Rollover Study of VX-770 in Cystic Fibrosis Subjects

NCT01117012 | Completed Phase 3 Results DMC

• 2 other identifiers: VX08-770-105PERSIST
• Study Type: interventional
• Target: 192
• Locations: 8 countries
• Sites: 58

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.

Conditions

Cystic Fibrosis

Outcome Measures

Primary Outcomes (1)

• 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.

  Study 105: Day 1 up to Week 168

Secondary Outcomes (6)

• Annualized Rate of Decline From Study 105 Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 96

  Study 105: Baseline through Week 96

• Absolute Change From Study 105 Baseline in Percent Predicted FEV1 Through Week 96

  Study 105: Baseline through Week 96

• Absolute Change From Study 105 Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 96

  Study 105: Baseline through Week 96

• Annualized Pulmonary Exacerbation Event Rate

  Study 105: Day 1 through Week 96

• Annualized Duration of Pulmonary Exacerbation Events

  Study 105: Day 1 through Week 96

Study Arms (1)

VX-770

EXPERIMENTAL

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

Drug: Ivacaftor

Interventions

Ivacaftor DRUG

Also known as: VX-770

VX-770

Eligibility Criteria

• Age: 6 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Vertex Pharmaceuticals Incorporated: lead
• Cystic Fibrosis Foundation: collaborator

Study Sites (58)

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Birmingham, Alabama, United States

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Oakland, California, United States

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Palo Alto, California, United States

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San Diego, California, United States

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Denver, Colorado, United States

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Atlanta, Georgia, United States

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Boise, Idaho, United States

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Chicago, Illinois, United States

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Indianapolis, Indiana, United States

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Iowa City, Iowa, United States

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Baltimore, Maryland, United States

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Boston, Massachusetts, United States

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Ann Arbor, Michigan, United States

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Detroit, Michigan, United States

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Minneapolis, Minnesota, United States

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Kansas City, Missouri, United States

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St Louis, Missouri, United States

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Omaha, Nebraska, United States

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Long Branch, New Jersey, United States

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Buffalo, New York, United States

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New Hyde Park, New York, United States

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Syracuse, New York, United States

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Cincinnati, Ohio, United States

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Cleveland, Ohio, United States

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Columbus, Ohio, United States

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Toledo, Ohio, United States

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Portland, Oregon, United States

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Hershey, Pennsylvania, United States

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Philadelphia, Pennsylvania, United States

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Pittsburgh, Pennsylvania, United States

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Knoxville, Tennessee, United States

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Nashville, Tennessee, United States

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Salt Lake City, Utah, United States

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Chrlottesville, Virginia, United States

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Seattle, Washington, United States

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Morgantown, West Virginia, United States

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Westmead, New South Wales, Australia

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Chermside, Queensland, Australia

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Herston, Queensland, Australia

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South Brisbane, Queensland, Australia

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Parkville, Victoria, Australia

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Nedlands, Western Australia, Australia

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Subiaco, Western Australia, Australia

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Vancouver, British Columbia, Canada

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Halifax, Nova Scotia, Canada

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Toronto, Ontario, Canada

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Montreal, Quebec, Canada

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Prague, Czechia

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Paris, France

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Roscoff, France

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Erlangen, Germany

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Jena, Germany

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Munich, Germany

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Würzburg, Germany

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Cork, Ireland

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Dublin, Ireland

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Belfast, Northern Ireland, United Kingdom

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London, United Kingdom

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Related Publications (1)

• 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.

  PMID: 25311995 DERIVED

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

ivacaftor

Condition Hierarchy (Ancestors)

Pancreatic Diseases; Digestive System Diseases; Lung Diseases; Respiratory Tract Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infant, Newborn, Diseases

Results Point of Contact

• Title: Medical Monitor
• Organization: Vertex

medicalinfo@vrtx.com

617-444-6777

Study Officials

• Edward McKone, MD

  St Vincent's University Hospital, Ireland

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: GT60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 3, 2010
• First Posted: May 5, 2010
• Study Start: July 1, 2010
• Primary Completion: May 1, 2014
• Study Completion: May 1, 2014
• Last Updated: July 7, 2015
• Results First Posted: July 7, 2015

Record last verified: 2015-06

Locations

CA (4); CZ (1); IE (2); GB (2); DE (4); AU (7); FR (2); US (36)