Clinical Trials and Research Studies at Inova

Medical treatments have come a long way in the last 30 years. Today, many more patients are surviving serious illnesses that they might not have years ago. This is due to improvements in medical treatments and recognition of the special medical and nursing needs of patients. These improvements have come about through research, both in the laboratory and in the clinical setting. However, there are still many questions to be answered about treatments for patients and the care they receive. Finding answers to these questions is why research is an essential part of Inova.

Inova conducts two types of clinical trials: industry-sponsored and investigator-initiated. Research projects at Inova hospitals provide answers to questions regarding the care of patients. They also contribute to the growing body of medical knowledge about different conditions and treatments.

You may be asked to take part in a research trial or to give permission for a family member who is not able to make the decision for themselves. Because this request sometimes comes at a time of emotional stress, we would like to provide some general information explaining research, to help you make an informed decision.

A clinical trial is a thorough and carefully controlled evaluation of a new medical test or treatment.

Before a clinical trial begins, the treatment must show that it has potential benefit. It also must meet rigorous government standards and scientific requirements for safety, and have acceptable side effects.

Ultimately, the only way to determine the true benefit and safety of a treatment is to test it on people with the disease or condition for which the treatment was developed. Clinical trials are one of the steps in determining how effective a drug or treatment is in combating a disease or medical condition.

There are many different types of clinical trials and many ways patients and their families can participate. A clinical trial can:

• Compare two medications or treatments to learn which works better for patients
• Examine how well a medication works and what side effects may occur
• Observe the general condition of the patient to learn about a specific disease process or group of symptoms, and how they affect patients
• Test new medical devices

Physicians, nurses and other researchers follow strict rules when conducting clinical trials. These rules protect the interests of the patient and remove any bias from the study.

The best available research design is known as a randomized, controlled, double-blind clinical trial. Here's how it works:

• Patients who have agreed to participate in the study are assigned to one or another treatment or medication by random choice.
• When a new medication is being studied, it may be compared to an existing medication, or to a placebo. A placebo is a harmless substance with no medical effect. It is manufactured to look identical to the medication being tested.
• No one directly involved – the patient, the nurses and the physicians – know whether the placebo or the study medication is being used. This is called a placebo-controlled double-blind study.
• The study medication is given to one group; the placebo is given to the other. Patients in both groups are watched carefully during the study and the reactions of both groups recorded.
• At the end of the study, the reactions to the treatment are compared to see if one group responded better than the other. This information is used to determine if the new medication was effective in treating the disease. Only after the trial is completed do researchers find out which patients were in which group.

An investigator-initiated research study is also a thorough and carefully controlled study. However, in this type of study, the concept and the protocol is conceived by our own clinician rather than a pharmaceutical or device company.

These are the earliest trials in the life of a new drug or treatment. They are usually small trials, involving up to 30 patients (often a lot fewer). When initial laboratory research shows that a new treatment might help treat a specific disease, Phase 1 trials are done to find out more about:

• Safe dose range
• Side effects
• How the body copes with the drug

Phase 1 clinical trials may be done on healthy subjects or those with a specific disease. The aim of Phase 1 clinical trial is to look at different doses and side effects. This work has to be done first, before we can test the potential new treatment to see if it works. Phase 1 trials are important because they are the first step in finding new treatments for the future. Subjects in Phase 1 clinical trials will be given a small dose of the drug being tested. If all goes well, the next subject will get a higher dose. With each subject taking part, the dose will gradually be increased and the effect that it has will be carefully monitored. Any side effects will be recorded. In a Phase 1 trial, you may have lots of blood tests as the researchers look at how the drug is affecting you, and how your body copes with the drug.

About 7 out of every 10 new treatments tested at Phase 1 make it to Phase 2 trials. These trials may be done on people who all have similar diseases. Phase 2 trials are done to find out:

• If the new treatment works well enough to test in Phase 3
• More about side effects
• More about the most effective dose to use in Phase 3 clinical trials

Although these treatments have been tested at Phase 1, there may still be side effects that are not known. Drugs can effect people in different ways.

These trials compare new treatments to the best currently available treatment and/or placebo (the standard treatment). They may compare:

• A completely new treatment with the standard treatment
• Different doses or ways of giving a standard treatment
• A new radiotherapy schedule with the standard one
• A new device that can be tested vs. standard treatment

Phase 3 trials are usually much larger than Phases 1 or 2. Because differences in success rates may be small, many patients are needed in the trial to show the difference.

Phase 3 trials sometimes involve thousands of patients in many different hospitals, and even different countries. Generally, Phase 3 trials are required before approval of a new medication or treatment.

Systematic reviews and meta analysis
Systematic reviews are studies that combine the results of multiple Phase 2 and 3 trials of a new treatment or diagnostic tests. Another type of study that will combine research results is meta analysis. The idea is to get a broader picture of how well a treatment works and how many side effects it potentially has. The more data there is, the more accurate such studies are likely to be.

Phase 4 trials are done after a drug has been shown to work and has been approved. In Phase 4 clinical trials, more long-term data about the efficacy and safety of a drug or device are gathered. Phase 4 clinical trials are run to find out more about long-term side effects and the safety of the drug or device, what the long-term risks and benefits are to the patients, and how well the drug/device works when it is used in clinical practice. Phase 4 trials look at drugs/devices that are already available for doctors to prescribe, rather than new ones being developed.