Gemphire Therapeutics halts phase 2a trial in NAFALD.

Gemphire Therapeutics  is halting its phase 2a trial in pediatric nonalcoholic fatty liver disease (NAFLD) because some patients’ disease got worse.

This report on fiercebiotech describes how an increase in liver fat was deemed an unexpected problem by the trial investigator. It was an unexpected consistent pattern of worsening of the disease, rather than improvement. The investigators believed this effect was due to the drug, and created risk to the patients,

The trial started early this year, patients were dosed with 300 mg of gemcabene daily. The primary endpoint was a change in serum alanine transaminase (ALT), which is an an enzyme biomarker of liver function, after 12 weeks of treatment. Secondary endpoints included a change in hepatic steatosis, the buildup of fat in the liver.

The first three patients that completed 12 weeks of treatment had an increase in liver fat content, as well as elevated ALT levels.



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The creation of the NASH Data Commons will provide a unified data system that promotes sharing of genomic and clinical data, forming the basis for a comprehensive knowledge system that centralises, standardises and makes accessible data. These datasets will lead to a much deeper understanding of which therapies are most effective for individual patients. With each new dataset added e.g. additional ‘omics’ data, it will evolve into a smarter, more comprehensive knowledge system that will foster important discoveries in chronic liver disease and increase the success of treatments for patients.

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