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    Clinical Trial Results:
    A Phase 2, Open-Label, Multicenter, Multi-cohort Study to Investigate the Pharmacokinetics, Safety and Efficacy of Sofosbuvir/Velpatasvir/Voxilaprevir Fixed Dose Combination in Adolescents and Children with Chronic HCV Infection

    Summary
    EudraCT number
    		 2018-000480-87
    Trial protocol
    		 DE   GB   PL   IT  
    Global end of trial date
    19 Feb 2020

    Results information
    Results version number
    		 v1
    This version publication date
    29 Aug 2020
    First version publication date
    29 Aug 2020
    Other versions
    		 v2

    Trial information

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    Trial identification
    Sponsor protocol code
    GS-US-367-1175
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 -
    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-001822-PIP01-15
    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
    19 Feb 2020
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    04 Dec 2019
    Global end of trial reached?
    Yes
    Global end of trial date
    19 Feb 2020
    Was the trial ended prematurely?
    Yes
    General information about the trial
    Main objective of the trial
    The primary objective of this study was to evaluate the steady-state pharmacokinetics (PK) and confirm the age-appropriate dose of sofosbuvir/velpatasvir/voxilaprevir (SOF/VEL/VOX) fixed-dose combination (FDC) in pediatric participants with chronic hepatitis C virus (HCV) infection.
    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
    28 Jan 2019
    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
    Italy: 13
    Country: Number of subjects enrolled
    Poland: 4
    Country: Number of subjects enrolled
    United Kingdom: 4
    Worldwide total number of subjects
    21
    EEA total number of subjects
    21
    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)
    0
    Adolescents (12-17 years)
    21
    Adults (18-64 years)
    0
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

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    Recruitment
    Recruitment details
    		 Participants were enrolled at study sites in Europe. The first participant was screened on 28 January 2019. The last study visit occurred on 19 February 2020.

    Pre-assignment
    Screening details
    		 Participants were planned to be enrolled into Cohorts 1, 2, and 3. The study was terminated because EMA granted Gilead a waiver for SOF/VEL/VOX in children less than 12 years old. Cohorts 2 and 3 were not enrolled.

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

    Arms
    Arm title
    		 SOF/VEL/VOX FDC
    Arm description
    		 Direct-acting antiviral (DAA) - naive participants without cirrhosis (12 to < 18 years old) received sofosbuvir/velpatasvir/voxilaprevir (SOF/VEL/VOX) fixed-dose (FDC) combination (400/100/100 mg tablet) orally once daily in nonfasting state for 8 weeks. A single placebo to match tablet was administered during screening until Day 1 to confirm the participant was able to swallow SOF/VEL/VOX 400/100/100 mg tablets.
    Arm type
    		 Experimental

    Investigational medicinal product name
    SOF/VEL/VOX FDC
    Investigational medicinal product code
    Other name
    Vosevi ®
    Pharmaceutical forms
    Film-coated tablet
    Routes of administration
    Oral use
    Dosage and administration details
    400/100/100 mg administered once daily

    Number of subjects in period 1
    		SOF/VEL/VOX FDC
    Started
    21
    Completed
    21

    Baseline characteristics

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    Baseline characteristics reporting groups
    Reporting group title
    SOF/VEL/VOX FDC
    Reporting group description
    		 Direct-acting antiviral (DAA) - naive participants without cirrhosis (12 to < 18 years old) received sofosbuvir/velpatasvir/voxilaprevir (SOF/VEL/VOX) fixed-dose (FDC) combination (400/100/100 mg tablet) orally once daily in nonfasting state for 8 weeks. A single placebo to match tablet was administered during screening until Day 1 to confirm the participant was able to swallow SOF/VEL/VOX 400/100/100 mg tablets.

    Reporting group values
    		 SOF/VEL/VOX FDC Total
    Number of subjects
    		 21 21
    Age categorical
    Units: Subjects
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 14 ( 1.2 ) -
    Gender categorical
    Units: Subjects
        Female
    		 13 13
        Male
    		 8 8
    Race
    Units: Subjects
        White
    		 16 16
        Black or African American
    		 1 1
        Asian
    		 2 2
        Other
    		 2 2
        American Indian or Alaska Native
    		 0 0
        Native Hawaiian or Pacific Islander
    		 0 0
    Ethnicity
    Units: Subjects
        Hispanic or Latino
    		 2 2
        Not Hispanic or Latino
    		 19 19
        Unknown or Not Reported
    		 0 0
    Cirrhosis
    Units: Subjects
        Yes
    		 0 0
        No
    		 21 21
    Baseline Alanine aminotransferase (ALT) Category
    Units: Subjects
        ≤ 1.5 x (Upper Limit of Normal) ULN
    		 16 16
        > 1.5 x ULN
    		 5 5
    Baseline HCV Ribonucleic acid (RNA)
    Units: log10 IU/mL
        arithmetic mean (standard deviation)
    		 5.9 ( 0.70 ) -

    End points

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    End points reporting groups
    Reporting group title
    SOF/VEL/VOX FDC
    Reporting group description
    		 Direct-acting antiviral (DAA) - naive participants without cirrhosis (12 to < 18 years old) received sofosbuvir/velpatasvir/voxilaprevir (SOF/VEL/VOX) fixed-dose (FDC) combination (400/100/100 mg tablet) orally once daily in nonfasting state for 8 weeks. A single placebo to match tablet was administered during screening until Day 1 to confirm the participant was able to swallow SOF/VEL/VOX 400/100/100 mg tablets.

