A Study of Lumacaftor in Combination With Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Who Are Homozygous for the F508del-CFTR Mutation (TRANSPORT)

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier:
NCT01807949
First received: March 4, 2013
Last updated: February 24, 2014
Last verified: February 2014

March 4, 2013
February 24, 2014
March 2013
May 2014   (final data collection date for primary outcome measure)
Absolute change in percent predicted forced expiratory volume in 1 second (FEV1) [ Time Frame: From Baseline at Week 24 ] [ Designated as safety issue: No ]
Relative change in percent predicted forced expiratory volume in 1 second (FEV1) [ Time Frame: From Baseline through Week 24 ] [ Designated as safety issue: No ]
Complete list of historical versions of study NCT01807949 on ClinicalTrials.gov Archive Site
  • Relative change in percent predicted FEV1 [ Time Frame: From Baseline at Week 24 ] [ Designated as safety issue: No ]
  • Absolute change in body mass index (BMI) [ Time Frame: From Baseline through Week 24 ] [ Designated as safety issue: No ]
  • Number of pulmonary exacerbations [ Time Frame: Through Week 24 ] [ Designated as safety issue: No ]
  • Absolute change in Cystic Fibrosis Questionnaire - Revised (CFQ R) respiratory domain score [ Time Frame: From Baseline at Week 24 ] [ Designated as safety issue: No ]
  • Absolute change in BMI z-score [ Time Frame: From Baseline at Week 24 ] [ Designated as safety issue: No ]
  • Absolute change in body weight [ Time Frame: From Baseline at Week 24 ] [ Designated as safety issue: No ]
  • Time-to-first pulmonary exacerbation [ Time Frame: Through Week 24 ] [ Designated as safety issue: No ]
  • Event of having at least 1 pulmonary exacerbation [ Time Frame: Through Week 24 ] [ Designated as safety issue: No ]
  • Absolute change in EuroQol 3 Level (EQ 5D 3L) score [ Time Frame: From Baseline at Week 24 ] [ Designated as safety issue: No ]
  • Absolute change in Treatment Satisfaction Questionnaire for Medication (TSQM) domains [ Time Frame: From Baseline at Week 24 ] [ Designated as safety issue: No ]
  • Safety and tolerability assessments based on adverse events (AEs), clinical laboratory values (hematology, serum chemistry, coagulation studies, and urinalysis), standard digital electrocardiograms (ECGs), vital signs, and pulse oximetry [ Time Frame: From Baseline and up to 28 Weeks ] [ Designated as safety issue: Yes ]
  • PK parameters of lumacaftor, M28 lumacaftor, ivacaftor, M1 ivacaftor, and M6 ivacaftor [ Time Frame: Baseline and Week 16 ] [ Designated as safety issue: No ]
  • Absolute change in body mass index (BMI) [ Time Frame: From Baseline and at Week 24 ] [ Designated as safety issue: No ]
  • Number of pulmonary exacerbations [ Time Frame: From Baseline through Week 24 ] [ Designated as safety issue: No ]
  • Absolute change in Cystic Fibrosis Questionnaire-Revised (CFQ R) respiratory domain score [ Time Frame: From Baseline through Week 24 ] [ Designated as safety issue: No ]
  • Safety and tolerability assessments based on adverse events (AEs), clinical laboratory values (hematology, serum chemistry, coagulation studies, and urinalysis), standard digital electrocardiograms (ECGs), pulse oximetry, and vital signs [ Time Frame: Baseline and up to 28 weeks ] [ Designated as safety issue: Yes ]
Not Provided
Not Provided
 
A Study of Lumacaftor in Combination With Ivacaftor in Cystic Fibrosis Subjects Aged 12 Years and Older Who Are Homozygous for the F508del-CFTR Mutation (TRANSPORT)
A Phase 3, Randomized, Double Blind, Placebo Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of Lumacaftor in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Homozygous for the F508del CFTR Mutation

The purpose of this study is to evaluate the efficacy and safety of lumacaftor in combination with ivacaftor in persons 12 years and older with Cystic Fibrosis who are homozygous for the F508del mutation.

Not Provided
Interventional
Phase 3
Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Investigator)
Primary Purpose: Treatment
Cystic Fibrosis, Homozygous for the F508del CFTR Mutation
  • Drug: Ivacaftor
    Other Name: VX-770
  • Drug: Lumacaftor
    Other Name: VX-809
  • Drug: Ivacaftor Placebo
  • Drug: Lumacaftor Placebo
  • Experimental: Treatment Arm 1
    Subjects randomized to study drug will take 600 mg of lumacaftor once daily (qd) and 250 mg of ivacaftor every 12 hours (q12h)
    Interventions:
    • Drug: Ivacaftor
    • Drug: Lumacaftor
  • Experimental: Treatment Arm 2
    Subjects randomized to study drug will take 400 mg of lumacaftor every 12 hours (q12h) and 250 mg of ivacaftor every 12 hours (q12h)
    Interventions:
    • Drug: Ivacaftor
    • Drug: Lumacaftor
  • Experimental: Treatment Arm 3
    Subjects randomized to placebo will remain on placebo through 24 weeks. Subjects will be given tablets that match both lumacaftor and ivacaftor and will follow the same dosing regimen as subjects receiving the study drug
    Interventions:
    • Drug: Ivacaftor Placebo
    • Drug: Lumacaftor Placebo
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
563
Not Provided
May 2014   (final data collection date for primary outcome measure)

Inclusion Criteria:

  • Males and females, aged 12 years or older on the date of informed consent or, where appropriate, date of assent
  • Confirmed diagnosis of CF
  • Homozygous for the F508del CFTR mutation
  • FEV1 ≥40% and ≤90% of predicted normal for age, sex, and height
  • Willing to remain on a stable CF medication regimen through Week 24 or, if applicable, the Safety Follow up Visit

Exclusion Criteria:

  • An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 4 weeks before first dose of study drug
  • History of solid organ or hematological transplantation
  • History of alcohol or drug abuse in the past year
  • Ongoing or prior participation in an investigational drug study (including studies investigating lumacaftor and/or ivacaftor) within 30 days of screening.
  • Use of strong inhibitors, moderate inducers, or strong inducers of CYP3A , including consumption of certain herbal medications (e.g., St. John's Wort) and certain fruit and fruit juices within 14 days before Day 1 of dosing
Both
12 Years to 65 Years
No
Contact information is only displayed when the study is recruiting subjects
United States,   Australia,   Austria,   Belgium,   Canada,   Denmark,   France,   Germany,   Spain,   United Kingdom
 
NCT01807949
VX12-809-104
Yes
Vertex Pharmaceuticals Incorporated
Vertex Pharmaceuticals Incorporated
Not Provided
Not Provided
Vertex Pharmaceuticals Incorporated
February 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP