Trial Outcomes & Findings for Study to Investigate Pharmacokinetics, Safety and Efficacy of Sofosbuvir/Velpatasvir/Voxilaprevir (SOF/VEL/VOX) Fixed Dose Combination (FDC) in Adolescents and Children With Chronic Hepatitis C Virus (HCV) Infection (NCT NCT03820258)
NCT ID: NCT03820258
Last Updated: 2020-10-23
Results Overview
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.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
21 participants
Primary outcome 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)
Results posted on
2020-10-23
Participant Flow
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.
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. As per the protocol, participants could have received 8 or 12 weeks of treatment, depending on their prior treatment and disease status, however all participants received treatment for 8 weeks.
Participant milestones
| Measure |
SOF/VEL/VOX FDC
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.
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|---|---|
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Overall Study
STARTED
|
21
|
|
Overall Study
COMPLETED
|
21
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Study to Investigate Pharmacokinetics, Safety and Efficacy of Sofosbuvir/Velpatasvir/Voxilaprevir (SOF/VEL/VOX) Fixed Dose Combination (FDC) in Adolescents and Children With Chronic Hepatitis C Virus (HCV) Infection
Baseline characteristics by cohort
| Measure |
SOF/VEL/VOX FDC
n=21 Participants
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.
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|---|---|
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Age, Continuous
|
14 years
STANDARD_DEVIATION 1.2 • n=5 Participants
|
|
Sex: Female, Male
Female
|
13 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
8 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
19 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Race · White
|
16 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Race · Black or African American
|
1 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Race · Asian
|
2 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Race · Other
|
2 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Race · American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Race · Native Hawaiian or Pacific Islander
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
Italy
|
13 Participants
n=5 Participants
|
|
Region of Enrollment
Poland
|
4 Participants
n=5 Participants
|
|
Region of Enrollment
United Kingdom
|
4 Participants
n=5 Participants
|
|
Cirrhosis
Yes
|
0 Participants
n=5 Participants
|
|
Cirrhosis
No
|
21 Participants
n=5 Participants
|
|
Baseline HCV Ribonucleic acid (RNA)
|
5.9 log10 IU/mL
STANDARD_DEVIATION 0.70 • n=5 Participants
|
|
Baseline Alanine aminotransferase (ALT) Category
≤ 1.5 x (Upper Limit of Normal) ULN
|
16 Participants
n=5 Participants
|
|
Baseline Alanine aminotransferase (ALT) Category
> 1.5 x ULN
|
5 Participants
n=5 Participants
|
PRIMARY outcome
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)Population: The Pharmacokinetic Analysis Set included all enrolled adolescent participants who received at least 1 dose of study drug and for whom at least 1 nonmissing PK concentration value was available from all types of PK sampling.
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.
Outcome measures
| Measure |
SOF/VEL/VOX FDC
n=21 Participants
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.
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|---|---|
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Pharmacokinetic (PK) Parameter: AUCtau of SOF, GS-331007 (Metabolite of SOF), VEL, and VOX
SOF
|
2474.8 hours•nanogram/milliliter
Standard Deviation 1247.28
|
|
Pharmacokinetic (PK) Parameter: AUCtau of SOF, GS-331007 (Metabolite of SOF), VEL, and VOX
GS- 331007 (metabolite of SOF)
|
14890.2 hours•nanogram/milliliter
Standard Deviation 3126.35
|
|
Pharmacokinetic (PK) Parameter: AUCtau of SOF, GS-331007 (Metabolite of SOF), VEL, and VOX
VEL
|
6773.0 hours•nanogram/milliliter
Standard Deviation 2367.30
|
|
Pharmacokinetic (PK) Parameter: AUCtau of SOF, GS-331007 (Metabolite of SOF), VEL, and VOX
VOX
|
2205.8 hours•nanogram/milliliter
Standard Deviation 1403.45
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SECONDARY outcome
Timeframe: First dose date up to the last dose date (maximum: 8 Weeks) plus 30 daysPopulation: The Safety Analysis Set included participants who received at least 1 dose of study drug.
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.
Outcome measures
| Measure |
SOF/VEL/VOX FDC
n=21 Participants
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.
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|---|---|
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Percentage of Participants Who Permanently Discontinued Study Drug Due to an Adverse Event
|
0 percentage of participants
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SECONDARY outcome
Timeframe: Posttreatment Week 12Population: 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).
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.
Outcome measures
| Measure |
SOF/VEL/VOX FDC
n=21 Participants
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.
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|---|---|
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Percentage of Participants With Sustained Virologic Response (SVR) 12 Weeks After Discontinuation of Therapy (SVR12)
|
100.0 percentage of participants
Interval 83.9 to 100.0
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SECONDARY outcome
Timeframe: Posttreatment Week 4Population: Participants in the Full Analysis Set were analyzed.
SVR was defined as HCV RNA \< LLOQ (ie, \< 15 IU/mL) 4 weeks after discontinuation of the study drug.
Outcome measures
| Measure |
SOF/VEL/VOX FDC
n=21 Participants
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.
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|---|---|
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Percentage of Participants With HCV RNA < LLOQ 4 Weeks After Discontinuation of Therapy (SVR4)
|
100.0 percentage of participants
Interval 83.9 to 100.0
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SECONDARY outcome
Timeframe: Posttreatment Week 24Population: Participants in the Full Analysis Set were analyzed.
SVR was defined as HCV RNA \< LLOQ (ie, \< 15 IU/mL) 24 weeks after discontinuation of the study drug.
Outcome measures
| Measure |
SOF/VEL/VOX FDC
n=21 Participants
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.