    Primary: Pharmacokinetic (PK) Parameter: AUCtau of SOF, GS-331007 (Metabolite of SOF), VEL, and VOX

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    End point title
    		 Pharmacokinetic (PK) Parameter: AUCtau of SOF, GS-331007 (Metabolite of SOF), VEL, and VOX [1]
    End point description
    AUCtau was defined as concentration of drug over time (the area under the concentration verses time curve over the dosing interval). For participants with separate consent to participate in the optional intensive PK substudy, intensive serial PK blood samples were collected at Week 2 or Week 4. Sparse PK samples were collected from all participants at Weeks 1, 2, 4, and end of treatment/Week 8. Plasma concentration data from all PK samples (intensive and sparse) were combined and used to generate PK parameters of SOF, GS-331007, VEL, and VOX for all participants using a population PK modeling approach.
    End point type
    Primary
    End point timeframe
    Sparse PK Sample (all participants): At Weeks 1 and 8 at any time, Weeks 2 and 4 (predose and between 15 minutes and 4 hours postdose). Intensive PK Sample [PK Substudy (N=14)]: Week 2 or Week 4 (0 (predose), 0.5, 1, 2, 3, 4, 6, 8, and 12 hours postdose).
    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: No statistical comparison was planned or performed.
    End point values
    		 SOF/VEL/VOX FDC
    Number of subjects analysed
    21
    Units: hours•nanogram/milliliter
    arithmetic mean (standard deviation)
        SOF
    2474.8 ( 1247.28 )
        GS-331007 (metabolite of SOF)
    14890.2 ( 3126.35 )
        VEL
    6773.0 ( 2367.30 )
        VOX
    2205.8 ( 1403.45 )
    No statistical analyses for this end point

    Secondary: Percentage of Participants Who Permanently Discontinued Study Drug Due to an Adverse Event

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    End point title
    		 Percentage of Participants Who Permanently Discontinued Study Drug Due to an Adverse Event
    End point description
    Treatment-emergent Adverse Events (TEAE) were defined as events that met 1 or both of the following criteria as any AEs with onset dates on or after the study drug start date and no later than 30 days after the permanent discontinuation of study drug. It also includes the AEs that leads to premature discontinuation of study drug. The Safety Analysis Set included participants who received at least 1 dose of study drug.
    End point type
    Secondary
    End point timeframe
    First dose date up to the last dose date (maximum: 8 Weeks) plus 30 days
    End point values
    		 SOF/VEL/VOX FDC
    Number of subjects analysed
    21
    Units: percentage of participants
        number (not applicable)
    0
    No statistical analyses for this end point

    Secondary: Percentage of Participants With Sustained Virologic Response (SVR) 12 Weeks After Discontinuation of Therapy (SVR12)

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    End point title
    		 Percentage of Participants With Sustained Virologic Response (SVR) 12 Weeks After Discontinuation of Therapy (SVR12)
    End point description
    SVR was defined as hepatitis C virus (HCV RNA) < Lower limit of quantification (LLOQ) (ie, < 15 IU/mL) 12 weeks after discontinuation of the study drug. The Full Analysis Set included all adolescent participants 12 to < 18 years old who were enrolled into the study and took at least 1 dose of study drug (SOF/VEL/VOX FDC).
    End point type
    Secondary
    End point timeframe
    Posttreatment Week 12
    End point values
    		 SOF/VEL/VOX FDC
    Number of subjects analysed
    21
    Units: percentage of participants
        number (confidence interval 95%)
    100.0 (83.9 to 100.0)
    No statistical analyses for this end point

    Secondary: Percentage of Participants With HCV RNA < LLOQ 4 Weeks After Discontinuation of Therapy (SVR4)

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    End point title
    		 Percentage of Participants With HCV RNA < LLOQ 4 Weeks After Discontinuation of Therapy (SVR4)
    End point description
    SVR is defined was HCV RNA < LLOQ (ie, < 15 IU/mL) 4 weeks after discontinuation of the study drug. Participants in the Full Analysis Set were analysed.
    End point type
    Secondary
    End point timeframe
    Posttreatment Week 4
    End point values
    		 SOF/VEL/VOX FDC
    Number of subjects analysed
    21
    Units: percentage of participants
        number (confidence interval 95%)
    100.0 (83.9 to 100.0)
    No statistical analyses for this end point

    Secondary: Percentage of Participants With HCV RNA < LLOQ 24 Weeks After Discontinuation of Therapy (SVR24)

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    End point title
    		 Percentage of Participants With HCV RNA < LLOQ 24 Weeks After Discontinuation of Therapy (SVR24)
    End point description
    SVR is defined was HCV RNA < LLOQ (ie, < 15 IU/mL) 24 weeks after discontinuation of the study drug. Participants in the Full Analysis Set were analysed.
    End point type
    Secondary
    End point timeframe
    Posttreatment Week 24
    End point values
    		 SOF/VEL/VOX FDC
    Number of subjects analysed
    21
    Units: percentage of participants
        number (confidence interval 95%)
    100.0 (83.9 to 100.0)
    No statistical analyses for this end point

    Secondary: Percentage of Participants With Overall Virologic Failure

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    End point title
    		 Percentage of Participants With Overall Virologic Failure
    End point description
    Overall Virologic Failure comprises of on-treatment virologic failure and relapse. On-treatment virologic failure (breakthrough, rebound, and nonresponse) and relapse were defined as follows: Breakthrough (confirmed HCV RNA ≥ LLOQ after having previously had HCV RNA < LLOQ while on treatment), Rebound (confirmed > 1 log10IU/mL increase in HCV RNA from nadir while on treatment), or Nonresponse (HCV RNA persistently ≥ LLOQ through 8 weeks of treatment) and Relapse (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 analysed.
    End point type
    Secondary
    End point timeframe
    Up to Posttreatment Week 24
    End point values
    		 SOF/VEL/VOX FDC
    Number of subjects analysed
    21
    Units: percentage of participants
        number (not applicable)
    0
    No statistical analyses for this end point

    Secondary: Percentage of Participants with HCV RNA < LLOQ on Treatment

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    End point title
    		 Percentage of Participants with HCV RNA < LLOQ on Treatment
    End point description
    Percentage of participants with HCV RNA < LLOQ (15 IU/mL) while on treatment by analysis visit. Participants in the Full Analysis Set were analysed.
    End point type
    Secondary
    End point timeframe
    Weeks 1, 2, 4, and 8
    End point values
    		 SOF/VEL/VOX FDC
    Number of subjects analysed
    21
    Units: percentage of participants
    number (confidence interval 95%)
        Week 1
    52.4 (29.8 to 74.3)
        Week 2
    81.0 (58.1 to 94.6)
        Week 4
    90.5 (69.6 to 98.8)
        Week 8
    100.0 (83.9 to 100.0)
    No statistical analyses for this end point

