BAY63-2521 in adult DeltaF508 homozygous Cystic Fibrosis patients. 1
Research type
Research Study
Full title
Multi-center, randomized, double-blind, placebo-controlled phase 2 study to assess the safety, tolerability and early signs of efficacy of tid orally administered BAY63-2521 in adult DeltaF508 homozygous Cystic Fibrosis patients
IRAS ID
152931
Contact name
Richard Sharp
Contact email
Eudract number
2013-004595-35
Duration of Study in the UK
0 years, 8 months, 0 days
Research summary
Cystic Fibrosis is a genetic disorder caused by several damages (mutations) of a single gene called CFTR gene. Deletion of phenylalanine (F) at position 508 (DeltaF508) is the most common. The CFTR gene is needed to produce chloride channels in cell walls that are important for transporting chloride across cell walls. In Cystic Fibrosis, these channels do not function properly, and mucus secretions become thick and can lead to serious medical problems.
A new indication of Riociguat is being assessed (even though it is already on the market) in this first trial in Cystic Fibrosis patients who are homozygous for the DeltaF508 mutation because research indicates that it may act as a disease modifier by correcting DeltaF508-CFTR function.
Approximately 40 participants will take part in the study in the European Union, United States and Canada. The study drug, BAY63-2521/Riociguat is marketed as Adempas® and is approved in these countries for the treatment of pulmonary artery hypertension and of chronic thromboembolic pulmonary hypertension.
Participants will take part in the study for 9 weeks and shall attend 9 visits to the study center. The study is divided into three periods: screening, treatment, and follow-up.
In order to see if it works, the study drug will be compared to a “placebo” (substance looking identical to Riociguat, but not containing the active drug). This will be given as a capsule taken (with or without food) by mouth three times a day for 28 days. The dose of the study drug will be increased after the first 14 days of treatment. So in case participants receive study drug, they will receive two different doses. Overall, a lower and a higher dose group will be tested.
Study procedures include (but are not limited to) physical examinations, ECGs, lung function tests, blood and urine tests and questionnaires.
REC name
London - Chelsea Research Ethics Committee
REC reference
14/LO/1034
Date of REC Opinion
11 Sep 2014
REC opinion
Further Information Favourable Opinion