeks
    Number of subjects analysed
    13
    39
    13
    28
    5
    8
    Units: percentage of participants
        number (not applicable)
    0
    0
    0
    0
    0
    0
    No statistical analyses for this end point

    Secondary: For the Treatment Phase, Percentage of Participants Experiencing Viral Relapse

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    End point title
    		 For the Treatment Phase, Percentage of Participants Experiencing Viral Relapse
    End point description
    Viral relapse was defined as having confirmed HCV RNA ≥ LLOQ during the posttreatment period having achieved HCV RNA < LLOQ at last on-treatment visit. Participants in the Full Analysis Set were analyzed.
    End point type
    Secondary
    End point timeframe
    Up to Posttreatment Week 24
    End point values
    		 12 to < 18 Years Old - SOF+RBV 12 Weeks 12 to < 18 Years Old - SOF+RBV 24 Weeks 6 to < 12 Years Old - SOF+RBV 12 Weeks 6 to < 12 Years Old - SOF+RBV 24 Weeks 3 to < 6 Years Old - SOF+RBV 12 Weeks 3 to < 6 Years Old - SOF+RBV 24 Weeks
    Number of subjects analysed
    13
    39
    13
    28
    5
    8
    Units: percentage of participants
        number (not applicable)
    0
    0
    0
    0
    0
    0
    No statistical analyses for this end point

    Secondary: For the Treatment Phase, Change From Baseline in HCV RNA

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    End point title
    		 For the Treatment Phase, Change From Baseline in HCV RNA
    End point description
    Participants in the Full Analysis Set with available data were analyzed. 999 = Not Applicable; Participants from the 12 Weeks groups were not analyzed for Change at Weeks 16, 20, and 24 because they were only treated for 12 weeks.
    End point type
    Secondary
    End point timeframe
    Baseline; Weeks 1, 2, 4, 8, 12, 16 (24 Week groups only), 20 (24 Week groups only), and 24 (24 Week groups only)
    End point values
    		 12 to < 18 Years Old - SOF+RBV 12 Weeks 12 to < 18 Years Old - SOF+RBV 24 Weeks 6 to < 12 Years Old - SOF+RBV 12 Weeks 6 to < 12 Years Old - SOF+RBV 24 Weeks 3 to < 6 Years Old - SOF+RBV 12 Weeks 3 to < 6 Years Old - SOF+RBV 24 Weeks
    Number of subjects analysed
    13
    39
    13
    28
    5
    8
    Units: log10 IU/mL
    arithmetic mean (standard deviation)
        Change at Week 1 (N = 13, 38, 13, 28, 4, 8)
    -4.25 ( 0.986 )
    -4.12 ( 0.733 )
    -4.12 ( 0.692 )
    -3.88 ( 0.928 )
    -4.12 ( 0.632 )
    -3.53 ( 0.347 )
        Change at Week 2 (N = 13, 39, 13, 27, 4, 8)
    -4.74 ( 0.980 )
    -4.86 ( 0.702 )
    -4.55 ( 0.812 )
    -4.51 ( 1.102 )
    -4.31 ( 0.846 )
    -3.94 ( 0.422 )
        Change at Week 4 (N = 13, 39, 13, 28, 4, 8)
    -4.74 ( 0.980 )
    -5.01 ( 0.760 )
    -4.68 ( 0.843 )
    -4.56 ( 1.171 )
    -4.42 ( 1.006 )
    -3.97 ( 0.453 )
        Change at Week 8 (N = 13, 39, 13, 28, 4, 8)
    -4.74 ( 0.980 )
    -5.02 ( 0.771 )
    -4.68 ( 0.843 )
    -4.54 ( 1.191 )
    -4.52 ( 1.177 )
    -3.97 ( 0.453 )
        Change at Week 12 (N = 13, 39, 13, 28, 4, 8)
    -4.74 ( 0.980 )
    -5.02 ( 0.771 )
    -4.68 ( 0.843 )
    -4.57 ( 1.178 )
    -4.52 ( 1.177 )
    -3.97 ( 0.453 )
        Change at Week 16 (N = NA, 39, NA, 28, NA, 8)
    999 ( 999 )
    -5.02 ( 0.771 )
    999 ( 999 )
    -4.57 ( 1.178 )
    999 ( 999 )
    -3.97 ( 0.453 )
        Change at Week 20 (N = NA, 39, NA, 28, NA, 8)
    999 ( 999 )
    -5.02 ( 0.771 )
    999 ( 999 )
    -4.57 ( 1.178 )
    999 ( 999 )
    -3.97 ( 0.453 )
        Change at Week 24 (N = NA, 39, NA, 28, NA, 8)
    999 ( 999 )
    -5.02 ( 0.771 )
    999 ( 999 )
    -4.57 ( 1.178 )
    999 ( 999 )
    -3.97 ( 0.453 )
    No statistical analyses for this end point

    Secondary: For the Treatment Phase, Percentage of Participants With HCV RNA < LLOQ While On Treatment