    Secondary: Percentage of Participants Who Developed Viral Resistance to SOF, VEL, and/or VOX During Treatment

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    End point title
    		 Percentage of Participants Who Developed Viral Resistance to SOF, VEL, and/or VOX During Treatment
    End point description
    Plasma samples were collected and stored for potential HCV sequencing. Impact on the treatment outcomes of SVR12 and SVR24 were observed during the study. Baseline nonstructural protein (NS)3, NS5A, and NS5B deep sequencing analysis was performed for all participants. Sequencing for the HCV NS5A and NS5B regions was performed for all enrolled participants at baseline and for participants with virologic failure. Resistance Analysis Population was defined as all participants in the Safety Analysis Set with a virologic outcome. No on-treatment virologic breakthrough or relapse was observed. Therefore, no participants qualified for resistance testing.
    End point type
    Secondary
    End point timeframe
    Up to End of Treatment (Week 8)
    End point values
    		 SOF/VEL/VOX FDC
    Number of subjects analysed
    21
    Units: percentage of participants
        number (not applicable)
    0
    No statistical analyses for this end point

    Secondary: Percentage of Participants Who Developed Viral Resistance to SOF, VEL, and/or VOX When Treatment is Discontinued

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    End point title
    		 Percentage of Participants Who Developed Viral Resistance to SOF, VEL, and/or VOX When Treatment is Discontinued
    End point description
    Plasma samples were collected and stored for potential HCV sequencing. Impact on the treatment outcomes of SVR12 and SVR24 were observed during the study. Baseline nonstructural protein (NS)3, NS5A, and NS5B deep sequencing analysis was performed for all participants. Sequencing for the HCV NS5A and NS5B regions was performed for all enrolled participants at baseline and for participants with virologic failure. Resistance Analysis Population was defined as all participants in the Safety Analysis Set with a virologic outcome. No on-treatment virologic breakthrough or relapse was observed through posttreatment Week 12 or posttreatment Week 24.
    End point type
    Secondary
    End point timeframe
    Up to Posttreatment Week 24
    End point values
    		 SOF/VEL/VOX FDC
    Number of subjects analysed
    21
    Units: percentage of participants
        number (not applicable)
    0
    No statistical analyses for this end point

    Secondary: Change in HCV RNA from Day 1 Through End of Treatment

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    End point title
    		 Change in HCV RNA from Day 1 Through End of Treatment
    End point description
    Participants in the Full Analysis Set with available data were analysed.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1); Weeks 1, 2 ,4, and 8
    End point values
    		 SOF/VEL/VOX FDC
    Number of subjects analysed
    21
    Units: log10 IU/mL
    arithmetic mean (standard deviation)
        Change at Week 1 (N = 20)
    -4.55 ( 0.525 )
        Change at Week 2
    -4.65 ( 0.621 )
        Change at Week 4 (N = 20)
    -4.71 ( 0.661 )
        Change at Week 8
    -4.77 ( 0.698 )
    No statistical analyses for this end point

    Secondary: Percentage of Participants with Alanine Aminotransferase (ALT) Normalization

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    End point title
    		 Percentage of Participants with Alanine Aminotransferase (ALT) Normalization
    End point description
    ALT normalization was defined as ALT > upper limit of normal (ULN) at baseline and ALT ≤ ULN at each visit. Participants in the Full Analysis Set with available data were analysed. It includes participants with ALT >ULN at Baseline.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1); Week 1, 2, 4, 8, and Posttreatment/Follow-up Week 4 (FU-4)
    End point values
    		 SOF/VEL/VOX FDC
    Number of subjects analysed
    5
    Units: percentage of participants
    number (not applicable)
        Week 1 (N = 4)
    50.0
        Week 2 (N = 4)
    75.0
        Week 4
    100.0
        Week 8
    100.0
        FU - 4 (N = 4)
    100.0
    No statistical analyses for this end point

    Secondary: Change from Baseline in Height Percentiles as a Measurement of Growth and Development

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    End point title
    		 Change from Baseline in Height Percentiles as a Measurement of Growth and Development
    End point description
    An age- and sex-specific percentile was derived for each weight, height, and body mass index (BMI) measurement according to the statistical analysis system (SAS) program available on the Centers for Disease Control and Prevention (CDC) website using the year 2000 growth charts. Participants in the Safety Analysis Set with available data were analysed.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1); Weeks 1, 2, 4, 8, FU - 4, Posttreatment/Follow-up Week 12 (FU-12), and Posttreatment/Follow-up Week 24 (FU-24)
    End point values
    		 SOF/VEL/VOX FDC
    Number of subjects analysed
    21
    Units: percentile
    median (inter-quartile range (Q1-Q3))
        Baseline
    47.9 (32.4 to 63.2)
        Change at Week 8
    0.0 (-3.0 to 1.3)
        Change at FU-12
    0.0 (-2.2 to 3.8)
        Change at FU-24
    -0.2 (-3.1 to 4.3)
    No statistical analyses for this end point

    Secondary: Change From Baseline in Weight Percentiles as a Measurement of Growth and Development

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    End point title
    		 Change From Baseline in Weight Percentiles as a Measurement of Growth and Development
    End point description
    An age- and sex-specific percentile was derived for each weight, height, and body mass index (BMI) measurement according to the statistical analysis system (SAS) program available on the Centers for Disease Control and Prevention (CDC) website using the year 2000 growth charts. Participants in the Safety Analysis Set with available data were analysed.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1); Weeks 1, 2, 4, 8, FU-4, FU-12, and FU-24
    End point values
    		 SOF/VEL/VOX FDC
    Number of subjects analysed
    21
    Units: percentile
    median (inter-quartile range (Q1-Q3))
        Baseline
    54.0 (30.9 to 75.9)
        Change at Week 8
    -2.5 (-3.5 to -1.2)
        Change at FU-12
    -2.7 (-7.1 to 0.3)
        Change at FU-24
    -1.6 (-5.2 to 4.3)
    No statistical analyses for this end point

