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Study of VX-809 in Cystic Fibrosis Subjects With the ∆F508-CFTR Gene Mutation

NCT ID: NCT00865904

Last Updated: 2015-08-28

Study Results

Results available

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

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

93 participants

Study Classification

INTERVENTIONAL

Study Start Date

2009-03-31

Study Completion Date

2009-12-31

Brief Summary

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The primary objective of the study was to evaluate the safety and tolerability of VX-809 in participants with cystic fibrosis (CF) who are homozygous for the F508del mutation on the CF transmembrane conductance regulator (CFTR) gene.

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Detailed Description

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This was a Phase 2, randomized, double-blind, placebo-controlled, multiple-dose study of orally-administered VX-809 in participants with CF who are homozygous for the specific CFTR mutation known as ∆F508 or F508del. Enrollment was planned for 90 participants at approximately 20 centers. Participants were planned to be randomized in a 4:1 ratio to receive 1 of 4 doses of VX-809 or placebo once a day for 28 days in a parallel design. Participants were outpatients during the study, except for overnight stays on Day 1 and 28.

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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Placebo

Placebo matched to VX-809 capsule orally once daily for 28 days.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Placebo matched to VX-809 capsules.

VX-809, 25 mg

VX-809, 25 milligram (mg) capsule orally once daily for 28 days.

Group Type EXPERIMENTAL

VX-809

Intervention Type DRUG

Capsules

VX-809, 50 mg

VX-809, 50 mg capsule orally once daily for 28 days.

Group Type EXPERIMENTAL

VX-809

Intervention Type DRUG

Capsules

VX-809, 100 mg

VX-809, 100 mg capsule orally once daily for 28 days.

Group Type EXPERIMENTAL

VX-809

Intervention Type DRUG

Capsules

VX-809, 200 mg

VX-809, 200 mg capsule orally once daily for 28 days.

Group Type EXPERIMENTAL

VX-809

Intervention Type DRUG

Capsules

Interventions

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VX-809

Capsules

Intervention Type DRUG

Placebo

Placebo matched to VX-809 capsules.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Confirmed diagnosis of CF with ∆F508-CFTR mutation in both alleles
* Forced expiratory volume in 1 second (FEV1) greater than or equal to (\>=) 40 percent (%) of predicted normal for age, gender, and height
* Weight \>=40 kilograms (kg) and body mass index greater than or equal to 18.5 kilogram per square meter (kg/m\^2)
* Screening laboratory values, tests, and physical examination within acceptable ranges
* Negative pregnancy test (for women of child-bearing potential)
* Able and willing to follow contraceptive requirements
* Willing to remain on a stable medication regimen for the duration of study participation

Exclusion Criteria

* History of any illness, or any ongoing acute illness, that could impact the safety of the study participant or may confound results of study
* Pulmonary exacerbation or changes in therapy for pulmonary disease within 14 days before receiving the first dose of study drug
* Impaired hepatic or renal function
* History of organ or hematological transplant
Minimum Eligible Age

18 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

Principal Investigators

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Medical Monitor

Role: STUDY_DIRECTOR

Vertex Pharmaceuticals Incorporated

Locations

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Birmingham, Alabama, United States

Site Status

Palo Alto, California, United States

Site Status

San Diego, California, United States

Site Status

Aurora, Colorado, United States

Site Status

Atlanta, Georgia, United States

Site Status

Chicago, Illinois, United States

Site Status

Iowa City, Iowa, United States

Site Status

Baltimore, Maryland, United States

Site Status

Boston, Massachusetts, United States

Site Status

Minneapolis, Minnesota, United States

Site Status

St Louis, Missouri, United States

Site Status

Chapel Hill, North Carolina, United States

Site Status

Cincinnati, Ohio, United States

Site Status

Cleveland, Ohio, United States

Site Status

Columbus, Ohio, United States

Site Status

Philadelphia, Pennsylvania, United States

Site Status

Pittsburgh, Pennsylvania, United States

Site Status

Seattle, Washington, United States

Site Status

Brussels, , Belgium

Site Status

Leuven, , Belgium

Site Status

Toronto, Ontario, Canada

Site Status

Cologne, , Germany

Site Status

Hanover, , Germany

Site Status

Rotterdam, , Netherlands

Site Status

Utrecht, , Netherlands

Site Status

Countries

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United States Belgium Canada Germany Netherlands

References

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Heneghan M, Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2023 Nov 20;11(11):CD010966. doi: 10.1002/14651858.CD010966.pub4.

Reference Type DERIVED
PMID: 37983082 (View on PubMed)

Southern KW, Murphy J, Sinha IP, Nevitt SJ. Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del). Cochrane Database Syst Rev. 2020 Dec 17;12(12):CD010966. doi: 10.1002/14651858.CD010966.pub3.

Reference Type DERIVED
PMID: 33331662 (View on PubMed)

Clancy JP, Rowe SM, Accurso FJ, Aitken ML, Amin RS, Ashlock MA, Ballmann M, Boyle MP, Bronsveld I, Campbell PW, De Boeck K, Donaldson SH, Dorkin HL, Dunitz JM, Durie PR, Jain M, Leonard A, McCoy KS, Moss RB, Pilewski JM, Rosenbluth DB, Rubenstein RC, Schechter MS, Botfield M, Ordonez CL, Spencer-Green GT, Vernillet L, Wisseh S, Yen K, Konstan MW. Results of a phase IIa study of VX-809, an investigational CFTR corrector compound, in subjects with cystic fibrosis homozygous for the F508del-CFTR mutation. Thorax. 2012 Jan;67(1):12-8. doi: 10.1136/thoraxjnl-2011-200393. Epub 2011 Aug 8.

Reference Type DERIVED
PMID: 21825083 (View on PubMed)

Other Identifiers

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VX08-809-101

Identifier Type: -

Identifier Source: org_study_id

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