A study in people with Cystic Fibrosis ( a rare hereditary pulmonary disease) to assess the efficacy and safety of a combination of two experimental drugs

Phase 1

• Conditions: Cystic fibrosis in patients homozygous for the F508del-CFTR Mutation; MedDRA version: 20.0 Level: PT Classification code 10011762 Term: Cystic fibrosis System Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]

• Registration Number: EUCTR2012-003989-40-GB
• Lead Sponsor: Vertex Pharmaceuticals Incorporated

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2013-05-13
• Study Completion: 2014-04-29
• Last Update Posted: 28 February 2019

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Not specified
• Target Recruitment: 549

Inclusion Criteria

• Males and females, aged 12 years or older on the date of informed consent or, where appropriate, date of assent
• Confirmed diagnosis of CF
• Homozygous for the F508del CFTR mutation
• FEV1 =40% and =90% of predicted normal for age, sex, and height
• 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: 264
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 349
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

• An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 4 weeks before first dose of study drug
• History of solid organ or hematological transplantation
• History of alcohol or drug abuse in the past year
• Ongoing or prior participation in an investigational drug study (including studies investigating lumacaftor and/or ivacaftor) within 30 days of screening
• Use of moderate to strong inhibitors or inducers of CYP3A, including consumption of certain herbal medications (e.g., St. John's Wort) and certain fruit and fruit juices within 14 days before Day 1 of dosing

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified