NCT03750734

Target Validation and Discovery in Idiopathic Bronchiectasis

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 13 (actual)
• Sponsor: Papworth Hospital NHS Foundation Trust · Other Government
• Sex: All
• Age: 18 Years – 80 Years
• Healthy volunteers: Accepted

Summary

Bronchiectasis is a long-term lung condition where the airways become abnormally enlarged, leading to a build-up of mucus and inflammation that makes the lungs more susceptible to recurrent infection. Patients with bronchiectasis have subtle abnormalities in the way their airway cells respond to infection that are, in part, responsible for the development of their condition. At present there are no licensed treatments for bronchiectasis. This study will aim to characterise in depth some of these abnormalities with a view to future studies that will try to develop treatments that can directly target those abnormalities at a molecular level. Patients known to have bronchiectasis who have provided written informed consent will be enrolled alongside healthy volunteers and patients with chronic obstructive pulmonary disease and cystic fibrosis, for comparison. Participants will give a blood sample and have a bronchoscopy. This is a thin telescopic tube, passed through the nose or mouth, under sedation, into the airways that will allow a sample of bronchial epithelial cells to be taken. The main objective of the study is to achieve a greater understanding of some of the key biological processes/pathways and disease marker genes that play a role in the development of bronchiectasis. This is important because, at present, little is known about the underlying disease mechanisms and there are no licensed treatments for bronchiectasis. The investigator's hope this in-depth characterisation of specific bronchial epithelial cell abnormalities in bronchiectasis will shed light on novel targets for future drug discovery.

Conditions

• Bronchiectasis
• Idiopathic Bronchiectasis

Interventions

Type	Name	Description
DIAGNOSTIC_TEST	Bronchoscopy	Flexible bronchoscopy will be performed under sedation (typically a sedative such as intravenous midazolam and/or fentanyl) with local anaesthetic to the throat and vocal cords. Bronchial epithelial tissue (via bronchial brushing and biopsy forceps) and bronchial lavage samples will be taken for analysis.
DIAGNOSTIC_TEST	Blood test	Peripheral blood will be taken to collect peripheral blood monocytes, which will then allow the production of induced pluripotent stem cell derived bronchial epithelial tissues.

Timeline

• Start date: 2019-09-10
• Primary completion: 2021-08-09
• Completion: 2021-08-09
• First posted: 2018-11-23
• Last updated: 2022-04-04

Locations

1 site across 1 country: United Kingdom