Division of Infectious Diseases: HIV Clinical Trials Unit

What is the Goal of HIV Treatment?
The goal of anti-HIV treatment is to reduce the amount of HIV in the body as much as possible for as long as possible. Viral load tests are also used to measure how well anti-HIV treatments are lowering the amount of HIV in the body. Treatment should also increase your T4 cell count, and may help with any symptoms of HIV disease.

About T4 cells and Viral Load
HIV causes illness by damaging the immune system. Measuring your T4 cell count gives an idea of how the immune system is doing, and can also help you decide if and when HIV treatment is needed. The T4 is also called the CD4. The other important test is called the viral load test. The viral load test measures the amount of HIV in a blood sample. Measuring both the T4 cell count and the viral load can help you decide if you should start treatment.

What is a Clinical Trial?
A clinical trial is a carefully controlled experiment in which people take a drug to find out if it works, and if it is safe. In a clinical trial, one or more different drugs or combinations of drugs are compared to each other. In the past, when there weren't many treatments for HIV, a clinical trial often gave one group of people an experimental drug, while the other group got nothing (or a dummy pill called a placebo). Now, however, many clinical trials are looking at different combinations of approved and experimental drugs. Approved drugs are often used in clinical trials.

What Treatments are being studied?
Some clinical trials are trying to find out if taking an experimental drug along with an approved HIV treatment combination is safe and effective. Other trials compare one experimental drug to another. There are trials for people who have already tried many different drug combinations. There are also clinical trials that are looking at ways to prevent and treat opportunistic infections, cancers and other conditions.

Why are clinical trials done?
Drug trials are done to find out which treatments work, and which ones don't. If drugs are sold without being tested, the only information about the drug will come from unproven stories, or from the company that makes the drug. Drugs might be advertised and sold without proof that they work or are safe. Now that there are approved drugs for HIV, a clinical trial might be done to find out if there are easier ways to take the drug, or to find treatments for side effects of approved drugs.

How do Clinical Trials Work?
If a drug company wants to study a treatment, they must submit a proposal to The Food and Drug Administration (FDA). A clinical trial must follow well-established guidelines for testing drugs in human beings. Once the FDA approves the plan for the trial, which is called a protocol, the trial can start. Every drug sold in the US must be approved by The FDA. The FDA reviews the results of clinical trials to determine if the drug is safe and effective.

About Clinical Trials
After a drug has been tested in the lab and in animals, it is tested in people. You might not know exactly what drugs you're getting in a clinical trial. It depends on what treatments are already approved for your condition, and how much is already known about the treatments being tested in the clinical trial.

Is the Treatment Safe?
There are three types, or phases, of trials that are done with people, although sometimes phases are combined. In a Phase I trial everyone usually gets some amount of a drug, but since researchers need to know how the body reacts to the treatment, and how much should be given, people are often given different amounts, or doses. Phase I trials are short, usually less than 2 or 3 months long, and usually involve under a hundred people. Because a treatment is being tested for the first time ever in people, this type of clinical trial is usually riskier than the other phases.

Why would I want to be in a trial?
Deciding to join a trial can be difficult. There are always risks involved. Carefully weigh the risks and benefits of any trial you are thinking about joining. Discuss the trial with people who you trust to give you advice about what would be best for you and your health. Speak with your doctor and other health care providers, and with people who have been involved in clinical trials themselves. Find out if there are other at other places, or trials of other drugs that might be better for your situation.

Reasons to join a trial:
-There may be no drug approved to treat your condition, and you might be given a drug that you could not get anywhere else.
-You may not be able to take the drugs already approved for your condition.
-You are interested in helping with research that will lead to finding better treatments for AIDS.
-You want to be closely watched for the possible side effects or unexpected reactions of a treatment.
-You want to get a new drug that turns out to be a more effective or easier to take treatment.
-You might get a treatment for the side effects of another treatment you are already taking.

Reasons not to join a trial:
-You may have to stop taking other medications that may be helping you.
-Your condition may get worse.
-The treatments in the trial may have side effects, or be unsafe.
-If other treatments are approved they may not work as well, or at all, because of the treatments you took in a clinical trial.
-You don't have the energy, time or interest to participate in the trial.

