Canadian Observation Trial in CF Patients Undergoing Treatment With Ivacaftor

NCT ID: NCT03390985

Last Updated: 2018-01-05

Basic Information

• Recruitment Status: COMPLETED
• Total Enrollment: 18 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2013-04-23
• Study Completion Date: 2017-07-26

Brief Summary

Specific aims:

1. To elucidate the biological mechanism that leads to pulmonary and nutritional improvement in CF patients following treatment with ivacaftor using advanced techniques to assess changes of the pulmonary and nutritional status

2. To examine the relation between the individual response to ivacaftor and the presence of modifier genes associated with CF disease severity,

3. To assess altered CFTR function using new available in vivo tests,

4. To validate newly developed in vivo sweat tests with well established functional tests,

5. To establish correlation between the CFTR response to Vx-770 measured in a new ex vivo method (organoids) and the actual clinical and/or functional response in individual patients,

6. To examine response in other CF-specific features such as aqua wrinkling.

7. To examine if sleep/activity level changes.

8. To establish a biorepository to enable further investigations.

Conditions

Cystic Fibrosis

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Eligibility Criteria

Inclusion Criteria

• any patient being prescribed ivacaftor

Exclusion Criteria

• only relates to specific tests

• Minimum Eligible Age: 6 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Tanja Gonska

OTHER

Sponsor Role: lead

Responsible Party

Tanja Gonska

Principal Investigator

Responsibility Role: SPONSOR_INVESTIGATOR

Other Identifiers

1000036224

Identifier Type: -

Identifier Source: org_study_id