NCT01262352

Brief Summary

The purpose of this study is to evaluate the effect of ivacaftor (VX-770) on lung clearance index (LCI) in subjects aged 6 years and older with cystic fibrosis (CF) who have the G551D-CFTR mutation on at least 1 allele.

Trial Health

90
On Track

Trial Health Score

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

Enrollment
21

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Jan 2011

Shorter than P25 for phase_2

Geographic Reach
3 countries

8 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

December 15, 2010

Completed
2 days until next milestone

First Posted

Study publicly available on registry

December 17, 2010

Completed
15 days until next milestone

Study Start

First participant enrolled

January 1, 2011

Completed
10 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 1, 2011

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

November 1, 2011

Completed
1.2 years until next milestone

Results Posted

Study results publicly available

January 30, 2013

Completed
Last Updated

February 11, 2013

Status Verified

February 1, 2013

Enrollment Period

10 months

First QC Date

December 15, 2010

Results QC Date

October 31, 2012

Last Update Submit

February 4, 2013

Conditions

Cystic Fibrosis

Outcome Measures

Primary Outcomes (1)

  • Absolute Change From Baseline in Lung Clearance Index (LCI)

    Lung clearance index (LCI) is a measure of ventilation inhomogeneity that is derived from a multiple-breath washout test. The LCI was calculated as the number of lung volume turnovers (cumulative expired volume divided by the functional residual capacity \[FRC\]) required to reduce end-tidal SF6 concentration to 1/40th of the starting value.

    Baseline through Day 29

Secondary Outcomes (3)

  • Absolute Change From Baseline in Percent Predicted FEV1

    Baseline through Day 29

  • Change From Baseline in Sweat Chloride

    Baseline through Day 29

  • Change From Baseline in CF Questionnaire-Revised (CFQ-R) Score (Respiratory Domain Score, Pooled)

    Baseline through Day 29

Study Arms (2)

Treatment Sequence 1

EXPERIMENTAL

Ivacaftor administered in Treatment Period 1 and placebo administered in Treatment Period 2.

Drug: IvacaftorDrug: Placebo

Treatment Sequence 2

EXPERIMENTAL

Placebo administered in Treatment Period 1 and ivacaftor administered in Treatment Sequence 2.

Drug: IvacaftorDrug: Placebo

Interventions

IvacaftorDRUG

150 mg tablet, oral use, twice daily every 12 hours (q12h)

Treatment Sequence 1Treatment Sequence 2
PlaceboDRUG

Tablet, oral use, twice daily every 12 hours (q12h)

Treatment Sequence 1Treatment Sequence 2

Eligibility Criteria

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

You may qualify if:

  • Male or female subjects with confirmed diagnosis of CF
  • Must have the G551D-CFTR mutation in at least 1 allele
  • FEV1 \>90% of predicted normal for age, gender, and height

You may not qualify if:

  • Ongoing participation in another therapeutic clinical study or prior participation in an investigational drug study within the 30 days prior to screening
  • Use of inhaled hypertonic saline treatment within 2 weeks of the Period 1, Day 1 visit

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (8)

Unknown Facility

Stanford, California, United States

Location

Unknown Facility

Iowa City, Iowa, United States

Location

Unknown Facility

Durham, North Carolina, United States

Location

Unknown Facility

Pittsburgh, Pennsylvania, United States

Location

Unknown Facility

Toronto, Ontario, Canada

Location

Unknown Facility

Belfast, United Kingdom

Location

Unknown Facility

Edinburgh, United Kingdom

Location

Unknown Facility

London, United Kingdom

Location

Related Publications (1)

  • Davies J, Sheridan H, Bell N, Cunningham S, Davis SD, Elborn JS, Milla CE, Starner TD, Weiner DJ, Lee PS, Ratjen F. Assessment of clinical response to ivacaftor with lung clearance index in cystic fibrosis patients with a G551D-CFTR mutation and preserved spirometry: a randomised controlled trial. Lancet Respir Med. 2013 Oct;1(8):630-638. doi: 10.1016/S2213-2600(13)70182-6. Epub 2013 Sep 10.

    PMID: 24461666DERIVED

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
medicalinfo@vrtx.com
617-341-6777

Study Officials

  • Jane Davies

    Royal Brompton Hospital and Imperial College

    PRINCIPAL INVESTIGATOR

  • Felix Ratjen

    The Hospital for Sick Children

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

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

Study Record Dates

First Submitted

December 15, 2010

First Posted

December 17, 2010

Study Start

January 1, 2011

Primary Completion

November 1, 2011

Study Completion

November 1, 2011

Last Updated

February 11, 2013

Results First Posted

January 30, 2013

Record last verified: 2013-02

Locations

CA(1)US(4)GB(3)