Study of the Effect of VX-770 on Lung Clearance Index in Subjects With Cystic Fibrosis and the G551D Mutation

• Register: EUCTR
• Last refreshed on: 25 September 2012
• Main ID: EUCTR2010-020546-96-GB
• Date of registration: 17/08/2010
• Prospective Registration: Yes
• Primary sponsor: Vertex Pharmaceuticals Incorporated
• Public title: Study of the Effect of VX-770 on Lung Clearance Index in Subjects With Cystic Fibrosis and the G551D Mutation
• Scientific title: A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Crossover Study to Evaluate the Effect of VX-770 on Lung Clearance Index in Subjects with Cystic Fibrosis, the G551D Mutation, and FEV1 >90% Predicted -
• Date of first enrolment: 08/10/2010
• Target sample size: 16
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2010-020546-96
• 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: yes 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

Canada, 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 subjects 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 >90% of predicted normal for age, gender, and height at screening
- LCI threshold at screening greater than the established ULN of 7.4
- 6 years of age or older on the date of signed informed consent form (ICF), and where appropriate, date of assent
- Weight greater than or equal to 15 kg without shoes at screening
- Able to swallow tablets
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 6
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 0

Exclusion criteria:
- Abnormal renal function at screening, defined as creatinine clearance <75 mL/min/1.73 m2 using the Counahan-Barratt equation (for subjects 6 to 17 years of age) or <50 mL/min using the Cockcroft-Gault equation (for subjects 18 years of age or older)
- History of solid organ or hematological transplantation
- Colonization with organisms associated with a more rapid decline in pulmonary status (e.g., B cenocepacia, B dolosa, and M abcessus) at screening
- Ongoing participation in another therapeutic clinical study or prior participation in an investigational drug study within the 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 withheld treatment for 48 hours prior to the screening visit and have undergone a washout period of at least 2 weeks prior to the Period 1 Day 1 visit)
- Concomitant use of any inhibitors or inducers of cytochrome P450 (CYP) 3A, including consumption of certain herbal medications (e.g., St. John’s Wort), and grapefruit/grapefruit juice. Subjects must stop consuming these items 14 days prior to Period 1 Day 1.




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

Health Condition(s) or Problem(s) studied

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

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

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)

Main Objective: To evaluate the effect of VX-770 on lung clearance index (LCI) in subjects aged 6 years and older with cystic fibrosis (CF) who have the G551D CFTR mutation on at least 1 allele

Secondary Objective: - To evaluate the safety of VX-770 in subjects aged 6 years and older with CF who have the G551D CFTR mutation on at least 1 allele
- To evaluate the efficacy of VX-770 in subjects aged 6 years and older with CF who have the G551D CFTR mutation on at least 1 allele

Timepoint(s) of evaluation of this end point: 4 Weeks

Primary end point(s): Absolute change from baseline in Lung Clearance Index (LCI)

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: Safety: 16 weeks; Efficacy: 4 Weeks

Secondary end point(s): Safety as determined by adverse events, clinical laboratory values (chemistry, hematology, coagulation studies, and urinalysis), standard digital electrocardiograms (ECGs), and vital signs

Efficacy as determined by:
• Absolute change from baseline in percent predicted forced expiratory volume in 1 second (FEV1)
• Change from baseline in sweat chloride
• Change from baseline in CF Questionnaire-Revised (CFQ-R)

Secondary ID(s)

NCT01262352

VX10-770-106

Source(s) of Monetary Support

Vertex Pharmaceuticals Incorporated