Phase 2
Completed N=216
Study to Investigate the Safety and Efficacy of Sofosbuvir/Velpatasvir in Adolescents and Children With Chronic HCV Infection
Source: ClinicalTrials.gov NCT03022981 ↗Enrolled (actual)
216
Serious AEs
1.8%
Results posted
Oct 2020
Primary outcomePrimary: PK Lead-in Phase: AUCtau: Area Under the Plasma Concentration Versus Time Curve Over the Dosing Interval of Velpatasvir (VEL) — 4479.3; 3697.5; 4450.3 hours*nanograms per milliliter (h*ng/mL)
Summary
This study will have 2 parts: Pharmacokinetics (PK) Lead-in Phase and the Treatment Phase.
The primary objective of the PK Lead-in Phase is to evaluate the steady state PK and confirm the dose of sofosbuvir/velpatasvir (SOF/VEL) fixed-dose combination (FDC) in pediatric participants with chronic hepatitis C virus (HCV) infection.
The primary objective of the Treatment Phase is to evaluate the safety and tolerability of SOF/VEL for 12 weeks in pediatric participants with chronic HCV.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY PK Lead-in Phase: AUCtau: Area Under the Plasma Concentration Versus Time Curve Over the Dosing Interval of Velpatasvir (VEL) |
4479.3; 3697.5; 4450.3 | — |
| PRIMARY PK Lead-in Phase: AUCtau: Area Under the Plasma Concentration Versus Time Curve Over the Dosing Interval of Sofosbuvir (SOF) |
3020.1; 1764.5; 3306.2 | — |
| PRIMARY PK Lead-in Phase: AUCtau: Area Under the Plasma Concentration Versus Time Curve Over the Dosing Interval of GS-331007 (Metabolite of SOF) |
13852.9; 9913.8; 11604.0 | — |
| PRIMARY Treatment Phase: Percentage of Participants Who Discontinued Study Drug Due to Any Treatment-Emergent Adverse Event (TEAE) |
0.0; 2.7; 2.4 | — |
| SECONDARY PK Lead-in Phase: Change From Baseline in Hepatitis C Virus (HCV) Ribonucleic Acid (RNA) at Day 7 |
6.09; 5.84; 5.77; -4.48; -4.20; -3.94 | — |
| SECONDARY PK Lead-in Phase: Percentage of Participants Who Permanently Discontinued Study Drug Due to an Adverse Event (AE) |
0.0; 0.0; 0.0 | — |
| SECONDARY Treatment Phase: Percentage of Participants With Sustained Virologic Response (SVR) at 12 Weeks After Discontinuation of Therapy (SVR12) |
95.1; 93.2; 82.9 | — |
| SECONDARY Treatment Phase: Percentage of Participants With SVR at 4 Weeks After Discontinuation of Therapy (SVR4) |
96.1; 94.5; 82.9 | — |
| SECONDARY Treatment Phase: Percentage of Participants With SVR at 24 Weeks After Discontinuation of Therapy (SVR24) |
95.1; 93.2; 82.9 | — |
| SECONDARY Treatment Phase: Percentage of Participants With Virologic Failure |
1.0; 1.4; 0.0 | — |
| SECONDARY Treatment Phase: Percentage of Participants With HCV RNA < LLOQ On Treatment |
44.1; 39.7; 36.8; 96.1; 94.4; 91.4 | — |
| SECONDARY Treatment Phase: Percentage of Participants Who Develop Viral Resistance to SOF and/or VEL During Treatment and After Discontinuation of Treatment |
16.3; 10.2; 18.1; 5.1; 0.0; 3.0 | — |
| SECONDARY Treatment Phase: Change From Baseline in HCV RNA at Weeks 1, 4, 8, and 12 |
6.06; 5.87; 5.86; -4.46; -4.28; -4.06 | — |
| SECONDARY Treatment Phase: Quality of Life (QoL) and Neuropsychiatric Assessments as Measured by PedsQL™ Pediatric QoL Survey |
80.0; 79.7; 86.5; 82.4; 82.8; 87.3 | — |
| SECONDARY Treatment Phase: Change From Baseline in Growth and Development as Measured by Height Percentiles |
44.5; 41.9; 39.3; 0.0; 0.1; 0.5 | — |
| SECONDARY Treatment Phase: Change From Baseline in Growth and Development as Measured by Weight Percentiles |
67.2; 45.9; 64.6; 0.0; 0.1; -0.5 | — |
| SECONDARY Treatment Phase: Changes in Growth and Development as Measured by Tanner Stage Assessment From Baseline |
4; 31; 17; 3; 2; 0 | — |
| SECONDARY Treatment Phase: Growth and Development as Measured by Parental Height |
170.5; 170.0; 168.7 | — |
| SECONDARY Treatment Phase: Change From Baseline in Growth and Development as Measured by Bone Age |
15.5; 7.8; 4.8; 0.6; 1.0; 0.5 | — |
| SECONDARY Treatment Phase: Swallowability of SOF/VEL as Assessed by the Participant's Ability to Swallow SOF/VEL Placebo Tablets at Baseline |
92; 1; 10; 0; 10; 72 | — |
| SECONDARY Treatment Phase: Number of Participants With Acceptability of SOF/VEL as Measured by a Questionnaire to Assess Acceptability, Including Palatability at Day 1 |
61; 5; 42; 0; 8; 5 | — |
| SECONDARY Treatment Phase: Number of Participants With Acceptability of SOF/VEL as Measured by a Questionnaire to Assess Acceptability, Including Palatability at Week 12 |
49; 2; 33; 0; 8; 2 | — |
Eligibility Criteria
Key Inclusion Criteria
- Chronic HCV-infected, treatment-naive and treatment-experienced adolescent and pediatric individuals aged 3 to < 18 as determined at Day 1.
Note: Other protocol defined Inclusion/Exclusion criteria may apply.
Data sourced from ClinicalTrials.gov (NCT03022981). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.