Trial Outcomes & Findings for A Study to Test Whether Avenciguat Helps People With Liver Cirrhosis and High Blood Pressure in the Portal Vein (Main Vessel Going to the Liver) Who Had Bleeding in the Esophagus or Fluid Accumulation in the Belly (NCT NCT06082843)
NCT ID: NCT06082843
Last Updated: 2025-06-12
Results Overview
Percentage change in hepatic venous pressure gradient (HVPG) from baseline to Week 8.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
22 participants
Primary outcome timeframe
At baseline and at Week 8.
Results posted on
2025-06-12
Participant Flow
Multi-national, randomised, double-blind, parallel group, placebo-controlled trial to investigate the safety and tolerability of avenciguat on top of standard of care on portal hypertension. The trial was prematurely discontinued due to the sponsor's decision.
All participants were screened for eligibility prior to participation in the trial. Participants attended a specialist site which ensured that they (the participants) strictly met all inclusion and none of the exclusion criteria. Participants were not to be allocated to a treatment group if any of the entry criteria were violated.
Participant milestones
| Measure |
Placebo
Participants with stabilized decompensated cirrhosis due to non-cholestatic liver disease, following their first decompensation event, received 1 milligram (mg), 2 mg, or 3 mg film-coated tablets of placebo-matching avenciguat (BI 685509) orally twice daily (bid). Participants started the treatment with a 1 mg film-coated tablet of placebo-matching avenciguat administered bid. One week later (Visit 3), the dosage was increased to 2 mg film-coated tablets bid, and after another week, participants started on 3 mg film-coated tablet bid (Visit 4), which was maintained for the rest of the treatment.
|
Avenciguat
Participants with stabilized decompensated cirrhosis due to non-cholestatic liver disease, following their first decompensation event, received 1 milligram (mg), 2 mg, or 3 mg film-coated tablets of avenciguat (BI 685509) orally twice daily (bid), up to a total dose of 6 milligrams (mg). The treatment period began (Visit 2) with a 1 mg film-coated tablet of avenciguat administered bid. If the dose was well-tolerated, it was increased to 2 mg film-coated tablets bid after one week (Visit 3), followed by an increase to the maintenance dose of 3 mg film-coated tablet one week later (Visit 4). If the maintenance dose of avenciguat was not well-tolerated, it was reduced, with the participants remaining on the highest tolerated dose for the rest of the treatment period.
|
|---|---|---|
|
Overall Study
STARTED
|
12
|
10
|
|
Overall Study
COMPLETED
|
0
|
0
|
|
Overall Study
NOT COMPLETED
|
12
|
10
|
Reasons for withdrawal
| Measure |
Placebo
Participants with stabilized decompensated cirrhosis due to non-cholestatic liver disease, following their first decompensation event, received 1 milligram (mg), 2 mg, or 3 mg film-coated tablets of placebo-matching avenciguat (BI 685509) orally twice daily (bid). Participants started the treatment with a 1 mg film-coated tablet of placebo-matching avenciguat administered bid. One week later (Visit 3), the dosage was increased to 2 mg film-coated tablets bid, and after another week, participants started on 3 mg film-coated tablet bid (Visit 4), which was maintained for the rest of the treatment.
|
Avenciguat
Participants with stabilized decompensated cirrhosis due to non-cholestatic liver disease, following their first decompensation event, received 1 milligram (mg), 2 mg, or 3 mg film-coated tablets of avenciguat (BI 685509) orally twice daily (bid), up to a total dose of 6 milligrams (mg). The treatment period began (Visit 2) with a 1 mg film-coated tablet of avenciguat administered bid. If the dose was well-tolerated, it was increased to 2 mg film-coated tablets bid after one week (Visit 3), followed by an increase to the maintenance dose of 3 mg film-coated tablet one week later (Visit 4). If the maintenance dose of avenciguat was not well-tolerated, it was reduced, with the participants remaining on the highest tolerated dose for the rest of the treatment period.