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    End point title
    		 For the Treatment Phase, Percentage of Participants With HCV RNA < LLOQ While On Treatment
    End point description
    Participants in the Full Analysis Set with available data were analyzed. 999, 9999, 99999 = Not Applicable; Participants from the 12 Weeks groups were not analyzed for Weeks 16, 20, and 24 because they were only treated for 12 weeks.
    End point type
    Secondary
    End point timeframe
    Weeks 1, 2, 4, 8, 12, 16 (24 Week groups only), 20 (24 Week groups only), and 24 (24 Week groups only)
    End point values
    		 12 to < 18 Years Old - SOF+RBV 12 Weeks 12 to < 18 Years Old - SOF+RBV 24 Weeks 6 to < 12 Years Old - SOF+RBV 12 Weeks 6 to < 12 Years Old - SOF+RBV 24 Weeks 3 to < 6 Years Old - SOF+RBV 12 Weeks 3 to < 6 Years Old - SOF+RBV 24 Weeks
    Number of subjects analysed
    13
    39
    13
    28
    5
    8
    Units: percentage of participants
    number (confidence interval 95%)
        Week 1 (N = 13, 39, 13, 28, 4, 8)
    30.8 (9.1 to 61.4)
    30.8 (17.0 to 47.6)
    46.2 (19.2 to 74.9)
    39.3 (21.5 to 59.4)
    50.0 (6.8 to 93.2)
    37.5 (8.5 to 75.5)
        Week 2 (N = 13, 39, 13, 28, 4, 8)
    100.0 (75.3 to 100.0)
    74.4 (57.9 to 87.0)
    76.9 (46.2 to 95.0)
    78.6 (59.0 to 91.7)
    75.0 (19.4 to 99.4)
    87.5 (47.3 to 99.7)
        Week 4 (N = 13, 39, 13, 28, 4, 8)
    100.0 (75.3 to 100.0)
    92.3 (79.1 to 98.4)
    100.0 (75.3 to 100.0)
    96.4 (81.7 to 99.9)
    75.0 (19.4 to 99.4)
    100.0 (63.1 to 100.0)
        Week 8 (N = 13, 39, 13, 28, 4, 8)
    100.0 (75.3 to 100.0)
    100.0 (91.0 to 100.0)
    100.0 (75.3 to 100.0)
    96.4 (81.7 to 99.9)
    100.0 (39.8 to 100.0)
    100.0 (63.1 to 100.0)
        Week 12 (N = 13, 39, 13, 28, 4, 8)
    100.0 (75.3 to 100.0)
    100.0 (91.0 to 100.0)
    100.0 (75.3 to 100.0)
    100.0 (87.7 to 100.0)
    100.0 (39.8 to 100.0)
    100.0 (63.1 to 100.0)
        Week 16 (N = NA, 39, NA, 28, NA, 8)
    9999 (999 to 99999)
    100.0 (91.0 to 100.0)
    9999 (999 to 99999)
    100.0 (87.7 to 100.0)
    9999 (999 to 99999)
    100.0 (63.1 to 100.0)
        Week 20 (N = NA, 39, NA, 28, NA, 8)
    9999 (999 to 99999)
    100.0 (91.0 to 100.0)
    9999 (999 to 99999)
    100.0 (87.7 to 100.0)
    9999 (999 to 99999)
    100.0 (63.1 to 100.0)
        Week 24 (N = NA, 39, NA, 28, NA, 8)
    9999 (999 to 99999)
    100.0 (91.0 to 100.0)
    9999 (999 to 99999)
    100.0 (87.7 to 100.0)
    9999 (999 to 99999)
    100.0 (63.1 to 100.0)
    No statistical analyses for this end point

    Secondary: For the Treatment Phase, Percentage of Participants With Alanine Aminotransferase (ALT) Normalization

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    End point title
    		 For the Treatment Phase, Percentage of Participants With Alanine Aminotransferase (ALT) Normalization
    End point description
    ALT normalization was defined as ALT > the upper limit of normal (ULN) at baseline and ALT ≤ ULN at each visit. Participants in the Full Analysis Set with ALT > ULN at Baseline with available data were analyzed. 999 = Not Applicable; Participants from the 12 Weeks groups were not analyzed for Weeks 16, 20, and 24 because they were only treated for 12 weeks.
    End point type
    Secondary
    End point timeframe
    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
    End point values
    		 12 to < 18 Years Old - SOF+RBV 12 Weeks 12 to < 18 Years Old - SOF+RBV 24 Weeks 6 to < 12 Years Old - SOF+RBV 12 Weeks 6 to < 12 Years Old - SOF+RBV 24 Weeks 3 to < 6 Years Old - SOF+RBV 12 Weeks 3 to < 6 Years Old - SOF+RBV 24 Weeks
    Number of subjects analysed
    4
    22
    4
    16
    0 [4]
    2
    Units: percentage of participants
    number (not applicable)
        Week 1 (N = 4, 22, 4, 16, 2)
    50.0
    63.6
    25.0
    75.0
    100.0
        Week 2 (N = 4, 22, 3, 15, 2)
    100.0
    90.9
    100.0
    93.3
    100.0
        Week 4 (N = 3, 22, 4, 15, 2)
    100.0
    95.5
    100.0
    100.0
    100.0
        Week 8 (N = 4, 22, 3, 15, 2)
    100.0
    95.5
    100.0
    100.0
    100.0
        Week 12 (N = 4, 22, 4, 16, 2)
    100.0
    100.0
    100.0
    100.0
    100.0
        Week 16 (N = NA, 21, NA, 15, 2)
    999
    100.0
    999
    100.0
    100.0
        Week 20 (N = NA, 22, NA, 16, 2)
    999
    100.0
    999
    100.0
    100.0
        Week 24 (N = NA, 21, NA, 15, 2)
    999
    100.0
    999
    100.0
    100.0
        Posttreatment Week 4 (N = 4, 16, 3, 12, 2)
    100.0
    100.0
    100.0
    100.0
    100.0
    Notes
    [4] - 1 participant had ALT > ULN at Baseline, but no other available data.
    No statistical analyses for this end point

    Secondary: For the Treatment Phase, Change From Baseline in Height

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    End point title
    		 For the Treatment Phase, Change From Baseline in Height
    End point description
    Participants in the Safety Analysis Set with available data were analyzed. 999 = Not Applicable; Participants from the 12 Weeks groups were not analyzed for Change at Weeks 16, 20, and 24 because they were only treated for 12 weeks.
    End point type
    Secondary
    End point timeframe
    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
    End point values
    		 12 to < 18 Years Old - SOF+RBV 12 Weeks 12 to < 18 Years Old - SOF+RBV 24 Weeks 6 to < 12 Years Old - SOF+RBV 12 Weeks 6 to < 12 Years Old - SOF+RBV 24 Weeks 3 to < 6 Years Old - SOF+RBV 12 Weeks 3 to < 6 Years Old - SOF+RBV 24 Weeks
    Number of subjects analysed
    13
    39
    13
    28
    5
    8
    Units: centimeters
    arithmetic mean (standard deviation)
        Change at Week 1 (N = 13, 39, 13, 28, 4, 8)
    0.1 ( 1.10 )
    0.0 ( 0.57 )
    0.0 ( 0.57 )
    0.0 ( 0.73 )
    0.3 ( 1.12 )
    0.3 ( 0.75 )
        Change at Week 2 (N = 13, 39, 13, 28, 4, 8)
    0.1 ( 1.07 )
    -0.1 ( 0.59 )
    0.1 ( 0.66 )
    0.0 ( 0.54 )
    0.7 ( 1.09 )
    0.3 ( 0.50 )
        Change at Week 4 (N = 13, 39, 13, 28, 4, 8)
    0.2 ( 1.20 )
    0.2 ( 0.78 )
    0.3 ( 0.67 )
    0.4 ( 0.74 )
    0.9 ( 0.87 )
    0.5 ( 0.67 )
        Change at Week 8 (N = 13, 39, 13, 28, 4, 8)
    0.3 ( 1.27 )
    0.2 ( 0.85 )
    1.0 ( 1.00 )
    0.4 ( 0.62 )
    0.9 ( 0.72 )
    0.8 ( 0.51 )
        Change at Week 12 (N = 12, 39, 13, 28, 4, 8)
    0.5 ( 1.68 )
    0.3 ( 0.95 )
    0.8 ( 0.81 )
    0.7 ( 0.68 )
    1.5 ( 0.45 )
    1.7 ( 1.84 )
        Change at Week 16 (N = NA, 38, NA, 28, NA, 8)
    999 ( 999 )
    0.4 ( 0.91 )
    999 ( 999 )
    1.1 ( 0.93 )
    999 ( 999 )
    1.8 ( 0.79 )
        Change at Week 20 (N = NA, 39, NA, 28, NA, 8)
    999 ( 999 )
    0.5 ( 1.10 )
    999 ( 999 )
    1.6 ( 1.15 )
    999 ( 999 )
    2.5 ( 1.01 )
        Change at Week 24 (N = NA, 36, NA, 27, NA, 8)
    999 ( 999 )
    0.8 ( 1.54 )
    999 ( 999 )
    2.1 ( 0.88 )
    999 ( 999 )
    2.7 ( 1.05 )
        Change at PT Week 4 (N = 13, 38, 13, 28, 4, 8)
    0.5 ( 1.79 )
    0.9 ( 1.70 )
    1.4 ( 0.80 )
    2.6 ( 1.17 )
    1.6 ( 0.91 )
    3.5 ( 0.85 )
        Change at PT Week 12 (N = 12, 38, 13, 28, 4, 8)
    1.4 ( 1.65 )
    1.3 ( 1.90 )
    2.4 ( 1.02 )
    3.8 ( 1.30 )
    2.8 ( 1.81 )
    4.0 ( 0.95 )
        Change at PT Week 24 (N = 13, 37, 13, 27, 3, 8)
    1.6 ( 1.89 )
    1.8 ( 2.83 )
    4.2 ( 1.11 )
    5.1 ( 1.59 )
    5.0 ( 1.61 )
    5.7 ( 1.27 )
    No statistical analyses for this end point

