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

This early-phase trial is testing a new bioartificial liver device that uses liver cells grown from stem cells. It will be given to 12 people with liver failure or small-for-size syndrome. The main goal is to see if the treatment is safe and tolerable, while also checking for any…

This early-stage trial is testing whether giving a mix of healthy gut bacteria (from donors) to people hospitalized with serious liver disease is safe and tolerable. The study involves 24 adults with liver failure or cirrhosis who have low levels of certain gut metabolites. Resea…

This Phase 2 trial tests whether KL1333 can reduce fatigue and improve leg strength in adults with primary mitochondrial disease, a genetic condition that affects energy production. About 180 participants will receive either KL1333 or a placebo twice daily for 48 weeks. The study…

This study at the National Institutes of Health looks at how infections can worsen symptoms in people with mitochondrial disease, a group of disorders that affect energy production in cells. Researchers will evaluate participants' immune systems through blood tests, physical exam…

This study creates a global registry for people with mitochondrial disorders—rare diseases that affect energy production in cells. By collecting health data from 6,000 participants worldwide, researchers aim to understand how these diseases progress and identify the best ways to …

This study is creating a large registry and tissue bank for people with mitochondrial disorders. Researchers will collect medical information and samples from up to 1,000 participants, including those diagnosed with or suspected to have a mitochondrial disease. The goal is to gat…

This study collects blood and skin samples from 100 people with primary mitochondrial diseases and healthy volunteers aged 3 to 85. Researchers will study how different mitochondrial mutations affect cell function and look for biomarkers. The samples will also help test a new the…