Conditions: Cystic Fibrosis
Keywords: Cystic fibrosis delta F508 Safety Tolerability Sweat Chloride Pharmacokinetics
Allocation: RANDOMIZED | Intervention model: PARALLEL
Enrollment: 21
Healthy volunteers: no

Brief summary:
Assessment of the safety, tolerability and early signs of efficacy of three times a day orally administered BAY63-2521 in adult delta F508 homozygous Cystic Fibrosis patients not on treatment with Orkambi

Detailed description:
The study consists of two parts. The first part is double-blind, randomized and placebo-controlled. The second part has an open-label study design. In part 1 patients on Orkambi or other CFTR-modulators are excluded. In part 2 patients on Orkambi are allowed to be included under certain conditions.

Arms:
arm 1: Participants received 0.5 mg BAY63-2521 three times daily (tid) for 14 days. The dose would be increased to 1 mg BAY63-2521 for an additional 14 days, if this was considered safe and tolerable on the basis of the available data for a given patient. arm 2: Participants received matching placebo tid.

Interventions: intervention 1: Riociguat (Adempas, BAY63-2521) intervention 2: Placebo
Intervention descriptions: intervention 1: Participants received 0.5 mg BAY63-2521 three times daily (tid) for 14 days. The dose would be increased to 1 mg BAY63-2521 for an additional 14 days, if this was considered safe and tolerable on the basis of the available data for a given patient. intervention 2: Participants received matching placebo tid.

Protocol document: