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Drug & research development » Clinical trials » NCT01117012

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

Clinical Trials gov number: NCT01117012

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Purpose: To enrol subjects with CF who have completed Study VX08-770-102 (Study 102) and Study VX08-770-103 (Study 103) to further evaluate the safety and efficacy of long-term ivacaftor treatment. Patients who were previously enrolled in Study 102 and Study 103; and have met certain criteria are eligible to enrol in this study. Study VX08-770-105 (Study 105) also offers an opportunity for subjects who received placebo in Study 102 and Study 103 to receive ivacaftor treatment.

Phase: 3

Type: Interventional, open-label, single group assignment

Study sponsor: Vertex Pharmaceuticals Incorporated; Cystic Fibrosis Foundation Therapeutics

  • Have completed the assigned study treatment Study 102 or Study 103
  • Females of childbearing potential must have a negative urine pregnancy test on Day 1 (first dose of ivacaftor)
  • Able to understand and comply with protocol requirements, restrictions, and instructions and likely to complete the study as planned, as judged by the investigator
  • Subjects with child bearing potential and who are sexually active must meet the contraception requirements
  • Subjects must sign the informed consent form (ICF), and where appropriate, assent must be obtained

Intervention
Ivacaftor

Geographical Location
Australia, Canada, Czech Republic, France, Germany, Ireland, United Kingdom, United States

Number of Participants
101-1000 (≥6 years)

Primary Endpoint

Long-term safety of ivacaftor at 96 weeks

Secondary Endpoint

At 96 weeks

  • Rate of decline in percent predicted forced expiratory volume in 1 second (FEV1)
  • Absolute change from Day 1 of Study VX08-770-105 (Study 105) in FEV1
  • Absolute change from Day 1 of previous VX-770 study in FEV1
  • Change from Day 1 of Study 105 in Cystic Fibrosis Questionnaire-Revised (CFQ-R)
  • Change from Day 1 of previous VX-770 study in CFQ-R
  • Pulmonary exacerbations

– Rate of change in weight

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  • Experimental arm: VX-770 150 mg tablet, oral use, twice daily every 12 hours (q12h)

Link:

http://www.ncbi.nlm.nih.gov/pubmed/25311995

Reference:

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):902-10. doi: 10.1016/S2213-2600(14)70218-8. Epub 2014 Oct 9. PubMed PMID: 25311995.

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