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

A multicentre, OBSERVational, case control study to determine the efficacy and safety of Lumacaftor/Ivacaftor in patients with severe lung disease and Cystic Fibrosis

Status

Recruiting

Phases

Unknown

Study type

Observational

Source

ANZCTR

Registry ID

ACTRN12619000411145

Enrollment

Registered

2019-03-13

Start date

2018-01-16

Completion date

2019-07-08

Last updated

2019-07-15

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

None listed

Brief summary

Aim To determine the safety and efficacy of LUM/IVA in subjects >12 years of age with CF, homozygous for F508del mutation of CFTR and an FEV1<40% of predicted normal, by comparing those patients treated with LUM/IVA with a cohort of age and sex matched CF controls with another set of mutations that lead to severe CFTR dysfunction (belonging to Class I, II or III), with an FEV1<40%5.

Interventions

Patients with CF, homozygous Phedel508, prescribed Ivacaftor/lumacaftor 100/125mg taken orally twice daily, under the Vertex compassionate access scheme in Australia. Participants must have taken Ivacaftor/Lumacaftor for at least 12 months under this scheme. 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. To take part they need to consent for anonymysed data to be avaialble for researchers to access. The patient is not required to do anything. Only data that has already been recorded will be used.

Eligibility

Sex/Gender

All

Age

6 Years to 85 Years

Healthy volunteers

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.

Outcome results