Clinical trials are research studies in which patients may volunteer to take part. Phoenix Children’s uses clinical trials to find better ways to prevent, diagnose and treat childhood diseases and injuries. Doctors use treatment trials to learn more about how to fight cancer, cardiovascular, endocrine and neurological disease. This guide is for patients who may join a treatment trial.

Clinical trials are part of a long, careful process, which may take many years. First, doctors study a new treatment in the lab. Then they often study the treatment in animals. If a new treatment shows promise, doctors then test the treatment in people. Doctors do this in three to four steps, or phases described below, and may offer patients a clinical trial as a treatment option.

Phases of Clinical Trials

Most clinical research involves the testing of a new drug in steps, called phases. This allows researchers to ask and answer questions in a way that results in reliable information about the drug and protects the participants. Clinical trials are usually classified into the following phases:

  • Phase I trials are the first step in testing a new therapy in humans. In these studies, researchers evaluate how a new drug should be given (by mouth, injected into the blood or injected into the muscle), how often and what dose is safe. A Phase I trial usually enrolls only a small number of participants, sometimes as few as a dozen.
  • Phase II trials focus on evaluating how well the new drug works for the disease or condition being studied and continues to test its safety.
  • Phase III trials compare the results of people taking the new therapy with results of people taking standard or care treatment. A participant will usually be assigned to the standard treatment group or the new clinical trial therapy group at random (called randomization). Phase III trials often enroll large numbers of participants and may be conducted at many doctors' offices, clinics and research institutions nationwide.
  • Phase IV trials examine the treatment after it has been approved and is being marketed. Phase IV trials evaluate the side effects, risks and benefits of a drug over a longer period of time and in a larger number of participants than in phase III clinical trials. Thousands of people are involved in a phase IV trial.

Types of Clinical Trials

  • Treatment trials test new therapies (like new drugs, new approaches to surgery or medical devices).
  • Prevention trials test new approaches, such as medicines, vitamins, minerals or other supplements that doctors believe may lower the risk of certain medical conditions. These trials look for the best way to prevent medical conditions for patients who have never had them. They also look at how to prevent medical conditions from coming back or a new condition.
  • Screening trials test the best way to find certain medical conditions such as cancer, especially in its early stages.
  • Quality of life trials explore ways to improve comfort and quality of life for patients.
  • Diagnostic trials are conducted to find better tests or procedures for diagnosing a particular medical condition.

How We Protect Patients

Our most important job is to protect patients.

First, Phoenix Children's protects patients in clinical trials by following well-planned protocols. A protocol:

  • Explains the treatment plan
  • Lists the medical tests patients will receive
  • Gives the number of how many patients will take part in the clinical trial
  • Lists eligibility criteria, which are guidelines to decide who may join the clinical trial
  • Explains safety information

Second, Phoenix Children's protects patients by using a careful informed consent process.

Third, our Institutional Review Boards (IRBs) protect patients by reviewing protocols and monitoring trials. The IRBs are committees of doctors, nurses, chaplains, social workers, lawyers and community members. They make sure that trials follow federal laws and that patients are protected.
The U.S. Food and Drug Administration (FDA) audits the IRBs’ files. Also, FDA officials may visit Phoenix Children's at any time and review anything they choose related to clinical trials.

Frequently Asked Questions

Patients choose to participate in a clinical trial for many reasons:

  • Patients are given access to the latest available treatments and/or a new drug, procedure or device which is being studied. There is no guarantee that these new treatments will be effective. However, many clinical trial participants decide that the potential benefits of the new treatment outweigh the risks.
  • Clinical trials are the most important tool that doctor-researchers have in their effort to find new and more effective ways to treat and possibly cure various medical conditions. Many of today’s medical treatments began in clinical trials.
  • Many patients feel that participating in a clinical trial can be empowering because it allows them to take an active role in their treatment options. They also know their participation in a trial might be helping to find a new treatment or cure and can benefit future generations.

Taking part in a clinical trial is voluntary. However, all clinical trials have guidelines about who can and cannot participate. Before enrolling in a clinical trial, a patient must qualify for the study. Some research studies seek participants with illnesses or conditions to be studied in the clinical trial, while others need healthy participants.

The factors that allow a patient to participate in a clinical trial are called "inclusion criteria" and those that do not allow a patient to participate are called "exclusion criteria." Meeting inclusion and exclusion criteria is an important principle of clinical research that helps to produce reliable results. These criteria are based on factors such as: 

Overall health:

  • Age and gender
  • Results of medical tests
  • Medicines a patient is taking
  • Any other health problems

 Medical condition:

  • Condition type and stage (if applicable)
  • Other treatments a patient may have had
  • How long it has been since a patient was last treated

It is important to note that inclusion and exclusion criteria are not used to reject people personally. Instead, the criteria are used to identify appropriate participants and keep them safe. These criteria help ensure that researchers will be able to answer the questions they plan to study, while ensuring that all measures to maintain efficacy, safety and equity exist.

