Types of Clinical Trials for Children with Cancer

The majority of children newly diagnosed with cancer are enrolled on clinical trials. Clinical trials are conducted by physicians, or other medical personnel, to evaluate safety and effectiveness of a particular intervention – typically a treatment, prevention, or detection strategy for a disease.

Patients enrolled on clinical trials are followed closely by both their individual physicians as well as the investigators in charge of the trial. Research protocols, written by experts in that particular disease based upon results of previous clinical trials or other studies, also provide a standard treatment outline for all pediatric oncologists to follow. As a result in large part of enrolling most children with cancer on clinical trials, the cure rate from childhood cancer, which was dismal 30 years ago, is now about 75%.

Though a marked improvement, this is still not good enough. Therefore, clinical trials, in addition to providing the best in available pediatric oncology care, continue to be integral to improving old strategies as well as developing new medicines and other innovative approaches to children with cancer.

Types of clinical trials

Phase 1
This is the first clinical study performed for a new treatment. The trial’s primary purpose is to determine toxicity (side effects) and safety of the treatment. Different dosages are tried to determine the best dose to use for the Phase 2 study. The Phase 2 dose is often the maximally tolerated dose (MTD) during the Phase 1 study, based on toxicity. While effectiveness of the treatment will also be evaluated, that is not the main purpose of the trial. Patients are typically enrolled in a Phase 1 trial when no Phase 3 or Phase 2 trials or other known effective treatments are available.

Phase 2
This is the second stage for evaluating a new treatment. It is a trial to see if the treatment has any effect on the particular disease. Toxicity (side effects) is still monitored quite closely. Patients are typically enrolled on a Phase 2 trial when no Phase 3 or other known effective treatments are available.

Phase 3
This is the third stage for evaluating a new treatment. It is a comparison study between at least two different treatments. It often compares a known, standard treatment to a new treatment strategy, which is hoped to improve outcome. Often, it includes a new drug shown to be effective in a Phase 2 study. Patients are randomly assigned (like flipping a coin) to one of the treatments. If it becomes clear in the middle of the trial that one treatment is better, then the trial will be stopped and patients switched to the better treatment. Most newly diagnosed children with cancer, as well as some at relapse, are treated in Phase 3 clinical trials.

This is a clinical trial involving a small number of patients of a new treatment strategy. It is often performed just before bringing the new treatment strategy to a large randomized Phase 3 trial. The main purposes are to assess feasibility of the new approach and to evaluate for any unexpected toxicity (side effects) before enrolling large numbers of patients.

This is a translational research trial that uses patient samples, such as tumor, blood, or bone marrow, to give researchers more information about diseases and treatments. These trials usually involve laboratory studies conducted on the molecular level. Phase 1, 2, 3, and pilot studies often have biological components.

This type of trial typically involves the patient or family filling out a written questionnaire. The purpose is to look for genetic or environmental factors that affect why a patient gets a type of cancer and/or how well they respond to treatment.

Quality of Life (QOL)
This type of trial may involve written questionnaires, interviews, or other surveillance tools to assess patients’ quality of life. These trials differ from trials which concentrate on evaluating the direct medical impact of certain treatments or interventions. Instead, the researchers here focus on how a disease and/or intervention for that disease impact on patients’ overall quality of life.

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