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
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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:
Building Partnerships: We contributed a comprehensive chapter to a new medical textbook for nurses and allied health professionals, ensuring that the patient perspective is integrated into clinical education.
Building Partnerships: We participated in a planning meeting for the update of national clinical guidelines for PSC.
Building Partnerships: We prepared strategic questions for an upcoming international forum titled “Putting Patients First: From Research to Treatment.”
Organisational Excellence: We updated our web details to make it less likely information from us sent by email ends up in your spam folders.
Progressing Research: We are collaborating with European partners to develop a simplified research registry for pregnancy outcomes, building the evidence base needed to improve medical guidance for women with PSC.
Progressing Research: We completed a formal submission to the Scottish Medicines Consortium to advocate for the value of a potential new treatment for PSC patients.
Progressing Research: We reviewed the participant information for a new academic clinical trial to ensure it is clear and accessible for patients.