Safety Study of VX 770 in Subjects With Cystic Fibrosis - Full Text View

Sponsor:	Vertex Pharmaceuticals Incorporated
Collaborator:	Cystic Fibrosis Foundation
Information provided by:	Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier:	NCT00457821

Purpose

Safety study of VX 770 in subjects with Cystic Fibrosis.

Condition	Intervention	Phase
Cystic Fibrosis	Drug: VX-770 Drug: Placebo	Phase II

Study Type:	Interventional
Study Design:	Allocation: Randomized Endpoint Classification: Safety Study Intervention Model: Crossover Assignment Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor) Primary Purpose: Treatment
Official Title:	A Phase 2a, Randomized, Double-Blind, Placebo-Controlled Study of VX-770 to Evaluate Safety, Pharmacokinetics, and Biomarkers of CFTR Activity in Cystic Fibrosis (CF) Subjects With Genotype G551D

Resource links provided by NLM:

Genetics Home Reference related topics: cystic fibrosis Help Me Understand Genetics

MedlinePlus related topics: Cystic Fibrosis

U.S. FDA Resources

Further study details as provided by Vertex Pharmaceuticals Incorporated:

Primary Outcome Measures:

Safety and tolerability assessments based on clinical evaluations, laboratory assessments, and adverse events during administration of VX-770 [ Time Frame: up to 28 days ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:

Change in Nasal Potential Difference from baseline [ Time Frame: up to 28 days ] [ Designated as safety issue: No ]
Change in Sweat Chloride from baseline [ Time Frame: up to 28 days ] [ Designated as safety issue: No ]
Change in Forced Expiratory Volume in 1 Second (FEV1) from baseline [ Time Frame: up to 28 days ] [ Designated as safety issue: No ]
PK Parameters, including concentration and exposure, of VX-770 [ Time Frame: up to 28 days ] [ Designated as safety issue: No ]
Cystic Fibrosis Questionnaire (CFQ-R, Part 2 only) [ Time Frame: up to 28 days ] [ Designated as safety issue: No ]

Enrollment:	38
Study Start Date:	April 2007
Study Completion Date:	August 2008
Primary Completion Date:	August 2008 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Experimental: 1 VX-770 dose A or placebo	Drug: VX-770 tablet Drug: Placebo tablet
Experimental: 2 VX-770 dose B or placebo	Drug: VX-770 tablet Drug: Placebo tablet
Experimental: 3 VX-770 dose C or placebo	Drug: VX-770 tablet Drug: Placebo tablet

Eligibility

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

Criteria

Inclusion Criteria:

Confirmed diagnosis of Cystic Fibrosis
G551D mutation in at least 1 allele (any known or unknown mutations allowed in second allele with the exception of R117H or 2789+5G-->A mutation).
Age 18 years or older.
FEV1 40% of predicted normal for age, gender, and height (Hankinson Standards): pre-bronchodilator value.
Willing to use 2 effective methods of birth control including 1 barrier method during the study and for 90days after the last dose of study drug.
Willing to remain on a stable medication regimen for the duration of study participation.

Exclusion Criteria:

Ongoing acute illness including acute respiratory or lower respiratory infections.
Pregnant, planning a pregnancy, or breast-feeding.
Abnormal liver function ≥3x upper limit of normal: (AST, ALT, GGT, ALP, total bilirubin).
History of abnormal renal function, prolonged QT intervals, solid organ or hematological transplantation.
History of alcohol abuse or drug addiction (including cannabis, cocaine, opiates).
Ongoing participation in another therapeutic clinical trial, or prior participation in an investigational drug without appropriate washout.
Unable to have nasal potential difference (NPD) performed due to physical or other constraints.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00457821

Locations

United States, Alabama
University of Alabama Hospital
Birmingham, Alabama, United States
United States, California
Stanford University Medical Center
Palo Alto, California, United States, 94304
United States, Colorado
Children's Hospital of Denver
Denver, Colorado, United States
United States, Iowa
Roy J. and Lucille A. Carver College of Medicine
Iowa City, Iowa, United States
United States, Maryland
Johns Hopkins Hospital
Baltimore, Maryland, United States
United States, Massachusetts
Children's Hospital of Boston
Boston, Massachusetts, United States, 02215
Mass. General Hospital
Boston, Massachusetts, United States, 02111
United States, Minnesota
Minnesota CF Center
Minneapolis, Minnesota, United States
United States, North Carolina

Chapel Hill, North Carolina, United States
United States, Ohio
Rainbow Babies and Children's Hospital
Cleveland, Ohio, United States
United States, Pennsylvania

Philadelphia, Pennsylvania, United States
Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, United States, 15213
United States, Washington
Dr. Moira Aitken
Seattle, Washington, United States, 98195

Sponsors and Collaborators

Vertex Pharmaceuticals Incorporated

Cystic Fibrosis Foundation

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):

Accurso FJ, Rowe SM, Clancy JP, Boyle MP, Dunitz JM, Durie PR, Sagel SD, Hornick DB, Konstan MW, Donaldson SH, Moss RB, Pilewski JM, Rubenstein RC, Uluer AZ, Aitken ML, Freedman SD, Rose LM, Mayer-Hamblett N, Dong Q, Zha J, Stone AJ, Olson ER, Ordoñez CL, Campbell PW, Ashlock MA, Ramsey BW. Effect of VX-770 in persons with cystic fibrosis and the G551D-CFTR mutation. N Engl J Med. 2010 Nov 18;363(21):1991-2003.

Responsible Party:	Robert Kauffman, M.D., Ph.D., Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier:	NCT00457821 History of Changes
Other Study ID Numbers:	VX06-770-101
Study First Received:	April 5, 2007
Last Updated:	October 11, 2010
Health Authority:	United States: Food and Drug Administration

Keywords provided by Vertex Pharmaceuticals Incorporated:

G551D mutation

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 February 09, 2012