Lumacaftor/Ivacaftor Combination Therapy in Subjects With CF Who Have an A455E CFTR Mutation

Cystic Fibrosis Clinical Trial

Official title:

A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Crossover Study to Evaluate the Efficacy of Lumacaftor/Ivacaftor Combination Therapy in Subjects With Cystic Fibrosis Who Have an A455E-CFTR Mutation

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03061331
• Other study ID #: VX15-809-111
• Secondary ID: 2016-001585-29
• Status: Completed
• Phase: Phase 2
• Start date: January 31, 2017
• Est. completion date: October 4, 2017

Study information

• Verified date: September 2018
• Source: Vertex Pharmaceuticals Incorporated
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This is a Phase 2, randomized, double-blind, placebo-controlled, multicenter, crossover study that will evaluate the efficacy of LUM/IVA in subjects with CF 12 years of age and older who have at least one A455E mutation.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 20
• Est. completion date: October 4, 2017
• Est. primary completion date: September 6, 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years and older

Eligibility

Inclusion Criteria:

• Male or female with confirmed diagnosis of CF

• All subjects must have an A455E mutation on at least 1 CFTR allele; no more than 10 subjects may have an F508del mutation on 1 CFTR allele.

• Forced expiratory volume in one second (FEV1) =30% of predicted and =90% of predicted at the Screening Visit, based on the Global Lung Function Initiative (GLI)-2012 multi ethnic all-age reference equations.

• Stable CF disease as judged by the investigator.

• Willing to remain on a stable medication regimen for CF from 4 weeks before Day 1 through the Follow up Visit.

Exclusion Criteria:

• History of any comorbidity reviewed at the Screening Visit 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.

• A G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R, or R117H mutation on at least one CFTR allele.

• Pregnant or breastfeeding.

• Any abnormal laboratory values at the Screening Visit.

• History of cataract/lens opacity, or evidence of cataract/lens opacity determined to be clinically significant by the ophthalmologist or optometrist during the ophthalmologic examination at the Screening Visit.

• Use of strong inhibitors or strong inducers of CYP3A, including consumption of certain herbal medications and certain fruit and fruit juices, within 14 days before Day 1

• Sexually active subjects of reproductive potential who are not willing to follow the contraception requirements

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Drug:
• LUM/IVA
LUM 400 mg/IVA 250 mg every 12 hours (q12h)
• Placebo
No Active Drug

Locations

Country	Name	City	State
Netherlands	HagaZiekenhuis	Den Haag
Netherlands	University Medical Center, Utrecht, Department of Pulmonology and Tuberculosis	Heidelberglaan

Sponsors (1)

Lead Sponsor	Collaborator
Vertex Pharmaceuticals Incorporated

Country where clinical trial is conducted

Netherlands, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Absolute Change From Study Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (ppFEV1) Through Week 8	FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration.	Study Baseline, Through Week 8