Study of VX-770 in Cystic Fibrosis Subjects Age 12 and Older Homozygous for the F508del-CFTR Mutatio
- Conditions
- Cystic FibrosisMedDRA version: 13.1Level: PTClassification code 10011762Term: Cystic fibrosisSystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- EUCTR2009-010261-23-Outside-EU/EEA
- Lead Sponsor
- Vertex Pharmaceuticals Incorporated
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- A
- Sex
- All
- Target Recruitment
- 140
- Male or female with confirmed diagnosis of CF
- Must be homozygous for the F508del-CFTR mutation.
- FEV1 40% or greater of predicted normal
- 12 years of age or older
- Females of child-bearing potential must have a negative serum pregnancy test at Screening
- Subjects of child-bearing potential and who are sexually active must meet the contraception requirements
Are the trial subjects under 18? yes
Number of subjects for this age range: 50
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 90
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
- 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
- An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 4 weeks before Day 1
- History of solid organ or hematological transplantation
- History of alcohol, medication, or illicit drug abuse within one year prior to Day 1
- 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 CYP3A4
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To evaluate the safety and efficacy of VX-770 in subjects with cystic fibrosis (CF) who are homozygous for the F508del-cystic fibrosis transmembrane conductance regulator (CFTR) mutation for 16 weeks;Secondary Objective: To investigate the pharmacokinetics of 16 weeks of treatment with VX-770 ;Primary end point(s): - Absolute change in percent predicted forced expiratory volume in 1 second (FEV1) from baseline through Week 16<br><br>- Safety assessments based on adverse events, clinical laboratory values (serum chemistry, hematology, coagulation studies, and urinalysis), standard digital electrocardiograms (ECGs), 24-hour ambulatory ECGs, vital signs, and physical examinations;Timepoint(s) of evaluation of this end point: Week 16
- Secondary Outcome Measures
Name Time Method Secondary end point(s): - Change from baseline in sweat chloride through Week 16 <br>- Change from baseline in CF Questionnaire-Revised (CFQ-R) through Week 16 <br>- Rate of change in weight through Week 16 <br>- Pharmacokinetic (PK) parameters of VX-770 <br>;Timepoint(s) of evaluation of this end point: Week 16