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Study to Investigate the Safety and Efficacy of Sofosbuvir/Velpatasvir in Adolescents and Children With Chronic HCV Infection

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ClinicalTrials.gov Identifier: NCT03022981
Recruitment Status : Completed
First Posted : January 18, 2017
Results First Posted : October 8, 2020
Last Update Posted : October 8, 2020
Sponsor:
Information provided by (Responsible Party):
Gilead Sciences

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Hepatitis C Virus Infection
Intervention Drug: SOF/VEL
Enrollment 216
Recruitment Details Participants were enrolled at 28 study sites in Belgium, Italy, the United Kingdom, and the United States. The first participant was screened on 26 January 2017. The last study visit occurred on 26 February 2020.
Pre-assignment Details 221 participants were screened.
Arm/Group Title 12 to < 18 Years Old 6 to < 12 Years Old 3 to < 6 Years Old
Hide Arm/Group Description

Pharmacokinetic (PK) Lead-in Phase: Sofosbuvir/Velpatasvir (SOF/VEL) fixed-dose combination (FDC) 400/100 mg tablets once daily for 7 days. Participants who completed the PK lead-in phase, continued into the treatment phase with no interruption of study drug administration and additional participants were enrolled into the treatment phase once the appropriateness of the dose was confirmed by PK results from the PK lead-in phase.

Treatment Phase: SOF/VEL 400/100 mg (adult and smaller size tablets based on swallowability assessment) once daily for 12 weeks.

PK Lead-in Phase: SOF/VEL FDC 200/50 mg tablets or oral granules once daily for 7 days. Participants who completed the PK lead-in phase, continued into the treatment phase with no interruption of study drug administration and additional participants were enrolled into the treatment phase once the appropriateness of the dose was confirmed by PK results from the PK lead-in phase.

Treatment Phase: SOF/VEL FDC 200/50 mg tablets or oral granules once daily for 12 weeks.

PK Lead-in Phase: SOF/VEL FDC 200/50 mg oral granules once daily for 7 days for participants who weighed ≥ 17 kg. SOF/VEL FDC 150/37.5 mg oral granules once daily for 7 days for participants who weighed < 17 kg. Participants who completed the PK lead-in phase, continued into the treatment phase with no interruption of study drug administration and additional participants were enrolled into the treatment phase once the appropriateness of the dose was confirmed by PK results from the PK lead-in phase.

Treatment Phase: SOF/VEL FDC 200/50 mg oral granules once daily for 12 weeks for participants who weighed ≥ 17 kg. SOF/VEL FDC 150/37.5 mg oral granules once daily for 12 weeks for participants who weighed < 17 kg.

Period Title: Overall Study
Started 102 73 41
Participated in PK Lead-in (PKL) Phase 17 [1] 20 [2] 19 [3]
Completed 96 69 35
Not Completed 6 4 6
Reason Not Completed
Adverse Event             0             1             0
Investigator's Discretion             0             1             2
Lost to Follow-up             6             2             1
Non-Compliance With Study Drug             0             0             2
Withdrew Assent By Parent/Guardian             0             0             1
[1]
All 17 participants completed treatment in the PK Lead-in phase and continued into treatment phase.
[2]
All 20 participants completed treatment in the PK Lead-in phase and continued into treatment phase.
[3]
All 19 participants completed treatment in the PK Lead-in phase and continued into treatment phase.
Arm/Group Title 12 to < 18 Years Old 6 to < 12 Years Old 3 to < 6 Years Old Total
Hide Arm/Group Description

PK Lead-in Phase: Sofosbuvir/Velpatasvir (SOF/VEL) fixed-dose combination (FDC) 400/100 mg tablets once daily for 7 days. Participants who completed the PK lead-in phase, continued into the treatment phase with no interruption of study drug administration and additional participants were enrolled into the treatment phase once the appropriateness of the dose was confirmed by PK results from the PK lead-in phase.

Treatment Phase: SOF/VEL 400/100 mg (adult and smaller size tablets based on swallowability assessment) once daily for 12 weeks.

PK Lead-in Phase: SOF/VEL FDC 200/50 mg tablets or oral granules once daily for 7 days. Participants who completed the PK lead-in phase, continued into the treatment phase with no interruption of study drug administration and additional participants were enrolled into the treatment phase once the appropriateness of the dose was confirmed by PK results from the PK lead-in phase.

Treatment Phase: SOF/VEL FDC 200/50 mg tablets or oral granules once daily for 12 weeks.

PK Lead-in Phase: SOF/VEL FDC 200/50 mg oral granules once daily for 7 days for participants who weighed ≥ 17 kg. SOF/VEL FDC 150/37.5 mg oral granules once daily for 7 days for participants who weighed < 17 kg. Participants who completed the PK lead-in phase, continued into the treatment phase with no interruption of study drug administration and additional participants were enrolled into the treatment phase once the appropriateness of the dose was confirmed by PK results from the PK lead-in phase.

Treatment Phase: SOF/VEL FDC 200/50 mg oral granules once daily for 12 weeks for participants who weighed ≥ 17 kg. SOF/VEL FDC 150/37.5 mg oral granules once daily for 12 weeks for participants who weighed < 17 kg.

