Functional Respiratory Imaging and Orkambi in CF

Cystic Fibrosis Clinical Trial

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT03956589
• Other study ID #: 2018-001573-24
• Status: Terminated
• Phase: Phase 4
• Start date: October 1, 2018
• Est. completion date: April 1, 2020

Study information

• Verified date: April 2020
• Source: University Hospital, Antwerp
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 12
• Est. completion date: April 1, 2020
• Est. primary completion date: April 1, 2020
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Intervention

Drug:
• Orkambi
Open label of Orkambi treatment during 3 months

Locations

Country	Name	City	State
Belgium	Antwerp University Hospital	Edegem

Sponsors (1)

Lead Sponsor	Collaborator
University Hospital, Antwerp

Country where clinical trial is conducted

Belgium, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in specific image-based airway resistance (siRaw)	Change in CFD-based airway resistance normalized by the lung volume	baseline and after three months of therapy
Primary	Change in specific image-based airway volumes (siVaw)	change in CT-based airway volumes normalized by the lung volume	baseline and after three months of therapy
Secondary	Internal Airflow Distribution	calculated using thorax HRCT	baseline and after three months of therapy
Secondary	Air Trapping	calculated using thorax HRCT	baseline and after three months of therapy
Secondary	Airway Wall Volume	calculated using thorax HRCT	baseline and after three months of therapy
Secondary	Aerosol Deposition	calculated using thorax HRCT	baseline and after three months of therapy
Secondary	Dynamic lung volumes	calculated using spirometry	baseline and after three months of therapy
Secondary	Static lung volumes	calculated using body plethysmography	baseline and after three months of therapy
Secondary	airway resistances	calculated using body plethysmography	baseline and after three months of therapy
Secondary	Lung clearance index	Marker of lung ventilation inhomogeneity	baseline and after three months of therapy
Secondary	6-minute walking test	Marker of fitness for daily activities	baseline and after three months of therapy
Secondary	Sweat chloride test	Chloride values in sweat chloride test	baseline and after three months of therapy
Secondary	CFQ-R	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.	baseline and after three months of therapy
Secondary	Digital lung auscultation	Digital analysis of digital lung sounds obtained by a digital stethoscope	baseline and after three months of therapy
Secondary	Exacerbation frequency	Number of cystic fibrosis exacerbations	baseline and after three months of therapy