Study of VX-770 in Cystic Fibrosis Subjects Age 12 and Older With the G551D Mutation

• Register: EUCTR
• Last refreshed on: 10 July 2015
• Main ID: EUCTR2008-007416-15-IE
• Date of registration: 14/05/2009
• Prospective Registration: Yes
• Primary sponsor: Vertex Pharmaceuticals Incorporated
• Public title: Study of VX-770 in Cystic Fibrosis Subjects Age 12 and Older With the G551D Mutation
• Scientific 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 Mutation - STRIVE
• Date of first enrolment: 11/08/2009
• Target sample size: 100
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2008-007416-15
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: yes Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no Number of treatment arms in the trial: 2

Countries of recruitment

Australia, Canada, Czech Republic, France, Germany, Ireland, United Kingdom, United States

Contacts

• Name: Mark De Rosch, PhD
• Affiliation: Vertex Pharmaceuticals Incorporated

• Name: Mark De Rosch, PhD
• Affiliation: Vertex Pharmaceuticals Incorporated

Key inclusion & exclusion criteria

Inclusion criteria:
- Male or female with confirmed diagnosis of CF
- Must have the G551D-CFTR mutation in at least 1 allele (any known or unknown mutations allowed in second allele).
- FEV1 40% to 90% (inclusive)
- 12 years of age or older
- Females of child-bearing potential must have a negative serum pregnancy test at Screening
- Subjects of child-bearing potential and who are sexually active must follow contraception requirements

Are the trial subjects under 18? yes
Number of subjects for this age range: 36
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 125
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 0

Exclusion criteria:
- History of any illness or condition that might confound the results of the study or pose an additional risk in administering study drug to the subject
- An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy for pulmonary disease within 4 weeks before Day 1 (first dose of study drug)
- Abnormal liver function
- Abnormal renal function
- History of solid organ or hematological transplantation
- History of alcohol, medication or illicit drug abuse within one year prior to Day 1 (first dose of study drug)
- Ongoing participation in another therapeutic clinical study or prior participation in an investigational drug study within 30 days prior to Screening.
- 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 4 weeks prior to Day 1 [first dose of study drug])
- Concomitant use of any inhibitors or inducers of CYP 3A4







Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Cystic Fibrosis
MedDRA version: 14.0 Level: PT Classification code 10011762 Term: Cystic fibrosis System Organ Class: 10010331 - Congenital, familial and genetic disorders

Intervention(s)

Product Name: VX-770
Product Code: VX-770, VRT-813077
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: ivacaftor
CAS Number: 873054-44-5
Current Sponsor code: VX-770
Other descriptive name: VRT-813077
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 150-
Pharmaceutical form of the placebo: Tablet
Route of administration of the placebo: Oral use

Primary Outcome(s)

Primary end point(s): Absolute change from baseline in percent predicted forced expiratory volume in 1 second (%predicted FEV1) through Week 24

Secondary Objective: To evaluate the safety of VX-770 after 24 and 48 weeks of treatment in subjects with CF who have the G551D CFTR mutation on at least 1 allele

To evaluate the efficacy of VX-770 after 48 weeks of treatment in subjects with CF who have the G551D CFTR mutation on at least 1 allele

Timepoint(s) of evaluation of this end point: Week 24

Main Objective: To evaluate the efficacy of VX-770 after 24 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

Secondary Outcome(s)

Secondary end point(s): Efficacy as determined by:
• Absolute change from baseline in percent predicted FEV1 through Week 48
• Change from baseline in sweat chloride through Weeks 24 and 48
• Change from baseline in Cystic Fibrosis Questionnaire-Revised (CFQ-R) through Weeks 24 and 48
• Rate of change in weight through Weeks 24 and 48
• Time to first pulmonary exacerbation through Weeks 24 and 48
Safety as determined by:
Adverse events, clinical laboratory values (serum chemistry, hematology, coagulation studies, and urinalysis), standard digital
electrocardiograms (ECGs), ambulatory ECGs, vital signs, and physical examinations

Timepoint(s) of evaluation of this end point: Week 24 and Week 48

Secondary ID(s)

VX08-770-102

NCT00909532

Source(s) of Monetary Support

Vertex Pharmaceuticals Incorporated