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Together is a new resource for anyone affected by pediatric cancer - patients and their parents, family members, and friends.

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Clinical Trials

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Clinical trials look for new and better treatments.

What are clinical trials?

Clinical trials are carefully designed research studies. Therapeutic clinical trials test treatments in people with certain diseases. Some clinical trials do not offer treatments. Non-therapeutic clinical trials collect information to help us learn more about the disease or its effects. A non-therapeutic trial may not offer a medical benefit to the patient. But what scientists learn from the study may help other patients in the future.

Clinical trials often use experimental treatments. An experimental treatment has not been fully tested and is not approved by the Food and Drug Administration (FDA). It is not part of standard medical care.

Clinical trials may study:

  • New medicines
  • Existing medicines for a different disease or group of patients
  • Different medicine doses or schedules
  • How to make other therapies more effective
  • Different combinations of treatments
  • New methods, devices, or equipment

Many current treatments were first studied in clinical trials. Those who take part in clinical trials can help in the search for new treatments.

Clinical trials help find:

  • Cures for diseases
  • Better treatments with fewer side effects
  • Ways to support long-term health and quality of life

Michele and Joseph, parents to a child with cancer, discuss reasons to consider a clinical trial, questions to ask, and hopeful thoughts. Watch their story.

Clinical trials versus standard medical care

Clinical trials are different from standard medical care.

Therapeutic clinical trials seek to improve treatments. Researchers must follow a specific research plan or protocol during a clinical trial.

Differences between clinical trials and standard care

Clinical trials

  • Treatment is experimental
  • Treatment follows the research plan
  • Treatment benefits are not fully known
  • Side effects are not fully known
  • Patients may get standard care and experimental treatment or experimental treatment only

Standard medical care

  • Treatment has been tested 
  • Treatment based on individual patient’s needs
  • Treatment benefits are known
  • Side effects of the treatment are known
  • Patient only gets standard care

Safety in clinical trials

Clinical trials have steps to ensure safety. A review board must review and approve every clinical trial. This board is an independent group of experts.

The review board also:

  • Monitors the study as it happens
  • Makes sure the study is ethical
  • Makes sure risks are reasonable compared to potential benefits

Key points about clinical trials

  • Clinical trials are an important part of finding cures.
  • Most treatments today were part of clinical trials.
  • Independent boards review and approve trials to make sure they are safe and ethical.
  • Taking part in a trial may benefit you. It will also help researchers create better treatments for future patients.

Reviewed: October 2023