    Secondary: Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development

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    End point title
    		 Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
    End point description
    Tanner Pubertal Staging were assessed for pubic hair growth and genitalia development (males) and for pubic hair growth and breast development (females) in stages 1 to 5. It was used to evaluate the onset and progression of pubertal changes from stage 1 (pre-pubertal) to stage 5 (adult). If a participant had reached Tanner stage 5, no further assessments were done. Pubic hair growth: Tanner stages (1: No hair, 2: Downy hair, 3: Coarse and curly hair, 4: Adult-like hair quality; 5: Hair extends to medial surface of the thighs); Breast development: Tanner stages (1: No glandular tissue, 2: Breast bud forms,3: More elevated, outside areola, 4: Increased breast size,5: Final adult-size breasts); Genitalia development: Tanner stages (1: Testes, scrotum, and penis about same size, 2: Enlargement of scrotum and testes, Penis (10.5-12.5); 3: Enlargement of penis (11.5-14); 4: Penis size (13.5-15); 5: Genitalia adult in size and shape). Participants in the Safety Analysis Set were analysed.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1); Weeks 8, FU-12, and FU-24
    End point values
    		 SOF/VEL/VOX FDC
    Number of subjects analysed
    21
    Units: participants
    number (not applicable)
        Pubic Hair (Male); Baseline; Stage 1 (N=8)
    1
        Pubic Hair (Male); Baseline; Stage 2 (N=8)
    1
        Pubic Hair (Male); Baseline; Stage 3 (N=8)
    0
        Pubic Hair (Male); Baseline; Stage 4 (N=8)
    4
        Pubic Hair (Male); Baseline; Stage 5 (N=8)
    2
        Pubic Hair (Male); Week 8; Stage 1 (N=8)
    0
        Pubic Hair (Male); Week 8; Stage 2 (N=8)
    2
        Pubic Hair (Male); Week 8; Stage 3 (N=8)
    0
        Pubic Hair (Male); Week 8; Stage 4 (N=8)
    4
        Pubic Hair (Male); Week 8; Stage 5 (N=8)
    2
        Pubic Hair (Male); FU-12; Stage 1 (N=8)
    0
        Pubic Hair (Male); FU-12; Stage 2 (N=8)
    2
        Pubic Hair (Male); FU-12; Stage 3 (N=8)
    0
        Pubic Hair (Male); FU-12; Stage 4 (N=8)
    3
        Pubic Hair (Male); FU-12; Stage 5 (N=8)
    3
        Pubic Hair (Male); FU-24; Stage 1 (N=8)
    0
        Pubic Hair (Male); FU-24; Stage 2 (N=8)
    1
        Pubic Hair (Male); FU-24; Stage 3 (N=8)
    1
        Pubic Hair (Male); FU-24; Stage 4 (N=8)
    2
        Pubic Hair (Male); FU-24; Stage 5 (N=8)
    4
        Genitalia (Male); Baseline; Stage 1 (N=8)
    1
        Genitalia (Male); Baseline; Stage 2 (N=8)
    1
        Genitalia (Male); Baseline; Stage 3 (N=8)
    0
        Genitalia (Male); Baseline; Stage 4 (N=8)
    4
        Genitalia (Male); Baseline; Stage 5 (N=8)
    2
        Genitalia (Male); Week 8; Stage 1 (N=8)
    0
        Genitalia (Male); Week 8; Stage 2 (N=8)
    2
        Genitalia (Male); Week 8; Stage 3 (N=8)
    0
        Genitalia (Male); Week 8; Stage 4 (N=8)
    4
        Genitalia (Male); Week 8; Stage 5 (N=8)
    2
        Genitalia (Male); FU-12; Stage 1 (N=8)
    0
        Genitalia (Male); FU-12; Stage 2 (N=8)
    2
        Genitalia (Male); FU-12; Stage 3 (N=8)
    0
        Genitalia (Male); FU-12; Stage 4 (N=8)
    3
        Genitalia (Male); FU-12; Stage 5 (N=8)
    3
        Genitalia (Male); FU-24; Stage 1 (N=8)
    0
        Genitalia (Male); FU-24; Stage 2 (N=8)
    1
        Genitalia (Male); FU-24; Stage 3 (N=8)
    1
        Genitalia (Male); FU-24; Stage 4 (N=8)
    2
        Genitalia (Male); FU-24; Stage 5 (N=8)
    4
        Pubic Hair (Female); Baseline; Stage 1 (N=13)
    0
        Pubic Hair (Female); Baseline; Stage 2 (N=13)
    2
        Pubic Hair (Female); Baseline; Stage 3 (N=13)
    2
        Pubic Hair (Female); Baseline; Stage 4 (N=13)
    5
        Pubic Hair (Female); Baseline; Stage 5 (N=13)
    4
        Pubic Hair (Female); Week 8; Stage 1 (N=13)
    0
        Pubic Hair (Female); Week 8; Stage 2 (N=13)
    2
        Pubic Hair (Female); Week 8; Stage 3 (N=13)
    1
        Pubic Hair (Female); Week 8; Stage 4 (N=13)
    5
        Pubic Hair (Female); Week 8; Stage 5 (N=13)
    5
        Pubic Hair (Female); FU-12; Stage 1 (N=13)
    0
        Pubic Hair (Female); FU-12; Stage 2 (N=13)
    2
        Pubic Hair (Female); FU-12; Stage 3 (N=13)
    1
        Pubic Hair (Female); FU-12; Stage 4 (N=13)
    5
        Pubic Hair (Female); FU-12; Stage 5 (N=13)
    5
        Pubic Hair (Female); FU-24; Stage 1 (N=13)
    0
        Pubic Hair (Female); FU-24; Stage 2 (N=13)
    1
        Pubic Hair (Female); FU-24; Stage 3 (N=13)
    2
        Pubic Hair (Female); FU-24; Stage 4 (N=13)
    4
        Pubic Hair (Female); FU-24; Stage 5 (N=13)
    6
        Breasts (Female); Baseline; Stage 1 (N=13)
    0
        Breasts (Female); Baseline; Stage 2 (N=13)
    2
        Breasts (Female); Baseline; Stage 3 (N=13)
    2
        Breasts (Female); Baseline; Stage 4 (N=13)
    4
        Breasts (Female); Baseline; Stage 5 (N=13)
    5
        Breasts (Female); Week 8; Stage 1 (N=13)
    0
        Breasts (Female); Week 8; Stage 2 (N=13)
    2
        Breasts (Female); Week 8; Stage 3 (N=13)
    1
        Breasts (Female); Week 8; Stage 4 (N=13)
    5
        Breasts (Female); Week 8; Stage 5 (N=13)
    5
        Breasts (Female); FU-12; Stage 1 (N=13)
    0
        Breasts (Female); FU-12; Stage 2 (N=13)
    2
        Breasts (Female); FU-12; Stage 3 (N=13)
    1
        Breasts (Female); FU-12; Stage 4 (N=13)
    5
        Breasts (Female); FU-12; Stage 5 (N=13)
    5
        Breasts (Female); FU-24; Stage 1 (N=13)
    0
        Breasts (Female); FU-24; Stage 2 (N=13)
    1
        Breasts (Female); FU-24; Stage 3 (N=13)
    2
        Breasts (Female); FU-24; Stage 4 (N=13)
    4
        Breasts (Female); FU-24; Stage 5 (N=13)
    6
    No statistical analyses for this end point

