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 - Study Results

Study Type:	Interventional
Study Design:	Intervention Model: Single Group Assignment; Masking: Open Label; Primary Purpose: Treatment
Condition:	Hepatitis C
Interventions:	Drug: Telaprevir Drug: Peginterferon alfa-2b Drug: Ribavirin

Participant Flow

Recruitment Details

Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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Pre-Assignment Details

Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
The study was planned to be conducted in 2 parts (Part A and Part B), which would use separate groups of participants. However, the study was terminated early (12 weeks after last dose of study drug in Part A) and Part B was not conducted.

Reporting Groups

	Description
Cohort 1 (13-17 Years): Telaprevir + Peg-IFN-alfa-2b + RBV	Participants aged 13 to 17 years received telaprevir twice daily for 12 weeks either as a film coated tablet (15 milligram per kilogram [mg/kg] of body weight) or as a chewable tablet (14 mg/kg of body weight) in combination with pegylated interferon alfa 2b (Peg-IFN-alfa-2b) 60 microgram per meter square (mcg/m^2) subcutaneous injection weekly and ribavirin (RBV) 200 mg capsules or 40 milligram per milliliter (mg/mL) solution orally twice daily with a maximum dose of 1200 mg per day. Peg-IFN-alfa-2b and RBV were administered for 24 weeks in participants who achieved extended rapid virologic response (eRVR) or for 48 weeks in participants who did not achieve eRVR. eRVR was defined as undetectable hepatitis C virus ribonucleic acid (HCV RNA) levels at Week 4 and Week 12.
Cohort 2 (7-12 Years): Telaprevir + Peg-IFN-alfa-2b + RBV	Participants aged 7 to 12 years received telaprevir twice daily for 12 weeks as a chewable tablet (16 mg/kg of body weight) in combination with Peg-IFN-alfa-2b 60 mcg/m^2 subcutaneous injection weekly and RBV 200-mg capsules or 40 mg/mL solution orally twice daily with a maximum dose of 1200 mg per day. Peg-IFN-alfa-2b and RBV were administered for 24 weeks in participants who achieved eRVR or for 48 weeks in participants who did not achieve eRVR. eRVR was defined as undetectable HCV RNA levels at Week 4 and Week 12.
Cohort 3 (3-6 Years): Telaprevir + Peg-IFN-alfa-2b + RBV	Participants aged 3 to 6 years received telaprevir twice daily for 12 weeks as a chewable tablet (18 mg/kg of body weight) in combination with Peg-IFN-alfa-2b 60 mcg/m^2 subcutaneous injection weekly and RBV 200-mg capsules or 40 mg/mL solution orally twice daily with a maximum dose of 1200 mg per day. Peg-IFN-alfa-2b and RBV were administered for 24 weeks in participants who achieved eRVR or for 48 weeks in participants who did not achieve eRVR. eRVR was defined as undetectable HCV RNA levels at Week 4 and Week 12.

Participant Flow: Overall Study

	Cohort 1 (13-17 Years): Telaprevir + Peg-IFN-alfa-2b + RBV	Cohort 2 (7-12 Years): Telaprevir + Peg-IFN-alfa-2b + RBV	Cohort 3 (3-6 Years): Telaprevir + Peg-IFN-alfa-2b + RBV
STARTED	13	19	10
COMPLETED	13	18	8
NOT COMPLETED	0	1	2
Lost to Follow-up	0	1	0
Withdrawal by Subject	0	0	1
Unspecified	0	0	1

Baseline Characteristics

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Population Description

Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
Full analysis set (FAS) included all enrolled participants who received at least 1 dose of study drug.

