Participating in a Clincial Trial - Questions and Answers

Rapid advances in our knowledge about Alzheimer's Disease (AD) have led to the development of many new drugs and treatment strategies. However, before these new strategies can be adopted, they must be shown to work on patients. This means that clinical trials - studies using humans to rigorously test how well a treatment works - have become an increasingly important part of AD research. Advances in treatment are only possible through the participation of patients and family members in these clinical trials.

Clinical trials are the primary way that researchers find out if a promising treatment is safe and effective for patients. Clinical trials also tell researchers which treatments are more effective than others. Trials take place at private research facilities, teaching hospitals, specialized AD research centers, and doctors' offices.

Participating in a clinical trial is a big step for people with AD and their caregivers. That's why physicians and clinical trials staff spend lots of time talking with participants about what it's like to be in a trial as well as the pros and cons of participation. Here are some things that potential participants might want to know about clinical trials.

> WHAT KINDS OF TRIALS ARE THERE?

Treatment trials with existing drugs assess whether an already approved drug or compound is useful for other purposes. For example, one current trial is testing whether anti-inflammatory drugs already used to treat arthritis might help to prevent AD.

Treatment trials with experimental drugs or strategies find out whether a brand new drug or treatment strategy can help improve cognitive function or lessen symptoms in people with AD, slow the progression of AD or even prevent it. Experimental drugs tested in these trials are developed from knowledge about the mechanisms involved in the AD disease process. These compounds are rigorously tested using tissue cultures and animals to discover their results. Safety and effectiveness studies are also conducted with animals before the compounds are tested on humans.

> WHAT ARE THE PHASES OF CLINICAL TRIALS

During Phase I trials, a study team gives the treatment to a small number of volunteers and examines its action in the body, its safety, and its effects at various doses. Phase I trials generally last only a few months.

If results show that the treatment appears safe, it will be tested in Phase II and Phase III clinical trials. These trials involve larger numbers of people over longer periods of time. In these trials, the study team wants to know whether the treatment is safe and effective and what side effects it might have.

After these phases are complete and investigators are satisfied that the treatment is safe and effective, the study team may submit its data to the Food and Drug Administration (FDA) for approval. The FDA reviews the data and decides whether to approve the drug or treatment for use on patients.

> WHAT HAPPENS WHEN A PERSON SIGNS UP FOR A CLINICAL TRIAL?

First it is important to learn about the study. Study staff explain the trial in detail to potential research participants and describe possible risks and benefits. Staff also talk about the participants' rights as research volunteers, including their right to leave the study at any time. Participants and their family members are entitled to have this information repeated and explained until they feel they understand the nature of the study and any potential risks.

Once all questions are answered and there is still interest in being a part of the study, a patient participant is asked to sign an informed consent form. Laws and regulations regarding the informed consent form differ across States and institutions, but all are intended to ensure that patient participants are protected and well cared for.

In some cases, a patient participant may not be able to provide informed consent because of problems with memory and confusion. In such cases, it is still possible for an authorized representative (usually a family member) to give permission for the patient to participate. Even so, it is still important that patients assent to be in the study, even if they can no longer formally consent to it. Different States have different laws about who is a legal representative. These laws are in a state of flux as researchers and the public grapple with the ethical issues of proxy consent.

Next, patients go through a screening process to see if they qualify to participate in the study. If they qualify and can safely participate, they can proceed with the other parts of the study.

> WHAT HAPPENS DURING THE TRIAL?

If the screening process shows they're a good match, participants have a "baseline" visit with the study staff. This visit generally involves a full physical exam and extensive cognitive and physical tests. This gives the study team information against which to measure future mental and physical changes. Participants also receive the test drug or treatment. As the study progresses, participating patients and family members must usually follow strict medication or treatment instructions and keep detailed records of symptoms. Every so often, participants visit the clinic or research center to have physical and cognitive exams, give blood and urine samples, and talk with study staff. These visits allow the investigators to assess the effects of the test drug or treatment, see how the disease is progressing, and see how the participant and the caregiver are doing.

In most clinical trials, participants are randomly assigned to a study group. One group, the test group, receives the experimental drug. Other groups may receive a different drug or a placebo (an inactive substance that looks like the study drug). Having the different groups is important because only by comparing them can researchers be confident that changes in the test group are the result of the experimental treatment and not some other factor. In many trials, no one - not even the study team - knows who is getting the experimental drug and who is getting the placebo or other drug. This is called "masking" meaning that the patient/family member and the staff are "blind" to the treatment being received.

> WHAT SHOULD PEOPLE CONSIDER BEFORE PARTICIPATING IN A CLINICAL TRIAL?

Clinical trials generally don't have miraculous results. The test drug or treatment may relieve a symptom, change a clinical measurement, or reduce the risk of death. With a complex disease like AD, it is unlikely that one drug will cure or prevent the disease. Some people choose not to participate or drop out of a study because this reality does not meet their expectations. Others participate because they realize that even if the benefit to them may be slight, they are making a valuable contribution to knowledge that will help future patients.

Some clinical trials want participants who are cognitively healthy or have only mild symptoms because they are testing a drug that might delay the decline in cognitive function. Other trials are interested in working with persons who have more advanced AD because they are testing a drug that might lessen behavioral symptoms, or they are testing new strategies to help caregivers. Even though a participant may not be eligible for one trial, another trial may be just right.

Many families find that the biggest benefit of participating in a clinical trial is the regular contact with the study team. These visits provide an opportunity to get state-of-the-art AD care and also to talk on an ongoing basis with experts in AD who have lots of practical experience and a broad perspective on the disease. The study team understands and can provide advice on the emotional and physical aspects of the person with AD and the caregivers' experience. They can suggest ways to cope with the present and give insights into what to expect in the future. They also can share information about support groups and other helpful resources.

For a list of clinical trials on AD and dementia currently in progress at centers throughout the US, go the ADEAR Clinical Trials Database.

You may also wish to visit the Alzheimer's Association website at www.alz.org.

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