    Secondary: Change from Baseline in Radiographic Bone Age Assessment as a Measurement of Growth and Development

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    End point title
    		 Change from Baseline in Radiographic Bone Age Assessment as a Measurement of Growth and Development
    End point description
    For radiographic bone age assessment, a single x-ray of the left wrist, hand, and fingers was performed and assessed by changes from baseline through end of treatment period. Participants in the Safety Analysis Set were analysed.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1); Week 8
    End point values
    		 SOF/VEL/VOX FDC
    Number of subjects analysed
    21
    Units: years
    median (inter-quartile range (Q1-Q3))
        Baseline
    14.5 (13.7 to 16.0)
        Change at Week 8
    0.0 (0.0 to 0.5)
    No statistical analyses for this end point

    Secondary: Change from Baseline in C-Type Collagen Sequence (CTX) Bone Turn-Over Biochemical Marker as a Measurement of Growth and Development

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    End point title
    		 Change from Baseline in C-Type Collagen Sequence (CTX) Bone Turn-Over Biochemical Marker as a Measurement of Growth and Development
    End point description
    Fasting blood samples for baseline values for bone age biomarkers CTX and change from baseline were recorded. Participants in the Safety Analysis Set were analysed.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1); FU-24
    End point values
    		 SOF/VEL/VOX FDC
    Number of subjects analysed
    21
    Units: ng/mL
    median (inter-quartile range (Q1-Q3))
        Baseline
    1.35 (1.05 to 2.35)
        Change at FU - 24
    -0.22 (-0.55 to -0.04)
    No statistical analyses for this end point

    Secondary: Change from Baseline in Procollagen Type 1 N-Terminal Propeptide (P1NP) Bone Turn-Over Biochemical Marker as a Measurement of Growth and Development

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    End point title
    		 Change from Baseline in Procollagen Type 1 N-Terminal Propeptide (P1NP) Bone Turn-Over Biochemical Marker as a Measurement of Growth and Development
    End point description
    Fasting blood samples for baseline values for bone age biomarkers P1NP and change from baseline were recorded. Participants in the Safety Analysis Set with available data were analysed.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1); FU-24
    End point values
    		 SOF/VEL/VOX FDC
    Number of subjects analysed
    20
    Units: ng/mL
    median (inter-quartile range (Q1-Q3))
        Baseline
    383.25 (175.00 to 944.20)
        Change at FU-24
    -101.50 (-359.00 to -24.98)
    No statistical analyses for this end point

    Secondary: Percentage of Participants in Each Swallowability Category of Able to Swallow or Unable to Swallow SOF/VEL/VOX 400/100/100 mg Size Tablets

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    End point title
    		 Percentage of Participants in Each Swallowability Category of Able to Swallow or Unable to Swallow SOF/VEL/VOX 400/100/100 mg Size Tablets
    End point description
    Swallowability for SOF/VEL/VOX FDC placebo to match (PTM) tablets was summarized based on the participants present in each swallowability category of Able to Swallow or Unable to Swallow a placebo tablet on one occasion during screening until Day 1. Participants in the Safety Analysis Set were analysed.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1)
    End point values
    		 SOF/VEL/VOX FDC
    Number of subjects analysed
    21
    Units: percentage of participants
    number (not applicable)
        Able to Swallow PTM Tablet
    100.0
        Unable to Swallow PTM Tablet
    0
    No statistical analyses for this end point

    Secondary: Percentage of Participants With Acceptability Questionnaire Responses as assessed by the Study Participant

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    End point title
    		 Percentage of Participants With Acceptability Questionnaire Responses as assessed by the Study Participant
    End point description
    A questionnaire was administered to participants to assess acceptability, including palatability, of the formulation. Acceptability and palatability were assessed by questions about how the study drug tasted, how easy it was to swallow the study drug, and also at the end of treatment about how it was to take the study drug and, as they all received a single tablet daily, how they felt about the number of tablets they had to swallow.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1); Week 8
    End point values
    		 SOF/VEL/VOX FDC
    Number of subjects analysed
    21
    Units: percentage of participants
    number (not applicable)
        Did Not Taste Study Drug; Day 1
    23.8
        Taste (Very Bad); Day 1
    9.5
        Taste (Bad); Day 1
    9.5
        Taste (Maybe Bad/ Maybe Good); Day 1
    38.1
        Taste (Good); Day 1
    19.0
        Taste (Very Good); Day 1
    0
        Swallow (Very Hard); Day 1
    9.5
        Swallow (Hard); Day 1
    9.5
        Swallow (Maybe Hard/ Maybe Easy); Day 1
    9.5
        Swallow (Easy); Day 1
    23.8
        Swallow (Very Easy); Day 1
    47.6
        Did Not Taste Study Drug; Week 8 (N=20))
    25.0
        Taste (Very Bad); Week 8 (N=20)
    0
        Taste (Bad); Week 8 (N=20)
    25.0
        Taste (Maybe Bad/ Maybe Good); Week 8 (N=20)
    40.0
        Taste (Good); Week 8 (N=20)
    5.0
        Taste (Very Good); Week 8 (N=20)
    5.0
        Swallow (Very Hard); Week 8 (N=20)
    0
        Swallow (Hard); Week 8 (N=20)
    5.0
        Swallow (Maybe Hard/ Maybe Easy); Week 8 (N=20)
    20.0
        Swallow (Easy); Week 8 (N=20)
    25.0
        Swallow (Very Easy); Week 8 (N=20)
    50.0
        Take (Very Hard); Week 8 (N=20)
    0
        Take (Hard); Week 8 (N=20)
    0
        Take (Maybe Hard/Maybe Easy); Week 8 (N=20)
    25.0
        Take (Easy); Week 8 (N=20)
    50.0
        Take (Very Easy); Week 8 (N=20)
    25.0
        Number of Tablets(Very Hard); Week 8 (N=20)
    0
        Number of Tablets(Hard); Week 8 (N=20)
    0
        NumberofTablets(Maybe Hard/Maybe Easy);Week8(N=20)
    45.0
        Number of Tablets(Easy); Week 8 (N=20)
    25.0
        Number of Tablets (Easy); Week 8 (N=20)
    30.0
    No statistical analyses for this end point