    Secondary: For the Treatment Phase, Change From Baseline in Weight

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    End point title
    		 For the Treatment Phase, Change From Baseline in Weight
    End point description
    Participants in the Safety Analysis Set with available data were analyzed. 999 = Not Applicable; Participants from the 12 Weeks groups were not analyzed for Change at Weeks 16, 20, and 24 because they were only treated for 12 weeks.
    End point type
    Secondary
    End point timeframe
    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
    End point values
    		 12 to < 18 Years Old - SOF+RBV 12 Weeks 12 to < 18 Years Old - SOF+RBV 24 Weeks 6 to < 12 Years Old - SOF+RBV 12 Weeks 6 to < 12 Years Old - SOF+RBV 24 Weeks 3 to < 6 Years Old - SOF+RBV 12 Weeks 3 to < 6 Years Old - SOF+RBV 24 Weeks
    Number of subjects analysed
    13
    39
    13
    28
    5
    8
    Units: kilograms
    arithmetic mean (standard deviation)
        Change at Week 1 (N = 13, 39, 13, 28, 4, 8)
    -0.7 ( 1.28 )
    -0.4 ( 1.36 )
    0.2 ( 0.82 )
    0.0 ( 0.55 )
    0.2 ( 0.10 )
    -0.2 ( 0.51 )
        Change at Week 2 (N = 13, 39, 13, 28, 4, 8)
    -0.2 ( 0.87 )
    -0.3 ( 0.99 )
    0.3 ( 1.08 )
    0.0 ( 0.64 )
    0.5 ( 0.21 )
    -0.1 ( 0.50 )
        Change at Week 4 (N = 13, 39, 13, 28, 4, 8)
    -0.2 ( 1.67 )
    -0.1 ( 1.15 )
    0.4 ( 0.90 )
    0.1 ( 0.90 )
    0.0 ( 0.22 )
    -0.1 ( 0.49 )
        Change at Week 8 (N = 13, 39, 13, 28, 4, 8)
    -0.5 ( 2.39 )
    0.0 ( 1.53 )
    0.6 ( 1.57 )
    0.1 ( 1.21 )
    0.3 ( 0.14 )
    0.0 ( 0.55 )
        Change at Week 12 (N = 13, 39, 13, 28, 4, 8)
    -0.4 ( 2.62 )
    -0.1 ( 2.01 )
    0.9 ( 2.15 )
    0.5 ( 1.35 )
    0.3 ( 0.22 )
    0.1 ( 0.85 )
        Change at Week 16 (N = NA, 38, NA, 28, NA, 8)
    999 ( 999 )
    0.2 ( 2.35 )
    999 ( 999 )
    0.6 ( 1.34 )
    999 ( 999 )
    0.1 ( 0.76 )
        Change at Week 20 (N = NA, 39, NA, 28, NA, 8)
    999 ( 999 )
    0.1 ( 2.65 )
    999 ( 999 )
    0.9 ( 1.59 )
    999 ( 999 )
    0.4 ( 0.86 )
        Change at Week 24 (N = NA, 36, NA, 27, NA, 8)
    999 ( 999 )
    0.5 ( 3.13 )
    999 ( 999 )
    1.2 ( 1.91 )
    999 ( 999 )
    0.4 ( 1.03 )
        Change at PT Week 4 (N = 13, 38, 13, 28, 4, 8)
    -0.3 ( 3.19 )
    0.9 ( 3.24 )
    1.2 ( 2.20 )
    1.3 ( 2.19 )
    0.5 ( 0.27 )
    0.7 ( 1.05 )
        Change at PT Week 12 (N = 13, 38, 13, 28, 4, 8)
    0.9 ( 2.69 )
    2.1 ( 3.73 )
    2.1 ( 3.28 )
    2.2 ( 2.21 )
    1.0 ( 0.88 )
    0.9 ( 1.12 )
        Change at PT Week 24 (N = 13, 37, 13, 27, 3, 8)
    2.5 ( 4.98 )
    3.0 ( 4.62 )
    3.8 ( 3.67 )
    3.5 ( 2.42 )
    1.3 ( 1.10 )
    1.7 ( 1.28 )
    No statistical analyses for this end point

    Secondary: For the Treatment Phase, Number of Male Participants With a Change From Baseline in Tanner Stage for Pubic Hair

