January and February 2016 Clinical Trials

CFF provided information on the following clinical trial:

01/16/2016– Study of In-line Digestive Enzyme Cartridge (RELIZORB) in people with CF who are currently receiving enteral tube feedings

This Phase 4, multi-center trial will look at the safety and effectiveness RELIZORB, a new enteral tube feeding in-line digestive enzyme cartridge. This cartridge is used with tube feedings to deliver the digestive enzyme lipase, which helps the body digest fats.

Researchers will test this device’s effectiveness by measuring fat absorption. The goal of the study is to show that RELISORB helps people with pancreatic insufficiency using tube feedings better digest fats with ease of use and without complications. This trial is for people with cystic fibrosis ages 4 years and older who are currently doing tube feedings at least 4-5 times a week. The trial will last one month and will have four study visits.

02/02/2016 – Phase 1 exploratory study on the effect of the drug QR-010 on CFTR function in the nasal tissue

This is a phase 1b non blinded interventional trial. This exploratory trial will study the use of QR-010 administered within the nose 3 times per week for 4 weeks. Researchers will test the drug in people with cystic fibrosis who have one or two copies of the F508del-CFTR mutation.

This new type of drug is designed to repair the RNA that creates the CFTR protein. In this trial researches will look to see if QR-010 administered within the nose can increase the movement of salt out of the nasal cells. It will also look at the safety and tolerability of the drug. This trial will require nasal potential difference (NPD) tests, blood draws, lung function tests, and urine samples. It will last 7 weeks and is for people who are over 18 years old.

02/09/2016 – Vertex 661 and ivacaftor (VX-661-111) in adults with cystic fibrosis and two copies of the delta F508 mutation

This is a Phase 2, placebo controlled trial. This trial will study the effectiveness of the drug VX-661 in combination with ivacaftor in people who have two of the delta F508 mutation. Researchers will test the drug’s effectiveness by measuring several different health outcomes. These outcome measures include lung function, sweat chloride, mucociliary clearance (MCC), and nasal potential difference (NPD).

This trial is for people ages 18 years and older with two copies of the F508del-CFTR mutation. The trial will last approximately 2 months. It will require sweat tests, NPD tests, MCC tests and lung function tests.