Cystic Fibrosis
Conditions
Brief summary
Open-label study to investigate the effects of Orkambi in CF patients homozygous for the F508del mutation by functional respiratory imaging. Primary endpoints in this study are the changes in Specific airway volumes (siVaw) and Specific Airway resistance (siRaw). A total of 20 ORKAMBI-naive patients with Cystic Fibrosis, homozygous for the F508del mutation will be included in this open label study and will be followed through 3 months of treatment. The treatment will be started after all assessments are performed at visit 1. After the start of the treatment some baseline measurements will be repeated throughout the 3-month treatment period. The patient will be asked to visit the hospital monthly. All study visits should be scheduled around the same time.
Interventions
DRUGOrkambi
Open label of Orkambi treatment during 3 months
Sponsors
University Hospital, Antwerp
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
12 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Documented diagnosis of CF (homozygous for the F508del mutation must be present, this should be documented in the medical history). * Age ≥ 12 years * FEV1 \> 50% * Signed informed consent. If patient is a minor, parents/guardians must give written informed consent * Patient must be on a stable regimen of CF medication for 4 weeks prior to Visit
Exclusion criteria
* FEV1 \< 50% * Anticipated requirement for hospitalization within the next three weeks * History of pneumothorax within the past 6 months prior to Visit 1 * History of haemoptysis requiring embolization within the past 12 months prior to Visit 1 * Unable or unwilling to complete study visits or provide follow-up data as required per the study protocol * Has taken Intravenous (IV) antibiotics within the past 4 weeks prior to Visit 1 * Has ongoing exacerbation or Allergic bronchopulmonary aspergillosis (ABPA) * Pregnant or lactating female * Posttransplant patients * Patients with severe hepatic impairment
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in specific image-based airway resistance (siRaw) | baseline and after three months of therapy | Change in CFD-based airway resistance normalized by the lung volume |
| Change in specific image-based airway volumes (siVaw) | baseline and after three months of therapy | change in CT-based airway volumes normalized by the lung volume |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Airway Wall Volume | baseline and after three months of therapy | calculated using thorax HRCT |
| Aerosol Deposition | baseline and after three months of therapy | calculated using thorax HRCT |
| Dynamic lung volumes | baseline and after three months of therapy | calculated using spirometry |
| Static lung volumes | baseline and after three months of therapy | calculated using body plethysmography |
| airway resistances | baseline and after three months of therapy | calculated using body plethysmography |
| Internal Airflow Distribution | baseline and after three months of therapy | calculated using thorax HRCT |
| 6-minute walking test | baseline and after three months of therapy | Marker of fitness for daily activities |
| Sweat chloride test | baseline and after three months of therapy | Chloride values in sweat chloride test |
| CFQ-R | baseline and after three months of therapy | standardized measures of quality of life will be administered to subject and to parents of subjects under 18 years of age. Disease-specific instrument designed to measure impact on overall health, daily life, perceived well-being and symptoms. Developed specifically for use in patients with a diagnosis of cystic fibrosis. Scores range from 0 to 100, with higher scores indicating better health. |
| Digital lung auscultation | baseline and after three months of therapy | Digital analysis of digital lung sounds obtained by a digital stethoscope |
| Exacerbation frequency | baseline and after three months of therapy | Number of cystic fibrosis exacerbations |
| Lung clearance index | baseline and after three months of therapy | Marker of lung ventilation inhomogeneity |
| Air Trapping | baseline and after three months of therapy | calculated using thorax HRCT |
Countries
Belgium
Outcome results
None listed