Drug-Induced Liver Injury During a Glaucoma Neuroprotection Clinical Trial.

Abstract: There are several ongoing, worldwide clinical trials with a cumulative target enrollment of over 1300 participants on the role of nicotinamide (a specific form of vitamin B3) as a therapeutic neuroprotective treatment for glaucoma. We describe a serious adverse event of drug-induced liver injury (DILI) likely related to the use of 3 g/day nicotinamide in a glaucoma clinical trial (clinicaltrials.gov identifier: NCT05695027) based in the United States. This report is important to share with the medical community, as other participants in glaucoma nicotinamide trials globally may have similar adverse events and many patients are using nicotinamide as a health supplement without medical supervision. We recommend that investigators, physicians, and patients remain vigilant about DILI as they seek novel vision-preserving neuroprotective therapies.