In short
Clinical trials are studying Vonafexor in people with impaired kidney function and suspected MASH, as well as patients with Alport syndrome at risk of disease progression. These studies mainly look at safety, tolerability, and whether Vonafexor can affect kidney-related measures.
Key points
- Vonafexor has been studied in two completed Phase 2 clinical trials. One trial looked at people with impaired kidney function and suspected MASH, while the other studied patients with Alport syndrome at risk of progression. The main goals were to assess safety, tolerability, and kidney-related outcomes. One study measured changes in GFR, which shows how well the kidneys filter blood. The other study tracked treatment-emergent adverse events and changes in physical exams, vital signs, laboratory tests, and lipid profile. Both studies were interventional and focused on specific patient groups.
Trial overview
Two Phase 2 interventional studies were listed for Vonafexor, and both were marked completed. These studies looked at different patient groups: one with impaired renal function and suspected MASH, and one with Alport syndrome at risk of progression.
Impaired renal function and suspected MASH
The study with NCT ID 2023-509192-16-00 enrolled 50 people and was completed. It studied people with impaired renal function and suspected MASH, which means a liver condition with fat and inflammation, along with reduced kidney function. The brief summary said the goal was to determine the effect of Vonafexor on renal function in people with suspected MASH and mild to moderately reduced GFR.
The primary outcome was the change from baseline in mGFRiohexol and eGFRcreat at week 16. In simple terms, the study checked whether kidney filtering changed after treatment compared with the start of the study. The listed interventions included Vonafexor by mouth at 25 mg and 100 mg, along with rosuvastatin and iohexol used for measurement.
Alport syndrome study
The study with NCT ID 2023-509638-20-00 enrolled 24 patients and was also completed. It focused on patients with Alport syndrome who were at risk of progression. The brief summary said the purpose was to assess the safety and tolerability of Vonafexor both during treatment and after treatment stopped.
The primary outcome tracked the number of treatment-emergent adverse events from the first dose until 2 weeks after the last dose. It also measured changes in physical examinations, vital signs, laboratory variables, and lipid profile compared with baseline, which is the starting point before treatment begins. The intervention listed for this study was oral Vonafexor 100 mg.
What was measured in the studies
The kidney-focused study measured GFR, which is a way to see how well the kidneys filter blood. It used both a measured method with iohexol and an estimated method based on creatinine. The Alport syndrome study focused more on safety checks, including adverse events, physical exams, vital signs, laboratory values, and blood fats.
- Baseline means the starting point before treatment or study procedures begin.
- Vital signs are basic body measurements such as blood pressure and pulse.
- Laboratory variables are blood or urine test results that help show how the body is working.
- Lipid profile is a blood test that measures fats such as cholesterol.
Who the studies focused on
These trials were not broad studies of all patients with kidney or liver disease. They focused on specific groups: people with suspected MASH and reduced kidney function, and people with Alport syndrome at risk of getting worse. This makes the results more relevant to those exact patient groups, but not necessarily to everyone with similar symptoms.