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Rollover Study of VX-770 in Cystic Fibrosis Subjects

Primary Purpose

Cystic Fibrosis

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Ivacaftor
Sponsored by
Vertex Pharmaceuticals Incorporated
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Cystic Fibrosis

Eligibility Criteria

6 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  1. Participants who have completed the assigned study treatment in Study 102 or Study 103
  2. Participants who are females of childbearing potential must have a negative urine pregnancy test on Day 1 (first dose of VX-770)
  3. Participants who are able to understand and comply with protocol requirements, restrictions, and instructions and likely to complete the study as planned, as judged by the investigator
  4. Participants of child bearing potential and who are sexually active must meet the contraception requirements
  5. Participants must sign the informed consent form (ICF), and where appropriate, assent must be obtained

Exclusion Criteria:

  1. Participants with a history of any illness or condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject
  2. Participants with a history of study treatment intolerance as observed in their previous VX-770 study that, in the opinion of the investigator, might pose an additional risk in administering study drug to the subject
  3. Participants who are pregnant, planning a pregnancy, breast-feeding, or not willing to follow contraception requirements
  4. Participants taking any inhibitors or inducers of Cytochrome P450 3A4 (CYP3A4), including certain herbal medications (for example, St. John's Wort) and grapefruit/grapefruit juice

Sites / Locations

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

VX-770

Arm Description

VX-770 (ivacaftor) 150 milligram (mg) tablet orally twice daily (q12h).

Outcomes

Primary Outcome Measures

Number of Participants With Non-Serious Adverse Events (AEs) and Serious Adverse Events (SAEs)
Adverse Event: any untoward medical occurrence in a participant during the study, including any unfavorable and unintended sign, symptom, or disease whether or not it was considered to be study drug related. This included any newly occurring event or previous condition that increased in severity or frequency after obtaining informed consent and assent (where applicable). SAE: medical event or condition, which resulted in any of following, regardless of its relationship to the study drug: death, life threatening adverse experience, in-patient hospitalization/prolonged hospitalization, persistent/significant disability/incapacity, congenital anomaly/birth defect, important medical event. Non-Serious AEs included all AEs except SAEs.

Secondary Outcome Measures

Annualized Rate of Decline From Study 105 Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 96
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Predicted FEV1 (for age, gender, and height) was calculated using the Knudson method. Baseline was defined as Study 105 Day 15.
Absolute Change From Study 105 Baseline in Percent Predicted FEV1 Through Week 96
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Predicted FEV1 (for age, gender, and height) was calculated using the Knudson method. Baseline was defined as the most recent measurement prior to intake of the first dose of study drug in Study 105. Absolute Change at Week 48 and Week 96 are reported.
Absolute Change From Study 105 Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 96
The CFQ-R is a validated patient-reported outcome measuring health-related quality of life for subjects with cystic fibrosis. Respiratory domain assessed respiratory symptoms (for example, coughing, congestion, wheezing), score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life. Absolute Change at Week 48 and Week 96 are reported.
Annualized Pulmonary Exacerbation Event Rate
Annualized event rate was calculated by regression with negative binomial distribution.
Annualized Duration of Pulmonary Exacerbation Events
Absolute Change From Study 105 Baseline in Weight Through Week 96
Weight is a measurement of nutritional status. Absolute change in weight, measured in kilograms (kg), at Week 48 and Week 96 are reported.

Full Information

First Posted
May 3, 2010
Last Updated
June 12, 2015
Sponsor
Vertex Pharmaceuticals Incorporated
Collaborators
Cystic Fibrosis Foundation
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1. Study Identification

Unique Protocol Identification Number
NCT01117012
Brief Title
Rollover Study of VX-770 in Cystic Fibrosis Subjects
Official Title
An Open-Label, Rollover Study to Evaluate the Long Term Safety and Efficacy of VX-770 in Subjects With Cystic Fibrosis
Study Type
Interventional

2. Study Status

Record Verification Date
June 2015
Overall Recruitment Status
Completed
Study Start Date
July 2010 (undefined)
Primary Completion Date
May 2014 (Actual)
Study Completion Date
May 2014 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Vertex Pharmaceuticals Incorporated
Collaborators
Cystic Fibrosis Foundation

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
The primary objective of the study was to evaluate the safety of long-term VX-770 treatment in participants with cystic fibrosis (CF). The secondary objective of the study was to evaluate the efficacy of long-term VX-770 treatment in subjects with CF.
Detailed Description
This open-label, rollover study of orally administered VX-770 was conducted in participants with CF to evaluate the safety and efficacy of long-term VX-770 treatment. Participants who were previously enrolled in Study 102 (VX08-770-102/NCT00909532) and Study 103 (VX08-770-103/NCT00909727), and met certain criteria were eligible to enroll in this study (Study 105/VX08-770-105/NCT01117012).

