A Two-Part, Open-Label, Single-Arm Phase 1/2 Study of Safety, Pharmacokinetics, and Efficacy of Telaprevir in Combination With Peginterferon Alfa-2b and Ribavirin in Pediatric Subjects Aged 3 to 17 Infected With Genotype 1 Hepatitis C Virus
Overview
- Phase
- Phase 1
- Intervention
- Ribavirin
- Conditions
- Hepatitis C
- Sponsor
- Vertex Pharmaceuticals Incorporated
- Enrollment
- 42
- Primary Endpoint
- Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
- Status
- Terminated
- Last Updated
- 9 years ago
Overview
Brief Summary
The purpose of this study is to assess the safety, efficacy, and pharmacokinetics in a carefully monitored cohort of pediatric subjects infected with hepatitis C virus (HCV) on a telaprevir-based regimen in Part A and with dose adjustments if needed before Part B.
Investigators
Eligibility Criteria
Inclusion Criteria
- •Males or females ages 3 to 17 years of age
- •Chronic hepatitis C
- •Hepatitis C virus genotype 1a or b at the Screening Visit
- •Subject is judged to be in good health (besides HCV infection) in the opinion of the investigator.
- •Signed informed consent form (ICF), and where appropriate, signed Assent Form
Exclusion Criteria
- •History of or prior evidence of a medical condition associated with chronic liver disease other than HCV
- •Body weight \<15 kg or \>90 kg
- •Prior evidence of hepatic decompensation
- •Contraindications to pegylated interferon/ribavirin (Peg-IFN/RBV)
- •History or other evidence of severe retinopathy or clinically significant ophthalmological disorder
- •History of non-genotype 1 HCV
- •Participation in investigational drug study as described in Study Protocol
- •Use of prohibited drugs within 7 days or 5 half-lives before the first dose of study drug
Arms & Interventions
Treatment-Naive or Prior Partial/Null Response
telaprevir + Peginterferon alfa-2b + Ribavirin
Intervention: Ribavirin
Treatment-Naive or Prior Partial/Null Response
telaprevir + Peginterferon alfa-2b + Ribavirin
Intervention: Telaprevir
Treatment-Naive or Prior Partial/Null Response
telaprevir + Peginterferon alfa-2b + Ribavirin
Intervention: Peginterferon alfa-2b
Outcomes
Primary Outcomes
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time Frame: Baseline up to Week 52
AE: any adverse change from the participant's baseline (pre-treatment) condition, including any adverse experience, abnormal recording or clinical laboratory assessment value which occurs during the course of the study, whether it is considered related to the study drug or not. An adverse event includes any newly occurring event or previous condition that has increased in severity or frequency since the administration of study drug. SAE: medical event or condition, which falls into any of the following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, in-patient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. "Study drug" includes all investigational agents administered during the course of the study.
Secondary Outcomes
- Percentage of Participants With Sustained Viral Response 24 Weeks After Last Planned Dose of Study Drug (SVR24)(24 weeks after last planned dose of study drug (up to Week 72))
- Percentage of Participants With Rapid Virologic Response (RVR)(Week 4)
- Percentage of Participants With Sustained Viral Response 12 Weeks After Last Planned Dose of Study Drug (SVR12)(12 weeks after last planned dose of study drug (up to Week 60))
- Percentage of Participants With Extended Rapid Virologic Response (eRVR)(Week 4 and Week 12)
- Percentage of Participants With Undetectable HCV RNA at Week 12(Week 12)
- Percentage of Participants With On-treatment Virologic Failure(Baseline up to Week 48)
- Number of Participants With Telaprevir Resistant HCV Variant at Non-Structural Viral Protein 3-4A (NS3-4A) Region(Baseline, On treatment (up to Week 48))
- Maximum Plasma Concentration (Cmax) of Telaprevir(Cohort 1: Pre-dose and 0.5, 1.0, 2.0, 3.0, 4.0, 5.0, 6.0, and 8.0 hours post-dose on Day 7, Cohort 2: Pre-dose and 0.5, 2.0, 4.0, 5.0, 6.0, and 8.0 hours post-dose on Day 7, Cohort 3: Pre-dose and 1.5, 4.0, and 8.0 hours post-dose on Day 7)
- Time to Reach Maximum Plasma Concentration (Tmax) of Telaprevir(Cohort 1: Pre-dose and 0.5, 1.0, 2.0, 3.0, 4.0, 5.0, 6.0, and 8.0 hours post-dose on Day 7, Cohort 2: Pre-dose and 0.5, 2.0, 4.0, 5.0, 6.0, and 8.0 hours post-dose on Day 7, Cohort 3: Pre-dose and 1.5, 4.0, and 8.0 hours post-dose on Day 7)
- Percentage of Participants With Virologic Relapse(12 weeks after planned EOT (up to Week 60))
- Area Under the Plasma Concentration Versus Time Curve (AUC) of Telaprevir(Cohort 1: Pre-dose and 0.5, 1.0, 2.0, 3.0, 4.0, 5.0, 6.0, and 8.0 hours post-dose on Day 7, Cohort 2: Pre-dose and 0.5, 2.0, 4.0, 5.0, 6.0, and 8.0 hours post-dose on Day 7, Cohort 3: Pre-dose and 1.5, 4.0, and 8.0 hours post-dose on Day 7)
- Elimination Half-Life (T1/2) of Telaprevir(Cohort 1: Pre-dose and 0.5, 1.0, 2.0, 3.0, 4.0, 5.0, 6.0, and 8.0 hours post-dose on Day 7, Cohort 2: Pre-dose and 0.5, 2.0, 4.0, 5.0, 6.0, and 8.0 hours post-dose on Day 7, Cohort 3: Pre-dose and 1.5, 4.0, and 8.0 hours post-dose on Day 7)