There are two general types of clinical trials:
-
Therapeutic clinical trials are used to evaluate new treatments for childhood cancer as well as other diseases. These studies are classified as three different types or phases. Each phase has different goals.
-
Non-therapeutic trials are those that involve other aspects of childhood cancer such as:
- Epidemiology (incidence and causes)
- Cancer Control (how best to deal with side effects)
- Late Effects (long-term complications of treatment)
Therapeutic Clinical Trials
Phase I
Goals:
- Test a new treatment in humans that has been tested in the laboratory and in animal studies.
- Learn how the human body responds to a new treatment and what the side effects are.
- Find the best way to give a new treatment.
- Find the highest dose of a new treatment with acceptable side effects.
Method:
- Patients are given a low dose of the treatment and are observed for side effects.
- The doses are increased according to a set plan in groups of patients until side effects become potentially dangerous.
Patients:
- Trial is offered only to patients whose disease has not improved with other less experimental treatments.
- Usually relatively few patients are enrolled in any one of the trials.
Potential Benefits:
- A few patients may respond to the new treatment and show improvement of their disease (usually less than 1 in 20).
- Future patients benefit from what is learned in this type of trial.
Potential Risks
- Side effects of the new treatment may be worse than expected.
- Patients may spend more time in the hospital or clinic to receive the new treatment or have tests done to evaluate the response.
- Most patients do not directly benefit from these studies.
Phase II
Goal:
- Learn if a new treatment works in patients with specific types of cancer.
- Learn more about how a new treatment affects the human body.
Method:
- Patients with different types of cancer receive a new treatment using the highest but still safe dose found in a Phase I trial.
- Patients are observed for improvements in their cancer and any side effects.
Patients:
- Trial is offered to patients who have not improved with other available treatments.
Potential Benefits:
- A few patients may respond well to the new treatment and show definite improvement of their disease.
- Future patients will benefit if the treatment works better than current treatments.
Potential Risks
- Benefits of the treatment are not known.
- Patients may spend more time in hospital or clinic to receive the new treatment and to have tests done to study their response to the treatment.
- Many Individual patients do not directly benefit from these studies.
Phase III
Goal:
- Learn if a new treatment that has been shown effective in treating a specific type of cancer in a Phase II trial is better in some way (more cures, longer control of disease, fewer or less serious side effects, or fewer days in the hospital) than the best current treatment for the disease.
- These trials are usually offered to patients who have just been diagnosed with cancer.
Method:
- Patients are assigned by a method called randomization to one of two or more treatment plans, one of which is the standard treatment.
Patients:
- Patients with the same type of cancer who otherwise would receive best current treatment.
Potential Benefits:
- Trial offers the most up-to-date treatments with the best-known results for improvement or cure of their disease.
- Some patients may respond better to the new treatment or show the same anti-cancer effects but with fewer side effects.
- Future patients will benefit if the treatment works better than current treatments.
Potential Risks
- The new treatment being compared is considered to be at least as good as the standard treatment, but it is not known for certain whether the new treatment will be better than the standard treatment either in curing more patients or in producing fewer side effects.
- Patients may spend more time in hospital or clinic to have tests done to study and compare their responses to the treatment.
- Sometimes new side effects are revealed with the new treatment, which means the new treatment might have to be modified.
More Clinical Trials Information
Catherine Bourne, RN, BHSc(N) Barbara Pugh, RN, MS Margaret Hussong, RN, MS, PNP Elizabeth Gilger, MSN, RN, CPON, CPNP