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|---|---|
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Percentage of Participants With HCV RNA < LLOQ 24 Weeks After Discontinuation of Therapy (SVR24)
|
100.0 percentage of participants
Interval 83.9 to 100.0
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SECONDARY outcome
Timeframe: Up to Posttreatment Week 24Population: Participants in the Full Analysis Set were analyzed.
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).
Outcome measures
| Measure |
SOF/VEL/VOX FDC
n=21 Participants
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.
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|---|---|
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Percentage of Participants With Overall Virologic Failure
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0 percentage of participants
|
SECONDARY outcome
Timeframe: Weeks 1, 2, 4, and 8Population: Participants in the Full Analysis Set were analyzed.
Percentage of participants with HCV RNA \< LLOQ (15 IU/mL) while on treatment by analysis visit.
Outcome measures
| Measure |
SOF/VEL/VOX FDC
n=21 Participants
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.
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|---|---|
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Percentage of Participants With HCV RNA < LLOQ on Treatment
Week 1
|
52.4 percentage of participants
Interval 29.8 to 74.3
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|
Percentage of Participants With HCV RNA < LLOQ on Treatment
Week 2
|
81.0 percentage of participants
Interval 58.1 to 94.6
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|
Percentage of Participants With HCV RNA < LLOQ on Treatment
Week 4
|
90.5 percentage of participants
Interval 69.6 to 98.8
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|
Percentage of Participants With HCV RNA < LLOQ on Treatment
Week 8
|
100.0 percentage of participants
Interval 83.9 to 100.0
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SECONDARY outcome
Timeframe: Up to End of Treatment (Week 8)Population: 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.
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.
Outcome measures
| Measure |
SOF/VEL/VOX FDC
n=21 Participants
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.
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|---|---|
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Percentage of Participants Who Developed Viral Resistance to SOF, VEL, and/or VOX During Treatment
|
0 percentage of participants
|
SECONDARY outcome
Timeframe: Up to Posttreatment Week 24Population: 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.
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.
Outcome measures
| Measure |
SOF/VEL/VOX FDC
n=21 Participants
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.
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|---|---|
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Percentage of Participants Who Developed Viral Resistance to SOF, VEL, and/or VOX When Treatment is Discontinued
|
0 percentage of participants
|
SECONDARY outcome
Timeframe: Baseline (Day 1); Weeks 1, 2 ,4, and 8Population: Participants in the Full Analysis Set with available data were analyzed.
Outcome measures
| Measure |
SOF/VEL/VOX FDC
n=21 Participants
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.
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|---|---|
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Change in HCV RNA From Day 1 Through End of Treatment
Change at Week 1
|
-4.55 log10 IU/mL
Standard Deviation 0.525
|
|
Change in HCV RNA From Day 1 Through End of Treatment
Change at Week 2
|
-4.65 log10 IU/mL
Standard Deviation 0.621
|
|
Change in HCV RNA From Day 1 Through End of Treatment
Change at Week 4
|
-4.71 log10 IU/mL
Standard Deviation 0.661
|
|
Change in HCV RNA From Day 1 Through End of Treatment
Change at Week 8
|
-4.77 log10 IU/mL
Standard Deviation 0.698
|
SECONDARY outcome
Timeframe: Baseline (Day 1); Week 1, 2, 4, 8, and Posttreatment/Follow-up Week 4 (FU-4)Population: Participants in the Full Analysis Set with available data were analyzed. It includes participants with ALT \>ULN at Baseline.
ALT normalization was defined as ALT \> upper limit of normal (ULN) at baseline and ALT ≤ ULN at each visit.
Outcome measures
| Measure |
SOF/VEL/VOX FDC
n=5 Participants
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.
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|---|---|
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Percentage of Participants With Alanine Aminotransferase (ALT) Normalization
Week 1
|
50.0 percentage of participants
|
|
Percentage of Participants With Alanine Aminotransferase (ALT) Normalization
Week 2
|
75.0 percentage of participants
|
|
Percentage of Participants With Alanine Aminotransferase (ALT) Normalization
Week 4
|
100.0 percentage of participants
|
|
Percentage of Participants With Alanine Aminotransferase (ALT) Normalization
Week 8
|
100.0 percentage of participants
|
|
Percentage of Participants With Alanine Aminotransferase (ALT) Normalization
FU-4
|
100.0 percentage of participants
|
SECONDARY outcome
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)Population: Participants in the Safety Analysis Set with available data were analyzed.
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.
Outcome measures
| Measure |
SOF/VEL/VOX FDC
n=21 Participants
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.
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|---|---|
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Change From Baseline in Height Percentiles as a Measurement of Growth and Development
Change at FU-24
|
-0.2 percentile
Interval -3.1 to 4.3
|
|
Change From Baseline in Height Percentiles as a Measurement of Growth and Development
Baseline
|
47.9 percentile
Interval 32.4 to 63.2
|
|
Change From Baseline in Height Percentiles as a Measurement of Growth and Development
Change at Week 8
|
0.0 percentile
Interval -3.0 to 1.3
|
|
Change From Baseline in Height Percentiles as a Measurement of Growth and Development
Change at FU-12
|
0.0 percentile
Interval -2.2 to 3.8
|
SECONDARY outcome
Timeframe: Baseline (Day 1); Weeks 1, 2, 4, 8, FU-4, FU-12, and FU-24Population: Participants in the Safety Analysis Set with available data were analyzed.
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.
Outcome measures
| Measure |
SOF/VEL/VOX FDC
n=21 Participants
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.
