Clinical Trials

What is a clinical trial?

A clinical trial is a research study that tests the current, most promising treatments for diseases. The goal of a clinical trial is to identify treatments that will provide the highest success rates or the best chance of a cure with the least amount of side effects. Find our about registering for a clinical trial.

  • Asthma Clinical Trial

    Is your child coughing and wheezing? Learn more about a research study testing how antibiotics and steroid therapy affect asthma.


How are clinical trials developed and approved?

A new trial is developed by a team of doctors, nurses and others with expertise in treating a particular disease. Some trials may be organized and conducted at the local or institutional level while other trials may be created and administered nationally. The clinical trial team creates a new plan for how children should be treated for a particular disease, such as a congenital heart defect. The team uses information from past clinical trials to develop a new plan that will hopefully further improve a child’s chance of survival.

How does a child benefit from being on a clinical trial?

We want to provide each child with the best possible medical care. New clinical trials build upon the results of past trials, past treatments and past information learned about a disease. A clinical trial may allow us to provide more advanced care. Any decision to place a child on a clinical trial will be made by the parent and child’s doctor, based on the child’s unique needs.

When the child is treated on a clinical trial, he receives the most up-to-date treatment available. Studies have shown that patients who participate on clinical trials do better than those children who are not treated on a trial.

The child is closely followed by doctors, nurses and researchers who look for:

  • How well the treatment works
  • How it affects healthy cells in the body over time
  • How other children in the future might benefit from the treatment—just as the child has been helped from clinical trials of the past. In some instances, a trial may not result in a direct benefit to the child but may provide some information that will aid in the treatment of future children with the same disease

Are clinical trials voluntary?

Yes, the patient and family may choose not to enter a clinical trial. The family may also choose for the child to be removed from a trial at any time. However, it is important to consider whether the child should begin a clinical trial initially. A study can be seriously damaged by patients who change their mind in the middle of treatment and decide to be removed from the trial. The decision on whether to participate in a study will not affect how the healthcare team feels about the child. The child will still receive the same quality of care.

Who is eligible for a clinical trial?

Every clinical trial has a specific set of criteria that patients must meet before they can participate in a study. Parents should consult with their child’s doctor to determine if their child is eligible for an active clinical trial. The decision will be based upon a number of factors, including the type and stage of the disease, pre-existing conditions affecting the child and potential side effects of the treatment.

The child’s doctor will also help determine if the clinical trial is the best decision to meet the child’s unique medical care needs.

Is a second opinion on the recommended treatment important?

Some families may want to get a second opinion before starting treatment. We fully support patients and their families getting all of the information necessary to be comfortable with the treatment plan. We are happy to help coordinate second opinions and can recommend other experts around the country who would be the most helpful to patients and families. We can also provide second opinions to patients receiving care at other children’s cancer hospitals.

Parents may want to have a doctor who is not a part of their child’s current healthcare team look at their child’s history, lab findings and exams. The doctor can then review his opinion about the treatment plan with patient families. In addition, some insurance companies require a second opinion. The child’s primary care doctor may suggest another expert doctor for parents to call.

What are the phases of a clinical trial?

Clinical trials are developed in a series of steps, called phases. Clinical trials have three phases:

  • Phase I trials are usually intended for patients with disease that has returned or failed to respond and for which there is no other known effective treatment. Phase I trials seek to determine the maximum dose of a drug that can be safely tolerated by patients and how the body breaks down a new drug. Clinical response is not the main focus of a phase I trial and less than 10 percent of patients are expected to respond in a Phase I trial. Phase I trials usually involve between 10 and 30 patients who are placed in groups of three and are closely monitored for side effects. If your child is part of a Phase I trial, he probably has not responded to standard treatments. The benefit is that the child may be among the first to receive a new treatment.
  • Phase II trials are typically offered to patients with disease that has returned, failed to respond or that has a very poor response to initial treatment. Phase II trials usually seek to determine how effective the optimal dose of a new drug is against a variety of tumors. These trials usually involve between 30 and 100 patients. Doctors can learn more about the effectiveness, side effects and safety of the particular treatment.
  • Phase III trials are typically the initial trials offered to newly diagnosed patients. They usually involve a relatively large number of patients and are most commonly conducted at many different hospitals and clinics around the country. In this phase, the new treatment is often compared to a standard treatment. The child may be randomly assigned to either the new treatment or current standard treatment. Doctors monitor to see if the new treatment is better than standard treatment. The patient and family will know which type of treatment the child is given.