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: 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: EUCTR2014-004838-25-AT

Lead Sponsor: Vertex Pharmaceuticals Incorporated

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 155

Inclusion Criteria

• Heterozygous for F508del-CFTR mutation and a second CFTR allele with a gating defect that is clinically demonstrated to be ivacaftor responsive
• FEV1 =40% and =90% of predicted normal for age, sex, and height during screening
• Stable CF disease as judged by the investigator.
Are the trial subjects under 18? yes
Number of subjects for this age range: 20
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 135
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.
• Pregnant and nursing females (females of childbearing potential must have a negative pregnancy test at Screening and Week -4 Visits).
• 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

Primary Outcome Measures

Name	Time	Method
Main Objective: To evaluate the efficacy of VX-661 in combination with ivacaftor in subjects with CF who are heterozygous for the F508del mutation on the CFTR gene and a second CFTR allele with a gating defect that is clinically demonstrated to be ivacaftor responsive ;Secondary Objective: To evaluate the safety of VX-661 in combination with ivacaftor <br><br>To investigate the pharmacokinetics (PK) of VX-661 and its metabolites, M1 and M2 (M1-661 and M2-661, respectively) and ivacaftor and its metabolite M1 (M1 ivacaftor)<br>;Primary end point(s): Absolute change in percent predicted forced expiratory volume in 1 second (FEV1) from baseline;Timepoint(s) of evaluation of this end point: Week 8

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): • Relative change in percent predicted FEV1 from baseline through Week 8<br>• Absolute change in sweat chloride from baseline through Week 8<br>• Absolute change in Cystic Fibrosis Questionnaire – Revised (CFQ-R) respiratory domain score from baseline through Week 8<br>• Safety and tolerability assessments based on adverse events (AEs), clinical laboratory values, standard 12-lead electrocardiograms (ECGs), vital signs, and pulse oximetry; from screening through 4 weeks after receiving last dose<br>• PK parameters of VX-661, M1-661, M2-661, ivacaftor, and M1 ivacaftor through Week 8;Timepoint(s) of evaluation of this end point: Weeks 8 and 12

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