Clinical trials that are well-designed and well-executed are the best approach for eligible participants to:

  • Play an active role in their own health care decision process along with their provider
  • Gain access to new research therapies before they are widely available
  • Obtain expert medical care at leading health care facilities during the trial
  • Help others by contributing to medical research

The risks and side effects to each research treatment are outlined for each trial and can vary from trial to trial.

  • A participant's doctor and study coordinator will review all the risks that are specific to the trial.
  • There may be unpleasant, serious or even life-threatening side effects to experimental therapy, such as headaches, nausea, hair loss, skin irritation or other physical problems the doctor does not expect or desire.
  • In clinical trials, the research treatment may not be effective for the participant.
  • The clinical trial may require more time and attention than standard care treatment, including trips to Phoenix Children's, more hospital stays or complex dosage requirements.

Patients should know as much as possible about the clinical trial and feel comfortable asking the members of the health care team questions about it, the care expected while in a trial, and the cost of the trial. It is recommended to:

  • Plan ahead and write down possible questions to ask.
  • Ask a friend or relative to come along for support and to hear the responses to the questions.
  • Bring a tape recorder or cell phone to record the discussion to replay later.

The following questions might be helpful for the patient to discuss with the health care team. Some of the answers to these questions are found in the informed consent document.

  • What is the purpose of the study?
  • Who is going to be in the study?
  • Why do researchers believe the experimental therapy being tested may be effective? Has it been tested before?
  • What kinds of tests and experimental therapy(ies) are involved?
  • How do the possible risks, side effects, and benefits in the study compare with my current treatment?
  • How might this clinical trial affect my daily life?
  • How long will the clinical trial last?
  • Will hospitalization be required?
  • Who will pay for the experimental drug(s) or device?
  • Will I be reimbursed for other expenses?
  • What type of long-term follow-up care is required for this study?
  • How will I know that the experimental therapy is working? Will results of the trials be provided to me?
  • Who will be in charge of my care?

To help a patient decide whether or not to participate, the doctors and nurses involved in the trial explain the details of the study. If the patient's native language is not English, translation assistance will be provided.

The research team provides a consent document that includes details about the study, such as its purpose, how long it will last, required procedures and key contacts. Risks and potential benefits are explained in the consent document.

The patient then decides whether or not to sign the document. Participation in any trial is completely voluntary. Informed consent is not a contract, and the patient may withdraw from the trial at any time.

 

 

Patients should look for the following information on their informed consent form:

Treatment

  • The reason for the clinical trial (what the doctors hope to learn)
  • Who is eligible to take part in the clinical trial
  • What is known about the type of treatment being studied
  • Possible risks and benefits (based on what is known so far)
  • Other treatments that may be options

Tests

  • Types of tests
  • How often tests and meetings with the doctor are needed

Costs

  • Who pays for the costs of the clinical trial
  • If the clinical trial causes the need for more medical care, who pays for those costs

Other

  • A statement about conflicts of interest (any direct financial benefit to Phoenix Children’s or the doctor from the sponsor of the trial)
  • A statement about the participant's privacy is protected
  • Who to call if there are any questions

The clinical trial team includes doctors, nurses and other health care professionals. They check the health of participants during the trial, give specific instructions for participating in the trial and guide them through the process. They monitor the participants carefully during the trial and stay in touch after the trial is completed.

Some clinical trials involve more tests and doctor visits than the participant would normally have for an illness or condition. For all types of trials, the participant works closely with the research team. Clinical trial participation is most successful when the study guidelines are carefully followed and there is frequent contact with the research staff.

If the doctor-researcher determines that a therapy is harmful to a participant, they will be taken off the study immediately. The participant may then receive other treatments from their own doctor.

A participant's primary care provider will still care for them. In a clinical trial, participants will also have a:

  • Principal Investigator (PI): The PI is usually a doctor who runs the clinical trial and makes sure that the health care team follows the plan.
  • Research Nurse: The research nurse teaches patients about the trial and collects data from patients on the trial. The research nurse is a good contact for questions during a clinical trial.

Most clinical trials provide short-term therapies related to a designated illness or condition, but do not provide extended or complete primary health care. By having the participant's primary care provider work with the research team, they can ensure that other medications or treatments will not conflict with the protocol.

Patients would continue to see their primary care provider for treatment and follow-up care after the clinical trial.

A patient’s insurance will usually cover all standard of care treatments such as exams, laboratory work, X-ray, etc. (standard of care is the routine care provided for a disease). All additional clinical trial related therapies or laboratory studies may be paid for by the sponsor of the clinical trial, such as a pharmaceutical company. Patients should ask their doctor what is covered by their insurance to ensure that their out-of-pocket costs have been explained fully.

Clinical trials are sponsored or funded by a variety of organizations or individuals such as physicians, medical institutions, foundations, voluntary groups, and pharmaceutical companies, in addition to federal agencies such as the National Institutes of Health (NIH), the Department of Defense (DOD) and the Department of Veterans Affairs (VA). Trials can take place in a variety of locations, such as hospitals, universities, doctors' offices or community clinics.

Yes, a participant can leave a clinical trial at any time. When withdrawing from the clinical trial, the participant should let the research team know about it, and the reasons for leaving the study.