A Roll-over study in children with Cystic Fibrosis (a rare hereditary pulmonary disease) to assess the long-term safety and pharmacodynamics of ivacaftor
- Conditions
- cystic fibrosisMedDRA version: 18.0 Level: PT Classification code 10011762 Term: Cystic fibrosis System Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
- Registration Number
- EUCTR2012-000386-20-GB
- Lead Sponsor
- Vertex Pharmaceuticals Incorporated
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Not specified
- Target Recruitment
- 33
Inclusion Criteria: Ivacaftor Arm
1.Completed the last study visit of the treatment period of the previous study
2.Hematology, serum chemistry, and vital signs results on Day 1 (most recent visit from the previous study on which these data are available) with no clinically significant abnormalities that would interfere with the study assessments, as judged by the investigator
3. As judged by the investigator, parent or legal guardian must be able to understand protocol requirements, restrictions, and instructions and the parent or legal guardian should be able to ensure that the subject assents to participation in the study to the degree the subject can assent, and that the subject will comply with and is likely to complete the study as planned
4. Parent or legal guardian must sign the ICF and corresponding assent must be obtained from the subject, as applicable
Inclusion Criterion: Observational Arm
Subjects who completed their assigned study drug treatment in the previous study and elected not to enroll in the ivacaftor arm and subjects who prematurely discontinued treatment in the previous study and received at least 1 dose of study drug treatment in the previous study will be eligible for enrollment in the observational arm.
Are the trial subjects under 18? yes
Number of subjects for this age range: 35
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: Ivacaftor Arm
1.Subjects who prematurely discontinued from the previous study
2.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
3.Subjects with a history of study treatment intolerance as observed in their previous study that, in the opinion of the investigator, might pose an additional risk in administering study drug to the subject
4.Subjects receiving commercially-available ivacaftor treatment
5.Subject was unable to complete an adequate slit-lamp examination at the last ophthalmologic examination in the previous study
Exclusion Criterion: Observational Arm
1.Subjects receiving ivacaftor treatment will not be eligible for enrollment in the observational arm.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To evaluate the long-term safety of ivacaftor treatment ;Secondary Objective: To evaluate the pharmacodynamics (PD) of long-term ivacaftor treatment in subjects who completed the previous study ;Primary end point(s): Safety, as determined by adverse events, clinical laboratory values, 12-lead electrocardiogram (ECGs), vital signs, and ophthalmologic examinations;Timepoint(s) of evaluation of this end point: Through week 84
- Secondary Outcome Measures
Name Time Method <br> Secondary end point(s): •Safety after stopping ivacaftor treatment as determined by serious adverse events (SAEs) and an ophthalmologic examination approximately 24 weeks after the last dose of study drug in the previous study<br> •Absolute change from baseline of the previous study in sweat chloride <br> •Absolute change from baseline in sweat chloride<br> •Absolute change from baseline of the previous study in weight <br> •Absolute change from baseline in weight <br> •Absolute change from baseline of the previous study in stature <br> •Absolute change from baseline in stature <br> •Absolute change from baseline of the previous study in body mass index (BMI) <br> •Absolute change from baseline in BMI <br> ;Timepoint(s) of evaluation of this end point: Through 84 weeks