- Clinical Trials: What You Need to Know
- Why do we need clinical trials?
- What happens before clinical trials?
- Some facts about clinical trials: Important points to keep in mind
- What are the phases of clinical trials?
- Phase 0 clinical trials: Exploring if and how a new drug may work
- Phase I clinical trials: Is the treatment safe?
- Phase II clinical trials: Does the treatment work?
- Phase III clinical trials: Is it better than what’s already available?
- Submission for FDA approval: New drug application (NDA)
- Phase IV clinical trials: What else do we need to know?
- Who sponsors and runs clinical trials?
- Should I think about taking part in a clinical trial?
- Answers to some common questions about clinical trials
- Other questions you should ask your research team
- What protects the study participants?
- What’s out there? Finding clinical trials
- How do I figure out which study is for me?
- What about cost? Will my insurance cover it?
- What would it be like to take part in a clinical trial?
- What if I’m not eligible for a clinical trial?
- Summing it all up
Phase I clinical trials: Is the treatment safe?
These studies are usually the first studies of a new drug that involve people. Although the treatment has been tested in lab and animal studies, the side effects in people can’t always be predicted. For this reason, these studies usually include a small number of people (15 to 50) and may be reserved for those who do not have other good treatment options. Often, people with different types of cancer are eligible for the same study. These studies are usually done in major cancer centers.
The main reasons for doing phase I studies are to find the highest dose of the new treatment that can be given safely without serious side effects. These studies also help to decide on the best way to give the new treatment. The first few people in the study often get a low dose of the treatment and are watched very closely. If there are only minor side effects, the next few patients may get a higher dose. This process continues until doctors find the dose that’s most likely to work while having an acceptable level of side effects.
Safety is the main concern at this point because this is usually the first time the treatment has been used in people. Doctors keep a close eye on how the people in the study are doing. They watch for any serious side effects. Because of the small numbers of people in phase I studies, rare side effects may not be seen until later. Special tests, such as blood tests to measure levels of the drug in the body at certain time points, are often a part of these clinical trials. Some studies may require time in a hospital. Placebos (sham or inactive treatments) are not part of phase I trials.
These studies are not designed to find out if the new treatment works against cancer. Overall, phase I trials are the ones with the most potential risk. And only phase 0 has a smaller chance of helping you than phase I. But phase I studies do help some patients. For those with life-threatening illnesses, weighing the potential risks and benefits carefully is key.
Here’s an example of how a typical phase I clinical trial might work:
Bruce was diagnosed with cancer 4 years ago. He was first treated with radiation therapy, but the cancer was later found to have spread to distant parts of his body. His doctor told him chemotherapy drugs A and B might help him. Bruce’s cancer shrank for a short time while he was taking drug A, but then it began to grow again. Drug B did not work for him.
Because Bruce is still fairly young, his doctor suggests he might want to consider trying a new form of treatment, “EX-1,” which is being studied in a phase I clinical trial at a nearby university hospital.
Bruce talks with the doctor conducting the study. The doctor explains that the drug being studied showed some promise in lab tests, but exactly how well it will work in people is still unknown. What’s more, it may have side effects that haven’t been seen yet. After getting all of his questions answered and weighing his options, Bruce decides to take part in the study.
Because 3 people have already enrolled in the study and have had no major side effects, Bruce will be the first person to get a higher dose of the treatment. He will need to stay in the hospital overnight on the first night. This is both to watch for any unexpected reactions and to take blood samples every few hours so that doctors can figure out how long the treatment stays in his body. He will get to go home the next day, but must return regularly over the next few weeks to be watched closely until it’s time for the next treatment.
Last Medical Review: 09/21/2012
Last Revised: 09/21/2012