A Study to Evaluate the Safety and Efficacy of VX-371 in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del-CFTR Mutatio

Phase 1

• Conditions: Cystic Fibrosis; MedDRA version: 20.0Level: PTClassification code 10011762Term: Cystic fibrosisSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]

• Registration Number: EUCTR2015-004841-13-IE
• Lead Sponsor: Vertex Pharmaceuticals Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2016-06-07
• Last Update Posted: 13 November 2017

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 150

Inclusion Criteria

•Willing and able to use the delivery device as directed by the study manual
•Confirmed diagnosis of CF, defined as a sweat chloride value =60 mmol/L by quantitative pilocarpine iontophoresis.
•Homozygous for the F508del CFTR mutation. If the CFTR screening genotype result is not received before randomization, a previous CFTR genotype lab report may be used to establish eligibility.
•Percent predicted FEV1 of =40 to <90 percentage points adjusted for age, sex, and height according to the Global Lung Initiative (GLI) at the Screening Visit.
•Willing to discontinue physician-prescribed HS use.
•Female subjects of childbearing potential with a negative serum pregnancy test at the Screening Visit
Are the trial subjects under 18? yes
Number of subjects for this age range: 35
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 115
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

•History of any comorbidity, which 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.
•Any clinically significant laboratory abnormalities at the Screening Visit that would interfere with the study assessments or pose an undue risk for 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).
•A 12 lead ECG demonstrating QTcF >450 msec at the Screening Visit.
•History of solid organ or hematological transplantation.
•Used diuretics or renin-angiotensin aldosterone system antihypertensive drugs in the 28 days prior to Screening or an anticipated need for any of these medications during the study.
•Ongoing or prior participation in an investigational drug study within 30 days of the Screening Visit.
•Inability to withhold short-acting, long-acting, or once-daily, long-acting bronchodilator use for 4, 12, or 24 hours prior to clinic visit, respectively.
•History of significant intolerance to inhaled HS, or intolerance to the single dose of HS at Screening
•Known hypersensitivity or history of intolerance to Orkambi.
•Pregnant and nursing females.
•Subjects who have participated in Parion Sciences Study PS-G201.

Study & Design

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