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    End point title
    		 For the Treatment Phase, Number of Male Participants With a Change From Baseline in Tanner Stage for Pubic Hair
    End point description
    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. Male participants in the Safety Analysis Set with available data were analyzed.
    End point type
    Secondary
    End point timeframe
    Baseline; End of Treatment (either Week 12 or 24), Posttreatment Week 12, and Posttreatment Week 24
    End point values
    		 Males 12 to < 18 Years Old - SOF+RBV 12 or 24 Weeks Males 6 to < 12 Years Old - SOF+RBV 12 or 24 Weeks Males 3 to < 6 Years Old - SOF+RBV 12 or 24 Weeks
    Number of subjects analysed
    31
    11
    3
    Units: participants
        End of Treatment - No Change (N = 31, 11, 3)
    23
    10
    3
        End of Treatment - Increase (N = 31, 11, 3)
    8
    1
    0
        End of Treatment - Decrease (N = 31, 11, 3)
    0
    0
    0
        Posttreatment Week 12 - No Change (N = 30, 11, 3)
    20
    9
    3
        Posttreatment Week 12 - Increase (N = 30, 11, 3)
    10
    2
    0
        Posttreatment Week 12 - Decrease (N = 30, 11, 3)
    0
    0
    0
        Posttreatment Week 24 - No Change (N = 31, 11, 3)
    20
    9
    3
        Posttreatment Week 24 - Increase (N = 31, 11, 3)
    11
    2
    0
        Posttreatment Week 24 - Decrease (N = 31, 11, 3)
    0
    0
    0
    No statistical analyses for this end point

    Secondary: For the Treatment Phase, Number of Male Participants With a Change From Baseline in Tanner Stage for Genitalia Development

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    End point title
    		 For the Treatment Phase, Number of Male Participants With a Change From Baseline in Tanner Stage for Genitalia Development
    End point description
    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. Male participants in the Safety Analysis Set with available data were analyzed.
    End point type
    Secondary
    End point timeframe
    Baseline; End of Treatment (either Week 12 or 24), Posttreatment Week 12, and Posttreatment Week 24
    End point values
    		 Males 12 to < 18 Years Old - SOF+RBV 12 or 24 Weeks Males 6 to < 12 Years Old - SOF+RBV 12 or 24 Weeks Males 3 to < 6 Years Old - SOF+RBV 12 or 24 Weeks
    Number of subjects analysed
    31
    11
    3
    Units: participants
        End of Treatment - No Change (N = 31, 11, 3)
    24
    11
    3
        End of Treatment - Increase (N = 31, 11, 3)
    7
    0
    0
        End of Treatment - Decrease (N = 31, 11, 3)
    0
    0
    0
        Posttreatment Week 12 - No Change (N = 30, 11, 3)
    21
    10
    3
        Posttreatment Week 12 - Increase (N = 30, 11, 3)
    9
    1
    0
        Posttreatment Week 12 - Decrease (N = 30, 11, 3)
    0
    0
    0
        Posttreatment Week 24 - No Change (N = 31, 11, 3)
    20
    9
    3
        Posttreatment Week 24 - Increase (N = 31, 11, 3)
    11
    2
    0
        Posttreatment Week 24 - Decrease (N = 31, 11, 3)
    0
    0
    0
    No statistical analyses for this end point

    Secondary: For the Treatment Phase, Number of Female Participants With a Change From Baseline in Tanner Stage for Pubic Hair

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    End point title
    		 For the Treatment Phase, Number of Female Participants With a Change From Baseline in Tanner Stage for Pubic Hair
    End point description
    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. Female participants in the Safety Analysis Set with available data were analyzed.
    End point type
    Secondary
    End point timeframe
    Baseline; End of Treatment (either Week 12 or 24), Posttreatment Week 12, and Posttreatment Week 24
    End point values
    		 Females 12 to < 18 Years Old - SOF+RBV 12 or 24 Weeks Females 6 to < 12 Years Old - SOF+RBV 12 or 24 Weeks Females 3 to < 6 Years Old - SOF+RBV 12 or 24 Weeks
    Number of subjects analysed
    21
    30
    8
    Units: participants
        End of Treatment - No Change (N = 21, 30, 8)
    20
    24
    8
        End of Treatment - Increase (N = 21, 30, 8)
    1
    6
    0
        End of Treatment - Decrease (N = 21, 30, 8)
    0
    0
    0
        Posttreatment Week 12 - No Change (N = 20, 29, 8)
    18
    23
    8
        Posttreatment Week 12 - Increase (N = 20, 29, 8)
    2
    6
    0
        Posttreatment Week 12 - Decrease (N = 20, 29, 8)
    0
    0
    0
        Posttreatment Week 24 - No Change (N = 20, 30, 8)
    18
    22
    8
        Posttreatment Week 24 - Increase (N = 20, 30, 8)
    2
    8
    0
        Posttreatment Week 24 - Decrease (N = 20, 30, 8)
    0
    0
    0
    No statistical analyses for this end point

    Secondary: For the Treatment Phase, Number of Female Participants With a Change From Baseline in Tanner Stage for Breast Development

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    End point title
    		 For the Treatment Phase, Number of Female Participants With a Change From Baseline in Tanner Stage for Breast Development
    End point description
    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. Female participants in the Safety Analysis Set with available data were analyzed.
    End point type
    Secondary
    End point timeframe
    Baseline; End of Treatment (either Week 12 or 24), Posttreatment Week 12, and Posttreatment Week 24
    End point values
    		 Females 12 to < 18 Years Old - SOF+RBV 12 or 24 Weeks Females 6 to < 12 Years Old - SOF+RBV 12 or 24 Weeks Females 3 to < 6 Years Old - SOF+RBV 12 or 24 Weeks
    Number of subjects analysed
    21
    30
    8
    Units: participants
        End of Treatment - No Change (N = 21, 30, 8)
    20
    24
    8
        End of Treatment - Increase (N = 21, 30, 8)
    1
    5
    0
        End of Treatment - Decrease (N = 21, 30, 8)
    0
    1
    0
        Posttreatment Week 12 - No Change (N = 20, 29, 8)
    17
    24
    8
        Posttreatment Week 12 - Increase (N = 20, 29, 8)
    3
    5
    0
        Posttreatment Week 12 - Decrease (N = 20, 29, 8)
    0
    0
    0
        Posttreatment Week 24 - No Change (N = 20, 30, 8)
    16
    19
    8
        Posttreatment Week 24 - Increase (N = 20, 30, 8)
    4
    11
    0
        Posttreatment Week 24 - Decrease (N = 20, 30, 8)
    0
    0
    0
    No statistical analyses for this end point

