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

Phase 1

• Conditions: Cystic Fibrosis; MedDRA version: 17.1Level: PTClassification code 10011762Term: Cystic fibrosisSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]

• Registration Number: EUCTR2014-004837-13-IT
• Lead Sponsor: Vertex Pharmaceuticals Incorporated

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2015-03-27
• Last Update Posted: 3 April 2017

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 490

Inclusion Criteria

• 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: 190
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 300
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

• 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