Drug-Induced Liver Injury During a Glaucoma Neuroprotection Clinical Trial

Area of Science:

• Ophthalmology
• Neuroprotection
• Clinical Pharmacology

Background:

• Nicotinamide (vitamin B3) is being investigated globally for neuroprotective effects in glaucoma treatment.
• Numerous clinical trials are underway, with over 1300 participants targeted for enrollment.
• Glaucoma patients may also use nicotinamide as a supplement without medical supervision.

Purpose of the Study:

• To report a serious adverse event of drug-induced liver injury (DILI) associated with nicotinamide in a glaucoma clinical trial.
• To raise awareness within the medical community about potential DILI risks.
• To encourage vigilance regarding DILI in patients undergoing nicotinamide therapy for glaucoma.

Main Methods:

• Case report of a serious adverse event in a clinical trial (NCT05695027).
• Assessment of the likely relationship between nicotinamide dosage (3 g/day) and the adverse event.
• Review of ongoing global clinical trials involving nicotinamide for glaucoma.

Main Results:

• A participant in a US-based glaucoma clinical trial experienced drug-induced liver injury (DILI).
• The DILI was likely related to the administration of 3 grams of nicotinamide daily.
• This highlights a potential safety concern for patients in nicotinamide trials.

Conclusions:

• Vigilance for DILI is crucial for investigators, physicians, and patients in nicotinamide glaucoma trials.
• The findings underscore the importance of medical supervision when using nicotinamide, even as a supplement.
• Further safety monitoring is warranted for nicotinamide as a potential neuroprotective therapy for glaucoma.