Acute infantile liver failure due to synthesis defect of mtDNA-encoded proteins

This study tested a new drug called OMT-28 in 28 people with primary mitochondrial disease, a condition that affects how cells produce energy. Participants took the drug once daily for 6 months, and researchers measured safety, blood markers of inflammation, and symptoms like fat…

This study looked at the safety of vatiquinone in 101 people with inherited mitochondrial disease who had already taken the drug in a previous study or treatment plan. The goal was to track any side effects until the drug became commercially available or the program ended. Partic…

This study investigates how a single dose of mavorixafor is processed in people with mild to moderate liver impairment compared to healthy volunteers with normal liver function. Researchers will measure drug levels in the blood and monitor safety and tolerability. The goal is to …

This study looked at how the body handles a drug called BI 1584862 in people with different levels of liver function. 43 adults with mild, moderate, or severe liver problems, plus a group with healthy livers, took a single dose of the drug. Researchers measured drug levels in the…

This study looked at how the experimental drug iclepertin (BI 425809) is processed in people with and without liver problems. 29 adults aged 18-79 took a single dose of the drug. Researchers measured drug levels in the blood and monitored safety to see if liver impairment changes…

This pilot study looked at 38 liver transplant patients to see if liver failure lowers blood levels of pyrophosphate, a natural substance that prevents calcium buildup in tissues. Researchers measured pyrophosphate before and after transplant and checked liver tissue for a relate…

This study looks at how the drug BI 1015550 is absorbed and processed in the blood of people with mild or moderate liver problems compared to those with healthy livers. About 28 adults took a single dose and had blood samples taken over several days. The goal is to understand if …

This completed Phase 1 study looked at how imlunestrant, an oral drug, enters the bloodstream and leaves the body in women with and without liver impairment. 27 female participants took a single dose, and researchers measured drug levels over time. The goal is to understand if li…