A Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-CFTR Mutation

NCT ID: NCT02516410

Last Updated: 2018-06-12

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

168 participants

Study Classification

INTERVENTIONAL

Study Start Date

2015-08-31

Study Completion Date

2016-06-07

Brief Summary

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Study to evaluate the efficacy of VX-661 in combination with ivacaftor (IVA, VX-770) through Week 12 in participants with cystic fibrosis (CF) who are heterozygous for the F508del mutation on the CF transmembrane conductance regulator (CFTR) gene and with a second CFTR mutation that is not likely to respond to VX-661 and/or IVA therapy (F508del/not responsive \[NR\]).

Detailed Description

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Conditions

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Cystic Fibrosis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Caregivers Investigators

Study Groups

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VX-661/IVA

VX-661 100 milligram (mg) plus IVA 150 mg fixed dose combination (FDC) tablet administered orally in the morning and IVA 150 mg film-coated tablet administered orally in the evening up to Week 12.

Group Type EXPERIMENTAL

VX-661 plus ivacaftor combination

Intervention Type DRUG

Ivacaftor

Intervention Type DRUG

Placebo

Placebo matched to VX-661 plus IVA FDC tablet administered orally in the morning and placebo matched to IVA film-coated tablet administered orally in the evening up to Week 12.

Group Type PLACEBO_COMPARATOR

Placebo (matched to VX-661 plus ivacaftor combination)

Intervention Type DRUG

Placebo (matched to ivacaftor)

Intervention Type DRUG

Interventions

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VX-661 plus ivacaftor combination

Intervention Type DRUG

Ivacaftor

Intervention Type DRUG

Placebo (matched to VX-661 plus ivacaftor combination)

Intervention Type DRUG

Placebo (matched to ivacaftor)

Intervention Type DRUG

Other Intervention Names

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IVA VX-770

Eligibility Criteria

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Inclusion Criteria

* Confirmed diagnosis of CF defined as a sweat chloride value greater than or equal to (\>=)60 millimole per liter (mmol/L) by quantitative pilocarpine iontophoresis.
* Heterozygous for the F508del-CFTR mutation and with a second CFTR mutation that is not likely to respond to VX-661 and/or ivacaftor therapy, genotype to be confirmed via assessment at the Screening Visit.
* Forced Expiratory Volume in 1 Second (FEV1) \>=40 percent (%) and less than or equal to (\<=)90% of predicted normal for age, sex, and height at Screening Visit.

Exclusion Criteria

* History of any comorbidity 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 participant.
* An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 28 days before Day 1 (first dose of study drug).
* History of solid organ or hematological transplantation.
* Ongoing or prior participation in an investigational drug study or use of commercially available CFTR modulator within 30 days of screening.
* Pregnant or nursing females.
Minimum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Vertex Pharmaceuticals Incorporated

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Birmingham, Alabama, United States

Site Status

La Jolla, California, United States

Site Status

Los Angeles, California, United States

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Denver, Colorado, United States

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Miami, Florida, United States

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Atlanta, Georgia, United States

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Chicago, Illinois, United States

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Indianapolis, Indiana, United States

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New Orleans, Louisiana, United States

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Ann Arbor, Michigan, United States

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Detroit, Michigan, United States

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Minneapolis, Minnesota, United States

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Kansas City, Missouri, United States

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Omaha, Nebraska, United States

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New York, New York, United States

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Cincinnati, Ohio, United States

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Memphis, Tennessee, United States

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Austin, Texas, United States

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Dallas, Texas, United States

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Richmond, Virginia, United States

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Seattle, Washington, United States

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Spokane, Washington, United States

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Brisban, Queensland, Australia

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Chermside, , Australia

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Herston, , Australia

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Innsbruck, , Australia

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Westmead, , Australia

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Graz, , Austria

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Innsbruck, , Austria

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Salzburg, , Austria

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Vancouver, British Columbia, Canada

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Montreal, , Canada

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Bron, , France

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Montpellier, , France

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Paris, , France

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Haifa, , Israel

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Hashomer, , Israel

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Jerusalem, , Israel

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Petah Tikva, , Israel

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Barcelona, , Spain

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Valencia, , Spain

Site Status

Countries

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United States Australia Austria Canada France Israel Spain

References

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Munck A, Kerem E, Ellemunter H, Campbell D, Wang LT, Ahluwalia N, Owen CA, Wainwright C. Tezacaftor/ivacaftor in people with cystic fibrosis heterozygous for minimal function CFTR mutations. J Cyst Fibros. 2020 Nov;19(6):962-968. doi: 10.1016/j.jcf.2020.04.015. Epub 2020 Jun 13.

Reference Type DERIVED
PMID: 32546431 (View on PubMed)

Other Identifiers

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2014-004787-37

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

VX14-661-107

Identifier Type: -

Identifier Source: org_study_id

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