NCT03956589

Functional Respiratory Imaging and Orkambi in CF

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

• Status: Terminated
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 12 (actual)
• Sponsor: University Hospital, Antwerp · Academic / Other
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Cystic Fibrosis

Interventions

Type	Name	Description
DRUG	Orkambi	Open label of Orkambi treatment during 3 months

Timeline

• Start date: 2018-10-01
• Primary completion: 2020-04-01
• Completion: 2020-04-01
• First posted: 2019-05-20
• Last updated: 2020-04-08

Locations

1 site across 1 country: Belgium