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

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

38 participants

Study Classification

INTERVENTIONAL

Study Start Date

2017-07-18

Study Completion Date

2018-12-18

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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.

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

A Study to Evaluate Efficacy and Safety of Ivacaftor in Subjects With Cystic Fibrosis Aged 3 Through 5 Years Who Have a Specified CFTR Gating Mutation

NCT02742519

Cystic Fibrosis

TERMINATED PHASE3

Pilot Study Testing the Effect of Ivacaftor on Lung Function in Subjects With Cystic Fibrosis and Residual CFTR Function

NCT01685801

Cystic Fibrosis

COMPLETED PHASE2

Study of Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older With the G551D Mutation

NCT00909532

Cystic Fibrosis

COMPLETED PHASE3

An Efficacy and Safety Study of Ivacaftor in Patients With Cystic Fibrosis and Two Splicing Mutations

NCT02934698

Cystic Fibrosis

COMPLETED PHASE3

Roll-Over Study of Ivacaftor in Cystic Fibrosis Pediatric Subjects With a CF Transmembrane Conductance Regulator Gene (CFTR) Gating Mutation

NCT01946412

Cystic Fibrosis

COMPLETED PHASE3

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Cystic Fibrosis

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Sequence 1: First Ivacaftor (IVA) Then Placebo

Participants received IVA 150 milligram (mg) every 12 hours (q12h) for 8 weeks in treatment period 1 followed by placebo matched to IVA for 8 weeks in treatment period 2. A washout period of 8 weeks was maintained between the 2 treatment periods.

Group Type EXPERIMENTAL

Ivacaftor

Intervention Type DRUG

IVA 150 mg tablet.

Placebo

Intervention Type DRUG

Placebo matched to IVA tablet.

Sequence 2: First Placebo Then IVA

Participants received placebo matched to IVA for 8 weeks in treatment period 1 followed by IVA 150 mg q12h for 8 weeks in treatment period 2. A washout period of 8 weeks was maintained between the 2 treatment periods.

Group Type EXPERIMENTAL

Ivacaftor

Intervention Type DRUG

IVA 150 mg tablet.

Placebo

Intervention Type DRUG

Placebo matched to IVA tablet.

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Ivacaftor

IVA 150 mg tablet.

Intervention Type DRUG

Placebo

Placebo matched to IVA tablet.

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

VX-770 IVA

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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.

Minimum Eligible Age

6 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No