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Restore CFTR Protein Completed with Results
Phase 2 study of riociguat in adults with CF (Bayer BAY 63-2521/ 17020)
This study evaluated the safety, tolerability, and effectiveness of the oral drug riociguat in adults with CF who have two copies of the F508del mutation and were not receiving CFTR modulator therapy.
This was a two-part study. In Part 1, a 28-day study, participants were randomly assigned to receive oral riociguat or placebo (0.5 mg three times daily) for the first 14 days, and then participants received 1.0 mg three times daily for the last 14 days. Part 2, an open-label extension, was cancelled because the first part of the study did not show efficacy.
Eligibility
See other primary eligibility criteria for more information.
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Age:
18 Years and Older -
Mutation(s):
Two Copies F508del -
FEV1% Predicted:
60 to 90%
For more information about the results of this study and where it was conducted, visit ClinicalTrials.gov.
Other Primary Eligibility Criteria
Adult F508del homozygous Cystic Fibrosis patients are only included
Study Results
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What We Learned:
The study found that riociguat after 28 days of dosing did not result in meaningful changes in sweat chloride or lung function at doses up to 1.0 mg when compared with placebo. The most common riociguat-related adverse event (AE) was headache occurring in three of fourteen participants (21%). A serious AE of intestinal obstruction requiring hospitalization occurred in one participant receiving riociguat. The two-part study was stopped early because the first part of the study did not show efficacy.
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Primary Findings:
Effectiveness:
This study was conducted between September 2014 and January 2017. The study enrolled 21 participants: 14 received riociguat and 7 received placebo. Two participants who received riociguat were discontinued from the study due to adverse events (AEs), one related to intestinal obstruction and one related to severe headache.
The primary efficacy endpoint was the mean change from baseline to Day 14 and Day 28 in sweat chloride concentration in participants receiving riociguat compared with placebo. This study did not meet the primary endpoint as riociguat (doses up to 1.0mg) did not reduce sweat chloride concentration when compared with placebo. On Day 28, the change from baseline in sweat chloride concentration was + 3.4 [11.0] mmol/L in the riociguat group (n = 9) compared with + 9.0 (12.7) mmol/L in the placebo group (n = 7).
No significant differences in lung function were observed between participants receiving riociguat compared with placebo. After 28 days, the change in lung function (mean ppFEV1) with riociguat was –0.8% (6.0) and for placebo was + 2.4% (9.6).
Safety:
The most common riociguat-related AE was headache occurring in three of fourteen participants (21%). A serious AE of intestinal obstruction requiring hospitalization occurred in one participant receiving riociguat.
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Citation:
J Cyst Fibros 2021;DOI 10.1016/j.jcf.2021.07.015
For current information about the overall development status of this drug, please check the Drug Development Pipeline.
Study Design
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Study Type: ?more info
Interventional -
Randomized Study: ?more info
Yes -
Placebo Controlled: ?more info
Yes -
Length of Participation:
28 days -
Number of Study Visits:
9
Additional Information
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Phase: ?more info
Phase Two -
Study Sponsor: ?more info
Bayer -
Study Drugs:
Eligibility
See other primary eligibility criteria for more information.
-
Age:
18 Years and Older -
Mutation(s):
Two Copies F508del -
FEV1% Predicted:
60 to 90%
For more information about the results of this study and where it was conducted, visit ClinicalTrials.gov.
Other Primary Eligibility Criteria
Adult F508del homozygous Cystic Fibrosis patients are only included
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