Phase 2 Completed N=34 Treatment

Safety and Efficacy of Sofosbuvir + Ribavirin in Adolescents and Children With Genotype 2 or 3 Chronic HCV Infection

Source: ClinicalTrials.gov NCT02175758 ↗

Enrolled (actual)

Serious AEs

0.9%

Results posted

Apr 2019

Primary outcomePrimary: For Participants in the PK Lead-in Phase, Pharmacokinetic (PK) Parameter: AUCtau of GS-331007 (Metabolite of SOF) — 9106.0; 7651.2; 10293.7 h*ng/mL

Summary

This study will have two parts as follows: The PK Lead-in Phase of the study will evaluate the steady state pharmacokinetics (PK) and confirm the dose of sofosbuvir (SOF) in hepatitis C virus (HCV)-infected pediatric participants. The PK Lead-in Phase will also evaluate the safety and tolerability of 7 days of dosing of SOF+ribavirin (RBV) in HCV-infected pediatric participants. The Treatment Phase will be initiated by age cohort after confirmation of age-appropriate SOF dosage levels. Participants from the PK Lead-in Phase will immediately rollover into the Treatment Phase with no interruption of study drug administration. The Treatment Phase will evaluate the antiviral efficacy, safety, and tolerability of SOF+RBV for 12 or 24 weeks in pediatric participants with genotype 2 or 3 HCV infection, respectively.

Outcome Measures

Outcome	Result	p-value
PRIMARY For Participants in the PK Lead-in Phase, Pharmacokinetic (PK) Parameter: AUCtau of GS-331007 (Metabolite of SOF)	9106.0; 7651.2; 10293.7	—
PRIMARY Percentage of Participants Who Permanently Discontinued Any Study Drug Due to an Adverse Event During the PK Lead-in Phase or the Treatment Phase	0; 0; 0; 0; 20.0; 0	—
PRIMARY For the Treatment Phase, Percentage of Participants With SVR at 12 Weeks After Discontinuation of Therapy (SVR12)	98.1; 98.1; 100.0; 97.4; 100.0; 100.0	<0.001 sig
SECONDARY For Participants in the PK Lead-in Phase, Change From Baseline in HCV RNA	-3.98; -4.23; -3.82; -3.78; -4.12; -3.53	—
SECONDARY Percentage of Participants Who Permanently Discontinued Any Study Drug Due to an Adverse Event During the PK Lead-in Phase	0; 0; 0	—
SECONDARY For the Treatment Phase, Percentage of Participants With Sustained Virologic Response (SVR) at 4 Weeks After Discontinuation of Therapy (SVR4)	100.0; 100.0; 100.0; 100.0; 80.0; 100.0	—
SECONDARY For the Treatment Phase, Percentage of Participants With SVR at 24 Weeks After Discontinuation of Therapy (SVR24)	100.0; 97.4; 100.0; 100.0; 80.0; 100.0	—
SECONDARY For the Treatment Phase, Percentage of Participants Experiencing Viral Breakthrough	0; 0; 0; 0; 0; 0	—
SECONDARY For the Treatment Phase, Percentage of Participants Experiencing Viral Relapse	0; 0; 0; 0; 0; 0	—
SECONDARY For the Treatment Phase, Change From Baseline in HCV RNA	-4.25; -4.12; -4.12; -3.88; -4.12; -3.53	—
SECONDARY For the Treatment Phase, Percentage of Participants With HCV RNA < LLOQ While On Treatment	30.8; 30.8; 46.2; 39.3; 50.0; 37.5	—
SECONDARY For the Treatment Phase, Percentage of Participants With Alanine Aminotransferase (ALT) Normalization	50.0; 63.6; 25.0; 75.0; 100.0; 100.0	—
SECONDARY For the Treatment Phase, Change From Baseline in Height	0.1; 0.0; 0.0; 0.0; 0.3; 0.3	—
SECONDARY For the Treatment Phase, Change From Baseline in Weight	-0.7; -0.4; 0.2; 0.0; 0.2; -0.2	—
SECONDARY For the Treatment Phase, Number of Male Participants With a Change From Baseline in Tanner Stage for Pubic Hair	23; 10; 3; 8; 1; 0	—
SECONDARY For the Treatment Phase, Number of Male Participants With a Change From Baseline in Tanner Stage for Genitalia Development	24; 11; 3; 7; 0; 0	—
SECONDARY For the Treatment Phase, Number of Female Participants With a Change From Baseline in Tanner Stage for Pubic Hair	20; 24; 8; 1; 6; 0	—
SECONDARY For the Treatment Phase, Number of Female Participants With a Change From Baseline in Tanner Stage for Breast Development	20; 24; 8; 1; 5; 0	—
SECONDARY For the Treatment Phase, Palatability of SOF Granules at Day 1 as Assessed by the Percentage of Participants Able/Unable to Taste the SOF Oral Granules	0; 42.9; 75.0; 100.0; 57.1; 25.0	—

Eligibility Criteria

Key Inclusion Criteria

Consent of parent or legal guardian required
Chronic HCV infection genotype 2 or 3
Screening laboratory values within defined thresholds
PK Lead-in only: all individuals must be treatment naive

Key Exclusion Criteria

History of clinically significant illness or any other medical disorder that may interfere with subject treatment, assessment or compliance with the protocol
Co-infection with HIV, acute hepatitis A virus, or hepatitis B virus
Clinical hepatic decompensation (ie, ascites, encephalopathy or variceal hemorrhage)
Pregnant or nursing females
Known hypersensitivity to study medication
Use of any prohibited concomitant medications as within 28 days of the Day 1 visit

Note: Other protocol defined Inclusion/Exclusion criteria may apply.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02175758). 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.