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

Phase 1

• Conditions: Cystic fibrosis in patients homozygous or heterozygous for the F508del-CFTR Mutation; MedDRA version: 16.0Level: PTClassification code 10011762Term: Cystic fibrosisSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]

• Registration Number: EUCTR2013-000604-41-ES
• Lead Sponsor: Vertex Pharmaceuticals Incorporated

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2013-10-08
• Last Update Posted: 17 May 2016

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 1000

Inclusion Criteria

? 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

Exclusion Criteria

? 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