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Canadian Observation Trial in CF Patients Undergoing Treatment With Ivacaftor — G551D  

Cystic Fibrosis Clinical Trial

Official title:
Canadian Observation Trial in CF Patients Undergoing Treatment With Ivacaftor

Clinical Trial 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.

Clinical Trial Description

n/a

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT03390985
Study type Observational
Source The Hospital for Sick Children
Contact
Status Completed
Phase N/A
Start date April 23, 2013
Completion date July 26, 2017
View Details
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