    Secondary: Percentage of Participants With Acceptability Questionnaire Responses as assessed by the Parent/Legal Guardian

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    End point title
    		 Percentage of Participants With Acceptability Questionnaire Responses as assessed by the Parent/Legal Guardian
    End point description
    A questionnaire was administered to the parent/legal guardian of participants to assess acceptability, including palatability, of the formulation. Acceptability and palatability were assessed by questions about how the study drug tasted, how easy it was to swallow the study drug, about how it was to take the study drug and, as they all received a single tablet daily, how they felt about the number of tablets they had to swallow.
    End point type
    Secondary
    End point timeframe
    Week 8
    End point values
    		 SOF/VEL/VOX FDC
    Number of subjects analysed
    21
    Units: percentage of participants
    number (not applicable)
        Did Not Taste Study Drug
    61.9
        Taste (Very Bad)
    0
        Taste (Bad)
    23.8
        Taste (Maybe Bad/ Maybe Good)
    9.5
        Taste (Good)
    4.8
        Taste (Very Good)
    0
        Swallow (Very Hard)
    4.8
        Swallow (Hard)
    9.5
        Swallow (Maybe Hard/ Maybe Easy)
    14.3
        Swallow (Easy)
    33.3
        Swallow (Very Easy)
    38.1
        Take (Very Hard)
    0
        Take (Hard)
    9.5
        Take (Maybe Hard/ Maybe Easy)
    19.0
        Take (Easy)
    47.6
        Take (Very Easy)
    23.8
        Number of Tablets (Very Hard)
    0
        Number of Tablets (Hard)
    4.8
        Number of Tablets (Maybe Hard/Maybe Easy)
    28.6
        Number of Tablets (Easy)
    47.6
        Number of Tablets (Very Easy)
    19.0
    No statistical analyses for this end point

    Secondary: Neuropsychiatric Assessments Based on Questionnaire as Completed by Participant

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    End point title
    		 Neuropsychiatric Assessments Based on Questionnaire as Completed by Participant
    End point description
    Neuropsychiatric safety assessment was done using the Pediatric Quality of Life (PedsQL™ 4.0 SF15). The PedsQL™ 4.0 SF15 Questionnaires was completed by all participants. It is a comprehensive and widely used patient-reported outcome survey designed to measure health-related quality of life in healthy children and adolescents. It is presented for each of the 4 domains of the SF-15 (physical functioning, emotional functioning, social functioning, and school functioning), the psychosocial health summary (emotional, social, and school functioning domains), physical health summary and the Total Score. Scores are transformed on a scale from 0 to 100. Higher scores indicate better health-related quality of life (HRQOL). A positive change from end of treatment period indicates improvement. Participants in the Full Analysis Set with available data were analysed.
    End point type
    Secondary
    End point timeframe
    Weeks 8, FU-12, and FU-24
    End point values
    		 SOF/VEL/VOX FDC
    Number of subjects analysed
    21
    Units: score on a scale
    arithmetic mean (standard deviation)
        Physical Functioning; Week 8
    88.1 ( 17.06 )
        Physical Functioning; Change at FU-12
    -0.7 ( 7.29 )
        Physical Functioning; Change at FU-24
    -0.9 ( 6.40 )
        Emotional Functioning; Week 8
    78.0 ( 19.33 )
        Emotional Functioning; Change at FU-12
    -3.0 ( 21.43 )
        Emotional Functioning; Change at FU-24
    -1.8 ( 18.56 )
        Social Functioning; Week 8 (N=20)
    95.0 ( 10.95 )
        Social Functioning; Change at FU-12 (N=20)
    -2.9 ( 8.67 )
        Social Functioning; Change at FU-24 (N=20)
    -0.4 ( 6.88 )
        School Functioning; Week 8
    80.2 ( 22.89 )
        School Functioning; Change at FU-12
    -4.8 ( 18.37 )
        School Functioning; Change at FU-24
    -3.6 ( 16.15 )
        Physical Health; Week 8
    88.1 ( 17.06 )
        Physical Health ; Change at FU-12
    -0.7 ( 7.29 )
        Physical Health; Change at FU-24
    -0.9 ( 6.40 )
        Psychosocial Health; Week 8
    83.6 ( 15.04 )
        Psychosocial Health; Change at FU-12
    -3.6 ( 12.38 )
        Psychosocial Health; Change at FU-24
    -2.4 ( 11.51 )
        Total Score; Week 8
    85.1 ( 14.13 )
        Total Score; Change at FU-12
    -2.6 ( 9.32 )
        Total Score; Change at FU-24
    -1.9 ( 8.83 )
    No statistical analyses for this end point

    Secondary: Neuropsychiatric Assessments Based on Questionnaire as Completed by Parent/Legal Guardian

