Functional Respiratory Imaging and Orkambi in CF

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03956589
• Date of registration: 02/05/2019
• Prospective Registration: No
• Primary sponsor: University Hospital, Antwerp
• Public title: Functional Respiratory Imaging and Orkambi in CF
• Scientific 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
• Date of first enrolment: October 1, 2018
• Target sample size: 12
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT03956589
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 4

Countries of recruitment

Belgium

Contacts

• Name: Stijn Verhulst, MD, PhD
• Affiliation: University Hospital, Antwerp

Key inclusion & exclusion 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



Age minimum: 12 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: Orkambi

Primary Outcome(s)

Change in specific image-based airway resistance (siRaw) [Time Frame: baseline and after three months of therapy]

Change in specific image-based airway volumes (siVaw) [Time Frame: baseline and after three months of therapy]

Secondary Outcome(s)

6-minute walking test [Time Frame: baseline and after three months of therapy]

Air Trapping [Time Frame: baseline and after three months of therapy]

Aerosol Deposition [Time Frame: baseline and after three months of therapy]

Internal Airflow Distribution [Time Frame: baseline and after three months of therapy]

Airway Wall Volume [Time Frame: baseline and after three months of therapy]

Exacerbation frequency [Time Frame: baseline and after three months of therapy]

airway resistances [Time Frame: baseline and after three months of therapy]

Digital lung auscultation [Time Frame: baseline and after three months of therapy]

Dynamic lung volumes [Time Frame: baseline and after three months of therapy]

Lung clearance index [Time Frame: baseline and after three months of therapy]

CFQ-R [Time Frame: baseline and after three months of therapy]

Static lung volumes [Time Frame: baseline and after three months of therapy]

Sweat chloride test [Time Frame: baseline and after three months of therapy]

Secondary ID(s)

2018-001573-24

Source(s) of Monetary Support

Please refer to primary and secondary sponsors