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|---|---|
|
Change From Baseline in Weight Percentiles as a Measurement of Growth and Development
Baseline
|
54.0 percentile
Interval 30.9 to 75.9
|
|
Change From Baseline in Weight Percentiles as a Measurement of Growth and Development
Change at Week 8
|
-2.5 percentile
Interval -3.5 to -1.2
|
|
Change From Baseline in Weight Percentiles as a Measurement of Growth and Development
Change at FU-12
|
-2.7 percentile
Interval -7.1 to 0.3
|
|
Change From Baseline in Weight Percentiles as a Measurement of Growth and Development
Change at FU-24
|
-1.6 percentile
Interval -5.2 to 4.3
|
SECONDARY outcome
Timeframe: Baseline (Day 1); Weeks 8, FU-12, and FU-24Population: Participants in the Safety Analysis Set were analyzed.
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. Tanner stages will be 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 Tanner pubertal stage assessments were to be completed.Pubic hair growth: Tanner stages (1: No hair, 2: Downy hair, 3: More 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).
Outcome measures
| Measure |
SOF/VEL/VOX FDC
n=21 Participants
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.
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|---|---|
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Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Breasts (Female); Baseline · Stage 2
|
2 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Breasts (Female); FU-12 · Stage 2
|
2 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Pubic Hair (Male); Baseline · Stage 1
|
1 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Pubic Hair (Male); Baseline · Stage 2
|
1 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Pubic Hair (Male); Baseline · Stage 3
|
0 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Pubic Hair (Male); Baseline · Stage 4
|
4 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Pubic Hair (Male); Baseline · Stage 5
|
2 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Pubic Hair (Male); Week 8 · Stage 1
|
0 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Pubic Hair (Male); Week 8 · Stage 2
|
2 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Pubic Hair (Male); Week 8 · Stage 3
|
0 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Pubic Hair (Male); Week 8 · Stage 4
|
4 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Pubic Hair (Male); Week 8 · Stage 5
|
2 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Pubic Hair (Male); FU-12 · Stage 1
|
0 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Pubic Hair (Male); FU-12 · Stage 2
|
2 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Pubic Hair (Male); FU-12 · Stage 3
|
0 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Pubic Hair (Male); FU-12 · Stage 4
|
3 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Pubic Hair (Male); FU-12 · Stage 5
|
3 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Pubic Hair (Male); FU-24 · Stage 1
|
0 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Pubic Hair (Male); FU-24 · Stage 2
|
1 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Pubic Hair (Male); FU-24 · Stage 3
|
1 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Pubic Hair (Male); FU-24 · Stage 4
|
2 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Pubic Hair (Male); FU-24 · Stage 5
|
4 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Genitalia (Male); Baseline · Stage 1
|
1 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Genitalia (Male); Baseline · Stage 2
|
1 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Genitalia (Male); Baseline · Stage 3
|
0 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Genitalia (Male); Baseline · Stage 4
|
4 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Genitalia (Male); Baseline · Stage 5
|
2 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Genitalia (Male); Week 8 · Stage 1
|
0 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Genitalia (Male); Week 8 · Stage 2
|
2 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Genitalia (Male); Week 8 · Stage 3
|
0 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Genitalia (Male); Week 8 · Stage 4
|
4 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Genitalia (Male); Week 8 · Stage 5
|
2 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Genitalia (Male); FU-12 · Stage 1
|
0 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Genitalia (Male); FU-12 · Stage 2
|
2 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Genitalia (Male); FU-12 · Stage 3
|
0 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Genitalia (Male); FU-12 · Stage 4
|
3 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Genitalia (Male); FU-12 · Stage 5
|
3 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Genitalia (Male); FU-24 · Stage 1
|
0 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Genitalia (Male); FU-24 · Stage 2
|
1 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Genitalia (Male); FU-24 · Stage 3
|
1 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Genitalia (Male); FU-24 · Stage 4
|
2 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Genitalia (Male); FU-24 · Stage 5
|
4 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Pubic Hair (Female); Baseline · Stage 1
|
0 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Pubic Hair (Female); Baseline · Stage 2
|
2 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Pubic Hair (Female); Baseline · Stage 3
|
2 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Pubic Hair (Female); Baseline · Stage 4
|
5 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Pubic Hair (Female); Baseline · Stage 5
|
4 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Pubic Hair (Female); Week 8 · Stage 1
|
0 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Pubic Hair (Female); Week 8 · Stage 2
|
2 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Pubic Hair (Female); Week 8 · Stage 3
|
1 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Pubic Hair (Female); Week 8 · Stage 4
|
5 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Pubic Hair (Female); Week 8 · Stage 5
|
5 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Pubic Hair (Female); FU-12 · Stage 1
|
0 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Pubic Hair (Female); FU-12 · Stage 2
|
2 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Pubic Hair (Female); FU-12 · Stage 3
|
1 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Pubic Hair (Female); FU-12 · Stage 4
|
5 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Pubic Hair (Female); FU-12 · Stage 5
|
5 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Pubic Hair (Female); FU-24 · Stage 1
|
0 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Pubic Hair (Female); FU-24 · Stage 2
|
1 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Pubic Hair (Female); FU-24 · Stage 3
|
2 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Pubic Hair (Female); FU-24 · Stage 4
|
4 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Pubic Hair (Female); FU-24 · Stage 5
|
6 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Breasts (Female); Baseline · Stage 1
|
0 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Breasts (Female); Baseline · Stage 3
|
2 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Breasts (Female); Baseline · Stage 4
|
4 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Breasts (Female); Baseline · Stage 5
|
5 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Breasts (Female); Week 8 · Stage 1
|
0 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Breasts (Female); Week 8 · Stage 2
|
2 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Breasts (Female); Week 8 · Stage 3
|
1 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Breasts (Female); Week 8 · Stage 4
|
5 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Breasts (Female); Week 8 · Stage 5
|
5 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Breasts (Female); FU-12 · Stage 1
|
0 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Breasts (Female); FU-12 · Stage 3
|
1 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Breasts (Female); FU-12 · Stage 4
|
5 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Breasts (Female); FU-12 · Stage 5
|
5 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Breasts (Female); FU-24 · Stage 1
|
0 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Breasts (Female); FU-24 · Stage 2
|
1 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Breasts (Female); FU-24 · Stage 3
|
2 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Breasts (Female); FU-24 · Stage 4
|
4 Participants
|
|
Number of Participants by Tanner Stage Assessment as a Measurement of Growth and Development
Breasts (Female); FU-24 · Stage 5
|
6 Participants
|
SECONDARY outcome
Timeframe: Baseline (Day 1); Week 8Population: Participants in the Safety Analysis Set were analyzed.