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    End point title
    		 Neuropsychiatric Assessments Based on Questionnaire as Completed by Parent/Legal Guardian
    End point description
    Neuropsychiatric safety assessment was done using the Pediatric Quality of Life (PedsQL™ 4.0 SF15). The PedsQL™ 4.0 SF15 Questionnaires was completed by the parent/ legal guardian of the participants. It is a comprehensive and widely used patient-reported outcome survey designed to measure health-related quality of life in healthy children and adolescents. It is presented for each of the 4 domains of the SF-15 (physical functioning, emotional functioning, social functioning, and school functioning), the psychosocial health summary (emotional, social, and school functioning domains), physical health summary and the Total Score. Scores are transformed on a scale from 0 to 100. Higher scores indicate better HRQOL. A positive change from end of treatment period indicates improvement. Participants in the Full Analysis Set were analysed.
    End point type
    Secondary
    End point timeframe
    Weeks 8, FU-12, and FU-24
    End point values
    		 SOF/VEL/VOX FDC
    Number of subjects analysed
    21
    Units: score on a scale
    arithmetic mean (standard deviation)
        Physical Functioning; Week 8
    87.4 ( 17.29 )
        Physical Functioning; Change at FU-12 (N=20)
    -2.5 ( 9.39 )
        Physical Functioning; Change at FU-24
    -1.7 ( 12.97 )
        Emotional Functioning; Week 8
    78.6 ( 21.06 )
        Emotional Functioning; Change at FU-12 (N=20)
    -0.3 ( 22.53 )
        Emotional Functioning; Change at FU-24
    -2.4 ( 17.95 )
        Social Functioning; Week 8
    87.7 ( 17.80 )
        Social Functioning; Change at FU-12 (N=20)
    4.2 ( 18.04 )
        Social Functioning; Change at FU-24
    0.8 ( 14.41 )
        School Functioning; Week 8
    74.2 ( 27.63 )
        School Functioning; Change at FU-12 (N=20)
    3.3 ( 20.84 )
        School Functioning; Change at FU-24
    3.2 ( 16.77 )
        Physical Health; Week 8
    87.4 ( 17.29 )
        Physical Health ; Change at FU-12 (N=20)
    -2.5 ( 9.39 )
        Physical Health; Change at FU-24
    -1.7 ( 12.97 )
        Psychosocial Health; Week 8
    80.0 ( 17.46 )
        Psychosocial Health; Change at FU-12 (N=20)
    2.1 ( 15.82 )
        Psychosocial Health; Change at FU-24
    0.2 ( 9.61 )
        Total Score; Week 8
    82.5 ( 16.10 )
        Total Score; Change at FU-12 (N=20)
    0.6 ( 12.67 )
        Total Score; Change at FU-24
    -0.4 ( 7.87 )
    No statistical analyses for this end point

    Secondary: Change from Baseline in Neuropsychiatric Assessments as Completed by Participant

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    End point title
    		 Change from Baseline in Neuropsychiatric Assessments as Completed by Participant
    End point description
    Neuropsychiatric safety assessment was done using the Pediatric Quality of Life (PedsQL™ 4.0 SF15). The PedsQL™ 4.0 SF15 Questionnaires was completed by all the participants. It is a comprehensive and widely used patient-reported outcome survey designed to measure health-related quality of life in healthy children and adolescents. It was presented for each of the 4 domains of the SF-15 (physical functioning, emotional functioning, social functioning, and school functioning), the psychosocial health summary (emotional, social, and school functioning domains), physical health summary and the Total Score. Scores are transformed on a scale from 0 to 100. Higher scores indicate better HRQOL. A positive change from baseline indicates improvement. Participants in the Full Analysis Set with available data were analysed.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1); Weeks 8, FU-12, and FU-24
    End point values
    		 SOF/VEL/VOX FDC
    Number of subjects analysed
    21
    Units: Score on a scale
    arithmetic mean (standard deviation)
        Physical Functioning; Baseline
    90.7 ( 13.81 )
        Physical Functioning; Change at Week 8
    -2.6 ( 10.20 )
        Physical Functioning; Change at FU-12
    -3.3 ( 11.87 )
        Physical Functioning; Change at FU-24
    -3.5 ( 12.72 )
        Emotional Functioning; Baseline
    73.2 ( 20.07 )
        Emotional Functioning; Change at Week 8
    4.8 ( 10.63 )
        Emotional Functioning; Change at FU-12
    1.8 ( 19.78 )
        Emotional Functioning; Change at FU-24
    3.0 ( 15.64 )
        Social Functioning; Baseline
    93.3 ( 12.53 )
        Social Functioning; Change at Week 8 (N=20)
    0.4 ( 9.16 )
        Social Functioning; Change at FU-12
    -2.0 ( 10.17 )
        Social Functioning; Change at FU-24
    -0.8 ( 8.70 )
        School Functioning; Baseline
    75.0 ( 24.58 )
        School Functioning; Change at Week 8
    5.2 ( 17.97 )
        School Functioning; Change at FU-12
    0.4 ( 12.21 )
        School Functioning; Change at FU-24
    1.6 ( 11.06 )
        Physical Health; Baseline
    90.7 ( 13.81 )
        Physical Health; Change at Week 8
    -2.6 ( 10.20 )
        Physical Health; Change at FU-12
    -3.3 ( 11.87 )
        Physical Health, Change at FU-24
    -3.5 ( 12.72 )
        Psychosocial Health; Baseline
    79.8 ( 14.16 )
        Psychosocial Health; Change at Week 8
    3.8 ( 7.47 )
        Psychosocial Health; Change at FU-12
    0.2 ( 9.74 )
        Psychosocial Health; Change at FU-24
    1.4 ( 8.81 )
        Total Score; Baseline
    83.4 ( 12.40 )
        Total Score; Change at Week 8
    1.7 ( 5.30 )
        Total Score; Change at FU-12
    -1.0 ( 8.83 )
        Total Score; Change at FU-24
    -0.2 ( 8.58 )
    No statistical analyses for this end point

    Secondary: Change from Baseline in Neuropsychiatric Assessments as Completed by Parent/Legal Guardian

