Purpose of this page.

Someone with an illness that does not have a totally successful treatment may find help in a clinical trial. The purpose of this page is to provide an introduction to clinical trials and resources for people to use if they wish to participate in a clinical trial.

It is sometimes difficult to find and recruit suitable participants for clinical trials. To overcome this difficulty, MIU is maintaining a registry of persons who wish to participate in clinical trials. This information will be made available to bona-fide medical researchers as they need recruits.

How to be placed on the MIU register.

Fill in the MIU Clinical Trials Registry Form.

If you are a medical researcher.

MIU will make this database available to you if you are implementing a clinical trial. Please e-mail us at trials@medinfo4u.com. or phone us at 1-541-267-2205.

Where to get information about specific clinical trials.

MIU can perform a search for a specific clinical trial.for a specific disease. If you would like such a search, please go to the Medical Informaion Search page.

CenterWatch Clinical Trials Listing ServiceTM (www.centerwatch.com) is an excellent information resource. Of special interest to health consumers are:

1. An international listing of ongoing clinical trials by disease and geographic area.
2. A patient notification service for persons who wish to keep abreast of new trials dealing with a particular disease.
3. Profiles of research centers conducting trials.
4. Information about drugs newly approved by the FDA.

Definition of clinical trials.

Clinical trials are scientific studies of the effects of a new treatment or medication in humans. Clinical trials are often the most effective way to determine the efficacy and safety of the proposed therapeutic. Before use in a clinical study, a new treatment is subjected to many laboratory tests. If the results are favorable, the treatment may be tested in people.

In addition to testing new treatments, clinical trials may test new combinations of existent therapies, new ways of administering conventional therapeutics, or how lifestyle changes may improve or prevent disease. Clinical trials may also focus on methods of detection and diagnosis of disease, improvement of patient comfort and quality of life, and the psychological impact of disease.

In the USA, a clinical trial begins only after the Food and Drug Administration (FDA) determines that the treatment is safe for humans. The FDA has strict guidelines for how proposed trials are evaluated. All legal and ethical medical codes that regulate medical practice apply equally to clinical trials. A chief tenant is that the potential benefits will always outweigh the risks, and no harm will knowingly be done to any patient. Additionally, an Institutional Review Board (IRB) at the participating site must certify that the study is well designed and the safety of the participants are protected. The actual clinical trials proceed in phases (with the exception of supportive and preventive types, q.i.), progressing from a small to a large group of people. Researchers eventually compare the results of different treatments in hundreds of patients. Results are used to guide future treatment decisions.

Patients may be eligible for different phases of a trial depending upon their overall health, the type and stage of illness and the nature of treatment that they have already received.

Why you might choose to join a clinical trial.

1. If you are ill, and there is no satisfactory treatment available, the treatment tested in the clinical trial may help you.
2. Participating may give you the satisfaction of helping speed scientific investigation that may help to treat future patients.
3. Patients who choose to participate in clinical trials may receive promising new therapeutics before they are generally available to others.
4. This may be the only way that you can get a promising new treatment.
5. Treatments received in a clinical trial are usually free.
6. Some studies give you free examinations, laboratory tests, medications; and in general, they may make you more healthy by curing or preventing disease.
7. Some studies pay you to participate. This is usual only when the study requires an exceptional amount of commitment from the volunteer, and no medical benefit is likely to accrue to the participant.

The risks of clinical trials.

1. Harmful side effects pose the biggest risks. When the medical researchers designed the study, they planned on contingencies should a participant have ill effects from the therapeutic. If you are taking a medication as part of the test, you might be switched to a lower dose, a different medication, or be asked to stop taking the medication.
2. Although every effort is made to ensure the safety of participants, it can never be guaranteed that every clinical trial is absolutely safe.

Definition of a placebo.

A placebo used in a clinical trial is a substance inert in relationship to a desired effect but otherwise identical in appearance to the material being studied. In a double blind study, the researchers and the volunteers do not know who receives the placebo. In a single blind study, only the researcher knows. Usually, a placebo-controlled study is used only when there is no known effective treatment. Commonly, all volunteers in the study receive the active drug if it has been shown to be safe and effective. In all events, the control groups are monitored as carefully as the groups receiving the new therapeutic. For more information, please see Behavior and Immune Regulation:Psychoneuroimmunology.

Rights of the participants.

1. Before you consent to join the study you have the right to have all your questions answered about the study. You should know all the potential benefits and risks. Legally, before you sign up for a study you must have all the facts about the study explained to you. When you feel that you understand the facts, you will be asked to sign an informed consent form stating that you do understand and are joining of your own free will. This form is not a contract.
2. Even though you have signed the consent form of your own volition, you may quit at any time.
3. You may say no to any test, procedure, or medication.
4. You can not be refused any other treatment that might benefit you if you decide not to participate in the study.
5. Your medical records will be confidential.
6. You may know the names of anyone with whom you speak.

Important questions to have answered.

1. What are the goals of the trial?

2. What exactly will be done to the participants?

• What procedures will be performed?
• What are the known risks?
• What are the suspected risks?

3. What exactly is required of the participants?

• How long will the study last?
• Will I have to pay anything? If so, how much will it cost, and where can I get information on ways to allay expenses?
• Will I have to be admitted to a hospital? If so, how frequently and for how long?