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.
Outcome measures
| Measure |
SOF/VEL/VOX FDC
n=21 Participants
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.
|
|---|---|
|
Change From Baseline in Radiographic Bone Age Assessment as a Measurement of Growth and Development
Change at Week 8
|
0.0 years
Interval 0.0 to 0.5
|
|
Change From Baseline in Radiographic Bone Age Assessment as a Measurement of Growth and Development
Baseline
|
14.5 years
Interval 13.7 to 16.0
|
SECONDARY outcome
Timeframe: Baseline (Day 1); FU-24Population: Participants in the Safety Analysis Set were analyzed.
Fasting blood samples for baseline values for bone age biomarkers CTX and change from baseline were recorded.
Outcome measures
| Measure |
SOF/VEL/VOX FDC
n=21 Participants
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.
|
|---|---|
|
Change From Baseline in C-Type Collagen Sequence (CTX) Bone Turn-Over Biochemical Marker as a Measurement of Growth and Development
Baseline
|
1.35 ng/mL
Interval 1.05 to 2.35
|
|
Change From Baseline in C-Type Collagen Sequence (CTX) Bone Turn-Over Biochemical Marker as a Measurement of Growth and Development
Change at FU-24
|
-0.22 ng/mL
Interval -0.55 to -0.04
|
SECONDARY outcome
Timeframe: Baseline (Day 1); FU-24Population: Participants in the Safety Analysis Set with available data were analyzed.
Fasting blood samples for baseline values for bone age biomarkers P1NP and change from baseline were recorded.
Outcome measures
| Measure |
SOF/VEL/VOX FDC
n=20 Participants
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.
|
|---|---|
|
Change From Baseline in Procollagen Type 1 N-Terminal Propeptide (P1NP) Bone Turn-Over Biochemical Marker as a Measurement of Growth and Development
Baseline
|
383.25 ng/mL
Interval 175.0 to 944.2
|
|
Change From Baseline in Procollagen Type 1 N-Terminal Propeptide (P1NP) Bone Turn-Over Biochemical Marker as a Measurement of Growth and Development
Change at FU-24
|
-101.50 ng/mL
Interval -359.0 to -24.98
|
SECONDARY outcome
Timeframe: Baseline (Day 1)Population: Participants in the Safety Analysis Set were analyzed.
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.
Outcome measures
| Measure |
SOF/VEL/VOX FDC
n=21 Participants
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.
|
|---|---|
|
Percentage of Participants in Each Swallowability Category of Able to Swallow or Unable to Swallow SOF/VEL/VOX 400/100/100 mg Size Tablets
Able to Swallow PTM Tablet
|
100.0 percentage of participants
|
|
Percentage of Participants in Each Swallowability Category of Able to Swallow or Unable to Swallow SOF/VEL/VOX 400/100/100 mg Size Tablets
Unable to Swallow PTM Tablet
|
0 percentage of participants
|
SECONDARY outcome
Timeframe: Baseline (Day 1); Week 8Population: Participants in the Safety Analysis Set with available data were analyzed.
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.
Outcome measures
| Measure |
SOF/VEL/VOX FDC
n=21 Participants
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.
|
|---|---|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Study Participant
Number of Tablets(Very Easy); Week 8
|
30.0 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Study Participant
Number of Tablets( Maybe Hard/ Maybe Easy); Week 8
|
45.0 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Study Participant
Did Not Taste Study Drug; Day 1
|
23.8 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Study Participant
Taste (Very Bad); Day 1
|
9.5 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Study Participant
Taste (Bad); Day 1
|
9.5 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Study Participant
Taste (Maybe Bad/ Maybe Good); Day 1
|
38.1 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Study Participant
Taste (Good); Day 1
|
19.0 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Study Participant
Taste (Very Good); Day 1
|
0 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Study Participant
Swallow (Very Hard); Day 1
|
9.5 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Study Participant
Swallow (Hard); Day 1
|
9.5 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Study Participant
Swallow (Maybe Hard/ Maybe Easy); Day 1
|
9.5 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Study Participant
Swallow (Easy); Day 1
|
23.8 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Study Participant
Swallow (Very Easy); Day 1
|
47.6 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Study Participant
Did Not Taste Study Drug; Week 8
|
25.0 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Study Participant
Taste (Very Bad); Week 8
|
0 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Study Participant
Taste (Bad); Week 8
|
25.0 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Study Participant
Taste (Maybe Bad/ Maybe Good); Week 8
|
40.0 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Study Participant
Taste (Good); Week 8
|
5.0 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Study Participant
Taste (Very Good); Week 8
|
5.0 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Study Participant
Swallow (Very Hard); Week 8
|
0 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Study Participant
Swallow (Hard); Week 8
|
5.0 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Study Participant
Number of Tablets(Easy); Week 8
|
25.0 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Study Participant
Swallow (Maybe Hard/ Maybe Easy); Week 8
|
20.0 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Study Participant
Swallow (Easy); Week 8
|
25.0 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Study Participant
Swallow (Very Easy); Week 8
|
50.0 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Study Participant
Take (Very Hard); Week 8
|
0 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Study Participant
Take (Hard); Week 8
|
0 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Study Participant
Take (Maybe Hard/Maybe Easy); Week 8
|
25.0 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Study Participant
Take (Easy); Week 8
|
50.0 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Study Participant
Take (Very Easy); Week 8
|
25.0 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Study Participant
Number of Tablets(Very Hard); Week 8
|
0 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Study Participant
Number of Tablets(Hard); Week 8
|
0 percentage of participants
|
SECONDARY outcome
Timeframe: Week 8Population: Participants in the Safety Analysis Set were analyzed.
