Rollover Study of VX-770 in Cystic Fibrosis Subjects

Vertex Pharmaceuticals

Status and phase: Completed; Phase 3

Conditions: Cystic Fibrosis

Treatments: Drug: Ivacaftor

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT01117012

PERSIST

VX08-770-105

Details and patient eligibility

About

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.

Full 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).

Enrollment: 192 patients

Sex: All

Ages: 6+ years old

Volunteers: No 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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

---

192 participants in 1 patient group

VX-770

Experimental group

Description:

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

Treatment:

Drug: Ivacaftor