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Bone Marrow/Stem Cell Transplant Frequently Asked Questions
Helpful information for patients, caregivers and families
(Bilingual Spanish/English)

18. What is a clinical trial?

According to the U.S. Department of Health and Human Services, a clinical trial is "a research study conducted to evaluate a medical procedure or medical product, such as a drug." New treatment approaches for transplant-related side-effects and complications are also being tested through clinical trials.

Clinical trials have three phases:

  • Phase 1: Determines whether the treatment is safe, the best way to give the treatment and the right dose that causes the fewest side effects
  • Phase 2: Determines the effectiveness of treatment and whether there are less common side effects which may appear when more patients receive the treatment
  • Phase 3: Compares/contrasts the new treatment with standard treatment

Benefits of a clinical trial may include lower treatment costs, additional monitoring and follow-up above the standard of care, and potential availability of newer treatments not currently available to treat your malignancy. Drawbacks may include unknown side-effects and insurance issues.

Discuss the pros and cons of participating in a clinical trial with your doctor. For information on Clinical Trials, see Resource Listing under National Cancer Institute and National Institutes of Health.

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