4. What are my options?

• Are there any standard treatments for my disease?
• If there are standard treatments, what is likely to happen to me with those treatments? What are the advantages and disadvantages?
• If there are no standard treatments, what is likely to happen to me if I don’t sign up for this experimental treatment?
• What will likely happen to me if I take this experimental treatment? What are the advantages and disadvantages?

5. If I need to drop out of the study because of adverse effects, what kind of follow-up treatmentwill I receive?

6. After the trials are over, are there any provisions for long-term follow up?

Responsibilities of the participants.

1. You must be honest with the researchers.
2. You must follow the instructions and take all medications or treatments exactly as they are prescribed.
3. You must accurately report any changes in your well being.
4. You must make every effort to keep scheduled appointments.

The phases of clinical trials.

Clinical trials in the USA are designed to be divided into four phases. If a treatment demonstrates outstanding results, a clinical trial may eliminate phase III and go directly to phase IV. Also, a trial may be called off in any phase if the safety or value is brought into serious question. The results of each phase are used to guide the administration of each successive phase. The FDA carefully reviews all the data for each phase. It may take between 1 and 10 years for a new therapeutic to be approved by the FDA for standard treatment. The phases for a typical clinical trial of a new treatment are as follows.

Phase I:

• The highest risk because this is the first time the treatment is used in humans.
• Usually the patients chosen for this phase are those most severely afflicted with the disease that the treatment is attempting to ameliorate. And, these patients are not likely to be helped by any other therapy.
• Few people enrolled.
• If a drug is being tested, dosage and route of administration are determined. The regimen is begun with a very low level of the drug and gradually increased as more patients are admitted to the study.
• Side effects are scrutinized.
• Once the best dose of medication or optimal treatment relative to side effects has been identified, the trial may go to phase II.

Phase II:

• Risk is less.
• Patients other than the highest risk are admitted to the study.
• More people are enrolled.
• Because more people are enrolled, there is more chance to observe uncommon side effects.
• Careful monitoring determines if the therapy is actually working.
• If a treatment shows efficacy, the trial then moves into phase III.

Phase III:

• During this phase, the treatment used in phase I and II is compared to other treatments.
• A large number of people are enrolled.
• Participants are randomly grouped as either the control group or the treatment group.
• The control group gets the conventional treatments.
• If there is no other existing treatments, the control group gets a placebo.
• If there are compelling reasons to believe the new therapeutic is superior to the conventional treatment, or clearly better than no treatment at all if no conventional treatment exists, the FDA allows this phase to be skipped.

Phase IV:

• To get to phase IV, the therapeutic must have proven its efficacy, but toxicity and utility are continued to be monitored.
• The new treatment may be designated a standard treatment option unless complications are revealed.
• The new treatment may be provisionally approved as a standard treatment if no other treatment is available.

Less common types of clinical trials.

The most common use of a clinical trial is to test a new treatment. However, there are other types of clinical trials: prevention studies, early detection studies, adjuvant studies, neoadjuvant studies, and supportive care studies.

Prevention Studies:

1. People at high risk of developing a particular disease (e.g., breast cancer in women with a significant family history) are studied.
2. Usually, one group will receive no special treatment, and the other will be given a medication or a diet in an attempt to prevent the disease from occurring.
3. The populations are surveyed for many years to determine what is the relative incidence of disease in the two groups.

Early Detection Studies:

1. These studies investigate methods for disease screening. If the disease can be discovered at a sufficiently early stage, treatment may be more effective and morbidity may be less.
2. Screening may involve testing blood or other body fluids, the use of imaging techniques such as x-rays or ultrasound, or other non-invasive techniques.
3. Studies may or may not last for many years and don’t have to be phased.

Adjuvant Studies:

1. These studies investigate the effect of an adjuvant on the return of cancer in a post- surgical patient. An adjuvant is a treatment added to a standard treatment to improve the chances for a complete cure. It is usually given after the surgery.
2. One group might receive surgery for cancer and the other group would receive surgery plus a chemotherapeutic.
3. These studies progress through the typical phases.

Neoadjuvant Studies:

1. These studies investigate the effects of an adjuvant given before standard therapy.
2. An example is giving chemotherapy to shrink a large tumor that can not be safely removed with surgery. The mass may then be safely removed.
3. These studies progress through the typical phases.

Supportive care studies:

1. These studies try to determine good ways of coping with side effects caused by treatment or symptoms of the disease itself. These new treatments may also be psychological, e.g., support groups for the patient or the family.
2. Studies that test pharmaceutical treatments will be phased studies. Studies that investigate psychological interventions will not be phased.

Group C and treatment center studies.

After therapeutics have passed through clinical trials but are awaiting FDA approval for sale, they may be available from some physicians. Patients who receive these drugs are regularly monitored for adverse effects just as if they were in a clinical trial. There is a gradation on the availability of chemotherapeutics based upon the number of doctors that are allowed to prescribe them. From least to most available they are Treatment Referral Center Studies, Modified Group C and Group C.

Copyright© 1996-2002 by Medical Information Unlimited, Inc. All rights reserved.
Revised: May 28, 1999.