Total of all reporting groups
Overall Number of Baseline Participants 102 73 41 216
Hide Baseline Analysis Population Description
The Safety Analysis Set included all participants who were enrolled into the study and received at least 1 dose of study drug (SOF/VEL FDC).
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 102 participants 73 participants 41 participants 216 participants
15  (1.9) 8  (1.6) 4  (0.8) 10  (4.5)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 102 participants 73 participants 41 participants 216 participants
Female 52 38 24 114
Male 50 35 17 102
Race/Ethnicity, Customized   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Race Number Analyzed 102 participants 73 participants 41 participants 216 participants
White 74 66 32 172
Black or African American 9 4 3 16
Asian 11 1 0 12
Other 5 2 5 12
American Indian or Alaska Native 2 0 0 2
Not Permitted 1 0 1 2
Native Hawaiian or Pacific Islander 0 0 0 0
[1]
Measure Description: Not Permitted = local requirements did not permit collection of race or ethnicity information.
Race/Ethnicity, Customized   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Ethnicity Number Analyzed 102 participants 73 participants 41 participants 216 participants
Hispanic or Latino 14 7 4 25
Not Hispanic or Latino 83 64 36 183
Not Permitted 5 2 1 8
[1]
Measure Description: Not Permitted = local requirements did not permit collection of race or ethnicity information.
Region of Enrollment  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 102 participants 73 participants 41 participants 216 participants
United States 77 57 38 172
Italy 17 5 2 24
United Kingdom 5 9 1 15
Belgium 3 2 0 5
1.Primary Outcome
Title PK Lead-in Phase: AUCtau: Area Under the Plasma Concentration Versus Time Curve Over the Dosing Interval of Velpatasvir (VEL)
Hide Description AUCtau is defined as concentration of drug over time (the area under the concentration versus time curve over the dosing interval).
Time Frame Day 7: 0 (predose), 0.5, 1, 2, 3, 4, 6 (Cohorts 1 and 2 only), 8, and 12 hours postdose
Hide Outcome Measure Data
Hide Analysis Population Description
The Lead-in Phase Pharmacokinetic (PK) Analysis Set included all PK lead-in phase participants with available data who received at least 1 dose of study drug and whom at least one non-missing PK concentration data value is available from the PK Lead-in Phase intensive sampling.
Arm/Group Title 12 to < 18 Years Old 6 to < 12 Years Old 3 to < 6 Years Old
Hide Arm/Group Description:
PK Lead-in Phase: Sofosbuvir/Velpatasvir (SOF/VEL) fixed-dose combination (FDC) 400/100 mg tablets once daily for 7 days.
PK Lead-in Phase: SOF/VEL FDC 200/50 mg tablets or oral granules once daily for 7 days.
PK Lead-in Phase: SOF/VEL FDC 200/50 mg oral granules once daily for 7 days for participants who weighed ≥ 17 kg. SOF/VEL FDC 150/37.5 mg oral granules once daily for 7 days for participants who weighed < 17 kg.
Overall Number of Participants Analyzed 16 17 18
Mean (Standard Deviation)
Unit of Measure: hours*nanograms per milliliter (h*ng/mL)
4479.3  (2105.66) 3697.5  (1653.25) 4450.3  (3285.75)
2.Primary Outcome
Title PK Lead-in Phase: AUCtau: Area Under the Plasma Concentration Versus Time Curve Over the Dosing Interval of Sofosbuvir (SOF)
Hide Description AUCtau is defined as concentration of drug over time (the area under the concentration versus time curve over the dosing interval).
Time Frame Day 7: 0 (predose), 0.5, 1, 2, 3, 4, 6 (Cohorts 1 and 2 only), 8, and 12 hours postdose
Hide Outcome Measure Data
Hide Analysis Population Description
Participants in the Lead-in Phase PK Analysis Set with available data were analyzed.
Arm/Group Title 12 to < 18 Years Old 6 to < 12 Years Old Cohort 3 (3 to < 6 Years Old)
Hide Arm/Group Description:
PK Lead-in Phase: SOF/VEL FDC 400/100 mg tablets once daily for 7 days.
PK Lead-in Phase: SOF/VEL FDC 200/50 mg tablets or oral granules once daily for 7 days.
PK Lead-in Phase: SOF/VEL FDC 200/50 mg oral granules once daily for 7 days for participants who weighed ≥ 17 kg. SOF/VEL FDC 150/37.5 mg oral granules once daily for 7 days for participants who weighed < 17 kg.
Overall Number of Participants Analyzed 16 17 17
Mean (Standard Deviation)
Unit of Measure: h*ng/mL
3020.1  (1162.56) 1764.5  (690.12) 3306.2  (3499.49)
3.Primary Outcome
Title PK Lead-in Phase: AUCtau: Area Under the Plasma Concentration Versus Time Curve Over the Dosing Interval of GS-331007 (Metabolite of SOF)
Hide Description AUCtau is defined as concentration of drug over time (the area under the concentration verses time curve over the dosing interval).
Time Frame Day 7: 0 (predose), 0.5, 1, 2, 3, 4, 6 (Cohorts 1 and 2 only), 8, and 12 hours postdose
Hide Outcome Measure Data
Hide Analysis Population Description
Participants in the Lead-in Phase PK Analysis Set with available data were analyzed.
Arm/Group Title 12 to < 18 Years Old 6 to < 12 Years Old 3 to < 6 Years Old
Hide Arm/Group Description:
PK Lead-in Phase: SOF/VEL FDC 400/100 mg tablets once daily for 7 days.
PK Lead-in Phase: SOF/VEL FDC 200/50 mg tablets or oral granules once daily for 7 days.
PK Lead-in Phase: SOF/VEL FDC 200/50 mg oral granules once daily for 7 days for participants who weighed ≥ 17 kg. SOF/VEL FDC 150/37.5 mg oral granules once daily for 7 days for participants who weighed < 17 kg.
Overall Number of Participants Analyzed 16 17 18
Mean (Standard Deviation)
Unit of Measure: h*ng/mL
13852.9  (3565.85) 9913.8  (3071.79) 11604.0  (2732.57)
4.Primary Outcome
Title Treatment Phase: Percentage of Participants Who Discontinued Study Drug Due to Any Treatment-Emergent Adverse Event (TEAE)
Hide Description An AE is any untoward medical occurrence in a clinical study participant administered a medicinal product, which does not necessarily have a causal relationship with the treatment. An AE can therefore be any unfavorable and/or unintended sign, symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product. TEAEs were defined as 1 or both of the following: Any AEs with an onset date on or after the study drug start date and no later than 30 days after permanent discontinuation of study drug and/or Any AEs leading to premature discontinuation of study drug.
Time Frame From first dose through last dose of the study drug (Up to 12 weeks) plus 30 days
Hide Outcome Measure Data
Hide Analysis Population Description
The Safety Analysis Set included all participants who were enrolled into the study and received at least 1 dose of study drug (SOF/VEL FDC).
Arm/Group Title 12 to < 18 Years Old 6 to < 12 Years Old 3 to < 6 Years Old
Hide Arm/Group Description:
Treatment Phase: SOF/VEL 400/100 mg (adult and smaller size tablets based on swallowability assessment) once daily for 12 weeks.
Treatment Phase: SOF/VEL FDC 200/50 mg tablets or oral granules once daily for 12 weeks.
Treatment Phase: SOF/VEL FDC 200/50 mg oral granules once daily for 12 weeks for participants who weighed ≥ 17 kg. SOF/VEL FDC 150/37.5 mg oral granules once daily for 12 weeks for participants who weighed < 17 kg.
Overall Number of Participants Analyzed 102 73 41
Measure Type: Number
Unit of Measure: percentage of participants
0.0 2.7 2.4
5.Secondary Outcome
Title PK Lead-in Phase: Change From Baseline in Hepatitis C Virus (HCV) Ribonucleic Acid (RNA) at Day 7
Hide Description [Not Specified]
Time Frame Baseline; Day 7
Hide Outcome Measure Data
Hide Analysis Population Description
Participants in the Lead-in Phase PK Analysis Set with available data were analyzed.
Arm/Group Title 12 to < 18 Years Old 6 to < 12 Years Old 3 to < 6 Years Old
Hide Arm/Group Description:
PK Lead-in Phase: SOF/VEL FDC 400/100 mg tablets once daily for 7 days.
PK Lead-in Phase: SOF/VEL FDC 200/50 mg tablets or oral granules once daily for 7 days.
