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Health Condition(s) or Problem
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Cystic Fibrosis
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Lay Summary
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The purpose of this study is to evaluate the efficacy and safety of ivacaftor in subjects
with cystic fibrosis (CF) who have the R117H-CFTR mutation.
(from ClinicalTrials.gov)
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Who can enter the trial
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Inclusion Criteria:
- Male or female with confirmed diagnosis of CF
- Must have at least 1 allele of the R117H CFTR mutation
- FEV1 40% to 90% (for subjects aged 12 years or older) or 40% to 105% (for subjects
aged 6 to 11 years) 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
Exclusion Criteria:
- Any 1 of the following CFTR gating mutations: G551D, G178R, S549N, S549R, G551S,
G970R, G1244E, S1251N, S1255P, G1349D
- 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
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Who cannot enter the trial
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Inclusion Criteria:
- Male or female with confirmed diagnosis of CF
- Must have at least 1 allele of the R117H CFTR mutation
- FEV1 40% to 90% (for subjects aged 12 years or older) or 40% to 105% (for subjects
aged 6 to 11 years) 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
Exclusion Criteria:
- Any 1 of the following CFTR gating mutations: G551D, G178R, S549N, S549R, G551S,
G970R, G1244E, S1251N, S1255P, G1349D
- 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
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What will happen
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Drug; Ivacaftor; 150 mg, oral use, administered twice a day (q12h); Ivacaftor; Kalydeco; VX-770; Drug; Placebo; oral use, administered twice a day (q12h); Placebo
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Primary aim
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Absolute change from baseline in percent predicted forced expiratory volume in 1 second (FEV1)
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Secondary Aim
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Change from baseline in body mass index (BMI); At Week 24; No; Change from baseline in sweat chloride; Through Week 24; No; Change from baseline in the respiratory domain of the Cystic Fibrosis Questionnaire Revised (CFQ-R); Through Week 24; No; Time to first pulmonary exacerbation; Through Week 24; No
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Participant Information Sheet
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Sorry, not currently available
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Website
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Sorry, not currently available
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Recruitment Status
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Recruiting
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Nation
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Northern Ireland, Scotland
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Location
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Belfast, Edinburgh
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