A Phase 2 clinical trial is now underway to evaluate sofosbuvir (Sovaldi) plus ribavirin for children and adolescents, ages 3-17 years, with hepatitis C virus genotypes 2 or 3. Another study will follow to test the forthcoming sofosbuvir/ledipasir coformulation in pediatric patients.
The development of direct-acting antiviral agents has brought about a revolution in hepatitis C treatment, especially with the advent of interferon-free regimens. All-oral combinations containing the recently approved sofosbuvir and other agents now awaiting approval have demonstrated sustained virological response or cure rates in the 90% to 100% range. But the new treatments have not been tested in children, for whom lengthy and poorly tolerated interferon-based therapy remains the standard of care.
The sofosbuvir plus ribavirin study will include a lead-in phase that will evaluate the pharmacokinetics and confirm age-appropriate doses of sofosbuvir for pediatric patients and assess the safety and tolerability of sofosbuvir plus ribavirin administered for 7 days.
The treatment phase of this open-label study will then evaluate the antiviral efficacy, safety, and tolerability of sofosbuvir plus ribavirin for 12 weeks in children with HCV genotype 2, or 24 weeks for those with genotype 3. Participants will be divided into 2 age cohorts: 3-11 years and 12-17 years.
The study is or soon will be enrolling participants at 16 study sites throughout the U.S., as well as sites in Germany, Italy, the U.K., Russia, Australia, and New Zealand.
For complete information, including full inclusion and exclusion criteria and study site contact information, see study NCT02175758 on ClinicalTrials.gov.
Gilead Sciences is also planning to start a second Phase 2 trial to evaluate a fixed-dose coformulation of sofosbuvir/ledipasvir, without ribavirin, in children and adolescents with chronic hepatitis C. This study — which is not yet recruiting — will also have multiple sites in the U.S. as well as the U.K., Australia, and New Zealand.
For complete information, see study NCT02249182 on ClinicalTrials.gov.
“The previous treatments were arduous. People would often start the treatment, but eventually drop out. This new therapy is a revolution that affects people around the world,” said Jeffrey Teckman of Saint Louis University, a pediatric gastroenterologist and principal investigator at one of the study sites for the sofosbuvir plus ribavirin trial. “A lot of times studies for children don’t get approved. The exciting part with our study is that the medication has already been tested in adults, and we are moving rapidly to test in children.”
ClinicalTrials.gov. Safety and Efficacy of Sofosbuvir + Ribavirin in Adolescents and Children With Genotype 2 or 3 Chronic HCV Infection. Clinical. Identifier NCT02175758.
ClinicalTrials. Gov. Safety and Efficacy of Ledipasvir/Sofosbuvir Fixed Dose Combination in Adolescents and Children With Chronic HCV-Infection. Identifier NCT02249182.
Saint Louis University. SLU Researcher to Study New Hepatitis C Medication in Children. Press release. September 19, 2014.