Resources and information for parents of children with cancer . . . by parents of children with cancer.

this page last modified

Clinical Trials

Note: This section has health/medical information. It was not written by a health care professional. The medical references are:

Historically, childhood cancer patients enter clinical trials more often than do adult cancer patients. A child diagnosed with cancer is usually referred to a major treatment facility, such as a Children's Hospital, because childhood cancer is rare and is best treated by the specialists at these facilities. These pediatric oncology specialists are often members of Children's Oncology Group, or COG. COG sponsors clinical trials for most childhood cancers. St Judes, Dana Farber, Duke, and several other institutions also sponsor childhood cancer trials (see below).

Please note: fewer adolescents than young children enroll in clinical trials. See the Teen Cancer Care section.

What is a clinical trial?

A cancer treatment clinical trial is an investigative study of a particular treatment plan. It can study new drugs, combinations of drugs and therapies, modes of drug administration, etc. The clinical trial documentation given to all the doctors administering the trial is a detailed protocol so that exactly the same treatment will be given to all patients.

The National Institute of Health (NIH) announced the launch of a new web site "Children and Clinical Studies" in 2008. This is a great reference, covering a multitude of issues concerning the participation of children in clinical trials. It includes FAQs and videos.

Clinical trial phases

Clinical trials are one of three types, called "phases", as defined below.

Phase I. Studies brand new drugs. In other words, they really don't know whether or not the drugs will kill people or cancer first. In this phase, they are trying to figure out what doses of drugs or therapies can be given to humans, extrapolating from animal trials.

Phase I studies are true experiments. Laboratory tests have suggested that the new drugs might be effective against cancer, but the researchers do not know if they will help, and the doses that children will tolerate are unknown. Parents often enroll their children in these trials in the hope that a new and untried drug will be effective, but they need to recognize that the chances are low.

Phase II. Studies drugs that graduated from Phase I studies, and the trial is testing if the drug is active against a specific cancer. They try them on a few more people and a few different doses. The drugs probably won't kill the patient.

Phase II trials are sometimes chosen by parents when their child has relapsed. Occasionally, Phase II trials are designed to test an exceptionally promising agent against a tumor for which other effective therapies exist. While the treatments are experimental, there is usually reason to believe that patients enrolled may benefit.

Phase III. Studies the standard protocol - the one most often currently used for a particular type of cancer - with drugs that have graduated from Phase II studies. This doesn't mean that they throw out the whole standard protocol and use the new drugs, it means that they "tweak" the current combination of drugs used by slipping in some new ones and/or changing the doses or combinations of current drugs and/or the length of drug treatment. Often the researchers are trying to maintain survival rates while lowering the toxicity of the treatment.

Two Candlelighters/ACCO Newsletters have excellent articles on clinical trials:

Check this external link for the official National Cancer Institute description of clinical trials:

Standard of Care

In a phase III clinical trial, treatment is usually divided into several "arms". One arm is the currently accepted "best" treatment for the cancer. The other arms have slightly different drugs, combinations of drugs, treatment schedules, etc. The currently accepted best treatment is called the "standard arm", or "standard of care". The other arms might be better, they might be worse, but the differences are likely to be only a few percentage points.

If parents enroll their child in the phase III trial, the child will be computer "randomized" to one of the treatment arms. The purpose of computer assignment is to assure that patients are evenly assigned to each treatment plan without bias from physicians or families.

Informed consent

Informed consent does not mean that the pediatric oncologist briefly explains the clinical trial and then the parents (or of-age child) signs the documents. True informed consent requires:

All questions should be answered in language that is clearly understood by the parents and child, and there should be no pressure to enroll the child in the study. The objective of the informed consent process is that the participants are comfortable with their choice and can comply with it.

The Candlelighters journal has an excellent article on informed consent:

Second opinions

Second opinions can be obtained from physicians at other institutions in your community or region, or any of the larger pediatric cancer centers. It is most useful to get a second opinion from a center that treats significant numbers of patients with your child’s diagnosis.

