Canadian Observation Trial in CF Patients Undergoing Treatment With Ivacaftor

Cystic Fibrosis Clinical Trial

— G551D  

Official title:

Canadian Observation Trial in CF Patients Undergoing Treatment With Ivacaftor

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03390985
• Other study ID #: 1000036224
• Status: Completed
• Phase: N/A
• First received: December 18, 2017
• Last updated: December 28, 2017
• Start date: April 23, 2013
• Est. completion date: July 26, 2017

Study information

• Verified date: December 2017
• Source: The Hospital for Sick Children
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 18
• Est. completion date: July 26, 2017
• Est. primary completion date: July 26, 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Years and older

Eligibility

Inclusion Criteria:

• any patient being prescribed ivacaftor

Exclusion Criteria:

• only relates to specific tests

Related Conditions & MeSH terms

• Cystic Fibrosis

Locations

Country	Name	City	State
n/a

Sponsors (1)

Lead Sponsor	Collaborator
Tanja Gonska

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Functional in vivo Cystic fibrosis transmembrane conductor regulator (CFTR) tests: Nasal Potential difference (NPD) measurements	NPD is measured to assess CFTR function as difference in mV between post and pre-drug start	7 study visits over a 2 year period
Primary	Functional in vivo test: Beta-adrenergic sweat secretion test	The beta-adrenergic sweat secretion test is measured as ratio of beta-adrenergic/cholinergic sweat secretion as difference between post and pre-drug start	7 study visits over a 2 year period
Primary	Functional in vitro test: Rectal tissue biopsies to measure CFTR function in vitro using Ussing chamber studies and organoids	Rectal tissue specimen is being used to measure the response to ivcaftor of individual CF patients in vitro	one study visit
Primary	Functional in vitro test: Collect nasal brushes for CFTR to generate nasal cell cultures for the assessment of ivacaftor response and CFTR function	Nasal cultures are used to measure the response to ivcaftor in vitro as difference of the forskolin-induced response of CFTR to ivacaftor	2 study visits in 6 months
Secondary	Measurement of aqua wrinkling	CF patients response to submerge in water for 5 min to develop a skin phenomenon known as aqua wrinkling is being measured subjectively pre and post-drug	2 study visits in 3 months
Secondary	Measurement of exhaled fractionated nitrogen oxide (FeNo)	the fraction of the exhaled NO in % In the expiration of CF patients is being measured pre-and post-drug	5 study visits in 1 year
Secondary	Measurements of the lung clearance index	Multiple breath wash-out technique is being used to measure the lung clearance index (no unit) pre and post-drug	5 study visits in 1 year
Secondary	Measurements of the pancreas function using fecal elastase test	Fecal elastase in stool is determined in microg/g stool pre and post-drug	4 study visits in 2 years
Secondary	Measurements of the pancreas function using serum trypsinogen	Serum trypsinogen is determined in nmol/L pre and post-drug	4 study visits in 2 years
Secondary	Assessment of the nutritional status	Nutritional status is assessed using the BODPOD in kg fat or non-fat mass change or percentage fat or non-fat mass change pre and post-drug	4 study visits in 2 years
Secondary	Measurements of the resting energy expenditure	The resting energy expenditure is measured using indirect calorimetry in kcal/d pre and post-post	4 study visits in 2 years
Secondary	Assessment of pulmonary radiological changes using high resolution CT	Radiological pulmonary changes are being assessed using high resolution CT pre and post-drug (descriptive and Bhalla score)	4 study visits in 2 years
Secondary	Assessment of glucose tolerance	Glucose tolerance is being assessed using the organ glucose tolerance test and glucose levels 2 hrs post in mmol/L will be compared pre and post-drug	4 study visits in 2 years