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.
Outcome measures
| Measure |
SOF/VEL/VOX FDC
n=21 Participants
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.
|
|---|---|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Parent/Legal Guardian
Did Not Taste Study Drug
|
61.9 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Parent/Legal Guardian
Number of Tablets (Very Hard)
|
0 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Parent/Legal Guardian
Take (Very Easy)
|
23.8 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Parent/Legal Guardian
Taste (Very Bad)
|
0 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Parent/Legal Guardian
Taste (Bad)
|
23.8 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Parent/Legal Guardian
Taste (Maybe Bad/ Maybe Good)
|
9.5 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Parent/Legal Guardian
Taste (Good)
|
4.8 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Parent/Legal Guardian
Taste (Very Good)
|
0 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Parent/Legal Guardian
Swallow (Very Hard)
|
4.8 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Parent/Legal Guardian
Swallow (Hard)
|
9.5 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Parent/Legal Guardian
Swallow (Maybe Hard/ Maybe Easy)
|
14.3 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Parent/Legal Guardian
Swallow (Easy)
|
33.3 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Parent/Legal Guardian
Swallow (Very Easy)
|
38.1 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Parent/Legal Guardian
Take (Very Hard)
|
0 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Parent/Legal Guardian
Take (Hard)
|
9.5 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Parent/Legal Guardian
Take (Maybe Hard/ Maybe Easy)
|
19.0 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Parent/Legal Guardian
Take (Easy)
|
47.6 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Parent/Legal Guardian
Number of Tablets (Hard)
|
4.8 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Parent/Legal Guardian
Number of Tablets (Maybe Hard/Maybe Easy)
|
28.6 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Parent/Legal Guardian
Number of Tablets (Easy)
|
47.6 percentage of participants
|
|
Percentage of Participants With Acceptability Questionnaire Responses as Assessed by the Parent/Legal Guardian
Number of Tablets (Very Easy)
|
19.0 percentage of participants
|
SECONDARY outcome
Timeframe: Weeks 8, FU-12, and FU-24Population: Participants in the Full Analysis Set with available data were analyzed.
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. Total scores as well as each of the subscale scores are transformed on a scale from 0 to 100. Higher scores in each case indicate better health-related quality of life (HRQOL). A positive change from end of treatment period indicates improvement.
Outcome measures
| Measure |
SOF/VEL/VOX FDC
n=21 Participants
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.
|
|---|---|
|
Neuropsychiatric Assessments Based on Questionnaire as Completed by Participant
Physical Functioning; Week 8
|
88.1 score on a scale
Standard Deviation 17.06
|
|
Neuropsychiatric Assessments Based on Questionnaire as Completed by Participant
Physical Functioning; Change at FU-12
|
-0.7 score on a scale
Standard Deviation 7.29
|
|
Neuropsychiatric Assessments Based on Questionnaire as Completed by Participant
Physical Functioning; Change at FU-24
|
-0.9 score on a scale
Standard Deviation 6.40
|
|
Neuropsychiatric Assessments Based on Questionnaire as Completed by Participant
Emotional Functioning; Week 8
|
78.0 score on a scale
Standard Deviation 19.33
|
|
Neuropsychiatric Assessments Based on Questionnaire as Completed by Participant
Emotional Functioning; Change at FU-12
|
-3.0 score on a scale
Standard Deviation 21.43
|
|
Neuropsychiatric Assessments Based on Questionnaire as Completed by Participant
Emotional Functioning; Change at FU-24
|
-1.8 score on a scale
Standard Deviation 18.56
|
|
Neuropsychiatric Assessments Based on Questionnaire as Completed by Participant
Social Functioning; Week 8
|
95.0 score on a scale
Standard Deviation 10.95
|
|
Neuropsychiatric Assessments Based on Questionnaire as Completed by Participant
Social Functioning; Change at FU-12
|
-2.9 score on a scale
Standard Deviation 8.67
|
|
Neuropsychiatric Assessments Based on Questionnaire as Completed by Participant
Social Functioning; Change at FU-24
|
-0.4 score on a scale
Standard Deviation 6.88
|
|
Neuropsychiatric Assessments Based on Questionnaire as Completed by Participant
School Functioning; Week 8
|
80.2 score on a scale
Standard Deviation 22.89
|
|
Neuropsychiatric Assessments Based on Questionnaire as Completed by Participant
School Functioning; Change at FU-12
|
-4.8 score on a scale
Standard Deviation 18.37
|
|
Neuropsychiatric Assessments Based on Questionnaire as Completed by Participant
School Functioning; Change at FU-24
|
-3.6 score on a scale
Standard Deviation 16.15
|
|
Neuropsychiatric Assessments Based on Questionnaire as Completed by Participant
Physical Health; Week 8
|
88.1 score on a scale
Standard Deviation 17.06
|
|
Neuropsychiatric Assessments Based on Questionnaire as Completed by Participant
Physical Health ; Change at FU-12
|
-0.7 score on a scale
Standard Deviation 7.29
|
|
Neuropsychiatric Assessments Based on Questionnaire as Completed by Participant
Physical Health; Change at FU-24
|
-0.9 score on a scale
Standard Deviation 6.40
|
|
Neuropsychiatric Assessments Based on Questionnaire as Completed by Participant
Psychosocial Health; Week 8
|
83.6 score on a scale
Standard Deviation 15.04
|
|
Neuropsychiatric Assessments Based on Questionnaire as Completed by Participant
Psychosocial Health; Change at FU-12
|
-3.6 score on a scale
Standard Deviation 12.38
|
|
Neuropsychiatric Assessments Based on Questionnaire as Completed by Participant
Psychosocial Health; Change at FU-24
|
-2.4 score on a scale
Standard Deviation 11.51
|
|
Neuropsychiatric Assessments Based on Questionnaire as Completed by Participant
Total Score; Week 8
|
85.1 score on a scale
Standard Deviation 14.13
|
|
Neuropsychiatric Assessments Based on Questionnaire as Completed by Participant
Total Score; Change at FU-12
|
-2.6 score on a scale
Standard Deviation 9.32
|
|
Neuropsychiatric Assessments Based on Questionnaire as Completed by Participant
Total Score; Change at FU-24
|
-1.9 score on a scale
Standard Deviation 8.83
|
SECONDARY outcome
Timeframe: Weeks 8, FU-12, and FU-24Population: Participants in the Full Analysis Set with available data were analyzed.