Consultations can be done over the phone, by e-mail, or in person. Be prepared to send information such as scan results or tissue samples. Often parents get different advice and opinions from each oncologist that they consult, and it can be trying deciding which advice is the best. If you feel that you are not getting good advice, keep looking.

Second opinions can also be arranged through the Childhood Cancer Ombudsman Program, which uses volunteer specialists to provide free help to families considering the range of treatment options and informed consent process.

Childhood Cancer Ombudsman Program

The full clinical trial protocol

The clinical trial documentation given to all the doctors administering the trial is a detailed protocol so that exactly the same treatment will be given to all patients. The protocol document lists the drugs, dosages, and tests for each segment of treatment and follow-up. It usually also contains a 5-25 page diagram that shows when each drug and test is given; the familiy might be given this or an abbreviated version (1-2 pages) to provide quick and easy reference on a daily basis. This part of the protocol is frequently called the "roadmap".

The entire trial document (or protocol) often exceeds 100 pages and covers the following topics: study hypothesis, experimental design, scientific background and rationale with relevant references from the scientific literature, patient eligibility and randomization, therapy for each arm of the study, required observations, pathology guidelines, radiation therapy guidelines, supportive care guidelines, specific information about each drug, relapse therapy guidelines, statistical considerations, study committee, record-keeping, reporting of adverse drug reactions, and consent form.

The full protocol is intended for the use of specialists in oncology medicine and nursing, and is not written in lay language. It is highly technical, and can be confusing and/or overwhelming. But some parents throw themselves into research to better understand their child’s illness, and the full protocol contains such a wealth of information all in one place that they will want a copy of this protocol. Since parents have a right to review all literature and information related to their child’s treatment, if you wish to read all of the details of the study, ask that a copy be provided to you. Informed consent documents for Children’s Oncology Group protocols specifically inform families that they will receive a copy of the full protocol upon request. The protocol is not for general distribution, because use of these protocols outside a controlled research setting can be dangerous and is unethical. Thus, if you receive a copy, it should is your personal use only.

Where to find clinical trials

Cancer.gov

This link takes you directly to the main search page on cancer.gov. Select the type of cancer, then select whether the trial is open or closed and the Protocol ID. You can also search for all available trials by entering in other applicable information and leaving the Protocol ID field empty.

ClinicalTrials.gov

The U.S. National Institutes of Health, through its National Library of Medicine, has developed ClinicalTrials.gov to provide patients, family members and members of the public current information about clinical research studies.

St. Jude.org

Select the cancer type for St. Jude's clinical trials and protocols.

Dana-Farber.org

From this page you can find the open clinical trials for pediatric cancers that are offered by Dana-Farber/Harvard Cancer Center.

MD Anderson

From this page you can find out about pediatric clinical trials offered at MD Anderson. Or use this direct link to their trials.

Memorial Sloan-Kettering

A listing of the newest clinical trials at MSKCC.

EmergingMed

EmergingMed gather strial and trial site information from ClincialTrials.gov, trial sponsors, medical centers, advocates and many other sources. On the site or over the phone, one fills out a profile with the details of their cancer; this profile is then matched to the eligibility criteria of all the cancer clinical trials in the EmergingMed database.

TrialCheck

The Coalition of Cancer Cooperative Groups hosts TrialCheck, a nonbiased cancer clinical trial matching service with precise screening that locates trials near patients' homes. This free system was developed by a nonprofit with the goal to make the clinical trial process more accessible for patients.

Center Watch

A clinical trials listing service.

VirtualTrials.com

For brain tumors: Al Musella's Clinical Trials Page.

Burzynski's home page, clinical trials

Burzynski is on the borderline between "alternative" and "conventional" treatment for cancer; his trials do have FDA approval.

Duke Comprehensive Cancer Center

A list of pediatric cancer trials at Duke.

Trials Central

Summaries of evidence from trials around the world.

General Disclaimer

These pages are intended for informational purposes only and are not intended to render medical advice. The information provided on Ped Onc Resource Center should not be used for diagnosing or treating a health problem or a disease. It is not a substitute for professional care. If you suspect your child has a health problem, you should consult your health care provider.

about this site | privacy statement | terms of use | contact