NCT00865904

Brief Summary

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.

Trial Health

93
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
93

participants targeted

Target at P50-P75 for phase_2

Timeline
Completed

Started Mar 2009

Shorter than P25 for phase_2

Geographic Reach
5 countries

25 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

March 1, 2009

Completed
17 days until next milestone

First Submitted

Initial submission to the registry

March 18, 2009

Completed
1 day until next milestone

First Posted

Study publicly available on registry

March 19, 2009

Completed
9 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2009

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2009

Completed
5.7 years until next milestone

Results Posted

Study results publicly available

August 28, 2015

Completed
Last Updated

August 28, 2015

Status Verified

June 1, 2015

Enrollment Period

9 months

First QC Date

March 18, 2009

Results QC Date

August 1, 2015

Last Update Submit

August 1, 2015

Conditions

Cystic Fibrosis

Keywords

∆F508F508delCystic Fibrosis Transmembrane Conductance RegulatorCFTRPancreatic diseasesLung diseasesGenetic disease, inbornInfant, newborn, diseases

Outcome Measures

Primary Outcomes (1)

  • Safety and Tolerability Based on Adverse Events (AEs)

    AE: any untoward medical occurrence in a participant during the study; the event does not necessarily have a causal relationship with the treatment. This includes any newly occurring event or previous condition that has increased in severity or frequency after the informed consent form is signed. AE includes serious as well as Non-serious AEs. Serious adverse event (SAE) (subset of AE): medical event or condition, which falls into any of the following categories, regardless of its relationship to the study drug: death, life threatening adverse experience, in-patient hospitalization/prolongation of hospitalization, persistent/significant disability or incapacity, congenital anomaly/birth defect, important medical event. Number of participants with AEs and SAEs are reported. An AE that started at or after initial dosing of study drug, or increased in severity after initial dosing of study drug visit is considered treatment-emergent.

    Up to 14 days after last dose (last dose = Day 28)

Secondary Outcomes (9)

  • Change From Baseline in Forced Expiratory Volume in 1 Second (FEV1) at Day 28

    Baseline, Day 28

  • Change From Baseline in Percent Predicted FEV1 at Day 28

    Baseline, Day 28

  • Change From Baseline in Forced Vital Capacity (FVC) at Day 28

    Baseline, Day 28

  • Change From Baseline in Forced Expiratory Flow Over the Middle Half of the FVC (FEF25-75) at Day 28

    Baseline, Day 28

  • Change From Baseline in Sweat Chloride at Day 28

    Baseline, Day 28

  • +4 more secondary outcomes

Study Arms (5)

Placebo

PLACEBO COMPARATOR

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

Drug: Placebo

VX-809, 25 mg

EXPERIMENTAL

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

Drug: VX-809

VX-809, 50 mg

EXPERIMENTAL

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

Drug: VX-809

VX-809, 100 mg

EXPERIMENTAL

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

Drug: VX-809

VX-809, 200 mg

EXPERIMENTAL

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

Drug: VX-809

Interventions

VX-809DRUG

Capsules

VX-809, 100 mgVX-809, 200 mgVX-809, 25 mgVX-809, 50 mg
PlaceboDRUG

Placebo matched to VX-809 capsules.

Placebo

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (25)

Unknown Facility

Birmingham, Alabama, United States

Location

Unknown Facility

Palo Alto, California, United States

Location

Unknown Facility

San Diego, California, United States

Location

Unknown Facility

Aurora, Colorado, United States

Location

Unknown Facility

Atlanta, Georgia, United States

Location

Unknown Facility

Chicago, Illinois, United States

Location

Unknown Facility

Iowa City, Iowa, United States

Location

Unknown Facility

Baltimore, Maryland, United States

Location

Unknown Facility

Boston, Massachusetts, United States

Location

Unknown Facility

Minneapolis, Minnesota, United States

Location

Unknown Facility

St Louis, Missouri, United States

Location

Unknown Facility

Chapel Hill, North Carolina, United States

Location

Unknown Facility

Cincinnati, Ohio, United States

Location

Unknown Facility

Cleveland, Ohio, United States

Location

Unknown Facility

Columbus, Ohio, United States

Location

Unknown Facility

Philadelphia, Pennsylvania, United States

Location

Unknown Facility

Pittsburgh, Pennsylvania, United States

Location

Unknown Facility

Seattle, Washington, United States

Location

Unknown Facility

Brussels, Belgium

Location

Unknown Facility

Leuven, Belgium

Location

Unknown Facility

Toronto, Ontario, Canada

Location

Unknown Facility

Cologne, Germany

Location

Unknown Facility

Hanover, Germany

Location

Unknown Facility

Rotterdam, Netherlands

Location

Unknown Facility

Utrecht, Netherlands

Location

Related Publications (3)

  • 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.

    PMID: 37983082DERIVED

  • 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.

    PMID: 33331662DERIVED

  • 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.

    PMID: 21825083DERIVED

MeSH Terms

Conditions

Cystic FibrosisPancreatic DiseasesLung DiseasesGenetic Diseases, InbornInfant, Newborn, Diseases

Interventions

lumacaftor

Condition Hierarchy (Ancestors)

Digestive System DiseasesRespiratory Tract DiseasesCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

Title
Medical Monitor
Organization
Vertex Pharmaceuticals Incorporated
medicalinfo@vrtx.com
617-341-6777

Study Officials

  • Medical Monitor

    Vertex Pharmaceuticals Incorporated

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
TRIPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 18, 2009

First Posted

March 19, 2009

Study Start

March 1, 2009

Primary Completion

December 1, 2009

Study Completion

December 1, 2009

Last Updated

August 28, 2015

Results First Posted

August 28, 2015

Record last verified: 2015-06

Locations

CA(1)NL(2)US(18)DE(2)BE(2)