Clinical trial NCT03022981: Study to Investigate the Safety and Efficacy of Sofosbuvir/Velpatasvir in Adolescents and Children With Chronic HCV Infection
Official title: A Phase 2, Open-Label, Multicenter, Multi-cohort Study to Investigate the Safety and Efficacy of Sofosbuvir/Velpatasvir in Adolescents and Children With Chronic HCV Infection
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Hepatitis C Virus Infection
Lead sponsor: Gilead Sciences
Sponsor class: INDUSTRY
First posted: 2017-01-18
Enrollment: 216
Sex: ALL
Minimum age (years): 3
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief 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.


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.

Primary outcomes:
measure: PK Lead-in Phase: AUCtau: Area Under the Plasma Concentration Versus Time Curve Over the Dosing Interval of Velpatasvir (VEL)
timeFrame: Day 7: 0 (predose), 0.5, 1, 2, 3, 4, 6 (Cohorts 1 and 2 only), 8, and 12 hours postdose
measure: PK Lead-in Phase: AUCtau: Area Under the Plasma Concentration Versus Time Curve Over the Dosing Interval of Sofosbuvir (SOF)
timeFrame: Day 7: 0 (predose), 0.5, 1, 2, 3, 4, 6 (Cohorts 1 and 2 only), 8, and 12 hours postdose
measure: PK Lead-in Phase: AUCtau: Area Under the Plasma Concentration Versus Time Curve Over the Dosing Interval of GS-331007 (Metabolite of SOF)
timeFrame: Day 7: 0 (predose), 0.5, 1, 2, 3, 4, 6 (Cohorts 1 and 2 only), 8, and 12 hours postdose
measure: Treatment Phase: Percentage of Participants Who Discontinued Study Drug Due to Any Treatment-Emergent Adverse Event (TEAE)
timeFrame: From first dose through last dose of the study drug (Up to 12 weeks) plus 30 days
Secondary outcomes:
measure: PK Lead-in Phase: Change From Baseline in Hepatitis C Virus (HCV) Ribonucleic Acid (RNA) at Day 7
timeFrame: Baseline; Day 7
measure: PK Lead-in Phase: Percentage of Participants Who Permanently Discontinued Study Drug Due to an Adverse Event (AE)
timeFrame: First dose date up to Day 7
measure: Treatment Phase: Percentage of Participants With Sustained Virologic Response (SVR) at 12 Weeks After Discontinuation of Therapy (SVR12)
timeFrame: Posttreatment Week 12
measure: Treatment Phase: Percentage of Participants With SVR at 4 Weeks After Discontinuation of Therapy (SVR4)
timeFrame: Posttreatment Week 4
measure: Treatment Phase: Percentage of Participants With SVR at 24 Weeks After Discontinuation of Therapy (SVR24)
timeFrame: Posttreatment Week 24
measure: Treatment Phase: Percentage of Participants With Virologic Failure
timeFrame: Up to Posttreatment Week 24
measure: Treatment Phase: Percentage of Participants With HCV RNA < LLOQ On Treatment
timeFrame: Weeks 1, 4, 8, and 12
measure: Treatment Phase: Percentage of Participants Who Develop Viral Resistance to SOF and/or VEL During Treatment and After Discontinuation of Treatment
timeFrame: First dose date up to Posttreatment Week 24
measure: Treatment Phase: Change From Baseline in HCV RNA at Weeks 1, 4, 8, and 12
timeFrame: Baseline; Weeks 1, 4, 8, and 12
measure: Treatment Phase: Quality of Life (QoL) and Neuropsychiatric Assessments as Measured by PedsQL™ Pediatric QoL Survey
timeFrame: Baseline; Week 12, End of Treatment (EOT), Posttreatment/Follow-up (FU) Week-12 (FU-12), and FU Week-24 (FU-24)
measure: Treatment Phase: Change From Baseline in Growth and Development as Measured by Height Percentiles
timeFrame: Baseline; Weeks 1, 4, 8, 12, Follow-up (FU) Week 4 (FU-4), FU-12, and FU-24
measure: Treatment Phase: Change From Baseline in Growth and Development as Measured by Weight Percentiles
timeFrame: Baseline; Weeks 1, 4, 8, 12, FU-4, FU-12, and FU-24
