Clinical Research Details

Descriptive Information
Sofosbuvir/Valpatasvir for Hepatitis C Genotype 1-6 in Patients with Transfusion-Dependent Thalassemia: An Open-Label Trial

Ala Sharara


Clinical Research - Clinical Trials (phase 0, 1,2 3 & 4)  

Phase 4  

  • Ali Taher
  • Louma Basma Rustam
  • Gilead Sciences
Louma Rustam
Extension: 5909
Conditions and Keywords
Hepatitis C Virus,Thalassemia Major
Hepatitis C Virus,Thalassemia Major,Transfusion Dependent Thalassemia,Genotype 1,Genotype 4,sofosbuvir/Velpatasvir
Study Design
Safety and/or Efficacy Study
Open Label
Single Group
N/A: Single arm study
N/A: Not Applicable
Eligibility and IRB
Min: 18

This is an open label prospective observational randomized controlled trial taking place at the American University of Beirut Medical Center aimed at assessing the efficacy of Sofosbuvir/Velpatasvir in transfusion-dependent thalassemia patients suffering from HCV Genotype 1-6. 

  1. Sustained Viral Response following 12 weeks of therapy (primary objective)
  2. Assessment of transfusion requirements (secondary objective)
  3. Adverse events (secondary objective)
  4. Efficacy in treatment naiive vs relapsers vs null responders (secondary objective)
  5. Efficacy in patients with advanced fibrosis/cirrhosis vs F1, F2 by elastography (secondary objective)

10 adult patients suffering from transfusion dependent thalassemia major from the American University of Beirut Medical Center will be enrolled in this open label trial. Patients will be enrolled at the private clinics or endoscopy unit of the AUBMC

Patients who have both HCV genotype 1 or 4 and transfusion dependent thalassemia major will be enrolled in this study. Consecutive patients will be introduced to the study, consented, and supplied with 3 months worth of Sofosbuvir/Ledipasvir.

  1. Age 18 or older
  2. transfusion dependent thalassemia
  3. HCV genotype 1-6
  4. no evidence of HCC on ultrasound
  5. adequate iron chelation therapy
  6. compensated liver disease

  1. Age younger than 18
  2. non transfusion dependent thalassemia
  3. HCV genotype other than 1-6
  4. HCC on ultrasound
  5. inadequate iron chelation therapy
  6. uncompensated liver disease