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. Total scores as well as each of the subscale scores are transformed on a scale from 0 to 100. Higher scores in each case indicate better HRQOL. A positive change from end of treatment period indicates improvement.
Outcome measures
| Measure |
SOF/VEL/VOX FDC
n=21 Participants
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.
|
|---|---|
|
Neuropsychiatric Assessments Based on Questionnaire as Completed by Parent/Legal Guardian
Total Score; Change at FU-24
|
-0.4 score on a scale
Standard Deviation 7.87
|
|
Neuropsychiatric Assessments Based on Questionnaire as Completed by Parent/Legal Guardian
Physical Functioning; Week 8
|
87.4 score on a scale
Standard Deviation 17.29
|
|
Neuropsychiatric Assessments Based on Questionnaire as Completed by Parent/Legal Guardian
Physical Functioning; Change at FU-12
|
-2.5 score on a scale
Standard Deviation 9.39
|
|
Neuropsychiatric Assessments Based on Questionnaire as Completed by Parent/Legal Guardian
Physical Functioning; Change at FU-24
|
-1.7 score on a scale
Standard Deviation 12.97
|
|
Neuropsychiatric Assessments Based on Questionnaire as Completed by Parent/Legal Guardian
Emotional Functioning; Week 8
|
78.6 score on a scale
Standard Deviation 21.06
|
|
Neuropsychiatric Assessments Based on Questionnaire as Completed by Parent/Legal Guardian
Emotional Functioning; Change at FU-12
|
-0.3 score on a scale
Standard Deviation 22.53
|
|
Neuropsychiatric Assessments Based on Questionnaire as Completed by Parent/Legal Guardian
Emotional Functioning; Change at FU-24
|
-2.4 score on a scale
Standard Deviation 17.95
|
|
Neuropsychiatric Assessments Based on Questionnaire as Completed by Parent/Legal Guardian
Social Functioning; Week 8
|
87.7 score on a scale
Standard Deviation 17.80
|
|
Neuropsychiatric Assessments Based on Questionnaire as Completed by Parent/Legal Guardian
Social Functioning; Change at FU-12
|
4.2 score on a scale
Standard Deviation 18.04
|
|
Neuropsychiatric Assessments Based on Questionnaire as Completed by Parent/Legal Guardian
Social Functioning; Change at FU-24
|
0.8 score on a scale
Standard Deviation 14.41
|
|
Neuropsychiatric Assessments Based on Questionnaire as Completed by Parent/Legal Guardian
School Functioning; Week 8
|
74.2 score on a scale
Standard Deviation 27.63
|
|
Neuropsychiatric Assessments Based on Questionnaire as Completed by Parent/Legal Guardian
School Functioning; Change at FU-12
|
3.3 score on a scale
Standard Deviation 20.84
|
|
Neuropsychiatric Assessments Based on Questionnaire as Completed by Parent/Legal Guardian
School Functioning; Change at FU-24
|
3.2 score on a scale
Standard Deviation 16.77
|
|
Neuropsychiatric Assessments Based on Questionnaire as Completed by Parent/Legal Guardian
Physical Health; Week 8
|
87.4 score on a scale
Standard Deviation 17.29
|
|
Neuropsychiatric Assessments Based on Questionnaire as Completed by Parent/Legal Guardian
Physical Health ; Change at FU-12
|
-2.5 score on a scale
Standard Deviation 9.39
|
|
Neuropsychiatric Assessments Based on Questionnaire as Completed by Parent/Legal Guardian
Physical Health; Change at FU-24
|
-1.7 score on a scale
Standard Deviation 12.97
|
|
Neuropsychiatric Assessments Based on Questionnaire as Completed by Parent/Legal Guardian
Psychosocial Health; Week 8
|
80.0 score on a scale
Standard Deviation 17.46
|
|
Neuropsychiatric Assessments Based on Questionnaire as Completed by Parent/Legal Guardian
Psychosocial Health; Change at FU-12
|
2.1 score on a scale
Standard Deviation 15.82
|
|
Neuropsychiatric Assessments Based on Questionnaire as Completed by Parent/Legal Guardian
Psychosocial Health; Change at FU-24
|
0.2 score on a scale
Standard Deviation 9.61
|
|
Neuropsychiatric Assessments Based on Questionnaire as Completed by Parent/Legal Guardian
Total Score; Week 8
|
82.5 score on a scale
Standard Deviation 16.10
|
|
Neuropsychiatric Assessments Based on Questionnaire as Completed by Parent/Legal Guardian
Total Score; Change at FU-12
|
0.6 score on a scale
Standard Deviation 12.67
|
SECONDARY outcome
Timeframe: Baseline (Day 1); Weeks 8, FU-12, and FU-24Population: Participants in the Full Analysis Set with available data were analyzed.