    Secondary: 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

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    End point title
    		 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
    End point description
    Participants were asked if they were able to taste the SOF oral granules. Participants in the Safety Analysis Set who performed the palatability assessment were analyzed.
    End point type
    Secondary
    End point timeframe
    Day 1
    End point values
    		 12 to < 18 Years Old - SOF+RBV 12 or 24 Weeks 6 to < 12 Years Old - SOF+RBV 12 or 24 Weeks 3 to < 6 Years Old - SOF+RBV 12 or 24 Weeks
    Number of subjects analysed
    1
    7
    12
    Units: percentage of participants
    number (not applicable)
        Able to Taste SOF Granules: Yes
    0
    42.9
    75.0
        Able to Taste SOF Granules: No
    100.0
    57.1
    25.0
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    Adverse Events: Up to 12 or 24 weeks (depending on group) plus 30 days; All-Cause Mortality: Up to Posttreatment Week 24
    Adverse event reporting additional description
    Safety Analysis Set included all participants who took at least 1 dose of study drug.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    20.1
    Reporting groups
    Reporting group title
    12 to < 18 Years Old - SOF+RBV 12 Weeks
    Reporting group description
    		 Participants 12 to < 18 years of age with HCV genotype 2 received SOF 400 mg (1 x 400 mg tablet, 4 x 100 mg tablets, or 8 x 50 mg oral granules) once daily + RBV capsules or oral solution (up to 1400 mg, dose depending on weight) for 12 weeks.

    Reporting group title
    12 to < 18 Years Old - SOF+RBV 24 Weeks
    Reporting group description
    		 Participants 12 to < 18 years of age with HCV genotype 3 received SOF 400 mg (1 x 400 mg tablet, 4 x 100 mg tablets, or 8 x 50 mg oral granules) once daily + RBV capsules or oral solution (up to 1400 mg, dose depending on weight) for 24 weeks.

    Reporting group title
    6 to < 12 Years Old - SOF+RBV 12 Weeks
    Reporting group description
    		 Participants 6 to < 12 years of age with HCV genotype 2 received SOF 200 mg (2 x 100 mg tablets or 4 x 50 mg oral granules) once daily + RBV capsules or oral solution (up to 1400 mg, dose depending on weight) for 12 weeks.

    Reporting group title
    6 to < 12 Years Old - SOF+RBV 24 Weeks
    Reporting group description
    		 Participants 6 to < 12 years of age with HCV genotype 3 received SOF 200 mg (2 x 100 mg tablets or 4 x 50 mg oral granules) once daily + RBV capsules or oral solution (up to 1400 mg, dose depending on weight) for 24 weeks.

    Reporting group title
    3 to < 6 Years Old - SOF+RBV 12 Weeks
    Reporting group description
    		 Participants 3 to < 6 years of age with HCV genotype 2 received SOF (weight ≥ 17 kg: 200 mg granules; weight < 17 kg: 150 mg granules) once daily + RBV capsules or oral solution (up to 1400 mg, dose depending on weight) for 12 weeks.

    Reporting group title
    3 to < 6 Years Old - SOF+RBV 24 Weeks
    Reporting group description
    		 Participants 3 to < 6 years of age with HCV genotype 3 received SOF (weight ≥ 17 kg: 200 mg granules; weight < 17 kg: 150 mg granules) once daily + RBV capsules or oral solution (up to 1400 mg, dose depending on weight) for 24 weeks.

