Cystic fibrosis
Conditions
Interventions
Start of orkambi is part of routine care.
Sponsors
University of Amsterdam
Eligibility
Inclusion criteria
Inclusion criteria: In order to be eligible to participate in this study, a subject must meet all of the following criteria: • Patients with a (predominant) class II mutation (almost exclusively homozygous Phe508del) for cystic fibrosis. • Start with Ivacaftor/Lumacaftor (Orkambi) therapy
Exclusion criteria
Exclusion criteria: A potential subject who meets any of the following criteria will be excluded from participation in this study: • None For bronchoscopy the following patients will be excluded: • Pre-lung transplant trajectory • No informed consent for the procedure • Deemed inappropriate by the treating physician.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| •Bacterial sequences in sputum, broncho-alveolar lavage fluid and oral and nasal wash. •Metabolic profiles in broncho-alveolar lavage fluid and oral wash. •Volatile metabolites in breath. | — |
Secondary
| Measure | Time frame |
|---|---|
| • Bacterial culture result from sputum and broncho-alveolar lavage fluid. • Long function test results (FEV1/FVC, etc). • Plasma parameters of (chronic) inflammation: IgG, CRP, leukocyte count. • Quality of life assessments. | — |
Contacts
Public ContactLieuwe D.J. Bos
Academic Medical Center, Room G3-228 Meibergdreef 9
Outcome results
None listed