Status:
COMPLETED
Study of Lumacaftor in Combination With Ivacaftor in Subjects 6 Through 11 Years of Age With Cystic Fibrosis, Homozygous for the F508del-CFTR Mutation
Lead Sponsor:
Vertex Pharmaceuticals Incorporated
Conditions:
Cystic Fibrosis
Eligibility:
All Genders
6-11 years
Phase:
PHASE3
Brief Summary
This is a Phase 3, 2-part (Part A and Part B), open-label, multicenter study to evaluate the pharmacokinetics, safety, and tolerability of lumacaftor in combination with ivacaftor in subjects with cys...
Eligibility Criteria
Inclusion
- Confirmed diagnosis of CF defined as: with 2 CF-causing mutations, chronic sinopulmonary disease or gastrointestinal/nutritional abnormalities
- Subjects who weigh ≥15 kg without shoes at Screening Visit
- Subjects who are homozygous for the F508del-CFTR mutation
- Subjects with percent predicted forced expiratory volume in 1 second (FEV1) of 70% to 105% (inclusive) (Part A) or ≥40% (Part B) at Screening Visit where the predicted values are adjusted for age, sex, and height using the Wang equation
- Subjects with stable CF disease and who are willing to remain on stable CF medication regimen
- Able to swallow tablets
Exclusion
- History of any illness or condition that might confound the results of the study or pose an additional risk in administering study drug to the subject
- Acute respiratory infection, pulmonary exacerbation, or changes in therapy for pulmonary disease within 28 days before Day 1 of the study
- Abnormal liver function as defined in the protocol at Screening Visit
- Abnormal renal function as defined in the protocol at Screening Visit
- History of solid organ or hematological transplantation
- Ongoing participation in an investigational drug study or prior participation in an investigational drug study within 30 days prior of Screening Visit
- History or evidence of lens opacity or cataract at Screening Visit
- Colonization with organisms associated with a more rapid decline in pulmonary status at Screening Visit (Part A only)
- A standard 12-lead ECG demonstrating QTcF \>450 msec at Screening Visit
Key Trial Info
Start Date :
July 1 2013
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 1 2015
Estimated Enrollment :
62 Patients enrolled
Trial Details
Trial ID
NCT01897233
Start Date
July 1 2013
End Date
October 1 2015
Last Update
June 20 2017
Active Locations (20)
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1
Birmingham, Alabama, United States
2
Tucson, Arizona, United States
3
Long Beach, California, United States
4
Palo Alto, California, United States