PK Lead-in Phase: SOF/VEL FDC 200/50 mg oral granules once daily for 7 days for participants who weighed ≥ 17 kg. SOF/VEL FDC 150/37.5 mg oral granules once daily for 7 days for participants who weighed < 17 kg.
Overall Number of Participants Analyzed 17 20 19
Mean (Standard Deviation)
Unit of Measure: log10 international units per mL (IU/mL)
Baseline 6.09  (0.569) 5.84  (0.656) 5.77  (1.275)
Change from Baseline at Day 7 -4.48  (0.656) -4.20  (0.642) -3.94  (1.082)
6.Secondary Outcome
Title PK Lead-in Phase: Percentage of Participants Who Permanently Discontinued Study Drug Due to an Adverse Event (AE)
Hide Description An AE is any untoward medical occurrence in a clinical study participant administered a medicinal product, which does not necessarily have a causal relationship with the treatment. An AE can therefore be any unfavorable and/or unintended sign, symptom, or disease temporally associated with the use of a medicinal product, whether or not considered related to the medicinal product.
Time Frame First dose date up to Day 7
Hide Outcome Measure Data
Hide Analysis Population Description
Participants in the Lead-in Phase PK Analysis Set were analyzed.
Arm/Group Title 12 to < 18 Years Old 6 to < 12 Years Old 3 to < 6 Years Old
Hide Arm/Group Description:
PK Lead-in Phase: SOF/VEL FDC 400/100 mg tablets once daily for 7 days.
PK Lead-in Phase: SOF/VEL FDC 200/50 mg tablets or oral granules once daily for 7 days.
PK Lead-in Phase: SOF/VEL FDC 200/50 mg oral granules once daily for 7 days for participants who weighed ≥ 17 kg. SOF/VEL FDC 150/37.5 mg oral granules once daily for 7 days for participants who weighed < 17 kg.
Overall Number of Participants Analyzed 17 20 19
Measure Type: Number
Unit of Measure: percentage of participants
0.0 0.0 0.0
7.Secondary Outcome
Title Treatment Phase: Percentage of Participants With Sustained Virologic Response (SVR) at 12 Weeks After Discontinuation of Therapy (SVR12)
Hide Description SVR12 was defined as HCV RNA < the lower limit of quantitation (LLOQ; ie, 15 IU/mL) at 12 weeks after stopping study treatment.
Time Frame Posttreatment Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
The Full Analysis Set included all participants who were enrolled into the study and received at least 1 dose of study drug (SOF/VEL FDC).
Arm/Group Title 12 to < 18 Years Old 6 to < 12 Years Old 3 to < 6 Years Old
Hide Arm/Group Description:
Treatment Phase: SOF/VEL 400/100 mg (adult and smaller size tablets based on swallowability assessment) once daily for 12 weeks.
Treatment Phase: SOF/VEL FDC 200/50 mg tablets or oral granules once daily for 12 weeks.
Treatment Phase: SOF/VEL FDC 200/50 mg oral granules once daily for 12 weeks for participants who weighed ≥ 17 kg. SOF/VEL FDC 150/37.5 mg oral granules once daily for 12 weeks for participants who weighed < 17 kg.
Overall Number of Participants Analyzed 102 73 41
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
95.1
(88.9 to 98.4)
93.2
(84.7 to 97.7)
82.9
(67.9 to 92.8)
8.Secondary Outcome
Title Treatment Phase: Percentage of Participants With SVR at 4 Weeks After Discontinuation of Therapy (SVR4)
Hide Description SVR4 was defined as HCV RNA < LLOQ (ie, 15 IU/mL) at 4 weeks after stopping study treatment.
Time Frame Posttreatment Week 4
Hide Outcome Measure Data
Hide Analysis Population Description
Participants in the Full Analysis Set were analyzed.
Arm/Group Title 12 to < 18 Years Old 6 to < 12 Years Old 3 to < 6 Years Old
Hide Arm/Group Description:
Treatment Phase: SOF/VEL 400/100 mg (adult and smaller size tablets based on swallowability assessment) once daily for 12 weeks.
Treatment Phase: SOF/VEL FDC 200/50 mg tablets or oral granules once daily for 12 weeks.
Treatment Phase: SOF/VEL FDC 200/50 mg oral granules once daily for 12 weeks for participants who weighed ≥ 17 kg. SOF/VEL FDC 150/37.5 mg oral granules once daily for 12 weeks for participants who weighed < 17 kg.
Overall Number of Participants Analyzed 102 73 41
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
96.1
(90.3 to 98.9)
94.5
(86.6 to 98.5)
82.9
(67.9 to 92.8)
9.Secondary Outcome
Title Treatment Phase: Percentage of Participants With SVR at 24 Weeks After Discontinuation of Therapy (SVR24)
Hide Description SVR 24 was defined as HCV RNA < LLOQ (ie, 15 IU/mL) at 24 weeks after stopping study treatment.
Time Frame Posttreatment Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
Participants in the Full Analysis Set were analyzed.
Arm/Group Title 12 to < 18 Years Old 6 to < 12 Years Old 3 to < 6 Years Old
Hide Arm/Group Description:
Treatment Phase: SOF/VEL 400/100 mg (adult and smaller size tablets based on swallowability assessment) once daily for 12 weeks.
Treatment Phase: SOF/VEL FDC 200/50 mg tablets or oral granules once daily for 12 weeks.
Treatment Phase: SOF/VEL FDC 200/50 mg oral granules once daily for 12 weeks for participants who weighed ≥ 17 kg. SOF/VEL FDC 150/37.5 mg oral granules once daily for 12 weeks for participants who weighed < 17 kg.
Overall Number of Participants Analyzed 102 73 41
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
95.1
(88.9 to 98.4)
93.2
(84.7 to 97.7)
82.9
(67.9 to 92.8)
10.Secondary Outcome
Title Treatment Phase: Percentage of Participants With Virologic Failure
Hide Description Virologic failure was defined as: On-treatment virologic failure - Breakthrough (confirmed HCV RNA ≥ LLOQ after having previously had HCV RNA < LLOQ while on treatment), or Rebound (confirmed > 1 log10 IU/mL increase in HCV RNA from nadir while on treatment), or Non-response (HCV RNA persistently ≥ LLOQ through 8 weeks of treatment); Virologic relapse: Confirmed HCV RNA ≥ LLOQ during the posttreatment period having achieved HCV RNA < LLOQ at last on-treatment visit.
Time Frame Up to Posttreatment Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
Participants in the Full Analysis Set were analyzed.
Arm/Group Title 12 to < 18 Years Old 6 to < 12 Years Old 3 to < 6 Years Old
Hide Arm/Group Description:
Treatment Phase: SOF/VEL 400/100 mg (adult and smaller size tablets based on swallowability assessment) once daily for 12 weeks.
Treatment Phase: SOF/VEL FDC 200/50 mg tablets or oral granules once daily for 12 weeks.
Treatment Phase: SOF/VEL FDC 200/50 mg oral granules once daily for 12 weeks for participants who weighed ≥ 17 kg. SOF/VEL FDC 150/37.5 mg oral granules once daily for 12 weeks for participants who weighed < 17 kg.
Overall Number of Participants Analyzed 102 73 41
Measure Type: Number
Unit of Measure: percentage of participants
1.0 1.4 0.0
11.Secondary Outcome
Title Treatment Phase: Percentage of Participants With HCV RNA < LLOQ On Treatment
Hide Description Percentage of participants with HCV RNA < LLOQ while on treatment by analysis visit.
Time Frame Weeks 1, 4, 8, and 12
Hide Outcome Measure Data
Hide Analysis Population Description
Participants in the Full Analysis Set were analyzed.
Arm/Group Title 12 to < 18 Years Old 6 to < 12 Years Old 3 to < 6 Years Old
Hide Arm/Group Description:
Treatment Phase: SOF/VEL 400/100 mg (adult and smaller size tablets based on swallowability assessment) once daily for 12 weeks.
Treatment Phase: SOF/VEL FDC 200/50 mg tablets or oral granules once daily for 12 weeks.
Treatment Phase: SOF/VEL FDC 200/50 mg oral granules once daily for 12 weeks for participants who weighed ≥ 17 kg. SOF/VEL FDC 150/37.5 mg oral granules once daily for 12 weeks for participants who weighed < 17 kg.
Overall Number of Participants Analyzed 102 73 41
Measure Type: Number
Number (95% Confidence Interval)
Unit of Measure: percentage of participants
Week 1
44.1
(34.3 to 54.3)
39.7
(28.5 to 51.9)
36.8
(21.8 to 54.0)
Week 4
96.1
(90.3 to 98.9)
94.4
(86.2 to 98.4)
91.4
(76.9 to 98.2)
Week 8
100.0
(96.4 to 100.0)
98.6
(92.3 to 100.0)
100.0
(89.7 to 100.0)
Week 12
100.0
(96.4 to 100.0)
98.6
(92.3 to 100.0)
100.0
(89.7 to 100.0)
12.Secondary Outcome
