What is a clinical trial?

Why do people participate in clinical trials for genetic eye disease?

People participate in clinical trials for different reasons. Some participate because they want to learn more about their disease. Others participate because they want to help with the development of new treatments that could help them and others in the future. If you take part in a clinical trial, you may be one of the first people to benefit from new study medication. However, there is always a chance that the new medication is not better, or worse, than the standard treatment (if a standard treatment exists) or that you will receive the sham procedure. A sham procedure is used studies evaluating intravitreal injections to mask the study participant to treatment assignment.

Are clinical trials safe?

Clinical trials follow a specific set of standards and are strictly regulated to help keep all participants safe. 

All clinical trials are reviewed and approved by a committee of independent experts (Ethics Committee or Institutional Review Board). During a clinical trial, participants are carefully monitored by the clinical trial team. In addition to eye tests, general safety tests may be performed during study visits, such as blood tests, blood pressure, heart rate and temperature.

What should I know before joining a clinical trial?

It is important to know as much as you can about a clinical trial before joining. 

You should carefully review any requirements and risks and talk with your clinical trial doctor to make sure participating in the clinical trial is a good choice for you. Remember the following when considering if you should join a clinical trial:

• Clinical trial participation can be time consuming. You may be expected to attend multiple visits at the study clinic where you will undergo several tests to monitor if there is any improvement and if the study treatment is safe.
• You may see different members of the clinical trial team, like technicians who specialize in specific eye tests. Your regular ophthalmologist may or may not be involved in the clinical trial but will be kept informed of your participation and progress. 
• Some clinical trials require you to stay overnight for some visits. In some trials, visits may be spread out over 2 or more days to increase convenience and reduce the burden of participation. Your clinical trial team will inform you exactly  what will be required from you during clinical trial participation.
• There may be restrictions on what you can and cannot do. For example, you may be asked to avoid or stop taking certain medications or to prevent pregnancy during study participation. 
• There is a chance that you may experience unknown side effects from the study treatment.

Who can join a clinical trial? 

Every clinical trial has a clinical trial plan, also known as a study protocol, which describes what will be done during the clinical trial, how it will be done, and why it is necessary. It also includes criteria on who can and cannot join. Some common criteria include: 

• age
• type of eye disease
• medical history 
• past treatments 
• specific genetic profile

You may also be reimbursed for time and travel during a clinical trial, but this depends on what is allowed at your study clinic. The informed consent form for the specific clinical trial will provide details on what reimbursement or payment you may receive for a clinical trial. 

Can I leave a clinical trial once it starts?

Clinical trial participation is completely voluntary. 

You do not need to take part in a clinical trial, and you can end your participation at any time, for any reason. If you leave a clinical trial early, the clinical trial doctor may ask that you return to the study clinic for a final visit. 

Can I join a ProQR clinical trial?

We are currently running several clinical trials in different inherited retinal diseases. Talk to your doctor or read more about the clinical trials below.