    Serious adverse events
    		 12 to < 18 Years Old - SOF+RBV 12 Weeks 12 to < 18 Years Old - SOF+RBV 24 Weeks 6 to < 12 Years Old - SOF+RBV 12 Weeks 6 to < 12 Years Old - SOF+RBV 24 Weeks 3 to < 6 Years Old - SOF+RBV 12 Weeks 3 to < 6 Years Old - SOF+RBV 24 Weeks
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 13 (0.00%)
    0 / 39 (0.00%)
    0 / 13 (0.00%)
    0 / 28 (0.00%)
    0 / 5 (0.00%)
    1 / 8 (12.50%)
         number of deaths (all causes)
    0
    0
    0
    0
    0
    0
         number of deaths resulting from adverse events
    0
    0
    0
    0
    0
    0
    Injury, poisoning and procedural complications
    Accidental overdose
         subjects affected / exposed
    0 / 13 (0.00%)
    0 / 39 (0.00%)
    0 / 13 (0.00%)
    0 / 28 (0.00%)
    0 / 5 (0.00%)
    1 / 8 (12.50%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 12 to < 18 Years Old - SOF+RBV 12 Weeks 12 to < 18 Years Old - SOF+RBV 24 Weeks 6 to < 12 Years Old - SOF+RBV 12 Weeks 6 to < 12 Years Old - SOF+RBV 24 Weeks 3 to < 6 Years Old - SOF+RBV 12 Weeks 3 to < 6 Years Old - SOF+RBV 24 Weeks
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    12 / 13 (92.31%)
    27 / 39 (69.23%)
    9 / 13 (69.23%)
    24 / 28 (85.71%)
    5 / 5 (100.00%)
    8 / 8 (100.00%)
    Surgical and medical procedures
    Tooth extraction
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 39 (0.00%)
    0 / 13 (0.00%)
    0 / 28 (0.00%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    0 / 13 (0.00%)
    4 / 39 (10.26%)
    3 / 13 (23.08%)
    5 / 28 (17.86%)
    1 / 5 (20.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    6
    3
    5
    1
    1
    Asthenia
         subjects affected / exposed
    1 / 13 (7.69%)
    5 / 39 (12.82%)
    1 / 13 (7.69%)
    0 / 28 (0.00%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    1
    6
    2
    0
    0
    0
    Pyrexia
         subjects affected / exposed
    0 / 13 (0.00%)
    0 / 39 (0.00%)
    0 / 13 (0.00%)
    4 / 28 (14.29%)
    1 / 5 (20.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    0
    9
    2
    0
    Influenza like illness
         subjects affected / exposed
    0 / 13 (0.00%)
    0 / 39 (0.00%)
    0 / 13 (0.00%)
    0 / 28 (0.00%)
    0 / 5 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Malaise
         subjects affected / exposed
    0 / 13 (0.00%)
    0 / 39 (0.00%)
    1 / 13 (7.69%)
    0 / 28 (0.00%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    1
    0
    0
    0
    Immune system disorders
    Seasonal allergy
         subjects affected / exposed
    0 / 13 (0.00%)
    0 / 39 (0.00%)
    0 / 13 (0.00%)
    0 / 28 (0.00%)
    1 / 5 (20.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Reproductive system and breast disorders
    Dysmenorrhoea
         subjects affected / exposed
    0 / 13 (0.00%)
    2 / 39 (5.13%)
    0 / 13 (0.00%)
    0 / 28 (0.00%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    3
    0
    0
    0
    0
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    0 / 13 (0.00%)
    3 / 39 (7.69%)
    1 / 13 (7.69%)
    7 / 28 (25.00%)
    1 / 5 (20.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    4
    1
    9
    1
    0
    Oropharyngeal pain
         subjects affected / exposed
    0 / 13 (0.00%)
    4 / 39 (10.26%)
    0 / 13 (0.00%)
    4 / 28 (14.29%)
    0 / 5 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    4
    0
    5
    0
    1
    Rhinorrhoea
         subjects affected / exposed
    1 / 13 (7.69%)
    2 / 39 (5.13%)
    1 / 13 (7.69%)
    3 / 28 (10.71%)
    0 / 5 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    1
    2
    1
    3
    0
    1
    Epistaxis
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 39 (2.56%)
    1 / 13 (7.69%)
    1 / 28 (3.57%)
    0 / 5 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    1
    1
    1
    0
    1
    Sinus congestion
         subjects affected / exposed
    0 / 13 (0.00%)
    0 / 39 (0.00%)
    0 / 13 (0.00%)
    2 / 28 (7.14%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    0
    2
    0
    0
    Pharyngeal erythema
         subjects affected / exposed
    0 / 13 (0.00%)
    0 / 39 (0.00%)
    0 / 13 (0.00%)
    0 / 28 (0.00%)
    0 / 5 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Upper respiratory tract congestion
         subjects affected / exposed
    0 / 13 (0.00%)
    0 / 39 (0.00%)
    0 / 13 (0.00%)
    0 / 28 (0.00%)
    1 / 5 (20.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Psychiatric disorders
    Insomnia
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 39 (2.56%)
    1 / 13 (7.69%)
    1 / 28 (3.57%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    1
    1
    0
    0
    Irritability
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 39 (0.00%)
    1 / 13 (7.69%)
    0 / 28 (0.00%)
    1 / 5 (20.00%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    1
    0
    1
    0
    Mood swings
         subjects affected / exposed
    0 / 13 (0.00%)
    2 / 39 (5.13%)
    0 / 13 (0.00%)
    1 / 28 (3.57%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    2
    0
    1
    0
    0
    Middle insomnia
         subjects affected / exposed
    0 / 13 (0.00%)
    0 / 39 (0.00%)
    0 / 13 (0.00%)
    0 / 28 (0.00%)
    1 / 5 (20.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Product issues
    Product taste abnormal
         subjects affected / exposed
    0 / 13 (0.00%)
    0 / 39 (0.00%)
    0 / 13 (0.00%)
    1 / 28 (3.57%)
    1 / 5 (20.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    0
    1
    1
    0
    Investigations
    Weight decreased
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 39 (0.00%)
    1 / 13 (7.69%)
    1 / 28 (3.57%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    1
    1
    0
    0
    Cardiac murmur
         subjects affected / exposed
    0 / 13 (0.00%)
    0 / 39 (0.00%)
    1 / 13 (7.69%)
    1 / 28 (3.57%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    1
    1
    0
    0
    Blood creatine phosphokinase increased
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 39 (0.00%)
    0 / 13 (0.00%)
    0 / 28 (0.00%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Injury, poisoning and procedural complications
    Contusion
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 39 (2.56%)
    0 / 13 (0.00%)
    3 / 28 (10.71%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    0
    3
    0
    0
    Arthropod bite
         subjects affected / exposed
    0 / 13 (0.00%)
    0 / 39 (0.00%)
    0 / 13 (0.00%)
    0 / 28 (0.00%)
    0 / 5 (0.00%)
    2 / 8 (25.00%)
         occurrences all number
    0
    0
    0
    0
    0
    2
    Fall
         subjects affected / exposed
    0 / 13 (0.00%)
    0 / 39 (0.00%)
    0 / 13 (0.00%)
    1 / 28 (3.57%)
    0 / 5 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    0
    1
    0
    1
    Scratch
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 39 (0.00%)
    0 / 13 (0.00%)
    1 / 28 (3.57%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    0
    1
    0
    0
    Ligament injury
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 39 (0.00%)
    0 / 13 (0.00%)
    0 / 28 (0.00%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Product use issue
         subjects affected / exposed
    0 / 13 (0.00%)
    0 / 39 (0.00%)
    0 / 13 (0.00%)
    0 / 28 (0.00%)
    1 / 5 (20.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Road traffic accident
         subjects affected / exposed
    0 / 13 (0.00%)
    0 / 39 (0.00%)
    0 / 13 (0.00%)
    0 / 28 (0.00%)
    0 / 5 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Nervous system disorders
    Headache
         subjects affected / exposed
    3 / 13 (23.08%)
    9 / 39 (23.08%)
    4 / 13 (30.77%)
    8 / 28 (28.57%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    5
    16
    4
    25
    0
    0
    Dizziness
         subjects affected / exposed
    0 / 13 (0.00%)
    4 / 39 (10.26%)
    0 / 13 (0.00%)
    1 / 28 (3.57%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    4
    0
    1
    0
    0
    Migraine
         subjects affected / exposed
    0 / 13 (0.00%)
    0 / 39 (0.00%)
    0 / 13 (0.00%)
    2 / 28 (7.14%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    0
    10
    0
    0
    Presyncope
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 39 (0.00%)
    0 / 13 (0.00%)
    0 / 28 (0.00%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Blood and lymphatic system disorders
    Lymphadenopathy
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 39 (2.56%)
    0 / 13 (0.00%)
    2 / 28 (7.14%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    0
    3
    0
    0
    Ear and labyrinth disorders
    Ear pain
         subjects affected / exposed
    0 / 13 (0.00%)
    0 / 39 (0.00%)
    1 / 13 (7.69%)
    1 / 28 (3.57%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    1
    1
    0
    0
    Motion sickness
         subjects affected / exposed
    0 / 13 (0.00%)
    0 / 39 (0.00%)
    0 / 13 (0.00%)
    1 / 28 (3.57%)
    0 / 5 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    0