Title Treatment Phase: Percentage of Participants Who Develop Viral Resistance to SOF and/or VEL During Treatment and After Discontinuation of Treatment
Hide Description Drug-resistant substitutions were analyzed as part of the Virology Study. 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 deep sequencing of the HCV nonstructural protein (NS)5A and NS5B genes was performed for all participants at the first time point after virologic failure if the plasma or serum sample was available. Pretreatment full-length NS5A deep sequencing data were obtained at a 15% assay cutoff for the Resistance Analysis Population which covered all NS5A and NS5B nucleoside inhibitor (NI) resistance-associated variants (RAVs).
Time Frame First dose date up to Posttreatment Week 24
Hide Outcome Measure Data
Hide Analysis Population Description
The Resistance Analysis Population with all adolescent or pediatric participants in the Safety Analysis Set with a virologic outcome with available data were analyzed.
Arm/Group Title 12 to < 18 Years Old 6 to < 12 Years Old 3 to < 6 Years Old
Hide Arm/Group Description:
Treatment Phase: SOF/VEL 400/100 mg (adult and smaller size tablets based on swallowability assessment) once daily for 12 weeks.
Treatment Phase: SOF/VEL FDC 200/50 mg tablets or oral granules once daily for 12 weeks.
Treatment Phase: SOF/VEL FDC 200/50 mg oral granules once daily for 12 weeks for participants who weighed ≥ 17 kg. SOF/VEL FDC 150/37.5 mg oral granules once daily for 12 weeks for participants who weighed < 17 kg.
Overall Number of Participants Analyzed 98 68 33
Measure Type: Number
Unit of Measure: percentage of participants
Pretreatment NS5A NI RAVs Number Analyzed 98 participants 68 participants 33 participants
16.3 10.2 18.1
Pretreatment NS5B NI RAVs Number Analyzed 66 participants 68 participants 33 participants
5.1 0.0 3.0
13.Secondary Outcome
Title Treatment Phase: Change From Baseline in HCV RNA at Weeks 1, 4, 8, and 12
Hide Description [Not Specified]
Time Frame Baseline; Weeks 1, 4, 8, and 12
Hide Outcome Measure Data
Hide Analysis Population Description
Participants in the Full Analysis Set with available data were analyzed.
Arm/Group Title 12 to < 18 Years Old 6 to < 12 Years Old 3 to < 6 Years Old
Hide Arm/Group Description:
Treatment Phase: SOF/VEL 400/100 mg (adult and smaller size tablets based on swallowability assessment) once daily for 12 weeks.
Treatment Phase: SOF/VEL FDC 200/50 mg tablets or oral granules once daily for 12 weeks.
Treatment Phase: SOF/VEL FDC 200/50 mg oral granules once daily for 12 weeks for participants who weighed ≥ 17 kg. SOF/VEL FDC 150/37.5 mg oral granules once daily for 12 weeks for participants who weighed < 17 kg.
Overall Number of Participants Analyzed 102 73 41
Mean (Standard Deviation)
Unit of Measure: log10 IU/mL
Baseline Number Analyzed 102 participants 73 participants 41 participants
6.06  (0.585) 5.87  (0.686) 5.86  (1.057)
Change from Baseline at Week 1 Number Analyzed 102 participants 71 participants 36 participants
-4.46  (0.661) -4.28  (0.632) -4.06  (0.914)
Change from Baseline at Week 4 Number Analyzed 102 participants 71 participants 34 participants
-4.89  (0.578) -4.64  (0.860) -4.49  (1.040)
Change from Baseline at Week 8 Number Analyzed 101 participants 70 participants 34 participants
-4.91  (0.588) -4.69  (0.678) -4.56  (1.066)
Change from Baseline at Week 12 Number Analyzed 101 participants 69 participants 34 participants
-4.91  (0.588) -4.70  (0.683) -4.56  (1.066)
14.Secondary Outcome
Title Treatment Phase: Quality of Life (QoL) and Neuropsychiatric Assessments as Measured by PedsQL™ Pediatric QoL Survey
Hide Description To evaluate the effect of treatment with SOF/VEL on general and disease-specific health-related QoL, the PedsQL™ Pediatric QoL Inventory V4.0 Short Form (SF15) was completed at Day 1, end of treatment, early termination (if applicable), and posttreatment Weeks 12 and 24. The SF15 questionnaire represented 4 domains: physical, emotional, social, and school functioning, with the emotional, social, and school functioning domains representing the psychosocial health summary. Neuropsychiatric assessment was conducted using the PedsQL™ Pediatric QoL Inventory V4.0 SF15 psychosocial domain-related scores. Items were calculated and transformed into an overall score with a range of 0 to 100 points, with more points indicating better QoL.
Time Frame Baseline; Week 12, End of Treatment (EOT), Posttreatment/Follow-up (FU) Week-12 (FU-12), and FU Week-24 (FU-24)
Hide Outcome Measure Data
Hide Analysis Population Description
Participants in the Full Analysis Set with available data were analyzed.
Arm/Group Title 12 to < 18 Years Old 6 to < 12 Years Old 3 to < 6 Years Old
Hide Arm/Group Description:
Treatment Phase: SOF/VEL 400/100 mg (adult and smaller size tablets based on swallowability assessment) once daily for 12 weeks.
Treatment Phase: SOF/VEL FDC 200/50 mg tablets or oral granules once daily for 12 weeks.
Treatment Phase: SOF/VEL FDC 200/50 mg oral granules once daily for 12 weeks for participants who weighed ≥ 17 kg. SOF/VEL FDC 150/37.5 mg oral granules once daily for 12 weeks for participants who weighed < 17 kg.
Overall Number of Participants Analyzed 102 73 41
Mean (Standard Deviation)
Unit of Measure: score on a scale
Parents Reports, Total Score at Baseline Number Analyzed 98 participants 73 participants 41 participants
80.0  (17.71) 79.7  (15.58) 86.5  (12.43)
Parents Reports, Total Score at Week 12 Number Analyzed 97 participants 68 participants 32 participants
82.4  (18.13) 82.8  (14.26) 87.3  (11.31)
Parents Reports, Total Score at EOT Number Analyzed 97 participants 70 participants 34 participants
82.4  (18.13) 82.2  (14.95) 87.0  (11.65)
Parents Reports, Total Score at Follow Up-12 Number Analyzed 94 participants 68 participants 35 participants
81.7  (17.39) 81.5  (15.34) 87.7  (14.11)
Parents Reports, Total Score at Follow Up-24 Number Analyzed 94 participants 69 participants 35 participants
80.8  (18.65) 79.7  (15.30) 88.3  (9.79)
Participants Reports, Total Score at Baseline Number Analyzed 100 participants 73 participants 18 participants
79.9  (15.22) 77.9  (13.33) 82.2  (12.47)
Participants Reports, Total Score at Week 12 Number Analyzed 101 participants 68 participants 21 participants
80.9  (16.38) 80.0  (14.21) 83.3  (11.62)
Participants Reports, Total Score at EOT Number Analyzed 101 participants 70 participants 21 participants
80.9  (16.38) 79.7  (14.13) 83.3  (11.62)
Participants Reports, Total Score at Follow Up-12 Number Analyzed 98 participants 68 participants 23 participants
82.5  (15.41) 81.1  (13.32) 80.5  (14.18)
Participants Reports, Total Score at Follow Up-24 Number Analyzed 98 participants 69 participants 25 participants
81.4  (16.34) 81.7  (15.43) 82.1  (12.56)
15.Secondary Outcome
Title Treatment Phase: Change From Baseline in Growth and Development as Measured by Height Percentiles
Hide 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.
Time Frame Baseline; Weeks 1, 4, 8, 12, Follow-up (FU) Week 4 (FU-4), FU-12, and FU-24
Hide Outcome Measure Data
Hide Analysis Population Description
Participants in the Safety Analysis Set with available data were analyzed.
Arm/Group Title 12 to < 18 Years Old 6 to < 12 Years Old 3 to < 6 Years Old
Hide Arm/Group Description:
Treatment Phase: SOF/VEL 400/100 mg (adult and smaller size tablets based on swallowability assessment) once daily for 12 weeks.
Treatment Phase: SOF/VEL FDC 200/50 mg tablets or oral granules once daily for 12 weeks.
Treatment Phase: SOF/VEL FDC 200/50 mg oral granules once daily for 12 weeks for participants who weighed ≥ 17 kg. SOF/VEL FDC 150/37.5 mg oral granules once daily for 12 weeks for participants who weighed < 17 kg.
Overall Number of Participants Analyzed 102 73 41
Median (Inter-Quartile Range)
