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Study of VX-770 in Cystic Fibrosis Subjects

This study is ongoing, but not recruiting participants.
Sponsor:
Collaborator:
Cystic Fibrosis Foundation
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier:
NCT01117012
First received: May 3, 2010
Last updated: January 3, 2012
Last verified: January 2012
History of Changes
  Purpose

Cystic Fibrosis (CF) is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The encoded protein, CFTR, is an epithelial chloride ion channel responsible for aiding in the regulation of salt and water absorption and secretion in various tissues. Although the disease affects multiple organs, the leading cause of mortality is the progressive loss of lung function. Obstruction of airways with thick mucous, chronic bacterial infection of the airways, and inflammatory response are all thought to play a role in causing lung damage. Through its function as a chloride channel, CFTR is believed to be integral in epithelial ion and water transport and hence, maintaining the normal hydration of lung secretions.

VX-770 is a potent and selective potentiator of wild-type, G551D, F508del, and R117H forms of human CFTR. Based on in vitro studies and pharmacologic, pharmacokinetic (PK), and safety profiles, VX-770 has been selected for clinical development as a possible treatment for patients with CF.

The current study will enroll subjects with CF who have completed Study VX08-770-102 (Study 102) and Study VX08-770-103 (Study 103) to further evaluate the safety and efficacy of long term VX-770 treatment. Patients who were previously enrolled in Study 102 and Study 103; and have met certain criteria are eligible to enroll in this study. Study VX08-770-105 (Study 105) also offers an opportunity for subjects who received placebo in Study 102 and Study 103 to receive VX-770 treatment.


Condition Intervention Phase
Cystic Fibrosis
 Drug: VX-770
 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:

MedlinePlus related topics: Cystic Fibrosis
Drug Information available for: Ivacaftor
U.S. FDA Resources

Further study details as provided by Vertex Pharmaceuticals Incorporated:

Primary Outcome Measures:
  • To evaluate the safety of long-term VX 770 treatment in subjects with CF [ Time Frame: 96 weeks ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Rate of decline in percent predicted forced expiratory volume in 1 second (FEV1) [ Time Frame: 96 weeks ] [ Designated as safety issue: No ]
  • Absolute change from Day 1 of Study VX08-770-105 (Study 105) in FEV1 [ Time Frame: 96 weeks ] [ Designated as safety issue: No ]
  • Absolute change from Day 1 of previous VX-770 study in FEV1 [ Time Frame: 96 weeks ] [ Designated as safety issue: No ]
  • Change from Day 1 of Study 105 in Cystic Fibrosis Questionnaire-Revised (CFQ-R) [ Time Frame: 96 weeks ] [ Designated as safety issue: No ]
  • Change from Day 1 of previous VX-770 study in CFQ-R [ Time Frame: 96 weeks ] [ Designated as safety issue: No ]
  • Pulmonary exacerbations [ Time Frame: 96 weeks ] [ Designated as safety issue: No ]
  • Rate of change in weight [ Time Frame: 96 weeks ] [ Designated as safety issue: No ]

Estimated Enrollment: 200
Study Start Date: July 2010
Estimated Study Completion Date: May 2013
Estimated Primary Completion Date: April 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: VX-770 Drug: VX-770
150 mg tablet, oral use, twice daily every 12 hours (q12h)

Detailed Description:

This open-label, rollover study of orally administered VX-770 will be conducted in subjects with CF to evaluate the safety and efficacy of long-term VX-770 treatment. Patients who were previously enrolled in Study 102 and Study 103; and have met certain criteria are eligible to enroll in this study. In Study 105, the treatment duration in the countries in which the study is conducted will be the sooner of approximately 96 weeks or until VX 770 is commercially available in each respective country.

  Eligibility

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

Inclusion Criteria:

  1. Subjects who have completed the assigned study treatment Study 102 or Study 103
  2. Subjects who are females of childbearing potential must have a negative urine pregnancy test on Day 1 (first dose of VX-770).
  3. Subjects 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. Subjects of child bearing potential and who are sexually active must meet the contraception requirements.
  5. Subjects must sign the informed consent form (ICF), and where appropriate, assent must be obtained.

Exclusion Criteria:

  1. Subjects 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. Subjects 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. Subjects who are pregnant, planning a pregnancy, breast-feeding, or not willing to follow contraception requirements.
  4. Subjects taking any inhibitors or inducers of CYP3A4, including certain herbal medications (e.g., St. John's Wort) and grapefruit/grapefruit juice.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01117012

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

No publications provided

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
Last Updated: January 3, 2012
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
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
 Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Pathologic Processes

ClinicalTrials.gov processed this record on May 23, 2013