A Phase 3, Randomized, Double Blind, Placebo Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous for the F508del CFTR Mutatio
- Conditions
- Cystic Fibrosis10083624
- Registration Number
- NL-OMON40417
- Lead Sponsor
- Vertex Pharmaceuticals
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 21
* 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
* 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 strong inhibitors, moderate inducers or strong 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
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Aboslute change in percent predicted forced expiratory volume in 1 second<br /><br>(FEV1) from<br /><br>baseline at Week 24</p><br>
- Secondary Outcome Measures
Name Time Method <p>Secondary Endpoints<br /><br>* Relative change in percent predicted forced expiratory volume in 1 second<br /><br>(FEV1) from baseline at Week 24<br /><br>* Absolute change in body mass index (BMI) from baseline at Week 24<br /><br>* Number of pulmonary exacerbations through Week 24<br /><br>* Absolute change in Cystic Fibrosis Questionnaire*Revised (CFQ-R) respiratory<br /><br>domain score from baseline at Week 24<br /><br>* Safety and tolerability assessments based on adverse events (AEs), clinical<br /><br>laboratory<br /><br>values (hematology, serum chemistry, coagulation studies, and urinalysis),<br /><br>standard<br /><br>digital electrocardiograms (ECGs), ambulatory ECGs, vital signs, and pulse<br /><br>oximetry</p><br>