Unit of Measure: percentile
Baseline Number Analyzed 102 participants 73 participants 41 participants
44.5
(16.2 to 74.1)
41.9
(20.5 to 65.2)
39.3
(13.9 to 70.1)
Change From Baseline at Week 1 Number Analyzed 102 participants 73 participants 36 participants
0.0
(-0.4 to 0.9)
0.1
(-0.7 to 2.4)
0.5
(-1.1 to 3.4)
Change From Baseline at Week 4 Number Analyzed 101 participants 71 participants 34 participants
0.0
(-0.7 to 1.7)
0.2
(-1.9 to 1.9)
1.4
(-0.3 to 3.7)
Change From Baseline at Week 8 Number Analyzed 101 participants 69 participants 34 participants
0.0
(-1.2 to 1.5)
-0.1
(-2.2 to 2.2)
0.3
(-2.1 to 3.6)
Change From Baseline at Week 12 Number Analyzed 100 participants 65 participants 28 participants
-0.1
(-1.4 to 1.0)
0.2
(-2.6 to 2.8)
0.2
(-2.0 to 5.1)
Change From Baseline at FU-4 Number Analyzed 99 participants 70 participants 34 participants
0.0
(-2.0 to 1.3)
0.2
(-2.3 to 2.5)
1.2
(-0.7 to 5.6)
Change From Baseline at FU-12 Number Analyzed 98 participants 68 participants 35 participants
-0.2
(-2.4 to 1.4)
0.1
(-1.7 to 2.6)
0.6
(-3.1 to 4.6)
Change From Baseline at FU-24 Number Analyzed 91 participants 67 participants 33 participants
-0.6
(-2.6 to 1.9)
-0.2
(-3.1 to 3.5)
0.3
(-2.1 to 6.0)
16.Secondary Outcome
Title Treatment Phase: Change From Baseline in Growth and Development as Measured by Weight Percentiles
Hide Description An age- and sex-specific percentile was derived for each weight, height, and BMI measurement according to the SAS program available on the CDC website using the year 2000 growth charts.
Time Frame Baseline; Weeks 1, 4, 8, 12, FU-4, FU-12, and FU-24
Hide Outcome Measure Data
Hide Analysis Population Description
Participants in the Safety Analysis Set with available data were analyzed.
Arm/Group Title 12 to < 18 Years Old 6 to < 12 Years Old 3 to < 6 Years Old
Hide Arm/Group Description:
Treatment Phase: SOF/VEL 400/100 mg (adult and smaller size tablets based on swallowability assessment) once daily for 12 weeks.
Treatment Phase: SOF/VEL FDC 200/50 mg tablets or oral granules once daily for 12 weeks.
Treatment Phase: SOF/VEL FDC 200/50 mg oral granules once daily for 12 weeks for participants who weighed ≥ 17 kg. SOF/VEL FDC 150/37.5 mg oral granules once daily for 12 weeks for participants who weighed < 17 kg.
Overall Number of Participants Analyzed 102 73 41
Median (Inter-Quartile Range)
Unit of Measure: percentile
Baseline Number Analyzed 102 participants 73 participants 41 participants
67.2
(31.5 to 84.1)
45.9
(23.3 to 78.6)
64.6
(38.0 to 86.7)
Change From Baseline at Week 1 Number Analyzed 102 participants 73 participants 36 participants
0.0
(-0.4 to 0.9)
0.1
(-0.6 to 1.2)
-0.5
(-2.7 to 0.7)
Change From Baseline at Week 4 Number Analyzed 102 participants 71 participants 34 participants
0.0
(-0.9 to 1.8)
0.2
(-1.4 to 1.5)
0.3
(-1.2 to 2.0)
Change From Baseline at Week 8 Number Analyzed 101 participants 69 participants 34 participants
0.0
(-1.3 to 2.1)
0.1
(-1.6 to 2.5)
0.2
(-3.7 to 2.5)
Change From Baseline at Week 12 Number Analyzed 100 participants 65 participants 28 participants
0.2
(-1.0 to 2.9)
0.5
(-1.7 to 2.5)
-1.7
(-4.7 to 0.6)
Change From Baseline at FU-4 Number Analyzed 99 participants 70 participants 34 participants
0.1
(-3.0 to 3.1)
0.0
(-2.3 to 2.6)
-0.8
(-4.9 to 2.4)
Change From Baseline at FU-12 Number Analyzed 98 participants 68 participants 35 participants
0.0
(-2.5 to 4.7)
0.5
(-2.2 to 4.9)
-0.8
(-6.6 to 2.2)
Change From Baseline at FU-24 Number Analyzed 91 participants 67 participants 33 participants
0.1
(-2.6 to 4.1)
1.4
(-1.2 to 5.7)
-1.3
(-3.5 to 2.9)
17.Secondary Outcome
Title Treatment Phase: Changes in Growth and Development as Measured by Tanner Stage Assessment From Baseline
Hide Description Tanner Pubertal Staging was 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 were 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 the 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, testes and penis, 3: Enlargement of penis, 4: Penis size enlargement, 5: Genitalia adult in size and shape).
Time Frame Baseline; EOT, FU-12 and FU-24
Hide Outcome Measure Data
Hide Analysis Population Description
Participants in the Safety Analysis Set with available data were analyzed. Overall Number of Participants Analyzed for each arm is the total of male plus female participants.
Arm/Group Title 12 to < 18 Years Old 6 to < 12 Years Old 3 to < 6 Years Old
Hide Arm/Group Description:
Treatment Phase: SOF/VEL 400/100 mg (adult and smaller size tablets based on swallowability assessment) once daily for 12 weeks.
Treatment Phase: SOF/VEL FDC 200/50 mg tablets or oral granules once daily for 12 weeks.
Treatment Phase: SOF/VEL FDC 200/50 mg oral granules once daily for 12 weeks for participants who weighed ≥ 17 kg. SOF/VEL FDC 150/37.5 mg oral granules once daily for 12 weeks for participants who weighed < 17 kg.
Overall Number of Participants Analyzed 102 73 41
Measure Type: Count of Participants
Unit of Measure: Participants
Pubic Hair (Male): Baseline, Stage 1 Number Analyzed 50 participants 34 participants 17 participants
4 31 17
Pubic Hair (Male): Baseline, Stage 2 Number Analyzed 50 participants 34 participants 17 participants
3 2 0
Pubic Hair (Male): Baseline, Stage 3 Number Analyzed 50 participants 34 participants 17 participants
10 1 0
Pubic Hair (Male): Baseline, Stage 4 Number Analyzed 50 participants 34 participants 17 participants
14 0 0
Pubic Hair (Male): Baseline, Stage 5 Number Analyzed 50 participants 34 participants 17 participants
19 0 0
Pubic Hair (Male): EOT, Stage 1 Number Analyzed 50 participants 32 participants 13 participants
2 28 13
Pubic Hair (Male): EOT, Stage 2 Number Analyzed 50 participants 32 participants 13 participants
4 3 0
Pubic Hair (Male): EOT, Stage 3 Number Analyzed 50 participants 32 participants 13 participants
7 1 0
Pubic Hair (Male): EOT, Stage 4 Number Analyzed 50 participants 32 participants 13 participants
13 0 0
Pubic Hair (Male): EOT, Stage 5 Number Analyzed 50 participants 32 participants 13 participants
24 0 0
Pubic Hair (Male): FU-12, Stage 1 Number Analyzed 47 participants 30 participants 14 participants
1 26 14
Pubic Hair (Male): FU-12, Stage 2 Number Analyzed 47 participants 30 participants 14 participants
5 3 0
Pubic Hair (Male): FU-12, Stage 3 Number Analyzed 47 participants 30 participants 14 participants
6 1 0
Pubic Hair (Male): FU-12, Stage 4 Number Analyzed 47 participants 30 participants 14 participants
10 0 0
Pubic Hair (Male): FU-12, Stage 5 Number Analyzed 47 participants 30 participants 14 participants
25 0 0
Pubic Hair (Male): FU-24, Stage 1 Number Analyzed 45 participants 32 participants 14 participants
1 29 14
Pubic Hair (Male): FU-24, Stage 2 Number Analyzed 45 participants 32 participants 14 participants
4 2 0
Pubic Hair (Male): FU-24, Stage 3 Number Analyzed 45 participants 32 participants 14 participants
2 1 0
Pubic Hair (Male): FU-24, Stage 4 Number Analyzed 45 participants 32 participants 14 participants
13 0 0
Pubic Hair (Male): FU-24, Stage 5 Number Analyzed 45 participants 32 participants 14 participants
25 0 0
Genitalia (Male): Baseline, Stage 1 Number Analyzed 50 participants 34 participants 17 participants
4 31 17
Genitalia (Male): Baseline, Stage 2 Number Analyzed 50 participants 34 participants 17 participants
3 2 0
Genitalia (Male): Baseline, Stage 3 Number Analyzed 50 participants 34 participants 17 participants
8 1 0
Genitalia (Male): Baseline, Stage 4 Number Analyzed 50 participants 34 participants 17 participants
16 0 0
Genitalia (Male): Baseline, Stage 5 Number Analyzed 50 participants 34 participants 17 participants
19 0 0
Genitalia (Male): EOT, Stage 1 Number Analyzed 50 participants 32 participants 13 participants
2 28 13
Genitalia (Male): EOT, Stage 2 Number Analyzed 50 participants 32 participants 13 participants
3 3 0
Genitalia (Male): EOT, Stage 3 Number Analyzed 50 participants 32 participants 13 participants