    4
    0
    1
    Gastrointestinal disorders
    Vomiting
         subjects affected / exposed
    1 / 13 (7.69%)
    3 / 39 (7.69%)
    2 / 13 (15.38%)
    11 / 28 (39.29%)
    3 / 5 (60.00%)
    3 / 8 (37.50%)
         occurrences all number
    1
    3
    3
    16
    4
    4
    Nausea
         subjects affected / exposed
    3 / 13 (23.08%)
    11 / 39 (28.21%)
    0 / 13 (0.00%)
    4 / 28 (14.29%)
    0 / 5 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    4
    13
    0
    4
    0
    1
    Diarrhoea
         subjects affected / exposed
    2 / 13 (15.38%)
    1 / 39 (2.56%)
    1 / 13 (7.69%)
    3 / 28 (10.71%)
    2 / 5 (40.00%)
    3 / 8 (37.50%)
         occurrences all number
    3
    1
    1
    4
    2
    4
    Abdominal pain upper
         subjects affected / exposed
    2 / 13 (15.38%)
    2 / 39 (5.13%)
    1 / 13 (7.69%)
    0 / 28 (0.00%)
    1 / 5 (20.00%)
    0 / 8 (0.00%)
         occurrences all number
    2
    2
    1
    0
    1
    0
    Abdominal pain
         subjects affected / exposed
    0 / 13 (0.00%)
    0 / 39 (0.00%)
    0 / 13 (0.00%)
    3 / 28 (10.71%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    0
    3
    0
    0
    Abdominal pain lower
         subjects affected / exposed
    0 / 13 (0.00%)
    3 / 39 (7.69%)
    0 / 13 (0.00%)
    0 / 28 (0.00%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    3
    0
    0
    0
    0
    Toothache
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 39 (2.56%)
    1 / 13 (7.69%)
    1 / 28 (3.57%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    1
    1
    0
    0
    Mouth ulceration
         subjects affected / exposed
    0 / 13 (0.00%)
    0 / 39 (0.00%)
    1 / 13 (7.69%)
    0 / 28 (0.00%)
    0 / 5 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    1
    0
    0
    1
    Anal pruritus
         subjects affected / exposed
    0 / 13 (0.00%)
    0 / 39 (0.00%)
    0 / 13 (0.00%)
    0 / 28 (0.00%)
    0 / 5 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Epigastric discomfort
         subjects affected / exposed
    0 / 13 (0.00%)
    0 / 39 (0.00%)
    0 / 13 (0.00%)
    0 / 28 (0.00%)
    0 / 5 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Gastrooesophageal reflux disease
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 39 (0.00%)
    0 / 13 (0.00%)
    0 / 28 (0.00%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Oral contusion
         subjects affected / exposed
    0 / 13 (0.00%)
    0 / 39 (0.00%)
    0 / 13 (0.00%)
    0 / 28 (0.00%)
    0 / 5 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Tooth loss
         subjects affected / exposed
    0 / 13 (0.00%)
    0 / 39 (0.00%)
    0 / 13 (0.00%)
    0 / 28 (0.00%)
    0 / 5 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Skin and subcutaneous tissue disorders
    Rash
         subjects affected / exposed
    1 / 13 (7.69%)
    3 / 39 (7.69%)
    0 / 13 (0.00%)
    2 / 28 (7.14%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    1
    3
    0
    2
    0
    0
    Pruritus
         subjects affected / exposed
    1 / 13 (7.69%)
    1 / 39 (2.56%)
    1 / 13 (7.69%)
    1 / 28 (3.57%)
    0 / 5 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    1
    1
    1
    1
    0
    1
    Alopecia
         subjects affected / exposed
    0 / 13 (0.00%)
    2 / 39 (5.13%)
    0 / 13 (0.00%)
    0 / 28 (0.00%)
    1 / 5 (20.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    2
    0
    0
    1
    0
    Acne
         subjects affected / exposed
    1 / 13 (7.69%)
    1 / 39 (2.56%)
    0 / 13 (0.00%)
    0 / 28 (0.00%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    1
    1
    0
    0
    0
    0
    Skin exfoliation
         subjects affected / exposed
    0 / 13 (0.00%)
    2 / 39 (5.13%)
    0 / 13 (0.00%)
    0 / 28 (0.00%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    2
    0
    0
    0
    0
    Erythema
         subjects affected / exposed
    0 / 13 (0.00%)
    0 / 39 (0.00%)
    0 / 13 (0.00%)
    0 / 28 (0.00%)
    0 / 5 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Rash papular
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 39 (0.00%)
    0 / 13 (0.00%)
    0 / 28 (0.00%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Musculoskeletal and connective tissue disorders
    Pain in extremity
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 39 (2.56%)
    0 / 13 (0.00%)
    2 / 28 (7.14%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    0
    2
    0
    0
    Arthralgia
         subjects affected / exposed
    0 / 13 (0.00%)
    0 / 39 (0.00%)
    0 / 13 (0.00%)
    2 / 28 (7.14%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    0
    2
    0
    0
    Muscle spasms
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 39 (0.00%)
    0 / 13 (0.00%)
    1 / 28 (3.57%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    0
    1
    0
    0
    Myalgia
         subjects affected / exposed
    0 / 13 (0.00%)
    0 / 39 (0.00%)
    0 / 13 (0.00%)
    2 / 28 (7.14%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    0
    2
    0
    0
    Joint swelling
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 39 (0.00%)
    0 / 13 (0.00%)
    0 / 28 (0.00%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Muscular weakness
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 39 (0.00%)
    0 / 13 (0.00%)
    0 / 28 (0.00%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    2
    0
    0
    0
    0
    0
    Infections and infestations
    Nasopharyngitis
         subjects affected / exposed
    0 / 13 (0.00%)
    3 / 39 (7.69%)
    1 / 13 (7.69%)
    4 / 28 (14.29%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    5
    1
    4
    0
    0
    Upper respiratory tract infection
         subjects affected / exposed
    0 / 13 (0.00%)
    2 / 39 (5.13%)
    0 / 13 (0.00%)
    2 / 28 (7.14%)
    0 / 5 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    2
    0
    2
    0
    1
    Oral herpes
         subjects affected / exposed
    1 / 13 (7.69%)
    2 / 39 (5.13%)
    0 / 13 (0.00%)
    1 / 28 (3.57%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    1
    2
    0
    1
    0
    0
    Ear infection
         subjects affected / exposed
    0 / 13 (0.00%)
    2 / 39 (5.13%)
    0 / 13 (0.00%)
    0 / 28 (0.00%)
    0 / 5 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    2
    0
    0
    0
    1
    Gastroenteritis
         subjects affected / exposed
    0 / 13 (0.00%)
    0 / 39 (0.00%)
    0 / 13 (0.00%)
    3 / 28 (10.71%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    0
    3
    0
    0
    Impetigo
         subjects affected / exposed
    0 / 13 (0.00%)
    2 / 39 (5.13%)
    0 / 13 (0.00%)
    0 / 28 (0.00%)
    0 / 5 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    2
    0
    0
    0
    1
    Influenza
         subjects affected / exposed
    0 / 13 (0.00%)
    2 / 39 (5.13%)
    0 / 13 (0.00%)
    1 / 28 (3.57%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    2
    0
    1
    0
    0
    Rhinitis
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 39 (2.56%)
    0 / 13 (0.00%)
    2 / 28 (7.14%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    0
    2
    0
    0
    Respiratory tract infection viral
         subjects affected / exposed
    0 / 13 (0.00%)
    0 / 39 (0.00%)
    1 / 13 (7.69%)
    1 / 28 (3.57%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    1
    1
    0
    0
    Tinea infection
         subjects affected / exposed
    0 / 13 (0.00%)
    1 / 39 (2.56%)
    0 / 13 (0.00%)
    0 / 28 (0.00%)
    1 / 5 (20.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    1
    0
    0
    1
    0
    Urinary tract infection
         subjects affected / exposed
    1 / 13 (7.69%)
    1 / 39 (2.56%)
    0 / 13 (0.00%)
    0 / 28 (0.00%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    1
    1
    0
    0
    0
    0
    Conjunctivitis viral
         subjects affected / exposed
    0 / 13 (0.00%)
    0 / 39 (0.00%)
    0 / 13 (0.00%)
    0 / 28 (0.00%)
    1 / 5 (20.00%)
    0 / 8 (0.00%)
         occurrences all number
    0
    0
    0
    0
    1
    0
    Folliculitis
         subjects affected / exposed
    0 / 13 (0.00%)
    0 / 39 (0.00%)
    0 / 13 (0.00%)
    0 / 28 (0.00%)
    0 / 5 (0.00%)
    1 / 8 (12.50%)
         occurrences all number
    0
    0
    0
    0
    0
    1
    Fungal skin infection
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 39 (0.00%)
    0 / 13 (0.00%)
    0 / 28 (0.00%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Pharyngitis
         subjects affected / exposed
    1 / 13 (7.69%)
    0 / 39 (0.00%)
    0 / 13 (0.00%)
    0 / 28 (0.00%)
    0 / 5 (0.00%)
    0 / 8 (0.00%)
         occurrences all number
    1
    0
    0
    0
    0
    0
    Metabolism and nutrition disorders
    Decreased appetite
         subjects affected / exposed
    1 / 13 (7.69%)
    2 / 39 (5.13%)
    2 / 13 (15.38%)
    2 / 28 (7.14%)
    0 / 5 (0.00%)
    3 / 8 (37.50%)
         occurrences all number
    1
    3
    2
    2
    0
    4

