Trial Phases
2
3
4
4
3
2
1
1
1
2
3
4
2
3
4
1
Phase 1
First-time testing, mainly to evaluate safety.
Typically includes up to 60 people with a particular cancer diagnosis or cancer mutation profile.
Every patient will receive the investigational drug (there is no placebo).
Since less is known about the investigational drug, there are more frequent visits, including additional blood tests and procedures to learn about the effects of the investigation drug.
A Phase 1 trial may test:
– Dosage range and schedule
– Side effects
– Early signs of effectiveness against the cancer
Phase 2
Test safety as well as whether an investigational treatment is effective for a specific type of cancer.
Study participants are typically assigned to different groups, and each group gets a different dose or schedule to provide information about which works best with tolerable side effects.
Phase 2 trials involve more study participants than the Phase 1 trials but usually include fewer than 100 people with cancer who meet specific requirements for entry into the trial.
Phase 3
Compare an investigational treatment to the current treatment(s) available for that type of cancer.
Much larger than a Phase 1 or 2 trial and can include thousands of study participants randomized to receive either the investigational treatment or the standard treatment for their type of cancer.
In most cases, neither the study investigators or the patient knows which cancer treatment they are receiving (called double-blind), so results cannot be influenced with conscious or unconscious bias.
Typically, a Phase 3 clinical trial needs to show that the investigational treatment is better than the existing treatment to obtain FDA approval.
Phase 4
Tested after it has been approved by the FDA for a specific use.
These trials are conducted to collect information about long-term effects on the patient’s quality of life, length of life and long-term side effects.
Phase 4 trials may also be used to test new combinations of approved therapies to determine whether outcomes are better.
