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 has been completed.
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier:
NCT01807949
First received: March 4, 2013
Last updated: September 2, 2015
Last verified: September 2015
Results First Received: August 1, 2015  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Double Blind (Subject, Investigator);   Primary Purpose: Treatment
Condition: Cystic Fibrosis, Homozygous for the F508del CFTR Mutation
Interventions: Drug: Placebo
Drug: Lumacaftor Plus Ivacaftor Combination
Drug: Ivacaftor

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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Reporting Groups
  Description
Placebo Placebo matched to lumacaftor (LUM, VX-809) and ivacaftor (IVA, VX-770) tablet every 12 hours (q12h), up to Week 24.
LUM 600 mg qd/IVA 250 mg q12h LUM 600 milligram (mg) plus IVA 250 mg fixed-dose combination (FDC) tablet in the morning and IVA 250 mg film-coated tablet in the evening, up to Week 24.
LUM 400 mg q12h/ IVA 250 mg q12h LUM 400 mg plus IVA 250 mg FDC tablet in the morning and in the evening, up to Week 24.

Participant Flow:   Overall Study
    Placebo     LUM 600 mg qd/IVA 250 mg q12h     LUM 400 mg q12h/ IVA 250 mg q12h  
STARTED     187     187     189  
COMPLETED     185     180     180  
NOT COMPLETED     2     7     9  
Adverse Event                 1                 2                 2  
Withdrawal by Subject                 1                 2                 2  
Non-Compliance                 0                 0                 1  
Undefined                 0                 1                 2  
Randomized But Not Treated                 0                 2                 2  



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
Full Analysis Set (FAS) included all randomized participants who received any amount of study drug.

Reporting Groups
  Description
Placebo Placebo matched to LUM and IVA tablet q12h, up to Week 24.
LUM 600 mg qd/IVA 250 mg q12h LUM 600 mg plus IVA 250 mg FDC tablet in the morning and IVA 250 mg film-coated tablet in the evening, up to Week 24.
LUM 400 mg q12h/ IVA 250 mg q12h LUM 400 mg plus IVA 250 mg FDC tablet in the morning and in the evening, up to Week 24.
Total Total of all reporting groups

Baseline Measures
    Placebo     LUM 600 mg qd/IVA 250 mg q12h     LUM 400 mg q12h/ IVA 250 mg q12h     Total  
Number of Participants  
[units: participants]
  187     185     187     559  
Age  
[units: years]
Mean (Standard Deviation)
  25.7  (10.02)     24.3  (8.31)     25.0  (9.03)     25.0  (9.16)  
Gender  
[units: participants]
       
Female     97     96     98     291  
Male     90     89     89     268  



  Outcome Measures
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1.  Primary:   Absolute Change From Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) at Week 24   [ Time Frame: Baseline, Week 16 and 24 ]

2.  Secondary:   Relative Change From Baseline in Percent Predicted FEV1 at Week 24   [ Time Frame: Baseline, Week 16 and 24 ]

3.  Secondary:   Absolute Change From Baseline in Body Mass Index (BMI) at Week 24   [ Time Frame: Baseline, Week 24 ]

4.  Secondary:   Absolute Change From Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score at Week 24   [ Time Frame: Baseline, Week 24 ]

5.  Secondary:   Percentage of Participants With Response Based on Percent Predicted FEV1   [ Time Frame: Week 16 and 24 ]

6.  Secondary:   Number of Pulmonary Exacerbation Events   [ Time Frame: through Week 24 ]

7.  Secondary:   Absolute Change From Baseline in Weight at Week 24   [ Time Frame: Baseline, Week 24 ]

8.  Secondary:   Absolute Change From Baseline in BMI-for-age Z-score at Week 24   [ Time Frame: Baseline, Week 24 ]

9.  Secondary:   Time-to-First Pulmonary Exacerbation   [ Time Frame: through Week 24 ]

10.  Secondary:   Percentage of Participants With At Least 1 Pulmonary Exacerbation Event   [ Time Frame: through Week 24 ]

11.  Secondary:   Absolute Change From Baseline in Euro Quality of Life Scale (EuroQol) 5-Dimension-3 Level (EQ-5D-3L) Index Score at Week 24   [ Time Frame: Baseline, Week 24 ]

12.  Secondary:   Absolute Change From Baseline in EQ-5D-3L VAS Score at Week 24   [ Time Frame: Baseline, Week 24 ]

13.  Secondary:   Absolute Change From Baseline in Treatment Satisfaction Questionnaire for Medication (TSQM) Domain Scores at Week 24   [ Time Frame: Baseline, Week 24 ]

14.  Secondary:   Number of Participants With Treatment-Emergent Adverse Events (AEs) and Treatment-Emergent Serious Adverse Events (SAEs)   [ Time Frame: up to Week 28 ]

15.  Secondary:   Pre-dose Concentration (Ctrough), Average Pre-dose Concentration (Ctrough,Avg), 3 to 6 Hours Post-dose Concentration (C3-6h), and Average 3 to 6 Hours Post-dose Concentration (C3-6h,Avg)   [ Time Frame: For C3-6h: 3 to 6 hours after morning dose on Day 1 and 15, Week 4 and 8; For C3-6h,avg 3 to 6 hours after morning dose on Day 15, Week 4 and 8; For Ctrough and Ctrough,avg: before morning dose on Week 4, 8, and 16 ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information
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Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: Medical Monitor
Organization: Vertex Pharmaceuticals Incorporated
phone: 617-341-6777
e-mail: medicalinfo@vrtx.com


Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT01807949     History of Changes
Other Study ID Numbers: VX12-809-104
Study First Received: March 4, 2013
Results First Received: August 1, 2015
Last Updated: September 2, 2015
Health Authority: United States: Food and Drug Administration
Canada: Health Canada
Spain: Ministry of Health and Consumption
Belgium: Federal Agency for Medicinal Products and Health Products
Denmark: Danish Medicines Agency
Austria: Austrian Medicines and Medical Devices Agency
Germany: Federal Institute for Drugs and Medical Devices
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
United Kingdom: Medicines and Healthcare Products Regulatory Agency
Australia: Department of Health and Ageing Therapeutic Goods Administration