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    10 Feb 2014
    ● Specified that subjects must be treatment naive for the PK lead-in phase ● Specified that subjects rolling over from the PK lead-in phase would not be required to perform laboratory assessments on Day 1 of the treatment phase ● Included the protocol number (GS-US-334-1113) for the long-term follow-up study ● Added coagulation testing to Day 7 of the PK lead-in phase ● Specified that subjects would be administered a dosing diary with instructions for the PK lead-in phase ● Removed SVR24 from the subgroup analyses ● Added creatine kinase as a laboratory assessment ● Added additional information regarding the development of SOF oral granular formulation ● Specified that the subject and/or legal guardian would be asked at screening if the subject was able to swallow and tolerate taking pills ● Added information regarding the SOF swallowability assessment on Day 1 of the PK lead-in phase or the treatment phase (as applicable) ● Identified the PedsQL™ Pediatric Quality of Life Inventory V4.0 Short Form (SF15) as the Quality of Life survey instrument ● Made additional administrative updates
    14 Mar 2014
    ● Added RBV dosing to the PK lead-in phase ● Specified that subjects rolling over from the PK lead-in phase would not be required to perform screening, Day 1, or Week 1 visits in the treatment phase ● Added REBETOL® as an RBV study drug and updated the weight-based dosing to reflect the approved dosing criteria ● Moved efficacy from a primary endpoint to a secondary endpoint ● Removed the Quality of Life survey as a secondary endpoint ● Clarified the safety endpoint and added historical SVR12 comparison ● Clarified that the swallowability assessment would be performed with a placebo tablet to assess ability to swallow the 400 mg or 100 mg SOF tablet and specified that it would occur from screening up to the Day 1 visit ● Added placebo tablet description and handling ● Added bone age assessment as part of the safety assessment ● Added virologic breakthrough futility assessment ● Added inclusion criteria for treatment-experienced subjects ● Modified the inclusion criteria for hemoglobin with levels for males ≥ 12 g/dL and females ≥ 11 g/dL ● Added exclusion criteria for the glomerular filtration rate to be < 90 mL/min/1.73m^2 as calculated by the Schwartz Formula, and added the Schwartz Formula ● Added dose reduction guidance of RBV per the REBETOL prescribing information ● Added laboratory assessment of amylase ● Made additional administrative changes
    10 Oct 2014
    ● Added India to study center countries ● Clarified the breakthrough futility assessment to specify that further enrollment would be suspended if 3 or more of the first 10 subjects enrolled had viral breakthrough at or prior to Week 8 ● Added SOF oral granule formulation/packaging information as well as a palatability assessment ● Included bioavailability data of SOF oral granules from Study GS-US-334-1111 ● Revised the table for RBV dose administration by weight to allow participants whose body weight is ≥ 47 kg to receive oral ribavirin doses ● Revised the SOF 400 mg pill count to be either 30 or 28 tablets per bottle ● Clarified that subjects who did not attain SVR24 will also be enrolled in the separate GS-US-334-1113 protocol ● Added clarification for pregnancy counseling of subjects < 12 years of age ● Added parental height to medical history ● Made additional administrative changes
    12 Nov 2014
    ● Added language to clarify the reconsent requirement for subjects who became adults while on the study ● Updated the RBV dosing chart to reflect accurate dosing of RBV for subjects ≥ 47 kg who may have utilized the RBV oral solution ● Clarified that Gilead Sciences (Gilead), not the sites, would calculate the height/weight percentiles according to the Centers for Disease Control and Prevention (CDC) reference charts ●Updated the statistical analysis secondary endpoint language to include previously omitted palatability assessment of SOF oral granules ● Made additional administrative changes
    29 May 2015
    ● Weight restrictions for Cohort 2 were added based on the Cohort 1 data analysis ● The dose for Cohort 2 was added based on the Cohort 1 data analysis ● Clarified the contraception language in the exclusion criteria ● Updated statistical methods to align with regulatory requirements ● Updated the table for dosage and administration of RBV to clarify the body weight range of 66 to 80 kgs (145 to 176 lbs) ● Made additional administrative changes
    26 Feb 2016
    ● The dose for Cohort 3 was added based on the Cohort 2 data analysis ● Updated PK and safety results ● Removed the collection and analysis of age of first menses ● Clarified the glomerular filtration rate calculation ● Made additional administrative changes

    Interruptions (globally)
    Were there any global interruptions to the trial? No

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported

    Online references
    http://www.ncbi.nlm.nih.gov/pubmed/28543053
    http://www.ncbi.nlm.nih.gov/pubmed/29193603
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