7 1 0
Genitalia (Male): EOT, Stage 4 Number Analyzed 50 participants 32 participants 13 participants
15 0 0
Genitalia (Male): EOT, Stage 5 Number Analyzed 50 participants 32 participants 13 participants
23 0 0
Genitalia (Male): FU-12, Stage 1 Number Analyzed 47 participants 30 participants 14 participants
1 26 14
Genitalia (Male): FU-12, Stage 2 Number Analyzed 47 participants 30 participants 14 participants
4 3 0
Genitalia (Male): FU-12, Stage 3 Number Analyzed 47 participants 30 participants 14 participants
7 1 0
Genitalia (Male): FU-12, Stage 4 Number Analyzed 47 participants 30 participants 14 participants
11 0 0
Genitalia (Male): FU-12, Stage 5 Number Analyzed 47 participants 30 participants 14 participants
24 0 0
Genitalia (Male): FU-24, Stage 1 Number Analyzed 45 participants 32 participants 14 participants
1 27 14
Genitalia (Male): FU-24, Stage 2 Number Analyzed 45 participants 32 participants 14 participants
2 4 0
Genitalia (Male): FU-24, Stage 3 Number Analyzed 45 participants 32 participants 14 participants
4 1 0
Genitalia (Male): FU-24, Stage 4 Number Analyzed 45 participants 32 participants 14 participants
13 0 0
Genitalia (Male): FU-24, Stage 5 Number Analyzed 45 participants 32 participants 14 participants
25 0 0
Pubic Hair (Female): Baseline, Stage 1 Number Analyzed 52 participants 38 participants 24 participants
2 31 24
Pubic Hair (Female): Baseline, Stage 2 Number Analyzed 52 participants 38 participants 24 participants
3 5 0
Pubic Hair (Female): Baseline, Stage 3 Number Analyzed 52 participants 38 participants 24 participants
9 2 0
Pubic Hair (Female): Baseline, Stage 4 Number Analyzed 52 participants 38 participants 24 participants
16 0 0
Pubic Hair (Female): Baseline, Stage 5 Number Analyzed 52 participants 38 participants 24 participants
22 0 0
Pubic Hair (Female): EOT, Stage 1 Number Analyzed 50 participants 37 participants 21 participants
2 29 21
Pubic Hair (Female): EOT, Stage 2 Number Analyzed 50 participants 37 participants 21 participants
2 5 0
Pubic Hair (Female): EOT, Stage 3 Number Analyzed 50 participants 37 participants 21 participants
6 2 0
Pubic Hair (Female): EOT, Stage 4 Number Analyzed 50 participants 37 participants 21 participants
10 1 0
Pubic Hair (Female): EOT, Stage 5 Number Analyzed 50 participants 37 participants 21 participants
30 0 0
Pubic Hair (Female): FU-12, Stage 1 Number Analyzed 51 participants 35 participants 21 participants
2 24 21
Pubic Hair (Female): FU-12, Stage 2 Number Analyzed 51 participants 35 participants 21 participants
1 7 0
Pubic Hair (Female): FU-12, Stage 3 Number Analyzed 51 participants 35 participants 21 participants
8 3 0
Pubic Hair (Female): FU-12, Stage 4 Number Analyzed 51 participants 35 participants 21 participants
8 1 0
Pubic Hair (Female): FU-12, Stage 5 Number Analyzed 51 participants 35 participants 21 participants
32 0 0
Pubic Hair (Female): FU-24, Stage 1 Number Analyzed 50 participants 35 participants 21 participants
2 24 20
Pubic Hair (Female): FU-24, Stage 2 Number Analyzed 50 participants 35 participants 21 participants
0 6 1
Pubic Hair (Female): FU-24, Stage 3 Number Analyzed 50 participants 35 participants 21 participants
8 1 0
Pubic Hair (Female): FU-24, Stage 4 Number Analyzed 50 participants 35 participants 21 participants
9 4 0
Pubic Hair (Female): FU-24, Stage 5 Number Analyzed 50 participants 35 participants 21 participants
31 0 0
Breasts (Female): Baseline, Stage 1 Number Analyzed 52 participants 38 participants 24 participants
1 29 24
Breasts (Female): Baseline, Stage 2 Number Analyzed 52 participants 38 participants 24 participants
5 6 0
Breasts (Female): Baseline, Stage 3 Number Analyzed 52 participants 38 participants 24 participants
6 3 0
Breasts (Female): Baseline, Stage 4 Number Analyzed 52 participants 38 participants 24 participants
17 0 0
Breasts (Female): Baseline, Stage 5 Number Analyzed 52 participants 38 participants 24 participants
23 0 0
Breasts (Female): EOT, Stage 1 Number Analyzed 50 participants 37 participants 21 participants
1 25 21
Breasts (Female): EOT, Stage 2 Number Analyzed 50 participants 37 participants 21 participants
2 9 0
Breasts (Female): EOT, Stage 3 Number Analyzed 50 participants 37 participants 21 participants
6 2 0
Breasts (Female): EOT, Stage 4 Number Analyzed 50 participants 37 participants 21 participants
14 1 0
Breasts (Female): EOT, Stage 5 Number Analyzed 50 participants 37 participants 21 participants
27 0 0
Breasts (Female): FU-12, Stage 1 Number Analyzed 51 participants 35 participants 21 participants
1 20 21
Breasts (Female): FU-12, Stage 2 Number Analyzed 51 participants 35 participants 21 participants
2 11 0
Breasts (Female): FU-12, Stage 3 Number Analyzed 51 participants 35 participants 21 participants
5 2 0
Breasts (Female): FU-12, Stage 4 Number Analyzed 51 participants 35 participants 21 participants
15 2 0
Breasts (Female): FU-12, Stage 5 Number Analyzed 51 participants 35 participants 21 participants
28 0 0
Breasts (Female): FU-24, Stage 1 Number Analyzed 50 participants 35 participants 21 participants
1 20 20
Breasts (Female): FU-24, Stage 2 Number Analyzed 50 participants 35 participants 21 participants
0 10 1
Breasts (Female): FU-24, Stage 3 Number Analyzed 50 participants 35 participants 21 participants
6 3 0
Breasts (Female): FU-24, Stage 4 Number Analyzed 50 participants 35 participants 21 participants
13 2 0
Breasts (Female): FU-24, Stage 5 Number Analyzed 50 participants 35 participants 21 participants
30 0 0
18.Secondary Outcome
Title Treatment Phase: Growth and Development as Measured by Parental Height
Hide Description Mid-parental height was calculated as the average of the biological father's and mother's heights. For boys, the sex-adjusted mid-parental height was calculated by adding 2.5 inches or 6.5 cm to the mean of the parents' heights. For girls, 2.5 inches or 6.5 cm was subtracted from the mean of the parents' heights.
Time Frame Day 1
Hide Outcome Measure Data
Hide Analysis Population Description
Participants in the Safety Analysis Set with available data were analyzed.
Arm/Group Title 12 to < 18 Years Old 6 to < 12 Years Old 3 to < 6 Years Old
Hide Arm/Group Description:
Treatment Phase: SOF/VEL 400/100 mg (adult and smaller size tablets based on swallowability assessment) once daily for 12 weeks.
Treatment Phase: SOF/VEL FDC 200/50 mg tablets or oral granules once daily for 12 weeks.
Treatment Phase: SOF/VEL FDC 200/50 mg oral granules once daily for 12 weeks for participants who weighed ≥ 17 kg. SOF/VEL FDC 150/37.5 mg oral granules once daily for 12 weeks for participants who weighed < 17 kg.
Overall Number of Participants Analyzed 53 39 23
Median (Inter-Quartile Range)
Unit of Measure: cm
170.5
(162.0 to 176.7)
170.0
(163.7 to 178.6)
168.7
(163.0 to 174.1)
19.Secondary Outcome
Title Treatment Phase: Change From Baseline in Growth and Development as Measured by Bone Age
Hide Description Bone age was determined based on x-ray of the left wrist, hand, and fingers. Baseline value is the last available value on or prior to first dose date of study drug.
Time Frame Baseline; FU-24
Hide Outcome Measure Data
Hide Analysis Population Description
Participants in the Full Analysis Set with available data were analyzed.
Arm/Group Title 12 to < 18 Years Old 6 to < 12 Years Old 3 to < 6 Years Old
Hide Arm/Group Description:
Treatment Phase: SOF/VEL 400/100 mg (adult and smaller size tablets based on swallowability assessment) once daily for 12 weeks.
Treatment Phase: SOF/VEL FDC 200/50 mg tablets or oral granules once daily for 12 weeks.
Treatment Phase: SOF/VEL FDC 200/50 mg oral granules once daily for 12 weeks for participants who weighed ≥ 17 kg. SOF/VEL FDC 150/37.5 mg oral granules once daily for 12 weeks for participants who weighed < 17 kg.
Overall Number of Participants Analyzed 102 73 41
