June 2015 Clinical Trials

The CF Foundation recently provided an update on the following three research trials.

06/25/15 – PROSPECT

This is a phase 4, multi-center trial. This trial aims to track CFTR function and disease progression. It will also look at the effect of ivacaftor/lumicaftor on those beginning treatment.

This is a two-part study. In parts A and B researchers will be looking for biomarkers that show the severity of disease and CFTR function. Part A will be enrolling three main groups of people: healthy participants that do not have CF, people with CF who have gene mutations that lead to partial CFTR function and people with CF whose mutations lead to no CFTR function. Part B will evaluate the impact of ivacaftor/lumicaftor in people with CF who have two copies of the F508del CFTR mutation.

This trial is for people ages 12 and older. It will last about 1 year and will require sweat tests, lung function tests and other assessments.

06/26/15 – Lumacaftor and ivacaftor (VX-809-109) in children ages 6-11

This is a Phase 3 trial. It will look at the safety and effectiveness of lumacaftor and ivacaftor combination therapy.

Researchers aim to determine if the drug is safe and effective in children ages 6-11 with cystic fibrosis and two copies of the F508del-CFTR mutation. The trial will last approximately 6 months. It will require lung function tests and other assessments.

06/28/15 – Vertex 661 and ivacaftor (Vx-661-107) in people with cystic fibrosis ages 12 and older

This is a Phase 3, multi-center trial that will look at the safety and effectiveness of the drug VX-661 in combination with ivacaftor.

Researchers will test the drug’s effectiveness by measuring participant’s lung function in addition to other outcome measures. This trial is for people ages 12 years and older with one copy of the F508del-CFTR mutation and a second CFTR Mutation that is not likely to respond to therapy. The trial will last approximately 6 months. It will require blood draws, sweat tests and lung function tests.