A study in people with Cystic Fibrosis (a rare hereditary pulmonary disease) to assess the long-term efficacy and safety of a combination of two experimental drugs
- Conditions
- Cystic fibrosis in patients homozygous or heterozygous for the F508del-CFTR MutationMedDRA version: 18.1Level: PTClassification code 10011762Term: Cystic fibrosisSystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]
- Registration Number
- EUCTR2013-000604-41-IE
- Lead Sponsor
- Vertex Pharmaceuticals Incorporated
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 1000
? Subjects entering the Part A Treatment Cohort must have completed 24 weeks of study drug treatment in Study 103 or Study 104 and wish to enroll in the Part A Treatment Cohort.
? Subjects entering the Part A Observational Cohort must have completed 24 weeks of study drug treatment in Study 103 or Study 104, but do not wish or do not qualify to enroll in the Part A Treatment Cohort.
? Treatment Cohort Subjects entering the Part B Treatment Cohort must have completed 56 days of study drug treatment in Cohort 4 of Study 102 and wish to enroll in the Part B Treatment Cohort.
? Willing to remain on a stable CF medication regimen through the end of study (Treatment Cohort only).
Are the trial subjects under 18? yes
Number of subjects for this age range: 389
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 611
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
? Any comorbidity or lab abnormality that might confound the results of the study or pose an additional risk in administering study drug to the subject.
? Pregnant and nursing females, sexually active subjects of reproductive potential who are not willing to follow the contraception requirements.
? History of drug intolerance in prior study that would pose an additional risk to the subject.
? History of poor compliance with study drug and/or procedures in prior study.
? Subjects may not participate in an investigational drug study until the completion of this study.
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 long term safety and tolerability of lumacaftor in combination with ivacaftor in subjects with cystic fibrosis (CF) who are homozygous or heterozygous for the for the F508del-CFTR mutation;Secondary Objective: To evaluate the long term efficacy and durability of lumacaftor in combination with ivacaftor for subjects in the Treatment Cohort<br><br>To evaluate the post treatment safety and tolerability of lumacaftor in combination with ivacaftor for subjects in the Observational Cohort<br>;Primary end point(s): Safety of long term treatment of lumacaftor in combination with ivacaftor based on adverse events (AEs), clinical laboratory values (serum chemistry, hematology, coagulation studies, and urinalysis), standard digital electrocardiograms (ECGs), vital signs, and pulse oximetry.;Timepoint(s) of evaluation of this end point: Through week 100
- Secondary Outcome Measures
Name Time Method Secondary end point(s): For the Treatment Cohort: Relative change from baseline in percent predicted forced expiratory volume in 1 second (FEV1), Absolute change from baseline in percent predicted FEV1, Absolute change from baseline in body mass index (BMI), Number of pulmonary exacerbations starting from the previous study (Part A only), Absolute change from baseline in Cystic Fibrosis Questionnaire Revised (CFQ R) respiratory domain score, Absolute change in BMI z score (for Part A only), Absolute change from baseline in body weight, Rate of change in percent predicted FEV1, Time-to-first pulmonary exacerbation including pulmonary exacerbations in the previous study (Part A only), Event of having at least 1 pulmonary exacerbation including pulmonary exacerbations in the previous study (Part A only)<br>For the Observational Cohort: Safety, as determined by serious adverse events (SAEs).;Timepoint(s) of evaluation of this end point: Through week 100