VX-770 Expanded Access Program (EAP)
VX-770, a compound being developed by Vertex Pharmaceuticals Incorporated (Vertex) for the
treatment of CF, is an orally bioavailable small molecule that targets the underlying defect
in CF, the dysfunctional CFTR protein. In Phase 3 studies of VX-770 in patients with CF and
a G551D CFTR mutation, improvements in CFTR function (measured by reduction in sweat
chloride concentration) and improvements in lung function were observed.
Patients who are interested in the VX-770 Expanded Access should contact their CF physician
Physicians interested in participating as a site should contact 800-745-4484.
Approved for marketing | Cystic Fibrosis | Site Unknown
A Prospective, 5-year Registry Study to Monitor the Susceptibility to Aztreonam of Pseudomonas aeruginosa (PA) Isolates from Patients with Cystic Fibrosis in the United States [AIR-CF5]
Pseudomonas aeruginosa (PA) is the most significant bacteria in patients with cystic fibrosis (CF). Once the PA is in the lungs it is very difficult to get rid of the bacteria. Therefore, CF patients must take medication either by oral or inhaled antibiotics for a very long time. The antibiotic called tobramycin inhalation solution (TOBI; TIS) has been on the market since 1997 and is widely used for management of CF in patients with PA;up to 69% of PA-positive CF patients were prescribed at least one course of TIS in 2008. In addition to TIS, IV tobramycin is also commonly used to treat acute lung infections of CF lung disease. Therefore, exposure in the CF population is extensive.A second aerosolized antibiotic is now available in the US. Cayston (aztreonam forinhalation solution; AZLI) is an inhaled monobactam antibacterial approved by the FDA in February 2010. It is indicated to improve respiratory symptoms in CF patients with PA. Undoubtedly, the introduction of a new inhaled antibiotic will change the profile of PA that is seen in CF patients with CF. The goal of this study is to track these changes over five years in a representational cohort of the US patient population. The main endpoint will be to evaluate the proportion of subjects who have sputum sample with PA. The secondary endpoints include:1) Annual change from baseline in lung function measured by the spirometry test. 2) The annual number of hospitalizations and the total number of hospitalizations at theend of each year3) The annual number of days hospitalized and the total number of hospitalization daysat the end of each year4) Annual change from the start of the study the patient's body weight change.5) Annual number of Cayston treatment courses per patient and the total number ofCayston treatment courses at the end of each year in patients that used CaystonNo formal hypotheses will be tested in this study. A sample size of 500 subjects was selected in order to provide a representative cross-section of subjects and to ensure that at least 300 subjects will have available data at the end of 5 years. Assuming the highest variability estimate of 0.25, the proportion of subjects whose least susceptible PA isolate has a 4-fold increase in aztreonam MIC over 1 year and is above the parenteral breakpoint (> 8 g/mL) can be estimated within the following ranges using a 95% confidence interval (CI).
A Point Prevalence Study to Evaluate the Prevalence of Antibodies to Selected Porcine Viruses in Patients with Cystic Fibrosis Who Are Receiving Porcine-Derived Pancreatic Enzyme Replacement Therapy: A Harmonized Protocol Across Sponsors
Pancreatic enzyme replacement therapy (PERT) with pig-derived pancreatic enzyme products is the cornerstone of nutritional management of pancreatic exocrine insufficiency (PEI) due to cystic fibrosis (CF) or other conditions. Pancreatic enzymes are extracted from the pig's pancreatic tissue. The pig populations are known to harbor certain viruses, some of which may have the potential to infect humans. Therefore, the possibility of contamination of starting materials with pig viruses capable of infecting humans must be considered. While no cases of transmission of an infectious illness associated with the use of pig pancreatic extracts have been reported to date, there remains a theoretical risk for transmission of viral disease, including diseases caused by an unidentified viruses. This study will be conducted in the United States (US) to establish the prevalence for antibodies to hepatitis E virus (HEV), pig parvovirus (PPV) and porcine circovirus 2 (PCV2) in a statistically representative cross-section of the CF population taking PERTs compared with age- and geography matched unexposed control patients. Assays for antibodies to PPV and PCV2. The following information will be collected:Screening and matching of Controls log Demographic data Medical historyTransfusion history PERT history (for CF patients)Diet history (vegetarian and vegan diets), including duration History of potential exposure to pig viruses (via questionnaire):Travel to or living in regions endemic for HEV for more than 1 week, including birthplace in such regions Past and present household pets (dogs, cats, other) Past or present consumption of organ meat (liver, kidney etc), and usual frequency of consumptionOccupational exposure (e.g., butcher or pig farmer) or parental occupationalexposure Use and frequency of injectable drugs Use and frequency of dietary supplements containing organ extracts such as pancreas, liver and brain Ever resided on a farm Specific data on blood sample collection and storage This study has 80% power and two-sided 5% Type I error to identify an antibody prevalence signal associated with PERT exposure characterized by an absolute increase in the point prevalence as well as the odds ratio for a range of antibody prevalence.
PROSPECTIVE EVALUATION OF BIOMARKERS OF AMINOGLYCOSIDE NEPHROTOXICITY IN ADULT PATIENTS WITH CYSTIC FIBROSIS
Aminoglycoside (a specific classs of antibiotics) are a key component of antibiotic therapy for treatment of acute lung infections in patients with cystic fibrosis. One of the major limiting factors associated with the aminoglycosides use is kidney damage. Although blood creatinine levels are frequently monitored, significant elevations only appear after substantial kidney damage has already occurred. The purpose of the study will evaluate the sensitivity and specificity new kidney blood and urine tests on identification of kidney damage. The study population is adult patients with cystic fibrosis that will be recruited to participate: 1) participants hospitalized for treatment of an acute lung infection and prescribed a beta-lactam (specific class of antibiotics), an aminoglycoside or fluoroquinolone antibiotic, and 2) outpatient controls (CF partcipants not experiencing a lung infection and not receiving intravenous antibiotics). These are the control subjects.Participants will be grouped into one of 3 arms of the study. A- beta-lactam and aminoglycoside, B-beta-lactam and fluroquinolone, C-No antibiotic stable CF participants.The study endpoint is to determine the sensitivity, specificity, and negative and positive predictive value of the new kidney blood/urine tests called a biomarkers. The kidney biomarkers may help in early identification of kidney injury in patients receiving aminoglycoside antibiotics.Statistics: The sensitivity to identify subjects treated with a known kidney damaging drug (aminoglycoside) will be determined sequentially using the pre-specified blinded adjudicated method that incorporates urine biomarker information and the conventional method using only sCr and BUN. The sensitivity of each method is determined as the number of true positives divided by the number of samples from aminoglycoside treated patient (true positives + false negatives). The specificity of each method is determined as the number of true negatives divided by the number of samples from control patients (true negative + false positive).