CHAPTER 4 Clinical Trials

The challenge in pediatric oncology remains clear: to strive
for the cure and health of all children through the
development of more effective yet less damaging treatment
for our young patients.
—Daniel M. Green, MD and Giulio J. D’Angio, MD
Late Effects of Treatment for Childhood Cancer
WITHIN DAYS OF ARRIVING at a major pediatric medical center with a child newly diag-
nosed with leukemia, parents are often asked to enroll their child in a clinical trial. A
clinical trial is a carefully controlled research study that uses human volunteers to
answer specific scientific questions. In order to accurately evaluate any new treat-
ment, large numbers of patients are needed in each clinical trial.
Pediatric clinical trials are all directed toward improving upon existing treatments. A
trial can involve a totally new approach that is thought to be promising, fine-tune
existing treatments, improve the results or reduce the toxicity of known treatments,
or develop new ways to assess response to treatments. More than half of all children
with cancer in North America are enrolled in clinical trials for part or all of their
treatment.
This chapter describes clinical trials and protocols and gives examples of how differ-
ent parents made decisions on this important issue. The trials discussed here are
treatment trials aimed at the leukemia itself. You may also be invited to participate in
other studies which are sponsored by pharmaceutical companies, especially those
designed to support patients through the effects of treatment. Such supportive care
trials evaluate antibiotics, antinausea drugs, and new agents to raise blood counts,
minimize pain, or control other symptoms. The oversight and control of these trials
involves an entirely different mechanism than the oncology treatment studies dis-
cussed in this chapter. Ask your doctor or nurse to discuss these studies with you if
you are invited to participate in them. Making an informed judgment on whether to
participate is crucial, because it will determine what treatment your child will receive
in the years to come.