Study of VX-809 in Cystic Fibrosis Subjects With the ∆F508-CFTR Gene Mutation
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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 |
- 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 ]
- 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 |
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
Show 25 Study Locations| 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 June 18, 2013