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. Total scores as well as each of the subscale scores are transformed on a scale from 0 to 100. Higher scores in each case indicate better HRQOL. A positive change from baseline indicates improvement.
Outcome measures
| Measure |
SOF/VEL/VOX FDC
n=21 Participants
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.
|
|---|---|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Participant
Physical Functioning; Baseline
|
90.7 score on a scale
Standard Deviation 13.81
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Participant
Physical Functioning; Change at Week 8
|
-2.6 score on a scale
Standard Deviation 10.20
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Participant
Physical Functioning; Change at FU-12
|
-3.3 score on a scale
Standard Deviation 11.87
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Participant
Physical Functioning; Change at FU-24
|
-3.5 score on a scale
Standard Deviation 12.72
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Participant
Emotional Functioning; Baseline
|
73.2 score on a scale
Standard Deviation 20.07
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Participant
Emotional Functioning; Change at Week 8
|
4.8 score on a scale
Standard Deviation 10.63
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Participant
Emotional Functioning; Change at FU-12
|
1.8 score on a scale
Standard Deviation 19.78
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Participant
Emotional Functioning; Change at FU-24
|
3.0 score on a scale
Standard Deviation 15.64
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Participant
Social Functioning; Baseline
|
93.3 score on a scale
Standard Deviation 12.53
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Participant
Social Functioning; Change at Week 8
|
0.4 score on a scale
Standard Deviation 9.16
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Participant
Social Functioning; Change at FU-12
|
-2.0 score on a scale
Standard Deviation 10.17
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Participant
Social Functioning; Change at FU-24
|
-0.8 score on a scale
Standard Deviation 8.70
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Participant
School Functioning; Baseline
|
75.0 score on a scale
Standard Deviation 24.58
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Participant
School Functioning; Change at Week 8
|
5.2 score on a scale
Standard Deviation 17.97
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Participant
School Functioning; Change at FU-12
|
0.4 score on a scale
Standard Deviation 12.21
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Participant
School Functioning; Change at FU-24
|
1.6 score on a scale
Standard Deviation 11.06
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Participant
Physical Health; Baseline
|
90.7 score on a scale
Standard Deviation 13.81
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Participant
Physical Health; Change at Week 8
|
-2.6 score on a scale
Standard Deviation 10.20
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Participant
Physical Health; Change at FU-12
|
-3.3 score on a scale
Standard Deviation 11.87
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Participant
Physical Health, Change at FU-24
|
-3.5 score on a scale
Standard Deviation 12.72
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Participant
Psychosocial Health; Baseline
|
79.8 score on a scale
Standard Deviation 14.16
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Participant
Psychosocial Health; Change at Week 8
|
3.8 score on a scale
Standard Deviation 7.47
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Participant
Psychosocial Health; Change at FU-12
|
0.2 score on a scale
Standard Deviation 9.74
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Participant
Psychosocial Health; Change at FU-24
|
1.4 score on a scale
Standard Deviation 8.81
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Participant
Total Score; Baseline
|
83.4 score on a scale
Standard Deviation 12.40
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Participant
Total Score; Change at Week 8
|
1.7 score on a scale
Standard Deviation 5.30
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Participant
Total Score; Change at FU-12
|
-1.0 score on a scale
Standard Deviation 8.83
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Participant
Total Score; Change at FU-24
|
-0.2 score on a scale
Standard Deviation 8.58
|
SECONDARY outcome
Timeframe: Baseline (Day 1); Weeks 8, FU-12, and FU-24Population: Participants in the Full Analysis Set with available data were analyzed.
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. Total scores as well as each of the subscale scores are transformed on a scale from 0 to 100. Higher scores in each case indicate better HRQOL. A positive change from baseline indicates improvement.
Outcome measures
| Measure |
SOF/VEL/VOX FDC
n=20 Participants
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.
