A Roll-over study in children with Cystic Fibrosis (a rare hereditary pulmonary disease) to assess the long-term safety and pharmacodynamics of ivacaftor

• Register: EUCTR
• Last refreshed on: 16 December 2019
• Main ID: EUCTR2012-000386-20-GB
• Date of registration: 26/04/2013
• Prospective Registration: Yes
• Primary sponsor: Vertex Pharmaceuticals Incorporated
• Public title: A Roll-over study in children with Cystic Fibrosis (a rare hereditary pulmonary disease) to assess the long-term safety and pharmacodynamics of ivacaftor
• 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: 31/07/2013
• Target sample size: 35
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2012-000386-20
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Canada, United Kingdom, 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:
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: 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.


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

Health Condition(s) or Problem(s) studied

cystic fibrosis
MedDRA version: 18.0 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)

Product Name: Ivacaftor
Product Code: VX-770
Pharmaceutical Form: Granules
INN or Proposed INN: IVACAFTOR
CAS Number: 873054-44-5
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 50-
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: 75-

Trade Name: Kalydeco
Product Name: ivacaftor
Product Code: VX-770
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: IVACAFTOR
CAS Number: 873054-44-5
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 150-

Primary Outcome(s)

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 Objective: To evaluate the pharmacodynamics (PD) of long-term ivacaftor treatment in subjects who completed the previous study

Main Objective: To evaluate the long-term safety of ivacaftor treatment

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: Through 84 weeks

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

Secondary ID(s)

VX11-770-109

Source(s) of Monetary Support

Vertex Pharmaceuticals Incorporated

Ethics review

Status: Approved
Approval date:
Contact:

Results

• Results available: Yes
• Date Posted: 02/07/2016
• Date Completed: 17/12/2015
• URL: https://www.clinicaltrialsregister.eu/ctr-search/trial/2012-000386-20/results