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

• Register: EUCTR
• Last refreshed on: 19 March 2012
• Main ID: EUCTR2008-006446-25-NL
• Date of registration: 10/03/2009
• Prospective Registration: Yes
• Primary sponsor: Vertex Pharmaceuticals Incorporated
• Public title: 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
• Scientific title: 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
• Date of first enrolment: 26/08/2009
• Target sample size: 90
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2008-006446-25
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: yes Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no

Countries of recruitment

Belgium, Germany, Netherlands

Key inclusion & exclusion criteria

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.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Cystic Fibrosis
MedDRA version: 9.1 Level: LLT Classification code 10011762 Term: Cystic fibrosis
MedDRA version: 9.1 Level: LLT Classification code 10011764 Term: Cystic fibrosis NOS

Intervention(s)

Product Name: VX-809
Product Code: VX-809
Pharmaceutical Form: Capsule, hard
CAS Number: 936727-05-8
Current Sponsor code: VX-809
Other descriptive name: VRT-826809
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 25-50
Pharmaceutical form of the placebo: Capsule, hard
Route of administration of the placebo: Oral use

Primary Outcome(s)

Primary end point(s): Safety and tolerability assessments based on adverse events, hematology, clinical chemistry, urinalysis, electrocardiograms (ECGs), vital signs, and physical examinations.

Secondary Objective: To evaluate the effect of VX-809 on biomarkers of CFTR activity, pulmonary function, and patient-reported outcomes (PROs) in subjects with CF who are homozygous for the DeltaF508-CFTR mutation

To evaluate the pharmacokinetics of VX-809 in subjects with CF who are homozygous for the DeltaF508-CFTR mutation

To determine a dose of VX-809 for further clinical study

Main Objective: To evaluate the safety and tolerability of VX-809 in CF patients who are homozygous for the DeltaF508-CFTR mutation

Secondary ID(s)

VX08-809-101