Clinical trials and cancer

Whether you've just learned the devastating news that your child has cancer, or your child has been dealing with cancer for some time, you may be offered the opportunity to take part in a clinical trial. Through research, our team is able to offer more options to patients and families in their battle with cancer and blood disorders. Offering such treatment options is our commitment to helping patients and families achieve the best possible outcomes.

Clinical trials have made tremendous progress in improving treatments and increased survival rates for children with cancer.  Research has also helped us decrease the short and long-term side effects of therapy.  Participation in research helps us to understand and test new ways to treat diseases and may give your child access to new treatment options that are not widely available.  Many of our patients and families enroll in clinical trials to help us advance cancer and blood disorders research and possibly help future generations of children.

At Children's Hospital of Wisconsin, with the support of the MACC Fund, we are able to offer many of clinical trials through a variety of sources.  Ask your child's doctor about clinical trial options that may be suitable for his or her condition.

Types of Clinical Trials

Clinical trials are mainly classified into Therapeutic and Nontherapeutic Trials.

Therapeutic trials

Therapeutic trials help determine the safety and effectiveness of treatment options for particular diseases. Therapeutic trials are conducted in phases, with the results of the previous phases leading to additional clinical trials. The following is an outline of the phases of a trial including a description of each phase's goals:

Phase I

  • Goal: To evaluate the maximum safest dose of a new treatment with minimum side effects.
  • The knowledge obtained from a Phase I trial may not benefit the individual patients enrolled in the trial, but can lead to improved treatment options for future children.

Phase II

  • Goal: To evaluate the effectiveness of a new treatment for a specific type of cancer and learn how the new treatment may affect the body.
  • Side effects are monitored very closely to determine how a treatment may affect the body.

Phase III

  • Goal: To evaluate whether a new treatment with fewer side effects is more effective than an existing standard treatment.
  • Patients may be assigned into different treatment groups (control group vs. investigational/experimental group) through a process called randomization.  
  • Randomization means that the treatment is assigned based on chance. It is like flipping a coin, except that it is done by a computer.
  • The Control Group receives the standard treatment and the Investigational/Experimental group receives the new test treatment.
  • Randomization ensures that individuals in each group have similar characteristics, so that any differences observed in treatment responses between the two groups are due to the treatments being studied and not influenced by anything else.

Nontherapeutic trials

Nontherapeutic trials do not provide a treatment to patients.  Instead, these trials study important factors that help advance the understanding of cancer.  These factors include: studies of cancer biology, causes of childhood cancer, the study of treatment side effects and how to reduce them, and quality of life during and after cancer treatment. These studies may involve filling out surveys, answering questions, or donating blood or tissue samples for future research.