Clinical Trial Phases:
To achieve FDA approval, all new treatments must typically pass through 3 phases of testing. Each phase is designed to test different aspects of a new treatment.
Phase 1 trials test a new agent on a small number of subjects for overall safety and to find the appropriate dose that can be safely given with acceptable side effects.
Phase 2 trials determine if a therapy has any activity against the cancer and can prevent tumor growth, progression, extend a patient’s life, or relieve symptoms.
Phase 3 trials compare promising treatments from Phase 2 against standard treatments to determine if the test treatment works better and has fewer or more manageable side effects. Phase 3 trials are typically large (hundreds of patients), randomized (each patient is randomly assigned to the standard treatment or the test treatment), and sometimes blinded (the patient and/or doctors are not told which treatment the patient is getting as a way to control for the “placebo effect”).
“Other” phase trials include: early phase 1 trials, which investigate how or whether a drug affects the body, and are not designed to evaluate therapeutic or diagnostic effects, phase 4 Trials, in which approved drugs are continually monitored for safety and efficacy, and “phase not applicable” trials which do not have a FDA-defined phase, such as trials that evaluate devices or behavioral interventions.
Clinical Trial Treatment Categories:
Anti-androgen: Prostate cancer is typically fueled by male hormones (“androgens”), and thus anti-androgen therapy is part of the standard of care for advanced and metastatic prostate cancer. FDA-approved anti-androgen therapies include androgen deprivation therapy (ADT) and second-generation anti-androgens such as abiraterone, enzalutamide, and apalutamide. Trials in this category are testing new types of anti-androgens, new ways or timing for using anti-androgens, and anti-androgens in combination with other types of treatments.
Targeted therapy: Trials in this section are testing treatments that target specific proteins expressed by tumor cells or other types of cells that support tumor growth. In some of these trials, patients will first need to be tested for whether their tumors express the target or have genetic alterations that make them likely to be sensitive to the treatment. There are currently no FDA-approved targeted therapies for prostate cancer.
Chemotherapy: Chemotherapies are treatments that kill cancer cells by targeting activities that are more prominent in cancer than normal cells, such as DNA replication, cell growth and cell division. Treatments in this category test FDA-approved and new chemotherapies in new ways in prostate cancer.
Immunotherapy: These are treatments that activate a person’s own immune system to target and kill their cancer. Types of immunotherapies include checkpoint immunotherapy, engineered immune cells, and anti-cancer vaccines. Two immunotherapies are currently FDA-approved for prostate cancer: Sipuleucel-T and pembrolizumab.
Drug: Trials in this category are testing drugs that do not target androgens, cancer proteins or immune cells.
Radiation: Radiation therapy is a standard-of-care treatment for localized prostate cancer. Trials in this category are testing new radiation therapy methods, new ways or timing for using radiation, and radiation in combination with other types of treatments.
Surgery: Surgical removal of the prostate (prostatectomy) is a standard-of-care treatment for localized prostate cancer. Trials in this category are testing new ways or timing for prostatectomy or surgical removal of metastatic lesions, and surgery in combination with other types of treatments.
Procedure: Trials in this category are testing other procedures that are not radiation or surgery, such as focal therapies for localized prostate cancer.
Other: Trials testing treatments that do not fall into any of the other categories.
Behavioral: Trials that test behavioral interventions such and diet, exercise, and counseling.
Diagnostic: Trials in this category are testing new types of imaging technologies for detection of prostate cancer, genetic testing, and other types of biomarkers that will help to improve understanding of the biology of prostate cancer and/or how to better select treatments for patients. These trials are not testing anti-cancer treatments. However, some are evaluating whether earlier detection of recurrent or metastatic disease can improve treatment and outcomes.