|
|---|---|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Parent/Legal Guardian
Physical Functioning; Baseline
|
85.3 score on a scale
Standard Deviation 22.56
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Parent/Legal Guardian
Physical Functioning; Change at Week 8
|
3.5 score on a scale
Standard Deviation 20.97
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Parent/Legal Guardian
Physical Functioning; Change at FU-12
|
2.1 score on a scale
Standard Deviation 20.90
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Parent/Legal Guardian
Physical Functioning; Change at FU-24
|
4.0 score on a scale
Standard Deviation 19.97
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Parent/Legal Guardian
Emotional Functioning; Baseline
|
73.4 score on a scale
Standard Deviation 18.57
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Parent/Legal Guardian
Emotional Functioning; Change at Week 8
|
7.8 score on a scale
Standard Deviation 18.57
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Parent/Legal Guardian
Emotional Functioning; Change at FU-12
|
6.6 score on a scale
Standard Deviation 14.50
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Parent/Legal Guardian
Emotional Functioning; Change at FU-24
|
4.7 score on a scale
Standard Deviation 12.80
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Parent/Legal Guardian
Social Functioning; Baseline
|
85.8 score on a scale
Standard Deviation 18.36
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Parent/Legal Guardian
Social Functioning; Change at Week 8
|
1.3 score on a scale
Standard Deviation 21.84
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Parent/Legal Guardian
Social Functioning; Change at FU-12
|
6.1 score on a scale
Standard Deviation 18.39
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Parent/Legal Guardian
Social Functioning; Change at FU-24
|
2.1 score on a scale
Standard Deviation 21.27
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Parent/Legal Guardian
School Functioning; Baseline
|
72.5 score on a scale
Standard Deviation 27.59
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Parent/Legal Guardian
School Functioning; Change at Week 8
|
0.4 score on a scale
Standard Deviation 23.64
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Parent/Legal Guardian
School Functioning; Change at FU-12
|
3.9 score on a scale
Standard Deviation 22.80
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Parent/Legal Guardian
School Functioning; Change at FU-24
|
3.8 score on a scale
Standard Deviation 20.50
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Parent/Legal Guardian
Physical Health; Baseline
|
85.3 score on a scale
Standard Deviation 22.56
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Parent/Legal Guardian
Physical Health; Change at Week 8
|
3.5 score on a scale
Standard Deviation 20.97
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Parent/Legal Guardian
Physical Health; Change at FU-12
|
2.1 score on a scale
Standard Deviation 20.90
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Parent/Legal Guardian
Physical Health, Change at FU-24
|
4.0 score on a scale
Standard Deviation 19.97
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Parent/Legal Guardian
Psychosocial Health; Baseline
|
76.8 score on a scale
Standard Deviation 17.34
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Parent/Legal Guardian
Psychosocial Health; Change at Week 8
|
3.7 score on a scale
Standard Deviation 17.39
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Parent/Legal Guardian
Psychosocial Health; Change at FU-12
|
5.7 score on a scale
Standard Deviation 15.31
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Parent/Legal Guardian
Psychosocial Health; Change at FU-24
|
3.7 score on a scale
Standard Deviation 15.52
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Parent/Legal Guardian
Total Score; Baseline
|
79.7 score on a scale
Standard Deviation 15.40
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Parent/Legal Guardian
Total Score; Change at Week 8
|
3.6 score on a scale
Standard Deviation 15.84
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Parent/Legal Guardian
Total Score; Change at FU-12
|
4.5 score on a scale
Standard Deviation 14.37
|
|
Change From Baseline in Neuropsychiatric Assessments as Completed by Parent/Legal Guardian
Total Score; Change at FU-24
|
3.8 score on a scale
Standard Deviation 14.89
|
Adverse Events
SOF/VEL/VOX FDC
Serious events: 1 serious events
Other events: 13 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
SOF/VEL/VOX FDC
n=21 participants at risk
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.
|
|---|---|
|
Vascular disorders
Hypotension
|
4.8%
1/21 • First dose date up to the last dose date (maximum: 8 Weeks) plus 30 days
The Safety Analysis Set included participants received at least 1 dose of study drug.
|
Other adverse events
| Measure |
SOF/VEL/VOX FDC
n=21 participants at risk
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.
|
|---|---|
|
Gastrointestinal disorders
Abdominal pain
|
23.8%
5/21 • First dose date up to the last dose date (maximum: 8 Weeks) plus 30 days
The Safety Analysis Set included participants received at least 1 dose of study drug.
|
|
Gastrointestinal disorders
Diarrhoea
|
14.3%
3/21 • First dose date up to the last dose date (maximum: 8 Weeks) plus 30 days
The Safety Analysis Set included participants received at least 1 dose of study drug.
|
|
Gastrointestinal disorders
Nausea
|
19.0%
4/21 • First dose date up to the last dose date (maximum: 8 Weeks) plus 30 days
The Safety Analysis Set included participants received at least 1 dose of study drug.
|
|
General disorders
Asthenia
|
9.5%
2/21 • First dose date up to the last dose date (maximum: 8 Weeks) plus 30 days
The Safety Analysis Set included participants received at least 1 dose of study drug.
|
|
General disorders
Fatigue
|
14.3%
3/21 • First dose date up to the last dose date (maximum: 8 Weeks) plus 30 days
The Safety Analysis Set included participants received at least 1 dose of study drug.
|
|
Infections and infestations
Rhinitis
|
14.3%
3/21 • First dose date up to the last dose date (maximum: 8 Weeks) plus 30 days
The Safety Analysis Set included participants received at least 1 dose of study drug.
|
|
Nervous system disorders
Dizziness
|
9.5%
2/21 • First dose date up to the last dose date (maximum: 8 Weeks) plus 30 days
The Safety Analysis Set included participants received at least 1 dose of study drug.
|
|
Nervous system disorders
Headache
|
23.8%
5/21 • First dose date up to the last dose date (maximum: 8 Weeks) plus 30 days
The Safety Analysis Set included participants received at least 1 dose of study drug.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
9.5%
2/21 • First dose date up to the last dose date (maximum: 8 Weeks) plus 30 days
The Safety Analysis Set included participants received at least 1 dose of study drug.
|
Additional Information
Gilead Clinical Study Information Center
Gilead Sciences
Phone: 1-833-445-3230 (GILEAD-0)
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee After conclusion of the study and without prior written approval from Gilead, investigators in this study may communicate, orally present, or publish in scientific journals or other media only after the following conditions have been met: * The results of the study in their entirety have been publicly disclosed by or with the consent of Gilead in an abstract, manuscript, or presentation form; or * The study has been completed at all study sites for at least 2 years
- Publication restrictions are in place
Restriction type: OTHER