• The first use of a new drug, treatment or screening procedure in humans.

• Used to test for safety and side effects and to determine how the drug should be given (by mouth or by injection into blood or muscle).

• Small numbers (20-80) of participants.

Phase II

•  Used to investigate the ideal dosing range of a new drug.

• The therapy is tested in a larger number of patients (50-300) to determine how well it works and its possible side effects.

Phase III

• Efficacy of treatment with a new drug is compared to that with common or standard therapies.

• Patients who participate in Phase III trials are randomized to receive the trial drug and/or standard therapy.

• A much larger group of patients is involved in Phase III studies (100-3,000+).

Phase IV

• Conducted to continue to evaluate for the safety and efficacy of cancer therapies that are FDA approved and available for general use.

• Not all clinical trials have Phase IV studies.

Currently, some Phase II and most Phase III clinical trials are designed as randomized, double-blind and placebo-controlled studies:

▪    Randomized: Each study subject is randomly assigned to receive either the study treatment or a placebo.*

▪    Blind and double-blind: The subjects involved in the study do not know which study treatment they receive. If the study is double-blind, the researchers also do not know which treatment is being given to any given subject. This ‘blinding’ is to prevent biases, since if a physician knew which patient was getting the study treatment and which patient was getting a placebo, he/she might be tempted to give the (presumably helpful) study drug to a patient who could more easily benefit from it. In addition, a physician might give extra care to only the patients who receive the placebos to compensate for their ineffectiveness. A form of double-blind study called a “double-dummy” design allows additional insurance against bias or placebo effect. In this kind of study, all patients are given both placebo and active doses in alternating periods of time during the study.*

Placebo-controlled: The use of a placebo (fake treatment) allows the researchers to isolate the effects of the clinical trial treatment.*

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* From http://en-wikipedia.org/wiki/clinical_trial# type.

Regulation of Clinical Trials

Clinical trials are regulated by a number of groups and processes to help protect patient safety and the ethicality of scientific research. Requirements of these regulatory groups and processes include:

1. Full disclosure: The pros and cons of a clinical trial must be explained to each patient considering enrolling in it.
2. Informed Consent:
   1. The Informed Consent form is designed to fully inform the patient and to facilitate communication between the patient and the research team. After talking with family, friends and doctors, it is up to the patient to decide whether to participate in the clinical trial.

1. If the patient decides to participate, she/he is asked to sign the Informed Consent form, once all the risks of the particular trial are fully understood.

1. c.     Participants can participate in clinical trials only after signing the informed consent, and they have the right to voluntarily withdraw from a clinical trial at any time, and for any reason.

1. Periodically, the research team will go over the form with the patient again to make sure the individual still wants to continue her/his participation. The patient’s health is continually monitored during the course of the treatment in the clinical trial.

Questions to Ask When Considering a Clinical Trial

1. What is the purpose of the trial?

1. Which type of research phase (I, II, III or IV) is the study?

1. What are the goals and objectives of the study–that is, why is the study being conducted?

1. Who is likely to benefit from the knowledge of the results? Also, how will the results be distributed or shared with participants and the public?

1. Who is sponsoring the trial?

1. To evaluate any conflicts of interests and who pays the study staff.

1. To determine possible benefits of the trial–for example, access to free medication?

1. What will be required of you?

1. Time burden, that is, duration of trial and frequency of visits.

1. Location, for consideration of transportation of site for trial.

1. Inpatient or outpatient treatment.

1. Personal costs to you.

1. Expectations (protocol)–for example, keep logs, collect specimens, schedule of medication).

1. Reimbursements or incentives for you.

1. What are the risks to you?

1. Invasiveness of procedures.

1. Known side effects.

1. Signs and symptoms of adverse effects (AEs).

1. Who to call and what to do if experience AEs.

Before you talk to the doctors and researchers for the first time, be sure to have a list of these and other questions that you want to ask them. As the patient, it is your right to ask any question that you have. It is the doctor’s and researcher’s job to answer your questions.