Medical Archives - Kids Cancer Resources

Tag: Medical

February 16, 2023

Expert Advice: What Are Clinical Trials?

What is important to know about clinical trials

Article by: Katie Sutton, M.D. – Pediatric Oncologist

Research studies and clinical trials are how doctors learn about cancers and how to treat them. Some studies simply collect information without guiding treatment. For example, for many cancers there are “biology studies” or registries. These studies allow us to learn about cancer. Some things we might learn from this type of study include:

How often a type of cancer occurs
What type of patients are most commonly affected (infants vs teenagers, boys vs girls, patients of a specific race or ethnicity?)
Information about the cancer such as what the cells look like under the microscope, common genetic changes, and common laboratory findings
How often the tumor spreads to other sites

If your child participates in this type of study, information about them and their cancer will be collected. For some studies, a blood or urine sample or a small leftover piece of tumor tissue may be saved or used in the laboratory. You and your child’s healthcare team may or may not receive any information back from the researchers in this type of study. This will be explained before you decide to participate.

What are the different stages of clinical trials?

Clinical trials help us understand how to treat cancers. They are research studies that test new medicines or combinations of medicines. There are different types of treatment studies called Phase 1, Phase 2, and Phase 3. Each type of study has a different purpose. Often before a medicine is tested in kids it goes through these steps in adults first.

Phase 1

These are the very earliest studies for a new drug. They involve a small number of patients. The goal is to test the drug’s safety and to find the best dose of the medicine (usually the highest dose with acceptable side effects). The investigators will be watching for signs that the medicine is working (“efficacy”), but this type of study is not designed to test how well the treatment works. If the medicine is tolerable and safe it can move to Phase 2.

Phase 2

These studies usually include more patients, although they are still small. They use the dose of the medicine from the Phase 1 study and begin to evaluate if the medicine works. The researchers will continue to monitor the safety of the treatment and what side effects patients have. If the treatment is promising, it will move on to Phase 3.

Phase 3

These studies include the largest number of patients and are trying to prove the new medicine works to treat the cancer. A Phase 3 study often compares the new medicine to the best known therapy (“standard of care”). If the new medicine is found to be better, it will become the new standard of care moving forward. Researchers continue to monitor for safety and side effects.

All phases of trials may also be trying to answer additional questions. For example, studies may evaluate a patient’s quality of life or may test whether certain blood tests are helpful during treatment. These will always be explained to you

For tips on how to talk with your child’s doctor about this and other issues, you can check out this blog for tips!

By Lexi Robertson

For Parents

February 15, 2023

Expert Advice: What Is The Process For Being On A Clinical Trial?

What Is The Process For Being on Clinical Trials?

Article by: Katie Sutton, Md – Pediatric Oncologist

Screening: In order to be enrolled on a clinical trial, your child’s healthcare team will do a thorough evaluation of their cancer (may be with images, labs, or biopsies – taking a sample of the tumor) as well as an evaluation of their current health overall. If they meet the criteria for study participation and your healthcare team believes the study is an appropriate option for your child, the study will be discussed with you.

Consent: All parts of the study should be thoroughly explained to you, and you should feel like you have plenty of time to ask questions. It’s your healthcare team’s job to make sure you understand everything about your child’s cancer and the study. You should understand your child’s diagnosis and prognosis. You should know what procedures are part of the study. You should be told that participation is voluntary and what alternative treatments exist. If you choose to participate (more on that later), you’ll sign a consent document – sometimes these are very long! You should always be provided with a copy. Even if you consent, you always have the right to withdraw from participation.

Enrollment: Your child will then be enrolled in the study. If it is a treatment clinical trial, there will usually be labs and other tests that have to happen when they start the therapy and then at set times throughout the study. In order for doctors to really answer the questions about whether a treatment is safe and effective, the study procedures and treatment must be given in a specific way and obtained at specific times. It may feel rigid, but it’s the only way to make sure the data is accurate and understandable. If you’re worried you might not be able to stick to the schedule, it’s important to tell your child’s healthcare team.

How to decide whether to participate in a study or clinical trial?

The decision to participate in research including clinical trials is a very personal one. Things one might consider:

How do you feel about research in general? As a doctor, I believe that research studies are how we’ve made such great progress in treating childhood cancer over the past 50 years, but sometimes due to past personal experiences or experiences of loved ones, families are uncomfortable with participation in research and THAT IS OK! Your healthcare team will support you.
Can you stick to the treatment plan and schedule of the study?
What other options exist for your child?
How common is your child’s cancer type? Is there a “standard of care?” Some cancers are so rare that we aren’t certain how to best treat them.
How is your child’s underlying health? How might the study impact their quality of life?
What Phase is the trial? A Phase 3 study of a medicine that has been already tested for safety and signs of efficacy (working) has a reasonable chance to directly impact your child and their cancer. It will be largely unknown whether a drug in a phase 1 study will work to treat your child’s cancer, but it may help other children in the future.

No Right or Wrong Way

Different families have different experiences, values, and hopes. For some, it is important to feel they have tried everything available to cure their child’s cancer. For others, their child’s quality of life is most important and so they want to pick a treatment that minimizes side effects or allows them to stay at home or avoid medicines. For some, knowing they are helping the “greater good” or kids in the future gives meaning to their child’s experience. There is no “right” or “wrong” way to approach the decision. Your child’s healthcare team should help you understand all of the options and how they align with your priorities for your child.