|
|---|---|---|
|
Overall Study
Withdrawal by Subject
|
0
|
2
|
|
Overall Study
Study terminated by sponsor
|
12
|
8
|
Baseline Characteristics
A Study to Test Whether Avenciguat Helps People With Liver Cirrhosis and High Blood Pressure in the Portal Vein (Main Vessel Going to the Liver) Who Had Bleeding in the Esophagus or Fluid Accumulation in the Belly
Baseline characteristics by cohort
| Measure |
Placebo
n=12 Participants
Participants with stabilized decompensated cirrhosis due to non-cholestatic liver disease, following their first decompensation event, received 1 milligram (mg), 2 mg, or 3 mg film-coated tablets of placebo-matching avenciguat (BI 685509) orally twice daily (bid). Participants started the treatment with a 1 mg film-coated tablet of placebo-matching avenciguat administered bid. One week later (Visit 3), the dosage was increased to 2 mg film-coated tablets bid, and after another week, participants started on 3 mg film-coated tablet bid (Visit 4), which was maintained for the rest of the treatment.
|
Avenciguat
n=10 Participants
Participants with stabilized decompensated cirrhosis due to non-cholestatic liver disease, following their first decompensation event, received 1 milligram (mg), 2 mg, or 3 mg film-coated tablets of avenciguat (BI 685509) orally twice daily (bid), up to a total dose of 6 milligrams (mg). The treatment period began (Visit 2) with a 1 mg film-coated tablet of avenciguat administered bid. If the dose was well-tolerated, it was increased to 2 mg film-coated tablets bid after one week (Visit 3), followed by an increase to the maintenance dose of 3 mg film-coated tablet one week later (Visit 4). If the maintenance dose of avenciguat was not well-tolerated, it was reduced, with the participants remaining on the highest tolerated dose for the rest of the treatment period.
|
Total
n=22 Participants
Total of all reporting groups
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|---|---|---|---|
|
Age, Continuous
|
60.3 Years
STANDARD_DEVIATION 10.6 • n=5 Participants
|
59.2 Years
STANDARD_DEVIATION 10.1 • n=7 Participants
|
59.8 Years
STANDARD_DEVIATION 10.2 • n=5 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
7 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
15 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
11 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
19 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
3 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
4 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
9 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
18 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Hepatic Venous Pressure Gradient (HVPG)
|
14.19 mmHg
STANDARD_DEVIATION 4.49 • n=5 Participants
|
11.55 mmHg
STANDARD_DEVIATION 3.94 • n=7 Participants
|
12.99 mmHg
STANDARD_DEVIATION 4.36 • n=5 Participants
|
PRIMARY outcome
Timeframe: At baseline and at Week 8.Population: As the trial was discontinued prematurely and no patient completed the 8-week treatment period, no data were available to assess the endpoint.
Percentage change in hepatic venous pressure gradient (HVPG) from baseline to Week 8.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: At baseline and at Week 8.Population: As the trial was discontinued prematurely and no patient completed the 8-week treatment period, no data were available to assess the endpoint.
Occurrence of a response, defined as at least a 10% reduction in the hepatic venous pressure gradient (HVPG) (measured in mmHg) after 8 weeks of treatment.
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Up to 46 days.Population: Treated set (TS): all trial participants who were randomized to the trial medication and were treated with at least one dose.
Number of participants with at least one decompensation events. Ascites, variceal hemorrhage (VH), and hepatic encephalopathy were defined as further decompensations events. As the trial was discontinued prematurely and no patient completed the 8-week treatment period, the data presented reflects the actual on-treatment period.
Outcome measures
| Measure |
Placebo
n=12 Participants
Participants with stabilized decompensated cirrhosis due to non-cholestatic liver disease, following their first decompensation event, received 1 milligram (mg), 2 mg, or 3 mg film-coated tablets of placebo-matching avenciguat (BI 685509) orally twice daily (bid). Participants started the treatment with a 1 mg film-coated tablet of placebo-matching avenciguat administered bid. One week later (Visit 3), the dosage was increased to 2 mg film-coated tablets bid, and after another week, participants started on 3 mg film-coated tablet bid (Visit 4), which was maintained for the rest of the treatment.
|
Avenciguat
n=10 Participants
Participants with stabilized decompensated cirrhosis due to non-cholestatic liver disease, following their first decompensation event, received 1 milligram (mg), 2 mg, or 3 mg film-coated tablets of avenciguat (BI 685509) orally twice daily (bid), up to a total dose of 6 milligrams (mg). The treatment period began (Visit 2) with a 1 mg film-coated tablet of avenciguat administered bid. If the dose was well-tolerated, it was increased to 2 mg film-coated tablets bid after one week (Visit 3), followed by an increase to the maintenance dose of 3 mg film-coated tablet one week later (Visit 4). If the maintenance dose of avenciguat was not well-tolerated, it was reduced, with the participants remaining on the highest tolerated dose for the rest of the treatment period.
