cystic fibrosis MedDRA version: 18.0 Level: PT Classification code 10011762 Term: Cystic fibrosis System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Name: Ivacaftor
Product Code: VX-770
Pharmaceutical Form: Granules
INN or Proposed INN: IVACAFTOR
CAS Number: 873054-44-5
Sponsors
Vertex Pharmaceuticals Incorporated
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 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
Exclusion criteria: 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 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
| Measure | Time frame |
|---|---|
| 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 •Absolute change from baseline of the previous study in sweat chloride •Absolute change from baseline in sweat chloride •Absolute change from baseline of the previous study in weight •Absolute change from baseline in weight •Absolute change from baseline of the previous study in stature •Absolute change from baseline in stature •Absolute change from baseline of the previous study in body mass index (BMI) •Absolute change from baseline in BMI ;Timepoint(s) of evaluation of this end point: Through 84 weeks | — |
Countries
Canada, United Kingdom, United States
Contacts
Public ContactClinical Trials and Medical Info
Vertex Pharmaceuticals Incorporated
Outcome results
None listed