Cystic Fibrosis Homozygous (homozygous for the F508del mutation) MedDRA version: 20.0 Level: PT Classification code 10011762 Term: Cystic fibrosis System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Trade Name: Orkambi
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: LUMACAFTOR
Other descriptive name: LUMACAFTOR
Concentration unit: mg milligram(s)
Concentration type: equal
Concentrati
Sponsors
Antwerp University Hospital
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Documented diagnosis of CF (homozygous for the F508del mutation must be present, this should be documented in the medical history). 2. Age = 12 years 3. FEV1 ³ 50% 4. Signed informed consent. If patient is a minor, parents/guardians must give written informed consent 5. Patient must be on a stable regimen of CF medication for 4 weeks prior to Visit 1 Are the trial subjects under 18? yes Number of subjects for this age range: 10 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 10 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. FEV1 < 50% 2. Anticipated requirement for hospitalization within the next three weeks 3. History of pneumothorax within the past 6 months prior to Visit 1 4. History of haemoptysis requiring embolization within the past 12 months prior to Visit 1 5. Unable or unwilling to complete study visits or provide follow-up data as required per the study protocol 6. Has taken Intravenous (IV) antibiotics within the past 4 weeks prior to Visit 1 7. Has ongoing exacerbation or Allergic bronchopulmonary aspergillosis (ABPA) 8. Pregnant or lactating female 9. Posttransplant patients 10. Patients with severe hepatic impairment
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary objective of this study is to assess the efficacy of ORKAMBI on lung functionality across multiple FRI parameters.;Secondary Objective: Secondary outcome variables are Patient Reported Outcome, lung function tests, digital lung auscultation, exercise tolerance and exacerbation frequencies.;Primary end point(s): FRI parameters: • Specific airway resistance (siRaw) • Specific Airway volumes (siVaw);Timepoint(s) of evaluation of this end point: HRCT scans will be taken at baseline (visit 1) and after 3 months of treatment (Visit 4). | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): FRI parameters: • Lung and lobe volumes • Internal airflow distribution • Airway wall thickness • Blood vessel volume • Air trapping • Deposition of inhaled medications Spirometry measurements: • FEV1 • FVC • FEV1/FVC Lung Clearance Index (LCI) Exercise Tolerance: • 6 Minute Walk Test Patient Reported Outcome (PRO): • Borg Category Ratio 10 Scale: measure of the present dyspnea and leg fatigue before and after exercise • Cystic Fibrosis Questionnaire- Revised (CFQ-R) respiratory domain score: measure Health-Related Quality of Life Exacerbation frequency: • Exacerbations requiring oral antibiotics • Exacerbations requiring intravenous antibiotics;Timepoint(s) of evaluation of this end point: HRCT scans will be taken at baseline (visit 1) and after 3 months of treatment (visit 4). Spirometry will taken at baseline (visit 1), after 1 month of tratement (visit 2), after 2 months of treatment (visit 3) and after 3 months of teratment (visit 4). LCI will be done at baseline (visit 1) and after 3 months of treatment (visit 4). 6 MWT and PRO will be done at baseline (visit 1) and after 3 months of treatment (visit 4). Exacerbation frequency will be checked at every visit (1,2,3, and 4). | — |
Countries
Belgium
Contacts
Public ContactKinderpneumologie UZA
Antwerp University Hospital
Outcome results
None listed