Median (Inter-Quartile Range)
Unit of Measure: years
Baseline Number Analyzed 102 participants 72 participants 40 participants
15.5
(13.5 to 17.0)
7.8
(6.8 to 9.0)
4.8
(4.2 to 5.5)
Change From Baseline at FU-24 Number Analyzed 92 participants 68 participants 35 participants
0.6
(0.0 to 1.0)
1.0
(0.0 to 1.2)
0.5
(0.0 to 1.1)
20.Secondary Outcome
Title Treatment Phase: Swallowability of SOF/VEL as Assessed by the Participant's Ability to Swallow SOF/VEL Placebo Tablets at Baseline
Hide Description A SOF/VEL FDC swallowability assessment was performed using placebo tablets at baseline.
Time Frame Baseline
Hide Outcome Measure Data
Hide Analysis Population Description
Participants in the Full Analysis Set with available data were analyzed. Swallowability assessment was performed in 12 to <18 years old and 6 to < 12 Years old only.
Arm/Group Title 12 to < 18 Years Old 6 to < 12 Years Old
Hide Arm/Group Description:
Treatment Phase: SOF/VEL 400/100 mg (adult and smaller size tablets based on swallowability assessment) once daily for 12 weeks.
Treatment Phase: SOF/VEL FDC 200/50 mg tablets or oral granules once daily for 12 weeks.
Overall Number of Participants Analyzed 102 73
Measure Type: Count of Participants
Unit of Measure: Participants
SOF/VEL 400/100mg Placebo Tablet, Able to Swallow Number Analyzed 102 participants 1 participants
92 1
SOF/VEL 400/100mg Placebo, Not Able to Swallow Number Analyzed 102 participants 1 participants
10 0
SOF/VEL 200/50mg Placebo, Able to Swallow Number Analyzed 10 participants 73 participants
10 72
SOF/VEL 200/50mg Placebo, Not Able to Swallow Number Analyzed 10 participants 73 participants
0 1
21.Secondary Outcome
Title Treatment Phase: Number of Participants With Acceptability of SOF/VEL as Measured by a Questionnaire to Assess Acceptability, Including Palatability at Day 1
Hide Description Acceptability was assessed by numeric response marked on line between numbers 0 - 100. Higher scores indicate better acceptability and/or palatability.
Time Frame Day 1
Hide Outcome Measure Data
Hide Analysis Population Description
Participants in the Full Analysis Set with available data were analyzed.
Arm/Group Title 12 to < 18 Years Old 12 to <18 Years Old 6 to < 12 Years Old 6 to <12 Years Old 3 to < 6 Years Old 3 to <6 Years Old
Hide Arm/Group Description:
Treatment Phase: SOF/VEL 400/100 mg (adult and smaller size tablets based on swallowability assessment) once daily for 12 weeks.
Treatment Phase: 2 * SOF/VEL 200/50 mg (adult and smaller size tablets based on swallowability assessment) once daily for 12 weeks.
Treatment Phase: SOF/VEL FDC 200/50 mg tablets once daily for 12 weeks.
Treatment Phase: SOF/VEL FDC 200/50 mg oral granules once daily for 12 weeks.
Treatment Phase: SOF/VEL FDC 200/50 mg oral granules once daily for 12 weeks for participants who weighed ≥ 17 kg.
SOF/VEL FDC 150/37.5 mg oral granules once daily for 12 weeks for participants who weighed < 17 kg.
Overall Number of Participants Analyzed 91 11 72 1 29 12
Measure Type: Count of Participants
Unit of Measure: Participants
Taste, Participants Who Did Not Taste Study Drug Number Analyzed 91 participants 11 participants 71 participants 1 participants 27 participants 12 participants
61 5 42 0 8 5
Taste, Participants Who Marked > 60 to 100 Number Analyzed 91 participants 11 participants 71 participants 1 participants 27 participants 12 participants
16 3 11 0 6 3
Taste, Participants Who Marked 40 to 60 Number Analyzed 91 participants 11 participants 71 participants 1 participants 27 participants 12 participants
11 1 6 0 4 1
Taste, Participants Who Marked 0 to < 40 Number Analyzed 91 participants 11 participants 71 participants 1 participants 27 participants 12 participants
3 2 12 1 9 3
Easy to Take, Participants Who Marked > 60 to 100 Number Analyzed 89 participants 11 participants 71 participants 1 participants 27 participants 12 participants
82 9 60 0 13 4
Easy to Take, Participants Who Marked 40 to 60 Number Analyzed 89 participants 11 participants 71 participants 1 participants 27 participants 12 participants
5 1 3 0 4 2
Easy to Take, Participants Who Marked 0 to < 40 Number Analyzed 89 participants 11 participants 71 participants 1 participants 27 participants 12 participants
2 1 8 1 10 6
22.Secondary Outcome
Title Treatment Phase: Number of Participants With Acceptability of SOF/VEL as Measured by a Questionnaire to Assess Acceptability, Including Palatability at Week 12
Hide Description Acceptability was assessed by numeric response marked on line between numbers 0 - 100. Higher scores indicate better acceptability and/or palatability.
Time Frame Week 12
Hide Outcome Measure Data
Hide Analysis Population Description
Participants in the Full Analysis Set with available data were analyzed.
Arm/Group Title 12 to < 18 Years Old 12 to <18 Years Old 6 to < 12 Years Old 6 to <12 Years Old 3 to < 6 Years Old 3 to <6 Years Old
Hide Arm/Group Description:
Treatment Phase: SOF/VEL 400/100 mg (adult and smaller size tablets based on swallowability assessment) once daily for 12 weeks.
Treatment Phase: 2 * SOF/VEL 200/50 mg (adult and smaller size tablets based on swallowability assessment) once daily for 12 weeks.
Treatment Phase: SOF/VEL FDC 200/50 mg tablets once daily for 12 weeks.
Treatment Phase: SOF/VEL FDC 200/50 mg oral granules once daily for 12 weeks.
Treatment Phase: SOF/VEL FDC 200/50 mg oral granules once daily for 12 weeks for participants who weighed ≥ 17 kg.
SOF/VEL FDC 150/37.5 mg oral granules once daily for 12 weeks for participants who weighed < 17 kg.
Overall Number of Participants Analyzed 91 11 72 1 29 12
Measure Type: Count of Participants
Unit of Measure: Participants
Taste, Participants Who Did Not Taste Study Drug Number Analyzed 90 participants 11 participants 70 participants 1 participants 23 participants 8 participants
49 2 33 0 8 2
Taste, Participants Who Marked > 60 to 100 Number Analyzed 90 participants 11 participants 70 participants 1 participants 23 participants 8 participants
24 5 14 0 4 1
Taste, Participants Who Marked 40 to 60 Number Analyzed 90 participants 11 participants 70 participants 1 participants 23 participants 8 participants
10 1 12 1 4 3
Taste, Participants Who Marked 0 to < 40 Number Analyzed 90 participants 11 participants 70 participants 1 participants 23 participants 8 participants
7 3 11 0 7 2
Easy to Take, Participants Who Marked > 60 to 100 Number Analyzed 89 participants 11 participants 70 participants 1 participants 23 participants 8 participants
83 9 65 1 20 6
Easy to Take, Participants Who Marked 40 to 60 Number Analyzed 89 participants 11 participants 70 participants 1 participants 23 participants 8 participants
3 0 5 0 1 1
Easy to Take, Participants Who Marked 0 to < 40 Number Analyzed 89 participants 11 participants 70 participants 1 participants 23 participants 8 participants
3 2 0 0 2 1
Feel About Taking Pills, Who Marked > 60 to 100 Number Analyzed 88 participants 11 participants 70 participants 1 participants 23 participants 12 participants
82 8 60 NA [1]  NA [1]  NA [1] 
Feel About Taking Pills, Who Marked 40 to 60 Number Analyzed 88 participants 11 participants 70 participants 1 participants 23 participants 12 participants
3 1 7 NA [1]  NA [1]  NA [1] 
Feel About Taking Pills, Who Marked 0 to < 40 Number Analyzed 88 participants 11 participants 70 participants 1 participants 23 participants 12 participants
3 2 3 NA [1]  NA [1]  NA [1] 
[1]
Data not applicable for the oral granules group.
Time Frame Adverse Events: From first dose through last dose of the study drug (Up to 12 weeks) plus 30 days; All-Cause Mortality: First dose date up to Posttreatment Week 24
Adverse Event Reporting Description The Safety Analysis Set included all participants who were enrolled into the study and received at least 1 dose of study drug (SOF/VEL FDC).
 
