Clinical trial NCT03956589: Functional Respiratory Imaging and Orkambi in CF
Official title: Functional Respiratory Imaging (FRI) to Assess the Short-term Effect of the Product ORKAMBI (Lumacaftor/ Ivacaftor) on Lung Function in ORKAMBInaive Patients With Cystic Fibrosis Homozygous for Phe508del
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE4
Conditions: Cystic Fibrosis
Lead sponsor: University Hospital, Antwerp
Sponsor class: OTHER
First posted: 2019-05-20
Enrollment: 12
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: Change in specific image-based airway resistance (siRaw)
timeFrame: baseline and after three months of therapy
measure: Change in specific image-based airway volumes (siVaw)
timeFrame: baseline and after three months of therapy
Secondary outcomes:
measure: Internal Airflow Distribution
timeFrame: baseline and after three months of therapy
measure: Air Trapping
timeFrame: baseline and after three months of therapy
measure: Airway Wall Volume
timeFrame: baseline and after three months of therapy
measure: Aerosol Deposition
timeFrame: baseline and after three months of therapy
measure: Dynamic lung volumes
timeFrame: baseline and after three months of therapy
measure: Static lung volumes
timeFrame: baseline and after three months of therapy
measure: airway resistances
timeFrame: baseline and after three months of therapy
measure: Lung clearance index
timeFrame: baseline and after three months of therapy
measure: 6-minute walking test
timeFrame: baseline and after three months of therapy
measure: Sweat chloride test
timeFrame: baseline and after three months of therapy
measure: CFQ-R
timeFrame: baseline and after three months of therapy
measure: Digital lung auscultation
timeFrame: baseline and after three months of therapy
measure: Exacerbation frequency
timeFrame: baseline and after three months of therapy
Other outcomes:
Locations:
city: Edegem
country: Belgium
facility: Antwerp University Hospital
Study officials:
affiliation: University Hospital, Antwerp
name: Stijn Verhulst, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: