Originally published in NKCF Update (June 2024).

Medical device businesses, pharmaceutical companies, the National Eye Institute, university medical centers, and even individual doctors may have a new device, drug, or treatment to evaluate. They want to compare their new product to the existing standard of care. At some point, human volunteers are needed to collect data on safety and effectiveness. This is the clinical trials process.

Investigators design a study protocol, a document that contains the recipe for treatment. Issues like drug dosages, length of treatment, schedule of tests, and the types of patients who are eligible to participate are outlined. The protocol is approved by a group of independent experts (called the Institutional Review Board or IRB) who determine if the plan is well constructed with the safety of the human study subjects as the top priority.

The National Institutes of Health do not regulate clinical trials. However, they do maintain an on-line registry of available clinical trials (https://clinicaltrials.gov/). If you have an interest in learning more about a particular clinical trial, you can type in a location or key word (like ‘keratoconus’) and you’ll find a database of active and recently completed studies.

Why Should I Consider a Clinical Trial?

Each person must weigh the pros and cons of enrolling in a clinical trial. In any clinical trial, study subjects can drop out at any time. By participating, you may have access to a new treatment or drug before it is widely available. Enrolling in a study often means you receive comprehensive exams and testing, so you will learn more about your disease.

You may be reimbursed for certain expenses you incur like travel or parking. In some cases, study subjects are paid to participate. Most importantly, what is learned by your participation may be critical to helping other patients and contribute to medical knowledge.

On the negative side, you may experience a reaction ro side effect to the treatment or drug. You may be expected to attend multiple follow-up visits over an extended period which may be an inconvenience. If you are participating in a double-blind study, neither you nor your doctor will know if you are receiving the actual investigative treatment or a placebo.

Two very important industry-sponsored clinical trials for keratoconus are underway. Glaukos, the company that offers the FDA-approved epithelium-off crosslinking procedure began a clinical trial in March 2022 to test the success of an epithelium-on treatment. 150 subjects between the ages of 18 and 55 were enrolled. Enrollment is closed and all subjects have received treatment. The protocol requires a 1-year follow-up, which is underway now. Data will be analyzed and submitted for FDA review in 2025.

Another large-scale epithelium-on crosslinking clinical trial is sponsored by Epion Therapeutics. The company began patient enrollment in October 2023 and enrollment is still open to eligible patients. Their goal is to treat 800 individuals between the ages of 8 and 45 years old who have been diagnosed with keratoconus but have not received any previous crosslinking. There are 24 study sites in 17 states. If you have questions about participating in this trial, email info@epiontx.com. Check the company website (epiontx.com) for an upcoming webinar and more information about clinical trial enrollment.