Hepatitis CPhase 1/2NCT01701063
An Open-Label Study of the Effect of Telaprevir in Combination With Peginterferon Alfa-2b and Ribavirin in Pediatric Subjects Infected With Hepatitis C Virus
A Phase 1/2 study testing Telaprevir, Peginterferon alfa-2b and Ribavirin in people with Hepatitis C, run by Vertex Pharmaceuticals Incorporated at 23 sites in 6 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 3 to 17 years
- Conditions the study is for
- Hepatitis C
- What is being tested?
- Telaprevir, Peginterferon alfa-2b and Ribavirin
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 42 participants
- Where
- 23 sitesin 6 countries
- Study dates
- Jan 2013to Apr 2015
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 3 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
23 sites in 6 countries.
23 sites in 6 countries
United States13 sites
Study site
Baltimore, Maryland
Study site
Denver, Colorado
Study site
Gainesville, Florida
Study site
Houston, Texas
Study site
Indianapolis, Indiana
Study site
Miami, Florida
Study site
New York, New York
Study site
Philadelphia, Pennsylvania
Study site
Phoenix, Arizona
Study site
San Francisco, California
Study site
Seattle, Washington
Study site
The Bronx, New York
Study site
Washington D.C., District of Columbia
United Kingdom3 sites
Study site
Birmingham
Study site
Leeds
Study site
London
About this study
Official title: 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
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.
- Conditions
- Hepatitis C
- Being tested
- Telaprevir, Peginterferon alfa-2b, Ribavirin
- Medical subject headings
- Hepatitis C
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TelaprevirDrug
100- and 250-mg chewable tablets or 375-mg film-coated tablets for oral administration
Peginterferon alfa-2bDrug
Also called PegIntron®
50 μg/0.5 mL, 80 μg/0.5 mL, 120 μg/0.5 mL, or 150 μg/0.5 mL for subcutaneous (SC) injection
RibavirinDrug
Also called Rebetol®
200-mg capsules or 40-mg/mL solution for oral administration
| Study group | Role | What they receive |
|---|---|---|
| Treatment-Naive or Prior Partial/Null Response | Experimental | telaprevir + Peginterferon alfa-2b + Ribavirin Receives: Drug: Telaprevir; Drug: Peginterferon alfa-2b; Drug: Ribavirin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
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.
Measured: Baseline up to Week 52
Secondary outcomes (12)
Percentage of Participants With Sustained Viral Response 12 Weeks After Last Planned Dose of Study Drug (SVR12)
SVR12 was defined as an undetectable Hepatitis C Virus (HCV) Ribonucleic Acid (RNA) Levels (less than [<] lower limit of quantification) at 12 weeks after last planned dose of study drug. The plasma HCV RNA level was measured using Roche COBAS TaqMan HCV/High Pure System (HPS) RNA assay version 2.0. The lower limit of quantification was 25 international units per milliliter (IU/mL).
Measured: 12 weeks after last planned dose of study drug (up to Week 60)
Percentage of Participants With Sustained Viral Response 24 Weeks After Last Planned Dose of Study Drug (SVR24)
SVR24 was defined as an undetectable HCV RNA Levels (< lower limit of quantification) at 24 weeks after last planned dose of study drug. The plasma HCV RNA level was measured using Roche COBAS TaqMan HCV/HPS RNA assay version 2.0. The lower limit of quantification was 25 IU/mL.
Measured: 24 weeks after last planned dose of study drug (up to Week 72)
Percentage of Participants With Rapid Virologic Response (RVR)
The plasma HCV RNA level was measured using Roche COBAS TaqMan HCV/HPS RNA assay version 2.0. The lower limit of quantification was 25 IU/mL. RVR was defined as an undetectable HCV RNA (<lower limit of quantification) 4 weeks after the start of study treatment.
Measured: Week 4
Percentage of Participants With Extended Rapid Virologic Response (eRVR)
The plasma HCV RNA level was measured using Roche COBAS TaqMan HCV/HPS RNA assay version 2.0. The lower limit of quantification was 25 IU/mL. eRVR was defined as an undetectable HCV RNA (<lower limit of quantification) at both 4 weeks and 12 weeks after the start of study treatment.
Measured: Week 4 and Week 12
Percentage of Participants With Undetectable HCV RNA at Week 12
The plasma HCV RNA level was measured using Roche COBAS TaqMan HCV/HPS RNA assay version 2.0. The lower limit of quantification was 25 IU/mL.
Measured: Week 12
Percentage of Participants With On-treatment Virologic Failure
On treatment virologic failure was defined as meeting any futility rule or completing assigned treatment duration and having detectable HCV RNA at EOT. The plasma HCV RNA level was measured using Roche COBAS TaqMan HCV/HPS RNA assay Version 2.0. The lower limit of quantification was 25 IU/mL. Futility rules: 1) HCV RNA >1000 IU/mL at Week 4; 2) HCV RNA >1000 IU/mL at Week 12; 3) Detectable HCV RNA after Week 12 to end of treatment.
Measured: Baseline up to Week 48
Percentage of Participants With Virologic Relapse
The plasma HCV RNA level was measured using Roche COBAS TaqMan HCV/HPS RNA assay Version 2.0. The lower limit of quantification was 25 IU/mL. Viral relapse was defined as having detectable HCV at follow-up in participants who had HCV RNA less than (<) lower limit of quantification (LLOQ) at planned EOT.
