About Clinical Studies

What is a clinical study?

Clinical research studies (also called clinical trials) are needed for advancing the treatment of medical conditions and diseases. All medications must be tested in clinical research studies before they can be approved by local regulatory health organizations and prescribed by doctors.

During a clinical study, researchers may want to find out the following about an investigational drug:

If it has manageable side effects
If it works the way it is expected to
If it doesn’t work as well, or works the same or better than other drugs
How it behaves in the human body (for example, how the study medication moves around and how quickly it passes through the body)

Clinical study phases

There are 4 steps in the clinical research process, called “phases.” Each phase has a different purpose to help researchers answer different questions.

Phase 1

We test the investigational drug in a small group of people, usually healthy volunteers. We’re looking at how safe it is and how the body handles it. This helps us find the right dose and learn more about side effects.

Phase 2

We give the investigational drug to people who have the condition we’re trying to treat. We check to see if it’s effective and continue to keep track of its safety. This phase helps us understand how well it works and refine the dosage.

Phase 3

We test the investigational drug in a much larger group of patients. We compare it to existing treatments or a placebo (inactive treatment). This phase confirms if it’s well-tolerated, meaning any side effects are manageable, and effective. If the investigational drug successfully finishes phase 3, it can be submitted to health authorities to get approval for more widespread use.

Phase 4

This happens after the investigational drug is approved and available to patients. We keep track of its long-term safety and effectiveness in real-world use. We might also study it for new uses or in different patient groups.

The ILLUMINATE Study is classified as a phase 3 study.

Who is involved in clinical studies?

Clinical studies take place in clinics, hospitals, or other healthcare facilities. For some studies, it is possible that some study assessments are carried out at the patient’s home with a qualified nurse.

During a clinical study, a dedicated team of researchers, doctors, and nurses will support you or your loved one. Each study team member is committed to you or your loved one’s health and well-being. At each phase of drug development, government agencies check the results to see whether the drug can continue to the next phase or whether the medication can also be used in other groups of patients or in other diseases.

By joining a clinical study like the ILLUMINATE Study, you or your loved one has the opportunity to possibly help other people living with Rett syndrome in the future.

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