Clinical trials, also called clinical studies, evaluate investigational methods of treating, diagnosing, and preventing cancer. These investigational methods include approaches to surgery or radiation therapy as well as gene therapy, vaccine therapy, experimental drugs, or various combinations of these treatments.
Clinical trials are among the final stages of a long and careful process of cancer research. Only those treatments that show promising results in tests done in the laboratory are used in clinical trials involving people.
There is no guarantee that the investigational approach will produce good results but, if the approach is effective, trial participants may be among the first to benefit. You will receive detailed information about the trial and should join a trial only after you understand both the possible risks and benefits. Everyone who joins a clinical trial at the National Cancer Institute (NCI) is cared for by a team of appropriate experts.
CCR Clinical Trials at the NIH Clinical Center
The clinical trials conducted by the NCI's Center for Cancer Research (CCR) on the National Institutes of Health (NIH) campus (Bethesda, Maryland) represent the core of our clinical program. At the CCR, basic and clinical science are seamlessly integrated with a mission to reduce the burden of cancer through exploration, discovery, and the translation of novel approaches into compassionate and effective care for all cancer patients. Our clinical studies are aimed at answering critical questions about a particular disease or disease process and at identifying promising new therapeutic interventions that can then be confirmed in larger studies carried out across the country at cancer centers participating in NCI-supported research.
NCI's trial-related treatment does not replace the care of a patient's regular physician/health care professional. The NCI:
- Encourages patients to maintain close contact with their physician/health care professional to ensure continuity of care
- Generates a letter updating the referring physician/health care professional after each patient visit
- Emphasizes the exchange of clinical information between investigators and community-based physicians/health care professionals
To make a referral, the physician/health care professional should follow one of the two procedures outlined below:
- Identify trials appropriate for your patient by reviewing the clinical trial summaries available at CCR Clinical Research.
- Contact a member of the research team listed on the trial summary of a particular trial for more information or to discuss a screening visit.
- Call the NCI Clinical Trials Referral Office at 1-888-NCI-1937 with your patient's medical history.
- Have the Clinical Trials Referral Office staff conduct a trial search and assist you in the preliminary evaluation of your patient's eligibility.
- Have the Clinical Trials Referral Office staff either forward the information to the research teams of appropriate trials or provide you with contact information for appropriate investigators.
Clinical Trial Phases
Clinical trials are usually conducted in a series of steps, called phases:
- Phase 0 trials – use investigational agents that have shown promise in laboratory studies and, for the first time, are given to a small group of patients (10 to 15). Small doses of the new agent are given over a short period to confirm that it works safely in humans in the way predicted by earlier lab studies. If the agent was designed to reach a specific target in cancer cells, tests will be done to check on its success. Any side effects are carefully monitored.
- Phase I trials – test investigational treatments in a small group of patients. In these studies, researchers evaluate what dose is safe, how a new agent should be given (by mouth, injected into a vein, or injected into the muscle), and how often. Researchers watch closely for any harmful side effects. Phase I trials usually enroll a small number of patients and take place at only a few locations. The dose of the new therapy or technique is increased a little at a time. The highest dose with an acceptable level of side effects is determined to be appropriate for further testing.
- Phase II trials – study the safety and effectiveness of an agent or intervention, and evaluate how it affects the human body. Phase II studies usually focus on a particular type of cancer, and include fewer than 100 patients.
- Phase III trials – compare a new agent or intervention (or new use of a standard one) with the current standard therapy. Participants are randomly assigned to the standard group or the new group, usually by computer. This method, called randomization, helps to avoid bias and ensures that human choices or other factors do not affect the study's results. In most cases, studies move into phase III testing only after they have shown promise in phases I and II. Phase III trials often include large numbers of people across the country.
- Phase IV trials – are conducted to further evaluate the long-term safety and effectiveness of a treatment. Phase IV trials usually take place after the treatment has been approved for standard use. Several hundred to several thousand people may take part in a phase IV study. These studies are less common than phase I, II, or III trials.
Clinical Investigator Profiles
Director, Medical Oncology Service
James L. Gulley, M.D., Ph.D., F.A.C.P.
