Clinical research is the main way that scientists, doctors and other health professionals gain new knowledge about diseases and conditions. Through the information obtained in clinical research studies we can learn the causes of diseases and how to prevent, treat and even cure them. All clinical trials are reviewed and approved by a special research ethics committee called an Institutional Review Board or IRB.

What is a clinical trial?

A clinical trial seeks to develop better treatments for children with cancer and blood disorders. Very often physicians know what is the best way to treat a particular cancer or blood problem. However even the current "best practice" fails to cure every child and often the treatment may be associated with serious potential side effects. Therefore pediatric hematologists/oncologists are constantly trying to improve treatments. In order to prove that a new treatment is better than established approaches physicians will conduct a clinical trial. Information from a large number of children treated the "new way" can be compared to the currently established practice to determine if the introduction of the new approach offers an advantage.

The type of information collected on clinical trial includes things like the age and gender of the child, the extent of the disease at diagnosis, the response to treatment (e.g. whether a child with cancer went into remission as well as the long term "cure rate") and the side effects of therapy.

Clinical trials fall into categories based on how experimental the treatment is. A new drug that has never been used to treat diseases in children, will be tested to determine optimal doses in children whose condition has not responded to all standard therapy. This is called a phase I clinical trial. A phase II clinical trial evaluates drugs that have been tested in phase I trials to see if it can benefit children with conditions that have failed standard approaches. What is the difference between a phase I and II clinical trial? The purpose of a phase I trial is to determine the right dose whereas the purpose of a phase II trial is to see if the drug actually works to improve the condition being treated.

A phase III trial is where the promising new drug or drug combination is directly compared to established approaches. Since physicians really don't know if the new approach is better (it may be worse) patients are randomized (like flipping a coin) to receive the new approach or the established treatment. If at any time it becomes clear that
one approach is better than the other, then the trial is stopped and all patients receive the better therapy. Unfortunately, without trials, it is impossible to know which new strategy will be the best.

Why not just add the new therapy without testing it?

The problem with any cancer therapy, no matter how promising, is that most cancer drugs have significant side effects. Each approach needs to be tested so that cure rates for children with cancer and other conditions continue to improve. Over the past three decades, the cure rate for childhood cancer has doubled using these principles. Today over seventy percent of children with cancer are cured, even those with far advanced cancers.

Who sponsors clinical trials?

A clinical trial may be sponsored by the government, for example by the National Institute of Health. Alternatively a pharmaceutical company or the School of Medicine may provide support. The majority of clinical trials in childhood cancer are performed by the Children's Oncology Group (COG). COG is a consortium of over 200 hospitals throughout the United States, Canada, and Australia that is dedicated to finding new ways to prevent, diagnose and treat children with cancer. The group is supported through a grant from the National Cancer Institute. As a study participant you will be informed of the sponsor of any clinical trial that you or your child is enrolled on.

What are my rights as a participant in a clinical trial?

As a participant you will be informed of the potential benefits and risks of the clinical trial. All your questions will be addressed at any time during the course of the work. You will be informed of any new unanticipated side effects that may occur and of any new findings suggest that one particular approach proves beneficial during the course of the study. In the event that one treatment proves to be beneficial or too toxic compared to other treatments then the trial is stopped immediately and all children receive the superior therapy.

Who sees my child's medical records?

All medical information is coded so that your child's name is never used in any public documents and the only people that have access to your child's records are the hospital staff and the study investigators. However, for the purpose of assuring that all studies are conducted in a scientifically rigorous manner, the representatives from the National Cancer Institute and the Food and Drug Administration may audit the records.

Where can I get additional information on clinical trials?

You can get additional information by asking your doctor directly or by contacting the following agencies:

Institutional Human Subjects Review Board
Mount Sinai Hospital and School of Medicine
Tisch Hospital and New York University School of Medicine
National Cancer Institute
Children's Oncology Group
American Cancer Society
Leukemia and Lymphoma Society

What will happen if I refuse to participate in a clinical trial or if I change my mind after agreeing to allow my child to be enrolled on a study?
It is your right to refuse to allow yourself or your child to participate in a clinical trial and there will be no penalty for refusing to do so. You are guaranteed the same excellent standard of care regardless of whether your child is enrolled in a clinical trial. If you decline participation, the treatment that will be recommended will be that judged to be the standard best medical practice. You have the right to discontinue participation in a clinical trial at any time.