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van CCT (UK)

van CCT (UK)

The effect of Ivacaftor in CF patients with a class III mutation

- candidate number20843
- NTR NumberNTR4873
- ISRCTNISRCTN no longer applicable
- Date ISRCTN created
- date ISRCTN requested
- Date Registered NTR30-okt-2014
- Secondary IDsNL50276.041.14 
- Public TitleThe effect of Ivacaftor in CF patients with a class III mutation
- Scientific TitleThe effect of Ivacaftor in CF patients with a class III mutation
- hypothesisTreatment with Ivacaftor can lead to a therapeutic level of restoration of the CFTR protein channel activity in patients with a class III, S1251N gating mutation. This level of restoration is comparable to a combination of the natural food components curcumin and genistein. Measurements in vitro (in organoids) can predict the individual treatment efficacy of Ivacaftor.
- Healt Condition(s) or Problem(s) studiedCystic Fibrosis (CF)
- Inclusion criteria• CFTR genotype compound/ S1251N
• Already had a rectal biopsy to produce an organoid
• Start a treatment with Ivacaftor
• Male and female patients, aged 6 years or older on the date of informed consent or;
• Signed informed consent form (IC).
- Exclusion criteria• Use of curcumin and or genistein at start or within two weeks prior to start of the study;
• Inability to follow instructions of the investigator.
- mec approval receivedno
- multicenter trialyes
- randomisedno
- groupFactorial
- TypeSingle arm
- Studytypeobservational
- planned startdate 1-dec-2014
- planned closingdate1-dec-2015
- Target number of participants10
- InterventionsThis study will be an observational study. We will observate the intervention of starting with a treatment with Ivacaftor.
- Primary outcomeSweat chloride concentration (SCC) before and after treatment with Ivacaftor.
- Secondary outcome• Pulmonary function (%FEV1) and airway resistance (Rint and bodybox).
• BMI (=weight (in Kg)/Length2 (in cm));
• Quality of life (measured with CFQ-questionnaire)
• Bile salt measurements in plasma and the feces.
• Elastase measurements in the feces
• Correlation between individual Ivacaftor induced CFTR function in vitro (organoid-based measurements) and the in vivo treatment effect;
• The CFTR stimulating ability of the concentration of Ivacaftor in the patient’s blood samples, examined by in vitro testing (in the organoid model). We will also determine the plasma levels of Ivacaftor.
- TimepointsBefore and after treatment with Ivacaftor.
- Trial web site
- statusstopped: trial finished
- Sponsor/Initiator University Medical Center Utrecht (UMCU)
- Funding
(Source(s) of Monetary or Material Support)
ZON-MW, The Netherlands Organization for Health Research and Development, NCFS (Nederlandse Cystic Fibrosis Stichting)
- Publications
- Brief summaryIn the Netherlands, the CFTR potentiator-drug Ivacaftor will be approved for the treatment of CF patients with a (class III) gating mutation, probably by the end of 2014. Primary objective is to objectively investigate the therapeutic potential of Ivacaftor in Dutch CF patients carrying the S1251N gating mutation. Children, adolescents and adults with Cystic Fibrosis who are indicated to start with Ivacaftor (6 years or older and have a compound/S1251N class III gating mutation) can participate in this multi center observational study. Main study parameter will be sweat chloride concentration before and after treatment with Ivacaftor.
- Main changes (audit trail)
- RECORD30-okt-2014 - 22-jun-2016

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