measure: Treatment Phase: Changes in Growth and Development as Measured by Tanner Stage Assessment From Baseline
timeFrame: Baseline; EOT, FU-12 and FU-24
measure: Treatment Phase: Growth and Development as Measured by Parental Height
timeFrame: Day 1
measure: Treatment Phase: Change From Baseline in Growth and Development as Measured by Bone Age
timeFrame: Baseline; FU-24
measure: Treatment Phase: Swallowability of SOF/VEL as Assessed by the Participant's Ability to Swallow SOF/VEL Placebo Tablets at Baseline
timeFrame: Baseline
measure: Treatment Phase: Number of Participants With Acceptability of SOF/VEL as Measured by a Questionnaire to Assess Acceptability, Including Palatability at Day 1
timeFrame: Day 1
measure: Treatment Phase: Number of Participants With Acceptability of SOF/VEL as Measured by a Questionnaire to Assess Acceptability, Including Palatability at Week 12
timeFrame: Week 12
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Children's Hospital Los Angeles
state: California
city: San Francisco
country: United States
facility: University of California, San Francisco
state: California
city: Aurora
country: United States
facility: Children's Hospital of Colorado
state: Colorado
city: Washington
country: United States
facility: Children's National Medical Center
state: District of Columbia
city: Gainesville
country: United States
facility: University of Florida Shands Medical
state: Florida
city: Orlando
country: United States
facility: Florida Gastroenterology Care for Children
state: Florida
city: Atlanta
country: United States
facility: Emory Children's Center
state: Georgia
city: Indianapolis
country: United States
facility: Riley Hospital for Children
state: Indiana
city: Louisville
country: United States
facility: Kosair Charities Pediatric Clinical Research Unit
state: Kentucky
city: Baltimore
country: United States
facility: Johns Hopkins University (JHU) - The Johns Hopkins Hospital (JHH)
state: Maryland
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Kansas City
country: United States
facility: Children's Mercy Hospital
state: Missouri
city: Omaha
country: United States
facility: Children's Hospital & Medical Center
state: Nebraska
city: New York
country: United States
facility: Mount Sinai Medical Center
state: New York
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
city: Columbus
country: United States
facility: Nationwide Children's Hospital- The Ohio State University (OSU)
state: Ohio
city: Philadelphia
country: United States
facility: The Children's Hospital of Philadelphia
state: Pennsylvania
city: Nashville
country: United States
facility: Monroe Carell Jr. Children's Hospital at Vanderbilt
state: Tennessee
city: Fort Worth
country: United States
facility: Cook Children's Medical Center
state: Texas
city: Houston
country: United States
facility: Baylor College of Medicine
state: Texas
city: Seattle
country: United States
facility: Seattle Children's Hospital
state: Washington
city: Brussels
country: Belgium
facility: Cliniques Universitaires Saint Luc
city: Bologna
country: Italy
facility: Azienda Ospedaliero-Universitaria di Bologna - Policlinico S. Orsola - Malpighi
city: Firenze
country: Italy
facility: Azienda Ospedaliera Universitaria Meyer
city: San Giovanni Rotondo
country: Italy
facility: Ospedale Casa Sollievo della Sofferenza
city: Leeds
country: United Kingdom
facility: The Leeds Teaching Hospitals NHS Trust
state: England
city: London
country: United Kingdom
facility: King's College Hospital NHS Trust
state: England
Study officials:
affiliation: Gilead Sciences
name: Gilead Study Director
role: STUDY_DIRECTOR
References:
citation: 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.