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Are Clinical Trials Safe?

U.S. clinical trials are designed to give patients the safest, potentially most effective clinical therapies. Clinical trials are conducted once researchers have shown in the laboratory and in animal research that a particular study treatment has a good chance of offering better outcomes for people with a specific disease.

Patients enrolled in cancer clinical trials are never treated as “guinea pigs.” In fact, patients are given either

  • The best treatment currently available, or
  • A new and possibly more effective therapy.

Patients in clinical trials are watched closely by their doctor, as well as by other members of their medical team, to ensure their safety. Every trial has a precise treatment plan called a “protocol,” which must be followed. Patients get a lot of attention and receive excellent cancer care. The trial can be changed or stopped if there is a problem. Patients who take part in a clinical trial also have the option to leave the trial at any time.

In a cancer clinical trial, placebos (simple pills with no medication in them, often called “sugar pills”) are not used in place of a proven effective therapy.


Types of Studies

Many types of treatment are offered through blood cancer clinical trials. The different types of studies are outlined below.

Treatment Type What Are Researchers Studying? Questions Researchers Hope to Answer

Drug therapy

New chemotherapy drugs or new combinations of drugs

What is the best order and combinations of chemotherapy drug therapies?

Drug maintenance therapy

The benefits of continuing therapy after remission with the same drug(s) used to induce the remission

Is disease progression or overall survival any different with or without maintenance therapy?

Targeted drug therapy

Drugs designed to interfere with cell functions of specific cancer cells, resulting in cancer cell death

Does the targeted therapy used alone have better response rates and/or overall survival than standard therapy with fewer side effects? Should it be combined with other standard therapies to improve effectiveness?

Radiation therapy

New combinations of treatment types

Are outcomes improved compared with current outcomes for patients getting combined radiation and drug therapy?


Immunotherapy that can trigger the body's immune system to fight cancer cells

If there is a good response, should the therapy be combined with standard treatment or used alone?


The ability to deliver radiation to blood cancer cells by attaching a radioisotope to a monoclonal antibody

Can this therapy be used as a first-line therapy with fewer side effects and with equal or better overall survival time than current standard front-line treatment options?

Stem cell transplantation

New stem cell transplant procedures

When is the best time for stem cell transplant and which type of transplant is best (auto, allogeneic, reduced intensity allogeneic, tandem)? 

Supportive therapy

Treatments to reduce disease or treatment-related side effects such as nausea, vomiting, infection and fatigue

Does the supportive therapy reduce side effects and improve patients' quality of life? Does it interfere with or compromise the effects of cancer therapy?

Disease and treatment response monitoring

Better diagnostics and monitoring techniques

Are certain cytogenetic tests good indicators of the likelihood of progression of an indolent disease such as chronic lymphocytic leukemia or myeloma? How do imaging technologies such as MRI and PET scans compare in evaluating responses to therapy and recurrence of lymphoma and other blood cancers?