Do I need my own doctor?
Yes. Joining a clinical trial is not the same as having your own doctor. Clinical trials are not designed to provide people with treatment, so it's important that you have a regular doctor or clinic for regular checkups and lab tests while you are in the trial.

How are the drugs given?
A drug may be given in one of several ways. In some trials, different drugs are given in different ways. Here are some of the ways a drug might be given:

Oral - A pill or liquid that you swallow by mouth
Intramuscular - injected into a muscle
Intravenous - injected into a vein
Subcutaneous - injected under the skin
Inhaled - a spray that you breathe in
Topical - a cream or patch you place on your skin
Sometimes a treatment involves a procedure, such as being exposed to light from a machine.

When do I take the treatments?
In some trials you will take the treatments at home. In other trials, you will have to stay overnight or for longer while you are in the trial. You will be told exactly when and how to take the treatments in a trial. You should get a written description of what each drug looks like, and how often you are supposed to take it. Ask the study nurse to go over your daily schedule with you, so you remember how many of each pills you are supposed to take, and what you can or can't eat. If you think you may forget how you are supposed to take something make sure the study nurse knows this.

How often do I go to the site?
You may have to visit the site as little as once a month or as often as five times a week. At first, there may be many medical checkups to see what the drug is doing to you. Later in the trial there will usually be fewer checkups. Ask for a schedule.

What if I'm Pregnant?
For pregnant women with HIV, decisions about the best anti-HIV treatment to use should be based on the health of the mother. Because the chances of anti-HIV drugs harming the fetus are greatest during the first trimester of pregnancy, it may be a good idea to wait until after the first trimester before starting anti-HIV treatment, if this is possible.

For a woman who's already on combination anti-HIV treatment but who has a low risk of disease progression, consideration could be given to stopping anti-HIV treatment for the first trimester of pregnancy. If treatment is stopped, all anti-HIV drugs should be stopped at once to lower the chance of drug resistance. There is little information about the safety of any anti-HIV drugs in pregnancy apart from AZT. AZT has been shown to reduce the chances of transmitting HIV from mother to baby by about two-thirds.

What if I get sick while I'm in the trial?
If your health gets worse while you're in the trial, the people running the trial will try to find out if the drug is making you sick or if you're sick for some other reason. Keep your doctor informed about everything you are experiencing.

Some drugs have side effects, like headaches or stomachaches. Some drugs can lead to serious illness or death. If you get sick because of the drug, tell the people running the trial. You may either be taken off the drug or be given a different amount. If the trial is comparing two drugs, you may be offered the other drug. Discuss your options with your doctor.

If you get sick, but not because of the drug, you may have to leave the trial. It's important to get the phone number of a doctor or nurse involved with the trial who you can call 24 hours a day, in case you get sick in the middle of the night. Because the drugs in trials are experimental, a doctor in an emergency room may not know what to do if the drug makes you sick. This is another reason why your doctor should always know everything that is going on in a clinical trial. Ask both your doctor and the study nurse how you can be prepared if this happens.

What is Informed Consent?
At the first visit you will be asked to give your informed consent to join the trial. Informed Consent means that after being informed of all the possible risks and benefits of the trial, you consent (agree) to join. The Informed Consent Form explains the rules of the trial in plain terms.
Be sure that you understand and agree with everything about the trial before you sign this form. Keep a copy for yourself. Even if you sign, you can still leave the trial whenever you want and for any reason. If a child is joining a trial, the parent or guardian will be asked to sign the form, stating that all the risks and benefits for the child are understood. Joining a clinical trial means that you agree to follow the rules of the trial. The rules of the trial are called a protocol, which explains exactly how the trial will be run. If you don't feel comfortable with the rules - like not being able to make all the appointments, or going to a clinic where the waiting room is full of sick people - talk to the people running the trial.

How do I leave the trial?
You can choose to leave the trial at any time, and this should not affect the quality of care you get at the hospital or clinic in the future. If you get sick because of the drug and are asked to leave the trial, the people running the trial should make sure your medical needs are taken care of, but they might not pay for it.