|
|---|---|---|
|
Occurrence of Further Decompensation Events (Ascites, Variceal Hemorrhage (VH), and / or Overt Hepatic Encephalopathy (HE)) During the 8-week Treatment Period
Ascites
|
0 Participants
|
0 Participants
|
|
Occurrence of Further Decompensation Events (Ascites, Variceal Hemorrhage (VH), and / or Overt Hepatic Encephalopathy (HE)) During the 8-week Treatment Period
Variceal Hemorrhage
|
0 Participants
|
0 Participants
|
|
Occurrence of Further Decompensation Events (Ascites, Variceal Hemorrhage (VH), and / or Overt Hepatic Encephalopathy (HE)) During the 8-week Treatment Period
Hepatic encephalopathy
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Up to 46 days.Population: Treated set (TS): all trial participants who were randomized to the trial medication and were treated with at least one dose.
Number of participants with grade 3 or higher common terminology criteria for adverse events (CTCAE) hypotension or syncope base on the investigator judgment. As the trial was discontinued prematurely and no patient completed the 8-week treatment period, the data presented reflects the actual on-treatment period.
Outcome measures
| Measure |
Placebo
n=12 Participants
Participants with stabilized decompensated cirrhosis due to non-cholestatic liver disease, following their first decompensation event, received 1 milligram (mg), 2 mg, or 3 mg film-coated tablets of placebo-matching avenciguat (BI 685509) orally twice daily (bid). Participants started the treatment with a 1 mg film-coated tablet of placebo-matching avenciguat administered bid. One week later (Visit 3), the dosage was increased to 2 mg film-coated tablets bid, and after another week, participants started on 3 mg film-coated tablet bid (Visit 4), which was maintained for the rest of the treatment.
|
Avenciguat
n=10 Participants
Participants with stabilized decompensated cirrhosis due to non-cholestatic liver disease, following their first decompensation event, received 1 milligram (mg), 2 mg, or 3 mg film-coated tablets of avenciguat (BI 685509) orally twice daily (bid), up to a total dose of 6 milligrams (mg). The treatment period began (Visit 2) with a 1 mg film-coated tablet of avenciguat administered bid. If the dose was well-tolerated, it was increased to 2 mg film-coated tablets bid after one week (Visit 3), followed by an increase to the maintenance dose of 3 mg film-coated tablet one week later (Visit 4). If the maintenance dose of avenciguat was not well-tolerated, it was reduced, with the participants remaining on the highest tolerated dose for the rest of the treatment period.
|
|---|---|---|
|
Occurrence of CTCAE Grade 3 (or Higher) Hypotension or Syncope Based on Investigator Judgement During the 8-week Treatment Period
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: Up to 46 days.Population: Treated set (TS): all trial participants who were randomized to the trial medication and were treated with at least one dose.
Number of participants that discontinued avenciguat due to hypotension or syncope. As the trial was discontinued prematurely and no patient completed the 8-week treatment period, the data presented reflects the actual on-treatment period.
Outcome measures
| Measure |
Placebo
n=12 Participants
Participants with stabilized decompensated cirrhosis due to non-cholestatic liver disease, following their first decompensation event, received 1 milligram (mg), 2 mg, or 3 mg film-coated tablets of placebo-matching avenciguat (BI 685509) orally twice daily (bid). Participants started the treatment with a 1 mg film-coated tablet of placebo-matching avenciguat administered bid. One week later (Visit 3), the dosage was increased to 2 mg film-coated tablets bid, and after another week, participants started on 3 mg film-coated tablet bid (Visit 4), which was maintained for the rest of the treatment.
|
Avenciguat
n=10 Participants
Participants with stabilized decompensated cirrhosis due to non-cholestatic liver disease, following their first decompensation event, received 1 milligram (mg), 2 mg, or 3 mg film-coated tablets of avenciguat (BI 685509) orally twice daily (bid), up to a total dose of 6 milligrams (mg). The treatment period began (Visit 2) with a 1 mg film-coated tablet of avenciguat administered bid. If the dose was well-tolerated, it was increased to 2 mg film-coated tablets bid after one week (Visit 3), followed by an increase to the maintenance dose of 3 mg film-coated tablet one week later (Visit 4). If the maintenance dose of avenciguat was not well-tolerated, it was reduced, with the participants remaining on the highest tolerated dose for the rest of the treatment period.
