Ritivixibat (A3907) Phase 2 Clinical Trial Results
Ritivixibat (A3907) Phase 2 Clinical Trial Results
Safety results for the Phase 2 ritivixibat study (A3907) for PSC have been released
Results from the Phase 2 clinical study (A3907-002) evaluating the safety and tolerability of ritivixibat in adults with primary sclerosing cholangitis (PSC) are now available. The study, sponsored by Ipsen, found that ritivixibat was generally well-tolerated by participants over a 12-week treatment period.
What was the purpose of the ritivixibat study?
The primary goal was to assess the safety and tolerability of repeated doses of ritivixibat in people living with PSC. Researchers also investigated whether the drug could lower bile levels in the body by interfering with the process where bile is reabsorbed into the blood, which may help decrease liver damage.
What were the key safety findings?
Most participants (81%) experienced at least one medical problem during the 12 weeks. These include any health changes reported, whether or not they were caused by the drug.
- No deaths or serious side effects occurred.
- 39% of participants experienced side effects considered related to the treatment.
- Common side effects included diarrhoea (22%), stomach pain (11%), and passing gas (11%).
- Some participants (11%) saw an increase in certain liver proteins (ALT and AST) in their blood.
How did the amount of bile in blood and urine change from the start of treatment until the end of 12 weeks of treatment?
The researchers did not find any notable change in the amount of bile in blood and urine from the start of treatment until the end of 12 weeks of treatment. However, due to low number of participants, the researchers could not make a meaningful conclusion about these results.
Why was the study stopped early?
The sponsor stopped the study early because of difficulties in finding enough participants. Because of the small sample size (18 participants), researchers could not reach a meaningful conclusion regarding changes in bile levels. Currently, there are no future studies planned for ritivixibat in PSC.
PSC Research
At PSC Support, we highlight trial outcomes to ensure our community stays informed about the research landscape. Find out more: PSC research results and clinical trials that are looking for volunteers.
Turning Ambition into Action in 2026:
Our Fortnight in Focus
At PSC Support, we want a world without PSC. We work tirelessly behind the scenes to drive research and improve lives. Here is a snapshot of what we've been up to:
Organisational excellence: We simplified our personal data consent process and streamlined how we record patient stories to ensure that the lived experiences of our community are captured accurately and securely to inform future work.
Progressing Research: We coordinated a series of upcoming meetings with international researchers and pharmaceutical companies, which supports better informed decisions for people with PSC, research, or care. This groundwork helps keep PSC research focused on the primary needs of the patient community.
Progressing Research: We reviewed and contributed to scientific abstracts and posters for the upcoming EASL international liver congress, which strengthens the quality of future research for people with PSC, research, or care.
Progressing Research: We engaged with the MHRA to prepare for the upcoming national consultation on rare disease therapies, representing PSC patients in the wider community. Maintaining this direct dialogue ensures our organisation is prepared to respond formally and protect the interests of those living with PSC
Improving Care: We presented about what it is really like to live with PSC to gastroenterologists This first-hand insight equips healthcare professionals to better understand the day-to-day challenges of PSC.
Income Generation: We secured a place in the prestigious Big Give Small Charity Week match-funding campaign, so that we can maximise future donations from our supporters as every donation between 22 and 29 June will be doubled!
Progressing Research: We recruiting for a research and advocacy officer to expand our core team. This additional capacity ensures we can better support scientific studies and represent the patient voice at every level.