A study in people with Cystic Fibrosis ( a rare hereditary lung disease) to assess the efficacy and safety of a combination of two experimental drugs
- Conditions
- Cystic FibrosisMedDRA version: 18.0Level: PTClassification code 10011762Term: Cystic fibrosisSystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
- Registration Number
- EUCTR2014-004837-13-ES
- Lead Sponsor
- Vertex Pharmaceuticals Incorporated
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 510
Homozygous for the F508del CFTR mutation, genotype to be confirmed at the Screening Visit
Confirmed diagnosis of CF defined as a sweat chloride value = 60 mmol/L by quantitative pilocarpine iontophoresis
FEV1 = 40% and = 90% of predicted normal for age, sex, and height during screening
Stable CF disease as judged by the investigator
Willing to remain on a stable CF medication regimen through Week 24 or, if applicable, the Safety Follow up Visit
Are the trial subjects under 18? yes
Number of subjects for this age range: 122
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 368
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
History of any comorbidity 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 28 days before Day 1 (first dose of study drug)
Pregnant or nursing females (females of childbearing potential must have a negative pregnancy test at Screening and Day 1)
Sexually active subjects of reproductive potential who are not willing to follow the contraception requirements
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 efficacy of VX-661 in combination with ivacaftor through 24 weeks of treatment in subjects with cystic fibrosis (CF) who are homozygous for the F508del mutation on the CF transmembrane conductance regulator (CFTR) gene;Secondary Objective: To evaluate the safety of VX-661 in combination with ivacaftor through Week 24<br><br>To investigate the pharmacokinetics (PK) of VX-661 and its metabolites, M1 and M2 (M1-661 and M2-661, respectively) and ivacaftor and its metabolite M1 (M1 ivacaftor);Primary end point(s): Absolute change in percent predicted forced expiratory volume in 1 second (FEV1) from baseline through Week 24;Timepoint(s) of evaluation of this end point: Week 24
- Secondary Outcome Measures
Name Time Method Timepoint(s) of evaluation of this end point: Weeks 24 and 28;Secondary end point(s): Relative change in percent predicted FEV1 from baseline through Week 24<br>Number of pulmonary exacerbations through Week 24<br>Absolute change in body mass index (BMI) from baseline at Week 24<br>Absolute change in Cystic Fibrosis Questionnaire ? Revised (CFQ-R) respiratory domain score from baseline through Week 24<br>Safety and tolerability assessments based on adverse events (AEs), clinical laboratory values, standard 12-lead electrocardiograms (ECGs), vital signs, and pulse oximetry; from screening through 4 weeks after receiving last dose<br>Time to first pulmonary exacerbation through Week 24<br>Absolute change in sweat chloride from baseline through Week 24<br>Absolute change in BMI z-score from baseline at Week 24 (in subjects <20 years of age at time of screening)<br>Absolute change in body weight from baseline at Week 24<br>PK parameters of VX-661, M1-661, M2-661, ivacaftor, and M1 ivacaftor through week 24