|
|---|---|---|
|
Occurrence of Discontinuation Due to Hypotension or Syncope During the 8-week Treatment Period
|
0 Participants
|
0 Participants
|
Adverse Events
Placebo
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
Avenciguat
Serious events: 2 serious events
Other events: 3 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Placebo
n=12 participants at risk
Participants with stabilized decompensated cirrhosis due to non-cholestatic liver disease, following their first decompensation event, received 1 milligram (mg), 2 mg, or 3 mg film-coated tablets of placebo-matching avenciguat (BI 685509) orally twice daily (bid). Participants started the treatment with a 1 mg film-coated tablet of placebo-matching avenciguat administered bid. One week later (Visit 3), the dosage was increased to 2 mg film-coated tablets bid, and after another week, participants started on 3 mg film-coated tablet bid (Visit 4), which was maintained for the rest of the treatment.
|
Avenciguat
n=10 participants at risk
Participants with stabilized decompensated cirrhosis due to non-cholestatic liver disease, following their first decompensation event, received 1 milligram (mg), 2 mg, or 3 mg film-coated tablets of avenciguat (BI 685509) orally twice daily (bid), up to a total dose of 6 milligrams (mg). The treatment period began (Visit 2) with a 1 mg film-coated tablet of avenciguat administered bid. If the dose was well-tolerated, it was increased to 2 mg film-coated tablets bid after one week (Visit 3), followed by an increase to the maintenance dose of 3 mg film-coated tablet one week later (Visit 4). If the maintenance dose of avenciguat was not well-tolerated, it was reduced, with the participants remaining on the highest tolerated dose for the rest of the treatment period.
|
|---|---|---|
|
Cardiac disorders
Sinus arrest
|
0.00%
0/12 • All-cause mortality: From first drug administration until individual end of study. Up to 60 days. Adverse event reporting: From first administration until last drug administration plus 7-day residual effect period. Up to 53 days.
Treated set (TS): all trial participants who were randomized to the trial medication and were treated with at least one dose.
|
10.0%
1/10 • All-cause mortality: From first drug administration until individual end of study. Up to 60 days. Adverse event reporting: From first administration until last drug administration plus 7-day residual effect period. Up to 53 days.
Treated set (TS): all trial participants who were randomized to the trial medication and were treated with at least one dose.
|
|
Infections and infestations
Vulval abscess
|
0.00%
0/12 • All-cause mortality: From first drug administration until individual end of study. Up to 60 days. Adverse event reporting: From first administration until last drug administration plus 7-day residual effect period. Up to 53 days.
Treated set (TS): all trial participants who were randomized to the trial medication and were treated with at least one dose.
|
10.0%
1/10 • All-cause mortality: From first drug administration until individual end of study. Up to 60 days. Adverse event reporting: From first administration until last drug administration plus 7-day residual effect period. Up to 53 days.
Treated set (TS): all trial participants who were randomized to the trial medication and were treated with at least one dose.
|
Other adverse events
| Measure |
Placebo
n=12 participants at risk
Participants with stabilized decompensated cirrhosis due to non-cholestatic liver disease, following their first decompensation event, received 1 milligram (mg), 2 mg, or 3 mg film-coated tablets of placebo-matching avenciguat (BI 685509) orally twice daily (bid). Participants started the treatment with a 1 mg film-coated tablet of placebo-matching avenciguat administered bid. One week later (Visit 3), the dosage was increased to 2 mg film-coated tablets bid, and after another week, participants started on 3 mg film-coated tablet bid (Visit 4), which was maintained for the rest of the treatment.
|
Avenciguat
n=10 participants at risk
Participants with stabilized decompensated cirrhosis due to non-cholestatic liver disease, following their first decompensation event, received 1 milligram (mg), 2 mg, or 3 mg film-coated tablets of avenciguat (BI 685509) orally twice daily (bid), up to a total dose of 6 milligrams (mg). The treatment period began (Visit 2) with a 1 mg film-coated tablet of avenciguat administered bid. If the dose was well-tolerated, it was increased to 2 mg film-coated tablets bid after one week (Visit 3), followed by an increase to the maintenance dose of 3 mg film-coated tablet one week later (Visit 4). If the maintenance dose of avenciguat was not well-tolerated, it was reduced, with the participants remaining on the highest tolerated dose for the rest of the treatment period.
|
|---|---|---|
|
Gastrointestinal disorders
Abdominal distension
|
0.00%
0/12 • All-cause mortality: From first drug administration until individual end of study. Up to 60 days. Adverse event reporting: From first administration until last drug administration plus 7-day residual effect period. Up to 53 days.
