Non-proliferative diabetic retinopathy treated with runcaciguat
Research type
Research Study
Full title
A Phase 2 randomized, placebo-controlled, double-masked proof-of-concept and dose-finding study to investigate the efficacy and safety of runcaciguat (BAY 1101042) in patients with moderately severe to severe non-proliferative diabetic retinopathy
IRAS ID
1003479
Contact name
Abosede Cole
Contact email
Eudract number
2020-002333-15
Clinicaltrials.gov Identifier
Research summary
Research Summary -
Diabetic retinopathy is a medical condition characterised by deterioration of blood vessels in the retina, due to diabetes. Vision
remains almost normal in earlier stages of diabetic retinopathy, called non-proliferative diabetic retinopathy (NPDR).
Deterioration of visual acuity occurs later, when new blood vessels grow or proliferate, called proliferative diabetic retinopathy
(PDR). NPDR and PDR can be complicated by fluid accumulation in the retina, resulting in Diabetic Macular Oedema (DMO)
which may lead to sudden vision deterioration.
This study investigates the effects of runcaciguat in patients with moderately severe and severe NPDR, assessed with a scale of
pictures of the internal eye called Diabetic Retinopathy Severity Score (DRSS). The study aims to reverse NPDR severity based
on centrally assessed colour fundus photographs and prevention of vision threatening complications, including PDR and DMO,
compared with placebo (no treatment). The study also investigates the safety and pharmacokinetics of runcaciguat.
During the initial four weeks of the 96-week treatment period, daily doses will be titrated by 30mg increments to the target dose.
The primary outcome of 2-step DRSS improvement takes place after 24 weeks of treatment. Additional DRSS assessments are
planned after 48, 72, and 96 weeks of treatment.
This combined proof-of-concept and dose-finding study consists of two parts. Part A, 98 patients randomly assigned to two
study groups (120mg and placebo) is designed to (i) determine a safe dose titration scheme and well-tolerated maximum dose
of runcaciguat in patients with NPDR and to (ii) provide the proof of concept that NPDR can be effectively treated with an oral
daily dose of runcaciguat. Part B, 192 patients randomly assigned to 5 study groups (120, 90, 60, 30mg and placebo), is
designed to determine the dose-exposure response of runcaciguat in terms of efficacy and safety to support the dose selection
for subsequent Phase 3 studies with runcaciguat in NPDR.Lay Summary of Results -
Main results of this study This is a summary of the main results for 103 participants who took the study treatment as planned. Individual results from each participant might be different and are not included in this summary.
The results from several studies are needed to decide which treatments work best and are safest. Other studies may provide new information or different results. Always talk to a doctor before making any treatment decisions.How many participants had their DRSS score improved by at least 2 levels after 48 weeks of treatment in the study eye?
To answer this question, researchers calculated the number of participants who had a DRSS score that improved by at least 2 levels after 48 weeks of receiving runcaciguat compared with placebo. Researchers found that there was no improvement in the DRSS score for the participants who received runcaciguat compared with participants who received placebo after 48 weeks of treatment.Other results of this study
How many participants experienced changes associated with vision loss problems after 48 weeks of treatment in the study eye?
To answer this question, researchers calculated the number of participants who had experienced any of the following changes associated with vision loss problems:
► worsening of NPDR
► new abnormal blood vessels growth in the eye ► diabetic macular edema ► major vision lossREC name
London - Hampstead Research Ethics Committee
REC reference
21/LO/0016
Date of REC Opinion
2 Mar 2021
REC opinion
Further Information Favourable Opinion