Acute infantile liver failure due to synthesis defect of mtDNA-encoded proteins
MONDO:0013111Acute infantile liver failure due to mtDNA-encoded proteins synthesis defect is a very rare mitochondrial respiratory chain deficiency described in fewer than 10 infants, primarily of middle Eastern descent, and characterized clinically by transient but life-threatening liver failure with elevated liver enzymes, jaundice, vomiting, coagulopathy, hyperbilirubinemia, and lactic acidemia.
Also known as: acute infantile liver failure, LFIT, TRMU infantile liver failure, acute infantile liver failure due to synthesis defect of mitochondrial DNA-encoded proteins, acute infantile liver failure due to synthesis defect of mtDNA-encoded proteins, infantile liver failure caused by mutation in TRMU, liver failure, infantile, transient, liver failure, transient infantile
184 clinical trials for this condition and its sub-types.
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New drug aims to tame hard-to-control seizures in rare mitochondrial disorders
Disease control TerminatedThis study tested a drug called vatiquinone in 68 people with mitochondrial disease and epilepsy that doesn't respond to standard treatments. Participants were randomly assigned to receive either vatiquinone or a placebo for 24 weeks to see if the drug could reduce the number of …
Phase: PHASE2, PHASE3 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated Jun 27, 2026 12:03 UTC
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Blood RNA may reveal hidden liver cancers
Diagnosis TerminatedThis study looked at whether tiny bits of RNA in the blood can serve as markers for liver disease and liver cancer. Researchers collected blood samples from nearly 1,900 people with liver cancer, cirrhosis, or chronic liver disease. The goal was to see if these RNA markers could …
Sponsor: Mayo Clinic • Aim: Diagnosis
Last updated Jun 27, 2026 12:29 UTC
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Ear device aims to cut opioid use after liver transplant
Symptom relief TerminatedThis study tested a small device worn on the ear that uses mild electrical pulses to help control pain after a liver transplant. The goal was to see if it could reduce the need for strong painkillers like opioids. The study was stopped early and included 27 adults waiting for a l…
Phase: NA • Sponsor: Duke University • Aim: Symptom relief
Last updated Jun 27, 2026 13:00 UTC
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Liver study of cancer drug amcenestrant halted early
Knowledge-focused TerminatedThis early-stage study looked at how the drug amcenestrant is processed in the body of women aged 40-75 with mild or moderate liver problems, compared to women with healthy livers. The goal was to understand dosing and safety. However, the trial was terminated before completion, …
Phase: PHASE1 • Sponsor: Sanofi • Aim: Knowledge-focused
Last updated Jun 27, 2026 07:51 UTC