Canadian Observation Trial in CF Patients Undergoing Treatment With Ivacaftor (G551D)
28. december 2017 opdateret af: Tanja Gonska
Specific aims:
- 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
- To examine the relation between the individual response to ivacaftor and the presence of modifier genes associated with CF disease severity,
- To assess altered CFTR function using new available in vivo tests,
- To validate newly developed in vivo sweat tests with well established functional tests,
- 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,
- To examine response in other CF-specific features such as aqua wrinkling.
- To examine if sleep/activity level changes.
- To establish a biorepository to enable further investigations.
Studieoversigt
Status
Afsluttet
Betingelser
Undersøgelsestype
Observationel
Tilmelding (Faktiske)
18
Deltagelseskriterier
Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.
Berettigelseskriterier
Aldre berettiget til at studere
6 år og ældre (Barn, Voksen, Ældre voksen)
Tager imod sunde frivillige
Ingen
Køn, der er berettiget til at studere
Alle
Prøveudtagningsmetode
Ikke-sandsynlighedsprøve
Studiebefolkning
CF patients with any of the following mutations G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P, G1349D and G970R approved by Health Canada to receive ivacaftor.
Beskrivelse
Inclusion Criteria:
- any patient being prescribed ivacaftor
Exclusion Criteria:
- only relates to specific tests
Studieplan
Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.
Hvordan er undersøgelsen tilrettelagt?
Design detaljer
Hvad måler undersøgelsen?
Primære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
|
Functional in vivo Cystic fibrosis transmembrane conductor regulator (CFTR) tests: Nasal Potential difference (NPD) measurements
Tidsramme: 7 study visits over a 2 year period
|
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
|
|
Functional in vivo test: Beta-adrenergic sweat secretion test
Tidsramme: 7 study visits over a 2 year period
|
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
|
|
Functional in vitro test: Rectal tissue biopsies to measure CFTR function in vitro using Ussing chamber studies and organoids
Tidsramme: one study visit
|
Rectal tissue specimen is being used to measure the response to ivcaftor of individual CF patients in vitro
|
one study visit
|
|
Functional in vitro test: Collect nasal brushes for CFTR to generate nasal cell cultures for the assessment of ivacaftor response and CFTR function
Tidsramme: 2 study visits in 6 months
|
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
|
Sekundære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
|
Measurement of aqua wrinkling
Tidsramme: 2 study visits in 3 months
|
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
|
|
Measurement of exhaled fractionated nitrogen oxide (FeNo)
Tidsramme: 5 study visits in 1 year
|
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
|
|
Measurements of the lung clearance index
Tidsramme: 5 study visits in 1 year
|
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
|
|
Measurements of the pancreas function using fecal elastase test
Tidsramme: 4 study visits in 2 years
|
Fecal elastase in stool is determined in microg/g stool pre and post-drug
|
4 study visits in 2 years
|
|
Measurements of the pancreas function using serum trypsinogen
Tidsramme: 4 study visits in 2 years
|
Serum trypsinogen is determined in nmol/L pre and post-drug
|
4 study visits in 2 years
|
|
Assessment of the nutritional status
Tidsramme: 4 study visits in 2 years
|
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
|
|
Measurements of the resting energy expenditure
Tidsramme: 4 study visits in 2 years
|
The resting energy expenditure is measured using indirect calorimetry in kcal/d pre and post-post
|
4 study visits in 2 years
|
|
Assessment of pulmonary radiological changes using high resolution CT
Tidsramme: 4 study visits in 2 years
|
Radiological pulmonary changes are being assessed using high resolution CT pre and post-drug (descriptive and Bhalla score)
|
4 study visits in 2 years
|
|
Assessment of glucose tolerance
Tidsramme: 4 study visits in 2 years
|
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
|
Samarbejdspartnere og efterforskere
Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.
Sponsor
Datoer for undersøgelser
Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.
Studer store datoer
Studiestart (Faktiske)
23. april 2013
Primær færdiggørelse (Faktiske)
26. juli 2017
Studieafslutning (Faktiske)
26. juli 2017
Datoer for studieregistrering
Først indsendt
18. december 2017
Først indsendt, der opfyldte QC-kriterier
28. december 2017
Først opslået (Faktiske)
5. januar 2018
Opdateringer af undersøgelsesjournaler
Sidste opdatering sendt (Faktiske)
5. januar 2018
Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier
28. december 2017
Sidst verificeret
1. december 2017
Mere information
Begreber relateret til denne undersøgelse
Yderligere relevante MeSH-vilkår
Andre undersøgelses-id-numre
- 1000036224
Plan for individuelle deltagerdata (IPD)
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