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cystic Fibrosis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
192 (Actual)

8. Arms, Groups, and Interventions

Arm Title
VX-770
Arm Type
Experimental
Arm Description
VX-770 (ivacaftor) 150 milligram (mg) tablet orally twice daily (q12h).
Intervention Type
Drug
Intervention Name(s)
Ivacaftor
Other Intervention Name(s)
VX-770
Primary Outcome Measure Information:
Title
Number of Participants With Non-Serious Adverse Events (AEs) and Serious Adverse Events (SAEs)
Description
Adverse Event: any untoward medical occurrence in a participant during the study, including any unfavorable and unintended sign, symptom, or disease whether or not it was considered to be study drug related. This included any newly occurring event or previous condition that increased in severity or frequency after obtaining informed consent and assent (where applicable). SAE: medical event or condition, which resulted in any of following, regardless of its relationship to the study drug: death, life threatening adverse experience, in-patient hospitalization/prolonged hospitalization, persistent/significant disability/incapacity, congenital anomaly/birth defect, important medical event. Non-Serious AEs included all AEs except SAEs.
Time Frame
Study 105: Day 1 up to Week 168
Secondary Outcome Measure Information:
Title
Annualized Rate of Decline From Study 105 Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 96
Description
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Predicted FEV1 (for age, gender, and height) was calculated using the Knudson method. Baseline was defined as Study 105 Day 15.
Time Frame
Study 105: Baseline through Week 96
Title
Absolute Change From Study 105 Baseline in Percent Predicted FEV1 Through Week 96
Description
FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Predicted FEV1 (for age, gender, and height) was calculated using the Knudson method. Baseline was defined as the most recent measurement prior to intake of the first dose of study drug in Study 105. Absolute Change at Week 48 and Week 96 are reported.
Time Frame
Study 105: Baseline through Week 96
Title
Absolute Change From Study 105 Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 96
Description
The CFQ-R is a validated patient-reported outcome measuring health-related quality of life for subjects with cystic fibrosis. Respiratory domain assessed respiratory symptoms (for example, coughing, congestion, wheezing), score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life. Absolute Change at Week 48 and Week 96 are reported.
Time Frame
Study 105: Baseline through Week 96
Title
Annualized Pulmonary Exacerbation Event Rate
Description
Annualized event rate was calculated by regression with negative binomial distribution.
Time Frame
Study 105: Day 1 through Week 96
Title
Annualized Duration of Pulmonary Exacerbation Events
Time Frame
Study 105: Day 1 through Week 96
Title
Absolute Change From Study 105 Baseline in Weight Through Week 96
Description
Weight is a measurement of nutritional status. Absolute change in weight, measured in kilograms (kg), at Week 48 and Week 96 are reported.
Time Frame
Study 105: Baseline through Week 96

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Participants who have completed the assigned study treatment in Study 102 or Study 103 Participants who are females of childbearing potential must have a negative urine pregnancy test on Day 1 (first dose of VX-770) Participants who are able to understand and comply with protocol requirements, restrictions, and instructions and likely to complete the study as planned, as judged by the investigator Participants of child bearing potential and who are sexually active must meet the contraception requirements Participants must sign the informed consent form (ICF), and where appropriate, assent must be obtained Exclusion Criteria: Participants with a history of any illness or condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject Participants with a history of study treatment intolerance as observed in their previous VX-770 study that, in the opinion of the investigator, might pose an additional risk in administering study drug to the subject Participants who are pregnant, planning a pregnancy, breast-feeding, or not willing to follow contraception requirements Participants taking any inhibitors or inducers of Cytochrome P450 3A4 (CYP3A4), including certain herbal medications (for example, St. John's Wort) and grapefruit/grapefruit juice
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Edward McKone, MD
Organizational Affiliation
St. Vincent's University Hospital
Official's Role
Principal Investigator
Facility Information:
City
Birmingham
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Oakland
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Morgantown
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Westmead
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Chermside
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Herston
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South Brisbane
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Parkville
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Victoria
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Subiaco
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Cork
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Belfast
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London
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United Kingdom

12. IPD Sharing Statement

Citations:
PubMed Identifier
25311995
Citation
McKone EF, Borowitz D, Drevinek P, Griese M, Konstan MW, Wainwright C, Ratjen F, Sermet-Gaudelus I, Plant B, Munck A, Jiang Y, Gilmartin G, Davies JC; VX08-770-105 (PERSIST) Study Group. Long-term safety and efficacy of ivacaftor in patients with cystic fibrosis who have the Gly551Asp-CFTR mutation: a phase 3, open-label extension study (PERSIST). Lancet Respir Med. 2014 Nov;2(11):902-910. doi: 10.1016/S2213-2600(14)70218-8. Epub 2014 Oct 9.
Results Reference
derived

Learn more about this trial

Rollover Study of VX-770 in Cystic Fibrosis Subjects

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