Study of Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older With the G551D Mutation

Launched by VERTEX PHARMACEUTICALS INCORPORATED · May 26, 2009

Keywords

ClinConnect Summary

This was a phase 3 study in subjects with cystic fibrosis (CF) age 12 years and older who have a G551D-CFTR mutation and percent predicted forced expiratory volumn in 1 second (FEV1) between 40% and 90%.

Based on in vitro studies and pharmacologic, pharmacokinetic (PK), and safety profiles, ivacaftor was selected for clinical development as a possible treatment for patients with CF. Patients with the G551D mutation were the targeted population for this study because ivacaftor is a potentiator of the gating function of the CFTR protein, and the most prevalent mutation with a gating defect i...

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Confirmed diagnosis of cystic fibrosis (CF) and G551D mutation in at least 1 allele
• • Forced expiratory volume in 1 second (FEV1) of 40% to 90% (inclusive) of predicted normal for age, gender, and height at Screening.
• • No clinically significant abnormalities that would have interfered with the study assessments, as judged by the investigator
• • Willing to use highly effective birth control methods during the study
• Exclusion Criteria:
• • History of any illness or condition that might confound the results of the study or pose an additional risk in administering study drug to the subject
• • Acute respiratory infection, pulmonary exacerbation, or changes in therapy for pulmonary disease within 4 weeks of Day 1 of the study
• • History of alcohol, medication or illicit drug abuse within one year prior to Day 1
• • Abnormal liver function ≥ 3x the upper limit of normal
• • Abnormal renal function at Screening
• • History of solid organ or hematological transplantation
• • Pregnant, planning a pregnancy, breast-feeding, or unwilling to follow contraception requirements
• • Ongoing participation in another therapeutic clinical study or prior participation in an investigational drug study within 30 days prior to Screening
• • Use of inhaled hypertonic saline treatment
• • Concomitant use of any inhibitors or inducers of cytochrome P450 3A4 (CYP 3A4)