Efficacy and safety of 3-week response-guided triple direct-acting antiviral therapy for chronic hepatitis C infection: a phase 2, open-label, proof-of-concept study

Lancet Gastroenterol Hepatol. 2016 Oct;1(2):97-104. doi: 10.1016/S2468-1253(16)30015-2. Epub 2016 Jul 25.

Abstract

Background: To shorten the course of direct-acting antiviral agents for chronic hepatitis C virus (HCV) infection, we examined the antiviral efficacy and safety of 3 weeks of response-guided therapy with an NS3 protease inhibitor and dual NS5A inhibitor-NS5B nucleotide analogue.

Methods: In this open-label, phase 2a, single centre study, Chinese patients with chronic HCV genotype 1b infection without cirrhosis were randomly allocated by a computer program to one of three treatment groups (sofosbuvir, ledipasvir, and asunaprevir; sofosbuvir, daclatasvir, and simeprevir; or sofosbuvir, daclatasvir, and asunaprevir) until six patients in each group (1:1:1) achieved an ultrarapid virological response (plasma HCV RNA <500 IU/mL by day 2, measured by COBAS TaqMan HCV test, version 2.0). Patients with an ultrarapid virological response received 3 weeks of therapy. Patients who did not achieve an ultrarapid response were switched to sofosbuvir and ledipasvir for either 8 weeks or 12 weeks. The primary endpoint was the proportion of patients with a sustained virological response at 12 weeks (SVR12) after treatment completion, analysed in the intention-to-treat population. All patients who achieved an ultrarapid virological response were included in the safety analysis. This trial is registered with ClinicalTrials.gov, number NCT02470858.

Findings: Between April 5, 2015, and April 15, 2015, 26 eligible patients were recruited. 12 patients were assigned to sofosbuvir, ledipasvir, and asunaprevir; six to sofosbuvir, daclatasvir, and simeprevir; and eight to sofosbuvir, daclatasvir, and asunaprevir. Six patients in each group achieved an ultrarapid virological response (18 [69%]). All patients with an ultrarapid virological response who were given 3 weeks of triple therapy achieved SVR12. The most common adverse events were fatigue (one [17%] of six patients receiving sofosbuvir, ledipasvir, and asunaprevir; one [17%] of six patients receiving sofosbuvir, daclatasvir, and simeprevir; and two [33%] of six patients receiving sofosbuvir, daclatasvir, and asunaprevir) and headache (one [17%] patient in each group). No patients experienced any serious adverse events.

Interpretation: In this proof-of-concept study, all patients with chronic HCV without cirrhosis who achieved an ultrarapid virological response on triple direct-acting antiviral regimens by day 2 and received 3 weeks of treatment were cured, with excellent tolerability. By shortening the duration of therapy from the currently recommended 12 weeks to 3 weeks, we could drastically reduce the cost of therapy and the rate of adverse events. Further large-scale studies should be done to confirm our findings.

Funding: Center for AIDS Research, National Institutes of Health, US Department of Energy, National Center for Research Resources and the Office of Research Infrastructure Programs, Cheng Si-Yuan (China-International) Hepatitis Research Foundation, and Humanity and Health Medical Group.

Publication types

  • Clinical Trial, Phase II
  • Randomized Controlled Trial
  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, Non-P.H.S.

MeSH terms

  • Administration, Oral
  • Adult
  • Aged
  • Antiviral Agents / administration & dosage*
  • Antiviral Agents / therapeutic use
  • Benzimidazoles / administration & dosage
  • Benzimidazoles / therapeutic use
  • Carbamates
  • Drug Administration Schedule
  • Drug Therapy, Combination
  • Female
  • Fluorenes / administration & dosage
  • Fluorenes / therapeutic use
  • Follow-Up Studies
  • Hepatitis C, Chronic / drug therapy*
  • Hepatitis C, Chronic / virology
  • Humans
  • Imidazoles / administration & dosage
  • Imidazoles / therapeutic use
  • Intention to Treat Analysis
  • Isoquinolines / administration & dosage
  • Isoquinolines / therapeutic use
  • Male
  • Middle Aged
  • Proof of Concept Study
  • Pyrrolidines
  • Simeprevir / administration & dosage
  • Simeprevir / therapeutic use
  • Sofosbuvir / administration & dosage
  • Sofosbuvir / therapeutic use
  • Sulfonamides / administration & dosage
  • Sulfonamides / therapeutic use
  • Treatment Outcome
  • Valine / analogs & derivatives
  • Viral Load

Substances

  • Antiviral Agents
  • Benzimidazoles
  • Carbamates
  • Fluorenes
  • Imidazoles
  • Isoquinolines
  • Pyrrolidines
  • Sulfonamides
  • ledipasvir
  • Simeprevir
  • Valine
  • daclatasvir
  • asunaprevir
  • Sofosbuvir

Associated data

  • ClinicalTrials.gov/NCT02470858