Arm/Group Title 12 to < 18 Years Old 6 to < 12 Years Old 3 to < 6 Years Old
Hide Arm/Group Description

PK Lead-in Phase: Sofosbuvir/Velpatasvir (SOF/VEL) fixed-dose combination (FDC) 400/100 mg tablets once daily for 7 days. Participants who completed the PK lead-in phase, continued into the treatment phase with no interruption of study drug administration and additional participants were enrolled into the treatment phase once the appropriateness of the dose was confirmed by PK results from the PK lead-in phase.

Treatment Phase: SOF/VEL 400/100 mg (adult and smaller size tablets based on swallowability assessment) once daily for 12 weeks.

PK Lead-in Phase: SOF/VEL FDC 200/50 mg tablets or oral granules once daily for 7 days. Participants who completed the PK lead-in phase, continued into the treatment phase with no interruption of study drug administration and additional participants were enrolled into the treatment phase once the appropriateness of the dose was confirmed by PK results from the PK lead-in phase.

Treatment Phase: SOF/VEL FDC 200/50 mg tablets or oral granules once daily for 12 weeks.

PK Lead-in Phase: SOF/VEL FDC 200/50 mg oral granules once daily for 7 days for participants who weighed ≥ 17 kg. SOF/VEL FDC 150/37.5 mg oral granules once daily for 7 days for participants who weighed < 17 kg. Participants who completed the PK lead-in phase, continued into the treatment phase with no interruption of study drug administration and additional participants were enrolled into the treatment phase once the appropriateness of the dose was confirmed by PK results from the PK lead-in phase.

Treatment Phase: SOF/VEL FDC 200/50 mg oral granules once daily for 12 weeks for participants who weighed ≥ 17 kg. SOF/VEL FDC 150/37.5 mg oral granules once daily for 12 weeks for participants who weighed < 17 kg.

All-Cause Mortality
12 to < 18 Years Old 6 to < 12 Years Old 3 to < 6 Years Old
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/102 (0.00%)   0/73 (0.00%)   0/41 (0.00%) 
Hide Serious Adverse Events
12 to < 18 Years Old 6 to < 12 Years Old 3 to < 6 Years Old
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   2/102 (1.96%)   2/73 (2.74%)   0/41 (0.00%) 
Gastrointestinal disorders       
Constipation  1  0/102 (0.00%)  1/73 (1.37%)  0/41 (0.00%) 
Psychiatric disorders       
Bipolar disorder  1  1/102 (0.98%)  0/73 (0.00%)  0/41 (0.00%) 
Hallucination, auditory  1  0/102 (0.00%)  1/73 (1.37%)  0/41 (0.00%) 
Suicidal ideation  1  2/102 (1.96%)  0/73 (0.00%)  0/41 (0.00%) 
Suicide attempt  1  1/102 (0.98%)  0/73 (0.00%)  0/41 (0.00%) 
1
Term from vocabulary, MedDRA Version 22.1
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
12 to < 18 Years Old 6 to < 12 Years Old 3 to < 6 Years Old
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   65/102 (63.73%)   53/73 (72.60%)   26/41 (63.41%) 
Gastrointestinal disorders       
Abdominal pain  1  6/102 (5.88%)  9/73 (12.33%)  2/41 (4.88%) 
Abdominal pain upper  1  10/102 (9.80%)  3/73 (4.11%)  2/41 (4.88%) 
Diarrhoea  1  7/102 (6.86%)  6/73 (8.22%)  5/41 (12.20%) 
Nausea  1  17/102 (16.67%)  5/73 (6.85%)  0/41 (0.00%) 
Vomiting  1  9/102 (8.82%)  12/73 (16.44%)  11/41 (26.83%) 
General disorders       
Fatigue  1  22/102 (21.57%)  9/73 (12.33%)  5/41 (12.20%) 
Pyrexia  1  10/102 (9.80%)  8/73 (10.96%)  6/41 (14.63%) 
Infections and infestations       
Nasopharyngitis  1  6/102 (5.88%)  7/73 (9.59%)  1/41 (2.44%) 
Upper respiratory tract infection  1  3/102 (2.94%)  7/73 (9.59%)  2/41 (4.88%) 
Injury, poisoning and procedural complications       
Product use issue  1  0/102 (0.00%)  2/73 (2.74%)  4/41 (9.76%) 
Metabolism and nutrition disorders       
Decreased appetite  1  1/102 (0.98%)  2/73 (2.74%)  3/41 (7.32%) 
Musculoskeletal and connective tissue disorders       
Arthralgia  1  1/102 (0.98%)  4/73 (5.48%)  0/41 (0.00%) 
Nervous system disorders       
Dizziness  1  9/102 (8.82%)  2/73 (2.74%)  1/41 (2.44%) 
Headache  1  30/102 (29.41%)  11/73 (15.07%)  2/41 (4.88%) 
Respiratory, thoracic and mediastinal disorders       
Cough  1  10/102 (9.80%)  11/73 (15.07%)  6/41 (14.63%) 
Epistaxis  1  3/102 (2.94%)  6/73 (8.22%)  2/41 (4.88%) 
Nasal congestion  1  6/102 (5.88%)  4/73 (5.48%)  5/41 (12.20%) 
Oropharyngeal pain  1  9/102 (8.82%)  2/73 (2.74%)  0/41 (0.00%) 
Rhinorrhoea  1  4/102 (3.92%)  4/73 (5.48%)  6/41 (14.63%) 
Skin and subcutaneous tissue disorders       
Rash  1  0/102 (0.00%)  7/73 (9.59%)  1/41 (2.44%) 
1
Term from vocabulary, MedDRA Version 22.1
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

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.

Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Gilead Clinical Study Information Center
Organization: Gilead Sciences
Phone: 1-833-445-3230 (GILEAD-0)
EMail: GileadClinicalTrials@gilead.com
Publications of Results:
Jonas MM, Romero R, Sokal EM, Rosenthal P, Verucchi G, Lin CH, et al. The Safety and Efficacy of Sofosbuvir/Velpatasvir in Pediatric Patients 6 to < 18 years old with Chronic Hepatitis C Infection [Abstract]. AASLD; 2019 08-12 November; Boston, Massachusetts.
Layout table for additonal information
Responsible Party: Gilead Sciences
ClinicalTrials.gov Identifier: NCT03022981    
Other Study ID Numbers: GS-US-342-1143
2016-002446-23 ( EudraCT Number )
First Submitted: January 13, 2017
First Posted: January 18, 2017
Results First Submitted: August 24, 2020
Results First Posted: October 8, 2020
Last Update Posted: October 8, 2020