Study of VX-770 in Cystic Fibrosis Subjects Age 6 to 11 With the G551D Mutation

• Register: EUCTR
• Last refreshed on: 29 May 2012
• Main ID: EUCTR2008-007479-26-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 6 to 11 With the G551D Mutation
• Scientific title: A Phase 3, 2 Part, Randomized, Double-Blind, Placebo Controlled, Parallel Group Study to Evaluate the Pharmacokinetics, Efficacy and Safety of VX 770 in Subjects Aged 6 to 11 Years with Cystic Fibrosis and the G551D Mutation - ENVISION
• Date of first enrolment: 11/08/2009
• Target sample size: 40
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2008-007479-26
• 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, 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 105% (inclusive)
- 6 to 11 years of age (inclusive)
- Weight 15 kg without shoes at Screening
- 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 meet the contraception requirements










Are the trial subjects under 18? yes
Number of subjects for this age range: 52
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
- 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
- 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)

Secondary Objective: To evaluate the safety of VX-770 after 24 and 48 weeks of treatment in subjects 6 to 11 years of age 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 6 to 11 years of age with CF who have the G551D CFTR mutation on at least 1 allele

Main Objective: To evaluate the efficacy of VX-770 after 24 weeks of treatment in subjects 6 to 11 years of age with CF who have the G551D CFTR mutation on at least 1 allele

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

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

Secondary Outcome(s)

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

Secondary end point(s): Part A: Pharmacokinetic (PK) parameters of VX-770 (and metabolites M1 and M6, if possible)
Part B 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
PART A AND PART B: 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

Secondary ID(s)

VX08-770-103

NCT00909727

Source(s) of Monetary Support

Vertex Pharmaceuticals Incorporated