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: 18.0Level: PTClassification code 10011762Term: Cystic fibrosisSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Respiratory Tract Diseases [C08]

• Registration Number: EUCTR2014-004787-37-ES
• Lead Sponsor: Vertex Pharmaceuticals Incorporated

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2015-08-25
• Last Update Posted: 5 September 2016

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 300

Inclusion Criteria

Heterozygous for the F508del CFTR mutation and with a second CFTR mutation that is not likely to respond to VX-661 and/or ivacaftor therapy, genotype to be confirmed via assessment at the Screening Visit.

Confirmed diagnosis of CF defined as a sweat chloride value ?60 mmol/L by quantitative pilocarpine iontophoresis (as documented in the subject?s medical record OR from sweat chloride test result obtained at the Screening, if subject does not have a sweat chloride test result in the medical record).

FEV1 ?40% and ?90% of predicted normal for age, sex, and height at Screening Visit. Spirometry measurements must meet American Thoracic Society/European Respiratory Society criteria for acceptability and repeatability.

Stable CF disease as judged by the investigator.

Willing to remain on a stable CF medication regimen through Week 12 or, if applicable, the Safety Follow-up Visit.
Are the trial subjects under 18? yes
Number of subjects for this age range: 150
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 150
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.

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 efficacy of VX-661 in combination with ivacaftor through Week 12 in subjects with cystic fibrosis (CF) who are heterozygous for the F508del mutation on the CF transmembrane conductance regulator (CFTR) gene and with a second CFTR mutation that is not likely to respond to VX-661 and/or ivacaftor therapy (F508del/not responsive [NR]);Secondary Objective: To evaluate the safety of VX-661 in combination with ivacaftor and to investigate the pharmacokinetics (PK) of VX-661 and its metabolite M1-661, and ivacaftor and its metabolite M1-ivacaftor;Primary end point(s): Absolute change in percent predicted forced expiratory volume in 1 second (FEV1) from baseline through Week 12.;Timepoint(s) of evaluation of this end point: Week 12