NCT01614457

Brief Summary

The purpose of this study is to evaluate the efficacy and safety of ivacaftor in subjects with cystic fibrosis (CF) who have the R117H-CFTR mutation.

Trial Health

90
On Track

Trial Health Score

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

Enrollment
70

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started Jul 2012

Shorter than P25 for phase_3

Geographic Reach
2 countries

31 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

First Submitted

Initial submission to the registry

June 5, 2012

Completed
3 days until next milestone

First Posted

Study publicly available on registry

June 8, 2012

Completed
23 days until next milestone

Study Start

First participant enrolled

July 1, 2012

Completed
1.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 1, 2013

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

October 1, 2013

Completed
1.4 years until next milestone

Results Posted

Study results publicly available

February 12, 2015

Completed
Last Updated

February 12, 2015

Status Verified

January 1, 2015

Enrollment Period

1.3 years

First QC Date

June 5, 2012

Results QC Date

January 31, 2015

Last Update Submit

January 31, 2015

Conditions

Outcome Measures

Primary Outcomes (1)

  • Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 24

    FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Hankinson and Wang standards were used to calculate percent predicted FEV1 (for age, gender, and height). The Hankinson standard was used for male subjects 18 years and older and female subjects 16 years and older. The Wang standard was used for male subjects aged 6 to 17 years and for female subjects aged 6 to 15 years.

    Baseline, Week 24

Secondary Outcomes (5)

  • Change From Baseline in Body Mass Index (BMI) at Week 24

    Baseline, Week 24

  • Change From Baseline in Sweat Chloride Through Week 24

    Baseline, Week 24

  • Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 24

    Baseline, Week 24

  • Time to First Pulmonary Exacerbation

    Day 0 to 15, Day 16 to 56, Day 57 to 112, Day 113 to 168

  • Number of Subjects With Adverse Events (AEs) and Serious Adverse Events (SAEs)

    Baseline up to follow-up (3 to 4 weeks after last dose [last dose = Week 24])

Study Arms (2)

Ivacaftor

EXPERIMENTAL

Ivacaftor 150 milligram (mg) tablet orally twice daily for 24 weeks.

Drug: Ivacaftor

Placebo

PLACEBO COMPARATOR

Placebo matched to Ivacaftor tablet orally twice daily for 24 weeks.

Drug: Placebo

Interventions

Ivacaftor 150 milligram (mg) tablet orally twice daily for 24 weeks.

Also known as: Kalydeco, VX-770
Ivacaftor

Placebo matched to Ivacaftor tablet orally twice daily for 24 weeks.

Placebo

Eligibility Criteria

Age6 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Male or female with confirmed diagnosis of CF
  • Must have at least 1 allele of the R117H CFTR mutation
  • Percent predicted forced expiratory volume in 1 second (FEV1) 40 percent (%) to 90% (for subjects aged 12 years or older) or 40% to 105% (for subjects aged 6 to 11 years) predicted normal for age, sex, and height
  • years of age or older
  • Minimum weight of 15 kilogram (kg) at screening
  • Females of childbearing potential must not be pregnant
  • Willing to comply with contraception requirements

You may not qualify if:

  • CFTR gene mutation leading to CFTR channel with gating defect (that is, any 1 of the following mutations: G551D, G178R, G551S, S549N, S549R, G970R, G1244E, S1251N, S1255P, or G1349D)
  • History of any illness or condition that might confound the results of the study or pose an additional risk in administering ivacaftor to the subject
  • An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 4 weeks before the first dose of study drug
  • Abnormal liver function, at screening, defined as greater than or equal to (\>=) 3 time upper limit of normal (ULN), of any 3 or more of the following: serum aspartate transaminase (AST), serum alanine transaminase (ALT), gamma-glutamyl transpeptidase (GGT), serum alkaline phosphatase (ALP), total bilirubin
  • Colonization with organisms associated with a more rapid decline in pulmonary status (for example, Burkholderia cenocepacia, Burkholderia dolosa, and Mycobacterium abscessus) at screening
  • History of solid organ or hematological transplantation
  • History of alcohol, medication or illicit drug abuse within 1 year before the first dose of study drug
  • Ongoing participation in another therapeutic clinical study or prior participation in an investigational drug study within 30 days before screening
  • Any "non-CF-related" illness within 2 weeks before Day 1 (first dose of study drug). "Illness" was defined as an acute (serious or non-serious) condition (for example, gastroenteritis)
  • Use of any inhibitors or inducers of cytochrome (CYP) P450 3A

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (31)

Unknown Facility

Birmingham, Alabama, United States

Location

Unknown Facility

Palo Alto, California, United States

Location

Unknown Facility

Hartford, Connecticut, United States

Location

Unknown Facility

Tampa, Florida, United States

Location

Unknown Facility

Chicago, Illinois, United States

Location

Unknown Facility

Iowa City, Iowa, United States

Location

Unknown Facility

Lexington, Kentucky, United States

Location

Unknown Facility

Baltimore, Maryland, United States

Location

Unknown Facility

Boston, Massachusetts, United States

Location

Unknown Facility

Detroit, Michigan, United States

Location

Unknown Facility

Grand Rapids, Michigan, United States

Location

Unknown Facility

St Louis, Missouri, United States

Location

Unknown Facility

Omaha, Nebraska, United States

Location

Unknown Facility

New York, New York, United States

Location

Unknown Facility

Syracuse, New York, United States

Location

Unknown Facility

Valhalla, New York, United States

Location

Unknown Facility

Chapel Hill, North Carolina, United States

Location

Unknown Facility

Philadelphia, Pennsylvania, United States

Location

Unknown Facility

Pittsburgh, Pennsylvania, United States

Location

Unknown Facility

Charleston, South Carolina, United States

Location

Unknown Facility

Nashville, Tennessee, United States

Location

Unknown Facility

Dallas, Texas, United States

Location

Unknown Facility

Fort Worth, Texas, United States

Location

Unknown Facility

Houston, Texas, United States

Location

Unknown Facility

Salt Lake City, Utah, United States

Location

Unknown Facility

Richmond, Virginia, United States

Location

Unknown Facility

Seattle, Washington, United States

Location

Unknown Facility

Morgantown, West Virginia, United States

Location

Unknown Facility

Madison, Wisconsin, United States

Location

Unknown Facility

Belfast, United Kingdom

Location

Unknown Facility

Edinburgh, United Kingdom

Location

Related Publications (1)

  • Moss RB, Flume PA, Elborn JS, Cooke J, Rowe SM, McColley SA, Rubenstein RC, Higgins M; VX11-770-110 (KONDUCT) Study Group. Efficacy and safety of ivacaftor in patients with cystic fibrosis who have an Arg117His-CFTR mutation: a double-blind, randomised controlled trial. Lancet Respir Med. 2015 Jul;3(7):524-33. doi: 10.1016/S2213-2600(15)00201-5. Epub 2015 Jun 9.

MeSH Terms

Conditions

Cystic Fibrosis

Interventions

ivacaftor

Condition Hierarchy (Ancestors)

Pancreatic DiseasesDigestive System DiseasesLung DiseasesRespiratory Tract DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesInfant, Newborn, Diseases

Results Point of Contact

Title
Medical Monitor
Organization
Vertex Pharmaceuticals Incorporated

Study Officials

  • Richard Moss, MD

    Stanford University

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 5, 2012

First Posted

June 8, 2012

Study Start

July 1, 2012

Primary Completion

October 1, 2013

Study Completion

October 1, 2013

Last Updated

February 12, 2015

Results First Posted

February 12, 2015

Record last verified: 2015-01

Locations