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

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

20 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-01-31

Study Completion Date

2017-10-04

Brief Summary

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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.

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Detailed Description

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Conditions

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Cystic Fibrosis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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Treatment Sequence 1

LUM/IVA in Treatment Period 1; washout; placebo in Treatment Period 2

Group Type EXPERIMENTAL

LUM/IVA

Intervention Type DRUG

LUM 400 mg/IVA 250 mg every 12 hours (q12h)

Placebo

Intervention Type DRUG

No Active Drug

Treatment Sequence 2

Placebo in Treatment Period 1; washout; LUM/IVA in Treatment Period 2

Group Type EXPERIMENTAL

LUM/IVA

Intervention Type DRUG

LUM 400 mg/IVA 250 mg every 12 hours (q12h)

Placebo

Intervention Type DRUG

No Active Drug

Interventions

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LUM/IVA

LUM 400 mg/IVA 250 mg every 12 hours (q12h)

Intervention Type DRUG

Placebo

No Active Drug

Intervention Type DRUG

Other Intervention Names

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lumacaftor/ivacaftor

Eligibility Criteria

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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

Minimum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No