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

Cystic Fibrosis Clinical Trial

Official title:

A Randomized, Double-blind, Placebo-controlled, Crossover Study to Evaluate the Efficacy of Ivacaftor in Subjects With Cystic Fibrosis Who Are 6 Years of Age and Older and Have Either a 3849 + 10KB C→T or D1152H-CFTR Mutation

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03068312
• Other study ID #: VX16-770-127
• Secondary ID: 2017-000457-39
• Status: Completed
• Phase: Phase 3
• Start date: July 18, 2017
• Est. completion date: December 18, 2018

Study information

• Verified date: February 2020
• Source: Vertex Pharmaceuticals Incorporated
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 38
• Est. completion date: December 18, 2018
• Est. primary completion date: December 18, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Drug:
• Ivacaftor
IVA 150 mg tablet.
• Placebo
Placebo matched to IVA tablet.

Locations

Country	Name	City	State
Israel	Hadassah Medical Organization	Jerusalem

Sponsors (1)

Lead Sponsor	Collaborator
Vertex Pharmaceuticals Incorporated

Country where clinical trial is conducted

Israel, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in Lung Clearance Index 2.5 (LCI2.5)	LCI2.5 represents the number of lung turnovers required to reduce the end tidal inert gas concentration to 1/40th of its starting value.	From baseline through 8 weeks