Rollover Study of VX-770 in Cystic Fibrosis Subjects

This study has been completed.
Sponsor:
Collaborator:
Cystic Fibrosis Foundation Therapeutics
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier:
NCT01117012
First received: May 3, 2010
Last updated: June 12, 2015
Last verified: June 2015
  Purpose

The primary objective of the study was to evaluate the safety of long-term VX-770 treatment in participants with cystic fibrosis (CF). The secondary objective of the study was to evaluate the efficacy of long-term VX-770 treatment in subjects with CF.


Condition Intervention Phase
Cystic Fibrosis
Drug: Ivacaftor
Phase 3

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: An Open-Label, Rollover Study to Evaluate the Long Term Safety and Efficacy of VX-770 in Subjects With Cystic Fibrosis

Resource links provided by NLM:


Further study details as provided by Vertex Pharmaceuticals Incorporated:

Primary Outcome Measures:
  • Number of Participants With Non-Serious Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: Study 105: Day 1 up to Week 168 ] [ Designated as safety issue: Yes ]
    Adverse Event: any untoward medical occurrence in a participant during the study, including any unfavorable and unintended sign, symptom, or disease whether or not it was considered to be study drug related. This included any newly occurring event or previous condition that increased in severity or frequency after obtaining informed consent and assent (where applicable). SAE: medical event or condition, which resulted in any of following, regardless of its relationship to the study drug: death, life threatening adverse experience, in-patient hospitalization/prolonged hospitalization, persistent/significant disability/incapacity, congenital anomaly/birth defect, important medical event. Non-Serious AEs included all AEs except SAEs.


Secondary Outcome Measures:
  • Annualized Rate of Decline From Study 105 Baseline in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1) Through Week 96 [ Time Frame: Study 105: Baseline through Week 96 ] [ Designated as safety issue: No ]
    FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Predicted FEV1 (for age, gender, and height) was calculated using the Knudson method. Baseline was defined as Study 105 Day 15.

  • Absolute Change From Study 105 Baseline in Percent Predicted FEV1 Through Week 96 [ Time Frame: Study 105: Baseline through Week 96 ] [ Designated as safety issue: No ]
    FEV1 is the volume of air that can forcibly be blown out in one second, after full inspiration. Predicted FEV1 (for age, gender, and height) was calculated using the Knudson method. Baseline was defined as the most recent measurement prior to intake of the first dose of study drug in Study 105. Absolute Change at Week 48 and Week 96 are reported.

  • Absolute Change From Study 105 Baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) Respiratory Domain Score Through Week 96 [ Time Frame: Study 105: Baseline through Week 96 ] [ Designated as safety issue: No ]
    The CFQ-R is a validated patient-reported outcome measuring health-related quality of life for subjects with cystic fibrosis. Respiratory domain assessed respiratory symptoms (for example, coughing, congestion, wheezing), score range: 0-100; higher scores indicating fewer symptoms and better health-related quality of life. Absolute Change at Week 48 and Week 96 are reported.

  • Annualized Pulmonary Exacerbation Event Rate [ Time Frame: Study 105: Day 1 through Week 96 ] [ Designated as safety issue: No ]
    Annualized event rate was calculated by regression with negative binomial distribution.

  • Annualized Duration of Pulmonary Exacerbation Events [ Time Frame: Study 105: Day 1 through Week 96 ] [ Designated as safety issue: No ]
  • Absolute Change From Study 105 Baseline in Weight Through Week 96 [ Time Frame: Study 105: Baseline through Week 96 ] [ Designated as safety issue: No ]
    Weight is a measurement of nutritional status. Absolute change in weight, measured in kilograms (kg), at Week 48 and Week 96 are reported.


Enrollment: 192
Study Start Date: July 2010
Study Completion Date: May 2014
Primary Completion Date: May 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: VX-770
VX-770 (ivacaftor) 150 milligram (mg) tablet orally twice daily (q12h).
Drug: Ivacaftor
Other Name: VX-770

Detailed Description:

This open-label, rollover study of orally administered VX-770 was conducted in participants with CF to evaluate the safety and efficacy of long-term VX-770 treatment. Participants who were previously enrolled in Study 102 (VX08-770-102/NCT00909532) and Study 103 (VX08-770-103/NCT00909727), and met certain criteria were eligible to enroll in this study (Study 105/VX08-770-105/NCT01117012).

  Eligibility

Ages Eligible for Study:   6 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Participants who have completed the assigned study treatment in Study 102 or Study 103
  2. Participants who are females of childbearing potential must have a negative urine pregnancy test on Day 1 (first dose of VX-770)
  3. Participants who are able to understand and comply with protocol requirements, restrictions, and instructions and likely to complete the study as planned, as judged by the investigator
  4. Participants of child bearing potential and who are sexually active must meet the contraception requirements
  5. Participants must sign the informed consent form (ICF), and where appropriate, assent must be obtained

Exclusion Criteria:

  1. Participants with a history of any illness or condition that, in the opinion of the investigator, might confound the results of the study or pose an additional risk in administering study drug to the subject
  2. Participants with a history of study treatment intolerance as observed in their previous VX-770 study that, in the opinion of the investigator, might pose an additional risk in administering study drug to the subject
  3. Participants who are pregnant, planning a pregnancy, breast-feeding, or not willing to follow contraception requirements
  4. Participants taking any inhibitors or inducers of Cytochrome P450 3A4 (CYP3A4), including certain herbal medications (for example, St. John's Wort) and grapefruit/grapefruit juice
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01117012

  Show 58 Study Locations
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
Cystic Fibrosis Foundation Therapeutics
Investigators
Principal Investigator: Edward McKone, MD St. Vincent's University Hospital
  More Information

No publications provided by Vertex Pharmaceuticals Incorporated

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT01117012     History of Changes
Other Study ID Numbers: VX08-770-105, PERSIST
Study First Received: May 3, 2010
Results First Received: June 1, 2015
Last Updated: June 12, 2015
Health Authority: United States: Food and Drug Administration
United Kingdom: Medicines and Healthcare Products Regulatory Agency
Ireland: Irish Medicines Board
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
Germany: Federal Institute for Drugs and Medical Devices
Czech Republic: State Institute for Drug Control
Australia: Department of Health and Ageing Therapeutic Goods Administration
Canada: Health Canada

Additional relevant MeSH terms:
Cystic Fibrosis
Fibrosis
Digestive System Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Lung Diseases
Pancreatic Diseases
Pathologic Processes
Respiratory Tract Diseases

ClinicalTrials.gov processed this record on July 30, 2015