Gemphire Therapeutics Inc. has been recommended to stop a clinical trial by the Data and Safety Monitoring Board at Emory University School of Medicine. The clinical trial, which ran into unanticipated problems, was investigating gemcabene in paediatric patients with a diagnosis of non-alcoholic fatty liver disease. The source article can be read here, at Gemphire’s website.
About Non-Alcoholic Fatty Liver Disease (NAFLD)
NAFLD refers to a range of conditions that are associated with the build-up of fat in the liver. According to the NHS, an estimated one in three people living in the UK have early-stage NAFLD. In these early stages, the condition usually doesn’t cause harm, but if the condition develops it can lead to liver damage through processes such as scarring (cirrhosis).
Typically, NAFLD is not associated with symptoms at the early stages and is likely to first be identified through tests carried out for other reasons. However, at a later stage, it may be associated with fatigue, aching in the top right of the stomach, weight loss, and weakness, amongst other symptoms.
The Study Methodology
The study was a Phase 2a proof-of-concept clinical trial of gemcabene in paediatric patients who have NAFLD, and it began in early 2018. On the trial, patients were given 300mg of gemcabene once per day. Researchers were evaluating the change in an enzyme called ALT, which acts as a liver function biomarker, as well as other factors including changes in liver fat (measured through a form of MRI imaging).
The Trial Termination Recommendation
The trial investigator became concerned when the first three enrolled patients who had twelve weeks of treatment all showed a raised liver fat content and increases in the ALT biomarker enzyme. This was suggested that the condition was worsening, rather than improving, in these patients, and the trial investigator thought that these were likely to be effects of gemcabene.
Patients enrolled in the study have now been taken off the drug. In addition to the termination of the trial, the Data and Safety Monitoring Board have recommended that enrolled patients are followed up for more safety information, and, once all the data has been collected, a report will discuss the findings.
Gemcabene has been studied in previous Phase 1 and 2 trials involving just under 1,200 adult participants without serious adverse events thought to be related to the drug being reported. Gemphire’s CEO has affirmed the company’s intention to continue developing gemcabene for several indications in cardiometabolic patients.
