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:
Improving Care: We attended a major national clinical conference to equip general gastroenterologists and nurses with specialist knowledge, which helps ensure future work is better aligned with what people with PSC need by improving local care and pathways.
Progressing Research: Our 2024 investment into early-stage research to build necessary scientific evidence enabled Dr James Sun to secure major national funding to explore the genetic drivers of the disease. This foundational knowledge is essential for identifying potential new treatments.
Progressing Research: We supported the development of a research manuscript on nutrition in PSC, so that the findings can be effectively shared with the wider medical community. This will help us advocate for the needs of people with PSC to ensure they get the care they need at the right time.
Improving Care: We hosted a clinical consensus meeting to map out an ideal PSC care pathway, which helps ensure future PSC management is better aligned with what people with PSC need. Establishing standard protocols across the NHS is essential for eliminating regional variations in care.
Progressing Research: We contributed to the strategic planning of an international rare liver disease network, which supports progress towards better care for people with PSC. Broadening our influence ensures patient needs are integrated into wider clinical strategies.
Improving Care: We shared firsthand experiences of living with PSC at an international medical school event, which improves clinical understanding of the disease for healthcare professionals. This is important because ensuring future specialists understand the human impact of PSC helps drive patient-centered care and clinical excellence.
Progressing Research: After just over 48 hours on our Big Give Small Charity Week match funding initiative, your donations helped us get to 78% of the way to our £20,000 goal! Thank you! Can we reach 100%?