Measured: 12 weeks after planned EOT (up to Week 60)
Number of Participants With Telaprevir Resistant HCV Variant at Non-Structural Viral Protein 3-4A (NS3-4A) Region
Sequence analysis of the HCV NS3-4A region was performed to monitor telaprevir-resistant variants. HCV RNA was isolated from the plasma, amplified by reverse transcription-polymerase chain reaction (RT-PCR), and sequenced (sequencing assay limit of detection HCV RNA >=1000 IU/mL). Results of this outcome measure were to be reported for overall participants instead of by age.
Measured: Baseline, On treatment (up to Week 48)
Maximum Plasma Concentration (Cmax) of Telaprevir
Cmax was measured for telaprevir only.
Measured: 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
Tmax was measured for telaprevir only.
Measured: 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
Area Under the Plasma Concentration Versus Time Curve (AUC) of Telaprevir
AUC was measured for telaprevir only. AUC 0-t last was defined as the area under the concentration-time curve from the time of dosing to the last measurable concentration. AUC 0-12 hour (AUC 0-12h) was calculated by respecifying predose concentrations as 12 hour concentrations. AUC 0-24h was calculated as AUC 0-12h multiplied by 2. Dose adjusted AUC (AUC 0-24h_Adj) was calculated by multiplying AUC 0-24h by the dose adjustment factor to obtain projected exposures in participants who were misdosed. Data were presented for AUC 0-t last, AUC 0-12h, AUC 0-24h, AUC 0-24h_Adj.
Measured: 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
T1/2 was defined as the time required for the concentration or amount of drug in the body to be reduced by one-half.
Measured: 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
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Oct 2012 (estimated)
- Study startJan 2013
- Primary completionApr 2015
- Study completionApr 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Hepatitis CStudies of the same condition, open and under way.
- More trials run by Vertex Pharmaceuticals IncorporatedThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 42 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vertex Pharmaceuticals Incorporated
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Janssen Pharmaceuticals
- Responsible party
- Sponsor
- Investigators
- Medical Monitor, Study director, Vertex Pharmaceuticals Incorporated
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01701063
- Sponsor's protocol ID
- VX11-950-118
- Status last verified
- Apr 2016
- First submitted
- 27 Sep 2012
- Last updated
- 20 Jul 2016
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 20 Jul 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT01701063
- Acronym / sponsor ID
- VX11-950-118
- Conditions
- Hepatitis C
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Terminated
- Enrolment
- 42 participants (actual)
- Lead sponsor
- Vertex Pharmaceuticals IncorporatedIndustry (pharma, biotech, devices)
- Collaborators
- Janssen Pharmaceuticals
- Study start
- Jan 2013
- Primary completion
- Apr 2015 (actual)
- Study completion
- Apr 2015 (actual)
- First posted
- 4 Oct 2012 (estimated)
- Results
- Posted 20 Jul 2016
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment-Naive or Prior Partial/Null Response | Experimental | Drug: Telaprevir, Drug: Peginterferon alfa-2b, Drug: Ribavirintelaprevir + Peginterferon alfa-2b + Ribavirin |
Who may qualify
- Ages
- 3 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (8 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Telaprevir100- and 250-mg chewable tablets or 375-mg film-coated tablets for oral administration | Drug | Treatment-Naive or Prior Partial/Null Response |
| Peginterferon alfa-2bAlso: PegIntron®50 μg/0.5 mL, 80 μg/0.5 mL, 120 μg/0.5 mL, or 150 μg/0.5 mL for subcutaneous (SC) injection | Drug | Treatment-Naive or Prior Partial/Null Response |
| RibavirinAlso: Rebetol®200-mg capsules or 40-mg/mL solution for oral administration | Drug | Treatment-Naive or Prior Partial/Null Response |
Primary outcome
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.
12 secondary outcomes
Percentage of Participants With Sustained Viral Response 12 Weeks After Last Planned Dose of Study Drug (SVR12)
Time frame: 12 weeks after last planned dose of study drug (up to Week 60)
Percentage of Participants With Sustained Viral Response 24 Weeks After Last Planned Dose of Study Drug (SVR24)
Time frame: 24 weeks after last planned dose of study drug (up to Week 72)
Percentage of Participants With Rapid Virologic Response (RVR)
Time frame: Week 4
Percentage of Participants With Extended Rapid Virologic Response (eRVR)
Time frame: Week 4 and Week 12
Percentage of Participants With Undetectable HCV RNA at Week 12
Time frame: Week 12
Percentage of Participants With On-treatment Virologic Failure
Time frame: Baseline up to Week 48
Percentage of Participants With Virologic Relapse
Time frame: 12 weeks after planned EOT (up to Week 60)
Number of Participants With Telaprevir Resistant HCV Variant at Non-Structural Viral Protein 3-4A (NS3-4A) Region
Time frame: Baseline, On treatment (up to Week 48)
Maximum Plasma Concentration (Cmax) of Telaprevir
Time frame: 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
Time frame: 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
Area Under the Plasma Concentration Versus Time Curve (AUC) of Telaprevir
Time frame: 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
Time frame: 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
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Jul 2016.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Monitor study director
Vertex Pharmaceuticals Incorporated
23 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Telaprevir, Peginterferon alfa-2b and Ribavirin in people with Hepatitis C, run by Vertex Pharmaceuticals Incorporated at 23 sites in 6 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 20 Jul 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 23 sites in United States, Italy, United Kingdom, Spain, Belgium and 1 more country. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 20 Jul 2016). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) (time frame: Baseline up to Week 52). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Jul 2016.