Research Interests: prostate cancer, therapeutic cancer vaccines, immunotherapy, other solid tumors (breast, GI)
Medical Oncology Service and Affiliate Investigators
|Christina M. Annunziata, M.D., Ph.D.|
breast and ovarian cancer
|Andrea B. Apolo, M.D.|
|Philip Arlen, M.D.||prostate cancer, pancreatic cancer|
lung cancer, lymphoma, solid tumor
|Gideon Blumenthal, M.D.||lung cancer, breast cancer|
prostate cancer, breast cancer, solid tumors
|Neil Caporaso, M.D.||cancer epigenetics|
|Corey A. Carter, M.D.||lung cancer, aerodigestive cancers|
|Alice P. Chen, M.D.||solid tumors|
|Helen Chen, M.D.||solid tumors|
|Barbara Conley, M.D.||diagnostic markers, drug development, and aerodigestive cancers|
melanoma, immunotherapy, lymphoma
|Stephen Creekmore, M.D.|
|Barbara K. Dunn, M.D.|
|Michele K. Evans, M.D.|
kidney, cervical, and thyroid cancer
hematologic/blood cancer and kidney cancer
|Barry Gause, M.D.||lymphoma, melanoma|
bone marrow and stem cell transplant
|Neelam K. Giri, M.D.|
|James J. Goedert, M.D.|
solid tumor and thyroid cancer
|Mark Greene, M.D.|
|Udayan Guha, M.D.||lung cancer|
|prostate cancer, therapeutic cancer vaccines, immunotherapy, other solid tumors (breast, GI)|
|David Halverson, M.D.||stem cell transplantation, leukemia and lymphoma|
malignant mesothelioma and lung cancer
|Christopher Heery, M.D.||immunotherapy, therapeutic cancer vaccines, chordoma, gastrointestinal cancer, rare diseases|
|Lee Helman, M.D.||rhabdomyosarcoma, Ewing's sarcoma and osteosarcoma|
MonoMac and DOCK8 deficiency
|Richard Hodes, M.D.||thymus development, T cell receptors, tumor suppressor p53, ataxia telangiectasia mutated|
|Paul Kluetz, M.D.||prostate cancer|
Elise C. Kohn, M.D.
breast and ovarian cancer, fallopian tube cancer, primary peritoneal cancer, solid tumor
Herbert L. Kotz, M.D.
cervical and ovarian cancer
leukemia and lymphoma
colorectal cancer, head/neck cancer, hematologic/blood cancer, lymphoma, sarcoma, solid tumor
|Jung-Min Lee, M.D.||ovarian, breast and women's cancers|
|Stan Lipkowitz, M.D., Ph.D.||breast cancer|
prostate cancer, immunotherapy, solid tumors, thyroid cancer
|Phuong Mai, M.D.|
|Shakun Malik, M.D.|
|Gerald Marti, M.D.||chronic lymphocytic leukemia|
|Mary McMaster, M.D.|
|Lori Minasian, M.D.||breast and ovarian cancer|
|Anthony Murgo, M.D.|
|Yang-Min (Max) Ning, M.D.||prostate cancer|
|Bernard Parker, M.D.||breast and ovarian cancer|
|Howard Parnes, M.D.||prostate cancer, bladder cancer|
hematologic/blood cancer, graft-versus-host disease
|Richard Piekarz, M.D.||solid tumors, sarcomas|
|Sheila Prindiville, M.D.||breast and ovarian cancer|
|Arun Rajan, M.D.||lung, thymic and aerodigestive cancer|
|Mark Rochewski, M.D.||multiple myeloma, lymphoma|
|Gisele Sarosy, M.D.|
|Mark Schiffman, M.D.|
kidney cancer, Birt-Hogg-Dube syndrome, leiomyomatosis, von Hippel-Lindau syndrome
|Eva Szabo, M.D.||lung and upper aerodigestive cancers|
|Naoko Takebe, M.D.|
|Marc Theoret, M.D.||prostate cancer|
|Giovanna Tosato, M.D.||vascular biology, tumor angiogenesis, KSHV- and EBV-associated malignancies|
|Margaret Tucker, M.D.||familial cancers, etiology of melanoma, etiology of multiple primary cancers|
leukemia, lymphoma, melanoma, kidney cancer
|Jeffrey White, M.D.||adult T-cell leukemia/lymphoma (ATL), cutaneous T-cell lymphoma (CTCL)|
leukemia and lymphoma
human papillomavirus (HPV), hepatitis C, HIV, therapeutic vaccines
|John J. Wright, M.D.||prostate cancer, nanotechnology-based cancer therapeutics|
|Farah Zia, M.D.|
hematology/oncology, breast cancer, early drug development
|Jo Anne Zujewski, M.D.||breast cancer therapeutics|