Clinical Trials at UCSF Benioff Children's Hospitals

Frequently asked questions

• What is a clinical trial?

  A clinical trial is a study that evaluates the safety and effectiveness of an experimental treatment in human volunteers. By joining a clinical trial, patients may have access to new therapies before they're available to the general public.

  Researchers conducting a clinical trial must follow strict rules in order to protect participants. Typically, treatments are first tested in laboratory animals before being studied in humans.

  In a clinical trial, participants may be assigned to receive an experimental treatment, such as a medication, surgical procedure or medical device. During the trial, the researchers measure specific outcomes in order to determine the treatment's safety and efficacy.

  In another type of clinical study, called an observational study, participants aren't assigned to receive a specific treatment. Participants receive routine care and are monitored over a period of time, while the study team collects and examines data about them.

• How do clinical trials work?

  Clinical trials are done in phases. In each phase, researchers try to answer a different question.

  Phase I: Is it safe?

  In phase I, researchers test a new treatment in a small group of people for the first time. The main goal is to assess the treatment's safety. Participants may be healthy or may have the condition that the treatment is intended to help.

  Phase II: Does it work?

  Once researchers determine that a treatment is safe, they test it in a larger group of people to make sure it is also effective.

  Phase III: Is it better than what we have now?

  In phase III, researchers compare the new treatment with an existing treatment (or treatments) to see which is safer and works better. This phase usually involves a large number of participants. If the study shows that a treatment is safe and effective, the Food and Drug Administration (FDA) reviews it for approval.

  Phase IV: What else can we learn?

  After a treatment is approved by the FDA and available to the general public, researchers may do a phase IV study to look at whether the treatment is useful for other conditions or leads to problems in the long run.

• Who can join a clinical trial?

  People of all ages, genders and ethnic groups can participate in clinical trials. The kinds of participants needed for a trial depend on the trial itself and the questions researchers are trying to answer. Criteria for a trial may include age, sex, type and stage of a specific disease, previous treatments or other medical factors.

  Clinical trial participants can be healthy volunteers or people with a particular condition, known as patient volunteers.

• Why should I consider a clinical trial for my child?

  Clinical trials are at the heart of medical research: They help establish better treatments and bring better outcomes to patients. Healthy volunteers may choose to participate because they want to help others and to contribute to scientific research.

  Patient volunteers may also be motivated by the opportunity to get access to a promising treatment that isn't yet available to the general public. However, families must understand that their child's condition may not improve with the experimental treatment and that their child could experience adverse side effects, among other possible drawbacks.

  Participating is a personal choice. If you're considering a particular clinical trial for your child, make sure you know what the study involves; ask as many questions as you need to, in order to feel informed and comfortable; and weigh the potential benefits and drawbacks when deciding whether participating is a good choice for your child. Here are some questions to think about:

  • Does my child want to participate in this study? For children who are developmentally able to understand, it's important to explore their feelings and address their questions or concerns.
  • Do I believe the study will make a significant difference to my child's life or the lives of other children?
  • Do I fully understand the potential benefits and risks of participating?
  • How will being in the trial impact my family? Will my child have to miss school or other activities? Will I need to take time off work? Will I be reimbursed for expenses, such as transportation?

  You may withdraw your child from a study at any time and for any reason.

• I'm interested in a clinical trial – what next?

  If there's a clinical trial at UCSF that you believe may be a good choice for your child, here are the next steps:

  1. Use the link on the study's webpage to let the researcher know you're interested.
  2. The researcher will contact you and tell you more about the study. Please ask all the questions you have and make sure you understand the answers.
  3. The researcher will ask you some questions to see whether your child meets the criteria for the study. If your child is eligible and you decide to proceed, the researcher will enroll your child.
  4. You will review and sign a consent form. The consent form explains your rights, states possible benefits and harms of the study, and describes what participants need to do. This will likely include visits to UCSF for treatments and tests.