Richard B. Moss
Vertex 770-102 Study
Contact Information
Stanford University School of Medicine 300 Pasteur Drive Stanford, CA 94305Brief
This study would evaluate the efficacy of a study drug, VX-770, after 48 weeks of treatment in subjects with cystic fibrosis (CF) who have the G551D-cystic fibrosis transmembrane conductance regulator (CFTR) mutation on at least 1 allele.
Recruiting Status:
RecruitingStanford Recruiting Status:
RecruitingCondition(s):
Intervention(s):
- Drug: VX-770
Phase:
Phase 3Eligibility
Ages Eligible for Study:
12 years to Any AgeGenders Eligible for Study:
Male and FemaleHealth of Volunteers:
People with the conditions listed in this trial can participate as controls.Key Inclusion Criteria:
1. Male or female with confirmed diagnosis of CF,22 defined as:
? a sweat chloride value ≥60 mmol/L by quantitative pilocarpine iontophoresis OR a sweat sodium ≥60 mmol/L on at least 1 occasion OR 2 CF-causing mutations (all as documented in the subject?s medical record)
AND
chronic sinopulmonary disease
OR gastrointestinal/nutritional abnormalities.
2 Must have the G551D-CFTR mutation in at least 1 allele (any known or unknown mutations allowed in second allele).
3 FEV1 40% to 90% (inclusive) of predicted normal for age, gender, and height (Knudson standards23, see Section 12.3.1 for details) at Screening.
4 12 years of age or older on the date of signed Informed Consent Form (ICF), and where appropriate, date of assent
5 Females of child-bearing potential must have a negative serum pregnancy test at Screening and a negative urine pregnancy test on Day 1 (first dose of study drug) prior to randomization
6 Hematology, serum chemistry, coagulation, and urinalysis results, at Screening, with no clinically significant abnormalities that would interfere with the study assessments, as judged by the investigator
7 Able to understand and comply with protocol requirements, restrictions, and instructions and likely to complete the study as planned, as judged by the investigator
8 Subjects of child-bearing potential and who are sexually active must meet the contraception requirements outlined in the study protocol
9 Signed ICF, and where appropriate, signed Assent Form
Key Exclusion Criteria:
1 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 An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 6 weeks before Day 1 (first dose of study drug).
3 Pregnant, planning a pregnancy, breast-feeding, or not willing to follow contraception requirements outlined in the study Protocol
4 Hemoglobin <10 g/dL at Screening
5 Abnormal liver function, at Screening, defined as ≥3x upper limit of normal (ULN), of any 3 or more of the following: serum aspartate transaminase (AST), serum alanine transaminase (ALT), gamma-glutamyl transpeptidase (GGT), serum alkaline phosphatase, total bilirubin
6 Abnormal renal function at Screening, defined as creatinine clearance <89 mL/min/1.73 m2 using the Counahan-Barratt equation24 (for subjects 12 to 17 years of age) or <50 mL/min using the Cockcroft-Gault equation25 (for subjects 18 years of age or older)
7 History of prolonged QT/QTc interval with Fridericia?s correction (QTcF) >450 msec
8 History of solid organ or hematological transplantation
9 History of alcohol, medication or illicit drug abuse within one year prior to Day 1 (first dose of study drug)
10 Colonization with organisms associated with a more rapid decline in pulmonary status (e.g., B. cenocepacia, B. dolosa, and M. abcessus) at Screening
11 Ongoing participation in another therapeutic clinical study or prior participation in an investigational drug study within 30 days prior to Screening. A washout period of ≥5 terminal half-lives of the previous investigational study drug, or 30 days, whichever is longer, must elapse prior to Screening. Subjects who have previously participated in VX-770 studies will be eligible, providing criteria for study entry are met. Subjects who discontinue from this study after randomization will not be eligible to re-enroll.
12 Any ?non-CF-related? illness within 2 weeks prior to Day 1 (first dose of study drug). ?Illness? is defined as an acute (serious or non-serious) condition (e.g., gastroenteritis).
13 Use of inhaled hypertonic saline treatment. (Subjects who have stopped inhaled hypertonic saline treatment will be eligible to participate, but they must have undergone a wash-out period of 6 weeks prior to Day 1 [first dose of study drug])
14 Concomitant use of any inhibitors or inducers of CYP3A4, including consumption of herbal medications (e.g., St. John?s Wort) and grapefruit/grapefruit juice. Subjects
must stop consuming these items from
14 days prior to Day 1 (first dose of study drug) and refrain from consuming these items until the Week 48 or if applicable, the Early Termination Visit.
Additional Study Details
Official Title:
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of VX-770 in Subjects with Cystic Fibrosis and the G551D MutationAnticipated start date:
7/1/2009Lead Sponsor:
Vertex Pharmaceuticals, Inc.Investigator(s):
- Richard B Moss
- Paul K Mohabir
- Colleen Dunn RCP
- Carlos Milla
- Carol K. Conrad
- Zoe Davies RN
- David Weill
- Dr. Jacquelyn Marie Zirbes DNP
- Martha Hamilton
- Terry Earl Robinson
- Andrea Core
- Yan Ki Angela Leung
- Viji Mahathevan
Study Type:
InterventionalPurpose:
TreatmentAllocation:
RandomizedMasking:
Double BlindControl:
noneAssignment:
ParallelEndpoints:
Safety/EfficacyPrimary Outcomes:
- absolute change from baseline in percent predicted forced expiratory volume in 1 second (%predicted FEV1) through Week 24 & 48, the change from baseline in weight, time to first pulmonary exacerbation, sweat chloride, questionnaires.
Secondary Outcomes:
- the rate of decline in FEV1, change from baseline in oxygen saturation, hospitalizations for pulmonary exacerbation, outpatient sick visits to the clinic or hospital visits for CF that are unrelated to the study protocol, courses of intravenous (IV) antibiotics through Week 24 & 48.
- adverse events, clinical laboratory values (serum chemistry, hematology, coagulation studies, and urinalysis), standard digital electrocardiograms (ECGs), 24-hour ambulatory ECGs, vital signs, and physical examinations.
Total Number to be Enrolled:
80Total Number to be Enrolled at Stanford:
4More Information
Locations & Contacts
Stanford Locations & Contacts:
Stanford University School of Medicine 300 Pasteur Drive Stanford, CA 94305Non-Stanford Locations:
The Stanford website does not have any locations outside of Stanford listed for this trial. You may want to check clinicaltrials.gov for posible additional locations.
This listing was last updated:
8/24/2009PLEASE NOTE:
Study Coordinators and Research Nurses cannot give medical advice over the phone. Telephone numbers are provided for obtaining additional information on specific clinical research trials only. If you have specific questions which require clinical expertise, please call your primary care physician. If you do not have a primary care physician please feel free to call the SHC Physician Referral Service at (800) 756-9000 or send an email.
Help