Treatment 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…
ID
Bron
Verkorte titel
Aandoening
Cystic Fibrosis (CF)
Ondersteuning
2. The NCFS, patient association of CF patients, The Netherlands.
Onderzoeksproduct en/of interventie
Uitkomstmaten
Primaire uitkomstmaten
Sweat chloride concentration (SCC) before and after treatment with Ivacaftor.
Achtergrond van het onderzoek
In 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.
Doel van het onderzoek
Treatment 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.
Onderzoeksopzet
Before and after treatment with Ivacaftor.
Onderzoeksproduct en/of interventie
This study will be an observational study. We will observate the intervention of starting with a treatment with Ivacaftor.
Publiek
Huispostnummer KH 01.419.0
Postbus 85090
S. Michel
Utrecht 3508 AB
The Netherlands
+31 (0)88 75 537 25
s.michel-2@umcutrecht.nl
Wetenschappelijk
Huispostnummer KH 01.419.0
Postbus 85090
S. Michel
Utrecht 3508 AB
The Netherlands
+31 (0)88 75 537 25
s.michel-2@umcutrecht.nl
Belangrijkste voorwaarden om deel te mogen nemen (Inclusiecriteria)
• 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).
Belangrijkste redenen om niet deel te kunnen nemen (Exclusiecriteria)
• 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.
Opzet
Deelname
Opgevolgd door onderstaande (mogelijk meer actuele) registratie
Andere (mogelijk minder actuele) registraties in dit register
Geen registraties gevonden.
In overige registers
| Register | ID |
|---|---|
| NTR-new | NL4728 |
| NTR-old | NTR4873 |
| CCMO | NL50276.041.14 |
| OMON | NL-OMON41740 |