Roll-Over Study of Ivacaftor in Cystic Fibrosis Pediatric Subjects With a CF Transmembrane Conductance Regulator Gene (CFTR) Gating Mutation

• Register: ClinicalTrials.gov
• Last refreshed on: 19 October 2017
• Main ID: NCT01946412
• Date of registration: 16/09/2013
• Prospective Registration: Yes
• Primary sponsor: Vertex Pharmaceuticals Incorporated
• Public title: Roll-Over Study of Ivacaftor in Cystic Fibrosis Pediatric Subjects With a CF Transmembrane Conductance Regulator Gene (CFTR) Gating Mutation
• Scientific title: A Phase 3, 2-Arm, Roll-Over Study to Evaluate the Long-term Safety and Pharmacodynamics of Ivacaftor Treatment in Pediatric Subjects With Cystic Fibrosis and a CFTR Gating Mutation
• Date of first enrolment: December 2013
• Target sample size: 33
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01946412
• Study type: Interventional
• Phase: Phase 3

Countries of recruitment

Canada, United Kingdom, United States

Contacts

• Name: Adebayo Lawal, M.D.
• Affiliation: Vertex Pharmaceuticals Incorporated

Key inclusion & exclusion criteria

Inclusion Criteria (Ivacaftor Arm):

1. Completed the last study visit of the treatment period of the previous study
(NCT01705145)

2. Hematology, serum chemistry, and vital signs results on Day 1 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

Inclusion Criteria (Observational Arm):

1. Subjects who completed their assigned study drug treatment in the previous study
(NCT01705145) 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.

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 Criteria: (Observational Arm)

1. Subjects receiving ivacaftor treatment will not be eligible for enrollment in the
observational arm.



Age minimum: 2 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: Ivacaftor

Primary Outcome(s)

Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) [Time Frame: Day 1 up to Week 97 (for participants who completed study drug dosing); Day 1 up to 24 weeks after the last dose (up to Week 108, for participants who prematurely discontinued study drug dosing)]

Secondary Outcome(s)

Absolute Change From Baseline of Parent Study in Stature at Week 12, 24, 36, 48, 60, 72 and 84 [Time Frame: Baseline (study 108), Week 12, 24, 36, 48, 60, 72 and 84 (study 109)]

Absolute Change From Baseline of Parent Study in Body Mass Index (BMI) at Week 12, 24, 36, 48, 60, 72 and 84 [Time Frame: Baseline (study 108), Week 12, 24, 36, 48, 60, 72 and 84 (study 109)]

Absolute Change From Baseline of Study 109 in Sweat Chloride at Week 24, 48, 72 and 84 [Time Frame: Baseline (study 109), Week 24, 48, 72 and 84 (study 109)]

Absolute Change From Baseline of Parent Study in Sweat Chloride at Week 24, 48, 72 and 84 [Time Frame: Baseline (study 108), Week 24, 48, 72 and 84 (study 109)]

Absolute Change From Baseline of Parent Study in Weight at Week 12, 24, 36, 48, 60, 72 and 84 [Time Frame: Baseline (study 108), Week 12, 24, 36, 48, 60, 72 and 84 (study 109)]

Absolute Change From Baseline of Study 109 in Body Mass Index (BMI) at Week 12, 24, 36, 48, 60, 72 and 84 [Time Frame: Baseline (study 109), Week 12, 24, 36, 48, 60, 72 and 84 (study 109)]

Absolute Change From Baseline of Study 109 in Stature at Week 12, 24, 36, 48, 60, 72 and 84 [Time Frame: Baseline (study 109), Week 12, 24, 36, 48, 60, 72 and 84 (study 109)]

Absolute Change From Baseline of Study 109 in Weight at Week 12, 24, 36, 48, 60, 72 and 84 [Time Frame: Baseline (study 109), Week 12, 24, 36, 48, 60, 72 and 84 (study 109)]

Secondary ID(s)

VX11-770-109

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Cystic Fibrosis Foundation Therapeutics

Results

• Results available: Yes
• Date Posted: 01/02/2017
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01946412