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

NCT03022981 | Completed Phase 2 Results DMC FDA Drug

• 2 other identifiers: GS-US-342-11432016-002446-23
• Study Type: interventional
• Target: 216
• Locations: 4 countries
• Sites: 27

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.

Conditions

Hepatitis C Virus Infection

Outcome Measures

Primary Outcomes (4)

• PK Lead-in Phase: AUCtau: Area Under the Plasma Concentration Versus Time Curve Over the Dosing Interval of Velpatasvir (VEL)

  AUCtau is defined as concentration of drug over time (the area under the concentration versus time curve over the dosing interval).

  Day 7: 0 (predose), 0.5, 1, 2, 3, 4, 6 (Cohorts 1 and 2 only), 8, and 12 hours postdose

• PK Lead-in Phase: AUCtau: Area Under the Plasma Concentration Versus Time Curve Over the Dosing Interval of Sofosbuvir (SOF)

  AUCtau is defined as concentration of drug over time (the area under the concentration versus time curve over the dosing interval).

  Day 7: 0 (predose), 0.5, 1, 2, 3, 4, 6 (Cohorts 1 and 2 only), 8, and 12 hours postdose

• PK Lead-in Phase: AUCtau: Area Under the Plasma Concentration Versus Time Curve Over the Dosing Interval of GS-331007 (Metabolite of SOF)

  AUCtau is defined as concentration of drug over time (the area under the concentration verses time curve over the dosing interval).

  Day 7: 0 (predose), 0.5, 1, 2, 3, 4, 6 (Cohorts 1 and 2 only), 8, and 12 hours postdose

• Treatment Phase: Percentage of Participants Who Discontinued Study Drug Due to Any Treatment-Emergent Adverse Event (TEAE)

  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.

  From first dose through last dose of the study drug (Up to 12 weeks) plus 30 days

Secondary Outcomes (18)

• PK Lead-in Phase: Change From Baseline in Hepatitis C Virus (HCV) Ribonucleic Acid (RNA) at Day 7

  Baseline; Day 7

• PK Lead-in Phase: Percentage of Participants Who Permanently Discontinued Study Drug Due to an Adverse Event (AE)

  First dose date up to Day 7

• Treatment Phase: Percentage of Participants With Sustained Virologic Response (SVR) at 12 Weeks After Discontinuation of Therapy (SVR12)

  Posttreatment Week 12

• Treatment Phase: Percentage of Participants With SVR at 4 Weeks After Discontinuation of Therapy (SVR4)

  Posttreatment Week 4

• Treatment Phase: Percentage of Participants With SVR at 24 Weeks After Discontinuation of Therapy (SVR24)

  Posttreatment Week 24

Study Arms (3)

12 to < 18 Years Old

EXPERIMENTAL

PK Lead-in Phase: Sofosbuvir/Velpatasvir (SOF/VEL) 400/100 mg once daily for 7 days. Participants who complete the PK lead-in phase, continue into the treatment phase with no interruption of study drug administration and additional participants will be enrolled into the treatment phase once the appropriateness of the dose is confirmed by PK results from the PK lead-in phase. Treatment Phase: SOF/VEL 400/100 mg once daily for 12 weeks.

Drug: SOF/VEL

6 to < 12 Years Old

EXPERIMENTAL

PK Lead-in Phase: SOF/VEL 200/50 mg once daily for 7 days. Participants who complete the PK lead-in phase, continue into the treatment phase with no interruption of study drug administration and additional participants will be enrolled into the treatment phase once the appropriateness of the dose is confirmed by PK results from the PK lead-in phase. Treatment Phase: SOF/VEL 200/50 mg once daily for 12 weeks.

Drug: SOF/VEL

3 to < 6 Years Old

EXPERIMENTAL

PK Lead-in Phase: SOF/VEL 200/50 mg once daily for 7 days for participants who weigh ≥ 17 kg. SOF/VEL 150/37.5 mg once daily for 7 days for participants who weigh \< 17 kg. Participants who complete the PK lead-in phase, continue into the treatment phase with no interruption of study drug administration and additional participants will be enrolled into the treatment phase once the appropriateness of the dose is confirmed by PK results from the PK lead-in phase. Treatment Phase: SOF/VEL 200/50 mg once daily for 12 weeks for participants who weigh ≥ 17 kg. SOF/VEL 150/37.5 mg once daily for 12 weeks for participants who weigh \< 17 kg.

