A randomized, double-blind, placebo-controlled, multiple dose study of VX-809 to evaluate safety, pharmacokinetics, and pharmacodynamics of VX-809 in cystic fibrosis subjects homozygous for the deltaF508-CFTR gene mutation - Study VX08-809-101

• Conditions: Cystic Fibrosis; MedDRA version: 9.1Level: LLTClassification code 10011762Term: Cystic fibrosis; MedDRA version: 9.1Level: LLTClassification code 10011764Term: Cystic fibrosis NOS

• Registration Number: EUCTR2008-006446-25-BE
• Lead Sponsor: Vertex Pharmaceuticals Incorporated

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2009-05-12
• Last Update Posted: 19 March 2012

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 90

Inclusion Criteria

The most important inclusion criteria are: (1) Male or female with confirmed diagnosis of CF; must be accompanied by a sweat chloride value greater than or equal to 60 mmol/L or a sweat sodium greater than or equal to 60 mmol/L, by quantitative pilocarpine iontophoresis, on at least 1 occasion. (2) Must have the DeltaF508-CFTR mutation in both alleles. (4) Age 18 years or older. (5) Weight greater than or equal to 40 kg. (6) Body mass index (BMI) greater than or equal to 18.5. (8) FEV1 greater than or equal to 40% of predicted normal for age, gender, and height (Knudson standards). (9) Oxygen saturation (pulse oximetry) greater than or equal to 92% on room air. (13) Willing to remain on a stable medication regimen for the duration of study participation.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

The most important exclusion criteria are: (1) History of any illness that, in the opinion of the investigator or the subject’s general practitioner, might confound the results of the study or pose an additional risk in administering study drug to the subject. (2) Ongoing acute illness including acute respiratory or lower respiratory infections. Subjects should not have a pulmonary exacerbation or changes in therapy for pulmonary disease within 14 days before receiving the first dose of study drug. (3) Pregnant, planning a pregnancy, or breast-feeding. (6) Liver function test result greater than or equal to 3x upper limit of normal (ULN). (7) History of abnormal renal function: in the past year creatinine clearance less than 50 mL/min using Cockcroft-Gault equation. (8) History of prolongation of QT/QTcF interval (greater than 450 msec in males, greater than 470 msec in females). (10) History of solid organ or hematological transplantation. (12) Ongoing participation in another therapeutic clinical study, or prior participation in an investigational drug study without appropriate washout. (13) Illness within 14 days before receiving the first dose of study drug. Illness” is defined as a recent non-serious, non-acute condition, e.g., common cold. (14) Treatment with systemic antibiotics for an acute illness within 14 days before receiving the first dose of study drug. (15) Concomitant use of any inhibitors or inducers of CYP3A4.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified