Study of VX-809 in Cystic Fibrosis Subjects With the ∆F508-CFTR Gene Mutation

This study has been completed.
Sponsor:
Information provided by:
Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier:
NCT00865904
First received: March 18, 2009
Last updated: April 13, 2011
Last verified: April 2011
  Purpose

The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VX-809 in subjects with cystic fibrosis who have the ∆F508-CFTR gene mutation.


Condition Intervention Phase
Cystic Fibrosis
Drug: VX-809
Drug: VX-809 Placebo
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator)
Primary Purpose: Treatment
Official Title: A Randomized, Double-Blind, Placebo-Controlled, Multiple Dose Study of VX-809 to Evaluate Safety, Pharmacokinetics, and Pharmacodynamics of VX-809 in Cystic Fibrosis Subjects Homozygous for the ∆F508-CFTR Gene Mutation

Resource links provided by NLM:


Further study details as provided by Vertex Pharmaceuticals Incorporated:

Primary Outcome Measures:
  • Safety and tolerability assessments based on adverse events, hematology, clinical chemistry, urinalysis, electrocardiograms (ECGs), vital signs, and physical examinations. [ Time Frame: 28 days ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Pharmacodynamic assessments using spirometry (FVC, FEV1, and FEF25-75), sweat chloride, nasal potential difference (NPD), and the CF Questionnaire-Revised (CFQ-R), and evaluation of pharmacokinetic parameters. [ Time Frame: 28 days ] [ Designated as safety issue: No ]

Enrollment: 89
Study Start Date: March 2009
Study Completion Date: December 2009
Primary Completion Date: December 2009 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: 1 Drug: VX-809
Dose A VX-809 oral capsule once a day for 28 days
Other Name: CFTR corrector
Active Comparator: 2 Drug: VX-809
Dose B VX-809 oral capsule once a day for 28 days
Other Name: CFTR corrector
Active Comparator: 3 Drug: VX-809
Dose C VX-809 oral capsule once a day for 28 days
Other Name: CFTR corrector
Active Comparator: 4 Drug: VX-809
Dose D VX-809 oral capsule once a day for 28 days
Other Name: CFTR corrector
Placebo Comparator: 5 Drug: VX-809 Placebo
Placebo oral capsule once a day for 28 days
Other Name: CFTR corrector

Detailed Description:

Cystic fibrosis (CF) is an inherited disease resulting from defects to a gene known as the cystic fibrosis transmembrane conductance regulator (CFTR). CF affects approximately 70,000 children and adults worldwide (30,000 in the United States) and is the most common fatal genetic disease in persons of European descent. Despite progress in the treatment of CF with antibiotics and mucolytics, the predicted median age of survival for a person with CF is in the mid-30s. Though the disease affects multiple organs, most morbidity and mortality is caused by progressive loss of lung function.

This is a Phase 2, randomized, double-blind, placebo-controlled, multiple dose study of orally administered VX-809 in subjects with CF who are homozygous for the specific CFTR mutation known as ∆F508 or F508del. Enrollment is planned for approximately 90 subjects at approximately 22 clinical sites in the U.S., Canada, Belgium, Germany, and The Netherlands. Subjects will be randomized in a 4:1 ratio to receive 1 of 4 doses of VX-809 or placebo once a day for 28 days in a parallel design. Subjects will be outpatients during the study, except for overnight stays on Days 1 and 28.

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Confirmed diagnosis of CF with ∆F508-CFTR mutation in both alleles
  • FEV1 greater than or equal to 40% of predicted normal for age, gender, and height
  • Weight greater than or equal to 40 kg and body mass index greater than or equal to 18.5
  • Screening laboratory values, tests, and physical examination within acceptable ranges
  • Negative pregnancy test (for women of child-bearing potential)
  • Able and willing to follow contraceptive requirements
  • Willing to remain on a stable medication regimen for the duration of study participation

Exclusion Criteria:

  • History of any illness, or any ongoing acute illness, that could impact the safety of the study subject or may confound results of study
  • Pulmonary exacerbation or changes in therapy for pulmonary disease within 14 days before receiving the first dose of study drug
  • Impaired hepatic or renal function
  • History of organ or hematological transplant
  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: NCT00865904

  Show 25 Study Locations
Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
Investigators
Study Director: Medical Monitor Vertex Pharmaceuticals Incorporated
  More Information

No publications provided by Vertex Pharmaceuticals Incorporated

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Medical Monitor, Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT00865904     History of Changes
Other Study ID Numbers: VX08-809-101
Study First Received: March 18, 2009
Last Updated: April 13, 2011
Health Authority: United States: Food and Drug Administration
Canada: Health Canada
Germany: Federal Institute for Drugs and Medical Devices
Belgium: Federal Agency for Medicinal Products and Health Products
Netherlands: The Central Committee on Research Involving Human Subjects (CCMO)

Keywords provided by Vertex Pharmaceuticals Incorporated:
∆F508
F508del
Cystic Fibrosis Transmembrane Conductance Regulator
CFTR
Pancreatic diseases
Lung diseases
Genetic disease, inborn
Infant, newborn, diseases

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 August 01, 2014