Treated set (TS): all trial participants who were randomized to the trial medication and were treated with at least one dose.
|
10.0%
1/10 • All-cause mortality: From first drug administration until individual end of study. Up to 60 days. Adverse event reporting: From first administration until last drug administration plus 7-day residual effect period. Up to 53 days.
Treated set (TS): all trial participants who were randomized to the trial medication and were treated with at least one dose.
|
|
Metabolism and nutrition disorders
Hyperuricaemia
|
8.3%
1/12 • All-cause mortality: From first drug administration until individual end of study. Up to 60 days. Adverse event reporting: From first administration until last drug administration plus 7-day residual effect period. Up to 53 days.
Treated set (TS): all trial participants who were randomized to the trial medication and were treated with at least one dose.
|
0.00%
0/10 • All-cause mortality: From first drug administration until individual end of study. Up to 60 days. Adverse event reporting: From first administration until last drug administration plus 7-day residual effect period. Up to 53 days.
Treated set (TS): all trial participants who were randomized to the trial medication and were treated with at least one dose.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
0.00%
0/12 • All-cause mortality: From first drug administration until individual end of study. Up to 60 days. Adverse event reporting: From first administration until last drug administration plus 7-day residual effect period. Up to 53 days.
Treated set (TS): all trial participants who were randomized to the trial medication and were treated with at least one dose.
|
10.0%
1/10 • All-cause mortality: From first drug administration until individual end of study. Up to 60 days. Adverse event reporting: From first administration until last drug administration plus 7-day residual effect period. Up to 53 days.
Treated set (TS): all trial participants who were randomized to the trial medication and were treated with at least one dose.
|
|
Psychiatric disorders
Insomnia
|
8.3%
1/12 • All-cause mortality: From first drug administration until individual end of study. Up to 60 days. Adverse event reporting: From first administration until last drug administration plus 7-day residual effect period. Up to 53 days.
Treated set (TS): all trial participants who were randomized to the trial medication and were treated with at least one dose.
|
0.00%
0/10 • All-cause mortality: From first drug administration until individual end of study. Up to 60 days. Adverse event reporting: From first administration until last drug administration plus 7-day residual effect period. Up to 53 days.
Treated set (TS): all trial participants who were randomized to the trial medication and were treated with at least one dose.
|
|
Renal and urinary disorders
Polyuria
|
0.00%
0/12 • All-cause mortality: From first drug administration until individual end of study. Up to 60 days. Adverse event reporting: From first administration until last drug administration plus 7-day residual effect period. Up to 53 days.
Treated set (TS): all trial participants who were randomized to the trial medication and were treated with at least one dose.
|
10.0%
1/10 • All-cause mortality: From first drug administration until individual end of study. Up to 60 days. Adverse event reporting: From first administration until last drug administration plus 7-day residual effect period. Up to 53 days.
Treated set (TS): all trial participants who were randomized to the trial medication and were treated with at least one dose.
|
|
Vascular disorders
Hypotension
|
0.00%
0/12 • All-cause mortality: From first drug administration until individual end of study. Up to 60 days. Adverse event reporting: From first administration until last drug administration plus 7-day residual effect period. Up to 53 days.
Treated set (TS): all trial participants who were randomized to the trial medication and were treated with at least one dose.
|
10.0%
1/10 • All-cause mortality: From first drug administration until individual end of study. Up to 60 days. Adverse event reporting: From first administration until last drug administration plus 7-day residual effect period. Up to 53 days.
Treated set (TS): all trial participants who were randomized to the trial medication and were treated with at least one dose.
|
Additional Information
Boehringer Ingelheim, Call Center
Boehringer Ingelheim
Phone: 1-800-243-0127
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee Boehringer Ingelheim (BI) acknowledges that investigators have the right to publish the study results. Investigators shall provide BI with a copy of any publication or presentation for review prior to any submission. Such review will be done with regard to proprietary information, information related to patentable inventions, medical, scientific, and statistical accuracy within 60 days. BI may request a delay of the publication in order to protect BI's intellectual property rights.
- Publication restrictions are in place
Restriction type: OTHER