Understanding the Phases of Clinical Trials
What are Clinical Trials?
by Lou Ann Donovan
Clinical trials also called interventional clinical studies involve research using participants. Through clinical trials, doctors find new ways to improve treatments and quality of life for people diagnosed with a type of blood cancer Myeloproliferative Neoplasms (MPNs). The participants receive specific interventions, such as drugs according to the research plan or protocol created by the principal investigator.
For MPN clinical trials, the research team consists of an MPN medical doctor who is the principal investigator along with a team that may include doctors, nurses, and other health care professionals.
Phases of Clinical Trials
Clinical trials involve a series of steps, called phases. These phases are defined by the FDA, Food and Drug Administration. If a new drug is successful in an early phase then it will proceed to the next phase. Researchers monitor each phase for safety, dosage, side effects, and benefit to the patient.
The following list shows the purpose of each phase and the number of patients that participate.
Phase 0 or Early Phase 1 are exploratory studies and are not as common. They are very small clinical trials that help researchers decide if a new drug should be tested in a phase 1 trial.
Phase 1 purpose is to find a safe dose, decide how the new drug should be given by mouth or in a vein and see how the new drug affects the body and fights the cancer. The number of participants is 15-30.
Phase 2 purpose is to determine if the new drug has an effect on the MPN and see how the new drug affects the body and fights the cancer. The number of participants is less than 100.
Phase 3 purpose is to compare the new drug with the current standard drug used for MPN’s. The number of participants is 100 to several thousand.
Phase 4 can help researchers learn more about the treatment outside of a clinical trial and are not as common. This phase looks at the long-term safety and effectiveness and takes place after the new drug has been approved by the FDA and is on the market.
Some researchers may design clinical trials to combine two phases for example phase 1 and 2 or phase 2 and 3 in a single protocol. With combined phases, there is a seamless transition between trial phases, which may allow research questions to be answered more quickly or with fewer patients in the study.
Possible Benefits and Risks
Clinical trials are another treatment option for MPN cancer patients. Like all treatment options for cancer, many have possible benefits and risks. The following information can be used to assist you with making a decision on joining a clinical trial.
Benefits of clinical trials for patients:
- Access to a new treatment
- Will be closely monitored by research team
- May be more effective than previous treatments
- Helps scientists learn more about MPN’s
Risks of clinical trials for patients:
- May have worse or unexpected side effects
- More visits to the doctor with extra expenses for travel
- May need additional tests
- Health insurance may not cover all patient costs
Clinical trials need people like you
Clinical trials and all their individual phases are an important part of clinical research. They assess the safety and effectiveness of new drugs or treatments before being approved by the FDA for use. Trials are available for all stages of cancer. Before participating in a clinical trial, talk to your health care provider and learn more about the potential benefits and risks for your specific MPN diagnosis.
Publisher Note (David Wallace):
I find it very encouraging that a search of clinicaltrials.gov shows over 270 clinical trials on Myeloproliferative Neoplasm, MPN with recruiting status. Numerous drugs are in late stage trials, likely leading to new treatments in 2021 and beyond. The future looks bright as MPN has drawn world-wide attention, as novel therapies are being developed for a disease that presently has limited treatment options.
This article is sponsored by Bristol Myers Squibb and Incyte Corporation. MPN Cancer Connection has complete independence in content creation.