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    End point title
    		 Change from Baseline in Neuropsychiatric Assessments as Completed by Parent/Legal Guardian
    End point description
    Neuropsychiatric safety assessment was done using the Pediatric Quality of Life (PedsQL™ 4.0 SF15). The PedsQL™ 4.0 SF15 Questionnaires was completed by the parent/ legal guardian of the participants. It is a comprehensive and widely used patient-reported outcome survey designed to measure health-related quality of life in healthy children and adolescents. It is presented for each of the 4 domains of the SF-15 (physical functioning, emotional functioning, social functioning, and school functioning), the psychosocial health summary (emotional, social, and school functioning domains), physical health summary and the Total Score. Scores are transformed on a scale from 0 to 100. Higher scores indicate better HRQOL. A positive change from baseline indicates improvement. Participants in the Full Analysis Set with available data were analysed.
    End point type
    Secondary
    End point timeframe
    Baseline (Day 1); Weeks 8, FU-12, and FU-24
    End point values
    		 SOF/VEL/VOX FDC
    Number of subjects analysed
    20
    Units: score on a scale
    arithmetic mean (standard deviation)
        Physical Functioning; Baseline
    85.3 ( 22.56 )
        Physical Functioning; Change at Week 8
    3.5 ( 20.97 )
        Physical Functioning; Change at FU-12 (N=19)
    2.1 ( 20.90 )
        Physical Functioning; Change at FU-24
    4.0 ( 19.97 )
        Emotional Functioning; Baseline
    73.4 ( 18.57 )
        Emotional Functioning; Change at Week 8
    7.8 ( 18.57 )
        Emotional Functioning; Change at FU-12 (N=19)
    6.6 ( 14.50 )
        Emotional Functioning; Change at FU-24
    4.7 ( 12.80 )
        Social Functioning; Baseline
    85.8 ( 18.36 )
        Social Functioning; Change at Week 8
    1.3 ( 21.84 )
        Social Functioning; Change at FU-12 (N=19)
    6.1 ( 18.39 )
        Social Functioning; Change at FU-24
    2.1 ( 21.27 )
        School Functioning; Baseline
    72.5 ( 27.59 )
        School Functioning; Change at Week 8
    0.4 ( 23.64 )
        School Functioning; Change at FU-12 (N=19)
    3.9 ( 22.80 )
        School Functioning; Change at FU-24
    3.8 ( 20.50 )
        Physical Health; Baseline
    85.3 ( 22.56 )
        Physical Health; Change at Week 8
    3.5 ( 20.97 )
        Physical Health; Change at FU-12 (N=19)
    2.1 ( 20.90 )
        Physical Health, Change at FU-24
    4.0 ( 19.97 )
        Psychosocial Health; Baseline
    76.8 ( 17.34 )
        Psychosocial Health; Change at Week 8
    3.7 ( 17.39 )
        Psychosocial Health; Change at FU-12 (N=19)
    5.7 ( 15.31 )
        Psychosocial Health; Change at FU-24
    3.7 ( 15.52 )
        Total Score; Baseline
    79.7 ( 15.40 )
        Total Score; Change at Week 8
    3.6 ( 15.84 )
        Total Score; Change at FU-12 (N=19)
    4.5 ( 14.37 )
        Total Score; Change at FU-24
    3.8 ( 14.89 )
    No statistical analyses for this end point

    Adverse events

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    Adverse events information
    Timeframe for reporting adverse events
    First dose date up to the last dose date (maximum: 8 Weeks) plus 30 days.
    Adverse event reporting additional description
    The Safety Analysis Set included participants who received at least 1 dose of study drug.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    22.1
    Reporting groups
    Reporting group title
    SOF/VEL/VOX FDC
    Reporting group description
    		 Direct-acting antiviral (DAA) - naive participants without cirrhosis (12 to < 18 years old) received sofosbuvir/velpatasvir/voxilaprevir (SOF/VEL/VOX) fixed-dose (FDC) combination (400/100/100 mg tablet) orally once daily in nonfasting state for 8 weeks. A single placebo to match tablet was administered during screening until Day 1 to confirm the participant was able to swallow SOF/VEL/VOX 400/100/100 mg tablets.

    Serious adverse events
    		 SOF/VEL/VOX FDC
    Total subjects affected by serious adverse events
         subjects affected / exposed
    1 / 21 (4.76%)
         number of deaths (all causes)
    0
         number of deaths resulting from adverse events
    Vascular disorders
    Hypotension
         subjects affected / exposed
    1 / 21 (4.76%)
         occurrences causally related to treatment / all
    1 / 1
         deaths causally related to treatment / all
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 5%
    Non-serious adverse events
    		 SOF/VEL/VOX FDC
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    13 / 21 (61.90%)
    Nervous system disorders
    Headache
         subjects affected / exposed
    5 / 21 (23.81%)
         occurrences all number
    9
    Dizziness
         subjects affected / exposed
    2 / 21 (9.52%)
         occurrences all number
    2
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    3 / 21 (14.29%)
         occurrences all number
    5
    Asthenia
         subjects affected / exposed
    2 / 21 (9.52%)
         occurrences all number
    2
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    5 / 21 (23.81%)
         occurrences all number
    5
    Nausea
         subjects affected / exposed
    4 / 21 (19.05%)
         occurrences all number
    10
    Diarrhoea
         subjects affected / exposed
    3 / 21 (14.29%)
         occurrences all number
    13
    Respiratory, thoracic and mediastinal disorders
    Cough
         subjects affected / exposed
    2 / 21 (9.52%)
         occurrences all number
    2
    Infections and infestations
    Rhinitis
         subjects affected / exposed
    3 / 21 (14.29%)
         occurrences all number
    4

    More information

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    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    27 Mar 2019
    - The approximate amount of blood drawn at each visit was reduced to align with the pediatric EMA guidance “Guideline on the Investigation of Medicinal Products in the Term and Preterm Neonate” dated 25 June 2009. - Weight related inclusion criteria were added for the PK substudy to align with the pediatric EMA guidance “Guideline on the Investigation of Medicinal Products in the Term and Preterm Neonate” dated 25 June 2009. - Clarification was provided regarding the possible impact of the study drug on P-glycoprotein (P-gp), breast cancer resistance protein (BCRP), and organic anion-transporting polypeptide (OATP)1B1, OATP1B3, or OATP2B1 substrates, as described in the Investigator Brochure. - Details regarding dose calculations for Cohorts 2 and 3 (aged 3 to < 12 years old) were added. - Changes to the list of disallowed medications and concomitant medications to be used with caution were made to align with Vosevi EU summary of product characteristics.

    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
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