The OBSERVE Cystic Fibrosis (CF) Study, to assess the effect of orkambi on people with CF in Australia

Not Applicable

Recruiting

• Conditions: Cystic fibrosis; Human Genetics and Inherited Disorders - Cystic fibrosis

• Registration Number: ACTRN12619000411145
• Lead Sponsor: niversity of Newcastle

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2019-03-13
• Last Update Posted: 15 July 2019

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 144

Inclusion Criteria

•Cystic Fibrosis; defined by the presence of two mutations known to cause dysfunction in CFTR, aged greater than or equal to 12 years.
•LUM/IVA intervention arm, with be homozygous for Phe508del and have commenced treatment with LUM/IVA on the compassionate access programme. Participants will need to have commenced treatment prior to March 2017 to potentially have at least 12 months of data available.

Exclusion Criteria

Insufficient data available to make a comparison over 12 months.
Data confirming CF genotype not available.

Study & Design

• Study Type: Observational
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
umber of exacerbations of CF lung disease requiring the use of intravenous antibiotics during a 12 month observation period. This will include all exacerbations treated with IV antibiotics, including hospitalisation and hospital in the home treatment. data is obtained from electronic medical records by site.[Compared after 12 months of treatment or follow-up.]