Reporting Groups

	Description
Cohort 1 (13-17 Years): Telaprevir + Peg-IFN-alfa-2b + RBV	Participants aged 13 to 17 years received telaprevir twice daily for 12 weeks either as a film coated tablet (15 mg/kg of body weight) or as a chewable tablet (14 mg/kg of body weight) in combination with Peg-IFN-alfa-2b 60 mcg/m^2 subcutaneous injection weekly and RBV 200 mg capsules or 40 mg/mL solution orally twice daily with a maximum dose of 1200 mg per day. Peg-IFN-alfa-2b and RBV were administered for 24 weeks in participants who achieved eRVR or for 48 weeks in participants who did not achieve eRVR. eRVR was defined as undetectable HCV RNA levels at Week 4 and Week 12.
Cohort 2 (7-12 Years): Telaprevir + Peg-IFN-alfa-2b + RBV	Participants aged 7 to 12 years received telaprevir twice daily for 12 weeks as a chewable tablet (16 mg/kg of body weight) in combination with Peg-IFN-alfa-2b 60 mcg/m^2 subcutaneous injection weekly and RBV 200-mg capsules or 40 mg/mL solution orally twice daily with a maximum dose of 1200 mg per day. Peg-IFN-alfa-2b and RBV were administered for 24 weeks in participants who achieved eRVR or for 48 weeks in participants who did not achieve eRVR. eRVR was defined as undetectable HCV RNA levels at Week 4 and Week 12.
Cohort 3 (3-6 Years): Telaprevir + Peg-IFN-alfa-2b + RBV	Participants aged 3 to 6 years received telaprevir twice daily for 12 weeks as a chewable tablet (18 mg/kg of body weight) in combination with Peg-IFN-alfa-2b 60 mcg/m^2 subcutaneous injection weekly and RBV 200-mg capsules or 40 mg/mL solution orally twice daily with a maximum dose of 1200 mg per day. Peg-IFN-alfa-2b and RBV were administered for 24 weeks in participants who achieved eRVR or for 48 weeks in participants who did not achieve eRVR. eRVR was defined as undetectable HCV RNA levels at Week 4 and Week 12.
Total	Total of all reporting groups

Baseline Measures

	Cohort 1 (13-17 Years): Telaprevir + Peg-IFN-alfa-2b + RBV	Cohort 2 (7-12 Years): Telaprevir + Peg-IFN-alfa-2b + RBV	Cohort 3 (3-6 Years): Telaprevir + Peg-IFN-alfa-2b + RBV	Total
Overall Participants Analyzed [Units: Participants]	13	19	10	42

Age [Units: Years] Mean (Standard Deviation)	14.9 (2.06)	10.2 (1.57)	4.9 (0.88)	10.4 (4.05)

Gender [Units: Participants]
Female	11	13	4	28
Male	2	6	6	14

Outcome Measures

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1. Primary:

Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: Baseline up to Week 52 ]

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2. Secondary:

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) ]

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3. Secondary:

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) ]

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4. Secondary:

Percentage of Participants With Rapid Virologic Response (RVR) [ Time Frame: Week 4 ]

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5. Secondary:

Percentage of Participants With Extended Rapid Virologic Response (eRVR) [ Time Frame: Week 4 and Week 12 ]

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6. Secondary:

Percentage of Participants With Undetectable HCV RNA at Week 12 [ Time Frame: Week 12 ]

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7. Secondary:

Percentage of Participants With On-treatment Virologic Failure [ Time Frame: Baseline up to Week 48 ]

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8. Secondary:

Percentage of Participants With Virologic Relapse [ Time Frame: 12 weeks after planned EOT (up to Week 60) ]

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9. Secondary:

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) ]

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10. Secondary:

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 ]

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11. Secondary:

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 ]

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12. Secondary:

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 ]

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13. Secondary:

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 ]

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Serious Adverse Events

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Other Adverse Events

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Limitations and Caveats

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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
Study was terminated early because it was determined that telaprevir/Peg-IFN/RBV regimen did not present a meaningful therapeutic treatment over existing interferon-free regimens and was unlikely to be used for participants aged 3 to 17 years.

More Information

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Certain Agreements:

Principal Investigators are NOT employed by the organization sponsoring the study.

There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

The agreement is:

	The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
	The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
	Other disclosure agreement that restricts the right of the PI to discuss or publish trial results after the trial is completed. Restriction Description: PI is free to publish results of the study after (1) the first multi-center publication, (2) if the sponsor elects not to publish the results, or (3) 18 months after close of the study, whichever occurs first. Proposed publications are to be submitted to the sponsor for review and comment for a period of at least 45 days (which may be extended under certain circumstances related to protection of intellectual property); the sponsor cannot require changes to the proposed publications.

Results Point of Contact:

Name/Title: Medical Monitor
Organization: Vertex Pharmaceuticals Incorporated
phone: 617-341-6777
e-mail: medicalinfo@vrtx.com

Responsible Party:	Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier:	NCT01701063 History of Changes
Other Study ID Numbers:	VX11-950-118
Study First Received:	September 27, 2012
Results First Received:	April 18, 2016
Last Updated:	June 7, 2016