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: EUCTR2012-003990-24-GB

Lead Sponsor: Vertex Pharmaceuticals Incorporated

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: Not specified

Target Recruitment: 559

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: 273
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 319
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

Primary Outcome Measures

Name	Time	Method
Main Objective: To evaluate the efficacy of lumacaftor in combination with ivacaftor through Week 24 in subjects with Cystic Fibrosis who are homozygous for the F508del-CFTR mutation;<br> Secondary Objective: To evaluate the safety of lumacaftor in combination with ivacaftor through Week 24<br> <br> To investigate the pharmacokinetics (PK) of lumacaftor and its metabolite, M28 (M28 lumacaftor) and ivacaftor and its metabolites M1 and M6 (M1 ivacaftor and M6 ivacaftor)<br> ;Primary end point(s): Relative change in percent predicted forced expiratory volume in 1 second (FEV1) from baseline through Week 24;Timepoint(s) of evaluation of this end point: Week 24

Secondary Outcome Measures

Name	Time	Method
<br> Secondary end point(s): Absolute change in body mass index (BMI) from baseline at Week 24<br> <br> Number of pulmonary exacerbations from baseline through Week 24<br> <br> Absolute change in Cystic Fibrosis Questionnaire – Revised (CFQ-R) respiratory domain score from baseline through Week 24<br> <br> Safety and tolerability assessments based on adverse events (AEs), clinical laboratory values (hematology, serum chemistry, coagulation studies, and urinalysis), standard digital electrocardiograms (ECGs), pulse oximetry and vital signs<br> ;Timepoint(s) of evaluation of this end point: Weeks 24 and 28

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