Overview

A Study to Evaluate Efficacy of Ivacaftor in Subjects With Cystic Fibrosis Who Have a 3849 + 10KB C→T or D1152H CFTR Mutation

Status:
Completed
Trial end date:
2018-12-18
Target enrollment:
0
Participant gender:
All
Summary
This study will evaluate the efficacy of ivacaftor treatment in subjects with CF 6 years of age and older who have a 3849 + 10KB C→T or D1152H CFTR mutation.
Phase:
Phase 3
Accepts Healthy Volunteers?
No
Details
Lead Sponsor:
Vertex Pharmaceuticals Incorporated
Treatments:
Ivacaftor
Criteria
Inclusion Criteria:

- Confirmed diagnosis of CF based on protocol-specified clinical features and at least
one of the following: increased sweat chloride level, identification of 2 CF causing
mutations, or demonstration of abnormal nasal epithelial ion transport.

- A 3849 + 10KB C→T or D1152H mutation on at least 1 CFTR allele.

- FEV1 ≥40% of predicted and ≤105% of predicted at screening.

Exclusion Criteria:

- A G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R, or R117H
mutation.

- History of any illness or any clinical condition 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.

- Ongoing or prior participation in an investigational drug study within 30 days before
the Screening Visit.

- Protocol-specified abnormal laboratory values at the Screening Visit

- For subjects <18 years of age at the Screening Visit, evidence of cataract/lens
opacity determined to be clinically significant by the ophthalmologist or optometrist
during the ophthalmologic examination (OE) at the Screening Visit.

- Use of any moderate or strong inducers or inhibitors of cytochrome P450 (CYP) 3A,
including consumption of certain herbal medications and certain fruit and fruit
juices, within 14 days before Day 1.

- Pregnant, breastfeeding, or planning to become pregnant during the study.

- Sexually active subjects of reproductive potential must be willing to use appropriate
contraception.