The landscape of clinical trials research in inherited ophthalmic disease

ABSTRACT

Objective

To describe the current status of clinical trials of genetic eye diseases with identified molecular targets for future areas of research.

Method

Data analysis of the clinical trials database on clinicaltrials.gov with keywords for eight common, genetically tractable inherited eye diseases and their common molecular targets was performed during the period from 20 March 2021 to 31 December 2023.

Results

Two hundred and eighty-eight trials involving our keywords have been identified, excluding 25 (8.7%) trials which were unknown (verification expired with no update), 14 (4.9%) trials which were terminated early and 6(2.1%) trials which were withdrawn. In total there were 243 (84.4%) trials included. Out of the 243 trials, 120 trials were completed, 76 trials were active and still open to recruitment and 44 trials were active without any more recruitment on the way. There were only 32 (13.2%) trials with posted results.

Conclusions

A low percentage of results were posted for completed trials. However, current and future clinical trials in the genetic eye diseases with molecular targets identified, have a promising future. The results of these trials will enhance and allow a better understanding of the potential to develop treatments for these conditions.

KEYWORDS:

• Inherited ophthalmic disease
• genetics
• clinical trials
• gene therapy
• drug intervention

Disclosure statement

The authors declare that the research was conducted in the absence of any commercial or financial relationships that could be construed as a potential conflict of interest.

Supplemental data

Supplemental data for this article can be accessed online at https://doi.org/10.1080/13816810.2024.2378013.