Orkambi Exercise Study

Launched by UNIVERSITY OF BRITISH COLUMBIA · Jun 28, 2016

Keywords

ClinConnect Summary

Advances in therapies and patient care have led to dramatic improvements in cystic fibrosis (CF) survival. Consequently, CF patients are living longer with varying degrees of lung function impairment. Dyspnea is a commonly reported symptom in CF that adversely impacts quality of life. Recently, lumacaftor/ivacaftor (Orkambi), a combination drug therapy, was approved by Health Canada for use in CF patients.

The purpose of this study is to determine the various factors that cause shortness of breath (or dyspnea) in patients with CF and to determine how treatment with Orkambi can manipulate t...

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • Confirmed diagnosis of CF and homozygous for the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene
• • Plan to initiate Orkambi™ by the treating physician within 30 days of the enrolment visit
• • Aged 19 years or older
• • Stable clinical status based on clinical judgment of the treating physicians (Drs Wilcox, Quon or Goodwin)
• • Forced Expiratory Volume in 1 second (FEV1.0) \< 90% predicted
• • Body mass index greater than 16 or less than 30 kg/m2
• • Currently non-smoking or a past smoking history of less than 20 pack-years
• • Able to read and understand English
• Exclusion Criteria:
• • A disease other than CF that could importantly contribute to dyspnea or exercise limitation
• • Chronic airway infection with Mycobacterium abscessus, Burkholderia cepacia complex, or other organisms with infection control implications based on the treating physicians
• • Contraindications to clinical exercise testing
• • Use of supplemental oxygen or desaturation less than 85% with exercise
• • Diagnosis of pneumothorax in the past 4 weeks
• • History of organ transplantation