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The landscape of clinical trials research in inherited ophthalmic disease
Vincent Ng1, Cheuk Ying Li2, Paul Cornes3
1Department of Ophthalmology, University Hospitals Birmingham NHS Foundation Trust, Birmingham, UK.
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(verification expired with no update), 14 (4.9%) trials which were terminated early and 6(2.1%) trials which were withdrawn.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.
Insights
Clinical trials for genetic eye diseases show promise, but few completed studies have posted results. Future research on molecular targets could lead to new treatments for inherited vision conditions.
Area of Science:
- Ophthalmology
- Genetics
- Clinical Research
Background:
- Genetic eye diseases represent a significant challenge in ophthalmology.
- Identified molecular targets offer potential pathways for therapeutic development.
Purpose of the Study:
- To assess the current landscape of clinical trials for genetic eye diseases.
- To identify research gaps and future directions based on molecular targets.
Main Methods:
- A comprehensive analysis of clinical trials.gov was conducted.
- Keywords related to eight common inherited eye diseases and their molecular targets were used.
- Data was collected from March 20, 2021, to December 31, 2023.
Main Results:
- 243 trials were included in the analysis after exclusions.
- 120 trials were completed, 76 were active and recruiting, and 44 were active but not recruiting.
- Only 32 (13.2%) of completed trials had posted results.
Conclusions:
- A low percentage of results are reported for completed genetic eye disease trials.
- Despite this, clinical trials targeting molecular pathways show significant future potential.
- Further research will improve understanding and treatment development for these conditions.
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