Drug: SOF/VEL

Interventions

SOF/VEL DRUG

SOF/VEL fixed-dose combination (FDC) 400/100 mg tablets or SOF/VEL FDC 200/50 mg tablets (based on swallowability assessment)

Also known as: Epclusa®, GS-7977/GS-5816

12 to < 18 Years Old; 6 to < 12 Years Old

Eligibility Criteria

• Age: 3 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Chronic HCV-infected, treatment-naive and treatment-experienced adolescent and pediatric individuals aged 3 to \< 18 as determined at Day 1.

Sponsors & Collaborators

• Gilead Sciences: lead

Study Sites (27)

Children's Hospital Los Angeles

Los Angeles, California, 90027, United States

Location

University of California, San Francisco

San Francisco, California, 94158, United States

Location

Children's Hospital of Colorado

Aurora, Colorado, 80045, United States

Location

Children's National Medical Center

Washington D.C., District of Columbia, 20010, United States

Location

University of Florida Shands Medical

Gainesville, Florida, 32610, United States

Location

Florida Gastroenterology Care for Children

Orlando, Florida, 32803, United States

Location

Emory Children's Center

Atlanta, Georgia, 30322, United States

Location

Riley Hospital for Children

Indianapolis, Indiana, 46202, United States

Location

Kosair Charities Pediatric Clinical Research Unit

Louisville, Kentucky, 40202, United States

Location

Johns Hopkins University (JHU) - The Johns Hopkins Hospital (JHH)

Baltimore, Maryland, 21287, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

Children's Mercy Hospital

Kansas City, Missouri, 64108, United States

Location

Children's Hospital & Medical Center

Omaha, Nebraska, 68198-5331, United States

Location

Mount Sinai Medical Center

New York, New York, 10029, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Nationwide Children's Hospital- The Ohio State University (OSU)

Columbus, Ohio, 43205, United States

Location

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Monroe Carell Jr. Children's Hospital at Vanderbilt

Nashville, Tennessee, 37232, United States

Location

Cook Children's Medical Center

Fort Worth, Texas, 76104, United States

Location

Baylor College of Medicine

Houston, Texas, 77030, United States

Location

Seattle Children's Hospital

Seattle, Washington, 98105, United States

Location

Cliniques Universitaires Saint Luc

Brussels, 1200, Belgium

Location

Azienda Ospedaliero-Universitaria di Bologna - Policlinico S. Orsola - Malpighi

Bologna, 40138, Italy

Location

Azienda Ospedaliera Universitaria Meyer

Florence, 50139, Italy

Location

Ospedale Casa Sollievo della Sofferenza

San Giovanni Rotondo, 71013, Italy

Location

The Leeds Teaching Hospitals NHS Trust

Leeds, England, LS1 3EX, United Kingdom

Location

King's College Hospital NHS Trust

London, England, SE5 9RS, United Kingdom

Location

Related Publications (1)

• 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.

  RESULT

MeSH Terms

Conditions

Hepatitis C

Interventions

sofosbuvir-velpatasvir drug combination

Condition Hierarchy (Ancestors)

Blood-Borne Infections; Communicable Diseases; Infections; Hepatitis, Viral, Human; Virus Diseases; Flaviviridae Infections; RNA Virus Infections; Hepatitis; Liver Diseases; Digestive System Diseases

Results Point of Contact

• Title: Gilead Clinical Study Information Center
• Organization: Gilead Sciences

GileadClinicalTrials@gilead.com

1-833-445-3230 (GILEAD-0)

Study Officials

• Gilead Study Director

  Gilead Sciences

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 13, 2017
• First Posted: January 18, 2017
• Study Start: January 26, 2017
• Primary Completion: November 19, 2019
• Study Completion: February 26, 2020
• Last Updated: October 8, 2020
• Results First Posted: October 8, 2020

Record last verified: 2020-10

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP
• Time Frame: 18 months after study completion
• Access Criteria: A secured external environment with username, password, and RSA code.

Locations

GB (2); US (21); BE (1); IT (3)