Background: There is currently no FDA or EMA-approved standard of care for metastatic uveal melanoma.
Material and methods: A systematic review of all interventional uveal melanoma trials on the ClinicalTrials.gov database and EU Clinical Trials Register was conducted from January 15, 2019 through November 30, 2019. Categorical data analysis and descriptive statistics were generated.
Results: A total of 119 trials met inclusion criteria for this systematic review, of which 39 were active. Of all trials, 47% were NIH-funded while 59% of active trials were industry funded. Of all trials, 86% were concerned with treatment of metastasis, 7% with adjuvant therapy, and 8% with treatment of primary tumor. In trials treating metastasis, 62% reported response rates as their primary outcome measure. Non-randomized patient allocation to treatment arms was reported in 73% of trials, and 8% of trials were in phase 3. Pharmaceutical drugs were utilized by 69% of trials. Of the 6 negative randomized trials, all reported no significant effect from intervention compared to a control arm and were usually initiated on preclinical or early phase data.
Conclusion: Given decreased NIH funding for uveal melanoma trials, clinicians should consider industry funding partnerships. Standardization of primary outcome measures between trials will also allow for statistical meta-analyses of results in this rare patient population. Finally, clinicians should use single arm studies to establish significant treatment response rates before proceeding with randomized trials.