NCCN Guidelines for Patients™: Multiple Myeloma
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Treatments used in place
of standard treatments
A federal government
agency that regulates
drugs and food
A fake medicine
that has no active agents
physical or emotional
response to treatment
4.3 Phases of clinical trials
Tests and treatments aren’t offered to the public as soon as they’re made.
They need to go through a series of trials to make sure they’re safe and work.
Clinical trials have four phases. They are labeled with Roman numerals I – IV.
The phases are described next using the example of a new drug treatment:
Phase I trials aim to find the best dose of a new drug with the fewest side
effects. The drug has already been tested in lab and animal studies, but now
it needs to be tested in patients. Doctors start by giving very low doses of the
drug to the first group of patients. Higher doses are given to the next groups
until side effects appear or the desired effect is seen. The drug may help
patients, but phase I trials are to test a drug’s safety. If a drug is found to be
safe enough, it can be tested in a phase II clinical trial.
Phase II trials assess if a drug works for a specific type of cancer. They are
done in larger groups of patients for whom standard treatments aren’t working.
Often, new combinations of drugs are tested. Patients are closely watched to
see if the drug works. The treatment team also looks for side effects. If a drug
is found to work, it can be tested in a phase III clinical trial.
Phase III trials include large numbers of patients. Often, these trials are
randomized. This means that patients are put into a treatment group by chance.
There can be more than two treatment groups in phase III trials. The control
group gets the standard treatment and the other groups get a new treatment.
Sometimes, standard care is no active treatment, so a placebo is used. Neither
you nor your doctor can choose your group. Every patient in phase III studies
is watched closely. The study will be stopped early if the side effects of the new
drug are too severe or if one group has much better results. Phase III clinical