Clinical trial NCT01117012: 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
Overall status: COMPLETED
Phase: PHASE3
Conditions: Cystic Fibrosis
Lead sponsor: Vertex Pharmaceuticals Incorporated
Sponsor class: INDUSTRY
Collaborators: name: Cystic Fibrosis Foundation
First posted: 2010-05-05
Enrollment: 192
Sex: ALL
Minimum age (years): 6
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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).

Eligibility criteria: 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

Primary outcomes:
measure: Number of Participants With Non-Serious Adverse Events (AEs) and Serious Adverse Events (SAEs)
timeFrame: Study 105: Day 1 up to Week 168
Secondary outcomes:
measure: Annualized Rate of Decline From Study 105 Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 96
timeFrame: Study 105: Baseline through Week 96
measure: Absolute Change From Study 105 Baseline in Percent Predicted FEV1 Through Week 96
timeFrame: Study 105: Baseline through Week 96
measure: Absolute Change From Study 105 Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 96
timeFrame: Study 105: Baseline through Week 96
measure: Annualized Pulmonary Exacerbation Event Rate
timeFrame: Study 105: Day 1 through Week 96
measure: Annualized Duration of Pulmonary Exacerbation Events
timeFrame: Study 105: Day 1 through Week 96
measure: Absolute Change From Study 105 Baseline in Weight Through Week 96
timeFrame: Study 105: Baseline through Week 96
Other outcomes:
Locations:
city: Birmingham
country: United States
state: Alabama
city: Oakland
country: United States
state: California
city: Palo Alto
country: United States
state: California
city: San Diego
country: United States
state: California
city: Denver
country: United States
state: Colorado
city: Atlanta
country: United States
state: Georgia
city: Boise
country: United States
state: Idaho
city: Chicago
country: United States
state: Illinois
city: Indianapolis
country: United States
state: Indiana
city: Iowa City
country: United States
state: Iowa
city: Baltimore
country: United States
state: Maryland
city: Boston
country: United States
state: Massachusetts
city: Ann Arbor
country: United States
state: Michigan
city: Detroit
country: United States
state: Michigan
city: Minneapolis
country: United States
state: Minnesota
city: Kansas City
country: United States
state: Missouri
city: St. Louis
country: United States
state: Missouri
city: Omaha
country: United States
state: Nebraska
city: Long Branch
country: United States
state: New Jersey
city: Buffalo
country: United States
state: New York
city: New Hyde Park
country: United States
state: New York
city: Syracuse
country: United States
state: New York
city: Cincinnati
country: United States
state: Ohio
city: Cleveland
country: United States
state: Ohio
city: Columbus
country: United States
state: Ohio
city: Toledo
country: United States
state: Ohio
city: Portland
country: United States
state: Oregon
city: Hershey
country: United States
state: Pennsylvania
city: Philadelphia
country: United States
state: Pennsylvania
city: Pittsburgh
country: United States
state: Pennsylvania
city: Knoxville
country: United States
state: Tennessee
city: Nashville
country: United States
state: Tennessee
city: Salt Lake City
country: United States
state: Utah
city: Chrlottesville
country: United States
state: Virginia
city: Seattle
country: United States
state: Washington
city: Morgantown
country: United States
state: West Virginia
city: Westmead
country: Australia
state: New South Wales
city: Chermside
country: Australia
state: Queensland
city: Herston
country: Australia
state: Queensland
city: South Brisbane
country: Australia
state: Queensland
city: Parkville
country: Australia
state: Victoria
city: Nedlands
country: Australia
state: Western Australia
city: Subiaco
country: Australia
state: Western Australia
city: Vancouver
country: Canada
state: British Columbia
city: Halifax
country: Canada
state: Nova Scotia
city: Toronto
country: Canada
state: Ontario
city: Montreal
country: Canada
state: Quebec
city: Prague
country: Czech Republic
city: Paris
country: France
city: Roscoff
country: France
city: Erlangen
country: Germany
city: Jena
country: Germany
city: Munich
country: Germany
city: Wurzburg
country: Germany
city: Cork
country: Ireland
city: Dublin
country: Ireland
city: Belfast
country: United Kingdom
state: Northern Ireland
city: London
country: United Kingdom
Study officials:
affiliation: St Vincent's University Hospital
name: Edward McKone, MD
role: PRINCIPAL_INVESTIGATOR
References:
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.