Study of Ivacaftor in Subjects With Cystic Fibrosis Who Have a Non-G551D CFTR Gating Mutation

• Register: EUCTR
• Last refreshed on: 21 August 2017
• Main ID: EUCTR2012-000388-26-BE
• Date of registration: 20/04/2012
• Prospective Registration: Yes
• Primary sponsor: Vertex Pharmaceuticals Incorporated
• Public title: Study of Ivacaftor in Subjects With Cystic Fibrosis Who Have a Non-G551D CFTR Gating Mutation
• Scientific title: A Phase 3, Two-Part, Randomized, Double-Blind, Placebo-Controlled, Crossover Study With an Open-Label Period to Evaluate the Efficacy and Safety of Ivacaftor in Subjects With Cystic Fibrosis Who Have a Non-G551D CFTR Gating Mutation
• Date of first enrolment: 23/07/2012
• Target sample size: 40
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2012-000388-26
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: no 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
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Belgium, France, United States

Contacts

• Name: Clinical Trials and Medical Info
• Affiliation: Vertex Pharmaceuticals Incorporated

• Name: Clinical Trials and Medical Info
• Affiliation: Vertex Pharmaceuticals Incorporated

Key inclusion & exclusion criteria

Inclusion criteria:
- At least 1 allele of the following CFTR gating mutations: G178R, S549N, S549R, G551S, G970R, G1244E, S1251N, S1255P, G1349D
- FEV1 =40% predicted normal for age, sex, and height
- 6 years of age or older
- Minimum weight of 15 kg at screening
- Females of childbearing potential must not be pregnant
- Willing to comply with contraception requirements

Are the trial subjects under 18? yes
Number of subjects for this age range: 7
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 3
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
- G551D-CFTR mutation on at least 1 allele
- History of any illness or condition that might confound the results of the study or pose an additional risk in administering ivacaftor to the subject
- An acute upper or lower respiratory infection, pulmonary exacerbation, or changes in therapy (including antibiotics) for pulmonary disease within 4 weeks before the first dose of study drug
- History of solid organ or hematological transplantation
- History of alcohol, medication or illicit drug abuse within 1 year before the first dose of study drug
- Ongoing participation in another therapeutic clinical study or prior participation in an investigational drug study within 30 days before screening
- Use of inhaled hypertonic saline treatment
- Use of any inhibitors or inducers of cytochrome P450 (CYP) 3A
- Evidence of cataract or lens opacity at screening.


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

Health Condition(s) or Problem(s) studied

Cystic Fibrosis
MedDRA version: 14.1 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)

Trade Name: Kalydeco
Product Code: VX-770
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: IVACAFTOR
CAS Number: 873054-44-5
Current Sponsor code: VX-770
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 150-
Pharmaceutical form of the placebo: Film-coated 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 (FEV1)

Timepoint(s) of evaluation of this end point: Through 8 weeks and 24 weeks of treatment

Main Objective: To evaluate the efficacy of ivacaftor in subjects with cystic fibrosis (CF) who have a non-G551D CFTR gating mutation

Secondary Objective: - To evaluate the safety of ivacaftor in subjects with CF who have a non-G551D CFTR
gating mutation
- To evaluate the durability of efficacy of ivacaftor in subjects with CF who have a
non-G551D CFTR gating mutation

Secondary Outcome(s)

Secondary end point(s): - Change from baseline in body mass index (BMI)
- Change from baseline in sweat chloride
- Change from baseline in the respiratory domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R)
- Safety, as determined by adverse events, clinical laboratory values (serum chemistry, hematology, and coagulation), ophthalmologic examinations, ECGs, and vital signs

Timepoint(s) of evaluation of this end point: Through 8 weeks and 24 weeks of treatment and at week 36

Secondary ID(s)

VX12-770-111

Source(s) of Monetary Support

Vertex Pharmaceuticals Incorporated